Cathy was diagnosed with myelofibrosis after a routine blood test.
After her husband retired, she encouraged him to do a blood test with her to get a sense of their health status. Her husband’s blood test results turned out normal while hers showed a low platelet count and teardrop-shaped red blood cells.
She later got a spleen ultrasound, which revealed a slightly enlarged spleen measuring 18 cm, and a bone marrow biopsy, which revealed myelofibrosis. Her myelofibrosis story includes a stem cell transplant, clinical trials, support groups and GvHD.
In addition to Cathy’s narrative, The Patient Story offers a diverse collection of MPN stories. These empowering stories provide real-life experiences, valuable insights, and perspectives on symptoms, diagnosis, and treatment options for cancer.
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This interview has been edited for clarity and length. This is not medical advice. Please consult with your healthcare provider for treatment decisions.
I’m originally from Shanghai. I came to the US in 1990.
I’m married with a 19-year-old son and a 32-year-old stepdaughter who lives in L.A.
I worked in insurance for the last 19 years. My husband and I both did, but he did for a lot longer. I was a product manager and worked on the IT side, making sure my business had a robust and stable system.
I like running outdoors, working out, and doing weights.
Pre-diagnosis
Routine blood work
When my husband retired, I said, “Let’s go get a blood test,” because he doesn’t like to know about those numbers. His numbers came back all normal and mine was pretty much all normal except for the platelet, which was in the 50s. I didn’t understand it. I know it’s lower than the lower limit.
A couple of days later, my primary care physician from Kaiser emailed me, and said, “I want you to get a spleen ultrasound.” I wondered why. I did the ultrasound and my spleen is 18 cm. Then that’s when I started Googling and found out that 18 cm is 50% larger than normal so there must be something wrong with me.
I Googled it right away. What’s the purpose of a spleen ultrasound and what happens if it’s a certain size? I have a general idea why before I went in and when I got the result, it just confirmed my suspicion that it had something to do with a blood disorder.
A couple of days more, my doctor said, “You have teardrop-shaped red blood cells. Now you need to get a bone marrow biopsy.” I searched YouTube videos and saw the big needle with the big handle drilling in. I asked, “Do I really need to? Is there another option?” She said, “That’s pretty much the gold standard.” I thought, Gold standard for what?
I was nervous about the procedure itself, but it wasn’t as bad as I thought. They did have a little trouble getting the marrow out. Later, I found out it was actually a symptom because I’m kind of dry.
Reaction to the diagnostic procedures
I was more anxious. I’m the type of person who likes to know all the details and I felt I wasn’t fed every detail. From the teardrop-shaped red blood cell, it pointed me in the same direction as well. I’m already thinking, I think this is it, but I’m not sure, and going back and forth.
How detailed did you get in your research?
I saw primary myelofibrosis as one potential diagnosis.
I don’t feel like I have cancer. Other than I have quite a bit of a strange bruising on me, which is normal because I’m clumsy, but those are in areas that I don’t usually hit myself with like the back of my finger. I still feel normal.
On one hand, I thought, Maybe the numbers are pointing in that direction. On the other hand, I just don’t want to believe it. Maybe something is wrong.
Around late April, I had quite an episode of vertigo. Never happened to me before. I was driving home, probably 10 minutes away, when all of a sudden, I felt blood traveling from my left ear to the brain. It’s almost like I can hear it. Then my vision became blurry. I thought it was my contact lenses getting dry. I kept driving for a couple of blocks and the road just started moving. That’s when I got a little scared. I parked on the side of the street and closed my eyes. I couldn’t open my eyes. I called my husband to pick me up.
We went to the ER. They couldn’t find anything. I had a similar mini-episode about ten times for the next 2 or 3 weeks. I know when it’s coming. The noise comes then my head spins. If I keep my eyes closed, I’m okay.
I later read that it may have something to do with low platelets but I don’t hear too many people mention that. I didn’t make the connection until later on.
I still run and do weights. I work during Eastern time so I get off at 4 o’clock. I get really sleepy. I don’t usually take a nap in the afternoon but I would nap on the sofa for an hour and have him wake me up so I don’t sleep through dinner. That’s in the last month or two before the diagnosis.
Being referred to a hematologist
I’m the type of person who worries about a lot of small symptoms. I would email my PCP and she’d tell me if this is something to worry about. But this time, she acted very fast.
Three days after my blood test, I went back and looked at the history of our emails. She asked me to do the spleen ultrasound. A couple of days later, teardrop-shaped red blood cells. She said, “Read this.” In the paragraph, it already said leaning towards MPN and I had no idea what MPN is. Probably a week later, she said, “I’m going to refer you to a hematologist,” and that’s when my hematologist and I spoke.
Appointment with the hematologist
I think he had a hunch. My husband was listening to the call as well and I can tell he was trying to be very conservative. He asked me about my symptoms and was trying to gather information. Then he asked, “Do you have a sibling?” That was another clue for me.
He said, “I need a few blood tests.” He didn’t get into detail. I asked, “Am I going to get a chromosome test?” He said yes because I knew about the list of tests. I just want to make sure I’m getting all my tests.
As soon as I met my doctor, he said, “Don’t Google.” I’ll say the opposite but with a grain of salt. How would I be able to ask these questions or know what’s missing without knowing what tests pertain to my disease?
You have to look at reputable sites. I go to the Mayo Clinic and the American Society of Hematology. They’re hard to understand. I look at the article as if they’re a foreign language. I Google pretty much every word to understand.
I figured that it’s a disease so someone has to be able to understand it, no matter how complex. It boils down to something is wrong with you that’s causing this and therefore there’s a potential solution.
Diagnosis
How long did it take to get a diagnosis?
A month. It was the longest month.
During work, I was busy enough not to think about it. As soon as work was over, my brain just gravitated toward, “Could it be cancer? Could it be something else? What’s going to happen?”
I already looked at the life expectancy, but I didn’t know how severe I was. I did not find out about the scoring system so I don’t know how to score myself yet. What’s going to happen?
My son is 19. If I’m not here, financially, he will be okay. I planned this from when he was very young. But what if he needs guidance?
Who’s going to take care of my husband when he needs medical care? He is a lot older than me. How about my parents?
Eventually, my brain got really tired. I didn’t want to think about it anymore.
Getting the official diagnosis
I got the call with the diagnosis on July 8th. I remember when we had the call. Now, I think back, Would it have been better if it was in person or through video?
As soon as we got on the call, I could sense he was nervous. He wanted to be careful how to break the news to me. I told him, “I’ve already done my homework. I think this is what I have and if that’s the case, go ahead and tell me. I’d much rather know about it and get it over with.”
He explained the scoring system, my platelets, spleen size, and fibrosis. Mutation-wise, I’m okay.
As soon as we got off the call, I was Googling MIPSS70. Now it makes sense, but at the time, I didn’t know about it. All I know is I’m probably MF-3 but what that meant, I didn’t know.
Joining online support groups
Some are much more experienced. Some can read my bone marrow biopsy and tell me whether it’s good or bad because I couldn’t understand some of the terminologies. But a lot of time, people give you support and prayers, which are helpful, too.
Myelofibrosis scoring system
There are actually multiple scoring systems. China uses a slightly different scoring system, which doesn’t take into consideration the genetic mutation and the karyotype, which puts a person that potentially could be high risk into medium risk, which changes their life expectancy.
That was actually something I was trying to show in my Chinese group. The website is in English. People can actually click on the link and translate to Chinese. However, the algorithm stops working; it won’t do the addition for them, but it’s good to spread the word out there. You also want to look at those two as well, not just your blood count or your fibrosis level.
JAK2 gene mutation
JAK2 was my mutation type. I don’t remember what that karyotype was, but it was a deletion of one of the 44. That’s the part I don’t remember. But in the scoring system, it will tell you these are the bad ones so I fit into one of those.
Reaction to the cancer diagnosis
I wasn’t sure where the scoring system came from. I thought I’d done enough research. At first, I didn’t believe in the scoring system. But then when I started looking into it, I realized that they make sense and they’re very objective.
It’s either you have a platelet below a certain number or not, or you have this mutation or not. It’s black and white. Before this, I couldn’t tell. It’s just that I wasn’t prepared to have this number thrown at me.
Talking about the diagnosis with the family
My husband doesn’t talk much when it comes to this. I know he was trying to be very calm for me. He always said, “Maybe there’s a treatment out there. Maybe there’s hope.” He’s always trying to give me hope.
He likes to keep things to himself and I like to get it out of him so that’s our conversation. I would talk about my concerns and he doesn’t talk much.
My son, being a teenager, doesn’t talk much, but he occasionally would ask me or even his stepsister, “What’s going to happen to my mom? How many years can she live? Is it going to get bad very soon?”
You can tell he’s thinking about it, but he’s probably not comfortable coming up to me to talk. I wanted him to know right away because I think he’s old enough. I don’t want to hide this away from him.
Feeling healthy but knowing something was wrong
It was very hard. I was hoping to get a second opinion outside of Kaiser, but because it’s HMO, it’s different.
I was able to get a second opinion from a different hematologist within the Kaiser network. He took his time, looked through my numbers, and concurred with the diagnosis. I also reached out to four hospitals outside of Kaiser, some major cancer centers.
In our Facebook group, we always talked about MPN specialists. In my experience, it’s actually not that easy to get ahold of one.
My Kaiser hematologist isn’t an MPN specialist but his diagnosis was accurate. He said, “We handle one myelofibrosis patient each month,” but I didn’t know how to interpret that. Looking back, it’s probably a pretty significant number because Stanford handles about 25 myelofibrosis transplants a year.
Out of the four major hospitals, one of them got back to me. I can talk to a hematologist but not an MPN specialist and it will total about $5,000 out of pocket.
Understanding fibrosis
When your level is high, even after you’ve gone through a transplant, the fibrosis level is not going to decrease right away. It might take months before your fibrosis levels go down, which means your blood count may not go up as fast as other diseases so just be prepared.
One thing that I hear most commonly is, “Is there a drug out there that could shrink my spleen without lowering my blood count too much?” A lot of the time, by the time your spleen is enlarged, your blood count is already borderline. You’re getting close to being transfusion-dependent.
They’re all related to each other. When your fibrosis is high, your bone marrow is not making as much blood, therefore, your spleen is working extra hard to make up for it.
Some patients have spleens that are much bigger than mine and they couldn’t find a solution to reduce it. I feel bad for those patients because their spleen needs to be shrunken enough to get a transplant. I’m lucky enough that mine was just slightly enlarged. Some are 24 cm. Mine was 18.
He said that I might want to consider a transplant in the near future. I said, “Oh, that’s why you asked me about siblings last time.” He said, “You may not need it right away, but it’s better to consider when you’re still relatively healthy.”
He spoke very slowly just to make sure I could digest everything. I think he said I had at least 1 to 2 years. Maybe it’s five years because I didn’t feel that bad.
I didn’t digest it at that moment. I’m kind of a dinosaur when it comes to this kind of thing, but eventually, it kicks in.
I’m not too worried about dying myself, for some reason. I’m just worried about leaving people behind. What can I do now for these people? I don’t know why. It just never dawned on me. It’s not scary for me to die, but it’s scary to leave people behind.
Stem cell transplant
I pushed for the transplant. When I went in for my first appointment with the Stanford transplant doctor, after the discussion, I said, “I’m ready. Just give me a date.”
When I got the date, October 1st, I did a lot of preparation beforehand and even imagined what my room would look like.
I love the nurses there. I had about at least 15. I met 15 nurses because you have a morning shift and a night shift. I learned so much from just talking to the nurses. Why do we do this? Why is this way? Why does the chemo nurse have to wear another gown and there’s always another nurse there just to make sure I’m getting the right chemo?
They kind of became my family during those 23 days because my husband doesn’t talk much. He would spend a whole day sitting in the room with me, keeping me company, but we didn’t talk much. But when the nurse comes in, we talk, even in the middle of the night. I thought I would not have much sleep. I wake up every few hours, but I just went right back to sleep.
I started fludarabine on October 1st. I had four days of fludarabine followed by melphalan; that’s the day we need to chew ice. Forty-five minutes before, I was getting cups of ice chips from the nurse, chewing non-stop all the way through then 30 minutes after. No mouth sores at all throughout the process.
On day zero, I started to feel tired. On days one and two, barely ate, but I had fruits. The next few days, my husband pushed me to get up, walk around, and take a shower. We were supposed to walk around half an hour a day. There’s this pink mask that we wear when we go to the hallway, which makes it harder to breathe. But if I exercise in the room, then I don’t need to wear it.
I danced with my IV pole. I like dancing, but he is not into dancing so I danced with my IV pole because then I can grab the pole with me wherever I want to go. It was really fun because we played music. That was one of my exercises. I even brought in my 8-lb weight at some point so I did some weights, too, because I know I’m going to lose muscle.
Side effects of stem cell transplant
Overall, the experience was a lot smoother than I imagined.
I thought I would have a reaction to the chemo right away, but it wasn’t until 5 or 10 days after that my body started to feel them.
One way to describe the initial feeling of chemo was kind of like towards the end of pregnancy when your body is getting heavier and heavier. It’s harder to do basic stuff. Going to the bathroom takes a lot of energy. Taking a shower requires two people.
Then comes the nausea and diarrhea, but they can be controlled. What I found out later was Zofran (ondansetron) works for me. It doesn’t work for all patients. One of the nurses said you can have Zofran every eight hours so you never feel the nausea, you are ahead of the game. As soon as that happened, I was able to eat a lot more than before.
Possibility of graft-versus-host disease (GvHD)
I think age makes a difference. My brother being a full match made a total difference. Once I got my diagnosis, the first thing I did was call him. He was in China. I said, “I need you to go get an HLA typing done. I don’t want to wait for the doctor to send you the kit,” so he did and the results came back very fast.
Looking at the typing, it’s so easy to understand. They look like 12 sets of timestamps. You compare one against the other. If you match, you match. I didn’t think he would be a match because we have different blood types. He was 12 out of 12. Full match.
After that, he said, “Let me know when you can get your transplant scheduled. I need to buy the airplane ticket.” At the time, China was still on lockdown due to COVID. He lives in Shenzhen across from Hong Kong; that city was locked down so we were worried he could not leave. Fortunately, right before his departure, it opened up.
It was better than winning the lottery.You can’t buy a perfect donor match, especially one who’s related. It would be nice to win the lottery, too. But when you’re in that situation, this is definitely better because the lottery can’t save a life.
For the first three months, I was supposed to be hiding away upstairs. I didn’t completely follow that because I felt okay. I was weak. When I went upstairs, I felt my legs were heavy. I thought maybe I could use going up and down the stairs as an exercise to gain strength.
I was hungry all the time so at midnight, I would be eating mini saltine crackers. My husband said I sounded like a little mouse in the bedroom. At four o’clock, I’ll be hungry and crave traditional Chinese food. I would be cooking rice soup and steamed pao at 4 o’clock. He doesn’t know how to cook those.
For four or five meals a day for about a month or two, I ate whatever I wanted to eat and did not gain weight. That was probably the only time in my adult life I would not gain weight eating like that.
For the first three months, I went back to Stanford ITA once every three days to check my blood. I needed a transfusion a couple of times. After that, I was fine and they just checked if I needed any adjustment on my medication.
They want to make sure because, during those first three months, you could have acute GvHD. Your blood count can fluctuate quite a bit.
Advances in research & treatment options
I actually met a lot of patients who aren’t the best candidates for a transplant. There are definitely medications to bring up their platelet or their red blood cell count. I actually talked to a lot of families of patients like that.
There’s not a whole lot that can be done. A lot of times, the patients themselves are not optimistic about what could happen to them. It’s really hard for their families to get them to a different state unless the patients are ready to deal with it.
Follow-up protocol
Istill go in for follow-ups once a month. Probably at least for another year.
Community support
Importance of finding support in a community
I met a 71-year-old patient. She arrived at Stanford two weeks after me and had some complications. She ended up having to go back there and was actually hospitalized. One day, I met her in ITA and it’s like a reunion. It felt good to see someone who’s been through it with you.
That’s the reason why I started to blog online with hashtags specific to myelofibrosis. A lot of patients chime in. They know what I went through.
Sometimes they ask for my opinion or my suggestions. I didn’t post on a site where all my friends were because I didn’t want them to feel uncomfortable not knowing how to talk to me about this. I know some did get uncomfortable when they eventually found out. I don’t want to impose on them by doing that.
Supporting patients with different backgrounds
China has a very different dynamic. Patient-doctor relationships are a little different. A lot of times, patients are afraid to ask the doctor questions so they actually help each other.
The patient actually knows more about the disease than a lot of the patients here. Give them a bone marrow biopsy report and one person can look at the different numbers and decipher it for you.
The treatment options are slightly different. The philosophies are different there, too. China seems to believe in MAC approach versus the reduced intensity (RIC) approach. They want to clear all your marrows, make sure there are no bad cells in there, and then bring in the stem cell. But here, studies show that the reduced intensity produced just as good as a result.
As a result, when patients go through MAC, there is a lot of damage to their organs. They are in the hospital for two weeks of chemo and they have much stricter diet restrictions. I felt that slowed down the recovery.
There was actually one patient I met who was going through a myelofibrosis stem cell transplant. It’s very hard to find a full match for Asians. He couldn’t find any good match. I got lucky because my brother is a full match.
His two sisters flew in from China; they’re not good candidates. I think he ended up using his daughter, which is very rare. It’s very rare to use kids but the daughter pulled through. He’s going through a haploidentical transplant, which is a little harder. I think it’s a lot harder than mine because there’s more chemo involved to reduce GvHD. He had days of fever.
I message him sometimes. I message other patients as well or they message me. There are a couple that are about to go into transplant at Stanford. I want to share my experience and I want them to benefit from mine as much as they can.
Words of advice
Knowledge is power. There’s a lot of information that’s not valid. You just have to figure out which one pertains to you. Use information from Facebook groups because some of these patients did go through the transplant so they can validate one way or the other.
A lot of times they say you don’t need to know about it and just let the doctor take care of you, but you also need to be able to make calls. Is this treatment for me?
For example, when I was given the initial chemo protocol, it was part of a clinical trial with T-cell depletion, which doesn’t make sense for a full donor match so I questioned that and then it was changed to a different protocol. I’m not saying not to trust the doctors, but I think there’s a reason they put you in one versus the other. Do your homework. Make your own call.
Clyde’s Caregiver Story
What do you remember from the first time you saw Cathy?
We were both employed by the same company. I sized her up as being a very quiet, introverted, typical Asian lady, which she was. It turned out that once you get to know her, she’s a very assertive, intelligent person with a mind of her own. We started dating about two years after she started working there. It was only then that I found out she was very assertive.
What do you like doing together?
There’s a 17-year age difference between us. There’s a cultural difference so there’s not a whole lot that we have together in common.
She likes to dance; I like to sit by the wayside. She likes to eat Chinese food; I like to eat Mexican food. Our life has been a series of compromises more than anything else.
We like to do a little bit of cooking together. We like to help out with non-profit organizations. We went to Mexico once to build a house with our school church group, but age prohibited me from doing it again. Cathy and her son Tom made a second trip down there in 2022 to help build a house.
It’s a very satisfying experience and I would encourage other people to go and do it. It allows them to see how people who are not so fortunate live. They don’t have running water, they don’t have electricity, they don’t have flush toilets, or anything else.
Reaction to Cathy’s blood test results
My personality is not to be the knee-jerk, oh-my-gosh type of reaction. We’ve got something here that’s not very normal. Let’s just see where this goes first. I had a little bit of time to process.
For the next day or two, I was thinking, It could be absolutely nothing or it could be something a little bit more serious. As time rolled on and we did more and more tests, it became very obvious that this wasn’t something that was just going to be simple. It was something that was serious.
Later on, I realized that it was something that was extremely life-threatening. It hit me a little bit slower than it hit Cathy.
I think Cathy is the type of person to look at it and say, “Okay, I know exactly where this is going.” Maybe she’s a little bit too over the top; that’s her personality and for this situation, it worked out best for her.
Understanding myelofibrosis
Cathy is the type of person to do a lot of the research. As she was doing the research, she would tell me what was going on and then I would slowly process it, but there really wasn’t anything else that I could do.
I’d talk to her every once in a while, figure out where she’s going with this, learning what she learned because she’s a very good learner at this. I’m a very slow learner so I’d have to ask her question after question after question before it would sink in.
Impact of a life-threatening disease
I couldn’t fathom it. I couldn’t fathom losing my wife. She’s a lot younger than I am and it was just racing through my head that this isn’t fair. I should be the one that should go first. I was just so afraid that I’d lose her. That was the hardest thing for me to process.
Once I got over that, I realized that we weren’t throwing in the towel. Not now, not when we still have options. She’s a strong lady. She was going to deal with it.
Because we’re a team, we’re going to see this thing through together. I’m going to be there every step of the way. I’m going to give her encouragement and we’re going to fight this together.
I had a pretty good feeling that we were going to beat this. I say we because it is her and me. As a caregiver, maybe you’re not doing the actual fighting, but you’re doing all of the support.
Caregiver’s role in the fight against cancer
If any of you have ever been in the military, you’ll know that there are actual people fighting, but 90% of the forces are in a support position. Without those people, the soldier isn’t able to fight.
As the patient, Cathy’s responsibility is to get better. As the caregiver, your responsibility is to do everything else. She doesn’t have to do anything else except get better.
We’re responsible for making her feel comfortable, whether that’s rearranging her bedding, helping her with the clothes, or getting her food. Everything else is up to me, the caregiver.
What was the biggest surprise, lesson, or difficulty for you?
The hardest thing for me was to show strength. I knew I had the strength, but emotions got in the way. The emotions and the fear of losing somebody that you actually love.
I needed to be strong. I needed not to show my worry and my concerns that things might not go the way that they need to go.
I wanted to show her a positive attitude. I wanted to put on a good face for her and show her that I’m positive that things are going to get better and that eventually, we’ll get back to as near normal a life as we possibly can.
Not taking care of one’s self
I felt that I wasn’t taking care of myself. You hear this all the time. You have to take care of yourself before you can take care of somebody else and I forgot that. I was running around to the point where I was running myself ragged.
Cathy saw that and she said, “You need to stop coming in so early in the morning. Instead of coming in at 8:00 or 8:30, come in around 10:00 or 11:00, and give yourself a little bit of time.” I did and it got better.
You do have to take care of yourself. If the caregiver falls ill, then the caregiver can’t take care of the patient.
Early signs of caregiver burnout
Everybody’s different. Maybe you start to get body aches or something like that or maybe you’re not thinking straight.
For me, it was getting very irritable. I was getting tired and getting body aches.
Look out for triggers. Are there changes in their personality? Are they doing things differently? Are they forgetting things? Are they getting clumsy? Anything like that is probably a sign that you’re getting stressed out.
Differing perspectives between patient & caregiver
Cathy is a very intelligent person and she studied this quite extensively. She’s the patient. She has an idea of what she wants. Because it is her health, I usually will defer to what she has to say.
If I had a very strong opinion in the opposite way is the only time that I would speak up. But at the same time, I’d say what I had to say and then it would be her final decision.
Advantages of patient education
They say knowledge is power. But at the same time, you have to be careful when you start looking up information on the Internet. Take it maybe with a grain of salt.
If this website pops up and says you should be doing X, Y, and Z, you have to look at the website and ask yourself, “Is this a reputable website?” If you go to another website like Stanford Hospital or the Mayo Clinic, you put a little bit more weight into what they say.
I also think that once you get on these Facebook groups, they’re good and they’re bad. They’re good because they give you support. They tell you what other people have gone through.
At the same time, you have to realize that the majority of these people are on there because they have a special issue with something. When they say, “Oh my god, this is hurting me, this is doing this to me. I had this as a symptom,” maybe the majority of people don’t have those symptoms. Be aware that they can happen to you, but take it with a grain of salt.
Words of advice
Brightening up the room really helps the patient. We had these static window stickers of plants, leaves, and all these little things that would brighten up the room. When we got to the hospital room, someone had painted a little rainbow and sunshine on the windows and the stickers were still there. We just went there recently and they’re all still up there so other patients have been using them and it’s brightening up the room. It lifts your spirit more than anything else.
Bring a rolling suitcase; that’s more for the caregiver. because the caregiver is going to be taking things back and forth with them all the time. They’re going to be bringing in clean clothes, taking out dirty clothes, and maybe bringing in a little bit of food or other supplies that they maybe had forgotten the first time around. It’s just a lot easier bringing in a rolling suitcase.
In a hospital, you don’t need to worry about medication. The nurses are constantly monitoring that. But when you get home, again, it’s the caregiver’s responsibility to do virtually everything, and that includes monitoring the medicine.
A patient frequently has brain fog. They don’t know what they did. They don’t know what they should be doing. It’s up to the caregiver.
Get a two-week supply of pill bottles, each marked from Sunday all the way through Saturday. Load them all up with the morning, afternoon, and evening pills. Put them all in separate containers. That way, it’s one less thing for the caregiver to worry about.
By the time you go through the first week, you still have a one-week supply and you should be refilling the empty containers. If you can’t refill the empty containers, you know immediately that you need to order medication. Ordering medication immediately rather than waiting until they run helped us quite a bit.
Importance of diverse community support
It’s important both to Cathy and to the other patients. To Cathy, I think it’s a mental stimulation for her. Work is one thing, but work is just work.
Helping other people is something that I think she really feels gratified in doing. At the same time, with these other people, frequently, they’re at a loss. They don’t know who to turn to. A lot of these people are overseas in China and sometimes they don’t get as good information as we do over here in the United States.
What to Know about Common Misconceptions, Emerging Trials, and Biomarkers
Edited by: Katrina Villareal
What is it really like to find and participate in clinical trials as a colorectal cancer patient? Learn how to navigate the complex world of clinical trials from patients and experts who have been there.
Hear from Dr. Andrea Cercek of Memorial Sloan Kettering, David Fenstermacher, Senior Director of Research and Medical Affairs at the Colorectal Cancer Alliance, and patient advocates Allison Rosen, Julie Clauer, and Kelly Spill as they share the barriers, risks, and results from real-life clinical trial experiences.
*Watch the full video below!
This interview has been edited for clarity and length. This is not medical advice. Please consult with your healthcare provider for treatment decisions.
Allison Rosen: We’re going to talk about the latest in colorectal cancer trials, what to know, and what to ask. We’re going to hear from some experts in the field and some amazing patient advocates who have participated in clinical trials.
This discussion is hosted by The Patient Story, a platform where you can find hope, guidance, and supportive communities, explore insights on navigating life after a cancer diagnosis, discover promising treatments, and connect with people who truly understand what you’re experiencing.
I’m very, very excited to introduce the partners that help make this happen: Colorectal Cancer Alliance, Colon Cancer Coalition, and COLONTOWN. I’ve had the opportunity to volunteer with each and every one of them in one shape or form.
The mission of Colorectal Cancer Alliance is to empower a nation of allies and to provide support for patients and families, caregivers, and survivors. Their goal is to raise awareness of preventative measures and inspire efforts to fund critical research.
The mission of the Colon Cancer Coalition is to improve health outcomes by reducing barriers to complete colorectal cancer screening and educating the public to advocate for their own health through tailored, local, and grassroots solutions.
COLONTOWN is an online community of more than 120 private Facebook groups that provide education and peer support for colorectal cancer patient survivors and care partners on every aspect of living with colorectal cancer and beyond, including clinical trial understanding and experiences.
COLONTOWN has an amazing university that’s a public education resource with content created by patients and caregivers for patients and caregivers. It’s a library of original content where experts talk about various topics, including clinical trials.
The purpose of this conversation is to have a conversation. We want to humanize colorectal cancer clinical trials by hearing from people who have been through them and are now dedicated to advocating for others. We’re also going to answer questions and break myths that surround clinical trials in general but especially colorectal cancer clinical trials.
Allison Rosen
Allison: I’m a stage 2 colorectal cancer survivor and a very, very passionate advocate. I’ve worked in the field of oncology for about 17 years and have been an advocate ever since I was in remission.
David Fenstermacher
David Fenstermacher: I’m the senior director of research and medical affairs with the Colorectal Cancer Alliance. My role is to oversee our grant portfolio. I also work with the community to develop some patient-focused projects around biomarkers and equity. The other thing in my purview is a national survey, which we will be releasing later in 2023, for colorectal cancer patients and caregivers.
Dr. Andrea Cercek
Dr. Andrea Cercek: I’m a medical oncologist at Memorial Sloan Kettering Cancer Center. I’m very passionate about clinical trials and the hope for the future that they give us.
Kelly Spill
Kelly Spill: I was diagnosed with stage 3 colorectal cancer at age 28. At that time, I had an ulcerated 5 cm tumor and I received nine infusions of immunotherapy from March to August 2020. I was declared in remission that same August.
Julie Clauer
Julie Clauer: I’m a stage 4 colorectal cancer patient. I was diagnosed in March 2018 with liver mets and since then, I’ve had cancer jump throughout pretty much everywhere — lung, liver, lymph nodes, bone.
I’ve had an active disease since then and started participating in a clinical trial in July 2020. I was on the trial for two and a half years and it completely changed the trajectory of my disease. I might actually have my first scans with no evidence of disease. We’re hoping that after all these years, I’m finally NED.
All the drugs that we have now were at one point in a clinical trial and had to be tested on people in order to get approval.
Dr. Andrea Cercek
Misconceptions about clinical trials
Allison: I’m very, very excited because this is such an important conversation. People have all sorts of knowledge about clinical trials when they go in. When we talk about clinical trials, there are a lot of misconceptions.
Dr. Cercek, what are some misconceptions you’ve heard? When people reach out to me, one that I’ve heard is they think clinical trials are a last resort, only for the very sickest patients. But they aren’t, are they?
Dr. Cercek: No, definitely not. Clinical trials come in all shapes and sizes and in all stages so that’s a really, really important point. People often feel it’s a last resort. “I’m a guinea pig. Why should I do this? Will this matter at this point?”
Trials happen in early-stage disease. They can be as a first-line treatment, when someone is first diagnosed, and also with advanced disease, where the goal of the trial is how to improve the standard treatment either by adding new medication or changing medications completely based on data from studies in advanced disease.
It’s important to run these trials in all stages to find out which drugs work and at what stage they work best. That’s a really, really important point that we need to instill. It’s critical for people to consider clinical trials because that’s really what helps move science forward. That’s really what makes advances.
All the drugs that we have now were at one point in a clinical trial and had to be tested on people in order to get approval. We have super strict approvals for all drugs but especially for oncology drugs. We get approval through clinical trials.
Patients are afraid they’re going to be given a placebo. In oncology, that’s not true.
David Fenstermacher
Allison: That last point is one of the things that most people have no idea about. David, you speak to patients all the time. What are some of the questions and misconceptions about clinical trials that you hear?
David: The one I hear the most is patients are afraid they’re going to be given a placebo. In oncology, that’s not true. The care that you would get would be the standard of care for your disease at your particular stage and biomarker. That’s really important to understand.
There are all sorts of concerns that patients have. How do I find a trial? Is there a trial near me? What are the costs of a trial and who pays for the different aspects of that? If you participate, how is that going to alter the schedule of when you have to go to the doctor or the site where the clinical trial is being conducted? Do you need more tests? Do you need more imaging?
A lot of people don’t truly understand that when you go in, it’s very regimented. You will learn exactly what’s going to happen. You can ask questions of your doctor so that you can understand exactly what you’re getting yourself into by possibly participating in a trial.
Everything is your decision. You can withdraw from a clinical trial at any time. This is really about you and the care that you can get through these new therapies or new combinations that are being developed.
Allison: This is great information. If someone is in North Carolina, they want to go to Houston or New York for a trial. They want to go to the trial that’s best for them. They want to know about cost, lodging, parenting — all the different things.
I said, ‘You know what? I have nothing to lose. The side effects sound a lot less worse than chemo, radiation, and surgery. Let’s give it a shot.’
Kelly Spill
Kelly’s clinical trial story
Allison: Kelly, we want to hear about your clinical trial experience. What were your first treatment options and how did you end up hearing about clinical trials? Did your doctor bring this up or did you?
Kelly: My original treatment option was chemo, radiation, and surgery, which would have led me to have a colostomy bag for the rest of my life and likely not be able to carry another baby again.
I didn’t know anything about clinical trials. I’m actually still learning to this day and it’s very exciting to learn now that I’m in remission. I’m in a big learning era right now.
At the time, I was going to Memorial Sloan Kettering. If I didn’t go there, I would have never known about this clinical trial.
I was planning on checking out a couple of cancer centers at the time and once I hit Sloan, I felt like I was at home. I felt like a family member there. I felt very comfortable. They were very informative with everything that was going on and that I needed to know.
Thankfully, I had a research nurse come into my room right before we were about to set an appointment to start chemo. She said, “We may have another option for you. Would you like to hear it?” I said, “Of course, I want to hear another option.”
I had this other option specifically because my tumor type was an MMRD, mismatch repair-deficient, so it allowed me to be on this clinical trial.
I had my mom with me and I don’t know what I would do without her. It was really nice having someone with me who was able to understand what I was being told. Being so sick, not feeling well, and being young, I had no idea what a lot of these big words meant and what was going on at the time.
After I heard the pros and cons of the side effects, I looked at my mom and asked, “Should we do this?” She asked, “How do you feel about it?” I weighed out my options.
I said, “You know what? I have nothing to lose. The side effects sound a lot less worse than chemo, radiation, and surgery. Let’s give it a shot.” If that didn’t work for me, then I would have gone back and did the chemo, chemo-radiation, and surgery. I still had a backup.
Allison: Having a caregiver there is so important, someone you trust and know. We’re in a nervous state and going through something very, very traumatic so having someone else there to help guide you is so important. Thank you so much for sharing and letting everyone know how you came to that decision because it’s personal and hearing different viewpoints is so important for others.
Dr. Cercek, you co-led the trial that Kelly was on and know her well. Can you tell us a little bit more about this trial?
Our trial was specifically asking the question, ‘Can it work even better when the tumor is in an early stage when it’s localized before it spreads?’
Dr. Andrea Cercek
Dr. Cercek: This study is still open. It’s for patients with mismatch repair-deficient, a specific subtype in early-stage rectal cancer.
Normally, for early-stage rectal cancer, the treatment is chemotherapy, radiation, and surgery. Because it’s located down in the pelvis at the end of the large intestine, there are a lot of important organs that are affected by our normal treatment, like the ovaries and the uterus.
Although it’s a good treatment and it’s curative, people are left with a lot of toxicity from treatment, including not being able to have babies, bowel/bladder dysfunction, and potentially even a permanent bag in about a third of patients or so because of where the tumors are.
The idea of the study in this subset of patients with mismatch repair-deficient or MSI-high tumors is to try immunotherapy. We knew that in stage 4 disease, immunotherapy works very well for mismatch repair-deficient colon and rectal cancer. This was accomplished in a clinical trial where it was the same idea.
We have a good biomarker. Our trial was specifically asking the question, “Can it work even better when the tumor is in an early stage when it’s localized before it spreads?” The way that we designed it is that because the standard treatment was curative, we didn’t want to compromise the cure.
We gave immunotherapy first, watched the patients really closely, and then if the tumor didn’t completely go away, they would simply jump onto the normal standard of care regimen with chemotherapy, chemo-radiation, and surgery, depending on the clinical situation. Everyone would be offered a complete package.
Thankfully, we saw that immunotherapy alone is able to get rid of these tumors so it’s been really amazing. Now we’re treating all different early-stage cancers, including the esophagus and stomach that have mismatch repair deficiency or MSI High. As I mentioned, the rectal study is still ongoing.
Allison: It’s important to note that that trial was for that specific gene. It’s important to know your genes and your mutation so that you can figure out what trial works best for you. Talk to your doctor about that so you have that knowledge. I’ve heard about your research and now meeting Kelly and seeing the human side of it is just amazing. Thank you so much for doing what you’re doing and continue doing what you’re doing.
I’d had over 60 chemo treatments, five open surgeries, and multiple radiation therapies. My body was worn out.
Julie Clauer
Julie’s clinical trial story
Allison: Julie, we want to hear about your clinical trial experience. How did you learn about it and how much did you know before?
Julie: In my first appointment before I started treatment, I was told about a clinical trial. I thought that was just what happens is that you get offered clinical trials. I found out later that that’s not actually how it works.
At that time, I decided not to do the clinical trial. I knew with stage 4 and as extensive as the disease I had, I knew that the first line of treatment wasn’t going to work forever and I knew that this wasn’t going to be a one-shot deal for me.
I’m a planner by nature so I wanted to know what the options were before I needed them. It was actually while I was doing well in treatment that I did the most research on clinical trials. I felt like that was extremely helpful because I wasn’t in a panicked state. You’re looking at it very objectively. You’re not making a major life decision at that point.
Sometimes I’d find out about clinical trials from COLONTOWN. I’d hear about something from another patient or my doctor would tell me about something he was working on that was really exciting.
We dribbled the clinical trial conversation in which I’d bring something to an appointment and say, “I heard about this trial. What do you think?” I’d get his opinion. It was like talking and learning about clinical trials two minutes at a time over a long period of time.
Anytime I got news of progression and needed a treatment change, we’d already have a set of what would be next. I always wanted to have a few options. The clinical trial I ended up going on was always on that shortlist.
When I would come in with the results of a scan that wasn’t good, we’d say, “Given what we see now, are all those still options? How do we want to move forward?” We had already made the decision before I needed the decision.
This trial was one that was on the docket for a long time so I learned a lot about it. I learned how the early results were going. I learned almost too much because, by the time it was time to go on it, the trial was not looking very good in terms of the results. From a research perspective, it didn’t meet its endpoint.
But I had progression and I was really exhausted. I’d had over 60 chemo treatments, five open surgeries, and multiple radiation therapies. My body was worn out.
When it came time to decide what was next, I could have gone on the last line of the standard-of-care chemotherapy, a third-line treatment that was available, or two trials that were in the mix.
This trial was one that was on the docket for a long time so I learned a lot about it.
Julie Clauer
When I went to my doctor, we were talking about the trial that I ended up going on. It was a better option for me than the standard of care at the time. We talked about the fact that it wasn’t going to change the trajectory of my disease. I wasn’t going to be cured, but the short-term stability was pretty good.
He said, “It’s immunotherapy-based. The toxicity is less. It could give you a few months to regroup, have a chemo break, and hopefully get you to stability so we could figure out what’s next.” It was intended to be a bridge.
I said, “Okay, I think I want to do this, but we have that other trial that I’m more interested in so I want to go talk to that primary investigator.” He was at a different institution. I made an appointment with him, talked to him about the trial, was excited about it, but he said, “It’s not open for another six weeks and when I look at your counts, you’re not going to be able to wait that long.”
There’s also an element of having options because you don’t know what will be available and what will work for you. I was looking for a range of stability and I ended up being stable on it for two and a half years.
It opened up a lot. I was able to get surgery. It was much better than stable, in my opinion. In terms of the actual results of the trial, it was stable. It goes to show that you have to know what you’re looking for and have some options.
Those moments are scary. Having somebody else who can help talk you through it is fantastic. Not having all the information thrown at you at a time when you’re really scared and trying to figure out the logistics while you’re in the moment was key for me.
My clinical trial experience was fantastic. It out-delivered. I actually went off the trial to get surgery, which wasn’t an option before. My side effects were significantly better.
I’ve since gone on an off-label treatment because of how I responded to the trial. It became an option because of how I did. It really changed everything about my disease, but it definitely was not the cure.
Allison: It’s always very complicated. It’s never, “I’m going to do this then this and this.” You need to figure out what’s best for you at that time. Sometimes you have to wait or make a difficult choice about where to go next if one trial doesn’t open for a certain amount of time. Someone asked, “What exactly do you mean by stability? No tumor growth?”
Julie: No tumor growth. Technically, on a clinical trial, if you have between 30% shrinkage and 20% growth on certain tumors that they’re measuring, that means stability. Technically, I had stability. However, I had 29.4% shrinkage so I was right on the cusp. I had 11 mets and went down to four. I was nonsurgical and became surgical.
Those outcomes are so important for researchers to look at, to see the whole population and how people do to move medicine forward. But when you’re an individual patient, I think all that matters is what it means to you.
Deciding to join a clinical trial
Allison: You can see between these women that their stories are different. Trials are different and they’re happening all over the US. There’s a process and some people might not know the process. How do I find what’s best for me? What might go through the mind of someone thinking about a trial?
Kelly, what was the process like for you before deciding to join the trial? Did you have any concerns? What were some of the questions that you asked your doctor?
Kelly: To be honest, I was so sick sitting there that I was weighing out the side effects. I was so concerned about not being able to have another baby. I was so concerned about radiation.
I remember sitting and listening to what I would have experienced as side effects. My sex life would have never been the same. It really scared me. Radiation and surgery scared me a lot more than chemo at that time.
After hearing the side effects of this clinical trial, I said, “Why wouldn’t I try this?” I had a backup plan of chemo, radiation, and surgery. My mom and I looked at each other, I said, “Let’s go for it.”
I’m the only one in my family who has been diagnosed with colorectal cancer. No one in my family has cancer so I haven’t been surrounded by it. I didn’t know anything. In our eyes, it was more of a chance of should we do it or not.
Coming out of this, I want to be that advocate because so many young people are being diagnosed with colorectal cancer. If you don’t know anyone who goes through it, you don’t even know that there are any of these options or resources.
Keep an open line of communication at every point. Make sure everyone is informed that these are the possibilities that we might be thinking about at some point.
Dr. Andrea Cercek
Clinical trials for young-onset patients
Allison: We know early-onset colorectal cancer is on the rise. I was an adolescent young adult patient when I was diagnosed. I wouldn’t say epidemic, but there’s something going on. Researchers and oncologists are trying to figure out why.
There are a lot of young onset programs now. How do oncologists proactively counsel their patients about trials given most young-onset patients want to do trials before failing the second line?
Dr. Cercek: Epidemic is actually a really good word. It’s rising worldwide. We don’t know why it’s happening, but there’s a steady rise from year to year.
We opened centers dedicated to young people under the age of 50 with colorectal cancer for two reasons. First is for research to try to get data to figure out why this is happening and second is to help patients navigate this disease from diagnosis through treatment and into survivorship. Each stage is critically important and has different and unique challenges in our young patients, as everyone here is well aware.
We believe that this disease, as best as we can tell, is the same disease once someone has it. There might be slight nuanced differences, but in terms of available treatment, our chemotherapy and our trials are for all ages. There’s nothing specifically for early-onset colorectal cancer because the disease, once it’s present in any stage, behaves very similarly.
Young patients themselves are more motivated, perhaps more eager to participate in trials and think about the next steps. It depends on the person very much, but that’s probably true as a group.
Although someone is motivated, it’s not always easy to offer trials because sometimes the standard of care is better, which is an important thing to acknowledge and think about, too. We have certain good treatments that are already accepted as the standard of care.
There are trials for every stage so it’s an important thing to keep in mind, especially with our young patients. Often, people come armed with information.
It’s incredibly overwhelming how many trials there are out there so that’s a challenge in and of itself. It’s important to educate our patients at any age, but especially our young patients in terms of biomarkers, which are potential targets in the future.
If someone has a specific mutation, I’ll often say, “Listen, you have this. We’re not going to think about this now, but this might be something that we’ll think about a trial in the future,” or, “There’s an exciting new trial with combination immunotherapy. We don’t need it right now, but that’s something that we’ll think about.”
The challenge is always, “I’m ready now, I’m ready now,” and oftentimes, I’ll tell you, “Listen, you’re in a really good spot. You don’t need anything right now because we don’t have anything. The chemo that you’re on is actually working. Once, of course, we see that it’s not working, then we think about what are the next steps.”
The most important thing is to keep an open line of communication at every point. Make sure everyone is informed that these are the possibilities that we might be thinking about at some point.
Allison: Having an open line of communication is huge as well as trust. Patients can do their research but it’s shared decision making. It’s about what’s best at that time. Younger people might be eager, but we have to trust in the process.
You shouldn’t let cost, fear, or other things stop you from investigating and thinking about trials.
Allison Rosen
Costs involved in joining a clinical trial
Allison: Does insurance cover clinical trials? People can’t just pick up and go. There might be a trial down the street, but the best trial might be 500 miles away or a thousand miles away.
David: There are some things covered by the sponsor of the clinical trial. The study drug is covered. Any lab tests related to any endpoints that aren’t the primary endpoint are paid by the sponsor. Any imaging that the sponsor would ask to be done so that they can monitor the response to the therapy on the clinical trial very closely would also be covered by the sponsors.
The doctor’s visits, hospital stays, regular lab tests, and regular imaging that would be part of the standard of care, your insurance would pay for.
Some sponsors do provide support for things such as transport, housing, food, and child care. There are other resources like the Colorectal Cancer Alliance, where we also have resources where we can help patients with some of these financial needs.
We have navigators who can help you through the process of finding a clinical trial and how you can participate in a clinical trial based on some of the challenges that you have.
Allison: You shouldn’t let cost, fear, or other things stop you from investigating and thinking about trials.
I wasn’t giving up on the idea of a cure. I saw it more as a relay race.
Julie Clauer
Switching mindsets
Allison: Everyone wants to be cured and it’s hard not to be stuck on getting to a cure. Julie, how did you change your goal to stability? What was that like?
Julie: It depends a lot on where you are in your care. That changes every point. Overall, the long-term goal and desire is always cure.
When you look at where I was in treatment, I was on the third/fourth-ish line. My body was very depleted. My blood count numbers weren’t doing so well. My platelets were low. I wasn’t necessarily in a healthy spot to do trials.
One of the reasons why I couldn’t wait six weeks wasn’t because the cancer was out of control. It was because I probably wouldn’t be healthy enough to participate in a clinical trial. That’s another reason not to leave them as a last resort.
But at that moment, I wasn’t giving up on the idea of a cure. I saw it more as a relay race. For this segment, what I want is to get to stability, to be able to regroup, let my body recover a little bit so I can figure out the next step.
Any given treatment, if you look at it as a one-time treatment, depending on where you are in your disease, you could be cured with one treatment like Kelly was. Where I was in my disease, I knew it was going to be a series of multiple treatments.
Look at it as segments of the long race. In this segment, what I was looking for was stability so that I could be ready for the next segment. My goal would change then because if I’m stable, then my goal would be something different at the next point.
Allison: Clinical trials are a conversation and decision you make with your care team, your caregiver, and yourself. You have to think about a lot of different things. It’s not an easy decision.
Stay in contact, be honest with what’s going on, and be honest with yourself. If you have a question, ask. Don’t be scared.
Kelly Spill
Importance of advocating for yourself
Allison: Kelly, you mentioned your mom was a part of your decision making and you now strive to be an advocate to help others. What advice do you have for people as far as being their own best advocate when you’re talking to your care team?
Kelly: Before I started the trial, I was taking pictures of what was happening in the bathroom because sometimes it’s hard for me to describe what’s going on. I took pictures and showed my oncologist.
I ended up being very close with my oncologist and my research nurse. They were calling me pretty much all the time. I don’t know if that’s how it is in every hospital, but I was taken care of very closely.
We had a very open communication. I wasn’t holding anything back. I told him how I felt after every treatment, every little detail, not just physically, but mentally and emotionally. All of that plays into it.
As a mom, my mind is elsewhere, too, so I was writing in my Notes on my iPhone how I was feeling at home.
I was constipated a lot and not doing well. After my first or second treatment, I couldn’t stop going to the bathroom. It was a completely opposite problem that I had been having. I quickly called the research nurse and told her what was going on, and she said, “That’s actually great news. Everything is being released.”
Stay in contact, be honest with what’s going on, and be honest with yourself. If you have a question, ask. Don’t be scared. It’s easy to be scared when you’re going through something like that, but putting everything out on the table is important.
Allison: I didn’t think about taking pictures. A journal can help because you go through so many things and can’t remember everything. You only have a certain amount of time with your doctor so coming prepared with a list helps expedite the conversation, help them understand, and not forget something. Be as prepared as possible. Those are great tips.
When people are first diagnosed, it’s really important to know those biomarkers right away.
Dr. Andrea Cercek
Role of biomarkers in clinical trials
Allison: Dr. Cercek, can you talk about the role of biomarkers in clinical trials and how they can guide personalized treatment?
Dr. Cercek: Abiomarker, at this point, is probably one of the most important pieces of information in terms of clinical trials and general treatment of colorectal cancer. It’s definitely important in advanced disease and even in the early stage, as we saw from Kelly’s experience.
A biomarker basically is a change in the tumor, like a mutation, which we have a potential drug for. Important biomarkers in colorectal cancer are things like mismatch repair deficiency or MSI, where we can use immunotherapy or other combinations that are tested in clinical trials.
Important biomarkers include BRAF, V600E in particular, but BRAF is good enough to remember. We’ve made progress with BRAF targeting in clinical trials. There’s an approved therapy, but we’re trying to do better. It’s a really, really important marker in terms of treatment and trials as well.
Another one, which used to be a huge challenge but now we’re making progress, is KRAS or RAS. Typically, when we hear RAS mutated, we think there’s a treatment that we can’t use, like anti-EGFR therapy.
Now, there are RAS inhibitors or RAS-targeted therapies. RAS is the biomarker and then we have a targeted therapy for different types of KRAS. You might have heard KRAS G12C; that’s the new kid on the block. We recently had very good data in terms of responses when treating this biomarker in colon cancer. A newer one is G12D that’s now in clinical trials.
That’s an important thing to think about and to test. Most importantly, it’s something that everyone should have done when they’re diagnosed so that we know what the landscape is and what we can do, not only in terms of clinical trials but also the standard of care.
Another really important one is HER2 or ERBB2. We’ve studied that a lot in breast and stomach cancer, but now we’re seeing combinations work in colon cancer as well. There are ongoing trials looking at it in advanced disease and first-line treatment.
When people are first diagnosed, it’s really important to know those biomarkers right away. We’re also looking at it in early-stage disease and rectal cancer, similar to the study that Kelly was on but with HER2 targeting because it’s a very, very good biomarker for which we have a really good drug.
There are a number of biomarkers that we need to know. We encourage the next-generation sequencing of the tumor to know the mutational landscape. You can do it from the tumor. We could even do it from the blood in what’s called circulating tumor DNA. There are different ways to get it done, but it’s important for everyone to speak with their oncologists and have that done if you don’t already know that information.
Things have changed over the past few years in such an exciting way so you can treat the specific mutation of the tumor of that person. Asking those questions is one of the most important things.
Allison Rosen
Allison: The point that you made right there is so important. Not everyone has the knowledge. Ask about biomarker testing and circulating tumor DNA. Personalized medicine is so important now. Back when I was diagnosed, the standard of care was chemo, radiation, and surgery. A lot of this was on the horizon.
Things have changed over the past few years in such an exciting way so you can treat the specific mutation of the tumor of that person. Asking those questions is one of the most important things. A few years ago, only one mutation had a trial. Now there’s all this research ongoing. There are trials ongoing for a lot of this. Have that conversation with your doctor.
If you don’t know, that’s the first question you can ask your care team. Send them a message or call their team.
If they haven’t done it or they haven’t told you about genetic testing, biomarker testing, or circulating tumor DNA testing, have that conversation. It could change the trajectory of your whole treatment. You can find a trial that would work for you.
Julie: Biomarkers are so critical. As a vanilla girl who’s wild type with no targetable mutations, not having a mutation is valuable information as well. There are a lot of trials that are available to you, too. A lot of people will say, “I don’t have any of those mutations so I’m out of luck.” No, you are not and I’m proof of that right here.
Dr. Cercek: Julie, thank you for that point. There are many studies, many drugs, many trials, and many different ways of targeting colorectal cancer. Biomarker is just one of them.
There are different ways that drugs can enter the cell that have nothing to do with the mutation that are also explored and making progress. That’s really critical to know your mutational landscape because even that wild type makes a big difference in the standard of care, too.
Allison: The amount of information that’s being shared here is so valuable.
There’s a wealth of tools on the Internet that can help you find a trial. It’s more about making sure you’re finding the right trial that is also going to fit your lifestyle.
David Fenstermacher
Finding clinical trials
Allison: How do you get started on a trial and/or find trials and register? How often should a patient be searching?
David: ClinicalTrials.gov was brought up, which is a website run by the government that lists all clinical trials available. That can be a little bit daunting to navigate through. It’s a fairly complicated site, but that’s the place where you’re going to get the most. You have to use different search strategies.
There are tools out there that you can use. You don’t have to necessarily do it yourself. We have a system called BlueHQ. It’s a patient portal that allows you to learn more about your disease, especially if you understand your biomarkers. We have a trial finder embedded called Leal Health. You can put data in there and they will try to match you with a clinical trial.
I’ve worked at several comprehensive cancer centers and they will have on their website what clinical trials are available. There’s a wealth of tools on the Internet that can help you find a trial. It’s more about making sure you’re finding the right trial that is also going to fit your lifestyle.
ClinicalTrials.gov is going to be the most up-to-date because protocols have to be submitted to ClinicalTrials.gov. You don’t have to do it yourself.
We use something called Leal Health that you can get through BlueHQ that allows you to enter data into their platform and they will try to do the trial matching for you. They’ll contact you if you are eligible for a trial, talk to you about where the trial is being conducted, and then see if you’re interested.
There is so much going on right now in colorectal cancer. When you look at new types of drugs that are coming out, antibody-drug conjugates are one. Think about new immunotherapies and how to turn cold tumors, those that do not respond to immunotherapies like PD-1 and PD-L1 therapies, into hot tumors so that they would respond then to those immunotherapeutic drugs.
There are opportunities for things like oncological vaccines. If you’re in stage 2 or 3 and you have a resection done, a company will take your tumor, sequence it, and look for neoantigens. These are caused by mutations in the tumor.
The interesting thing here is is they give you a vaccine against most common neoantigens so that hopefully if you grow new tumor cells after your surgery, your immune system would already be primed to take care of them.
On a daily basis, there could be a new trial. It’s more important to work with your oncologist and let them know you’re interested so they can help with trying to find the right trial for you.
Not all oncologists are comfortable with clinical trials, especially in rural parts of the country. There are resources that you can reach out to online that would allow you to keep track of clinical trials more closely. You can work with other companies that can do clinical trial matching for you. They’re on the web so they’re available.
Work with your oncologist and let them know you’re interested so they can help with trying to find the right trial for you.
David Fenstermacher
Final takeaways
Dr. Cercek: The most important thing is you can be and should be your own advocate. Speak up for yourself. Open that line of communication.
If you’re having trouble, let your oncologist know. If you have questions, it’s okay. Ask questions. It’s not annoying. You’re in this fight together and that’s the most important way to think about this and to approach this disease.
Kelly: Ask questions. Be open. You know your body best and you know yourself best. Coming from someone who’s a big introvert, I always feel like I’m annoying people with all these questions. It’s so important to ask them. Trust your gut.
David: A clinical trial could be out there that is just right for you at your stage in your disease progress. Do not be afraid of the fact that it’s research. These are drugs that have gone through extensive pre-clinical testing. This could be the best standard of care that you could get. Keep an open mind.
Julie: There’s no perfect trial for everybody. They’re all studies. They’re all trying to learn. Know yourself and have those conversations. Understand what you’re willing to do before you need to make the decision. When you get to that point of having to make the decision, you don’t necessarily have enough time to make the best decision you can.
Allison: Thank you so much to these panelists for their time to share and educate. You will most certainly help save lives by being here to share so thank you to each and every one of you.
Ananda was diagnosed with stage 3B rectal cancer at 44.
Ananda knew something wasn’t right when she experienced rectal bleeding after the birth of her son in 2014. Even though her symptoms got worse, doctors dismissed her concerns as hemorrhoids for years.
It wasn’t until 2020, after the birth of another child, that Ananda was finally able to get a colonoscopy which revealed a large rectal tumor. She was then diagnosed with stage 3B rectal cancer at 44.
Knowing that she needed to see a specialist, she made the 10-hour drive to the Mayo Clinic where she subsequently had her surgery.
She shares the importance of being your own advocate and listening to your body. Leaning on her faith helped her through the most traumatic thing she had to go through.
In addition to Ananda’s narrative, The Patient Story offers a diverse collection of colorectal cancer stories. These empowering stories provide real-life experiences, valuable insights, and perspectives on symptoms, diagnosis, and treatment options for cancer.
This interview has been edited for clarity and length. This is not medical advice. Please consult with your healthcare provider for treatment decisions.
Name: Ananda P.
Diagnosis:
Rectal cancer
Staging:
3B
Initial Symptoms:
Intermittent rectal bleeding
Treatment:
Surgery
Chemotherapy: FOLFOX (folinic acid, fluorouracil, and oxaliplatin)
Introduction
I’m originally from Canada. I live in Rapid City, South Dakota. My husband, Joe, and I have eight kids from ages 3 to 20.
My background is in journalism. Writing and reading are my two big hobbies. I love people, ice skating, and a lot of different things.
Pre-diagnosis
Initial symptoms
My son, Reeve, who’s number six, was born in 2014. I had some rectal bleeding after he was born and it was really painful.
I went to the doctor. She did an exam and said, “You have a fissure so increase your fiber and drink lots of water to clear it up,” so I did. Magnesium was amazing; that really helped. It cleared up and went away.
Fast forward two years later, my son Avram was born in August 2016. I had some more bleeding so I started taking more magnesium, fiber, and lots of water, but it wasn’t clearing up.
The bleeding didn’t happen every time. It was just sporadic. I would take two magnesium, which was more than enough but I was still having this blood on and off.
In January 2017, five months after he was born, my uncle was diagnosed with stage 4 colon cancer and he died a month later. I thought, I really need to get on my issue.
I got pregnant again and had a late miscarriage so going to the doctor or having a colonoscopy was on the back burner.
In July 2018, I finally went and said, “My uncle died last year.” My primary care doctor asked, “Are you having any pain in your stomach or any other symptoms?” I said, “No, just blood every once in a while.” She said, “With your history of having all these babies, I’m so sure it’s hemorrhoids. Your risk of getting cancer at this age is so slim.” I was 42 at that point.
She did an exam and said, “Yep, I can feel the hemorrhoids,” so she gave me hemorrhoid medicine. Then she said, “If it doesn’t clear up, come back and see me.” We were in the middle of an interstate move so I thought that since it was hemorrhoids, I checked that off the list.
Symptoms worsen
It didn’t go away and it became more mucousy. There was one point where I passed a hard thing the size of a grape and it wasn’t poop.
Now I was pregnant again. I talked to a different doctor than I’d gone to before and she said, “After this baby’s born, we’ll get you in for a colonoscopy.”
Finally getting a colonoscopy
My son was born in January 2020, on the day the world announced COVID. I had trouble nursing him. As a mom, you always feel so guilty when you can’t nurse. A lot of moms feel that way and I felt since this is my 8th, I should be able to.
Because I had all these other kids, I couldn’t get in the right frame of mind to devote as much as I needed to nurse him. I put him on the bottle, which I felt guilty about but was a huge blessing because if I was nursing, I would have put the colonoscopy off for another year.
In April, I went in for a colonoscopy. The gastroenterologist said, “I’m so sure it’s hemorrhoids.”
I had a close friend diagnosed with colorectal cancer a year before me. She was exactly my age. When I told her my symptoms, she said, “Get in for that colonoscopy.”
Even though it was dismissed, I said, “I really want this.” I was 44 at that point and the gastroenterologist said, “I’m sure that it’s hemorrhoids. We’ll talk about it when you come out. We can do a banding procedure. It’s real simple.”
Diagnosis
Getting the official diagnosis
I went to sleep and when I woke up, the doctor was looking at me and she said, “I am so sorry. I did not expect to be telling you this, but there is a large mass in your rectum and I’m quite sure it’s cancer. It’s very hard and that’s generally a sign of malignancy.”
Reaction to the diagnosis
That was a huge shock. You’re never ready for that; that’s what everybody always says. I called my husband. He wasn’t there with me because he was watching the little ones.
I’m a pretty organized person, but when you get news like that, where do you go from there? They got a small enough sample for a biopsy because she was sending it to pathology.
She called, but it was not immediate. Although she did believe it was cancer, we didn’t know for sure.
Genetics
They’re seeing more and more younger patients. The doctor at Mayo said they were studying this pretty intensely. One particular doctor who’s studying it said, “I believe there is something that’s causing it. We just haven’t figured out what that thing is yet.”
I was told that a lot of times, at least with colon cancer, you don’t see blood. With rectal cancer, it’s more common because you’re closer to the opening.
They did genetic testing and there were no genetic markers, but I don’t know. My uncle died from it so maybe there’s some predisposition. My dad had polyps removed when he was young and forgot all about it.
My family got their colonoscopies and nobody had any issues. They were all good. There were a couple of polyps.
This doesn’t have to be a hopeless situation. There is hope in the midst of it.
Ananda P.
Treatment
Finding a cancer treatment center
My friend who had colorectal cancer went to the Cancer Treatment Centers of America in Tulsa and she had such a good experience so I called them.
We don’t have traditional insurance. We’re part of a healthcare sharing network so we’re handled as self-pay. We get it all reimbursed, but it’s not traditional. When I told CTCA that, they were really slow to call me back.
In Rapid City where I had gone for the colonoscopy, they referred me to a general surgeon. I immediately went online and found out that rectal surgery is very complicated and you really want a specialist doing it.
I called the Mayo Clinic and they were just starting to open up after going on restrictions with COVID. It was 4 o’clock in the afternoon and they said, “Can you be here at 8:30 tomorrow morning?” I said yes.
Honestly, I didn’t even know where Rochester, Minnesota, was. Being from Canada, my US geography is not wonderful. I told them I would come and found out it’s a 10-hour drive.
I called my husband and we figured out who would stay and who would go. I brought my 15-year-old daughter who had a learner’s permit so she could help drive and my three-month-old baby because he would be the most work.
Going to the Mayo Clinic
We drove through the night. She drove through the night to Minnesota on her learner’s permit. We got in at six or seven in the morning and I showered and went to this appointment.
They are so efficient at Mayo. I got in for the appointment with the gastroenterologist. They did a CT scan and an MRI. I don’t remember if they did the lower ultrasound at that point or if that was later; I think they did. By the end of the day, they know what you have.
I’m not sure exactly if everything happened all in one day or over the course of a couple of days. I know the results came in very quickly. We knew this was cancer.
Meeting with the rectal surgeon
I met with the surgeon first. He does rectal surgeries all day, every day and he teaches other people to do rectal surgeries. I remember him coming in wearing a suit and sitting on the bed. He was very professional but casual. He knew what he was doing.
He said, “Here’s what’s going on. We’re going to do the surgery a month from now,” so they scheduled it for June 1st. I said, “A month? What if the cancer spreads through my body?”
He explained that the kind of colorectal cancer I had was very slow-progressing. He said, “You’ve probably had this 6 to 8 years.” Back when I had that fissure those years ago, there was probably some rectal cancer already going on.
Surgery
We went back home and I came back on June 1st to have the surgery. It was quite a long surgery. They got the tumor out with clear margins.
When I went in, they thought it was stage 1 and that they caught it early. When they did the pathology post-surgery, they realized that there had been a small breakthrough through the rectal wall into the fatty tissue, which made it stage 3B.
Because of that, they recommended adjuvant chemo for four months to make sure it doesn’t come back. I would start in July 2020.
Adjuvant chemotherapy
That was the most traumatic thing that I had to go through. It’s the mental difficulty of knowing that you’re putting poison in your body and knowing I didn’t have cancer anymore.
They come into that chemo room in a hazmat suit because they can’t touch the chemo. The nurses are head-to-toe garbed up yet they put the needle into your body. It’s such poison and yet I’m willingly letting it be injected into my veins.
I really struggled with it. I wanted to quit. I did quit. I called my oncologist and said, “I’m done.” I was doing it in Rapid City. They basically explained chemo is chemo wherever you have it done so I decided to do it at home. But when I called her, she talked me back into it and explained the statistics.
Side effects of chemotherapy
I was doing oral chemo, which made me very sick and my stomach hurt really bad. Then they switched me to FOLXFOX. It’s a pack that you wear for two days then they take it out.
I didn’t get sick with that, which was amazing. That was an answered prayer because oral chemo was awful. I’ve heard other people say they liked oral chemo, but it was awful for me.
I remember one pharmacist said managing anxiety is an important part of your care plan. Up to that point, I felt guilty feeling so fearful.
Ananda P.
Managing anxiety
That time was really, really, really difficult. I felt like I should have been stronger, but I wasn’t. I would go outside, sit there, and breathe. It was hard to slow down my body to breathe.
They gave me Ativan for anxiety, which helped me sleep at night. I didn’t take it too much because I liked it and thought I would get addicted to it, but it did help me for a short time.
I’m very task-oriented so I go into the mode of what needs to be done. It was absolutely terrifying thinking I was two steps away, two stages away from terminal cancer. In between me and stage 4, there’s only 3C and I have a little baby and a lot of little kids at that point.
My teenage daughter was going through a really hard time. I couldn’t be there for her and that was so difficult. My son graduated from high school. He was homeschooling and he just finished his work. It was October and I was in the middle of chemo. I wanted to celebrate that, but I was in survival mode.
All these big things are happening and it was so difficult to function. It’s really good to talk about it right now because it was pretty traumatic. Processing the trauma takes a long time. You think you’re over it and then something will come up.
I rang the bell in November 2020 and the cancer has never returned. At the Mayo Clinic, they told me they had a very low recurrence rate with rectal cancer, which was the opposite of what I read online so that was really good.
Follow-up protocol
I regularly go for CT scans. It started with every six months and now it’s once a year. Once I get to five years, the chance of getting rectal cancer would be the same as anyone else getting it.
Words of advice
I think God gives us an instinct about our bodies. You don’t know what it is and you can’t pinpoint it, but something is wrong and that’s a real thing. That’s a real voice to listen to.
Be your own advocate. I know doctors don’t like it when you go online and do all this Dr. Google research, but I learned so much. My sister came down from Canada for five weeks and the two of us were reading all these medical journals nonstop.
We would call the doctors at Mayo and ask, “What about this?” We were pretty impressed with our own knowledge. I don’t say that boastfully, but my point is that you can learn about what’s going on with your body.
Not that doctors aren’t trustworthy; they are, but they’re not inside your body so they don’t feel that feeling of something is off. Only the patient feels that and that’s a real thing so listen to that.
What really threw me off, and I wish I had known, is all of the anxiety and fear. It’s not that you’re not strong enough to handle this. This is part of the process. My medical team was really good about explaining that to me, but I didn’t know it until I was far into the process.
I remember one pharmacist said managing anxiety is an important part of your care plan. Up to that point, I felt guilty feeling so fearful. I was so terrified and I thought, This could be it. This is what happens. People just all of a sudden get a diagnosis and then they die.
I was so scared for my kids. I had a friend who had never had kids and had never been married so she didn’t understand. She’s very kind, but she said, “You need to focus on yourself and not just be thinking about the kids,” and I couldn’t. I’m a mom. It’s instinctual to constantly be thinking about your kids.
That’s what I wish I had known. The anxiety is going to be crippling and it’s not your fault. It’s part of this journey.
Leaning on faith in God
The other thing I learned was to lean on my faith. I didn’t have the frame of mind to pray. I would put scriptures on my phone and read them in my desperation. Things like, “God is a refuge and strength, an ever-present help in times of trouble.”
I’m not in this alone. I have my sister, my sister-in-law, and my husband. I had friends. My friend Amy drove me to all my chemo appointments. She was amazing. I had a good human team and I had God who I relied on and we got through it.
There is an end, even for those who are diagnosed with stage 4, like my friend Julie was. It’s a day-to-day journey but there is strength and there is hope. This doesn’t have to be a hopeless situation. There is hope in the midst of it.
Thank you to The Patient Story for all you do. I watched so many videos and was on the website. It was really, really helpful to hear other people’s stories of what they’d gone through. You are doing a great thing and I’m really thankful for what you do.
Cancer Details: Rectal adenocarcinoma; 98% of rectal and colon cancers are adenocarcinomas 1st Symptoms: Blood in stool, constipation Treatment: Chemo, radiation, surgery, immunotherapy
Cancer Details: 7cm mass found 1st Symptoms: Fatigue, weight loss, fast heart rate, bladder infection Treatment: Chemo, radiation, surgery for tumor removal
Myelofibrosis experts Dr. John Mascarenhas (Mount Sinai) and Dr. Tania Jain (Johns Hopkins Medicine), and Clinical Trial Nurse Ashley Giacobbi (The Leukemia & Lymphoma Society) explain cutting-edge therapies. Hosted by The Patient Story Founder Stephanie Chuang and featuring insights from patient advocate Mary Linde, this empowering discussion will help you navigate all aspects of clinical trials.
The myelofibrosis panelists discuss momelotinib, pelabresib avtemadlin, selinexor, pelabresib, navitoclax, imetelstat and other myelofibrosis treatments.
Thank you to GSK and Karyopharm for their support of our patient education program! The Patient Story retains full editorial control over all content.
This interview has been edited for clarity and length. This is not medical advice. Please consult with your healthcare provider for treatment decisions.
Stephanie Chuang, The Patient Story: Hi, everyone! Welcome to the program hosted by The Patient Story and The Leukemia & Lymphoma Society.
I had a different blood cancer, non-Hodgkin lymphoma, and I went through treatment a few years ago, but I’m so passionate about these discussions. So many things are happening in the landscape of changing treatment options and they start with clinical trials.
We hear about clinical trials but the term is so daunting and overwhelming. Our goal is for you to have a much better understanding of what they are in human terms. Maybe some might be right for you that you can ask your own doctors about.
The Patient Story features hundreds of in-depth, authentic patient stories across cancers and we also feature top cancer specialists. Our goal is to humanize cancer and help you navigate life after a diagnosis, whether you’re a patient or a caregiver. You can join our community and you’ll get first access to programs like these with new updates and new stories.
We’re so proud to be co-hosting this with The Leukemia & Lymphoma Society. It is the world’s largest nonprofit health organization dedicated to funding blood cancer research as well as offering patient services and education.
They have great resources, if you haven’t checked them out yet. Their information specialists are just a phone call away to help you answer some cancer questions. They also offer help to pay for cancer costs through a co-pay assistance program, like travel to CAR T-cell therapy or clinical trials.
We also want to give special thanks to GSK and Karyopharm for supporting our free, independent patient education program. We want to stress that The Patient Story and the LLS retain full editorial control over the entire program.
This is not medical advice and not meant to be a substitute so please make sure to talk to your own healthcare team when you’re making any decisions.
Dr. Mascarenhas, really appreciate you being here. I heard you describe yourself as being tireless and working nights and weekends to help patients in this space. We really want to understand what drew you to MPNs and to continue to do this work for patients.
Dr. John Mascarenhas: Thanks for having me join you. My interest began in the laboratory doing leukemia research many years ago. It evolved over time into clinical research in which I was introduced to MPNs and I really gravitated towards it. This was at a point where we didn’t have JAK inhibitors.
I was at the right time when the discovery of the JAK2 mutation came out. I got involved in the early development of JAK inhibitors and watched the field blow up. It’s been really exciting and rewarding to see this transition from how we used to treat patients with myelofibrosis and other related MPNs to what it looks like today so I’m very enthusiastic and optimistic about the future.
Stephanie: Thank you, Dr. Mascarenhas. We know that the landscape has shifted very quickly in a short amount of time. Excited to talk about it.
Dr. Tanya Jain
Stephanie: We’re also really excited to have Dr. Tanya Jain here tonight, another MPN specialist. She is an assistant professor of oncology at Johns Hopkins Medicine and director of the Adult CAR-T Cell Therapy Program at the Sidney Kimmel Comprehensive Cancer Center with myeloproliferative disorders as a top area of interest and studying newer drugs in the early phase of development.
Dr. Jain, thank you for joining our discussion. We’d also love to understand what drew you to the MPN space. What’s the driver of doing this work for patients and their families?
Dr. Tania Jain: Thank you so much for having me. I’m absolutely delighted to be a part of this panel.
I don’t think it was a specific event. It was a natural transition during my fellowship under the mentorship of Dr. Ruben Mesa and Dr. Jeanne Palmer, who’ve obviously done a lot of work and continue to do so.
How that transitioned me was to recognize the significant unmet need in the space and the opportunity that existed to contribute to improve outcomes for patients and to improve the lives of patients and their families so that’s what we try to do.
Stephanie: Thank you so much. That means a lot for all of us who’ve gone through cancer.
Ashley Giacobbi
Stephanie: Speaking of incredible work, we have Ashley Giacobbi representing The Leukemia & Lymphoma Society’s Clinical Trial Support Center. It’s a really great resource because as we know, clinical trials can be very daunting and seem like a lot to wade through.
Ashley, thank you for being here. I have such a special place in my heart for nurses. I remember going through my treatment and nurses being such a lifeline so I really appreciate the work that you do. We’d love to understand more about yourself and what drew you to this calling.
Ashley Giacobbi: Thank you so much. I’m really honored to be here.
I have been a nurse for almost 20 years now and have enjoyed all of my time at the bedside.
I get extra excited when talking about new changes and how much has changed in the world of oncology over the last 20 years. It’s exciting to be able to help patients find clinical trials when they’re seeking the newest and next level of care and some of these new developments and advancements that we’ve had.
Stephanie: Thank you, Ashley. We’ll be talking about some of the top topics you’re getting from patients and care partners who are calling you and asking for your help in the clinical trial space.
Mary Linde
Stephanie: Finally, we’ve got Mary Linde, who I’m really blessed to be able to call a friend now. Mary, you’re a myelofibrosis patient advocate. You lead a group on Facebook with myelofibrosis patients and care partners. We’re so lucky to have you share your perspective as well. Can you describe yourself a little bit outside of cancer? As we know, we are so much more than a diagnosis.
Mary Linde: Thank you for that. I’m almost 61 years old. I’m a nurse as well and have both a bachelor’s and a master’s degree in nursing. I stopped clinical nursing for 25 years.
I’m currently the CEO of a small nonprofit foundation that mostly runs a retirement community here in San Francisco. I have always had a heart to serve the elderly.
I’m a mom to two amazing adult sons who are launched and living on their own. One is married and one is about to get married. I’m really hoping for grandchildren soon and to live long enough to really enjoy those grandchildren.
I have a three-year-old Coton de Tulear puppy who gets me out and moving. I walk 3 or 4 miles a day with him.
Mary’s myelofibrosis diagnosis
Stephanie: Like so many of us experience, a diagnosis changes our lives. What led you to figure out that something wasn’t right and how did you get your primary myelofibrosis diagnosis?
Mary: It wasn’t easy. I was about 54 1/2 when I started with vague symptoms. I had fatigue, dizziness, and sometimes vertigo to the tune of whoosh and then I fell. I felt like I needed to nap more frequently. I felt like if I walked up any incline, I needed to rest.
I knew something was wrong. I thought I had mono or Lyme disease. I kept complaining to my primary physician about these symptoms. He kept telling me I had menopause, which had long been through at that point.
I would repeatedly go back to my primary physician and say, “Please, let’s investigate what this is,” and even told him that my father had a rare blood cancer at around this same age.
Finally, six months into begging, I reached out my arm to the doctor and said, “I’m not leaving your office till you do a chem 7 and a CBC,” and he did.
Three days later, I got the results that I had platelets in the 800,000. Of course, Google became my friend. I called the doctor and he refused to talk to me. He said, “I’m sending your blood off for special testing. I don’t want to tell you because I don’t want to scare you.”
I started Googling and I was pretty certain I had an MPN. Three months later, I found out that I had the JAK2 mutation. I didn’t find out from my primary care physician; I found out when I got a call from the oncology center admitting me to services.
When I walked into the oncology center, I had a very strange experience. The introductory oncologist said to me, “I know you’re probably really afraid of the C-word so we’re not going to talk about it. Let’s talk about it like a blood disease.” I told her that I was a nurse so she didn’t have to do this. Then she said, “The other C word is an evil drug so we’re not going to talk about that either.”
I kindly asked her to get me another doctor and then sought a second opinion. At that time, I wasn’t so afraid of dying as much as I was afraid of not having the information I needed to live well. I didn’t know if my life was going to be shortened and I wanted as much information as I could get.
Stephanie: I really appreciate you sharing all that, Mary. I also love the message of self-advocacy and empowerment. It’s your life, you know your body, and you certainly didn’t let that fall through the cracks.
What is myelofibrosis?
Stephanie: Dr. Jain, what is myelofibrosis in layman’s terms? What are some of the more common first signs or symptoms?
Dr. Jain: What we heard from Mary’s story is something that I hear in the clinic fairly commonly. Having that fatigue and tiredness is something that often takes people to physicians to get tested. Delays are not uncommon either because fatigue or tiredness have several possible reasons. Some may be more common than others.
Mary also mentioned the JAK2 mutation, which is of relevance here. The premise of myelofibrosis starts with the overactivation of the JAK/STAT pathway, which is supposed to be a normal functional pathway that’s supposed to do regular stuff and make blood cells.
When it’s over-activated or activated without any restrictions, that’s when undesirable things happen, which can include affecting the bone marrow function in a negative way.
You could be making too many cells or you could alter the bone marrow function in the way that there is more scarring in the bone marrow, which is the fibrosis in the term myelofibrosis, which again affects the ability of the bone marrow to function normally whose job is to make a normal quantity and quality of blood cells.
As a result, patients can get anemic or have low hemoglobin, which can cause tiredness, fatigue, difficulty breathing, and related symptoms.
By virtue of the JAK/STAT pathway activation, this systemic or generalized inflammation that we often notice or patients leads them to a workup. What that results in is what we in our clinic call constitutional symptoms or symptoms that could be nonspecific or vague as sometimes labeled.
Those can include things like night sweats, low appetite, or other symptoms resulting from an enlarged spleen like abdominal discomfort, which are other symptoms that can sometimes lead to further investigations clinically.
Stephanie: Thank you. I know that’s a lot to cover in a short amount of time. We do hear lots of stories about how long it can take to figure out a myelofibrosis diagnosis so that’s not unique to Mary’s situation.
Landscape shifts in myelofibrosis treatments
Stephanie: Dr. Mascarenhas, you alluded to how optimistic you are because there are so many developments happening. How would you describe the landscape shifts that have happened in myelofibrosis treatments, especially in just the last few years?
Dr. Mascarenhas: Myelofibrosis is a stem cell-derived blood cancer and it could affect people in different ways. You don’t meet two people that walk the same path. They all come in different forms and fashions. Their clinical picture can be really varied and their course can be quite heterogeneous and variable, too. You have to understand the patient that you’re dealing with and the patient-specific goals of therapy.
For some patients, it could be alleviation of anemia. For other patients, it could be systemic symptoms like fevers, night sweats, weight loss, bone pain, and profound fatigue that can be quite debilitating. For many patients, it can be an enlarged spleen or liver that’s causing a lot of discomfort and challenging normal activities like bending over and doing things that would be normally easy to do.
Knowing how the disease is affecting the patient really informs and dictates how best to approach the patient. It’s typically trying to alleviate the symptomatology and reduce the spleen, and that’s usually using JAK inhibitors.
JAK inhibitors
Dr. Mascarenhas: Over the years, we’ve grown to have three clinically or commercially available JAK inhibitors: ruxolitinib since 2011, fedratinib since 2019, and pacritinib since 2022. And a fourth one, momelotinib, later in 2023.
This provides a lot of opportunities to try to address those symptoms. Those cytokine-driven symptoms that Dr. Jain explained are a result of this JAK/STAT pathway. These drugs are great in reducing a lot of that inflammatory cue that makes patients feel terrible, improving their sense of well-being and functionality, and reducing their spleen.
In doing so, patients have a better quality of life and, by virtue in most cases, will live longer because they can do the simple things that we need to do. They can move and eat. People who move and eat will simply live longer and do better than patients who cannot and that’s just generally true in oncology.
They have really revolutionized the quality of life aspect. As Dr. Jain pointed out, she worked with Dr. Mesa who was really instrumental in measuring quality of life and making that an endpoint for clinical trials and bringing attention to symptomatology and quality of life.
We have a great armamentarium that covers lots of different types of patients to improve their symptoms and quality of life.
We are developing drugs that are looking to try to improve anemia. Historically, we use drugs like PROCRIT (epoetin alfa) or Aranesp, erythropoiesis-stimulating agents to try to improve hemoglobins, get patients out of the transfusion suite, and give them some more energy back, or drugs like danazol, a synthetic male androgen.
There are drugs that we repurpose, which is a common theme in oncology, from other diseases like multiple myeloma. We use drugs called IMiDs or immunomodulatory drugs like thalidomide, lenalidomide, or pomalidomide. These can all be used in myelofibrosis off-label to improve hemoglobin with responses of about 20 to 30%.
The reality is we can improve hemoglobin in some patients, we can improve symptoms in the spleen, but there’s still really a lot left to do.
How JAK inhibitors work
Mary: Dr. Mascarenhas, could you tell us how JAK inhibitors work? What are their benefits and limitations?
Dr. Mascarenhas: JAK inhibitors are a class of agents interestingly invented in the setting of this disease and now have applications to a lot of different diseases that are not even malignant.
They intermittently reduce the JAK/STAT pathway that is inappropriately activated in the bone marrow cells. This pathway is responsible for causing the proliferation of blood cells and elaborating inflammatory mediators.
JAK inhibitors intermittently quell this cascade of events. That reduces the propensity to have this inflammatory state, this overproduction of blood cells, and seems to improve symptoms by reducing cytokines, which are inflammatory byproducts.
Patients feel better and can move and eat. It restores vitality for reasons that I personally don’t understand and I’ve never seen a good explanation for.
It also reduces the spleen. It’s a phenomenon I don’t really understand. It’s an interesting aspect of the drug. It reduces symptoms by reducing the activity of this pathway.
Unfortunately, these drugs don’t induce remissions. They don’t kill what I would call the maternal stem cell that gives rise to all of these abnormal cells. It doesn’t get rid of that cell.
It quiets down what’s happening in the bone marrow and the spleen allows for some degree of normalcy. The malignant cell population still remains in the body, in the bone marrow, and in the organs.
Unfortunately, we don’t see changes in the bone marrow that would lead us to believe that we’ve remitted the disease. Myelofibrosis is in the name. You have scarring in the bone marrow that typically remains even while patients enjoy the clinical benefits of the drug.
Then there’s the potential for downsides of the drugs. They often will exacerbate cytopenia or low blood count. Anemia and thrombocytopenia, low hemoglobin, and low platelet count can often get lower with these drugs.
We’ve pivoted to try to develop drugs that may not be as myelosuppressive, as count reducing and may even improve some of the blood counts.
Pacritinib can be delivered in patients with low blood counts and can improve hemoglobin in about 25% of patients. Momelotinib, a drug that has also been associated with improvements in blood counts.
What we now know, which we didn’t know when we started off in all of this, is these drugs are not just JAK inhibitors. They interfere with a lot of different pathways that may be relevant and irrelevant and cause toxicity.
For example, inhibiting ACVR1, which is another pathway that’s important for iron availability, might explain why pacritinib and momelotinib improve hemoglobins and why maybe ruxolitinib or Jakafi and fedratinib or INREBIC don’t.
There are a lot of nuances with these drugs that help us understand why they may fit niches and help certain people in certain ways and why you can provide serial JAK inhibitors.
If you fail one, it doesn’t necessarily mean that you wouldn’t enjoy some response to another. You can go from one to the other. The tailoring of the JAK inhibitor is somewhat of an art more than a science in some cases and requires knowing the patient, what the goals are, and trying to match the potential goals and potential toxicities to that patient.
Mary: Thank you for that. I’ve often heard that we’re not getting rid of our JAK2 but maybe turning the volume down on it a bit with the use of these drugs.
Determining the sequence of treatment
Mary: Dr. Jain, how do you know which JAK inhibitor might or might not work and which one to try first for a patient?
Dr. Jain: I’m going to piggyback on what Dr. Mascarenhas said and emphasize the point that no two patients with myelofibrosis will be the same. Everybody will have their own presentation or different things that you need to address.
Everybody will have a different trajectory in terms of their disease course and that’s what’s important to recognize as you’re thinking about what you’re going to start a patient on in terms of your choice of JAK inhibitor. We don’t have the same treatment for everyone. We’re looking at what we need to address in you as a patient.
If we’re trying to address high counts, a JAK inhibitor makes sense. If they have a big spleen and/or symptoms that will need to be addressed in combination with that, a JAK inhibitor would make sense based on the fact that we have the longest experience with it.
Ruxolitinib or Jakafi has been our first go-to. It was the first approved and we feel more comfortable with it. We know how to adjust the dose and move things around with it. If that doesn’t work, then the go-to next step in an ideal world would be a clinical trial.
If we don’t have one that would fit, it would either be fedratinib or pacritinib. The choice would depend on where the blood counts stand. If the platelets or the cell counts are on the lower side, pacritinib would probably be a better choice.
There are patients who have anemia as a major presenting symptom. In those situations, options such as Procrit, danazol, pacritinib to some extent, and hopefully momelotinib in the future are some of the options. Luspatercept is in clinical trials; we’ll see how that pans out. Those will be some of the options.
The third set of patients is those who come with more advanced disease in the way of more excess or a higher fraction of blasts or leukemic cells or very, very early cells in the bone marrow that indicate that these patients are headed towards the pathway of a more aggressive pattern of disease like leukemia.
In those situations, either in combination with JAK inhibitors or without something like hypomethylating agents could be considered usually en route to a bone marrow transplant, if that makes sense in terms of eligibility.
Stephanie: What’s clear is this is very individualized. There are so many considerations.
What are clinical trials?
Stephanie: Mary, can you bring us back to the day when you got the diagnosis? What was that like for you learning about cancer?
Mary: When I was initially diagnosed, it was over a long period of time so it wasn’t shocking exactly as I grew into it.
I thought back to my own father, who had a rare blood cancer in the late 1980s, had it for five years, and died at age 60. In fact, we were both diagnosed at age 55. I felt like I was somewhat prepared for how to manage this because I’m in the health field.
I had a bird’s eye view of it with my own father and I knew how to advocate for myself, especially since my dad had to advocate at a time when there really weren’t a lot of options for this cancer.
There’s a lot of fear and a lot of wondering. Will I be around to see my grandchildren? Will I finish my career? Will I make the contribution to society that I had hoped? Will I have a painful death?
I’m grateful over the years to have learned that I have so many treatment options available to me that I don’t look at the future as something grim at all. I just see it as my normal life.
I’m so grateful to learn of combination therapies because not all of us can make it to stem cell transplant, which is ultimately our cure.
There’s a whole lot more hope on the horizon for us with MPNs, particularly primary myelofibrosis. I’m just grateful for the research and the amount of research that’s being churned out these days, the clinical trials, and the combination therapies.
I’m so excited to be able to talk to experts in the industry about research and development towards not only treatment but cure for myelofibrosis.
Stephanie: We’ve talked a little bit about some of the drugs and combinations that are being studied now.
The first time I heard the term clinical trials, it felt a little daunting. There are lots of misconceptions about clinical trials. We do know that different stakeholders are trying to research what can be better to improve the standard of care for different patients and patient groups. One of the challenges is that people view clinical trials as a last resort.
Ashley, we know clinical trials might not be right for everybody, but they are an access to tomorrow’s treatment today. We’re sure you get lots of questions in your role at the LLS. What are clinical trials in layman’s terms and what are some of the top questions and misconceptions you hear from people?
Ashley: Clinical trials are carefully controlled research studies, which are conducted by doctors, researchers, and scientists. They may be investigating new therapies or therapies used in the past in a slightly different way or in combination with other therapies to find out which works better or might have fewer side effects. They’re looking at both tolerability as well as effectiveness of the treatments.
There are so many misconceptions out there, but one of the largest is that it’s for patients who have exhausted all options. That’s simply not the case. There are clinical trial options at all stages of diagnosis.
There are interventional trials, which test treatment options, as well as observational or registry trials, which help us learn more about myelofibrosis as a diagnosis but may not involve specific treatments.
Another concern that we regularly hear about is the use of placebos. Placebos have definitely gotten a bad rap. A placebo can be known as a sugar pill. They’re not very commonly used in cancer clinical trials because it just wouldn’t be right for us to use a sugar pill in place of somebody who needed active therapy for a serious or life-threatening disease.
When placebos are used, it would be in a setting where a patient doesn’t require any type of treatment at that particular moment or in combination with another therapy so that the patient is continuing to receive active therapy for the disease that they have.
Logistical concerns may come up as well. We always like to chat with patients about travel for clinical trials and what that might entail. We’ve talked about some of the academic medical centers and how important it is to have an MPN specialist.
Very often, investigators who are looking at clinical trials for myelofibrosis are in academic centers. We’re always encouraging and pushing for those clinical trials to be available in the community setting because traveling to and from those academic centers can be a real challenge for patients.
In addition to the logistical travel considerations, there are some misconceptions that clinical trial participation is free or that patients may even receive money for participating. Unfortunately, that’s not always the case. It’s really important that we explain that to patients.
Parts of the clinical trial that are investigational or are not approved will be provided by the clinical trial. Physician visits and lab work are things that we would expect a patient to undergo if they were receiving standard care. Those will still get billed back to the insurance company or may have to be paid for in another manner.
Each clinical trial is structured a little bit differently and it’s hard to know from an overview, but really it’s important to have a general understanding before diving into the specifics of any clinical trial.
If you are participating in a clinical trial, occasionally, there can be stipends available to support the patient or caregiver and that’s why it’s so important to reach out and find out more and make sure that you have all the information you need as you make a decision about participating.
Mary: Thank you, Ashley. It’s so important to hear that we do have access to medication, not necessarily the placebo. A lot of us do worry about that. We also need to check in to find out what is available to us in terms of financing.
Momelotinib, a new drug
Mary: Dr. Mascarenhas, let’s talk about single-drug therapy. There’s a new drug that was recently FDA-approved, momelotinib. What patient might it benefit?
Dr. Mascarenhas: Momelotinib is a JAK inhibitor so it’s much like ruxolitinib (Jakafi) except as I mentioned before, there are some nuances that make these drugs a little bit different. It also inhibits ACVR1, which is another pathway that regulates iron availability for red blood cell production. In its long development history, it’s differentiated itself from other drugs in large part by its ability to improve hemoglobins in a subset of patients.
It went to the FDA based on a study called MOMENTUM. The expected approval was in late June. It was delayed by the FDA, which is not uncommon, to September for re-review. My expectation is it will probably be commercially available in the pharmacies and available for prescription likely by mid to late September.
I encourage patients to discuss with their physicians if that drug might make sense for them or any of the other drugs that we’ve talked about, whether it’s fedratinib, ruxolitinib, or pacritinib. As we’ve said, it really has to be tailored to the patient.
I’m excited to see momelotinib come up. For a physician, it’s great because it gives us different options and allows us to tailor the treatment for each patient. That might be a great opportunity for some patients to either embark on that therapy or switch.
The clinical trials set the tone for the interactions with the FDA about what that label would look like based on the way the clinical trial was designed and run. We can only guess that there may be stipulations.
It may only be available after the use of a first-line JAK inhibitor or it might have to have certain requirements for depth of anemia level. There are some nuances that sometimes play into the decision-making and these are based on the clinical trials that led to the approval.
Best drug combination therapies
Mary: Dr. Jain, there’s combination therapy to think about. June F. asks, “What are the best drug combinations at present?” Can you introduce us to combination therapies?
Dr. Jain: I’m not sure if I can answer what the best drug combination is right now, but hopefully that is a question we will learn more about and address in the future.
There are drug combinations that are promising. It brings options that we can offer to patients. They’re mostly in clinical trials but hopefully, some will move the field forward.
Historically, we addressed the JAK/STAT pathway, which we have learned the most about in the last couple of decades. That is the primary pathway involved in the occurrence of myelofibrosis.
As we have learned more over the years, there are other pathways that are critical to the development of myelofibrosis and that is where the rationale for drug combinations comes in.
We’ve seen several clinical trials that have addressed that or used combination therapy in patients who don’t have a good response to Jakafi itself and need more than that. Many of those drugs have been tested in the first-line setting to see if patients would do better with combinations upfront rather than Jakafi alone, which has been the long-standing go-to first-line treatment for over a decade now.
Some of the drugs that you’ll hear about are Navtemadlin, for example, which is an MDM2 inhibitor. Dr. Mascarenhas presented data at EHA that showed a 35% spleen reduction in about one-third of patients in patients who weren’t responding to Jakafi or ruxolitinib itself.
There are BET inhibitors like pelabresib that I heard Dr. Mascarenhas talk about at ASH, which also is being tested in the first line in patients who are starting upfront treatment.
There are BCL-xL inhibitors like navitoclax, which have shown some improvement in the second-line setting. We’re awaiting a public announcement on the data for the first-line trial.
The premise is that you’re combining two mechanisms that have shown to work such that you do not have overlapping toxicities. That’s an important piece to remember.
JAK inhibitors can cause some decrease in blood counts; we’ve seen that with ruxolitinib. You want to add a drug that may not do that as much or may not do that to an extent that makes it prohibitive of a combination.
With pacritinib, for example, patients often would get gastrointestinal toxicity so don’t combine a drug that may also have gastrointestinal toxicity. That’s important to remember as we think about combinations, which we will see more and more in the future.
Logistics are certainly important to consider as we’re thinking about clinical trials, what the trial offers, the frequency of visits to the center, and whether that makes sense in terms of continuing to be on it or not.
When to consider combination therapy
Mary: Dr. Mascarenhas, what should patients ask when considering a combination therapy like Dr. Jain just talked about? How do we know when it’s right or the right time to start one?
Dr. Mascarenhas: The first question to ask is: Why is this combination for me? What would be the advantage in my case in terms of addressing my goals of therapy? Importantly, what are the potential risks?
As physicians, and I’m definitely included when I say this, we often focus on what we think is going to be the advantage of participating in a clinical trial. The reality is there are disadvantages sometimes. There can be toxicity associated with approved drugs and unapproved drugs that are in clinical trials.
Patients want to understand expectations of results and the potential toxicities that one could incur. What does the patient need to be aware of in terms of mitigating and reporting?
A trial is not really a passive experience. Patients are active participants. They’re not simply receiving a drug. They have to be very willing and engaged to report all symptoms and not just what they think might be important. They need to report everything to the study team.
That might involve calling ahead of their visit to let the team know what’s going on, making sure that all their medications are accounted for, and they’re following up. It’s an opportunity to assess whether a drug is active and adequately characterize the toxicities of a drug. As the drug moves along, it’s optimized in the delivery for future patients.
There are two objectives. How is this drug going to help me and what is my role in this whole process? It really should be a joint venture. It’s not a one-sided experience. We learn from the patients. Hopefully, the patient community learns from what we’re doing and everyone benefits.
Stephanie: Dr. Jain had set up the landscape of combination therapies and talked about different options. Dr. Mascarenhas, it’d be great to run through a few specific combinations.
Dr. Mascarenhas: Clinical trials today to a large degree are unlike what clinical trials were historically. These drugs are all rational with supporting pre-clinical data. People don’t always realize that they go through enormous amounts of testing through various aspects before they ever enter a human being.
There’s an enormous amount of regulatory burden that exists that’s really annoying but really important because it provides that sense of confidence in the investigators but also in the patients that what we’re introducing makes sense. We’re not just taking something off the counter and saying, “I wonder if this is going to work,” and throwing it into a clinical trial. These are rationally developed drugs.
An example is selinexor. Selinexor is a really interesting drug. It’s already approved for blood cancers that are not myeloid cancer like multiple myeloma and other B-cell lymphomas. This drug affects the shuttling of proteins in different compartments in the blood cell, in the nucleus, and in the cytoplasm.
Why does that matter? Because if you can affect where proteins are in a cell, you can ultimately affect the functioning of these proteins. Selinexor inhibits this shuttling protein and affects the way certain proteins exert their function, which might be important to the pathophysiology of the disease. This might even turn on other proteins and put them in an area where they can actually induce a good effect.
For example, there is probably a multitude of different changes when you use a drug like selinexor that shifts the cell’s functioning from one in which it’s acting inappropriately and nefariously into one in which you try to induce it to die through normal mechanisms.
There are normal mechanisms that are built into our cells that if they get corrupted by a virus or by cancer, they turn on this mechanism called apoptosis and undergo programmed cell death. Cancer cells have ingeniously figured out ways to get around that. We struggle each day to figure out ways to get them back on track, to encourage them to recognize that they’re inappropriate, and to undergo this programmed death.
Selinexor is a great example of a drug that affects multiple pathways and induces myelofibrosis, perhaps even the primordial cells, to undergo this process of death. It works best in combination with ruxolitinib and that’s a common theme that we’ve seen.
In oncology, it’s very rare, except for maybe CML or some other diseases, that we use monotherapies. In most of oncology, we use combination therapies to synergize together to get deeper responses and avoid overlapping toxicities.
That’s what we’re seeing with drugs like selinexor or navtemadlin, which has a great wealth of data at this point. These drugs are active as single agents, but they seem to be even more active in combination.
This is supported by studies that are done in mice that are engineered to have myelofibrosis with primary cells from people who have been generous enough to donate their blood cells. We experiment and figure out how we are going to make better drugs in the future.
I thank those patients. Seems like a small thing, but it’s a huge, huge aspect of how we move this whole machine forward in clinical research. All of that information allows us to say this combination really looks effective.
We’ve got JAK inhibitors that are commercially available. We’re going to add this drug that synergizes nicely in preclinical models and cells in the dish. We think it’s going to work better than each drug alone and we’ll figure out how to dose them and schedule them to minimize toxicity. That’s really where we’re seeing it.
Selinexor, navtemadlin, pelabresib (BET inhibitor), and navitoclax (BCL-xL inhibitor) all look good in combination with ruxolitinib. As Dr. Jain said, ruxolitinib has been around the longest and that’s the go-to drug that we combine drugs with.
We’re not even waiting anymore for patients to have failed the first line of therapy. We’re introducing the drugs earlier on. We get confidence in their ability and their toxicity profile. Why wait for patients to do worse? Why not try to get deeper responses earlier on?
The deeper we can reduce the disease burden, we believe that the better the patient will be overall. They will not simply feel better but have smaller spleens, which is important. Fewer disease cells in their body will hopefully mean longer progression-free survival.
Navtemadlin, selinexor, pelabresib, navitoclax, imetelstat — there’s a whole host of drugs that are aiming to really hit the clone and induce deeper responses.
Second-line treatment after Jakafi
Mary: For people who have been on Jakafi and, for whatever reason, the treatment wasn’t effective, what treatment options are available?
Dr. Jain: That’s a common situation we run into because as Dr. Mascarenhas pointed out, Jakafi or ruxolitinib has a very strong role. There are a lot of things that it does do, but there are a few things that it doesn’t do. Based on registration studies and clinical practice, if it works, it works for a few years and then we start losing that response.
A clinical trial available that would be a combination of a JAK inhibitor with something else or something added to Jakafi itself would be my go-to to consider. There are several of those going on at this time. There are also trials with single-agent drugs.
The way drug development goes is there’s testing of efficacy by using the drug alone. Then once we are comfortable with their efficacy and safety profile, we add it onto a ruxolitinib or a JAK inhibitor backbone.
If we see efficacy and safety in that combination, we move it up the ladder a little bit more and try to investigate if that would be a better option in the front line compared to Jakafi itself.
There are non-JAK inhibitor combinations that get added to Jakafi. Navtemadlin, an MDM2 inhibitor, has data. It restores the natural cell-killing pathway that should happen but is not working very well for some reason. We’ve seen patients respond in a post-ruxolitinib setting in terms of their spleen and symptoms.
There are other drugs like navitoclax, a BCL-xL inhibitor, that show responses in about one-third of the patients who were not having a good response to Jakafi or ruxolitinib by itself.
Pelabresib is a BET inhibitor. There are other BET inhibitors in the pipeline that are being developed with slightly different nuances to their conformation and how they inhibit the pathway. These are some of the categories of drugs that look promising and worth looking forward to.
Imetelstat has emerging data. It’s slightly different in the way it works. It inhibits the telomerase activity. It’s one of what we think happens in myelofibrosis or leads to that, even though the telomerase length may be not as long or in fact short. The telomerase activity tends to be high and that’s what imetelstat inhibits.
I’m sure I’m missing some here and that’s not for the lack of preference. Selinexor is another one that Dr. Mascarenhas already mentioned.
I would tell my patients that if we can get our hands on one of these combination clinical trials at that time, that would be my first go-to option.
If for any reason there are no clinical trial options available after ruxolitinib or Jakafi, then obviously there are other JAK inhibitors like fedratinib, pacritinib, and hopefully soon momelotinib. Patients may still benefit even though they stopped responding to or did not achieve benefit from Jakafi as a front-line JAK inhibitor.
When to get a second opinion
Mary: When is it right for a patient to seek a second opinion during this time of figuring out the right treatment options?
Dr. Mascarenhas: The right answer always is now. I don’t think it ever hurts to seek a second opinion at the time of diagnosis to confirm the diagnosis. There is a lot of heterogeneity in the way these diseases can present. There are overlaps and nuances.
It’s important to seek a second opinion because you may be a patient who should be considered for a bone marrow transplant, which is the only curative option we have. Sometimes that’s delayed too late and the patient misses the window of opportunity. For some patients, that may not be on their plate but having that discussion is really important.
I don’t recommend seeing lots of different people; that can be problematic. Getting a second opinion at each stage never hurts — at the time of diagnosis, if you’re not doing well on your first therapy, and when there’s consideration of second therapy.
If you have a physician who gives you pushback about getting a second opinion or comes across negatively, you might want to find another physician because it shouldn’t be an ego thing.
It’s your life. You’re dealing with cancer. You should feel free and confident that a second opinion is not a reflection of your treating physician. It’s getting more information and the more information you have, the better the outcome.
Dr. Jain: I agree with that 100%. The MPN community is small but also very well knit and I think we work very closely together. I’ve asked my patients to get second opinions and I’ve set them up for them if they need to go. I’ve sent patients to Dr. Mascarenhas and to others if there is, for example, a clinical trial opportunity that we may not have and they may have.
Sometimes it’s better for them to hear it from more than one person. We do a lot of things similarly, but there are some that we may have different perspectives about.
A common joke is if you ask 10 MPN experts, you’ll get 12 different opinions, and not because anything is right or wrong. There just may be a slight difference in our prior experience with a particular strategy or what our perspective about a particular strategy may be. All those are important things.
The role and timing of the transplant may benefit from a second opinion. Get another perspective as to what their center’s experience has been and how they may do something different. There are nuances to every transplant center and some of it may be more beneficial to you. Geography obviously plays a big role.
How to look for myelofibrosis clinical trials
Stephanie: We’ve talked about treatment options in myelofibrosis, but when you actually come down to needing to find one or stay on one, there are so many questions that we all encounter. We know that ClinicalTrials.gov is out there, but it’s a little bit difficult to navigate and that’s why the LLS’ Clinical Trial Support Center is so important. Ashley, what are your top recommendations in terms of looking for clinical trials?
Ashley: Often, people try to go about it on their own. ClinicalTrials.gov is the most comprehensive database of all clinical trials. In addition to cancer clinical trials, there are also clinical trials for all types of disorders and diagnoses.
It’s not always easy to hone in on what you need depending on past treatments you’ve had or medical history so it can be a challenging situation to find clinical trials that are actually applicable to you as an individual.
We always encourage reaching out to The Leukemia & Lymphoma Society. We have the Clinical Trial Support Center, which was developed as a response to help patients find clinical trials that are appropriate to them as unique individuals.
It’s a free service staffed by nurses who have expertise in blood cancers and clinical trials. Nurse navigators connect one-on-one with patients, caregivers, or even other healthcare providers who may need to learn more about potential clinical trial options.
We find out more about the patient’s diagnosis. We learn about any past treatments they may have had and find out about their medical history because all of those factors will play into a patient’s eligibility to participate in a clinical trial.
We also take a long time to find out about patient preferences and any unique obstacles that could hinder or drive participation in clinical trials. We help tailor some of the support and resources that we’re able to provide so that patients can overcome some of those barriers that may be in place.
After we’ve generated all of that information and really taken the time to get to know the patients, we produce an individualized clinical trial search. That could be related to geography and preferences. What matters most to the patient? Is this something that they’re really not interested in spending a long time in the hospital if they can avoid it?
We definitely look at all of those and produce a list that can be taken back and reviewed with their treatment team so that that can be part of the informed decision-making with the patient’s next steps.
We are available for all blood cancers, including myelofibrosis and other MPNs. It’s important to know that patients can reach out.
Half of the ongoing clinical trials are cancer clinical trials, but sadly, only about 5% of cancer patients actually enroll or participate. There’s so much opportunity out there and we’d really love to help overcome any barriers that patients might have so that they can consider participating.
The role of stem cell transplant
Stephanie: Transplant is such a huge topic for so many people and they hear so much about it. In the context of the landscape changing so quickly, do you feel that you and other specialists in the field will be recommending transplants less to people? This is probably a very individual response, but with everything happening, what’s the trend?
Dr. Jain: I actually think of it the other way. By virtue of having more effective treatment strategies to improve the spleen, symptoms, and get a disease response, we may be able to take more patients to transplant as a curative therapy.
A lot of times, lack of spleen response, poor performance by virtue of a lot of symptoms, or disease advancement into a more aggressive disease pattern become barriers to transplant. Having more options in the future will allow us to take more of these patients to transplant.
We’ve seen a lot of advances with a lot of these options. What we have not seen so far is the curative potential. Hopefully, we will in the future. All of these drugs have a lot of advantages in terms of improving spleen symptoms and cytopenias but we have not had definitive improvement or getting rid of the abnormal stem cell clone. That’s something that at least so far we’ve only achieved with a stem cell transplant.
At some point, as long as patients are eligible for transplant and that is something that would fit their life wishes in general, that would be a curative option to consider. Better non-transplant treatments will only get more patients to something more curative like a transplant.
Dr. Mascarenhas: I couldn’t have said it better myself.
Final takeaways
Stephanie: We’ve covered a lot of ground in a short amount of time. If there’s one thing you’d like for our audience of patients and care partners to walk away with, what would that be?
Dr. Mascarenhas: I hope to share my enthusiasm and optimism that we are making advances. There is a brighter future and I’m really genuinely excited about that.
Embrace the opportunity of clinical trials. Clinical trials are looked at for other people, but they may be important for you. Talk to your physicians. You may not go through with it but learning about it and exploring it is important for each patient.
Dr. Jain: It’s never a wrong time to see a specialist or to see someone else if something is not working out. Some symptoms are nonspecific. Never hesitate to be an advocate for yourself, to get things done, and to seek a second opinion.
As a transplanter, I’m obliged to pitch in that getting an opinion about transplant and at what course in your disease that makes sense is important to address. You don’t have to wait until you’re progressing or not responding to drugs. That needs to be addressed and outlined in an early appointment rather than waiting for too long.
Ashley: I really want to echo what’s already been said. It’s so important to consider clinical trials as part of the treatment plan. Even if they may not be an option at that particular moment, it’s important to be informed and look at all of those components as you’re making an informed decision about the treatment plan with your providers.
As a representative of The Leukemia & Lymphoma Society, I would really encourage any patient with myelofibrosis or any MPN to reach out. The Leukemia & Lymphoma Society has so many resources available, including clinical trial support but also so many others that we would really enjoy the opportunity to connect and provide those resources to patients.
Mary: Continue to have hope. Continue to want to thrive and live.
At the beginning of my journey with myelofibrosis six years ago, I was told I’d be dead by 72. Now they’re saying you could outlive this. You could die with this, not of it. Hearing both Dr. Jain and Dr. Mascarenhas talk about all these combination therapies and how they actually might get us to the cure was very exciting.
Conclusion
Stephanie: Thank you so much, Mary. Your story is so powerful. It’s also on our platform so feel free to check out Mary’s story at ThePatientStory.com.
Thank you to Ashley from The Leukemia & Lymphoma Society and to Dr. Jain and Dr. Mascarenhas for the work that you’re doing to push forward research and help patients and family members better understand their options in myelofibrosis and in MPNs altogether.
I hope you’re able to take away something that was really key for you, because that’s our goal, to empower you in your own care. We look forward to seeing you at a future program.
Special thanks again to GSK and Karyopharm for their support of our independent patient education content. The Patient Story retains full editorial control.
April was diagnosed with metastatic triple-negative breast cancer (TNBC) at age 40, shortly before the COVID-19 pandemic began. She initially found four lumps in her left breast and learned she was BRCA1+ like her mother, who passed away from ovarian cancer.
April underwent chemotherapy, a double mastectomy, and reconstructive surgery. Just 5 months later, the cancer returned and she had to do more chemo and surgery. After finally getting radiation, April was devastated to be diagnosed with stage 4 metastatic breast cancer in her lungs. She has continued to battle infections, side effects, financial hardship, and scanxiety.
Throughout her journey, April has leaned heavily on her faith, grace, and gratitude. She tries to focus on living life day by day, finding joy in her family, and setting future goals. April hopes sharing her story will encourage other cancer patients to have hope and know they can get through this difficult journey. Her resilience in the face of adversity is inspiring.
In addition to April’s narrative, The Patient Story offers a diverse collection of metastatic breast cancer stories. These empowering stories provide real-life experiences, valuable insights, and perspectives on symptoms, diagnosis, and treatment options for cancer.
This interview has been edited for clarity and length. This is not medical advice. Please consult with your healthcare provider for treatment decisions.
I’m an educator of over 10 years and an author, a mother, and a wife. I love to read, write, and paint.
Most of all, I love spending time with my family, serving in my local church, and, since I’ve been on this journey, being an advocate for other women who are on the same journey that I’m on.
Pre-diagnosis
Initial symptoms
Cancer has been a part of the majority of my adult life. My mother was diagnosed with breast cancer during my senior year in high school. I spent most of my 20s being her caretaker as she went through her journey.
She went into remission. Six years later, she developed ovarian cancer and that was a red flag for her doctors. They did gene testing and found that she was BRCA1 positive. This was back in the 90s when gene testing was just becoming more available.
She passed in 2007. They discussed my doing BRCA testing, but the preventative steps were to have preventative surgeries by having your breasts, ovaries, and uterus removed. At that time, I knew I wanted more children. I was early in my career. I was worried about how would it affect my ability to get life insurance and health insurance.
I had a really great OB-GYN when this happened and he said, “Let’s treat you as if you are BRCA positive and, every year, starting at age 26, you get a mammogram and a pelvic ultrasound, and stay on top of this until you have your next child. Then we can circle back around to these preventative surgeries,” so that’s exactly what I did.
For years, I tried to push that fear to the back of my mind. I ended up being blessed to have another child. I had my last son in 2015 and, ironically, my OB-GYN then was a two-time survivor so she was on me. She said, “Okay, you’ve had your child. Let’s get this BRCA test done and start looking at preventative surgeries.”
I did the BRCA gene test finally in 2019 and had my regular mammogram, which came back completely normal. About three months later, in January 2020, while on vacation for my husband’s 50th birthday, I was in the shower doing a breast self-exam. Because of my family history, I’ve always done breast self-exams and been very particular about making sure I know my body, what feels normal, and what feels different.
I felt four lumps along the side of my left breast that were not there before. My husband said, “Okay, don’t worry, we’ll just make an appointment when you get back and get it checked out,” so that’s what I did.
I made an appointment when we got back home. Within a week, I was in my primary care physician’s office. She felt it and was concerned so she immediately sent me over to the breast center. From there, it went from a mammogram the same day to an ultrasound.
The radiologist came in, looked at the ultrasound, and said, “We want you to come back in the next couple of days for a biopsy.” They did the biopsy, which was not a pleasant experience. About a week later, I was getting the call to come back into the breast center.
Diagnosis
Getting the official diagnosis
Ironically, my husband stopped to check the mail and the results of my BRCA gene tests were in. As we drove to get the results of the biopsy, I found out that I was BRCA1 positive.
We got to the breast center and I’m told that I have triple-negative breast cancer, which is very aggressive and fast-growing, which explains why I had a normal mammogram in October 2019 and then had these four lumps appear three months later.
My husband and I were in shock. We sat in the parking lot for about 15 minutes, cried, prayed, and tried to wrap our minds around how our life was about to change.
Being my mother’s caretaker, I was surprised that I wasn’t aware that there are so many different types of breast cancer. I thought breast cancer was breast cancer. I didn’t even realize that there were so many different types.
I was diagnosed on February 13, 2020, right before lockdown.
Treatment
Discussing the treatment plan
I first was sent to see the oncology surgeon. She explained to me all the details of what triple-negative was, that it doesn’t have receptors that a lot of oncologists target for treatment, and how aggressive it is.
The standard treatment for triple-negative was the same for everyone because there were not a lot of treatment options. For all triple-negative patients, it was chemotherapy with ADRIAMYCIN, which we call the Red Devil, a really strong chemo drug that you could only have once in your lifetime because it causes heart damage.
I did the carbo-Taxol-Adriamycin combo for six months and then I would come back to her so she could remove the tumor beds that were left.
She also explained to me that I was stage 2 because even though my cancer was still local to the breast, I had two lymph nodes involved, which automatically bumps you from stage 1 to stage 2, but I still caught it very, very early. It was just because of the lymph node involvement and how quickly it spread.
After surgery, the next step would be radiation. Because of my BRCA, they also wanted to go ahead with the preventive surgery and have my ovaries and uterus removed so that I would not end up developing ovarian cancer. BRCA increases my risk of having breast and ovarian cancer to up to 45 to 80%; the average person has a 12% risk.
The other thing I also learned is that it’s a huge myth that most cancers are genetic or hereditary. Most cancers are not. In my case, if you have the gene, it just gives you that extra tool to be more proactive in the beginning.
It was a lot to digest, particularly for me, because I had watched my mother go through it so I wasn’t going in completely blind. I knew what this was going to look like and I knew how bad it could get. I had to really fight those thoughts and memories that haunted me from being her caretaker and walking with her through the hardest moments of her journey.
I also had to deal with the fact that I had just turned 40. I felt like the year before was the best year of my life. We were doing all these things. My oldest had just graduated from high school.
It was just a shock that, at 40, this was what was about to happen. I was going to have a double mastectomy and deal with having a completely new body and all the side effects of the chemo.
My now eight-year-old was four back then. He needs his mother. Having lost my own mother, I can’t help but worry. Will I be here for him? That was one of my biggest fears. I need to be here for my children and for my husband.
I’m the type of person that I have less anxiety the more information I have. It helps my anxiety. I immediately started trying to educate myself and learn more about triple-negative, all of the treatments, and all of the surgeries.
The other fear for me, too, was that it was during the pandemic. I literally started my chemotherapy at the beginning of the lockdown. At that time, COVID was such an unknown. For my treatment team, it was one of their biggest fears.
This chemotherapy was so strong. They’d already told me it was going to basically wipe out my immune system. At a time when you need your immune system the most, they were going to have to completely wipe mine out so they were really afraid of what would happen if I did catch COVID.
My family and I had to be under very, very strict quarantine. Literally, no one left the house unless it was for gas or doctor’s appointments, not even groceries because we were doing deliveries for that. There was a huge fear. I felt like I was having to fight two monsters at the same time, pretty much.
Side effects of chemotherapy
With the Adriamycin and the Taxol, the first thing that happened was my hair fell out. I knew it was going to happen so I made the decision to cut my hair. I didn’t want to see large chunks of hair falling out.
I had a great group of girlfriends who lovingly came over to my house and brought a hairstylist. She cut my hair down to a buzz cut. In their own way, they found a way to keep my mind occupied and make it light and chat with me while she was doing it. They made me feel beautiful no matter what. They loved me and cared for me so much.
I carried that moment with me when I had to go to the infusion centers alone to remind myself that I wasn’t alone. Just because I was in the infusion center alone, I was still surrounded by so many people who love and care about me and have rallied around me.
After about the third treatment, even with the buzz cut, patches of it just started falling out. My husband ended up just shaving me completely bald.
I also started to feel the really hard hits to my body, which was the nausea. Even though they’ve come a long way with the pre-meds that they give you right before the infusion, I still was very nauseated.
The lining of my mouth, my esophagus, my stomach — everything felt on fire. The chemo attacks any of the fast-growing cells, which are your mucous membranes.
I couldn’t use the same restroom as my family because I’m toxic. I had to worry about kissing or hugging my child. On top of not feeling well, it was mentally and emotionally hard to deal with the side effects of how sick it was making me.
After each treatment, I would spend 2 to 3 days in bed because I was so fatigued. They gave me steroids to help with my appetite because, with everything on fire, you don’t really want to eat.
By the time the three days were over, I’d have two good days to be out of bed and be able to do things with my sons and my husband then it would be time for the next treatment. That became my life for six months straight.
Managing neuropathy due to chemotherapy
The chemo combo that I did could cause severe neuropathy.
At one breast cancer support group meeting, a survivor spoke about neuropathy and cold therapy kits. I heard that the neuropathy could be so bad that you’d have numbness and tingling so bad that you could lose the ability to button your clothes. There were even women who lost the ability to walk.
I love to paint, write, and create. I don’t want to lose the ability to do those things so it was really important for me if there was a way to help with that side effect to take advantage of it.
I reached out to her when I got ready to start this part of treatment. God bless this survivor. She basically delivered a cold therapy kit, which I had been looking for online. It was so expensive. She delivered this kit that had five sets of gloves and five sets of socks. There’s a special thermometer that has to measure the temperature of the gloves and you have to buy dry ice to go in there because they have to be kept at -15°F.
She delivered it to me and walked me through the process. It was amazing. I’m forever grateful and thankful for that because I don’t deal with neuropathy. I get tingling occasionally, but none of the severe neuropathy that I could have gotten. A fellow survivor was able to do that for me.
Testing positive for COVID during treatment
In the middle of chemotherapy, I ended up catching COVID. I went in to have chemo one day in July and my white blood cell count was too low to get chemotherapy. Then the next day, my whole family contracted COVID.
I initially thought it was a false positive because I didn’t have any symptoms and I don’t have an immune system. This has to be a false positive.
I had to go get tested for COVID every week for a month. I couldn’t go back to the infusion center until I tested negative. For a month straight, I tested positive for COVID. But somehow, by the grace of God, I remained asymptomatic and, to this day, my doctors have no explanation as to why.
It got scary for my husband. Thankfully, we all made it through that. When I finally finished chemo, it was August 2020 and I went to have the next step before radiation, which was to have a double mastectomy.
Surgery
Double mastectomy
When they did the double mastectomy, they also did immediate reconstruction with implants because it was either that or flat for me. They also took out several lymph nodes, not just the ones that were involved initially but several in that area.
Everything they removed, they take to the lab and test it to see if there’s any residual cancer. When I came back for the results in October 2020, everything that they took out came back clear for cancer so that was the best news. That’s the news that every cancer survivor wants to hear and I was super excited.
My surgeon suggested that I leave radiation on the table because, in her mind, there’s only so much radiation that your body can tolerate. She felt like with me being triple-negative and having a high chance of recurrence, that would leave more tools in the toolbox for later down the line, which made sense at the time.
My radiation doctor was not happy. She felt differently. She had her own formula that she called me to go over, but I trusted my surgeon. She was one of the best in the region and sat on a lot of breast cancer boards.
I honestly felt like it was an answer to a silent prayer. That’s one less thing I don’t have to do.
After seeing what my mom went through, I was determined to do everything that my doctor suggested. I was going to follow the entire treatment plan because I have my family to fight for. I wanted to be here for them so I wanted to do whatever it took.
Unfortunately, there was a little part at the bottom of the pathology report that I didn’t pay attention to that said that no biopsy clips were found in any of the tissue that was sent to the lab. Biopsy clips are little metal clips that they put into the areas where they biopsy to mark where they found active cancer cells
In my case, the chemo worked so well. Chemo works really well for triple-negative in that it shrinks the tumor all the way down to where it’s gone and you can’t see it. There’s really no way for the surgeon to see with the naked eye where it was unless there’s something there to be a place marker.
Even if the tumor is gone, it’s really important for them to go in and take out the tumor bed where it was because that’s where the microscopic cells can hide.
I got that pathology report in October. By March, the tumors were back in all the exact same places. Everyone on my treatment team was shocked that they were back so quickly, in five months or less.
1st relapse
Some breast cancer patients are able to get lumpectomies. In my case, from what my surgeon explained, because I had a double mastectomy, they felt pretty sure that they had gotten the tumor beds because all of my breast tissue was removed.
The only problem with that is my breast tumors were along the side of my breast, which was a blessing because it helped me to be able to find them early but it was also why it was important for those biopsy clips to be found.
Also, you have so many lymph nodes in your underarm so there’s no way to know if the correct lymph node was gotten or if there’s no more tumor left behind to see unless you’re counting those clips to say this many were placed and we took this many out.
As a patient, this is something that I’ve been educated about in hindsight. They say hindsight is 20/20. I didn’t know. All I saw was there was no cancer, thank God. I didn’t know that no clips meant that maybe there should have been further investigation at that time to find those clips and make sure that everything that needed to be removed had been removed.
Second round of treatment
I ended up having to go through a second round of chemotherapy in 2021 and another surgery. It was a different set of chemo drugs.
At that second surgery, the radiologist brought it up. She counted the clips and said, “She had this many clips and there were only this many sent to the lab. We need to do some more scans and figure out if there’s still some biopsy clips left to make sure that everything is removed.”
Side effects of chemotherapy
The side effects weren’t as bad. I didn’t lose my hair. I wasn’t as sick. I still had nausea and digestive issues, but it was a lot easier.
They added an immune therapy drug that was approved while I was going through treatment. I started having some side effects from that as well, but they were manageable.
The treatment was shortened because they were trying to get my surgery scheduled and it got moved up. You have to have a four-week break between chemo and surgery.
From that surgery, things spiraled for a bit because the clips were brought up and I had to have an extra surgery to retrieve those clips and that set the schedule back. It was 2022 by the time I started radiation.
Radiation
I was very blessed to get some of these newer options out there. I received a new type of radiation called proton therapy because I have left-sided breast cancer. Your heart sits on your left side.
Proton therapy is supposed to be more targeted and precise so that it can better avoid your heart, your lungs, and other important organs that may not be involved with breast cancer.
I had to go to downtown Atlanta every day for two months.
Side effects of radiation
I did really well. Even though I burned, it wasn’t severe. My skin turned very, very dark along my neck; it still is a different color. It never completely goes back to the original color. But when it burned, it turned very, very dark, dark brown then it started to peel.
Thankfully, because I was constantly putting different things on daily, it was already new skin underneath when it peeled. For some patients, it’s still raw and they have to do antibiotic ointments. I was blessed to not have that severe of a burn.
Compared to all of the other treatments, I have to say radiation was probably the easiest treatment that I had. It’s just that it does do a lot of unseen damage.
You know how when you sit in the sun and get a sunburn but you don’t feel anything and then afterward you get that peel? That’s how radiation felt. I didn’t feel anything. Maybe a little tired or fatigued afterward, but other than that, no major side effects until after the radiation was done.
I started to have side effects from the damage it does to your tissues internally. About two to three weeks after I finished, I ended up in the hospital with a major chest infection.
Radiation damages the tissues and the blood flow so without the blood flow, any normal bacteria that your body would fight off, that tissue can’t fight off. It doesn’t have blood flow, which means the white blood cells can’t get over there and all the things that your immune system sends to defend your body.
After I healed from the radiation, everything looked good. I went on the treadmill to try to get my energy back and get rid of some of the fatigue. When I was going through radiation, I didn’t wear bras because I was trying to be very gentle with my skin. But afterward, I thought it looked good so I put on a sports bra and got on the treadmill for a light workout.
My infectious disease doctor seems to think that because of the friction from the sports bra and because the skin was so thin, bacteria from the sweat got into the chest. I was in the hospital for almost ten days before they could get it under control. They were able to clear the infection. They gave me some really strong antibiotics intravenously.
2nd relapse
Getting a diagnosis of metastatic breast cancer
This was May 2022. While I was there, they were doing scans to look at the infection and saw nodules in my lungs. Initially, I thought this was just inflammation because I just finished radiation.
They did some more tests. The doctor explained that it was very suspicious for metastatic disease and that I was now considered stage 4 with lung nodules.
She explained that the only way to confirm it was to do a lung biopsy. But being in the hospital with a massive infection, I wasn’t in a state to have that done immediately. It was later done in September. I had follow-up scans between May and September.
Initially, they said to just monitor it and see if it grows. That will also be an indicator if this is cancer or not. In August, it showed growth. At that point, they said the only way to be sure was to go in and biopsy.
Surgery to remove nodules
It turned from a biopsy to a lung surgery because most of the nodules were very small. They don’t want to go digging in my lungs so they’re looking for one that was on the edge. The largest one that they felt comfortable to biopsy was right next to my heart. It turned into a surgery so that they could deflate the lung and make it fall off away from the heart.
They ended up taking that entire one out, which was good because it ended up being breast cancer in the lungs. They cut that little wedge out and took out that nodule.
Reaction to the metastatic cancer diagnosis
In September when they confirmed that I have metastatic breast cancer in my lungs, it was devastating. It wrecked us because we were now into year three; three straight years back to back with no remission. We had maybe a month or two here to come up and take a breath. No real relief from this constant fight against this disease. It felt very defeating.
It took some time to process and put it into perspective through my faith and understanding that there is still life after a metastatic diagnosis. It took a while because immediately after I got the results of the lung surgery, the chest infection flared back up, but this time, it came back with a vengeance.
Recurrence of chest infection
It was ten times worse than it was before. This time, I was in the hospital for nine days. They were giving me the strongest antibiotics, but they weren’t working. It got very scary when the antibiotics weren’t working.
My blood pressure dropped down to 40 every day and they could not get it up. That’s when they realized this was going south. This was not going in a good direction.
The surgeon that was assigned to me explained that she was going to have to do emergency surgery to take out my left implant so that she could manually wash out my chest wall and clear out some of this infection.
Thank God she did because she said, “If we don’t do this, you’re probably hours away from going septic. The fact that we’ve given you the strongest antibiotics we have, we’re not going to have anything to help you if you go septic and you can die from sepsis in hours,” and I felt it.
Surgery to clear out the infection
They did the emergency surgery, which was scary because I wasn’t even a year out from the radiation. There’s always a concern with wound healing so they don’t like to do surgery at all on radiated skin earlier than a year, but I didn’t have a choice. Thankfully, she was able to go in there and get out a lot of infection.
When they released me, I had dropped down to 125 lbs. I had to go home on IV antibiotics that I had to administer to myself every day for six weeks to get all of that infection out once and for all.
Adjunct therapies
Hyperbaric oxygen therapy
When I was in the hospital for the second infection, the infectious disease doctor who handled my case had a hyperbaric oxygen therapy center that he also ran. He suggested that I do hyperbaric oxygen therapy, which I had never heard of, ever. Apparently, it was something that he did for a lot of cancer patients who had radiation, especially those who had issues with wound healing.
It was a nerve-wracking experience, but it made a huge difference in healing. When I first got out, my entire chest felt like a statue. It was just so hard and there was just very little blood flow there that it didn’t even feel like flesh. I couldn’t lift my arm. There was so much scar tissue.
I started doing hyperbaric oxygen therapy. The experience with that is a lot like radiation in terms of having to go every day. For two hours, you lay in a pressurized chamber that forces high levels of oxygen into the cells, which forces your stem cells to regrow blood vessels and all these different things.
I did that for 44 treatments over the course of two months. Halfway through it, I started to feel my chest softening. I could feel the blood flow returning. That was the amazing part, being able to see the difference with my own eyes. He felt that would set me up in good standing, in case anything was to ever happen, like another surgery, or protect me against any future infection.
I’ve shared it with every survivor I’ve talked to who’s had radiation. I wish I had known about it sooner, but I’m glad that I was blessed to find out about it.
Vitamin C infusions
I go to a medical doctor to receive high doses of vitamin C by IV. I was 125 lbs. I had no appetite. I was super weak. I couldn’t stay out of bed for very long to do things.
The high dose of vitamin C helped me with the fatigue. It helped me regain energy. It gave me back my appetite. I gained my weight back. I’m up to 149 lbs now, which is much better
It helped with all of the side effects and gave me back my quality of life where I wasn’t weak and in bed all the time. That was an amazing game-changer for me, too.
The only negative side to it is that my insurance didn’t cover it because even though it’s given by a doctor, it’s considered a holistic treatment. They’re now approving certain things like acupuncture but this particular treatment wasn’t on the list so I had to find a way to pay for it myself. It’s been a struggle and continues to be, but it has made a huge difference.
A lot of patients come to this doctor and do it in conjunction with chemo or whatever treatment they’re on. It helps them with the side effects.
Financial toxicity due to cancer
I didn’t realize all the ways that cancer affects you and your family outside of the physical, mental, and emotional. It completely drains your finances even when you have good medical insurance.
There are patients out there who don’t have access to health care. I did have access, thankfully, through the Affordable Health Care Act. I was very blessed that my health insurance covered the majority of my medical expenses. I still had deductibles, copays, and things like that.
However, it’s also the loss of income and not just by the patient. We live in a city where we don’t have a lot of family. We have a church family and friends. They’re a great support, but during the pandemic, it fell on my husband to be my caretaker and take care of the kids. There was a lot of time that was taken away from him being able to earn income as well so we did what we could for as long as we could.
We worked hard throughout our lives to be good financial stewards. We had savings. We had retirement accounts. We did all the “right things,” but this cancer diagnosis wiped all of that out just trying to survive and not being able to go to work. A two-income household and me not being able to work is a whole hit to our finances.
Being unable to work coupled with being my caretaker and getting diagnosed the second time became overwhelming to him. Men handle stress differently. They tend to internalize and he ended up having a massive heart attack.
I can’t help but think that some of that is due to all the stress of being a caretaker and trying to figure things out, provide, and show up every day for his family. We almost lost him. It was a heart attack that they call a widowmaker. He went through his own recovery at the same time.
Now we’re both dealing with health issues and that level of financial toxicity reached a point where we had to make the hard decision to sell our home of 10 years. We raised our boys in that home and all of our memories were there, but it was a decision that we had to make.
We still had to find a way to pay for treatments that I had to have that insurance didn’t pay for. It was just what we had to do, but it was such a hard decision to make.
The last time I got out of the hospital, I had to come home and start packing because we had closed on the house. We had to be out within 30 days after I got out of the hospital. It’s so hard to be dealing with such a horrible diagnosis and then have to figure out financially how to get through this.
Even though there are so many organizations out there for research — and I’m so thankful for cancer research — I have a special place in my heart for the grassroots organizations that are boots-on-the-ground organizations that provide help to patients themselves who are currently on the journey.
There are so many things that you don’t think about. I’m helping the organization that helped me, My Style Matters, founded by Tiah Tomlin, the young lady who got me the icing kits. I’m helping her now with some of her programs and the things that you don’t think about that become a struggle with the diagnosis — school supplies, Christmas, things like that.
They’re not on the priority list, but as a parent, it’s way up on the priority list. Being able to take those burdens off of patients makes a huge difference. It’s all these little things that you just don’t even think about or realize.
Patients are overwhelmed enough. There is a huge need out there when it comes to managing breast cancer patients or patients in general.
Follow-up protocol
It took me a while to recover from the infections to be able to handle treatment. I’m still doing the vitamin C infusions weekly.
My doctor wants to start with a new targeted therapy recently approved for triple-negative breast cancer called PARP inhibitors, which you take daily. She wants to start with the least toxic option since I am metastatic and I’m in it for the long haul for now.
Both doctors agreed that I could do the PARP inhibitor and vitamin C together. It’ll be a mutual benefit because the fewer side effects, the more PARP inhibitor I can tolerate.
My scans have been every three months right now so that continues to be the plan. There have been really good results with this PARP inhibitor, especially for BRCA patients and for triple-negative patients as well so I’m thankful for that.
There are side effects that come with it, like nausea and digestive issues.
The main one is fatigue and severe anemia. It can kill red blood cells, which can cause severe fatigue and anemia. Sometimes you have to have transfusions. My oncologist is very good about titrating, monitoring lab work, and staying on top of those things.
In February, I had a scan that was looking really promising. Everything was stable from August of last year. In February, the scan, everything looked the same as in August, which was great. They actually said a couple of nodules were shrinking so that was great news.
I had another scan in July that said things were growing again, but that’s the nature of this metastatic journey. It’s ups and downs and I’m learning how to not be just thrown off course.
Words of advice
Importance of advocating for yourself
When you get diagnosed, if you don’t have a medical background or you’re not familiar with a lot of the terminology, it’s so important to not be intimidated and to educate yourself.
Google is not a doctor, but it can be a great translator when it comes to some of these medical terms. If there’s something that you don’t understand, ask questions.
Most importantly, read your charts, your notes, and your pathology reports. Don’t just rely on your doctors to tell you what’s going on. Read so that you can understand and be ready with questions.
It’s your body and no one has to live with the results. Doctors are great, but they’re also dealing with however many patients they have coming to their practice on a daily basis. They’re human.
It’s really important for us as patients to educate ourselves and be active members of the team, even though we’re not medical professionals. Make sure you are staying on top of things and understanding things for yourself.
Managing scanxiety
After three years of dealing with this, I had a social worker tell me at the radiation center, “There’s such a thing as medical PTSD,” and I’ve never heard it phrased that way, but that’s exactly what it feels like.
When I go into the infusion center, my body has a physical response to the sight of the infusion center and the smells so there is this high level of anxiety.
I usually manage anxiety pretty well. It definitely feels like medical PTSD because it’s out of my control and it’s an actual physical response.
I dread every piece of information. It’s just information. The doctors are not trying to give you doomsday information. It’s cancer and there’s nothing good about cancer. I do have to mentally prepare myself.
Leaning heavily on faith
I lean heavily on my faith in God, which has gotten me through this journey more than anything else.
Before I go in for a scan, I tell myself, “Today, I feel good. I have my health and strength today at this moment.”
When I walk in, I’m getting information and when I walk out, nothing is going to have changed besides having that information. I’m going to walk out the same way that I walked in — feeling good with my health and strength today.
I try to focus on today and take it moment by moment. There’s a scripture that says that God gives us enough grace and mercy for today and the future belongs to Him so I try to rest in that. Today is what I have and it’s a gift.
There are three things that I’ve used to get me through this and they have become my mantra and that is faith, grace, and gratitude. Those have helped me get through this journey in my hardest and most difficult moments.
I try to tackle everything from a place of faith and not fear. I try to give myself and others as much grace as I need and as they may need. Always try to find a reason to be thankful every day, every moment, no matter how bad, even on my worst days.
I allow myself the moment to cry and to grieve. We’re human. It’s important to allow yourself those moments to curl in a ball on the floor and just let it out, but I know I can’t stay there. I won’t allow myself to stay there. I won’t allow myself to be destroyed or conquered in that way.
Find something to be grateful for, whether it’s my son’s smile and his little face, walking outside and seeing my favorite red cardinal or blue jay flying by, or a sunny day. Take those things in with appreciation and gratitude.
Focusing on the future
I try to set future goals for myself so that I can keep my mind focused on my future.
When you get scan results, it can put you in a mental space where you feel overwhelmed and want to just give up. You think, What’s the point in planning for the future?There’s not going to be a future, why plan for one? To fight that, I set future goals to make my mind focus on having a future with my family.
The first time I went through chemotherapy, I decided to get my real estate license so that was something for me to focus on.
The second time I went through chemotherapy, I decided to write a book for my youngest son. I wanted to give him something that we could read together every night and would remind him no matter how hard things get, God promises He will give us what we need to get through those hard times to brighter days. There are always brighter days on the other side.
I wanted him to have that that daily reminder and it was something that we could do together. He helped me with the drawings and the pictures. It was good for us. It was also my prayer that it would help other families with young children who were going through similar hard times and encourage their children as well.
Encouraging other cancer patients
My prayer is that by sharing my journey, I’m able to help some other person out there dealing with a cancer diagnosis.
Be encouraged that no matter what your diagnosis is, it’s simply a diagnosis. It’s simply a journey that you’re on. It’s not a journey that anyone wants to be on, but there is still a joy to be found. There’s still life to be lived.
You’re here for a purpose. Every morning that you wake up, there is a reason that you are still here. I just want them hopefully to be encouraged by a little portion of my story, to have faith and know that with God, you can get through this. You can and you will get through this.
Symptoms: Four lumps on the side of the left breast Treatment: Chemotherapy (carboplatin, paclitaxel doxorubicin, surgery (double mastectomy), radiation (proton therapy), PARP inhibitors
Cancer details: Triple negative doesn’t have any receptors commonly found in breast cancer making it harder to treat 1st Symptoms: Lump in left breast Treatment: Mastectomy, chemotherapy, 2nd mastectomy
Melissa B., Stage 1 Breast CancerDiagnosis: Stage 1 Triple Negative Breast Cancer Symptoms: Pea-sized lumpTreatment: Chemotherapy, surgeries
Cancer details: Triple negative doesn’t have any receptors commonly found in breast cancer 1st Symptoms: Lump in left breast Treatment: Chemotherapy, surgery
Susan S., Recurrent Breast Cancer Diagnosis: Breast Cancer Symptoms: Lump, twisted and caved-in nipple Treatment: Double mastectomy, radiation, lumpectomy, chemo
The Hodgkin lymphoma treatment options live discussion took place in August 2023, hosted by The Leukemia & Lymphoma Society, Imerman Angels, and The Patient Story.
Sharing from real-life experience, the panelists were Stephanie Chuang, founder of The Patient Story and non-Hodgkin lymphoma survivor, Dr. Matthew Matasar, Hodgkin lymphoma specialist at Rutgers Cancer Institute, Dr. Samantha Siegel, both a doctor and Hodgkin lymphoma patient, and Chelsey Gomez, Hodgkin lymphoma patient advocate and artist behind Ohyouresotough.
The discussion covered an overview of Hodgkin lymphoma, standard, and emerging first-line treatments, options for relapsed/refractory patients including immunotherapy and stem cell transplants, managing side effects, the importance of doctor-patient relationships and shared decision-making, and key takeaways about community support and focusing on the quality of life during and after cancer treatment.
Thank you to Seagen for its support of our patient education program! The Patient Story retains full editorial control over all content.
This interview has been edited for clarity and length. This is not medical advice. Please consult with your healthcare provider for treatment decisions.
Stephanie Chuang, The Patient Story: Hi, everyone! I’m very excited to have everyone to join us. We’re hosted by The Leukemia & Lymphoma Society, Imerman Angels, and The Patient Story. We have an incredible group of panelists tonight.
I’m a non-Hodgkin lymphoma survivor, founder of The Patient Story, and first and foremost, a patient advocate. The Patient Story was born out of my own experience with cancer. At the time, as a patient, I was looking for humanized answers for what my life with cancer would look like.
Fast forward to today, The Patient Story has hundreds of in-depth conversations and stories with cancer patients, care partners, and top medical experts in video and across our platforms. You can find us on ThePatientStory.com, YouTube, and social media channels. The goal for us is to help navigate people after getting that diagnosis.
We’re proud to partner with The Leukemia & Lymphoma Society or the LLS, which is the world’s largest nonprofit health organization dedicated to funding blood cancer research. They also provide a lot of education and services and that includes their information specialists who are just one call away to help with your questions. They also have financial scholarships and we’ll talk about that at the very end as well.
Last but not least is Imerman Angels, a wonderful peer-to-peer support group program. I used Imerman while I was a cancer patient and they will connect cancer patients and caregivers with mentor angels. They will use things like age, gender, where you live, and experiences to try and make that match.
We also want to give a special thanks to Seagen for supporting our educational program and allowing us to really do the work that we want to do in true patient education, connection, and space and provide it for free; that’s really important to us.
We want to stress that The Patient Story, the LLS, and Imerman all retain full editorial control of the entire program. A reminder that this is not meant to be medical advice or a substitute for medical advice. It is educational and we’re hoping that you’re able to take away great information tonight back to your own doctors and healthcare team.
Introduction
Stephanie: First up, Dr. Matthew Matasar, someone we’ve been able to work with before. He’s the chief of the Division of Blood Disorders at Rutgers Cancer Institute. He’s been a medical oncologist specializing in lymphoma for more than 25 years and leads clinical trials to try and find new and better ways to treat diseases like Hodgkin lymphoma.
Dr. Matasar, what drew you to lymphoma? What inspires you to do the research that you do and dedicate yourself to patient care?
Dr. Matthew Matasar: First of all, thanks for having me. I’m really thrilled to have this opportunity. What we’re doing together really matters and makes a big difference so thanks for making this happen.
I started out as a philosophy major back when I was in college, wanted to go into medical ethics, and then got sucked into oncology.
I saw oncology in general and lymphoma, especially as a place where I could make a difference, where being a really good doctor or being a crummy doctor makes a difference. I wanted to be the kind of doctor who listens to his patients, works with them as individuals, and understands that when they’re coming to me, they’re having the worst day of their life. I want to try to make it a little bit better using my brain and my heart as best I can.
If I do my job well, it’ll be better than if I don’t. These things matter and what I do matters. I feel this pride in knowing that what I’m doing is making a difference for people in my clinic, individual by individual, and by trying to develop newer, more effective, and less toxic treatments.
Maybe I could leave a little bit broader mark on the world. Trying to make a difference. What we’re doing here is trying to make a difference.
Stephanie: Yes, and you’ve been doing that and we really appreciate that you go above and beyond to help patients and their families.
Next up, from one doctor to another, Dr. Samantha Siegel, both a doctor and a patient, which is a really interesting perspective. Sam, I’m really, really grateful to have you here and lucky to get to know you. Thank you for all that you do.
We will get into your Hodgkin lymphoma story shortly but, first, we’d love to hear more about you outside of the cancer diagnosis because as we know, we are so much more than that.
Dr. Sam Siegel: Thanks for having me. I’m so excited to be here and to connect with you and all of the patients, caregivers, and community members.
I am one-half of a sandwich. I’m married to another doctor named Sam, but we got married before med school. Sam squared, Samwich, he Sam she Sam — a lot of iterations of that that are fun and interesting.
I’m a proud mom of three kids. They’re my best teachers in this world. They’re so incredible. Parenting them through cancer and through medicine has been very interesting. It’s always exciting. Our house is never boring.
I love jogging, painting, and playing guitar. I’ve recently become an enthusiast of ecstatic dance. It’s like a nightclub but during the day. No booze and kids are allowed. It’s just freestyle dancing.
I used to dance growing up and I’ve gotten back into it lately as a way to connect with myself and my body. I found it really helpful in healing from chemo and chemo treatment. I love dancing, music, moving through music, cooking, and food.
I’m hoping to unify how other people enjoy aspects of being alive and how we can talk to our doctors about how to tailor our cancer treatment to what matters most to us. That’s really important to me because if I couldn’t jog anymore or I couldn’t play my guitar anymore or paint or work with my hands, that would be pretty devastating to me.
All of that matters when it comes to talking with my doctor. I’m excited to be here as a doctor, as a patient, as a person, as a human being, first and foremost so thank you.
Stephanie: Thank you, Sam. I couldn’t have put it better myself. It’s not just about extending life, it’s the quality of life and even after treatment.
We will talk about the long-term side effects because we all want to live and get back to living the way that we know how and maybe better.
Up next, another awesome rock star. You may know her as the genius behind Ohyouresotough, which is amazing artwork. Chelsea Gomez, thank you for being here tonight. As a patient advocate, you’ve grown such a community yourself. Can you also tell us more about you outside of the diagnosis?
Chelsey Gomez: Hi, everyone! I’m so happy to be here.
I’m from Florida. I just turned 33 and have a daughter.
I’m a professional artist. I own my own cancer awareness brand named Ohyouresotough. If you ever see anything of mine, you’ll see that I like to cope with hard things with humor. It’s really important to see the lighter side because a lot of the cancer world is not so fun.
I love all things art. I use clay, I paint, and I do digital art. When I’m not doing art, I’m running after my daughter to do whatever she wants to do, like play Barbies. I’m really excited to be here. Thank you for giving me the opportunity to share my story, too.
Stephanie: We appreciate it. Without voices like yours and Sam’s, we wouldn’t have the platform that we have today. Both of your stories are on The Patient Story so thank you for being here and for sharing your voice to help other people.
What is Hodgkin lymphoma?
Stephanie: Let’s get down to business. We’re going to try to avoid medical terminology as best as possible. Dr. Matasar, what is Hodgkin lymphoma?
Dr. Matasar: Working through things without terminology is theoretically what we’re supposed to be doing all day any day. When we’re talking to patients, families, and caregivers, we try to help people make sense of their illness.
What is lymphoma? Lymphomas are types of cancer. They’re cancers of cells called lymphocytes or immune cells. Lymphomas collectively are cancers that come from and are of the immune system.
That’s not to say somebody who has a lymphoma has a bad immune system — far from it. In fact, most people diagnosed with Hodgkin lymphoma have perfectly fine immune systems. They’re not constantly sick with infections.
For whatever reason, some cell mutated or changed in a way that makes it live too long and start making copies of itself. Then those copies live too long and they copy and the copies copy.
Compounding that, your body sees these cells that are copying that don’t belong there and views them as foreign or not right. In a similar way to an oyster that has a little grain of sand in it, it starts making a pearl around it. The body reacts to these cells and causes inflammation and scarring to try to wall off these weird cells causing even more swelling typically in lymph nodes, although that swelling can happen outside of lymph nodes in other parts of the body as well. It’s that swelling that usually leads to people being diagnosed with this type of specific cancer.
Sam’s Hodgkin lymphoma diagnosis
Stephanie: Sam and Chelsey, you had symptoms and red flags that helped you figure out something’s not right. Sam, what was your experience?
Sam: I wasn’t feeling right. In hindsight, it’s interesting to go back and piece things together, but I felt this vague sense of tiredness. I didn’t have enough gas in my tank. I was still running 10 miles on the weekends, but coughing a lot.
I didn’t have the steam for my usual level of physical exercise. I was coughing a lot, particularly at night. There were a lot of California wildfires at that time so I thought it was the air.
And, of course, I’m tired. Every parent during the pandemic is tired, especially with our kids doing homeschooling. It’s a whole different world. Then being a doctor at that time was very hard.
But then I got a rock-hard lump that appeared above my collarbone. It was painless and rapidly growing. As a doctor, I knew that I had cancer.
I got a scan and mine said something about possibly metastatic lung cancer or breast cancer. They weren’t sure so I needed to get a tissue biopsy. A little bit of time transpired between noticing the symptoms and getting the tissue biopsied.
Once that came back as Hodgkin’s lymphoma, I knew what the path ahead would be like. Around the time I was diagnosed in 2021, there was this big change that started happening with immunotherapy. It was the standard of what had been happening for a really long time, which is ABVD or a combination of four drugs.
I got on the eve of my 30th birthday. Then I started ABVD.
There was a question about my staging, whether I was stage 2AE or stage 4 because there was some lung infiltration. Long story short, that meant that I was going to get six months of ABVD.
After a month or two, the coughing went away and I started feeling better. My cough came back at about month 2 to 3. We thought it was the bleomycin. I’m a runner and that’s very, very bad. We ended up dropping the bleomycin and I continued on AVD alone for the remaining four months.
Coincidentally, there had been a trial around that time to say that that’s okay, the de-intensification of therapy. Dropping the bleomycin became a standard thing, if people got a scan after a couple of months that showed that the body’s responding.
My PET scan after two months looked really good. My cancer was responding and I was having that cough. We dropped the bleo and then I did four more months of AVD and boy, was it hard. It was really, really hard.
I had a lot of side effects. I struggled a lot and I struggled with that feeling. I hear people say about Hodgkin’s that this is the good one. It certainly didn’t feel that way. It felt really hard.
Stephanie: I hate when I hear whenever people say you got the good one.
Chelsey’s Hodgkin’s lymphoma diagnosis
Stephanie: Chelsey, how about you? What were the first symptoms for you or the red flags?
Chelsey: I was first diagnosed with Hodgkin’s in 2018 when I was 28. I was working a lot of hours so I was very tired, but I didn’t think much of it.
I had weight loss. I was trying, but it never worked before and then I suddenly started losing weight. I had shortness of breath and, at one point, I almost crashed my car because I had a vertigo episode. I ended up going to the doctor and they told me it was just stress.
The only reason I got diagnosed with Hodgkin’s was I eventually had a lump come up on the left side of the base of my neck. I went to urgent care. They told me that I just needed antibiotics, but there was something in their eyes that told me that wasn’t all I needed.
Eventually, my family forced me to go to the ER and we got a full biopsy done. I was in the hospital for the first time. Long story short, I had stage 2 Hodgkin’s and I also had ABVD. About halfway through, we had to drop below because I had toxicity.
I was re-diagnosed with Hodgkin’s in 2019 as well.
Dr. Matasar: First, hearing Chelsey, how you knew better, listened to your body, didn’t take no for an answer, and saw something that made more sense to you. The importance of that just can’t be overstated.
I meet so many patients who say, “Well, they told me not to worry,” or “My doctor told me to come back in six months and I was getting worse and worse, but I was told not to worry. I was told it was something else.” Congratulations on knowing better and I hope that people take heed and learn from the example that you set.
Chelsey: Especially when you’re young. It’s hard sometimes to speak up. But even if you’re young, you know your body.
Dr. Matasar: Maybe even especially if you’re young. We all know that old doctors sometimes don’t have the best reputation for listening to young people, trusting them, or taking them as seriously as they ought to be taken. Doubly so because of your youth at the time.
Changes in the treatment of Hodgkin lymphoma
Stephanie: Dr. Matasar, the treatment landscape in Hodgkin lymphoma has been changing quickly. Can you share about the evolution that’s been going on?
Dr. Matasar: Back in the ’80s, ’90s, and 2000s, clinical trials to try to make Hodgkin lymphoma better were all about intensification. How can I ratchet the treatment up to make it even stronger, even more toxic, but even stronger against lymphoma?
We were climbing that mountain of pushing to see just how much chemo we could cram into someone’s gullet in pursuit of a cure. All we had was chemo so what you want to do is more.
We’ve come over the other side of the mountain now and we’re in a very different place. As a discipline and lymphoma experts in the field, we’ve moved away from intensification.
We’re into de-intensification, personalization, and leveraging treatments other than chemotherapy as we try to help both maximize the chances of cure and minimize the short- and long-term risks of our treatment.
We’ve gone away from uniformly using ABVD, which remains a very good treatment and a very commonly used treatment.
More often, we’re now using other adjunctive treatments like brentuximab vedotin or ADCETRIS in lieu of bleomycin, which both Chelsey and Sam talked about potentially injuring their lungs, leading to cough or shortness of breath.
We’re finding ways to cure more people and, at the same time, cause less harm along the way and that’s really where the future of Hodgkin’s lymphoma is going to take us. We try to make even more progress on this mission towards more effective and less toxic treatments.
Stephanie: I love that that’s the trend and that we can continue going down that path.
First-line treatment for Hodgkin lymphoma
Stephanie: We’ll talk about some of these newer promising treatments and new directions, but can you tell us a little bit more about the standard first-line treatment for Hodgkin lymphoma?
Dr. Matasar: As oncologists, when we meet a Hodgkin’s patient for the first time and we work through their treatment choices, we think through those choices together with our patients and their families.
We split people into different categories as we understand the risk of their disease and the options that are presented because of that.
One first way to try to put people into categories to help them think through their choices together is based on stage. We talk about early-stage Hodgkin lymphoma and advanced-stage Hodgkin lymphoma.
People will often ask, “What’s my numerical stage? Is it stage 1, stage 2, stage 3, stage 4?” We heard from Sam that sometimes, it’s not even clear to us as oncologists exactly what the stage is. Is it a stage 2E or is it a stage 4?
The staging exercise, which sometimes feels very black and white, can have shades of gray. We do our best to try to put people into risk categories informed by their stage and then think through the treatments that we know are best for that stage of illness.
Early-stage Hodgkin lymphoma treatment
Dr. Matasar: For the vast majority of people with early-stage Hodgkin lymphoma, the standard of care remains to be ABVD. It’s been around longer than I’ve been a doctor. It’s an oldie but a goodie and it still will cure the vast majority of patients.
Sometimes we think about radiation therapy as part of that treatment and sometimes not. However, ABVD for early-stage Hodgkin lymphoma remains the standard of care.
Advanced-stage Hodgkin lymphoma treatment
Dr. Matasar: For most patients with advanced-stage Hodgkin lymphoma, we’ve moved away from ABVD. This is based on really powerful clinical research comparing ABVD to a program where the bleomycin was swapped out in lieu of this newer immunotherapy called brentuximab vedotin, which is a type of drug called an antibody drug conjugate.
An antibody is a protein that binds onto the surface of a cell and stapled to that is a toxin so this conglomerate attaches onto the Hodgkin cell, the Hodgkin cell absorbs it, and then the toxin is released inside the cell — like a Trojan horse sneaking into the city and releasing the soldiers inside.
This newer program where bleomycin was swapped out for brentuximab — so BV-AVD instead of ABVD — not only got more people into remission and kept them in remission longer, but actually led to a higher cure rate, people living longer, and everything good that we as doctors want for our patients.
Because of this important research, we really now use brentuximab plus AVD as our traditional standard of care treatment for patients with advanced-stage Hodgkin lymphoma in pursuit of a cure.
Role of radiation in Hodgkin lymphoma treatment
Stephanie: You mentioned radiation therapy as well and that has been its own sort of beast, if you will. There are a lot of considerations about long-term side effects and where the mass is located. What role do you think radiation therapy has been playing? What are your thoughts about whether it should still be used and in what situation?
Dr. Matasar: It’s a great question and you’re right, it is sort of its own beast. Back in the ’80s and early ’90s, almost everybody with early-stage Hodgkin lymphoma got radiation treatment as part of their care.
We knew then and now that using radiation therapy would cure a few more patients than not, but that slight improvement in cure rate never translated to people living longer.
It’s a trade-off. You may do a little bit better with Hodgkin lymphoma in some situations but you’re paying a steep price oftentimes in terms of long-term effects, late effects, and risks of health problems later in life.
That includes radiation therapy putting patients at a higher risk for other cancers later in their life, particularly younger women who need radiation therapy to the chest. If that radiation touches the chest wall and breast tissue, particularly for women under the age of 35 or especially under 30, it really does heighten the risk of breast cancer later in life.
Your heart also doesn’t like radiation therapy much more than the rest of you. We know that radiation therapy to the heart can lead to a higher risk of heart disease later in life.
There are all these consequences of the decisions that we make together in our pursuit of a cure. Because we know so well about these late effects of radiation therapy, increasingly, we try to be ultra choosy with whom we use radiation therapy.
We really restrict its use for what we think we really need to get a cure. For most patients, we can cure them just fine without using radiation therapy and putting people at risk for health problems later in life because of that treatment.
Use radiation therapy if you need to; it’s a great treatment. It can be life-saving when you need it but don’t use it willy-nilly.
Side effects of Hodgkin lymphoma treatment
Chelsey’s side effects of bleomycin
Stephanie: Chelsey, you experienced the toxicity and side effects of bleomycin. Can you talk to us about what that reaction was and what you did about it?
Chelsey: I had my fifth chemo right before New Year. I had chemo four other times, but I went home and spiked a really high fever all of a sudden. As a cancer patient, if it’s 100.4°F, you have to go. Mine was 102 and my husband said, “We’re getting in the car.” I was very lethargic and just not feeling well.
We ended up in the ER and they told me I was septic. Of course, my mom heard that as well. She was watching my daughter and she’s freaking out because it’s not good when you’re septic. They put me in the ICU and I was in the ICU for at least two days.
The symptoms dissipated after a few hours. I was feeling better, not 100%, but they started giving me antibiotics around the clock.
I had a feeling that it had something to do with bleomycin. I’ve researched what can happen with bleomycin because I have asthma and that was something I had to consider going into the treatments.
Everything I was seeing was adding up to it being toxicity, but there was nobody at that hospital who really treated cancer patients. They came in and out, but they weren’t there all the time.
It took about four days for my oncologist to actually come and see me. At that time, I pretty much diagnosed myself with bleomycin toxicity. I also stopped accepting the antibiotics because it wasn’t an infection. It was a reaction to this drug and it was confirmed later on.
I also had a pulmonary function test and I had a 30-point drop or something. I’m happy to report that I have regained a lot of my pulmonary function now many years out. For a lot of people, I know that’s something scary when you do go through this.
This was a local hospital and they had never seen this before. The doctor was saying, “You need to advocate for yourself, especially when you’re in a situation where you know your cancer probably better than the people that are there. It’s not your regular care team.” I had to speak up for myself and it was hard, but I did it.
Stephanie: I really appreciate that you spelled that out. Dr. Matasar talked about it earlier, too, and I know Sam’s a huge proponent of that as well.
You said it was hard and I think we’ve all experienced that as patients where you want to advocate for yourself. You hear it from other people. It can be a little bit difficult to speak up. Do you have any other tips for people who are on the fence about it, who aren’t sure if they’re supposed to speak up?
Chelsey: I wasn’t a great advocate for myself when I first got sick. The oncologist I ended up with was the one who was just walking by when I was in the ER initially and I thought it was fate. It wasn’t. Immediately upon meeting him in his office, he told me it was the good cancer.
I was like a unicorn of the Hodgkin’s. Nothing went how it was supposed to go. It wasn’t good at all. Sometimes I was quiet about it. I didn’t know what to do because I always look things up and he would call me Dr. Google but not in a nice way. I was scared to bring up a lot of things.
If you’re young and you’re scared or sometimes it’s when you’re female and you have a male doctor, it can be intimidating. Bring your family with you or somebody else you trust and have them speak up for you if you are scared to do it. I know a lot of people do that and it’s helpful.
When you’re inside that room, your mind goes fuzzy and you don’t even know what’s going on half the time. It can be the difference between having a good result or ending up pretty much needing a transplant like I did. I truly believe that if I had been a better advocate, I might have not needed one.
Stephanie: Thank you for going into all that. I can completely attest to blanking out in the doctor’s office and not hearing the same thing that my family heard. I appreciate that tip about bringing people in if you can.
Sam’s side effects
Stephanie: In terms of side effects, you worked with your doctor to manage them. Can you share anything that was helpful there?
Sam: I had a lot of side effects. I had a lot of nausea and vomiting, yet I gained a ton of weight during AVD from all the steroids and I was developing a lot of problems from that. My glucose and liver enzymes were increasing. I had inflammation in various organs.
In hindsight, I think that was also making my neuropathy really bad because once my glucose got better and I lost weight, my pain and my nerves got better, too, despite the fact that I was getting more medicine that theoretically should have been impacting the nerves.
All your systems can be impacted. Taking care of the whole body is really important. I talked to my doctor about diet, lifestyle, and supportive therapy. What are some complementary therapies that I could try while getting my traditional medicines?
I also took a lot of medicine at the time. I needed pain medicine. I needed nausea medicine. I needed all the tools in the toolbox to cope with living day-to-day because it was just really hard.
The medicines caused a lot of mood and neuropsychiatric side effects. I had problems thinking. At some points, I had problems driving or following a list of simple items and that was really tough for me. That was a really far fall in terms of functioning, going from being a doctor to having trouble shopping a grocery list of five items so that was pretty devastating.
When I found out that I could do brentuximab as therapy leading up to the transplant, that gave me a huge quality of life back. As those other drugs cleared out and my body healed, I could think again and started feeling myself in here again. I’m not gone, I’m still there, and that mattered so much to me. It was a huge gift.
Stephanie: I really appreciate you bringing that to life because sometimes, we feel lost in that fog and how important it is to get any semblance of that quality of life back of ourselves. Your story shares that so powerfully.
Immunotherapy for Hodgkin lymphoma
Stephanie: We’re going to shift to combination therapy for the front line. Patient Matthew S. asks, “How successful have immunotherapy trials been in regard to Hodgkin lymphoma?” Dr. Matasar, can you explain the idea behind combination drug approaches and for whom would this benefit?
Dr. Matasar: We’ve talked about the progress that we’ve made with swapping brentuximab vedotin for bleomycin.
Matthew’s question about immunotherapy is a really valuable one. This idea of immunotherapy has really been a revolution in a lot of different forms of cancer, but nowhere has it been more impactful than in Hodgkin lymphoma.
When we talk about immunotherapy in Hodgkin lymphoma, what we’re generally speaking about is a class of medicines called checkpoint inhibitors.
One of the ways that these Hodgkin’s cells survive in our body is they are able to effectively shield themselves from our normal healthy immune system cells. They put up these barricades and ways of shielding themselves or hiding or preventing our immune system from doing the trash.
There are two checkpoint inhibitors that are approved for Hodgkin lymphoma, which are very similar medicines truthfully: one called nivolumab and one called pembrolizumab — we’ll call them nivo and pembro for short. These medicines are able to strip those shields off of the Hodgkin lymphoma cells and allow your immune system to see what it was otherwise blind to.
The treatment itself does nothing to the cancer cells. It does not kill a single cell all by itself. What it does is re-enable your own body’s immune system to do the work. It’s a game-changer.
When we use these medicines by themselves and use it as a treatment for a patient who’s been failed by many different prior chemotherapies, they’re able to put people into remission more than half the time.
They’re actually able to cure some patients. Despite chemotherapy having failed again and again and again, this medicine is able to eradicate the lymphoma. It goes away and never comes back. That’s amazingly powerful to be able to say and it’s an amazingly powerful treatment modality for patients with Hodgkin lymphoma.
The more we learn about how good these medicines work, the more we want to use them for more patients to try to help cure more people. It went from being a last-ditch effort after everything else has failed to be part of the treatment when it comes back. Using it in that situation was able to help more patients.
We’re now seeing the initial results of our first trials of using it as part of the first treatment. Instead of ABVD or brentuximab plus AVD, we’re combining nivo or pembro with chemotherapy like AVD and we’re starting to see very promising early results.
BV-AVD vs. ABVD as first-line treatment
Stephanie: How much is brentuximab and AVD being used? Is it standard of care or does it just vary depending on the hospital system or the healthcare provider versus going to ABVD first-line?
Dr. Matasar: It’s a little bit hard. Everybody’s situation is a little bit different. There are standards of care, which means that all things being equal, it’s sort of a one-size-fits-all approach.
Medicine is never that clean or that easy. There are times when ABVD would be a standard for early-stage disease, but we have to use brentuximab instead of bleo. There are people with advanced-stage disease who even if BV-AVD would be the standard, I still want to use ABVD for this individual patient.
This happens because doctors listen to our patients and we take into account their personal priorities, preferences, and individual risk profile. All of these medicines have their own pros and cons and their own risks and rewards.
A treatment program is best when it’s personalized and done in the context of a doctor and a patient having meaningful and valuable conversations about what matters to that person. Is it just a cure regardless of side effects? Is it being able to play the guitar and run? What is it about the treatment that we need to take into account as we map a path toward cure?
Stephanie: Any idea of when we could see the FDA approval of checkpoint inhibitors in the front line?
Dr. Matasar: Certainly not anytime soon. The first results that we had were read out at major conferences. This is still very early data.
When it comes to Hodgkin lymphoma, doctors were very conservative. We don’t want to mess this up. We know that the stakes are high and that many people will be cured with traditional treatments. We don’t want to change gears until we’re really confident that we’re not hurting people in the process.
The initial safety readout of this combination using the checkpoint inhibitors in the first treatment looked better than any of us expected. There are a lot fewer immune-related side effects than we’re accustomed to seeing when using these immunotherapies. They do have their own immune-related side effects.
We want to see the data mature, as we say. We want to see how people do over time and make sure that there are no dangerous signals about increased late effects or late side effects happening as we gain more experience with this treatment approach.
Long story short, we don’t want to change anytime immediately soon. We’re probably looking a couple of years down the road.
Treatment for relapsed/refractory Hodgkin lymphoma
Stephanie: Dr. Matasar, how have the standard of care treatments for relapsed/refractory Hodgkin lymphoma patients been changing?
Dr. Matasar: Historically, when chemo failed, we would use other chemotherapy programs that use different chemotherapy medicines than the first cocktail. The most commonly used in America historically was a program called ICE. Different than ABVD because they’re different medicines, but still chemo.
We’ve moved away from ICE being the only standard of care and we’re using more of those other medicines — brentuximab, the checkpoint inhibitors. You can use one of them all by itself or in combination.
It’s informed, of course, by what we did the first time. If patients get brentuximab as part of their first treatment, we’re not going to rush into doing it a second time and want to do something a little different. Maybe we would use checkpoint inhibitors alone or combined with some milder chemotherapy programs.
This is the art of medicine. Trying to pick amongst a number of very effective choices and determining how to leverage those medicines and combine them to achieve our patient’s goals. Always with this view of maximizing the good and minimizing the bad.
Sam’s Hodgkin lymphoma relapse
Stephanie: Sam, you relapsed a month after finishing your first-line treatment. I can’t even imagine what a gut punch it was to go through all that and then find out that news. What was that conversation like about where you were going to go next?
Dr. Siegel: Gut punch is a really great way to put it because it was. I finished six months of ABVD, which then went down to AVD. Despite a scan saying that I had no evidence of disease, I felt pretty awful.
At that point, I wasn’t sure. Am I just feeling awful because this is just what a body feels like after you’ve had six months of this poison? Something in my gut was telling me that something wasn’t right yet, but my scan was clean so I just focused on recovery for a little bit.
Within a month, I started having symptoms that were eerily reminiscent of my initial symptoms — a wheeze only in the left upper part of my chest and a little pea-sized lump. That time around, I thought, Okay, I think I’m pretty clear what’s happening here.
I got a scan, which led to some biopsies and a diagnosis of a relapse. I’m already researching on Google the next treatment regimen that I’m going to have to go through. The whole while, I’m preparing myself that I’m going to have to go through ICE.
I’m thinking for sure I’m going to have to go through something called ICE, DHAP, or one of these other regimens that have been used longer term for relapsed/refractory Hodgkin’s or salvage therapy.
When the relapse was confirmed, my doctor said, “There’s this drug now, a targeted therapy called brentuximab. Instead of doing ICE, would you be open to trying that alone for a couple of months and then repeating a PET scan to see where we’re at?”
There was a pretty decent chance that if the brentuximab didn’t get me into remission before the transplant, I would have to get ICE, a multi-drug, more traditional chemo. I was willing to take that chance because I felt so beaten up by having to get all those months of traditional cytotoxic chemotherapy and all the side effects.
The decision made sense to me at that time whereas maybe other people may have been, “No, I want to hit it hard and do that right away.” For me, I was going to take the least amount of poison possible to get me into remission before transplant.
It worked. I got a strong remission before my transplant. I went on to get the bone marrow transplant then I took brentuximab. I did almost a year of post-transplant consolidation treatment.
Because those studies and the data were just coming through, my doctor said, “You know, this is kind of becoming a thing now based on the research and this seems like it might really fit you based on how bad you’re feeling,” so that was perfect for me. I was so grateful.
Stephanie: The timing matters, right? It just happened.
Stem cell transplant
Dr. Matasar: The first thing to say is that there are two types of transplants. What we’re talking about so far is a treatment called autologous, or from yourself, stem cell transplant.
To call it a transplant is actually wrong. There’s no transplantation going on; it’s just a word that we use. This treatment that you’re hearing from Chelsey and Sam is basically just a trick. It’s a way of letting us give a round of super strong treatment.
With regular strength treatment like ABVD or ICE, we give those treatments and then let people recover. Sometimes if the chemo is stronger, you might need to boost the recovery.
Here, we’re talking about a single course of treatment, usually six days, that is so strong that if I gave it to my patient, gave them a hug, and said, “I’ll see you later,” I wouldn’t see you later. It’s too strong.
We need a very powerful antidote for such a powerful treatment. The antidote that we use is actually a person’s own stem cells, these special Adam and Eve progenitor cells that live in our bodies and let us heal.
We filter the blood ahead of time with a process like a mini dialysis where we filter out a few of those special stem cells. We put them in the freezer, give people six days of chemotherapy, thaw out those cells, and give them back as an antidote.
The course of the six days of treatment and the stem cell re-infusion as a little mini transfusion, that’s the antidote. That is we are calling a transplant.
That’s different than an allogeneic or donor stem cell transplant where there really is transplantation going on. Your immune system is being put to sleep and a new immune system from a sibling or a stranger is put into your body to give you a new immune system. We then task that new immune system with attacking your cancer. For us, that’s a very different ball of yarn.
Chelsey’s Hodgkin lymphoma relapse
Stephanie: Patient Caitlin M. asks, “For relapsed/refractory patients, are there other options aside from chemo to get into remission before a stem cell transplant?”
Chelsey, you went through immunotherapy, but unlike Caitlin, you didn’t respond. After three cycles, your PET scan showed disease progression. I can’t imagine what that was like.
It would be great to understand more about your experience in terms of the conversation you were having with your doctor ahead of the transplant. Are there questions you wish you’d ask your doctor?
Chelsey: I relapsed in the latter part of 2019. I switched to the Mayo Clinic for my care. I had been researching through the relapsed/refractory Hodgkin’s groups on Facebook. I saw nivolumab, brentuximab, and all of those things, and they were a lot less harsh.
I asked about it when I went there and it was a newer thing at that time. My oncologist said, “Yeah, we can give it a try. It’s had really good results.”
Sam mentioned that the side effects of ICE and the side effects of brentuximab are a night-and-day difference. I don’t think anybody wouldn’t want to try the less harsh one leading up to the transplant.
I had three of those treatments, had a scan, and my cancer progressed. My oncologist was honestly shocked as well that it didn’t respond whatsoever and that made me actually ineligible for maintenance, like what Sam had.
We switched to ICE. ICE was the first chemo where I had to be inpatient for three days every time. It was intense.
When your hair falls out on ABVD, for the most part, it’s slowly coming out. With ICE, it was clumps of hair coming out. I was very sick. It was very, very harsh chemo.
Chemotherapy & immunotherapy pre-transplant
Stephanie: Dr. Matasar, for relapsed/refractory patients, can you explain this combination of chemo and immunotherapy in the context of a transplant, hopefully, what this might lead to?
Dr. Matasar: Up until now, the goal for patients who have their disease come back despite good first treatment is to get their disease into remission and then into a round of high-dose therapy or autologous transplant in an attempt to maximize the chances of cure.
Can we cure people reliably and consistently without a transplant by leveraging these immunotherapies either alone or in combination with chemotherapy? This remains a clinical trial-type question; this is not a DIY thing.
My hope for the future is to use these treatments, particularly immunotherapies, to really limit our need to take people through the rigors of high-dose chemotherapy, cure more people, and cause fewer problems.
Determining sequence of treatment
Stephanie: If the ABVD didn’t work, there’s a relapsed/refractory situation. Maybe there’s some radiation that’s been involved. You have studies about brentuximab, nivolumab, or pembro alone or in combination. How are doctors having that conversation about the right thing to do next? What is the most promising next course of option?
Dr. Matasar: This is the art standing next to the science. There is some artistry to what we try to do to figure out the right lid for each pot and how to help a patient navigate the course of their illness.
There is no one-size-fits-all anymore. It used to be ICE; that’s all we did. It worked well and it was awful. Now we have a range of choices.
You have to sit with a patient and think through it. How much disease is there that I’m trying to shrink away? How quickly is it growing? How do you feel? How sick is it making you? How quickly do we need to get you better? What was your first treatment? How well did it work? How badly did it not work?
Take all of these factors, try to cram it into your doctorly brain, and try to give some reasonable recommendations. Sometimes you’re going to be as gentle as brentuximab all by itself. Sometimes you’ll want to give more chemo. Sometimes you want to give checkpoint inhibitors alone or with BV. Sometimes you want to do checkpoint inhibitors plus chemo. These are all reasonable courses.
Ideally, what you’re doing as a doctor is working with your patient as a person, as an individual, and charting a course that makes sense for them, for their illness, and for their life.
Stephanie: It’s a really thoughtful response. Are some of the considerations the treatments you had before, how successful they were, what’s already been done… What about things like age or comorbidities or those kinds of factors?
Dr. Matasar: We try not to be ageist, but, truthfully, taking care of a 30-year-old is different than taking care of a 90-year-old and to not recognize that would be silly.
That being said, it’s always a matter of individualization — understanding an individual person’s goals, their preferences, what risks they’re willing to take, what matters to them, and then trying to figure out the best treatment for that person, given everything you know about them, about their value system, and about their goals of care.
Stephanie: Wonderful. I wish every doctor thought like you.
Long-term post-treatment side effects
Stephanie: We’ve talked a little bit about long-term or late-term side effects. Patient Ariadne J. asks, “What is known about long-term post-treatment side effects?”
Sam: I love this question because now I can plug survivorship, which is basically my newfound life passion as a patient-doctor. Cancer survivorship, even though a lot of people don’t necessarily identify with the word survivor, is an important thing to keep your finger on the pulse.
Survivorship applies to this area of medicine that’s changing and evolving. It’s anybody living after a cancer diagnosis and that could be during treatment if you’re on long-term treatment and that could be after your treatment is over.
There are a lot of ways that the cancer experience, even if the treatment goes perfectly, makes you question your life when you’re in your 30s and have your fertility changed. There are all these things that get impacted once you hear that big C word.
Survivorship is an evolving area in medicine that is trying to address all of that. For me, there was a lot. There was identity. There was this existential crisis. Death and dying and making sense of all of that.
The steroids and the other medicine that I had to take impacted my thinking and my emotions. I’m usually a pretty balanced, even person so that was a very hard roller coaster.
There’s some cardiac stuff for some people. Adriamycin, which is part of the initial treatment, is something that we really need to be thinking about. We’re giving people this medicine that’s toxic to the heart in their 20s, 30s, and 40s. We need to talk about intensive lifestyle interventions like a healthy diet and cardiovascular exercise.
An overall program that focuses on wellness is hugely important in managing long-term side effects and integrative medicine. I’m doing an integrative medicine fellowship. I’m trying to unify everything that I learned in medical school with everything I needed as a patient to get better from cancer and cancer treatment and hoping to offer that to other people.
Those are practitioners that you could consider going to or looking up. Make sure that you communicate everything you’re trying and doing with all of the people who are in charge of your care, including yourself. You’re an important part of that conversation. Cancer survivorship is important for managing long-term side effects.
Stephanie: Chelsey, what were the long-term side effects for you? You talked about going into menopause, for instance.
Chelsey: The main thing I struggle with that is the most apparent in my everyday life is the cognitive side effects of all the different treatments. I honestly think it was from the extreme stress that I was under for so long.
I was on this job in an insurance company and I had to balance things with legal documents. I know that if I were to go back to that job, I would not be able to do it now and that’s taken me a long time to see that that’s okay. I’m still me and I just have to work with things differently.
I have fatigue a lot of times and joint pain. I often joke that inside, I’m 80 years old but on the outside, I’m in my 30s so it feels like that.
As Sam mentioned, there’s a lot of identity crisis that you have as a cancer patient. Who am I now? What happened to the old me? What can I do?
I really want to encourage people to seek community. Even just hearing from Sam, I’m sure some people will say, “Oh, other people feel like that?” That’s definitely what inspired me to start making art and connecting with others. It can truly make a difference in your long-term mental health in survivorship.
Stephanie: Thank you so much, Chelsey. You capture a lot of that in your artwork.
Stephanie: Dr. Matasar, with the newer therapies that are either just approved or in the pipeline, are we addressing some of these long-term/late-term side effects that hopefully people can avoid even years later after treatment ends?
Dr. Matasar: We are. A lot of the drive to develop these newer treatments has been informed by our understanding of late effects, cancer survivorship, and the risks that survivors of Hodgkin lymphoma treated with more traditional treatments go on to face.
We still need to follow with these newer treatments to watch for the possibility of late effects. We don’t believe that they’re going to cause as many or as severe, but part of the work of survivorship is learning from our survivors and walking that path with them as we see how their lives unfold over the years and decades to come.
The number one thing that I would encourage survivors to do is to work with your care teams on developing a survivorship care plan. It can be a paper document or a digital document. It should be something that lives with you that says what your diagnosis was, what your treatments were, and what our understanding is of what you can be doing to safeguard your health in the years to come.
That’s informed by understanding the possible long-term consequences of the treatments that you received, how doctors think you’re best served by taking care of yourself, and what we can do as doctors to prevent or reduce the risk of those problems as you go on to live your lives.
Stephanie: Wonderful. That’s a great tip. It’s great that the trend of more focus on survivorship after treatment is going to do wonders for so many people.
Shared treatment decision-making
Stephanie: We all want to be more empowered in our care to be able to ask doctors the questions, to feel that we can, and to ask ones that will be impactful.
Dr. Matasar, you talked about how the right treatment for each patient depends on different things like age, health, transplant eligibility, and goals of therapy. How can a patient find out the best treatment options for them and in what order? From a doctor’s perspective, what should that conversation be to elicit the best response for patients and their family members?
Dr. Matasar: I was really disheartened, Chelsey, to hear your story of your doctor disparagingly calling you Dr. Google. Nobody should have to deal with this stuff anymore. We’ve got to be better than that.
Everybody should be empowered to come into a conversation with a doctor as an equal partner in this process. I tell my patients, “This is about you. It’s not about me. I’ve got no ego in this. This is your mountain that you’re climbing. I’m not climbing the mountain. I’m the Sherpa. I’m dragging your bags alongside you. But this is your climb and I’m here to help.”
As patients or as caregivers, if you aren’t feeling valued and heard, then you may not have found the right fit for you in terms of your care team. Everybody should always feel free to be getting a second opinion. I Not enough people take advantage of this sometimes. They don’t want to insult their doctor. I don’t want to be that guy or that gal and I don’t want to be a pain in the ass. Be a pain in the ass.
I like nothing more than when my patients are pains in the ass and they come in with lots of questions. It means they’re doing their homework and they’re really invested. They want to learn and take advantage of whatever I’ve got up in my brain. I love nothing more and any doctor should love nothing more than a patient who’s all in on partnering with me on making this thing work.
Be vocal. Do your research if you want to. You don’t have to. You shouldn’t have to. But if you want to and if it’s something that you value, then that should be celebrated by your care team and never put down.
Chelsey: Have open discussions with your oncologist. If you do find other studies or other treatments, they should be open to answering your questions about it and not being dismissive, even if it’s not an option for you. They should be able to explain to you why or why not.
If you’re a young person about to have a transplant, even if you’re not but if you’re in childbearing age, please ask about fertility. I’m now in menopause at 33 years old.
It was explained to me and I understood. We didn’t really have time to waste to get me to transplant. That’s a conversation you should bring up not only when you’re going into a transplant but also when you first get diagnosed with cancer.
Stephanie: Thank you, Chelsey, those are very important questions. I was lucky to have first-line treatment and be okay, but I asked that question before my intensive chemotherapy. It’s great whenever we’re reminded: advocate for yourself depending on what you want your life to look like.
Finding a Hodgkin lymphoma specialist
Stephanie: Dr. Matasar, it’s clear that for relapsed/refractory patients, and if people are experiencing multiple relapses, it’s hard to find one answer from one doctor. They seek different opinions or get different responses. It’s a very personal discussion to be had.
Is there a right time to find a Hodgkin lymphoma specialist? When should that happen? How can you have that conversation?
Dr. Matasar: It’s tough because it’s very personal. My general philosophy is that people deserve to have expertise in their corner. I also recognize that people want to receive care close to home and they should be able to receive good care close to home.
In an ideal world, everybody would have an oncologist who lives close to them and everybody would have access to an expert to support the decision-making and to support the care journey. Sometimes that would be the same person.
If you lived near an expert, perfect. If you don’t have an expert in your neck of the woods and you want it, then you should have the opportunity to seek out that expert as a consultant and as a backup.
That doctor would work collaboratively with your local oncologist as a team to make sure that they’re doing everything to the best of their ability. You have the expert on standby in case things go sideways. In my mind, that’s the ideal.
If patients aren’t comfortable with what they’re hearing, even from me as an expert, go see somebody else. Hear from another set of lips. Get a fresh perspective. Maybe there will be a better fit in terms of that critical doctor-patient relationship.
Any doctor who doesn’t want you to get a second opinion doesn’t deserve to be your first opinion. Find the care that feels right to you. Trust your gut. The importance of the doctor-patient relationship is too great. It’s too critical in a relationship to settle for anything less than the best.
Stephanie: It’s so resonant and it’s powerful to hear from someone like you, Dr. Matasar. If the doctor doesn’t support you getting a second opinion, they’re not worthy of being your first opinion. I really love that.
Final takeaways
Stephanie: If there’s anything else you want to add, what would you really want people to take away from this discussion?
Dr. Matasar: I’m just bringing it back to The Patient Story. What you’re doing, what we’re doing together is where modern medicine should be. We should be building community. We should be sharing experiences and stories and supporting one another.
Every patient’s journey is unique and yet we don’t walk these paths alone. If you can find ways to build community and be supported by various communities, it makes the journey a little bit less painful.
Chelsey: He said it really well. When I went to my second doctor, I felt comfort and care that I hadn’t gotten before. Make sure that you are having a good relationship with your doctor.
A sense of community is very important because a lot of society puts a little bit of an expectation on our shoulders to always be brave, positive, and strong warriors, and not everyone feels that way.
I just want you to know that’s okay. It’s okay to authentically be yourself and talk about the hard parts of cancer, not just the smiling, ringing the bells, and all of that. There’s a lot more to cancer than that. It’s okay for you to feel the way that you feel, however that is.
Sam: Having lots of questions doesn’t make you anxious so it’s okay to have lots of questions and concerns and to look things up. It’s okay to reject that label because you’re just appropriately concerned about your life and the quality of your life.
Some doctors may be more attuned to knowing to ask about what’s really important to you in your life. Some people may be really busy that they forget that. As patients, it’s up to us to tell our doctors what’s really important to us and things that we like doing.
Share with your doctor what’s really important to you. Not only will it help them to know you as a human being, but it will help inform treatment decisions.
There were times during my treatment and I’ve heard from other patients where it felt like to want anything more than not dying was greedy. We’re beyond that now in medicine.
We are in the era of personalization, community, and individualized care within the guidelines of all the new things that are being discovered. It’s not greedy to want to keep exercising to some degree or keep doing your art or whatever defines the quality of life for you.
Tell your doctor what’s important to you. Reject the anxiety label. Let’s shift the focus from mortality to vitality in cancer. It’s so much more than just not dying. It’s the living part.
Stephanie: You both have exemplified that so much. I’m really grateful to have had you, Chelsey, and Dr. Matasar doing your work in research and helping patients and families holistically. Thank you for the work that all three of you are doing as advocates.
Special thanks again to Seagen for its support of our independent patient education content. The Patient Story retains full editorial control.
Lindsay was diagnosed with a very rare appendix cancer called LAMN or low-grade appendiceal mucinous neoplasm. She found out that she had LAMN appendix cancer after discovering an ovarian cyst.
As she was training for a marathon, she noticed that she had an increasing urge to urinate and was going to the bathroom more often.
She had a history of really bad periods, including bad cramping and terrible bloating. Multiple doctors kept telling her to go on the pill or have an IUD inserted. Before she could get an IUD, her doctor wanted her to have an ultrasound done. Results showed what looked like a complex ovarian cyst.
While undergoing surgery to remove the cyst, her gynecologic oncologist recognized that something was going on with her appendix and abdominal cavity. She was familiar with appendix cancer and immediately knew what to remove, what to biopsy, what pathology to request, and what the next steps were.
Appendix cancer is often misdiagnosed as ovarian cancer or overlooked as a gynecological issue so she was lucky that she went to a gynecologic oncologist who was familiar with it.
In addition to Lindsay’s narrative, The Patient Story offers a diverse collection of patient stories. These empowering stories provide real-life experiences, valuable insights, and perspectives on symptoms, diagnosis, and treatment options for cancer.
This interview has been edited for clarity and length. This is not medical advice. Please consult with your healthcare provider for treatment decisions.
I’m from Manhattan. I live on the Upper West Side with my dog Charlie, who I adopted in 2020.
I love running. I’m really into Peloton. I love to travel. I equally love the beach and the mountains so I can never choose between one or the other.
I love trying new foods and new restaurants. Natural wines have been a new thing that I’ve been exploring. I love listening to live music and going to concerts.
Pre-diagnosis
Initial symptoms
I’ve always had really bad periods, including bad cramping and terrible bloating. In the last few years leading up to my diagnosis, I noticed that it was getting worse.
I had been speaking to multiple doctors to try and figure out what was going on with my period and what I thought was endometriosis. They kept telling me, “Take birth control or go on an IUD,” but I wanted to get down to the root of it.
I was actually at my healthiest. I was training for my fourth marathon when all of this started. I noticed I was going to the bathroom more often. I’d drink water and 10 minutes later, I immediately had to pee. I couldn’t hold anything.
Discovering an ovarian cyst
It turned out that I had an ovarian cyst. The only reason we found out was because my gynecologist told me I needed to get an IUD so I would need to have an ultrasound to make sure that everything was all right down there. Thank goodness because, if not, we would have never found this.
I got the ultrasound and it showed what looked like a complex cyst so they wanted me to get an MRI. This was the weekend of the marathon. The marathon was on a Sunday. I had my MRI appointment on Friday morning.
The biggest thing I was worried about at the time was that the contrast they were giving me was going to mess up my nutrition and my hydration plan.
I was ready to run this marathon. I had a goal time. I wasn’t just running it to complete it so I was feeling really good.
The cyst itself wasn’t painful. It was really mostly around the time of my period that it would get really painful.
When we were in the office pre-COVID, there were days that I’d be sitting at my desk thinking I’d have to leave early because of such bad period cramps. But it was coming from the smaller-sized cyst at that time.
Nearing my surgery, I started to feel where it was and it did get uncomfortable, but it wasn’t very painful. It was more just this bloated discomfort.
Lindsay with Coach Ramon pre-marathon
Ovarian cystectomy
A couple of days after the marathon, I got a call from my doctor saying that the cyst was huge and very complex with all of these bumps. It needed to be removed then he referred me to an oncologist. I thought, I don’t need to go to an oncologist. Everyone gets cysts.
No one really thought that a cyst was going to lead to cancer because I was so “young” and feeling healthy. I ended up going to an oncologist who removed it.
Thank goodness I did because, during surgery, she recognized that something was going on with my appendix and my abdominal cavity. Because she was familiar with appendix cancer, she immediately knew what to remove, what to biopsy, what pathology to request, and what the next steps were.
Feeling that something was wrong
I’ve been running with the same running group for at least 10 years and I’m very close with the coach. During training, we did a half marathon. It was the same 4 1/2-mile loop over and over. There were two porta-potties halfway through each loop and every time I passed the same porta-potty, I had to stop and pee.
I’ve run 15 or 20 half marathons. I’ve never once had to stop to go to the bathroom. I told my coach, “My pace was great, but my time is all messed up because for the first time ever I had to stop to pee every time I hit that same bathroom.” He joked, “Finally, after all these years, you followed the right hydration plan and hydrated properly; that’s why you had to go pee.”
It turned out the cyst was the size of a grapefruit or a softball, they said. I’m very petite so it was pushing against my bladder and that’s why I was constantly having to go to the bathroom.
I knew something was up with my body. I was feeling really bloated. It wasn’t weight gain because I was in the best shape of my life at that point. I knew something was up, but I just didn’t know what it was.
Gynecological issues get downplayed. Take a pain reliever, take a leave, or take birth control but that’s not really fixing it. It’s just putting a Band-Aid on it. I got frustrated after all these years and really wanted to get to the bottom of it.
Lindsay with Coach Ramon at the team pasta dinner
What if it’s cancer?
It’s ironic because I ran the marathon with the American Cancer Society. We had a team pasta dinner the Saturday night before the marathon. At dinner, a member of the team told her story.
She said, “At this time last year, I was sitting right where you were. I was trained for the marathon, ran the marathon, then a week later, I found a lump in my breast, and it turned out to be pre-cancer.”
I got up to go to the bathroom with my mom and said, “It’s kind of freaking me out because what if that’s me? What if I have cancer?” It was the first time that I ever really thought of that.
I’ve had an abnormal amount of friends and family over the years who have had cancer. It never once crossed my mind that what I was going through could have been cancer, which was very naive of me.
Biopsy
My primary care doctor referred me to a gynecologic oncologist. I met with a couple of other doctors, but I ended up loving this woman and having the surgery with her.
They removed the cyst through laparoscopic surgery. While she was inside, she noticed that my appendix was huge and had a tumor on it and that my abdominal cavity was filled with what looked like mucus.
She kept calling it a jelly-like substance. She had a hunch of what it could be, but she didn’t really get into detail. She said, “Let’s run pathology on it then we’ll figure it out. Let’s not worry about it.”
At that point, she removed my appendix, she removed as much as she could of the funky (as she called it), jelly-like substance. The cyst was so big that it had completely taken over my ovaries so she removed the right ovary as well and ran pathology on it.
I knew something was wrong because I had an appointment the following week with her PA as a follow-up to the surgery. It wasn’t an easy surgery because it was a lot to recover from, but they do these all the time. I didn’t need to see her again. They were going to look at these four holes, tell me that I was healing fine, and move on.
The oncologist called me the night before my appointment and said, “Can we change your appointment time? I want to see you because I want to talk to you about the pathology reports.” That’s when I knew something was wrong. That’s when it hit me.
I actually called my parents separately because I thought maybe she told them something that they didn’t tell me because she spoke to them immediately while I was in recovery. I thought maybe they were trying to hide something from me so I wouldn’t worry.
I called them both and said, “What did Dr. Villella tell you? Because now she wants to talk in person and she said that it’s exactly what she thought it was going to be.”
The reason I say I’m lucky and I’m glad that I went to her is that so many oncologists don’t know what appendix cancer is. Even if you’re not an oncologist, so many people just have their appendix removed.
She recognized that this could be coming. They did biopsies for ovarian cancer and everything else, but she knew to include appendix cancer.
Diagnosis
Getting the official diagnosis
My parents came with me to the appointment. I sat on a table and she put her hand on my knee and said, “You’re going to be okay, but this is going to be a difficult, difficult couple of months for you.”
Then she explained that it is this extremely rare appendix cancer, that it’s the best of them, that there are several different kinds, and that if I was going to get diagnosed with appendix cancer, this is the least invasive.
It’s all a blur, but the biggest takeaway I got was that it cannot spread the way other cancers spread so I don’t have to worry about this becoming breast cancer or lung cancer. It’s going to be very contained.
I was going to need another major, major surgery that sounded completely barbaric and foreign to me, something I’d never heard of. I would not need systemic chemo, but it would be another type of chemo treatment.
My parents cried. I was too in shock to cry.
The doctor printed out some medical journal articles and highlighted certain things. I was so thankful that she did. She said, “I think that you are smart enough that you could read through this and will understand a little bit more about LAMN. I wouldn’t have printed this or gone this deep into it for most patients, but I think that this would be useful for you.”
She even gave me printouts of photos that they took inside and showed me where the mucin was on my organs, what it looked like, and how much was there. I understood that part of it and that’s pretty much all I left with.
Reaction to the diagnosis
It was a shock. I remember it so well. It was in December. My mom stayed over that night. We tried to watch all these Christmas specials to take our minds off of it, but it was very overwhelming.
It’s horrible enough to be told you have cancer, but to be told you have a type of cancer that you’ve never even heard of before is just next level.
No one ever expects to hear, “You have cancer,” but to hear you have a type of cancer that you didn’t even know existed is another blow. It’s like getting hit in the gut and then getting punched even harder.
There’s no research on it, which was why she gave me printouts because she said it’s going to be hard to find data to help make treatment decisions. She recommended an oncologist that she worked with before, but she encouraged me to meet with as many other specialists.
She said, “Find as many people as you can that have heard of appendix cancer. Meet with them and hear what they have to say. You don’t have to have your next surgery with me and with this other guy. You could do it wherever you’re most comfortable, but find out what you can and come back and report it to me. I want to hear what you find out because this is fascinating.”
I think she actually said something like doctors are going to want to meet with you because they’re going to be so fascinated to meet someone that has appendix cancer because they probably never have or will.
Not having much research data available
It was really, really frustrating, especially when it came to the treatment, the fertility part of treatment, and certain decisions that had to be made with my treatment plan.
It wasn’t black and white. There’s not enough research that says if you do this, you will have this outcome or if you have this treatment, you will not be able to have children.
There are just so many pieces to it. I was extremely overwhelmed, but I was in major fight-or-flight mode, which I’m sure everyone gets into when they have a diagnosis like this.
Meeting with specialists
I met with a couple of top specialists in the city. I’m so thankful that I live in a city where I have access to these doctors and that you can meet virtually so I didn’t have to take a lot of time off from work. I would hop on a call with a doctor and get back to work.
I made it a point to meet with as many doctors as I could until I could fully understand what the treatment plan was and actually retell it to someone. Until I was able to explain fully what was getting done, I was not ready to make any decisions.
I was completely overwhelmed; that was the biggest feeling. I wasn’t sad or angry. It was just overwhelming how I had so much to do and no idea where to start.
I ended up sticking with the GYN oncologist who did my surgery at Lenox Hill. There’s a GI oncologist who she works with who has done this type of procedure before. I ended up moving forward with them.
I met with two other GI specialists. One is at Sloan-Kettering. He’s done incredible research for appendix cancer and he’s very well known in the appendix cancer world, but it just was not a fit for me.
Then I met with this other doctor at Mount Sinai who is very well-experienced in HIPEC. He was recommended by a friend of mine. Her husband went to him and he was really nice and answered all these questions.
At the end of the day, I felt like there was more of a team effort with the doctors at Lenox Hill in terms of discussing the plan. Other doctors were saying, “We should remove this, we should remove that while we’re inside. We should also get rid of your cervix. I’ve removed ovaries before. Once you’ve removed that, you can remove anything in a reproductive system.” It was not what you wanted to hear.
I ended up sticking with the original team, knowing that the GYN oncologist who discovered it would be part of the surgery. She’s already been inside me. She knows what to look for. She knows what’s there. She found it so that was where I was most comfortable. I’m so glad I went with them. They were fantastic.
Treatment
Discussing the treatment plan
Across the board, they all said that I would need cytoreductive surgery (CRS) and HIPEC, which is a heated chemotherapy bath in your abdominal cavity.
With cytoreductive surgery, they cut you open from your chest plate all the way down to your pelvic bone. The first part of the surgery is to remove any visible tumors, which are in the form of mucin, that jelly-like, funky substance. They go in and scrape out as much as they can. But mucin, because of its consistency, can get stuck to certain organs.
I was told that I would have my left ovary removed for sure because it was covered in mucin. They also recommended to remove my uterus and do a full hysterectomy.
A lot of doctors also recommend doing a bowel resection because the appendix is connected to the end of your bowel. They want to make sure that they get any cells that could have left the appendix and got in your colon or bowels.
My team at Lenox Hill said, “We will do what we have to do. We don’t remove organs just to remove organs. If we cannot clean off your uterus, we have to remove your uterus. But if we can salvage it, we will salvage it for you.” The other doctors did not say that. They immediately said we should get rid of this, this, and this.
Cytoreductive surgery (CRS)
I woke up from surgery thinking I was going to have no uterus and left ovary. They were able to clean all of the mucin off and save my left ovary. It does not work though. I’m in menopause after surgery, but it’s still there.
Cytoreductive surgery involves going in and removing what they can remove and that could include organs. It’s very common to have your spleen removed. I also had a piece of my omentum removed, which is like a sheet over your abdominal cavity. I think a part of my liver was also removed and a lot of mucin.
Hyperthermic intraperitoneal chemotherapy (HIPEC)
After everything visible is removed, they temporarily close up your abdominal cavity and pump you with a very hot chemotherapy bath. They rotate your body for 90 minutes to make sure that that chemo gets into every crevice of your abdominal cavity to kill anything left.
Now, that doesn’t work for every type of appendix cancer, but for my type, it does. With certain types of appendix cancer, it’s not mucin but a different type of tumor. It’s hard and won’t break it up.
When I first heard about it, I thought it sounded very barbaric. They’re going to cut me in half and remove stuff from inside my guts with your hands and then “shaking.” A doctor I know who’s a specialist jokes and calls it shake and bake.
Find a specialist who is well-versed in this because it’s not a common treatment. It’s more common in Europe and I believe they do it for ovarian cancers. There’s a trial for HIPEC in a certain type of colon cancer. A lot of people do feel strongly for and against it, but I am a big supporter.
Recovery from surgery
Recovery was awful. Everybody warned me that it was going to be bad. But until you actually go through it, you can’t understand how bad.
My doctors and people in my support group who’ve gone through it said, “You’re not going to feel like yourself for at least 3 to 6 months. It’s going to take a full year for you to fully recover.” It’s hard to understand without going through it.
Now that I’ve gone through it, I say that to other people. New patients don’t understand it.
I was in the hospital for a week and had 52 staples across my abdomen. My mom had to move in with me for a couple of weeks because I couldn’t do anything on my own. I couldn’t shower without holding on to someone.
I would have to decide if I wanted to take a walk or shower. My energy levels were so low that I couldn’t do both. There were days that I would just walk up and down my hallway. It was too much for me to go all the way downstairs and walk to the corner and back so it was complete exhaustion, really bad pain.
First walk in the hospital
Two weeks post-op. 52 staples removed!
I lost my appetite. I lost about 10 or 15 lbs. They eventually came back, which is how I knew that I was finally starting to recover. But zero appetite and hair loss, which they didn’t warn me about. I didn’t lose all my hair, but I would say at least 50 to 75% of my hair fell out. It was really thin. It’s traumatic when that happens. I think anyone would say that.
I was depressed. I went on Zoloft (sertraline) because I got to a point where I just had no motivation to do anything. I felt like the world was passing me by. I was sleeping on my couch all day long and didn’t feel like I was getting better.
It was painful. I slept on my couch for a few weeks because it was too painful to get in and out of bed. My couch was more comfortable.
I remember being in the hospital one day, looking up at the very sterile, ugly white walls, and that was when it hit me that I was a cancer patient. I was also very hyped up on pain meds. Even though I’m not your standard cancer patient who’s getting chemo and this and that, this is real.
I can’t get out of bed without someone helping me. I cried when I came home. I’m not a crier but I was crying nonstop because I went from being super independent to needing my mom to move in with me.
About a month after the surgery, I finally went back to work. I was working from home so I was able to adjust. My birthday was in June and that was my 40th. I remember hitting this wall of depression where I was thinking, I am not where I thought I would be at 40.
I could barely walk around the block. All of my friends are out celebrating their big 40th. We should be going on trips and this and that. I was just processing that I have cancer. This is going to take a lot longer to recover from. Thank goodness I didn’t have any complications, but it still was just a painful couple of months.
My oncologist said, “I think you should see a specialist like a psych-oncologist to talk through this stuff.” I did and he was very helpful in understanding what was going on
It was also a hormonal issue as well. I was going through surgical menopause and we couldn’t get the hormones right so I think all of it was messing with my head.
I’m over a year out and it’s still not easy because I always thought that I’ll just go back to normal. But it finally has hit me: there is no normal to go back to and it’s not necessarily a bad thing.
I absolutely love my life. I feel so blessed. So many of my friends really showed up for me. I reconnected with old friends. We didn’t have any falling out; we just drifted apart. When they heard what happened to me, they said, “What’s going on? I’m going to be here for you,” and they were. It was really incredible to reconnect with these people.
I’ve found this passion to advocate for women, to listen to your body, and for rare cancers.
I still sit here over a year later thinking, This sucks. I have to worry about scans every six months. I have another surgery because I have an incisional hernia, which is a remnant from my last surgery and that’s going to set me back another 6 to 8 weeks before I can get back to things.
I’m still processing it. What is this new normal and how do I adjust to it?
Fertility preservation
I feel so strongly that every woman should freeze their eggs at a young age whether they think they want kids or not because you never know what’s going to happen. It’s not an issue of whether you want kids or not. It’s an issue of having the option to have kids down the line.
I would rather say I don’t want kids but I have the option because I froze my eggs than say this cancer took away my ability to have kids. I was lucky in the sense that I had frozen my eggs a few years prior.
I did have time. They offered that if I wanted to do another round of retrievals, I could. But after having my ovary surgery, I was still really sore and tired. I knew I had this other big surgery coming up.
I was happy with the retrieval that I had. I was lucky because I know a lot of women are not even offered that because either their bodies can’t handle the hormones or they don’t have time.
Mine wasn’t an aggressive cancer so I had the luxury of taking a couple of months to really do my research and heal from my first surgery before my second one so I could have done a retrieval, but, physically, I could not put myself through something like that.
I had to make the decision of removing my uterus as a precaution or keeping it because I might want to have a kid down the line. However, after HIPEC, there is no research that says you can or cannot have a child. Yes, people have gotten pregnant after having HIPEC, but there are also people whose uterus can’t hold a baby.
Fertility-wise, am I cutting my nose to spite my face here? Am I keeping my uterus to possibly have a kid one day? I’m not even going to be able to have a kid because HIPEC is going to ruin my uterus. Then I keep my uterus and mucin can probably grow there and the cancer could come back.
Another school of thought is maybe the uterus is protecting other things in that area so if the mucin comes back, it will grow on the uterus and can be removed.
There are different schools of thought, but it all really came down to fertility for me and that’s what I told my doctors. I said, “Obviously, I know that your goal as my doctor is to keep me healthy and safe so if you have to remove it, remove it. But my hope is that we can keep it just in case I want to have a child down the line.”
Fertility is so important. You never know what can happen so preserve your eggs earlier so that you don’t have to think. I felt like I ticked something really big off my cancer list, having had done that years prior. I know it’s expensive and it’s not easy, but I think it needs to be talked about more. I think people don’t talk about it enough.
Chance of recurrence
I try to stay as up-to-date as possible with this stuff. It’s interesting because the research is changing. I was told that there is a very high chance of recurrence, but it would be within the first five years. The doctor in a recent webinar I listened to said that new research shows a much lower chance of recurrence for my specific type.
My doctor does six-month CAT scans for the first two years and then upgrades it to annual scans. But I know people in my support group who are 25 years out and still get annual scans just to be safe because it could come back.
We don’t say cancer-free or in remission. We say no evidence of disease (NED) because, right now, I have no evidence of disease, but at a cellular level, there could be some stuff down there that we don’t know about and it could come back.
We don’t have a standard timeline where if you have five years of clear scans, then you’re considered in remission because there’s just such a lack of research out there.
Knowing the possibility of recurrence
It gives me a lot of anxiety. The second I feel bloated or have a stomach ache or something, I think, Oh my goodness, it’s back. But then I bring myself back down. It’s so slow-moving. There’s no way that I’m going to wake up with a belly filled with this stuff overnight
It’ll be a slow thing, but it’s nerve-wracking. Honestly, I try and take it a day at a time. I make as many plans as I can so that I’m staying busy and getting back to things that I love, like traveling and running.
My support group actually helps a lot. We do it on Zoom. It’s all appendix cancer patients and caregivers globally. There are people from literally everywhere.
It’s a very small community because there are so few of us. But it’s helpful to talk through anxieties with them and hear other people. Sometimes you think, Am I exaggerating? Should I not be worried about this? But then you hear other people thinking about it and then you say, “Yeah, it’s not just me. This is kind of what we have to live with right now.”
Possible origin of appendix cancer
I’ve gotten a lot of different answers. This particular kind is so slow-moving that I’ve probably had this for a very long time because of the amount of mucin they found. Based on what I’ve learned, it makes it sound like it’s been there for a while.
There was a tumor in my appendix that was secreting mucin, which then created the ovarian cyst. When they did the pathology, there were traces of appendix cancer in the ovarian cyst. The cyst grew so big that it must have been there for a while. They don’t know what it’s from.
There are some new genetic tests that I keep hearing about in my support group, but I think that’s more for pathology than actually figuring out where it came from. There’s a lot of stuff going on.
For example, there’s a 9/11 thing where if you lived or worked near the World Trade Center during a certain period of time, there’s some research that says that might have affected certain types of cancers, including appendix cancer. Hopefully, we’ll have an answer one day.
Words of advice
If you feel something is off with your body, listen to it. You know your body best.
Don’t be intimidated by doctors. If you’re not getting the answers that you need, no matter how much you like that doctor, go to another doctor. Don’t worry about offending them. Some doctors have bigger egos than others. You have to take care of yourself.
Ask as many questions. Don’t feel embarrassed about asking questions and advocating for yourself. It’s so important. Many doctors who don’t know what’s going on might not know how to tell you they don’t know what’s going on. If you’re not confident with the answers you’re getting, go elsewhere.
There are limited resources and not a lot of research, especially with rare cancers. Find support groups and smaller groups that you can reach out to. There’s minimal research, but there’s some research and those organizations are extremely helpful.
I have bad days but pre-cancer, I wake up every morning and say this Jewish prayer where you say basically thank you for giving me another day. I’m here, I have another day.
I say that every day and now it just feels different. Every day, I wake up and say, “I’ve got another day. I don’t know what my purpose is, but I’m still here. The cancer didn’t get me today.”
There’s a woman in my support group who says this all the time. It didn’t get me today. It’s not going to get me tomorrow. It might get me some time. But I really just try and take it a day at a time and do things that I love doing.
I try not to take things too seriously anymore. With work, I used to get so stressed out. I don’t let that stuff bother me anymore and I feel like I’m doing really well with work right now. I’m the happiest I’ve been at work in a while and I think it’s just this new attitude.
Helping others is also something that gives me hope. I love being able to pay it forward and seeing people who have had it a lot worse than me thriving. So many people stepped up for me during this.
It is what it is. I try not to say why me or this or that. I do sometimes, but I don’t think that gets me anywhere. I try to just have a positive attitude.
When I can’t get out of a funk, I work out. I get on the Peloton and take a really hard class or go for a run to clear my mind.
Symptoms: None; found the cancers during CAT scans for internal bleeding due to ulcers Treatment: Chemotherapy (capecitabine + temozolomide), surgery (distal pancreatectomy, to be scheduled)
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Thank you to AbbVie and Genmab for their support of our patient education program! The Patient Story retains full editorial control over all content.
This interview has been edited for clarity and length. This is not medical advice. Please consult with your healthcare provider for treatment decisions.
Stephanie Chuang, The Patient Story: Hi, everyone! This discussion is hosted by The Leukemia & Lymphoma Society and The Patient Story.
I was diagnosed with diffuse large B-cell lymphoma a few years ago and went through hundreds of hours of good, old-fashioned chemotherapy.
Thankfully, our discussion is all about other options that are happening, that have been approved, and that are in the pipeline.
We hear about clinical trials and the term itself can be so daunting. What are clinical trials? Our goal is for you to walk away from this with a much better understanding of what a clinical trial is and if it’s a good option for you or your loved one.
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We’re proud to be hosting this also with The Leukemia & Lymphoma Society, the world’s largest nonprofit health organization dedicated to funding blood cancer research and offering patient services and education. It has great resources from information specialists who help answer cancer questions to help pay for cancer care costs, including travel for CAR T-cell therapy.
I’m really excited to introduce our panelists. We’re really, really grateful to have them with us.
First off, someone I was lucky to meet at one of these big conferences. A very busy guy. I’m really excited that he’s spending the time with us. Dr. Joshua Brody, director of the Lymphoma Immunotherapy Program at the Icahn School of Medicine at Mount Sinai.
Dr. Josh Brody: Thank you so much. This is a great opportunity. It’s really nice to try to reach out to talk to people as directly as we can.
I run the Lymphoma Immunotherapy Program here at the Icahn School of Medicine at Mount Sinai in New York. At Mount Sinai, we are very lucky to be an NCI-designated cancer center where one of our missions beyond patient care is trying to get the next generation of patient care, which is part of developing clinical trials and helping to get patients access to newer, hopefully, better and safer therapies.
I was committed to becoming a cancer doctor from the time I was six years old. As that developed, the plan was for me to become a cancer doctor who develops new immunotherapies for cancer.
We’re very lucky to have the best precedent of immunotherapies helping patients with lymphomas. Even what you called the standard old chemotherapy is not that old because rituximab is an immunotherapy that is part of that old chemotherapy and when I started doing this, rituximab was not even a therapy.
Immunotherapy has a great precedent. Using the patient’s own immune system to help fight their cancer has a great precedent in lymphoma and that is part of what got me interested in lymphoma.
I was doing my training at Stanford in California and it’s a very well-known lymphoma place so it was a great opportunity to hopefully become great at what they are great at. There were a few things converging to get me to become a lymphoma doctor and now I’ve been doing it for a while.
Stephanie: Really grateful that we have people like you who’ve been in this for so long and are so dedicated to helping to figure out what is effective and also what will help with quality of life.
Crissy Kus: Thanks, Stephanie. I became a nurse about 12 years ago and started my career early, moving around the units in the hospital every 4 to 6 weeks. After a year of doing that, I was allowed to choose a home unit. I knew pretty quickly that BMT was my home, working with patients with blood cancers.
I help patients specifically with CAR T-cell therapy and lymphoma. I’ve been with The Leukemia & Lymphoma Society in the Clinical Trial Support Center. We’re a team of nurses who help patients and their families explore clinical trial options, learn about their treatment options, and navigate that whole journey — whether that’s proceeding with the standard of care and learning about those details and logistics or identifying barriers to participating in clinical trials and helping them overcome any of the barriers in the way of them participating.
Stephanie: Thank you, Crissy.
I’m also really excited to introduce our final panelist tonight, Dr. Robyn Stacy-Humphries, who has such an incredible story. Someone I’ve gotten to know and I feel very lucky to know you.
You have such an incredible perspective as someone who is a physician who got your own diagnosis not just once but going through treatment three times.
Dr. Robyn Stacy-Humphries: First of all, hats off to Dr. Brody. We’re very lucky to have him on this panel.
Like Dr. Brody, I actually wanted to be a doctor from the time I was five or six years old. I went to medical school and residency in radiology and my subspecialty is cancer-focused. I do body imaging, PET-CT, and mammography. I diagnose cancers and do biopsies.
Ironically, I was diagnosed with lymphoma in my late 40s. Very much of a shock to be on both sides of it. Both my parents passed away from cancer of different kinds and then I developed one myself.
Navigating the cancer world as both patient and physician has been unusual and that’s one of the reasons I’m thankful to be here. One of my missions is to try to give back, to try to bridge the gap between doctors and patients so that they can learn to talk to each other better. Patients can learn to advocate for themselves and doctors can perhaps understand where they’re coming from.
Stephanie: Thank you for being here and for offering a perspective that is very unique. We have two physicians on tonight who really care about the patient’s perspective.
This is a personal topic for me as well. DLBCL is all over my medical charts. I was 31 when I was diagnosed and like you, Robyn, there’s that shock. I remember trying to understand: what do these letters mean? Lots of alphabet soup coming at you when you get diagnosed with cancer of any kind.
What is DLBCL?
Stephanie: Dr. Brody, in as human terms as possible, what is diffuse large B-cell lymphoma? Are there some common first red flags or symptoms?
Dr. Brody: For Stephanie, I have to apologize for the alphabet soup. That’s a thing we do. We make up code names with lots of letters but not just to be confusing. There’s so much we’ve learned in decades that we make subsets of subsets and then we need more alphabet soup to describe all the subsets. PMBCL, DLBCL, non-GC — it’s letters on top of letters.
Anything that we can say in medicine or science, we should be able to say it in English as well. None of it’s so complicated that we shouldn’t be able to say it in English, especially when we’re talking to our patients. We have to be able to say things in a way that is understandable.
In regular English, lymphoma is a cancer of lymph cells. What are lymph cells? This is really simple. You think about breast cancer, cancer of breast cells, prostate cancer, cancer of prostate cells. It’s a healthy cell that becomes a cancer.
What are lymphocytes? Lymphocytes are a certain type of blood cell. They are specifically blood cells that may live in your lymph nodes. When you say swollen glands because you had a cold or a sore throat or something, those glands we’re talking about are usually lymph nodes.
Lymph nodes are all around your body. That’s the first tricky thing because when you think breast cancer, prostate cancer, you think, “Oh, I know where the breast is, where the prostate is,” so that makes sense. Lymphoma comes from lymphocytes, which mostly live in lymph nodes, so that could be anywhere in the body.
Common symptoms of lymphoma
Dr. Brody: The common presenting symptoms of lymphoma depend on which lymph node area had a cell become cancerous. Amongst all of those possibilities, we still say that the most common presentation is a painless, swollen lymph node literally anywhere in the body.
There are subsets of lymphomas that don’t even show up in a lymph node per se. Sometimes we talk about primary mediastinal B-cell lymphoma. There are lymph nodes there, but they’re not in the common lymph node spots. We think of the neck, the underarms, and so forth. They just show up in the middle of the chest, the mediastinum. Nonetheless, the most common presenting symptom is a painless lymph node.
Lumps you get, because you had a sore throat, are not a painless lymph node. Usually, they are painful or tender lymph nodes. If you have a tender lymph node, that’s a little less likely to be lymphoma than inflammation as a result of a cold or sore throat.
Again, not every lymph node that gets a little bit swollen needs to be emergently evaluated. Otherwise, every person would have a swollen lymph node at some time or another.
The first thing we have to pour on this is a bit of common sense. You have a lymph node that’s a bit swollen for a few days. You can watch it for a few more days and if things start to recover on their own, probably you don’t need to go and get that swollen lymph node evaluated.
Lymph nodes that are swelling, usually painless, and getting worse over weeks and weeks and weeks need to be evaluated. Those are the most common presenting symptoms of diffuse large B-cell lymphoma, which is the highest incidence type of lymphoma.
Stephanie: I really appreciate you sharing the differences between what is more commonplace versus when you need to maybe get something checked out. People do get really worried.
Robyn’s DLBCL diagnosis
Stephanie: Robyn, you went through this yourself so it’d be great to hear from you. What was it for you that was a red flag that led to the diagnosis? Share a little bit about getting that diagnosis.
Robyn: I had a swollen lymph node, but it was an unusual location in what’s called your supraclavicular region. You never have normal lymph nodes there. When you have a cold, those don’t swell up. Usually, when you have a lymph node above your collarbone, it can be a symptom of lung cancer, gastric cancer, ovarian cancer, or even breast cancer.
I felt a lymph node literally while I was watching TV. I knew it was bad. It’s never good.
My process for diagnosis went very fast. I ended up with a CT the next day, which I looked at and found out I had lymphoma. I had lymph nodes all the way up and down my neck, which you couldn’t feel. They were all very small. There were too many of them.
I actually had some lymph nodes behind my nose and something called Waldeyer’s ring. I thought I had allergies. I was congested and that actually was lymphoma.
I really had no symptoms besides that. I was working 60 hours a week as a mother of three. I ran every day. I ate well.
Everything went really fast. It was a shock. Before I knew it, I had a CT scan, a PET scan, and a port put in, and immediately started on R-CHOP, which is the standard therapy. It was initially very successful for me. It didn’t work later, but it was very successful at first.
I did six rounds of R-CHOP and as Dr. Brody said, RITUXAN (rituximab) changed everything. When I was in medical school, non-Hodgkin’s lymphoma cure rate was very low, only about 30 or 40%. Once you added Rituxan, it’s over 70% in a lot of cases.
Things have progressed in the last 10 years. Now with the immunotherapies, things are totally different.
Stephanie: What a strange experience to have essentially self-diagnosed and have to read your own scans and images, but you do that for a living. I appreciate both of you talking about just how quickly things have changed in the last 10 to 15 years and even in the last five years or so with immunotherapy.
That’s what’s exciting to talk about but also why these programs are so necessary because it is a lot. It’s great to have people who specialize in this area so that there’s a depth of knowledge about all the different options and for whom these options are best.
Standard of care for DLBCL
Stephanie: Let’s talk about the standard of care. Dr. Brody, what has been the standard first-line treatment in DLBCL? We heard Robyn talk about R-CHOP.
Dr. Brody: For the past 20 years, there’s been a bit of an evolution. R-CHOP has been the standard of care. It doesn’t mean that everyone gets R-CHOP, but most people do — certainly more than 80%, probably more than 90%.
Some people may not get R-CHOP maybe because it can be a little tough. We have patients who are in their 90s and they may get a gentler version of R-CHOP or even slight variations of that. We do have patients in their 90s who still get R-CHOP, but it’s not gentle therapy.
We sometimes give gentler versions and sometimes even more aggressive versions. There’s a therapy called R-EPOCH. It’s a tougher version of R-CHOP.
In some ways, it seems to be “better” in that it’s maybe more effective, but in a big randomized trial comparing the two, there wasn’t a clear difference. We think that R-EPOCH is just good for certain super high-risk subsets of patients so 5 or 10% of patients get R-EPOCH instead of R-CHOP.
That was a big evolution from the CHOP of the 90s and easily added a 10-plus percentage increase in the cure rate to standard therapy for patients with DLBCL so it’s a big, big deal.
I was very lucky because Rituximab came out of Stanford and Biogen Idec-Genentech back in the early 2000s so it was an exciting time for us. We now enter the Rituximab era of treating patients with DLBCL. Now we’re still seeing comparably exciting evolutions to what may be the standard going forward.
Stephanie: Amazing to have all that history and to be front and center when it was all going on; that must have been exciting.
We have a recent approval, polatuzumab or POLIVY and R-CHP, which is R-CHOP without the O for first-line treatment. How much real-world data is there? What are your thoughts about it as another option for people?
Dr. Brody: Pola-R-CHP, we say, is another option. It’s not the right answer, but it might be the right answer in the future. We’ll see.
There’s a randomized trial, POLARIX, where half of the patients got R-CHOP and half got pola-R-CHP. Pola-R-CHP was a bit more effective where 6% more people stayed in remission for the first couple of years. It’s possible that could eventually translate to an increased cure rate, but we need more time to follow those folks and see how they do.
But a 6% increase, staying in remission for two years, that’s not nothing. If you were one of those six out of 100 people, that’d be a big deal for you. The only catch is it’s not “for free” in that there are some extra side effects but not too bad. The risk of infections during the therapy was moderate, but it was also a bit increased in the pola-R-CHP. Interestingly, it was about a 6% increase on that side as well.
Maybe 6% percent more people staying in remission and maybe 6% more people getting this significant side effect. The side effect lands you in the hospital so it’s not nothing. A little bit tougher on lymphoma, but a little tougher on patients as well.
I wouldn’t say it’s the right therapy for everybody, but certainly, every lymphoma doctor is weighing each patient. It’s supposed to be personalized for each patient. This patient might do well because they’re younger, healthier, and wouldn’t have a high risk of side effects and this other patient may not do well.
A 90-year-old probably wouldn’t get pola-R-CHP because R-CHOP is already tough therapy for them. Higher-risk patients and certain subsets of high-risk patients might get the most benefit from the pola-R-CHP. Probably in the near future, maybe half of the patients are going to get pola-R-CHP and half are going to get R-CHOP.
Risk of relapse with DLBCL
Stephanie: That’s great. Thank you for sharing that and interesting to hear. We’d like to take in one of the patient questions actually. Wilson asks, “What’s the risk of relapse with DLBCL and what is the leading treatment for those who relapse?”
Dr. Brody: As Dr. Stacy-Humphries said, we cure the majority of patients. This is awesome not just compared to the 90s, but compared to the 70s when I had relatives that had this disease. We cure the majority of patients and that’s wonderful. It’s not 100% and we would like to get it there.
Today, we are curing more than 65% of patients, depending on which study you look at. A third of patients could still relapse.
These studies focus on slightly younger, slightly healthier patients. If you look at everybody, it’s probably about 65, maybe a little bit above that with “standard” 2023 therapies. We see that increasing as well over the next few years.
Relapsed/refractory DLBCL treatment
Dr. Brody: The standard of care for them depends on a few things. It depends on when they relapse. If patients relapse very quickly, within the first year, the approach is a bit different. The idea is they just got chemo and it didn’t work very well so giving something that kills cancer in a different way might be better.
In the TRANSFORM trial and the ZUMA-7 trial, early relapsers and people that didn’t get a good remission at all or refractory patients, CAR T cells were superior to the old standard.
The old standard was basically more chemo and actually a lot of chemo, which was tough therapy. Those trials both showed that CAR T was better. If you read between the lines, they were also, I would say, safer and better tolerated. That’s a rare win-win, both better and safer. We don’t get a lot of those in new cancer therapies.
With pola-R-CHP, it wasn’t really a win-win. It was maybe better, but not safer. CAR T versus the old standard of more chemo was a real win-win. For early relapses and refractory patients, CAR T is the standard of care. It doesn’t mean every patient has to get it; there’s still some individualization. But for the early relapsers, that’s been the big change over the past year and two. That was not the standard of care even a couple of years ago.
For patients whose disease relapses later, it’s even more individualized. The old standard for younger patients with late relapses was this mega dose chemo approach called autologous stem cell transplant. Autologous means you give the stem cells to yourself. We want to distinguish it from the other kind of transplant where you get stem cells from another person called an allogeneic stem cell transplant.
For people who relapse a little later and who are young and tough enough to tolerate this tough therapy, that is still the standard but that doesn’t really apply to every patient. When we did those first studies, they were for people under 60 then we started doing it for patients under 65. Now we do it for patients under 70.
It’s tough therapy for people who are around the median age of this disease, which is upper 60s and 70s. It’s standard of care, but a lot of times for those late relapsing patients, they may not be eligible for that approach. Thankfully, there’s a whole bunch of other more targeted options.
Robyn’s 1st DLBCL relapse
Stephanie: Robyn, you had a good response to R-CHOP in the beginning. As you mentioned, it didn’t stay that way. We’d love to understand more about what happened. How far along were you until you realized something was not right again?
Robyn: I was actually in complete remission for four years when my relapse happened in 2015. Again, I felt a lymph node in my neck, which I knew wasn’t supposed to be there.
As Dr. Brody said, the standard of care at that point was an autologous stem cell transplant. First, they give you salvage chemotherapy; RICE is what I received. It’s very rough chemotherapy. I also got intrathecal chemotherapy to prevent the lymphoma from going to my brain.
When I was confirmed to be in remission, they went ahead and did the stem cell transplant. They harvest stem cells and oblate your bone marrow using the strongest chemo possible then give you your marrow back.
It’s really a rescue. You have no stem cells left. They give you your marrow then you have to wait for your marrow to come back.
The biggest risk is infection. I got called septic shock and was very, very sick in the intensive care unit on medicines and pressors.
When I survived that, because my case was slightly unusual, it was decided among several institutions that I get radiation, which was really horrible. Two thumbs down, don’t recommend it, but I’m still here.
I was very, very sick. I couldn’t eat solid food. I dropped from a BMI of 22 down to about 15 or 14. It was quite difficult. I was actually trying to work. It wasn’t great, but you do what you have to do to survive. I did get great care so my doctors were fantastic. It’s just I had some complications.
Robyn’s 2nd DLBCL relapse
Robyn: I was in remission for nine months after the autologous stem cell transplant when the lymphoma came back. This time, it came back even more aggressively. Not only was it in my neck, it was underneath my armpit and in my groin.
I had no match for an allogeneic transplant — no one was even close. The doctors were very shocked. But I had already decided I did not want to go through that again.
I researched on www.ClinicalTrials.gov on my own. We started looking at other options. I heard about T-cell therapies. They talked about killer T cells and CAR T. It had been in the news in 2015 or ’16.
I started looking into that and that’s how I ended up going into a clinical trial. I’m very, very grateful I did. It was a phase 2 trial. It was definitely a leap of faith, but I just had a feeling it was the right thing for me.
I was an excellent candidate because I was in really, really good health except for the lymphoma. For a clinical trial, this is perfect because I had no comorbidities. That way the doctors could actually figure out if this was going to really work without something else that would interfere with the procedure.
Stephanie: ClinicalTrials.gov is a great resource. It is not the easiest to navigate, especially for a layperson so that is where the LLS’ Clinical Trial Support Center is so wonderful. Resources like that help people really navigate what’s out there.
What is a clinical trial?
Stephanie: When we talk about clinical trials, the research is happening to hopefully help provide more options for people.
A lot of patients have talked to me about efficacy or how successful it is at saving your life or increasing your life span and safety, which is quality of life and side effects. I want to live longer, but how is my quality of life going to look?
Dr. Brody, you’ve been involved with so many clinical trials. What is a clinical trial?
Dr. Brody: We should take Dr. Stacy-Humphries’ example of this. Robyn got that in 2016. There were no FDA-approved CAR T cells in 2016. They got approved in 2017.
Clinical trials are lifesavers. They are an opportunity to get access to a thing that is not yet FDA approved or sometimes things are FDA approved but a newer version or a better version or a combination.
In Robyn’s case, that was the only way to get access to CAR T cells, which years later, we realize are in many ways superior to autologous transplant and RICE. Not in every setting but in some settings, blatantly superior.
Clinical trials are carefully regulated opportunities to get access to medicines or combinations of medicines that are just not yet FDA-approved. This is a preview of future medicine. We don’t want to oversell it because not every clinical trial is successful.
We can’t ever do a trial unless we get all of these things blessed by many different groups, institutional ethical boards, local ethical boards, and the FDA. It is a way of getting access to things that are not yet FDA approved or not yet FDA approved in that combination or in that specific way.
Lymphoma is the fifth most common cancer in America. Pretty common but not as common as other ones like breast cancer. We have more medicines for lymphoma because we’ve been lucky to have more successful clinical trials and more rapid progress.
Researching clinical trials
Dr. Brody: The great thing about clinical trials is the tough thing about them: finding which one is where and at what time. ClinicalTrials.gov is the same tool we use, but it’s not super user-friendly. You can look at it and you might get the answer. At the very least, it might guide you to who to call.
There is no one best way to find out what is the right clinical trial for me. Even if you ask your doctor, they know about some, but it’s hard to know about every single trial in America at any given moment because they change even from month to month.
Stephanie: Crissy, how can someone look up what clinical trials are out there on their own if that’s the direction they’d like to go in?
Crissy: ClinicalTrials.gov houses every cancer clinical trial and general treatment clinical trials in the United States and some outside of the United States as well. Patients can go on there and look up information about studies pertinent to their case if they’re interested.
I will give a fair warning. In my previous job, on at least a weekly basis, I had to help hematologists in the navigation of that database. These were really bright physicians and physicians who were the primary investigator on many of these trials and they had a hard time navigating the database.
I tell patients to say don’t get down on yourself. If you looked at that database and it was overwhelming, that’s where we come in. We have a database that sits on top of ClinicalTrials.gov and has all the information that it has. It allows our nurses to update and augment that information in real-time so that it’s as updated as possible and has additional logistical information so that we can best communicate the information to patients.
Additionally, most large cancer centers or NCI-designated centers have a page on their website that has all the clinical trials that they have at their site. If a patient is specifically interested in a certain institution in the US, they can likely go to their page and look at the clinical trials that they have.
It’s generally going to link them to ClinicalTrials.gov where they can look at more information about the trial, but most institutions have really good databases on their own that talk about the clinical trials that they have at their site.
Misconceptions about clinical trials
Stephanie: Crissy, you’re on the phone a lot with patients and their family members answering questions about clinical trials. What are the top questions that you’re getting and some of the misconceptions that you’re hearing about clinical trials?
Misconception #1: Getting a placebo
Crissy: One of the biggest misconceptions that I hear from patients almost daily is that they’re worried about participating in a clinical trial because what if they get the placebo part of the trial?
In the United States, placebos are very rarely used in clinical trials. When they are used, they’re not used alone. A patient with cancer would never get just a placebo and not some type of effective treatment. The way that a placebo would be used in a clinical trial would be in addition to something that is proven to already be effective.
A really simple example that I give patients using CAR T as an example is if they wanted to study a drug in addition to CAR T that will make CAR T more effective or have less toxicity.
The way that a placebo would be used in that setting would be if you had 100 patients all getting CAR T as the standard of care and 50 of the patients get CAR T and then they get a placebo and then the other 50 patients get CAR T and a drug that’s being studied to see if it makes CAR T more effective or less toxic.
Every patient in the clinical trial is still getting the minimum standard of care. Some patients are getting something to see if it makes it more effective or potentially less toxic and the other patients are getting a placebo.
Misconception #2: Clinical trial is free
Crissy: Another big misconception about clinical trials is that everything on a clinical trial is free or that if a patient doesn’t have insurance, they can participate in a clinical trial to get a drug for free. Unfortunately, while I wish that that was true, it’s not.
There are three buckets of costs that we describe to patients.
Bucket number one is things that are free, things that the patient is not responsible for, and their insurance is not billed for and those are things that are investigational. The study drug that’s being used in a clinical trial? Not billed to insurance. Any follow-up or monitoring specific to the study drug or needed just for the clinical trial? Covered by the sponsor of the trial, not billed to insurance.
The second bucket is things that are billed to the patient’s insurance and those are things that are routine, standard of care. If someone has lymphoma, they’re going to be seen by their physician routinely. Their labs are going to be monitored. They may need to be hospitalized for treatment or symptoms. Those are all billed to insurance because even if a patient wasn’t participating in a clinical trial, they would still be having those physician visits and having their labs checked routinely.
The third bucket is things that come out of a patient’s pocket like the general copays that you would have and travel and lodging to a site. If you needed to travel far away from home to participate in a study, that would be considered out-of-pocket for a patient.
Misconception #3: Clinical trial as last resort
Crissy: The other misconception that I hear quite frequently is that patients think that they can only participate in a clinical trial if they’ve exhausted all of their treatment options and that could not be further from the truth.
There are clinical trials for patients in every step of their diagnosis — from the time that they’re first diagnosed, all the way through having failed multiple lines of prior therapy. There’s a clinical trial for most patients at every stage of their treatment.
This could be standard of care, front-line treatment for a patient with another added drug to see if it makes it more effective. It could be things like studying CAR T in the front-line setting where half of the patients get front-line treatment like R-CHOP and half of the patients get CAR T to see if CAR T is more effective in front-line treatment. Then every step beyond that, including long-term monitoring for patients who are in remission.
What is CAR T-cell therapy?
Stephanie: We’d like to delve into CAR T a little bit more. We have been getting a lot of patient questions.
Dr. Brody, can you give us a general description of CAR T-cell therapy? What is it? What does it entail?
Dr. Brody: When I describe CAR T cells to my patients, they think I’m kidding. It sounds like science fiction, but it’s a real thing. They think, “Oh, what decade? When will that be here?” It’s already here.
CAR T-cell is a combination of gene therapy and immune therapy. We take out some of a patient’s immune cells, send them somewhere where they turn them from normal healthy T cells, and insert this new gene called CAR, a chimeric antigen receptor. Now we call it CAR T cells.
They ship the CAR T cells back to you. The patients have to get some chemotherapy before the CAR T cells are reinfused. We say we give that to make room for the new cells to come in. It’s a bit of a metaphor, but it’s conceptually fair.
The gene that we put in, CAR, helps those T cells to go to your diffuse large B-cell lymphoma and eliminate it more potently than any other single therapy that we’ve ever developed. Close to some other immunotherapies now, but it’s quite amazing.
They don’t do it perfectly and they don’t do it for 100% of people, but they induce remissions in a good majority of people. Depending on the setting, they may cure 35, 40, or maybe even 50%, but we usually say about 40% of patients. Again, it depends on what’s happened to that patient before and some other things about the patient.
For Dr. Stacy-Humphries, we used to call that an incurable setting — third-line, diffuse large B-cell lymphoma — and now we’re curing 40% or more of those patients. It’s miraculous and it’s not science fiction. It’s a real thing.
Robyn’s CAR T-cell therapy experience
Stephanie: Robyn, you went through this. You’re a physician so you have, I think, more of a know-how of navigating ClinicalTrials.gov or understanding what’s in the pipeline. How did you determine that this was what you wanted to do? You mentioned it was a leap of faith. Who brought it up? What was the experience like when you actually went through CAR T?
Robyn: I decided that I want to do CAR T because I didn’t have an allogeneic match. I could have had a haploidentical transplant, which is a half-match with my son. I didn’t do well with the autologous transplant so I didn’t think I was going to do well or I may not have survived an allogeneic transplant and I may not have had a normal life. I wanted to have that quality of life.
I’d read about CAR T in some scientific articles. I just Googled this. Nobody really told me anything. I just thought this made sense because right now, the basis of cancer therapy is you either cut it, you burn it, or you poison it. Taking your own immune cells is actually much nicer. It’s lovely as compared to the other ones, even though it has side effects.
When I researched clinical trials, I didn’t have that much of an insight. I just put in my diagnosis. I found all of the trials and we emailed every single investigator.
The reason I ended up in the trial was it was the only opening that existed. We looked all over the world. I was really ready to sell the house and move wherever. We were lucky to get one about nine hours away.
Fast forward, I signed up for CAR T. I’m the second person at this huge hospital who gets the treatment. When they took my T cells out, they gave me one chemotherapy agent, bendamustine — some people get FluCy — and had the CAR T cells infused. The infusion took about five minutes. Everyone in the room clapped and just left so it was very anticlimactic.
In my case, they started working very quickly. By the time I came in, I had a lot of lymph nodes and within 24 hours, they started melting. Within seven days, all my palpable lymph nodes were gone.
I developed cytokine release syndrome. In my case, it was a fever and low blood pressure. Most people feel really lousy, like the worst case of flu ever. I was hospitalized for three days from days 5 to 8. Other than that, I was in a rental condo and stayed at the hospital for a month. I didn’t feel great, but I didn’t feel awful.
Amazingly, I was able to go back to work four weeks later, only working two days a week, which is unusual. I did extremely well and I would say that that’s relatively unusual. I know a lot of people now who had CAR T who were working two weeks after.
Dr. Brody: After the stem cell transplant, how long were you out of work? Just for comparison.
Robyn: I was out of work for three months and when I went back to work, I was not able to eat solid food. I lived off smoothies. It was terrible.
Stephanie: What a comparison. You also said you were young and otherwise healthy except for the lymphoma so all these things may factor into a response.
Pros & cons of CAR T-cell therapy
Stephanie: Patient Gil R. asks, “What are the pros and cons of CAR T-cell therapy and what is its duration?” Dr. Brody, I think you can answer this question in several ways, but I think in talking about pros and cons, people want to compare. What are the side effects? In terms of duration, how long is the therapy itself and how long is the expected duration of response?
Dr. Brody: Pros and cons, very broadly.
Pro, if you compared it to what Dr. Stacy-Humphries described as the old standard before that, the chemo and stem cell transplant, CAR T cell therapy is both more effective, especially for some patients, and gentler. Robyn was out of work for 2 to 4 weeks, whereas after the transplant she was out of work for months and only on smoothies.
When you ask doctors about efficacy, we can give you numbers. When you ask about side effects, we describe them in more nebulous, vague ways because there are so many numbers you could give.
A great metric is how long were you out of work or unable to do the things you do. If you’re retired but you garden every day, how many weeks or months until you are gardening again? Those are great examples.
Let’s acknowledge the cons as well and Robyn pointed out one. Some of the side effects and the most common and one of the scariest ones is CRS, cytokine release syndrome.
It’s basically like having an infection, but there’s no infection there. You have fevers and sometimes dangerously low blood pressure. Even though Robyn was only hospitalized for a few days, the more common thing nowadays is folks are hospitalized for sometimes a couple of weeks on average.
Sometimes, it’s a boring hospitalization, which is the best kind of hospitalization, just to watch for that side effect to see if it occurs. Sometimes folks have to go to the intensive care unit just to treat that low blood pressure and fever. That is one of the biggest side effects.
One other scary side effect is called ICANS (immune effector cell-associated neurotoxicity syndrome) but we just call it neurotoxicity or encephalopathy. It means there’s a horrible problem with the brain and this side effect manifests in many different ways like hallucinations and loss of consciousness.
These things sound very scary so I should preface it by saying there’s a very defined time course to these side effects. They almost always happen within the first couple of weeks. There are a couple of rare exceptions of it happening on the third week, but they never happen two months later. Both CRS and neurotoxicity are scary side effects. They’re time-limited for the majority of people, but they’re a serious big deal.
Some patients have a higher risk of getting those side effects, like patients who are older or less healthy and patients who have greater tumor bulk. Patients with enough lymphoma have more of those side effects than patients with just a small amount of lymphoma when they go into CAR T therapy.
This therapy has a greater track record of keeping people in remission for years and years. We have some newer therapies that are as promising, but they don’t have the years and years of track record because they’re newer therapies.
These side effects are not nothing. If you were an 80-year-old patient with a bulky tumor, these cons are a serious thing and we have to start thinking about other alternatives for those kinds of patients.
What are bispecific antibodies?
Stephanie: Dr. Brody, let’s talk about bispecific antibodies. Some have described it very simplistically as an off-the-shelf CAR T option.
Dr. Brody: This is, I would say, the most exciting and transformational thing in lymphoma and DLBCL, certainly in the last couple of years. Maybe you might argue ever because my belief is their total impact in the next few years is going to be unparalleled.
Antibodies are magical little molecules, big long proteins that bind very specifically to one thing. We make antibodies against COVID after we get a vaccine or get COVID.
A company can make antibodies against certain targets on lymphoma cells. Rituximab is an antibody against lymphoma, but it just binds lymphoma.
Some brilliant pioneers from a few groups developed bispecific antibodies. They bind to lymphoma cells and one of your T cells.
Some people use the Lady and the Tramp with one spaghetti noodle metaphor. If you haven’t seen the movie, the spaghetti noodle brings them together except instead of the kiss between the Lady and the Tramp, this is the kiss of death because the T cell kills the lymphoma cell and kills it pretty well.
In a way, it’s sort of an off-the-shelf version of a T-cell therapy, like the CAR T cell. It’s very cool that CAR T-cell therapy is individualized. I send my T cells in, they send me back my CAR T cells. Logistically and practically, it’s very difficult and it takes a lot of time.
How much time it takes is a bit variable. It could take 2 or 3 weeks to make the product, but sometimes it takes a couple more weeks just to plan the whole thing out. Sometimes it takes more weeks just to get to see the doctor to plan that so there can be real delays there.
Whereas with these bispecific antibodies, you just inject it right into the patient. No delay there so they are in some ways a lot easier because they are off the shelf. You don’t have to make it for each person.
Efficacy of bispecific antibodies
Dr. Brody: The efficacy has been overall awesome. They are not perfect, but they are putting a big proportion of patients into complete remission and some of those patients are staying in complete remission for years so that’s fantastic. Maybe the numbers are a little less amazing than CAR T cells in efficacy, but they’re also a lot easier.
Side effects of bispecific antibodies
Dr. Brody: Overall, possible side effects are a lot gentler. Those CAR T cells were causing CRS or neurotoxicity in maybe 10 and 20% of patients, whereas these bispecifics caused bad side effects in maybe 1 to 3% of patients.
It opens up a lot of opportunities. A patient that was not eligible for CAR T, because it would have been too scary, could be eligible for bispecifics.
Access to bispecific antibodies
Dr. Brody: CAR T cells are available at a limited number of centers. That can be tricky because you’re not just going there for one day to get the therapy; you’re there for a while, as Robyn was saying.
Bispecifics were just FDA-approved so they may not be available everywhere today. We don’t want to oversell that, but they can be available everywhere and they’re now available at most major centers.
Eligibility for bispecific antibodies
Stephanie: Epcoritamab or EPINLY and glofitamab or COLUMVI are newer and newly approved. They are great as new options to share with patients.
How would someone know what questions to ask whether they’re a good candidate for bispecifics or which option to go for? As we know, it was approved for later lines of treatment and even after approval, they’re still in clinical trial because everyone likes to try and move it up earlier in lines of treatment.
Dr. Brody: Very exciting. These two medicines were just FDA-approved. There was one other that was approved for follicular lymphoma at the end of 2022, which was mosunetuzumab.
There are a lot of these getting studied and approved now. There will be several more approved as sort of a plan C, third-line therapy when Plan A or Plan B don’t work well enough.
If a patient asks, “Is this the right therapy for me?” Tough and detailed question, but the first thing you want to make sure of is that the doctor that you are working with is familiar with the option.
Even though they are available anywhere, there are a lot of new cancer medicines coming out every month and year. This is very tough, especially for community oncologists who take care of every kind of cancer. It would be impossible for them to be super familiar with every new medicine for every type of cancer.
It is not that every patient with lymphoma needs to be seen by a lymphoma specialist, but once Plans A and B have failed, this is not a great situation.
Let’s just be simple about it. Bluntly, it is worth the schlep to go and see a specialist who does nothing but take care of lymphoma patients. They are familiar with these medicines and it would be hard for most community oncologists to be familiar with them.
Community oncologists will be familiar with these medicines over the next couple of years, but they may not be today and they may not have access to administer them because there can be complexity in getting these medicines started in a new practice.
You need to make sure that you’re speaking with a doctor that has comfort around this. A lot of times, community oncologists are vaguely aware of it so they’ll call their local buddy who’s a specialist and say, “You tell me. Should I send this patient to you?”
But as you heard from Robyn, sometimes patients end up advocating for themselves. I’d like to say that there’s a better way, but that does happen. The function of The Patient Story, the LLS, and other patient advocacy groups is to try to be an intermediary so patients don’t have to advocate for themselves.
But if a patient is asking if bispecific antibodies are right for them, there’s a good chance the answer is yes so you should at least be talking to someone that has experience with these.
Choosing among treatment options
Stephanie: When there are different options and they’re new, how do you make a decision on which option to put a patient on?
Dr. Brody: Although these medicines are newly approved, we’ve been using these medicines for three years, give or take, so we have a lot of familiarity with them. Maybe we didn’t predict this, but epcoritamab and glofitamab are more similar than different.
For example, the complete remission rate for both of them was exactly 39%. What are the chances of that? The high-grade bad side effects were about 3% with both of those medicines.
There are a few nuanced differences. Epcoritamab is given subcutaneously whereas the glofitamab is given intravenously. It’s a little difference, not a huge, big deal.
The timing is a bit different. Epcoritamab has more visits and they might go on for longer. Glofitamab has fewer visits and is designed to be stopped after about nine months.
It doesn’t give you an answer to which one is better. We ultimately make a decision for each patient, but they’re pretty similar overall.
Stephanie: Let’s bring back CAR T. There are different approvals for different groups of people and there has to be that personal conversation between a patient and the provider to understand the best option.
Generally speaking, if you’re comparing CAR T-cell therapy versus bispecifics in terms of limitations and challenges, what would that summary look like for our patient audience?
Dr. Brody: I’m going to change the question slightly. The question is always which one’s better, CAR T or bispecifics? But that’s not actually the question people really care about because, unfortunately, neither of these cure 100% of people. As the patient, you want to get access to both, not A or B. You might want both this and that or that and then this.
The best question is: what’s the best order to give them? If you were going to get both, would you rather get CAR T and then bispecifics or bispecifics and then CAR T?
Sometimes it’s a moot point because CAR T is approved as a second-line therapy for some patients and so far, bispecifics are approved as a third-line therapy for patients. In some cases, you don’t have to make a choice because this one’s available and that one’s not — that’s going to change a lot in the next couple of years.
I have to give you my honest belief about this. CAR T has one thing that is definitely better — they have a longer, more tried, and true precedent of keeping people like Dr. Stacy-Humphries in remission for a long time. We say “curing” patients. It’s been proven for longer so you might want the most proven thing and CAR T is that.
It still fails for a majority of patients depending on the setting. If it cures 40%, that means there’s 60% that still we need to do better for.
If you really want to get both, if you were to get CAR T and it failed, you might not be able to get bispecifics because the chemo you get right before CAR T might not make the bispecifics work well. It wipes out many of your own immune cells so giving them in that order may not be great. Giving CAR T and bispecifics right afterward may not be a good option.
If you give bispecifics first and you’re in remission, great! You don’t need something else. But if they stop working, then you could get CAR T afterward. We’ve done that many times now.
It’s not which one is better; it’s what’s the best order to get them if you’re going to get both. For some situations, it’s going to be CAR T. There are a lot of situations for which you might want to get bispecifics first also because it is gentler and the risk of those bad side effects is lower. Some people find that appealing as well.
There’s no right answer, but I’m giving you a little bit of my prejudicial belief about what might be the best order.
Stephanie: Sequencing is a huge question. If bispecifics are approved for earlier lines, it will actually be a consideration for people.
Bispecific antibodies in the community setting
All things considered, part of the question, too, was accessibility. There’s this idea that if it’s off the shelf, more people will have access to it. Do you see any challenges there, even in the community setting, with bispecifics? What are some of the solutions?
Dr. Brody: I was explaining how much easier and gentler bispecifics are compared to CAR T cells. Robyn actually had a good experience, but she did have cytokine release syndrome and people do have to get hospitalized usually for that. Sometimes, for only a few days but most commonly for a couple of weeks.
Bispecifics are gentler. The risk of high-grade side effects is less, but there is still a 3% risk.
As of today, folks getting bispecifics still have to get hospitalized for at least one day just to watch them as they get the first dose of that new medicine. That’s not so bad. One day is less than a couple of weeks, but it’s not nothing.
Some community oncologists may have difficulty getting that hospital bed lined up. It sounds like a simple thing, but practically, it can still be difficult. We are trying to overcome that obstacle.
There’s an ongoing clinical trial of epcoritamab trying to give it at a gentler step-up dosing so patients don’t even have to get that one-day hospitalization. There’s a good chance that the trial will be successful so we’re looking forward to that.
But as of now, even getting a one-day hospitalization might make this an obstacle to getting this with your local oncologist.
Bispecific antibodies as a treatment option
Stephanie: Robyn, you lead this huge group online in the CAR T-cell therapy space. I’m curious if you’ve started to get questions about bispecifics. What is your take on where you think bispecifics might fill a gap or be a good option for folks?
Robyn: Every patient is unique. In community hospitals, most oncologists are very overwhelmed with trying to take care of more of the “bread and butter” type of therapy so I really don’t see bispecifics going into the community hospitals anytime soon, at least in the more rural areas. It’s going to have to be in a city center and often on a transplant unit.
For bispecifics and CAR T, I see them as very similar. On the CAR T site, we do have people who fail. About 40% have a long-term remission rate then there’s everyone else.
Most people after the second or third round should see a lymphoma specialist then they’ll have to decide what they would rather have or what’s best for them. With the new bispecifics, it changes the whole gamut.
Most of the people on the site are actually CAR T, but then when people fail, there’s another site that deals with relapses from CAR T. A lot of those patients go on to bispecifics and they’ve had some real success with that.
Everything’s very early. Patients need to be their own advocates and have to be honest. As much as they would like to stay in their rural hometown or be with their family doctor, if you have something serious like this, you really have to travel.
New DLBCL clinical trials
Stephanie: Dr. Brody, there are different clinical trials that are happening that we haven’t covered, but as great as these developments are, everything has some limitations. There are always more clinical trials to try and solve those problems. If you could talk a little bit about some of the newer clinical trials that are happening and what problems they’re trying to solve, especially in the bispecific space.
Dr. Brody: Rituximab targets CD20 and these bispecifics so far also target CD20, a marker that’s on lymphoma cells. The CAR T cells target another thing on lymphoma cells called CD19. There are a few of those markers that you could maybe target.
If you target something on the surface of lymphoma cells, if there were a couple of lymphoma cells that didn’t have CD20 or CD19 on them, those won’t get targeted by the therapy and would grow out, escape, and cause the relapse.
We call this antigen escape when that targeted antigen is lost by the cancer cells and then those cancer cells grow back. This is the main limitation of both CAR T cells and bispecific antibodies so there are a lot of ways we could try to solve this problem.
One of the simplest ones is to go after different targets. We now have bispecifics targeting another antigen on lymphoma cells called CD22. The numbers don’t even matter that much. It’s just going after something that wasn’t on the cancer cell initially so they’re still there after the first bispecific.
We have things beyond bispecifics called trispecifics, which are going after two things on the lymphoma cell and then getting the T cell. For example, going after CD20 and CD79 and then grabbing the T cell as well, the effector that’s going to give the kiss of death to that lymphoma cell.
These are good ways to prevent the antigen escape problem. We’re starting some of those trials now. We’ve been treating a lot of people and seeing patients going into complete remission so maybe these will be even better than the already standard of care that was the cutting edge just a month ago, the new bispecific antibodies.
Maybe we’ll have trispecific antibodies as the new standard of care to prevent that problem. Some limitations but some solutions coming up for those limitations so pretty encouraging.
Different phases of clinical trials
Stephanie: Crissy, a lot of your patients get worried when they hear that one of the studies is in phase 1.
Crissy: I hear that a lot. Patients call me and say, “I only want to participate in a later phase trial like a phase 3 or a phase 4.” There’s a long conversation that goes into explaining to patients that a drug doesn’t go through all four phases of a clinical trial before it’s approved.
Phase 1 clinical trial
Crissy: A phase 1 clinical trial is a dose-finding clinical trial where they find the proper dose and establish safety. How that’s done is in a step-up fashion. Generally, there are a couple of dose levels that are identified and they enroll a small number of patients into each dose level.
They start with dose level one and give that dose to around three patients. They assess those patients and monitor for toxicity for a set number of days, usually more than a month. If no unwanted toxicity is seen, they move up to the next dose level and beyond in that same fashion.
They move up to dose level two, give that to a small number of patients, and assess them for toxicity. If there are no unwanted toxicities, they move up to the next dose level. Same thing. A small number of patients monitor them for a period of time.
Phase 2 clinical trial
Crissy: Once they’ve reached either the maximum dose in the trial or toxicity, that dose level is identified and moved into phase 2. They give the dose level that was identified in phase 1 to a large number of patients in phase 2, usually around 100 patients. That’s when they’re assessing for effectiveness.
If you look at the way a clinical trial is listed on ClinicalTrials.gov, many clinical trials are listed as phase 1/2 and that design that I described is being done on the same clinical trial.
Sometimes, after phase 2 is complete, if the data is robust enough, they’ll submit to the FDA for approval at that point. Sometimes clinical drugs only go through phase 1 and phase 2 before being submitted to the FDA. However, a phase 2 clinical trial is considered the gold standard before being submitted to the FDA for approval.
Phase 3 clinical trial
Crissy: In a phase 3 clinical trial, drugs are being compared to the standard of care. A really good example of this is after CAR T was very first approved, it was only approved for patients who had lymphoma and had failed two or more lines of therapy. The FDA approved it for patients who had failed front-line treatment and then either salvaged with an autologous stem cell transplant or just salvage therapy alone.
A phase 3 clinical trial opened up where CAR T was compared to the standard of care second-line therapy. Half of the patients received CAR T and half of the patients received standard of care with an autologous transplant.
Phase 4 clinical trial
Crissy: A phase 4 clinical trial is long-term toxicity, side effect type of trial for drugs that are already on the market.
Low enrollment for adult clinical trials
Stephanie: This is where the LSS’s Clinical Trial Support Center can help. Can you explain more about the numbers that you’re seeing? The national enrollment for adult clinical trials is very low and the LLS is trying to get the numbers up.
Crissy: Many patients come to us having tried to look for clinical trials on their own or a doctor told them to look for clinical trials or they had a loved one who just decided to start looking for clinical trials on their own. They often come to us very overwhelmed, having tried to look at ClinicalTrials.gov or other databases for clinical trials.
You can certainly go on to ClinicalTrials.gov and look for trials yourself. What our department does is take that workload off of a patient and their families and does a personalized search for the patients that we work with.
The national average of patients that participate in clinical trials is around 3-5%. Nurse navigators in the Clinical Trial Support Center help patients identify clinical trials that they’re potentially eligible for, identify barriers that they may be facing — logistical barriers, insurance barriers, not knowing what options are available — and overcome those barriers.
Our average of patients that participate is around 22%. The patients that we work with have a higher chance of participating in clinical trials because of the work that we do to help them navigate the entire process.
On behalf of the patients that we work with who do participate in a clinical trial, our nurses do more than 20 interactions with healthcare providers or the trial sponsor. Our team and LLS in general really look at that as more than 20 opportunities for a patient to fall through the cracks if they weren’t working with a nurse on our team.
Travel costs in clinical trial participation
Stephanie: Patient Joy B. asks, “Are travel costs included for clinical trials?”
Crissy: Very good question. Around a quarter of clinical trials close because they don’t reach accrual goals. They have a set goal for how many patients will participate in the trial and they don’t reach it.
One of the reasons that happens is that while clinical trials are at academic sites, patients aren’t always located near an academic site so they have to travel to participate.
A lot of work has been done over the last 5 to 10 years to overcome that barrier for patients. If they have to travel to participate in a clinical trial, how do we decrease the burden for them with that travel? And that’s been identified as financial assistance.
A lot of clinical trials will provide financial assistance to patients who participate in a trial that has to travel beyond a certain distance. Generally, it’s more than 30 or so minutes.
If a patient has to travel more than 30 or so minutes to participate in a clinical trial, I highly, highly encourage them to ask the team at the treatment site if there is financial assistance for me to participate. Oftentimes there is.
How it looks is different for every clinical trial and every site. Sometimes it is a concierge service that books patients’ travel or lodging for them. Sometimes it’s a prepaid card that helps them pay for the hotel, food, and gas.
Other times, in not-so-ideal situations, it is done on a reimbursement basis where patients submit their mileage, food receipts, and lodging receipts then they’re paid back by the company for those expenses.
There’s been a lot of advocacy from LLS and other groups to change that model. A lot of patients don’t have the money to front that. If they can’t pay for a hotel room upfront, the reimbursement model doesn’t work for them.
Additionally, The Leukemia and Lymphoma Society has copay assistance and travel assistance for patients, whether that’s to get treatment or to participate in a clinical trial.
Clinical trial paperwork
Stephanie: Robyn, you went through this process. I would love to hear about figuring out logistics, but also you had to go through excessive paperwork before the clinical trial, is that right?
Robyn: The paperwork is very intimidating for most people and even for myself. Look through the paperwork but also talk with your doctor. The paperwork always has the worst things that can possibly happen with the clinical trial, but you also need to ask your doctor, “What is the alternative that I’m getting?” A lot of times, we don’t go into that.
For example, CAR T versus allogeneic transplant. People get a little overwhelmed. Sometimes, they don’t understand that the clinical trial has some risks but even the standard therapy might even have more risks.
Ask questions. Try not to get too overwhelmed with the paperwork. Be your own advocate. Knowledge is power. Read up and be ready to go. Don’t expect all your doctors to have all the answers right off the bat. You need to be able to ask the right questions.
Crissy: If I had the time like a day or so of leeway, I would call the patient ahead of time, get their email address, and send them a copy of the consent form beforehand.
I would tell them, “Look this over with your family. Highlight, write down questions, and make a list of questions that you have about the form before coming to the site.” It’s a long form; oftentimes over 50 pages and that can be a lot in a single doctor’s visit to look over.
Bring someone with you. That can be a village. If where you’re going allows, bring people with you for that visit or have them on video call so there are more ears hearing the information and people who can remember the information.
Lastly, the really important thing is you can sign that form and say that you want to participate, but it’s not legally binding. You can leave the clinic that day and say, “You know what, I changed my mind. I don’t want to participate.” You can do that at any point in the clinical trial — a day later, a week later, months later. You’re not legally bound by any means after signing that form.
I encourage patients to stay on the trial if possible. For data capturing purposes, we need patients to stay on the study to show the effectiveness or safety of the drug so that it can potentially get to many more people.
Stephanie: I love that message of self-advocacy. What we’ve seen at The Patient Story is people get motivated when they hear other people say, “I was an advocate for myself.” It almost gives a sort of permission to speak up.
Final takeaways
Stephanie: We’d love to understand the final takeaway from each of our panelists that they’d like for audiences to walk away from. Dr. Brody, what’s your biggest message?
Dr. Brody: Patients with lymphoma are unlucky to have lymphoma but so lucky to be here today with that lymphoma. Twenty years ago, we didn’t have access to medicines that cure patients and keep them in long-term remission to go and live their lives, hopefully just like they were before.
We only have those new medicines because patients join clinical trials. Patients who join clinical trials today sometimes are getting access to the medicines of the future.
These new bispecifics that just got FDA approved, our patients were getting access to them three years ago because they did this slightly scary thing and asked, “Are there any clinical trials available?” For some of them, thank goodness, because they’re still here to tell their story today.
Stephanie: Thank you, Dr. Brody, for being a leader in the space of clinical trials. We also know what a big advocate you are for patients and their families so thank you so much.
Crissy, I know that for you, there’s something about when to talk about these options with people’s doctors, right?
Crissy: Around 90% of adult patients with cancer are treated at community sites. The bulk of clinical trials, unfortunately, are not at community sites. They’re at large academic centers.
From the time a patient is diagnosed, they may not know that there are clinical trial options available to them if they’re not at the site where a patient is being treated. Generally, physicians are going to offer patients what they have at their site.
It’s really important to talk to your doctor. Ask them from the point of diagnosis and at relapse and beyond: What are all of my options beyond the treatment that you’re offering me here? Is there something that’s potentially better or other options that I could access somewhere else?
You’re the driver of your ship and so advocate for yourself. Ask questions. From the time you’re diagnosed, ask about the treatment options that are available to you now and what if. What’s our plan B? If this treatment doesn’t work, what’s next for me?
Explore second opinions. If you’re interested, go to another site and see an expert in your disease and ask what they would recommend to you. There are no ill feelings from a healthcare provider when a patient wants to see another physician in that space and get their input.
For patients who come to me worried that they’re going to offend their doctor, you’re generally not. The grand majority of physicians are not going to be offended if you want to get a second opinion from an expert in the field.
If you are with a physician who is offended by you getting a second opinion, that isn’t a physician I would personally want. I want a physician who wants their patients to advocate for themselves to know all of their options and explore every option that’s available to them.
Stephanie: Thank you so much, Crissy, for that and for your work at the CTSC. Patients and family members find a lot of comfort for sure in having someone to lean on through what can be a very overwhelming process so thank you again.
Speaking of this process, Robyn, you are such an inspiration for people about the power of a clinical trial. We know that it might not be for everyone and it’s a very personal decision, but you have such a wonderful voice to lend when it comes to what is right about a clinical trial.
Thank you also for participating in one because that helped to further the data and the experience so that it could lead to approval. You’re proof of thriving after a trial. What would you like your message to be to our audience?
Robyn: Don’t give up hope. Make sure you ask questions. Feel free to get other opinions. Really good doctors are not insulted when you get another opinion.
I’m living a great life. I’m working. I’m traveling. I saw my youngest child get married. It’s fantastic.
I’ve been a doctor for over 30 years and I’ve taken care of a lot of patients. I’ve read hundreds of thousands of films. I’ve done thousands of biopsies. I’ve held a lot of people’s hands. I’ve counseled a lot of people. But ironically, my biggest contribution to medicine is not as a doctor, but it’s been as a patient so I’m grateful to be here.
Thanks, Stephanie, for doing this. This is fantastic. I wish I had had something like this when I was going through my experience.
Stephanie: Thank you so much, Robyn, Crissy, and Dr. Brody — incredible discussion about clinical trials in the space of DLBCL. We hope you’re able to walk away tonight knowing more and feeling more comfortable about what clinical trials are.
Special thanks again to AbbVie and Genmab for their support of our independent patient education content. The Patient Story retains full editorial control.
Patients share what actually relieves their worries
Three cancer patients discuss how to deal with scanxiety, the overwhelming fear and anxiety that often accompanies medical scans and tests for cancer patients. Our cancer patient panelists Nick Mundy, Lainie Jones, and Matt Ode share their real-life experiences and offer practical tips and strategies for managing scanxiety and maintaining emotional well-being throughout the cancer journey.
This interview has been edited for clarity and length. This is not medical advice. Please consult with your healthcare provider for treatment decisions.
Alexis Moberger, The Patient Story: We are excited to have this conversation along with our three panelists.
The Patient Story features in-depth storytelling with cancer patients. All three of our panelists actually have shared their stories on our platform. We also have live discussions with top cancer specialists to get the latest information on top treatments and new therapies.
Nick Mundy: I live in the Cincinnati area. I’m 33 years old and a two-time thriver of non-Hodgkin lymphoma. I just finished chemotherapy on May 24th for a diagnosis of stage 4 diffuse large B-cell lymphoma with liver metastasis.
Right now, I get scans every three months. I’ve been getting scans and dealing with scanxiety for about the last decade, long before I ever knew what scanxiety was.
Matt Ode: This is such an important topic because no matter who we are, when we’re going through cancer, we all face this. We all face scanxiety. We all face some type of anxiety.
I’m 31 years old and from Cleveland, Ohio. At 24, I was diagnosed with stage 4 testicular cancer. The doctors found an 11 cm tumor in my small intestine. I went through five rounds of BEP (bleomycin, etoposide, and cisplatin).
I ended up having many complications after my initial surgery, which led to multiple surgeries and eventually to kidney and liver failure. I was in a coma for two weeks and ended up being in the ICU for over 40 days. I had to relearn to walk and relive my entire life again at the age of 25.
Lainie Jones: Thank you so much to The Patient Story for having us and for the amazing community that you have created. I’m so honored to be here. I’m excited to talk about this because it’s something that’s not talked about enough.
I’m 39 years old. I live in South Florida and I’m a five-time cancer survivor. My first cancer diagnosis was at 18 months old so the word “survivor” has stuck with me throughout my entire life. My next diagnoses were breast cancer at 24, melanoma at 25, thyroid cancer at 26, sarcoma, and another relapse of stage 0 adrenal cortical carcinoma.
What is scanxiety?
Nick: Scanxiety might be closely related to PTSD and/or the fear of relapse. According to the Dana-Farber Cancer Institute, common symptoms include irritability, sweaty palms, increased heart rate, and nausea in the time leading up to an exam.
I personally think I have all of those signs and symptoms when dealing with scanxiety. The irritability is off the charts, the sweaty palms, the increased heart rate, and even my bowels change.
We’re going to talk about how we deal with it. I would love to hear what scanxiety looks like for you.
Matt: When I was rebuilding my life, I’d go in and get scans every three months and it can be very stressful at times.
As you prepare yourself, as you do the things you can control in your life, and focus on the things you can control in your life, time will help heal through this process. And as we continue to heal through ourselves through cancer, we also heal through ourselves through anxiety and scanxiety as well.
Today, I am very blessed to say that I am six years cancer-free. I now only have to do one-year scans. For some of you, it may turn from three months to six months to once a year. Everyone’s at a different pace.
Lainie: Scanxiety is something I deal with all the time. My scan routine is every three months. It looks very different for every person, but because I live with a rare genetic mutation called Li-Fraumeni syndrome, I’m monitored very closely.
Something that I always experience is loss of sleep the night before a scan and about two weeks leading up to the scan.
How to deal with scanxiety
Nick: How do we deal with scanxiety? What are some of the tips, tricks, and resources that we use?
Lainie, you’ve got a beautiful outlook and you’ve been going through scans for a long, long time. How do you deal with scanxiety and how do you continue to maintain a positive mindset throughout all of this?
Live incrementally
Lainie: First of all, I want to say I’ve mastered the whole-body MRI. I think that that’s something to be really proud of because I can stay still for two hours.
I live by the motto that I live every day like it’s my first. A lot of people ask, “Wait, what? You live every day like you were just born?” I really do. As a cancer patient, cancer thriver, it’s a gift we’ve been given so that is my motto.
I get scans so frequently that I tell myself if there is something there, it’s only three months old. That’s really helped me stay positive.
I’ve had scans where things show up then you know it’s time to tackle that new adventure. I really think what helps is incrementally focusing on the times of your scans and staying on top of being your own advocate.
You know your body best. It’s so important to have that at the forefront of your mind. I live in three-month increments. Every time I get ready to go get my scans, I think, Okay, here I am, another three months. We’re going after it. Keep a positive mindset all the way through.
Nick: I love that. Living incrementally, living three months at a time. I’m going to take that and put it in my tool kit. I’m just entering the three-month scans so this is new to me. I’ve been through every six months, annually, or when something comes up. With that perspective and that re-framing, it gives you the power back so I really, really appreciate you sharing that.
Mind over matter
Nick: You’ve mastered the whole-body MRI. The cardiac MRI that I’ve gone through is usually about 90 minutes and every time I go through it, I’ve almost tapped. How do you manage to stay still through the two hours?
Lainie: Every single time I get strapped in, there’s an itch at the top of my toe. I think it’s really mind over matter. When I’m laying in that machine, I just put myself in the zone. I’ve mastered it to the point where I fall asleep and need to try to keep myself up.
I really make sure that I have a great playlist. It’s my Beating Cancer playlist on Spotify. Most of the time, institutions can play your Spotify list. I’m dancing mentally and giving myself good vibes. If I can’t play the playlist, I make sure I have some really good tunes to zone out. The noise is unbearable, but it’s mind over matter.
Nick: Truly some great nuggets of information. I really, really appreciate that, Lainie. Thank you for sharing.
Find the right support system
Nick: Matt has absolutely been through it. This man is a warrior and the definition of perseverance.
Matt, I know that you are big on controlling what you can control. Talk to us about how you deal with scanxiety. How do you control all the things that you’re going through and all the feelings that you’re feeling when dealing with scanxiety?
Matt: We have support systems such as our family and our friends, but our family and friends aren’t actually doing the scans. They’re not actually going through cancer. They’re there for us, but that can make us feel really lonely.
How do you find the right support system, especially when you’re feeling alone in the situation? You have this anxiety and you’re talking to people, but they’re not actually going through it. Sometimes you just feel like they’re just listening, but they might not understand exactly.
I was lucky enough that my hospital, the Cleveland Clinic, has what’s called the 4th Angel mentorship program. What they do is connect you with an individual who went through the exact same cancer as you. Ask your hospital if they have a similar program.
It gave me peace of mind when I was able to talk with this individual who had gone through pretty much the same cancer as me. Every time I’d get a scan, I would text him and say, “Hey man, I’m feeling this today,” and he’d walk me through how he was able to overcome it or what was able to help him. It was a tremendous help.
I built a Facebook group for cancer patients, survivors, and caregivers. The whole premise of the group is to help you along your journey with cancer. We have over 6,000 members in the group now. It’s an incredible family filled with love, support, and people who can relate to one another. You can pretty much search what type of cancer you have and I can guarantee you there’s somebody in that group that is going through something similar.
Ask questions
Matt: If you get really nervous and have so many questions, don’t be afraid to ask your doctor. A lot of times, the anxiety is made-up stories in my head. You have a scenario and you make it the worst possible scenario.
But when I would talk to my doctor and he’d say, “Hey, that’s not necessarily true, this is not necessarily true, and here’s how it actually works. Maybe here’s how you’re feeling and I dealt with so many patients like this,” that can give you a sense of ease.
Nick: That’s so powerful. Thank you so much for sharing that. I love your vulnerability. Sometimes as cancer patients, I think we can get blinded by the headlights and almost forget that we have the right to ask these questions. We forget that it’s okay to feel how we are feeling at this moment.
I love the Facebook group because it’s imperative to have a great support system and other people that you can express yourself authentically and honestly with that have walked a mile in your shoes.
We are all surrounded by people that love us. We have great family and friends and caregivers, but if they haven’t been in your shoes as a patient, it’s very hard sometimes to get the sincere empathy that you’re really looking for.
The group that you have sounds phenomenal. I’ve gone through this journey many years where I felt alone and I could have definitely used a Facebook group like that. I’m glad that you initiated that and created it.
Be vulnerable
Matt: One last thing is your vulnerability is not your weakness. Your vulnerability is actually your strength. I’m not saying to open up to the world. You don’t have to be a panelist like us and share your story everywhere. Being willing to open up to somebody that you can trust is like baggage getting off of your shoulders.
When I first got diagnosed, I did not want to share any of my emotions for a good month. I was stone cold, afraid to share anything, thinking I was weak, especially as a man. I felt like being a man, you can’t just express how you’re feeling.
When I was finally able to release some of my emotions and really express what I was going through, I could then take away what I wasn’t able to control and focus on what I can control, where I needed to go, and the steps to help heal me.
Lainie: And also, just to add to that, you’re helping other people while doing that. It’s so important. A lot of times, we hold things in and don’t realize that when you’re sharing your story, it’s going to help other people. It’s so important.
Nick: I love that we’re having this discussion because often there’s another person who’s got the same scanxiety, fear, worry, or same question that you have. By being vulnerable and leading the way with that, it gives other people permission to be vulnerable as well and so I’m really glad that you guys both highlighted that.
Name your emotions
Nick: There are a lot of different ways that I’ve dealt with scanxiety over the years, some healthy, some not so healthy. Today, I really lean more into anything that’s going to improve my wellness or give me the best chance at being here long term so I’m making decisions that are going to give me more life.
One of them is naming the feeling that I’m feeling. Scanxiety, as we mentioned earlier, can manifest in multiple different ways and lots of different feelings, physical symptoms, and mental symptoms.
If I can’t name what I’m feeling, I’ll pull up a feelings wheel, similar to one that you’ll see in a therapist’s office. There are different emotions in the middle and often, I’ll start off with angry. From there, I’ll look at the other things and really see what is it that I’m really feeling.
I know that I’m presenting as angry, but what’s really underneath that anger? There’s typically fear, sadness, and feeling withdrawn. I take the time to pull out the feelings wheel or just sit down and put pen to paper and write what I’m feeling.
Take it a step further and share with someone who is a safe and trusted person: a loved one, a friend, a family member, or a peer-to-peer mentor like the program that Matt was talking about. I know that The Leukemia & Lymphoma Society has a peer mentor program as well.
Get that feeling out and remove the power that it can grow when you keep it inside. Sometimes you feel like you have to be tough and put on a face for everybody. When you keep those feelings inside, it can bubble up and just explode. I can have an angry outburst, throw up from being so sick of how I’ve worked myself up, or have my knee shaking as I’m walking in before a scan.
I have found that naming the feeling, expressing it, and talking about it has taken the power away from it and it’s given me power back over the situation.
Manage your environment
Nick: The next thing that I try to do is manage my environment. Typically, when we hear environment, we’re thinking about the setting around us. If you’ve been going through scans like most of us have, there’s not much that you can do to manipulate your external environment.
But leading up to that scan, you can manipulate the environment of your home or office and create a safe place that feels very zen, relaxing, and peaceful. Spend time there and create a ritual for yourself.
As important as it is to make sure that you cultivate the proper external environment, I think it’s even more important to cultivate the right internal environment by being conscious of what you’re telling yourself. What’s the story that you’re telling yourself? What’s on repeat? Be really cognizant of that.
Managing my internal environment goes to telling myself the facts. I’m a master at spinning stories. When I do that, I get sick. I just keep going. I have to tell myself the facts. I do that leading up to my scans. I do that in the car. I do that while I’m in the MRI or PET scan machine.
What that looks like for me is, “I am safe. I am loved. I have everything I need to live a full life today at this moment. The only thing that worrying about this situation is going to do is change my health. It will not change the results. But if I continue to worry like this, it can and will impact my health over time.”
I want to live a very long and healthy life. I want to give myself the best chance and I know that me stressing myself out is going to lower my immune system. It’s going to open me up to infection. As someone who’s battled febrile neutropenia and been hospitalized countless times, I’m doing anything I can to boost my immune system.
We receive information from our doctors and that information changes from appointment to appointment. But the facts are still that I am safe, I am loved deeply by my family members, by my friends, and by my support system, and I have everything at this moment to lead a successful and meaningful life today.
And really, today is all I have control over. I don’t have control over the three months from now when I go back to get my scan. But today, I have everything I need to live a meaningful, fulfilling, and joyful life. I remind myself of that and I have to continue to replay that over and over until I eventually believe it and that becomes my default.
Practice deep breathing
Nick: Box breathing or breathwork, in general, is something I do to get myself nice and calm. One of my favorite sets of breathwork is a nice deep inhale through the nose for four seconds and then exhaling through the mouth for six. I’ll do that about 10 times.
By focusing on my breath, it brings me back to the here and now. It doesn’t allow me to project myself into the future, into the doctor’s office where he’s reading me the results about my end of life because that’s the way that my brain works.
When I start to spin these stories about what the doctor’s going to tell me when I see him or her next week, all of these things have helped me mentally, emotionally, and physically.
Lainie: That’s so awesome. I suffer from lymphedema in my left arm and I’ve had numerous cellulitis infections from it. There was a point when I was in the ER and my heart rate was so high because I was so worked up. I was covered in hives,
My husband said, “You have to calm down and take deep breaths and just put relaxing music on.” Your box breaths would have been so helpful at that moment. They said, “You’re going to be admitted into the cardiac area if your heart rate doesn’t drop.”
Those deep breaths and getting in the zone are so important. Your body can do crazy things, but make sure you’re in tune with it.
Make healthy choices
Matt: I love that. I would say one thing that has helped me tremendously is focusing on the foods that I eat and making sure I try to get some type of movement.
I know that for some people who are going through cancer, it’s tough to workout. Getting some type of movement in and eating the right foods will not just mentally, but also physically prepare you for the scans to come up. If you’re prepared, you will feel more confident, you will have more courage, and that usually leads to less anxiety.
I eat clean foods such as lean protein, vegetables, and fruits, and stay away from fast food and heavy carbs as much as I can. I’m not saying carbs are bad by any means, but some of these things make you bloated. It’s not good for your gut health and ultimately not good for your mindset.
When I feel like crap, guess what that means? It leads to more anxiety in my life. Going into that scan, I’d feel less prepared. When I’m eating clean and moving, I’m doing everything I can to try to heal my body.
You’re never going to be perfect. There are going to be ups and downs. I remember during my chemotherapy, there were some days when the only thing I could eat was a peanut butter and jelly sandwich, so I ate a peanut butter and jelly sandwich.
But if at least 80% of the time you really try to take control of your health, you’re going to feel so much better going into these scans. You’re going to feel confident and stronger. You’re going to feel like you’ve done everything you could control.
Whatever the outcome is, if you’re getting three-month scans, guess what? The worst possible scenario is they caught it within three months, but you did everything you possibly could to take care of your health. For me, that has made a massive difference when it comes to preparing for my scans as well.
I created a whole list of healthy food options, healthy snack options, and healthy vitamins. Talk to your physician before taking anything. It’s made a massive difference in my life and allowed me to feel more confident and more prepared going into my scans.
Anxiety while waiting for the results
Nick: There can also be some relief after you’ve gotten your results from the scans and found out that there was something there. We’re talking about scanxiety, but this is also another form of scanxiety that I could definitely relate to.
In 2022, I started feeling all the signs and symptoms of my non-Hodgkin’s lymphoma coming back. The fatigue was starting to set in. I was having wicked night sweats every single night, my beds were drenched, and my lymph nodes started to swell up.
I told my oncologist and the doctors in the ER, “I need a scan. Something’s wrong with me. I had cancer last year and I think I have cancer again.” Everybody said, “No, you’re fine. You look healthy. You’re strong. You’re in good shape. You’re doing all these physical activities.”
That made me feel like a crazy person until I got my scan. I had to beg to get the scan. When I got the results, I saw the words liver metastasis and lesions. From there, I had a biopsy a week later. Then a week after that, I found out that I had stage 4.
While I wasn’t relieved to know that I had stage 4 with liver metastasis, I was relieved to have my intuition recognized and validated. I know my body better than anybody else. I live in it 24/7.
I knew that something was off. For people to continuously tell me that something wasn’t wrong made me feel all of the signs and symptoms of scanxiety. There was a little bit of relief once the diagnosis came.
I say that to cover the spectrum of scanxiety. We could interview 15 other people and they are going to have different things that they do and different experiences with their scanxiety. I wanted to share and make somebody else who might be going through something similar feel seen. You can have scanxiety and almost relief or have some of these things come up as you do find things that are on your scans and your results.
Anxiety during treatment
Lainie: Treatment on its own can be anxiety-ridden. I’ve done chemo three different times and I’m on treatment for the rest of my life so I get infusions every 21 days.
When I was going through the aggressive portion of chemo, I would always try and make it fun. Turn lemons into lemonade. I know that sounds so cliché, but making an experience that’s maybe a little dark or not so fun helped put me at ease.
I would always have somebody come with me, a friend or family member, and just have fun. I know that sounds silly, but sometimes fun helps that anxiety go away. Make it into an experience. Every day is a memory so make it a memory.
Treat yourself after a scan
Nick: Do you have a go-to treat after your scan? You got to treat yourself. After you’ve gotten out of the two-hour MRI, what do you do? How do you celebrate or how do you at least sigh and let yourself just breathe again?
Lainie: The first thing is I go to the bathroom. That’s a treat on its own.
Matt: I love it. I still have anxiety until I get my results. My results usually don’t come for 3 to 4 days so even after I’m done with the scan, I’m still nervous. I got half of it done. Now I got to get the results.
Anybody who gets scans usually waits at least 12 hours, sometimes even 24 hours, before they can eat anything because they need to make sure their body is completely cleansed.
Once I get my results and get the all-clear, I take my wife and we usually go to a nice steak dinner. That’s how I do it. I celebrate when I get the actual results.
Lainie: I love that. I don’t really have a specific food I like to eat after. All I know is I just want a big cup of water to get rid of that crap that they inject in your body. Fortunately, for a whole-body MRI, there’s no fasting. I try to eat a little something before.
I go to MD Anderson and I just sit and wait. I don’t really have a celebratory meal. Maybe get a nice coffee.
But when I have surgeries, that’s a different story. I like to have my McDonald’s waiting for me when I’m done with surgery. It’s not healthy, but a guilty pleasure.
Matt: We all deserve a little guilty pleasure.
Lainie: Yeah, exactly.
I travel to Houston every three months for my scans. I always keep my medical ID band on. If my husband doesn’t come with me, when I get home or once we get the all-clear, he has to take it off. It’s like a celebratory tradition. Another three months to go! It’s always nice to have those little traditions as a cancer patient.
Staying strong before and after a scan
Nick: How do you not lose your will to fight?
I personally feel like there is no way that I can lose by showing up and living life to the fullest on a daily basis, being the best version of myself that I can possibly be.
I empty the tank every single day. I live boldly. I love boldly. I try to treat others with respect. I try to honor my values and my morals. This is outside of cancer, really.
At the end of the day, I really feel like if I operate like that on a daily basis, losing is not even an option. I’m going out, I’m giving it everything I’ve got every single day, and I think that’s the best way that I can possibly honor life.
We’re all going to die. We came into life the same way. We’re going to leave the same way gradually or suddenly. But in order for me to be prepared for that, I just need to live well. I try to live well on a daily basis. I feel like if I live well on a daily basis and I love well on a daily basis, then there’s no way that I can lose.
Matt: I love that, Nick, that was perfectly said. I really wouldn’t have to say another thing. Going through what we’re all going through makes life just a little more precious. It puts things into perspective.
The little things that I used to worry about before cancer, like certain arguments or feeling I might be losing certain friends, don’t really matter so much anymore. What does matter is the people who love me. Being the best possible version of myself, just like Nick said. Then to add a little cherry on top of that is giving back.
Personally, being able to serve and help others is what gives me true happiness and fulfillment in my life. I have a little saying and it’s actually an acronym called h.o.p.e. — help one person every day — and that is just the motto I live by.
If I can help one person every day, being a Christian, I feel like I’m able to serve God in the way that He wants me to. I’m not pushing religion on anybody, but that’s another thing.
My faith has helped me through a lot of this. I know a lot of people have some type of faith; that will help you through it as well. Being the best version of yourself, realizing that life is just a little more precious, not worrying about those little things, being of service to as many people as you can, and knowing when your time is up, you can say, when you meet your Creator, that you did everything you possibly could.
Lainie: Matt, I love that. For me, it’s one word: purpose. We are all here for a reason.
People know me as the girl who’s had five cancers; that’s not very hard to forget, but I don’t let it define who I am. I let it empower me. It’s so important to take your experience, empower others, and not look at cancer as a death sentence. Let this empower who you are and help inspire others because it’s so important.
Unfortunately, we have been given a gift that nobody wants. Take that gift to help inspire others and lead with purpose.
I have a social media platform that I use to share my journey. I truly believe that if I’m helping one person, that’s why I’m here, thriving, and living every single day like it’s my first.
Conclusion
Nick: I love it. Man, this is phenomenal. I am so grateful to The Patient Story for connecting us and for hosting events like this so we can have real talks and discussions with other cancer patients, caregivers, and family members who are in the trenches because we need it. Nobody fights alone.
Thank you, Lainie and Matt.
Alexis: Thank you, Nick, Matt, and Lainie, for joining our discussion. We definitely want to continue these live discussions and make sure that they are educational and helpful to patients and caregivers. You can also find the latest patient stories and the latest medical news on our platform.
