Navigating Brain Cancer While Pregnant: Caroline’s Grade 4 Glioblastoma Story
Glioblastoma during pregnancy is not a scenario most people ever imagine for themselves, yet that is exactly where Caroline found herself at 20 weeks pregnant. What began as something she dismissed as “pregnancy brain” and stress from work, escalated into slurred speech and the sudden inability to read from her own teaching materials in front of a full class.
Alarmed, she went home and searched “brain tumor while pregnant,” a phrase no expectant parent ever wants to type. An emergency room visit and MRI revealed a large mass on her language center, but because she was pregnant, her team could not yet fully characterize what they were seeing.
Caroline and her medical team wrestled with impossible choices, including whether to attempt an awake craniotomy while she was pregnant, knowing there were no guarantees of getting a clear diagnosis or preserving her speech. She chose to go home briefly and try speech therapy, but her symptoms sharply worsened. Within days, her language had deteriorated so much that she could not accurately say the month or year. A repeat scan showed the mass had nearly doubled in size, and an emergency craniotomy followed.
When she woke up, Caroline was relieved simply to be speaking again, even if imperfectly. But the pathology news was devastating: glioblastoma, a fast-growing grade 4 primary brain tumor. Because of how urgently she needed treatment, her doctors told her they could not both aggressively treat the tumor and safely continue the pregnancy. Ultimately, Caroline chose to terminate her pregnancy at 21 weeks, while grieving the possibility that she herself might live less than a year and her 22‑month‑old daughter would not remember her.
Treatment moved quickly. Caroline underwent six weeks of chemoradiation and then high‑dose chemotherapy, which she had to stop early when her platelets could no longer tolerate the regimen. She also used a wearable device with electrodes on her scalp.
Caroline reoriented her life completely around simply being present with her daughter and focusing on small daily goals, like getting out of bed and walking. Over time, her scans showed no evidence of disease or stable findings, and her neurologist encouraged her to use that window to heal while continuing close MRI surveillance. Knowing how low glioblastoma survival statistics can be, she told her doctor she intended to be among the small percentage who reach five years.
Caroline describes living with terminal cancer as both a blessing and a curse. The experience stripped away much of her lifelong anxiety and fear, leaving her more grounded in each day. She had already survived other trauma before cancer, but this diagnosis — and the agonizing decision to end a wanted pregnancy to pursue treatment — became a stark turning point.
Today, she frames her life less in terms of how long she has and more in terms of how fully she can love her daughter, inhabit her relationships, and refuse to be intimidated by a world that once felt overwhelming.
Key Story Takeaways:
Persistent or worsening symptoms should be checked by a doctor.
Patients are never to blame for how their bodies respond to disease or treatment; cancer and the limits of current therapies are at fault, not choices or willpower.
Caroline advocated for herself, accepted help, and defined success as time with her child and healing in her body.
Even with a terminal diagnosis and difficult statistics, Caroline focuses on becoming a five-year survivor and living one day at a time.
Caroline lives with both grief over ending a wanted pregnancy and deep gratitude that treatment has given her more time with her daughter.
Name: Caroline W.
Age at Diagnosis:
37
Diagnosis:
Glioblastoma
Grading:
Grade 4
Symptom:
Loss of the ability to speak and write
Treatments:
Surgery: craniotomy
Chemotherapy
Radiation
Wearable electric field therapy for glioblastoma
This interview has been edited for clarity and length. This is not medical advice. Please consult with your healthcare provider to make informed treatment decisions.
The views and opinions expressed in this interview do not necessarily reflect those of The Patient Story.
I started thinking that I was having pregnancy brain.
When I was diagnosed, I was exactly 20 weeks pregnant. They didn’t know if I would survive the pregnancy.
Caroline blamed her first symptoms on pregnancy and stress.
I was a massage therapist and a yoga instructor. I had my own business in the neighborhood for 10 years. I thought it was a little bit of stress, maybe.
Struggling with Speaking and Reading During Pregnancy
But her symptoms intensified.
I taught a class of about 30 students. During class, I was stumbling on my words. I knew something was wrong because when I went to read the book that I had made for the course, I couldn’t read it. I was able to perform the physical part completely, but that’s all I did for the rest of the class for two hours. I could respond, but I was slurring. I went home and searched on Google, “brain tumor while pregnant.”
Searching for Answers: Google and Emergency Room Visit
Caroline went to the emergency room. An MRI led to answers and more questions.
One of the residents came and he was excited. He said they found a mass on my language center. I was excited at first because it felt like validation that it wasn’t me having a mishap. It was a big mass right on my language center. But they didn’t know what it was. Is it a brain bleed? Is it a stroke? Is it a tumor? They couldn’t do an MRI with contrast since I was pregnant.
Discovering the Brain Mass and Facing Uncertainty
I stayed in the ICU for a couple of days. While they tried to decide whether they could do an awake craniotomy to see if they could do a biopsy, the problem was that they were afraid that the blood had already coagulated inside my brain and they would not be able to get a good sample.
ICU Stay and Difficult Diagnostic Decisions
My ability to speak was already declining every day. They gave me the option to try the awake craniotomy, knowing it may or may not work, or go home, go to speech therapy a couple of times a week to see if it was a stroke, and come back in three weeks to see if they can do something then. I chose that option because I wanted to go home.
Worsening Symptoms and Fast-Growing Brain Tumor
I was doing speech therapy and feeling good for a couple of days, but then it started going downhill quickly. My husband took me back to the hospital a few days later and the mass had nearly doubled in size.
Loss of Speech and Cognitive Confusion
By the time I got in, I didn’t know who the president was. I knew what month it was, but I couldn’t say it. I tried to say March and it would come out as May. If I tried to say 2022, it came out as 2002. I couldn’t get the words right.
We’re here now, so let’s get it taken care of. They said it could have been a hemorrhagic stroke. I was hoping for that because I can live with that. I might not be able to talk again, but I could live with that. They were also thinking it could be some sort of bleed.
Emergency Craniotomy and Return of Speech
After an emergency craniotomy, Caroline was able to speak again.
It wasn’t the best, but to go from not being able to speak to speaking again, I was so excited. When they brought me back to my room, I was told that it looked like it was a tumor and that it looked malignant. I said, “Okay, we’ll talk about it tomorrow.”
Devastating Diagnosis: Glioblastoma While Pregnant
In my heart, I felt it was going to be a primary brain tumor.
They came in and said it’s a glioma. I said, “Oh, no. That’s the worst one.” She said, “Not necessarily. It could be a grade 2. You could continue your pregnancy if it’s a grade 2.” I said, “What if it’s a grade 3 or 4?” She said, “We throw the kitchen sink at it.”
And it was grade 4.
Forced to Make a Heartbreaking Pregnancy Decision
Before she could fully comprehend her diagnosis, Caroline was forced to make a heart-wrenching decision.
When you hear that news, you think, “Is there any other way to get around this?” They said they wouldn’t be able to treat me if I kept the baby. It would have been too long between having the baby and being able to get chemotherapy.
Undergoing Pregnancy Termination for Cancer Treatment
When I was diagnosed, I was exactly 20 weeks pregnant. I was diagnosed on the same week as my 20-week scan and the scan was perfect. I met with the fetal medicine specialist and the genetics specialist to find out if there’s any way they could take my baby out sooner, but the answer was no.
Radiation would be too far away if I were to have the baby. They wouldn’t even do the radiation because it was too far from the surgery.
I was encouraged to have a termination because they felt like I needed to start treatment as soon as possible. That way, I could extend my life to maybe a year. The prognosis for glioblastoma is about a year to a year and a half. It’s usually six months to a year and a half, so they didn’t know if I would survive my pregnancy.
I scheduled my termination, which was a two-day procedure, and had it done at 21 weeks. Making that choice and knowing that I might not even live a year was the hardest thing. My other baby at home was only 22 months then. She won’t remember me. My biggest concern was that she wouldn’t remember who I am. Those two moments were definitely the hardest.
I was afraid of leaving my daughter, but I wasn’t afraid of dying. If it weren’t for my daughter, I probably wouldn’t have been as worked up.
Caroline’s fight continued, with no time to pause.
Caroline’s Fight Continued, with No Time to Pause
After the night I came home from my termination, I had to go back the next morning to be fitted for my radiation mask. It was fast. I did six weeks of chemoradiation. I was told that they wanted to do a year of high-dose chemo. The treatment would be five days on, three days off for a year. But I only made it six months because my platelets couldn’t take it anymore.
I had radiation scheduled at 7:40 a.m., Monday through Friday. We got up, got in the car, and listened to a glioblastoma podcast on the way there, trying to learn more.
Chemoradiation, Podcast Learning, and Side Effects
Fatigue was the worst part for me. I lost my hair not from the chemo, but from the radiation. I got a crazy radiation haircut. I lost my taste for a lot of different things that I used to like and different smells. I had nausea and constipation from chemo, which wasn’t as difficult as I imagined it would be, especially the lower-dose chemo. The higher dose is what got me.
Daily Life Adjustments During Treatment
After I shaved my head, I had something attached to my head and I had to shave every day to get all the prickles off. I put these electrodes on my head that came with a little backpack. It’s a little stimulation for the brain.
Obviously, I wasn’t going back to work, so I didn’t even close my own office. The main thing in my life that I wanted was to be with my baby. Keep the nanny as long as possible so she can take care of her and I can be here to see them, even if I can’t help with anything. In the beginning, my goal every day was to get out of bed and walk.
Support System: Focusing on Family and Motivation
Then, Caroline finally got encouraging news.
My original neurologist… Her idea was that I have no evidence of disease right now. My tumor was not showing up. It was either no evidence of disease or stable or unremarkable, whatever it was. I was getting MRIs every other month. She said, “If you want to take this time off to heal, then we’ll continue the MRI every other month. When your recurrence happens, you will go back on chemo.” I let my body heal and returned because my body was responsive to chemo.
Encouraging Medical News: No Evidence of Disease
I said to the doctor, “If I’m doing this, I’m going to be a five-year survivor,” because of the statistics. It’s hard to say. A certain percentage makes it to five years. I said that I would be in that 5%.
Five-Year Survival Hope and Living with Terminal Cancer
Having terminal cancer has been a blessing and a curse, but I live for each day now. I had experienced a lot of trauma growing up and I feel like I’ve been a survivor my whole life. I had bad anxiety and I don’t have that kind of anxiety anymore because I have gone through the hardest thing I could ever have gone through in my life. The world does not scare me anymore.
Strategies for Treatment, Trials, and Team Building
Chronic lymphocytic leukemia (CLL) care is getting more personalized than ever.
CLL patient advocate Jeff Folloder and expert hematologist-oncologist Dr. Nicole Lamanna (Columbia University Irving Medical Center) explain how today’s innovative targeted therapies, time-limited treatment options, and emerging clinical trials can help patients craft a care strategy that truly fits their needs. The conversation dives into how to assemble a strong, collaborative medical team, at both local hospitals and major centers, so patients and families feel empowered at every step.
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Building a CLL Game Plan: Strategies for Treatment, Trials, and Team Building
Hosted by The Patient Story Team | 1h 6m 44s
Chronic lymphocytic leukemia (CLL) care is more personalized than ever. Patient advocate Jeff Folloder and expert hematologist-oncologist Dr. Nicole Lamanna (Columbia University Irving Medical Center) explain how today’s innovative targeted therapies, time-limited treatment options, and emerging clinical trials can help patients craft a care strategy that truly fits their needs.
Thank you again to AbbVie for its support of our independent patient education program. The Patient Story retains full editorial control over all content.
Jeff Folloder: Hello and welcome to “Building a CLL Game Plan: Strategies for Treatment, Trials, and Team Building.” My name is Jeff Folloder. I’m a long-time passionate patient advocate. I’ve been on the chronic lymphocytic leukemia (CLL) journey for over 15 years. Because of all the wonderful things that have happened over those years, I am living an excellent life, and it’s my mission to make sure that everyone with CLL has the opportunity to live a great life.
In this discussion, we’ll talk about the latest treatment options for CLL, how to build the right medical team, how to access clinical trials even in community settings, and most importantly, how to make shared decisions that keep quality of life at the center.
Tremendous thanks to our sponsor, AbbVie. Without them, we can’t do programs like this, so their support is important, helping us host these educational programs for free. We want to note that The Patient Story does maintain full editorial control.
We hope that you find this helpful. Remember, this is not a substitute for medical advice. We hope it will inspire better discussions with your medical team.
We want to hear from you, so we can make these discussions even better. What did you enjoy? How can we improve? Please take the time to let us know. It’s how we get better.
It’s my pleasure to introduce my friend, Dr. Nicole Lamanna. She is the Judy Horrigan Professor of Medicine for the Leukemia Service and the director of the Chronic Lymphocytic Leukemia Program. She’s at the NewYork-Presbyterian/Columbia University Irving Medical Center. I have a very direct question for you, Dr. Lamanna. What drives you to show up to work every day?
Dr. Nicole Lamanna: It’s a pleasure to be here. I’ve been doing this for a long time, too. As he’s been an advocate for 15 years or so, I’ve been doing this for over 20 years. And I love what I do. I’m also a very staunch patient advocate and he knows this very well, which is why I do these kinds of programs. I love taking care of patients with this disease and trying to figure out therapies that will improve the quality of their lives and the efficacy of the treatments to deal with this disease.
Hopefully, at some point, we’ll have curative therapies. I think that we’re getting close with what we’re doing with all the great treatments we have now. It’s been a big change for somebody like me who started in the era of chemoimmunotherapy and to see this transition over 20 years is phenomenal.
That’s what drives me to come to work every day. You build long-lasting bonds with your patients. This is, in part, why I love dealing with this disease, because I get to make wonderful relationships with my patients, so it’s a lot of fun.
Newest Treatments for CLL
Jeff: You mentioned the old-school treatment programs, like chemoimmunotherapy. I’d like to think that for most of us, that’s pretty much in the rear-view mirror. What new treatments are available for CLL today, and how are they different from older treatments that patients used to get?
Dr. Lamanna: Some of you may have even received some of those older treatments, right? Twenty years ago, we used chemoimmunotherapy, which included drugs like fludarabine (Fludara), bendamustine (Treanda, Bendeka), cyclophosphamide (Cytoxan), all intravenous treatments.
Don’t get me wrong — they were great treatments. In fact, some of you might have had them and have done well on them, but they had a lot of side effects, so they were harder to give to older individuals. Remember, the median age for this disease is the 70s, although we have lots of younger people, too. But sometimes, chemotherapy can be rougher if you have lots of medical problems and are older, so it became prohibitive for many older folks.
As we started to understand the biology of this disease, how these abnormal B cells grow and live, and what supports them, understanding the pathway of their development led to a lot of targets. Over the last 10 to 12 years, we’ve had a slew of targeted therapies, so they’re not that new anymore.
We have two major classes. One is called Bruton tyrosine kinase (BTK) inhibitors, which many might be familiar with. They’re oral agents, and we now have three FDA-approved BTK inhibitors — ibrutinib (Imbruvica), acalabrutinib (Calquence), and zanubrutinib (Brukinsa) — which have dramatically transformed the landscape of treating this disease.
They’re much more tolerable therapies with great efficacy. In many randomized trials against the older forms of chemotherapy, they showed an improvement. In a way, what Jeff mentioned about moving away from chemotherapy is due to both toxicity as well as proven improvement in efficacy. We’ve moved away and pushed chemoimmunotherapy down the road for these targeted therapies.
The other big class is what they call BCL-2 inhibitors, which are agents that help with the killing and allow the CLL cells to die. There’s one that’s FDA-approved, which many of you might be familiar with. It’s an oral agent called venetoclax (Venclexta) and is often given in combination with other therapies, which also dramatically changed the way we treat this disease.
Now there are newer agents, Jeff. I don’t know if you want me to speak about all the exciting stuff in clinical trials now, but we can get to that later. But these are the ones that have removed chemoimmunotherapy and have moved patients into the newer era of targeted agents with improvement in disease control and better side effects.
Jeff: That’s fantastic to hear.
Continuous Therapy vs. Treatment for a Set Time
Jeff: I know a lot about BTK inhibitors and venetoclax (Venclexta), but I have also been hearing from our communities that these treatments don’t have to go on forever. It used to be that you had to take them for the rest of your life. What happened? What changed? And how do doctors decide who gets to do it short-term and who gets to do it for the rest of their life?
Dr. Lamanna: When we first started with targeted therapies, BTK inhibitors were given as a daily medication that you would take indefinitely. Unless you were having a side effect to the medicine or it was no longer working, people can be on these for years.
Then, when venetoclax (Venclexta), the BCL-2 inhibitor, became available, we started partnering it with a monoclonal antibody called rituximab (Rituxan), which is an intravenous but more targeted therapy than the older chemos. What we noticed is that people could get very deep remissions quicker than with just the BTK inhibitor that we were giving chronically.
Through clinical trials, it became a time-limited combination that people could take. This was for patients who had prior treatments and was given for two years. We knew it had deep responses that people could get off of treatment and take a break. It’s like what we do with chemotherapy, where people would get six cycles of a regimen and be done, but it was limited more so because of toxicity issues. You can’t continue chemo indefinitely, but you’d get treatment and get off treatment until you needed treatment again, maybe years down the road. Venetoclax (Venclexta) in combination with rituximab (Rituxan) became the first time-limited combination.
When we saw that, we moved it to the frontline with a different monoclonal antibody called obinutuzumab (Gazyva), which is a little bit more potent. Then it became a shorter duration in the frontline setting, meaning people who have never had treatment for their CLL. It was a year of therapy, and there were great responses.
Now we have good options for patients. Some patients, if they want, can take continuous therapy, and some people can take time-limited therapy.
Fast forward to more recent data — so it’s going to get a little bit busier — studies were saying, “Why don’t we combine BTK and BCL-2 and also do a time-limited approach with our two best therapies?” This oral-oral combination is not yet FDA-approved, but it’s in the NCCN Guidelines from a study that was conducted for several years and reported at one of our big meetings in 2024, looking at acalabrutinib (Calquence) and venetoclax (Venclexta). We have an oral-oral combination that will hopefully get approved by the FDA soon, but treatment is also time-limited. How do we choose?
This becomes a discussion between you and your provider. The good news is that both of these are great treatment options. For some patients, it depends on their other medical problems, or particularly if they’re frail. It depends on social circumstances. Do they have good support at home? Can they go back and forth to the medical center frequently?
There are two different treatment paradigms of continuous therapy with an oral drug versus combination therapy. They have different side effects and different monitoring schedules, so we have to take those into account with each patient that we see and have an open dialogue about preferences, side effects, and medical problems, so we can help guide you in making an informed decision.
There are some patients for whom a pill every day is an easy thing to do. Depending on their circumstance or how busy they are, like maybe they can’t go back and forth to have labs done frequently, then the medical center might choose a pill every day.
Some people want to be done with therapy. There’s an advantage to getting a break from treatment. They might say this works for them. They want to get a good response and hopefully have years off of treatment, so they don’t take medicine continuously that could have potentially chronic side effects.
We have a much longer discussion now in the clinic about treatment options because the good news is we now have these treatment options. We didn’t have them before. One of the important things that patients need to know is that they can switch. They can go from one drug to another. Choosing one treatment doesn’t preclude them from changing therapies in the future if they need them, which is important because patients ask what the better choice is. The answer is we don’t have one.
Randomized trials are looking to see if one treatment option is better if we start that first versus the other, but that will take years in follow-up to get data to say if one is truly better to start with before another or not. In general, we haven’t said that that’s the case because they’re both good options, and we haven’t shown that one is necessarily better than the other. It’s more of a discussion between the provider and the patient, taking into account all these other factors, like medical problems, other medicines that the patient might be on, and so on, to make an informed decision about which option might be better at that particular time.
Treatment Options: Newly Diagnosed vs. When CLL Comes Back
Jeff: Let me complicate the matrix a little bit. It sounds like there are an awful lot of options and components that you’re crunching with your patients to get to the treatment route. Is there a difference between someone who’s brand new to the CLL game versus someone who’s relapsed or refractory? Does that add even more complication to this process?
Dr. Lamanna: For someone who might be getting treatment for the first time, the discussion may be a little easier in the sense that they’re a clean slate since they haven’t had any other therapy yet.
Relapse absolutely is different because partly what you do next will also hinge on what you did prior. Did you have any particular side effects from said treatment that you might have gotten as your frontline treatment? Has your disease changed? Have the genetics of your disease changed? Are there any more aggressive features that might change what we’re thinking about what to treat you with?
In the relapse setting or for somebody who’s had multiple therapies, there’s no doubt that we’re thinking about the kinetics of their disease, the patient themselves, their newer medical problems, side effects from what they got before, and how well their previous treatment did. All of these play a role when we talk about subsequent lines of therapy.
It’s an individualized approach to therapy. I’m not sure we’ll get a one-size-fits-all. I think that would be impossible because even with a good regimen, people can encounter side effects, so you always have to adapt according to the person in front of you. Relapse can be a little bit more complicated because we’re thinking about what that person has gotten before and how well they did.
Will CLL Treatment Affect My Fertility?
Jeff: Paul has a great question, which I wish would get asked more often because it’s important. “Do different CLL treatments affect fertility for those of childbearing age?”
Dr. Lamanna: There’s no doubt that a lot of the treatments may impact fertility. Chemotherapy was probably the biggest offender when we talked about that, but the TKIs, not so much. There’s a paucity of data in CLL only because the median age is older, but we have tyrosine kinase inhibitors for another chronic leukemia called chronic myeloid leukemia (CML) that have been used, and we’ve shown that, over time, pregnancy is safe.
Now it does depend on the drugs that you’re getting, so we do talk about that. People of childbearing age with CLL who are on therapy need to talk to their provider. Obviously, our ideal preference would be not to be in therapy if they’re pregnant. If the childbearing person who has the CLL is the woman, it would ideally be nice not to be on therapy while they’re pregnant. If it’s a male CLL patient, I don’t think we have data. There’s probably insufficient data to say if it impacts sperm and their ability to impregnate a non-CLL person.
When I think about counseling some of my patients, I have not had that as a barrier for them to impregnate as a male CLL patient. With women, I have more of a discussion. We talk about the risks of having CLL and the impact on blood counts, which will happen as a pregnant female with CLL, because they often become anemic, and it can change the white blood cell count. We talk about the timing of therapy as well. For a woman, it’s a longer discussion and we try to see if we can plan a little bit.
There’s animal data and we have anecdotal data of these therapies. I’ve had several younger CLL women patients who have had successful pregnancies and deliveries. That’s the good news, but there’s a paucity of data in this regard only because the disease impacts older patients and most aren’t having children later in life. You need to talk to your provider, but you can plan for families.
Building the Best Medical Team
Jeff: When we were doing the introduction, one of the things that we mentioned was team building. The National Football League (NFL), for all intents and purposes, is one of the best team sports out there. Even more important than that, as far as I’m concerned, is team building for CLL. The reason why I say that is that I help manage a Facebook community that has over 18,000 patients and caregivers.
Dr. Lamanna: That’s a lot.
Jeff: That’s a lot of folks impacted with CLL. Invariably, when they join us, the first question they ask is, “Do I really need to see a CLL specialist?” And everybody, almost in unison — very rarely on the same pitch — will say, “You don’t have to go to one; you just have to see one once. You’ve got to get a CLL specialist on your team.” You’re a CLL specialist.
Dr. Lamanna: It’s a biased question.
Jeff: It is a biased question. How can a CLL patient go about putting together the right team? Not everyone has access to a major metropolitan CLL center with CLL specialists. Is there a difference between being treated at a local community hospital versus a big cancer center? How do we put all this stuff together?
Dr. Lamanna: I’m a CLL specialist, so, of course, I’m going to be biased. I work with lots of patients who aren’t able to come to big academic centers or to the city, which makes sense. You should feel very comfortable with your local physician. If your physician is a community physician, you want to be able to have an open dialogue and be able to ask questions.
I agree that you should see a CLL specialist. No doubt, this is a rare disease, so most of the doctors in the community — and I am not knocking the community docs; they work hard — might see a variety of different diseases, so they may not know the most up-to-date information about CLL per se if they’re treating many diseases. I think that it’s good to see somebody once or at critical times, perhaps if you’re requiring therapy or getting therapy again. It’s always good to touch base with a CLL specialist and have them part of your team because they can be helpful. They can bring up treatment ideas that maybe the local physician hasn’t thought of or can partner with the local physician. I think that’s important.
For my patients who come from afar, if they’re in another state and they have an emergency, I need to make sure that they can get care very quickly, so I want them to have a local physician. If their physician is comfortable with me, where I can talk to them and they can talk to me, then that’s a good sign. If they’re intimidated and have a problem with me, then that’s a problem. I don’t have a big ego. Jeff, do I have a big ego?
Jeff: You do not have a big ego.
Dr. Lamanna: I am just a humble CLL doc who wants to take good care of my patients. That’s my drive. Not all the other stuff. If you can have a meaningful conversation with your physician and they’re willing to work with a CLL specialist at critical times, that’s important. There might be aspects of your case that are complicated or new treatment options.
It’s great to have a CLL specialist on your team and to know that your local physician is the one managing you on a day-to-day basis. You should feel comfortable with that. You need to have a good rapport.
Remember, CLL is a journey. As Jeff noted, he’s had this for years. You’re going to have your physician, hopefully for years, so you want to have a good relationship with them and their team because that’s the one that you’re going to go to all the time. It’s great to have them like a primary care physician in a way. They’re there with you for years. You get to know them and they know all about you and your other medical problems. You form this long-term bond.
It’s always important to include a CLL specialist. If you’re able to do that, that’s great because they can be important at critical junctures during your journey, like treatment discussions, unusual side effects, or complications. Sometimes patients can be very complicated. The community physician will be there for you all the time, so you need to have a good relationship with them. If they’re comfortable working with a specialist if and when needed, then that’s a good sign. That’s a great sign.
How Can Patients Facilitate Communication Between Their Community Doctor & A CLL Specialist?
Jeff: Keeping the NFL metaphor going, having a CLL specialist as your quarterback seems to work out pretty well. It’s been my experience that in this day and age, most doctors have absolutely no problem cooperating with other doctors. We’re not in the ‘60s and ‘70s, where the doctor was the center of the universe and there was absolutely no room for anyone else. It’s a relationship.
As you said, CLL is long-term. There are so many wonderful choices available now and many more coming in the near future. You have to have that long-term relationship. Let’s talk about what the patient can do to facilitate the quarterback and the local team player. What questions should the patients be asking? What should they be taking note of and keeping track of? What’s the best way for them to share test results and treatments with all the doctors? How do we put everything on one plate?
Dr. Lamanna: Forums like this and online communities, like the one Jeff manages, are very helpful to have a basic understanding of your disease. Try to understand what it is to have CLL and how to look at your blood counts. You don’t have to be a scientist or a professional. It’s all about having an understanding, so that if you want to look at your blood counts, you know how to interpret a little bit about what they could potentially mean. That will help when doctors are talking to you about where you are on your journey. Are you progressing? Are you stable? Are things fine? What’s going on? Understanding your disease is important.
When you go to a doctor’s visit, it’s good to bring somebody if you can because you may not hear everything. Your helper will also hear things so that later, if you’re wondering about whether a doctor said something, you have somebody else as another set of ears. I always tell patients that if they have questions, they should write them down and bring them in, so we can go over them. They can also send messages through the patient portal. Many hospitals and centers have portals, so you can message your questions to your team.
It’s always important to write them down because you won’t always remember when you go to the office. Maybe you just had your blood drawn, so you’re anxious, which is totally normal during a visit, but you might forget things. Always bring a list of questions.
Spend some time getting a general education about your disease with your team. There are some patients who don’t want to do a lot of reading online and that’s okay.
Jeff: It’s okay.
Dr. Lamanna: There are some patients who, for them, it could be very anxiety-provoking. If you’re not at the point where you want to hear other people talk about their CLL or their treatments, then don’t. The internet can be good, but it could also be bad because you can Google things, and it just spits stuff out. Because CLL is a very heterogeneous disease, patients may be at a different point in their journey than you are, so that could be very anxiety-provoking, too. If you’re not ready for all that, then pull back and keep it very simple with your doctor.
Usually, when I first meet a patient with CLL, I spend a good amount of time educating them about their disease, and I hope they feel that way. I also give them some general reading material and guidelines. I give them a printout about healthcare maintenance, vaccines, and other things that they may not remember, so they can go home with a little package after they first meet me.
It takes many visits to understand the disease itself, what it means to look at your blood counts and the lymph nodes, and things like that. I would advise people to write things down, bring questions, and bring somebody with them, so you have an extra pair of ears. Write other questions. After the visit, if something comes up and you’re not sure, you can ask your provider or team.
Now, let’s say you have multiple people involved. Jeff, you asked about how to share information with other providers or with the CLL specialist. Most hospital systems nowadays are doing well with working through systems. Many hospitals even cross-communicate. You can ask your physician, “Do you partner? Are you online? Can my CLL specialist see this through one of these hospital systems or not?”
They’ll know because they’ll say, “Oh, yeah, they can see me and I can see them,” so then that’s easy. You can message the team and say, “Hey, I had a CT scan and the results are online,” or “I had blood work done.” If they don’t, then it’s still the old-fashioned way of uploading or sending by fax. That works, too.
Jeff: They still exist.
Dr. Lamanna: It still exists. And that’s okay too, right? It depends on where you are, what kind of system they have, and if they integrate with other systems, so that information can be shared between the physicians.
That goes not just for your CLL, but for other medical problems. This is common. I was in the clinic yesterday and my patient had a CT scan for a completely different reason. I said, “Hey, can I have access to that CT scan? I’d like to look at it. I’d like to see how your lymph nodes are doing, too.” It provides another opportunity. They weren’t linked in, but I said, “Don’t worry. We’re going to have you sign a release, so I can have my secretary work on getting that scan.”
There are different ways to communicate with the teams. You just need to ask your doctor. “Can my doctor see this through your system or not? If not, can we make sure that they get a copy of it?” Or you can upload yourself. A lot of my patients are very tech savvy, so they upload the lab results and reports into their own portal, so I can see them, but you don’t have to do that. You can have the physician’s office do that, too, if you’re not so savvy or can’t do that. You just need to request and then everybody can see the information.
Jeff: As far as what the medical teams need to know, I’m going to have to underline one particular plan of action for all male CLL patients. When you walk into the doctor’s office and the provider asks how you’re doing and you say, “Fine,” please bring your significant other, because “fine” is not the correct response. Your significant other will absolutely underline that you’re not fine. X, Y, and Z are going on along with ABC and one, two, three. We need to get this information in the hands of our medical providers so that they can be responsive to our CLL.
Dr. Lamanna: If you’re a non-talker, it’s always good to bring a partner if you’re not going to talk, as they know what’s going on with you. We always joke around because then the partner or spouse will start bringing up other stuff. They say, “You see? I’m in trouble. They’re talking about everything.” That happens.
Jeff: You just have to go with it.
Understanding the Roles of Your Team and Patient Advocacy Groups
Jeff: We talked a little bit about medical providers. What role do social workers, patient advocacy groups, and other support systems play? How does that work with the CLL care team? I mentioned 18,000 people on the CLL Support Group on Facebook. There are dozens of CLL groups on Facebook and throughout the country. How do these support roles fit into this program?
Dr. Lamanna: Let’s first talk about your medical team and then about the outside support. Besides the physician, many of you have probably already interacted with a nurse, a nurse practitioner, or a physician assistant. They’re part of the medical team, per se. They’re also very good at handling and putting out fires.
My NP is great at education. She’ll do educational sessions before somebody starts treatment. They do a whole video visit to educate about the drugs and side effects. They’re part of your medical team, too, and often may respond for the physician, depending on what’s going on.
We also have social workers who can get involved, depending on the person’s needs. Not everybody needs a social worker, but some people do. They have questions that are not necessarily medical but are tied in. I get them involved if I think that the patient needs extra resources. Sometimes that could have to do with financial resources or other social resources that they might need. The social worker can be very helpful with paperwork or facilitating other means that might help the patient if they’re on treatment, where they may need other support besides medical. Most practices, whether it’s academic or community, have a social worker.
Before we get outside the medical team, other things could be available to the practice, so it’s something you should ask your physician team. There are psychologists and psychiatrists. There might be other services in the medical practice, like nutritionists, art therapy, or music therapy. That’s something that you can ask during the first visit. You may not need those services right away, but maybe it’s something to think about.
Ask the practice what other internal resources you could take advantage of, so that if you need them, they’re available to you and the physician can get them involved as well. It’s important to know that there are internal groups. Sometimes people need psychiatric or psychological counseling to help them initially with their new diagnosis, besides talking with their physician or provider. Or maybe you feel uncomfortable dealing with just your partner because it feels too stressful, so you want someone else who can hear you out. There’s nothing wrong with any of these.
Then, as Jeff noted, there are external resources, like organizations and groups that have these forums. But also, we have the Lymphoma Research Foundation, Blood Cancer United (formerly The Leukemia & Lymphoma Society), and many online groups with resources available to patients that they could take advantage of, depending on their needs.
I give a list to my patients, even if they don’t need them right away, so that they have them already. These are things to bring up to your physician. If there’s something that you think you’re not getting assistance with, your physician can tap in or tell you where to go to help make your life a little easier. There are internal and external resources for CLL patients and for cancer patients in general, and patients may not know how to take advantage of them. If there’s something that you feel isn’t medical and you’re not sure if you could ask your doctor, bring it up with your physician. There are a lot of untapped resources for leukemia patients.
Clinical Trial Options for CLL
Jeff: At the beginning of this presentation, we talked about the exciting treatment options available and in the works. We’re now going to talk about what I think is the most important part of CLL: clinical trials. The reason why I think that is because without them, we wouldn’t have any of this new cool stuff to use for our CLL journeys.
I participated in a clinical trial more than a dozen years ago and I got great results from it, even though I’m currently relapsed. Without the knowledge that was gained from that clinical trial, we wouldn’t be able to build on the new stuff. What clinical trial options are available for CLL patients today and how do they fit into their personal treatment landscape?
Dr. Lamanna: I want to thank you, Jeff, for participating in a clinical trial; kudos and thank you. There’s a big misconception out there about clinical trials and, as Jeff noted, we can only move the field forward and bring new agents to patients through clinical trials. Patients get very fearful about clinical trials because they think they’re experimental and they’ll become a guinea pig.
There are different types of clinical trials, which are very important to note. There are trials where the drug hasn’t been studied, meaning it’s the first time it’s going to be in a patient, so we don’t know the dose yet or the potential side effects; that’s a phase 1 clinical trial. Usually, the patients who participate are those who have received all forms of standard care and other agents, so somebody who’s very relapsed and we’re looking for newer options for them.
Then some trials are further along in development, meaning we know the dose and side effects, they’re looking promising, and we’re moving them up in development. They’re expanding to see how well it works, what the results are, or compared to a standard of care treatment. Is this better than our current treatment? These are usually done on patients who are less heavily pretreated or newly diagnosed, where we’re comparing it against a standard of care and we think it might be better.
There are different types of clinical trial options. As a patient, how do you figure that out? Do you have access to clinical trials? Depending on where you’re at, your team may or may not have access to clinical trials or run clinical trials, so that’s one question. If so, can they partner with other places? Are you interested? This is where a CLL specialist is important because there might be a good option for you, depending on what you’ve had and where you are in your CLL journey, that perhaps a clinical trial could be a good option for you. You won’t know unless you inquire.
Now, clinical trials aren’t for everybody. Being in a clinical trial means you’re going to commit to more than you would normally. Jeff can probably tell you this better. Many clinical trials will do more blood work, more imaging, and more checking in because they’re following patients very closely to look at responses and side effects. They’re going to do more than they would usually as standard of care, so you have to be comfortable with that.
The first question is what clinical trials are available to you, so you want to talk to your provider about access to clinical trials if and when available… and if you need it. There are also ways that you could do online research. There are websites that talk about clinical trials, like ClinicalTrials.gov. Clinical trials change all the time. We open and close studies all the time, so what might be running at one point in time may no longer be open. We might be done with that trial and have moved on, so that’s important, too.
If you need treatment, whether it’s your first or subsequent lines, do you have any access and what are those clinical trials? It’s a good time to talk to a CLL specialist about that and your provider. If your provider has access to trials, then you could sit down and talk about them.
Usually, the provider is trying to make sense of each individual. Not every trial is right for every patient, which is very important. Some trials are very appropriate for a patient, depending on where they are with their disease, but not for another person. Your provider is supposed to help guide you based on where you are with your disease, what the best options of treatment are, and how a clinical trial might enhance that aspect or not.
It’s an important and critical time to talk to somebody if you’re interested in clinical trials because it might provide you with the next best therapy. I think that was what Jeff was getting at. It might provide a lifeline. If you’ve had all these other treatments, where do you go next?
Jeff: I love the way the clinical trial was presented to me. I was told that my participation would get me earlier access to the best stuff. We know that this is going to be great and this will get you quicker access to it, and I liked that approach.
Dr. Lamanna: Absolutely. Even the drugs we now consider standard of care were in clinical trials at one time.
We have to talk about the side effects or potential side effects. This is necessary whether the treatment is a new drug or part of a trial that compares a standard of care with a newer drug whose side effects we already know. We have to talk about all potential side effects, regardless of whether you’re on a clinical trial or not.
Obviously, with earlier phase studies, we may not know all the potential side effects of a newer treatment, especially if something has never been used in humans. But usually with more advanced clinical trials, we pretty much know a lot about the side effects and we need to talk about that. That’s what informed consent is all about. You might have other medical problems, so how does the treatment fit with your medical problems and other medications? You need to have an open dialogue about whether it’s right for you.
Again, whether it’s a part of a trial or not, we should be having that discussion. That’s informed consent, even if you’re not on a clinical trial. We need to talk about the side effects, so you’re aware. “If I have a side effect, who do I call? What’s the dialogue going to be when I’m on treatment, so that I can get access to you and your team and they can help me with my side effects? Should I stop the drug? Am I going to amend?” Whether you’re on a trial or not, it’s important to understand the potential side effects when you’re on treatment.
Remember, those commercials on TV where they talk about a drug and you hear all the side effects? Even with taking aspirin, there could be a bad outcome. Somebody could have died from taking something as simple as an aspirin. Informed consent is important, but the doctor needs to tell you the frequency and commonality of those side effects, so you can make sense of them and not panic. Otherwise, nobody would ever take treatment. Ever. You have to make sense of them, work with the team, and talk to them.
The clinical trial process is lengthier for sure. There’s more work to be involved in one because you’re going to be seeing the team much more often and doing much more. You’ll have more blood work and potentially more imaging, so you need to be comfortable with that.
Exciting Areas of CLL Research
Jeff: We’ve laid out clinical trials and I know you do a lot of work with clinical trials. I’m going to ask you to pull back the curtain a little bit. Tell me what’s exciting. What’s the coolest stuff that you’re working with in clinical trials right now?
Dr. Lamanna: As I noted, we have BTK inhibitors and a BCL-2 inhibitor, venetoclax (Venclexta). There are newer classes or newer mechanisms of action that we’re very excited about, which I hope will lead to newer FDA approvals in the near future.
BTK inhibitors bind to the Bruton tyrosine kinase protein. These are ibrutinib (Imbruvica), acalabrutinib (Calquence), and zanubrutinib (Brukinsa). We even have a newer one called pirtobrutinib (Jaypirca), which binds a little bit differently to BTK.
What does that mean? Unfortunately, these drugs are not curative, so patients can develop resistance to the site where they bind to BTK, all of these drugs. Eventually, people may develop resistance to their therapy. BTK degraders bind very differently to BTK, which is very important in the survival of CLL cells. It binds the whole complex and it degrades and gets rid of the whole complex differently. Theoretically, it could get around the resistant mutations that are on some of these BTK inhibitors, where they bind to one site and then the protein changes, so they no longer are effective because now they’ve developed a mutation at the site that they can’t bind to anymore.
The BTK degraders will bind as a complex and they’re looking very promising in clinical trials. There are several of them that are in development. Again, we usually start new drugs in patients who have received prior treatments that are working well for them. These trials are being done in patients who have had relapse or multiple lines of pretreated CLL.
The responses are looking encouraging. Trials are looking at them by themselves, but there are also going to be trials that will look at them in combination with other agents or in less heavily pretreated patients. I’m hoping that’s a different mechanism of a drug that will be available at some point in the future. We still need some time. It’s very early. That’s one very exciting group of agents being looked at.
Another group of agents that is very exciting is called bispecific monoclonal antibodies. Like rituximab (Rituxan), which I told you guys about earlier, or obinutuzumab (Gazyva), which binds to CD20 on your CLL cells, the bispecific antibody binds to one of the markers on your CLL cells and that could be CD20 or CD19, but it also binds to your T cells, so that’s why it’s called “bi.” The goal is to bring your T cells in close proximity to your B cells and try to target and kill these CLL cells.
They are also very encouraging in clinical trial development for CLL. They’re approved for use in other lymphomas, but show encouraging results in CLL. There are many clinical trials looking at these bispecifics either as a single agent or in combination with other drugs that are already approved for CLL.
These are going to further enhance what we can do for patients who get current standard of care drugs, like BTK inhibitors and venetoclax (Venclexta). If they relapse and need more therapy, they’ll hopefully have newer agents to choose from through the development of these clinical trials with BTK degraders and bispecifics. It’s very exciting.
There’s more I can talk about. I can go on and on and on. But those are some of the more promising ones in development that I’m very excited about. There are a lot of clinical trials surrounding those mechanisms that are going to be very important.
Jeff: Very cool. There are so many options available to us. When I first started this, there was plan A and plan B, and that was it. You were going to get either/or. Now we have to use up the entire alphabet.
Quality of Life and Treatment Decisions
Jeff: From what I’ve seen over the past few years, quality of life has become one of the most important components in this decision-making matrix. How do you personally help your patients navigate through this complex matrix? Let’s talk about how quality of life impacts decision-making.
Dr. Lamanna: Let’s talk about people who aren’t on treatment. We talk about the quality of life when they’re off treatment. It doesn’t mean that you may not have any symptoms from your disease. Sometimes these are harder to quantify, like fatigue and other things like that. It may be difficult to say this is just your CLL, CLL and other things, or a combination of CLL and other things that are going on in your life. We talk about patients who aren’t on treatment, getting exercise, going out, enjoying life, having a quality of life, and looking at it as a chronic disease that you currently don’t need treatment for.
I don’t like the term “watch and worry.” I think it’s a terrible term. We need to get rid of that because, yes, there’s anxiety and we’re not taking away that component. But if you don’t need treatment for your disease, it’s good to be on the sidelines because you get to see the development of all these treatments that may replace current treatments and potentially have fewer side effects. You might get different options that might be better.
Remember, all treatment has potential side effects. If you don’t need treatment, why get treatment? It’d be different if we had a treatment that cured everybody and then we wouldn’t be having this conversation. But until that occurs, until we prove that starting somebody on treatment earlier is going to do better for the group as a whole, or that it extends survival, we wouldn’t do that. We haven’t shown that yet in clinical trials, so this is why not everybody needs therapy and may not need therapy for years
The goal is to talk about their quality of life when they’re off therapy and if they need help with some of those issues. When you’re first diagnosed, there’s a lot of anxiety, so that’s understandable. Quality of life is one piece.
For people who are in therapy, it’s another discussion about quality of life. We talk about therapies, the side effects of those therapies, and how they relate to what’s going on in their lives at the time. We have great options and you can go from one to another in the future. You can go from a BTK inhibitor to venetoclax (Venclexta) or the other way around.
We talk about how a particular treatment option at the time may impact their life, depending on where they are in their life. Are they having kids? Do they have a young family? Are they working and traveling a lot? Those are important. You need to talk with your provider because these factor into what treatment option you might choose at the time.
Fifteen years ago, I had a very young patient who had a young family and was constantly working, so he chose to do a BTK inhibitor because doing other therapies was more complicated at the time. He said, “It’s easy for me to take a pill. I’m going to do that.” Believe it or not, he’s still on a BTK inhibitor and doing great. Those are some of the discussions you have with your provider because where you are currently in your life may feed into the side effect profile of the drugs and what you may choose, so that’s important.
In general, for all patients with CLL, we talk about the other stuff. We talk about diet, exercise, and even psychological counseling and support, if needed. People ask a lot of questions about food and healthy diets.
Other medical problems feed into this, so we talk about their other medical problems and how to manage them if they have other chronic health problems. CLL is chronic, like other medical problems, which is why you need to think about it. Like people with diabetes, heart disease, and high blood pressure, CLL is a chronic condition. That should give you hope because people are living very good quality of lives with their other medical problems, but they need to be well-managed, so sometimes, we need to partner with your other healthcare providers.
I do think, Jeff, it’s an important concern, particularly because we’re getting better at this. We have a long way to go, but it’s important to talk about it since there isn’t one option now. Survival continues to improve and gets better. There’s no single choice where this is your only choice and what you have to do, so discussions on quality of life become important.
How Do I Talk to My Doctor About Symptoms and Side Effects?
Jeff: I want to focus on quality of life a little bit more, if you’ll indulge me. This is part of the communication process that patients need to be hyper-aware of. Medical professionals can’t help you if they don’t know what’s going on. If you’re feeling tired all the time, don’t just deal with it. Let your medical providers know that you’re tired and that it’s more than just being tired after a workout. I’m tired all the time. If you’re getting sick all the time, you need to let your medical professionals know about this.
At the same time, if this is weighing heavily upon you and you’re dealing with stress to the point where it is making life difficult, you need to communicate that to your medical professionals because they can help you. This is part of the treatment matrix and part of deciding how to treat your CLL.
I know that you deal with this with your patients on a very regular basis. I want the patients and their caregivers to know that it’s okay to talk about this. This is part of the shared decision-making that must go on. Would you agree with that?
Dr. Lamanna: Oh, absolutely. Importantly, sometimes some of the symptoms, even though you think there’s nothing you can do about them, may be more than that. There may be something. If somebody tells me that they’re tired to the point that it’s inhibiting their daily activities and quality of life, then I will work that up because maybe there’s another reason that they’re tired. Maybe it is related to their CLL. Even if their numbers are okay, I might have to factor that in if they are debilitated, then we do have to talk about whether or not that means embarking on therapy earlier. Because somebody’s not textbook and their counts aren’t terrible, is there something else going on?
There’s a medical intervention potentially that needs to be worked up, or maybe there are other things going on and there’s a different diagnosis or support needed because of whatever symptom. Jeff’s absolutely right. It’s important to tell your provider and healthcare team what’s going on because it could potentially have some ramifications for your treatment or your medical care that you’re not even aware of. They need to work it up and say, “Okay, this is this, and this is why you’re feeling this or not.” They need to make sure that there’s nothing related and there’s an intervention potentially that could help you.
Infections are a big thing. I want to talk about this. Some providers want to know if you’re constantly sick and some providers might say, “I want you to call your internist.” This is a unique thing. I have my people call me because if somebody’s always sick all the time, is it because their immune system is run down? Do I need to factor that in with where they are with their CLL?
Patients who are always sick may need extra assistance, whether they need gammaglobulin or help. Maybe they’re getting sick all the time because their disease is slowly progressing, so maybe we should be treating them sooner. It’s important to share what’s going on with your providers and healthcare teams, so they can help navigate and see if it’s medical or not. Is there another diagnosis? What can we do to help support you through the issues that you’re going through?
Why Isn’t My Doctor Starting Treatment Right Away?
Jeff: I’m going to deliberately avoid the W words for what we do with CLL patients and use the totally stilted phrase “mindful surveillance.”
Dr. Lamanna: I say active observation and monitoring, but active surveillance is great. I use active surveillance too.
Jeff: Just justice tilted, in my opinion. Lee’s got a great question. “Is it a good idea or should I talk to my doctor about other options? Why do we have to wait? Why can’t we just go knock it out now?”
Dr. Lamanna: You’re referring specifically to why I can’t treat it right now, as soon as I’m diagnosed.
Jeff: Exactly.
Dr. Lamanna: As I alluded to earlier, we don’t have curative therapy yet. What we have shown, or at least run several clinical trials on, is that starting earlier treatment in CLL didn’t benefit the patient. There were increased side effects from the therapy and it ultimately didn’t improve survival. If we start therapy, we’re giving you treatment with potential side effects and not improving the outcome. That was one of the major takeaways.
But the other major takeaway is if you’re younger — this may have less relevance for older patients — we also know that when you start treatment, there’s always the potential of developing resistance to therapy.
Most of my patients are extremely nervous about their first treatment and all that goes with the first treatment, like the side effects. I’m thinking of not just your first treatment, but also your next treatment, the treatment after that, and the treatment after that.
I’m forecasting as your CLL doc in the future. When I look at you, this particular person with CLL, how do I get you to have a normal lifespan? How do I forecast the drugs in the pipeline, the drugs that I need to know, and how to get you there so you can enjoy all the life stuff? We know that if you don’t need therapy, we can potentially start the clock with resistance. I worry about resistance patterns and the ability to use those therapies later on down the road.
Until we prove that starting treatment earlier would benefit you, we will not change that paradigm. The one other cancer I can liken this to is prostate cancer, because many patients with prostate cancer also don’t need treatment if they have low-risk disease and are monitored for similar issues. It’s unusual because when we think about cancer, everybody thinks about how to get rid of it and be done with it.
We don’t have that yet in CLL because it’s technically not curative, although that’s also a very loaded term. There are patients whom I have treated 20 years ago and have never needed treatment, and for all intents and purposes, they may be cured of their CLL.
In general, we know that CLL is a chronic disease and can come back, so we think about that and about issues of resistance when we talk about treatment and somebody starting treatment. That’s why we don’t start everybody on treatment as soon as they get diagnosed, because we haven’t shown a benefit yet. We don’t want to take out potential agents that we need later on when you do need treatment.
Jeff: It does. Dr. Lamanna is always thinking three and four moves down the range.
Dr. Lamanna: I have to.
Jeff: Note to self: don’t ever play chess with her. Not a problem.
Conclusion
Jeff: I loved having this conversation with you. When I have these types of conversations, I do get excited. All of these choices that are in front of us right now are here because of clinical trials and because CLL specialists have made the deliberate effort to learn more about CLL. We have more choices today and we’re going to have even more choices tomorrow because we keep on researching.
I find comfort in the fact that as long as I’m on active monitoring, the longer I wait, the more likely it is that a drug that’s going to be developed will be the right one for me and that it could cure me… and that would be very cool.
Dr. Lamanna, I want to thank you for sharing your expertise with us. I love having these conversations with you. You’re a ray of sunshine in literally what can be a very depressing journey for a lot of people. I’m glad that you are so dedicated to making sure that people with CLL live great lives.
I want to thank AbbVie for its generous support of this program. The support helps us to host these educational programs for free. The Patient Story maintains full editorial control of these programs.
We hope that you find this helpful. Remember that this program is not a substitute for medical advice. We hope that it will inspire you to make better decisions and have better discussions with your medical team.
We want to hear from you to make these discussions even better. What would you like? What can we improve?
No matter where you’re treated, your voice matters. Ask the questions. Explore the options and remember that you’re a key member of your CLL care team.
Thank you again to AbbVie for its support of our independent patient education program. The Patient Story retains full editorial control over all content.
Why Clinical Trials Matter: How New AML Treatment Options Saved Shelley’s Life
Just after celebrating her 70th birthday, Shelley began feeling unusually tired on her walks. Her heart raced and she couldn’t go half a block without needing to rest. Concerned, she saw her doctor and soon learned she had chronic myelomonocytic leukemia (CMML). The diagnosis was frightening, but her daughter’s constant support helped her face it head-on.
Interviewed by: Taylor Scheib Edited by: Katrina Villareal
Months later, Shelley sensed something wasn’t right again. Her blood tests confirmed it: her CMML had transformed into acute myeloid leukemia (AML) with an NPM1 mutation. It was a devastating moment, yet she stayed determined, crediting her daughter and her care team for keeping her strong.
Shelley went onto chemotherapy in preparation for a stem cell transplant but her AML did not respond well. That is when her doctor approached her about clinical trials because her leukemia had the NPM1 biomarker and she decided to take part in a clinical trial designed for AML with that tumor marker. She appreciates the importance of research and data, and as importantly, a clinical trial means something deeply personal: hope for herself and others. She takes part knowing that her participation helps shape future AML treatments.
Shelley’s clinical trial participation lead to her being approved for a successful stem cell transplant. She now advocates for a better understanding of clinical trials. She reminds others that these studies don’t withhold treatment for people with active disease. Instead, they offer real opportunities for medical progress. Shelley believes her survival came from three things: her daughter, her medical team, and her clinical trial.
Today, she’s grateful for every day she can return to her music and live fully. Shelley’s story shows that knowledge, research, and hope can come together to make a real difference for people living with acute myeloid leukemia.
Key Story Takeaways
Shelley’s honest look at life after an acute myeloid leukemia diagnosis
How knowing her tumor’s genetic makeup helped her enroll in a clinical trial, making a stem cell transplant possible
The truth about placebos and real treatment in clinical trials
How research and resilience helped Shelley return to her music
Name: Shelley G.
Age at Diagnosis:
70
Diagnosis:
Chronic Myelomonocytic Leukemia (CMML) that progressed to Acute Myeloid Leukemia (AML)
Biomarker(s):
NPM1 mutation
Symptoms:
Fatigue
Rapid heartbeat
Shortness of breath
Low blood counts
Treatments:
Chemotherapy
Stem cell transplant
Clinical trial (oral medication)
Thank you to Kura Oncology for supporting our independent 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 to make informed treatment decisions.
The views and opinions expressed in this interview do not necessarily reflect those of The Patient Story.
I am Shelley. I was originally diagnosed with CMML just after my 70th birthday in August 2022. After more testing, by July of the following year, my diagnosis evolved into AML. There was a bone marrow biopsy in July 2023 that confirmed it. That’s the initial part of my diagnosis.
My work is my biggest passion
I’m most passionate about my job. I feel very lucky to have a great job—it’s very interesting. I work for a labor union that represents educational employees in Illinois. I’m assigned to a specific geographic area, covering about fourteen small local unions—teacher unions, support staff, custodians. I help them with all sorts of issues, mainly collective bargaining. I prepare and conduct trainings, sometimes specific (like First Amendment rights for educational employees). I am a lawyer by background. I’m extremely passionate about my work and can’t imagine not wanting to do it. I learned, when I was on medical leave, how bored I was without it.
My life outside of work
Aside from work, I do have outside interests. I’m interested in music. I play an instrument, though not a virtuoso, and have done photography for many years, with a lot of my photos on Flickr. Since being diagnosed, I haven’t taken many pictures or had time to use my big Nikon camera; I started using my cellphone camera, which really isn’t adequate for good photography. I’m also interested in politics (local, state, federal), history, and, strangely enough, economics. I like to read crime novels, mysteries, and watch movies- anything that gets me thinking.
My musical journey with the hammered dulcimer
The instrument I play is pretty ancient: it’s called a hammered dulcimer. Most people haven’t heard of it. It’s actually the great-great-grandmother of the piano. The Italian inventor of the piano adapted the mechanism from instruments like the hammered dulcimer, replacing pluckers with mechanical hammers. Most people don’t know the piano is actually a percussion instrument—a melodic percussion instrument.
I thought my leukemia symptoms were maybe a heart condition
After my 70th birthday in August, I was excited and celebrated with a surprise birthday party. A month later, in September, I noticed my heart would race and I was exhausted after walking only half a block. I thought it was a heart problem, so I saw my doctor. He ran tests, found nothing wrong with my heart, but my blood counts were dangerously low. He told me to go to the emergency room for a blood transfusion immediately.
My CMML diagnosis
At the hospital, they did more tests and soon a doctor told me, “We think you have leukemia.” They needed more testing, including a bone marrow biopsy, and scheduled follow-up appointments. I was released from the hospital and soon found out for sure.
After the doctor visit, Shelley was diagnosed with chronic myelomonocytic leukemia (CMML).
It was pretty scary. I thought, “Oh, no. What’s going to happen to me?” If my daughter weren’t there to say we were going to get through this, I don’t know what would have happened. I would have been pretty much at a loss. But she was there to back me up, so I bucked up and proceeded with the initial treatment.
Over time, Shelley and her care team began to grow concerned about her blood work again.
I tracked my numbers carefully and noticed my hemoglobin and platelets were going in the wrong direction. I thought that it wasn’t right, so I sent MyChart messages to the doctor through the portal. I asked, “Do I need to be concerned? I think I need to be concerned.” Finally, he agreed and said, “Yeah. You do need to be concerned.” So they scheduled another bone marrow biopsy.
Her CMML transformed to acute myeloid leukemia (AML), which happens in about 15 to 20% of patients over five years.
Emotions upon learning my AML diagnosis
I was very distressed and freaked out. It felt like a death sentence. The only image I had of leukemia came from the tearjerker movie “Love Story,” with these people who were madly in love and it was all very sad because eventually she got leukemia and she died. So that’s all I could think about. But my new care team gave me hope, and things started to change for the better.
I’m a lawyer, so I like to win
I don’t give up easily. I’m a lawyer, so I like to win.
I would say that one of the things that kept me going, more than anything else, was my daughter. I have to stay alive for my daughter. She has no brothers or sisters and no cousins. Her father died in 2017. I need to stay alive for her. And I want to stay alive for myself because I like living.
A clinical trial helped me prepare for a stem cell transplant
My doctor—the friend’s neighbor, a stem cell expert—said my best chance at survival was a stem cell transplant. In September 2023, I went on medical leave for work and started chemo in the hospital, but it didn’t work well. My leukemia doctor then offered a clinical trial, which I accepted immediately because I thought, “Why not?” Over a period of time, including several hospital stays, the clinical trial seemed to work well enough that they cleared me for the stem cell transplant. I underwent the procedure late February, stayed in the hospital for six weeks, and went home at the end of April 2024.
Clinical trials are crucial for science and medicine
I was aware that there was such a thing called clinical trials, and I always thought, “That’s great. That’s fantastic. Hopefully, it will save my life and help other people because of the data.” I’m familiar with data. I know they’ll be collecting data, and if this drug works, then it’s all going to be part of getting it eventually approved by the FDA.
The importance of understanding clinical trials
Clinical trials are absolutely necessary to develop any kind of cure or treatment to prolong life. I believe there are several factors that saved my life: the clinical trial, my daughter, and a great team of doctors.
If, God forbid, the person who has a problem understanding clinical trials should ever have a loved one who is sick with a terrible disease, there would be a real treatment plan for them because of a clinical trial. It’s all about saving lives. It’s all about eliminating, minimizing, or mitigating misery.
I’ve had this question asked of me: how do you know you’re not in the group that’s getting a placebo? That’s not what happens in a clinical trial. It would be medically unethical. They can’t do that. That’s not how clinical trials work. That’s for a different kind of research. You don’t tell someone with active disease, “I’m going to give you something that may be a sugar pill and, well, we’ll see what happens.” No, that’s not how it works.
Editor’s Note: In cancer trials, no one is given only a sugar pill when an effective standard treatment exists. Instead, participants typically receive either the current standard-of-care treatment or the standard-of-care plus a new therapy being studied, and everyone is closely monitored for safety and benefit. Many of the oncologists we interview describe cancer clinical trials as “getting tomorrow’s medicine today.”
Clinical trials are absolutely necessary to develop any kind of cure or treatment to prolong life… clinical trials saved my life.
Shelley G. – AML Patient
She’s devoted herself now to helping people understand how her clinical trial actually works.
I take three pills a day and I have a diary where I have to write down information. This is the data collection part. They prefer you do it at the same time of day if possible, but you have to do it within at least 12 hours. Sometimes it’s not perfect, depending on what time I wake up, but there are certain rules.
As Shelley looks ahead, she’s grateful for her care team and her treatment, which she hopes will help keep music in her life.
I don’t ever want to relapse. I always want to be on this medication because I want to live.
You can survive. Don’t give up. You have to be a fighter. You have to fight for a lot of things in this world. You need to be here for yourself and for other people, so fight on.
Living with hope and gratitude
I guess hope is a part of my vocabulary, even if I don’t think of it that way. We all have concerns about the world and the future, but for me, I focus energy on moving forward and helping others feel hopeful, too.
For anyone with AML or cancer, my advice is: with the right doctors, the right team, and the right treatment plan, you can survive. Don’t give up. Be a fighter for yourself and others. Fight on.
Thank you to Kura Oncology for supporting our independent 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 to make informed treatment decisions.
The views and opinions expressed in this interview do not necessarily reflect those of The Patient Story.
Love, Stress & Strength: What It’s Really Like to Be a Cancer Caregiver
Jamel Martin is a devoted son and caregiver for his father, Joseph, who was diagnosed with prostate cancer. When he first learned about the disease, Jamel researched and turned to his family and church community for strength. Joseph’s diagnosis brought them even closer, reminding Jamel of the importance of time, communication, and faith through uncertainty.
In this cancer vlog, Jamel and Joseph open up about caregiving, emotional healing, and what it means to support one another through fear and recovery. They share how community, love, and open conversation can help men overcome stigma around going to the doctor and seeking help. Their story is one of patience, gratitude, and the power of family connection.
Thank you to Pfizer for supporting our independent patient education content. The Patient Story retains full editorial control.
This interview has been edited for clarity and length. This is not medical advice. Please consult with your healthcare provider to make informed treatment decisions.
The views and opinions expressed in this interview do not necessarily reflect those of The Patient Story.
Jamel: My name is Jamel Martin, and I’m a caregiver for my dad, Joseph Martin.
The Diagnosis
Jamel: When I first found out, I didn’t know much about prostate cancer until they made me do a little research. I also started asking my mom to find out more about it. It doesn’t necessarily mean it’s detrimental. Once I found that out, I was a little bit more okay.
Joseph: I’m a happy-go-lucky guy. When something goes wrong with me, I try to uplift myself and not let things bring me down. I get back up and start all over again.
Learning About the Cancer Awareness Network (CAN)
Joseph: Once I found out that I had prostate cancer, we had Minister Loretta Herring at the church, who was with the Cancer Awareness Network. I got involved with her. She was one of my backbones and helped me walk through it and everything.
Cancer Awareness Network (CAN) is a non-profit community organization based in Birmingham, Alabama. CAN strives to empower cancer patients, survivors, and their caretakers. The organization has played an important role in the Martin family’s cancer journey.
Jamel: We have always been close and tight-knit. But going through that time made me value his life more because I realized that he wasn’t always going to be here with me. Any given day, he could be gone, and all I’d be left with are memories and reflections on what he taught me and instilled in me as a child. I take it wholeheartedly to try to spend more time with him. Once you grow older and have your own family, you tend to spend more time with your family, but you still need to make time for your parents as well. It helped me think about those things more, too.
Life as a Caregiver
Jamel: It was basically things that I was taught, like helping to take out the trash, cutting the grass, and cleaning the cars. I was helping move different things around for him that he couldn’t do. As a caregiver, what I was doing for him wasn’t as extensive as giving him a bath and picking him up from the tub. It was more about helping him around the house, so it wasn’t that much of a burden or very hard for me to turn into the role of a caregiver for him. Uplifting him was the hardest part. Keeping him happy and in a happy space.
Through Joe’s cancer journey, Jamel didn’t carry the weight of caregiving alone. He says his mother, a nurse, stepped in as Joe’s primary caregiver.
Joseph and His Wife Reflect on Their Shared Journey
Joe: You know what? I learned to be patient and to do what you ask me to do when you ask me to do it. I stopped being so stubborn and hardheaded. It all worked out well.
Wife: Anytime you work together, teamwork makes the dream work. We worked through the healing process, other side effects that occur with the surgery and how you dealt with them, and tried options for impotence and how you felt about that. We started out saying we’re going to get through it and that’s what we did. But still, we’re going to watch and follow up with the primary care physician annually. You always have to tell me when something is different about your body. You have to let me know.
I would say a lot of Black men don’t go to the doctor. However, I know you had to go because you didn’t have a choice but to go to find out what was going on. I think a lot of men are afraid to find out what may be going on and what the outcome will be. Just like your recent hospital stay, you didn’t know what the outcome was going to be and what was causing your problems.
Joe: That’s why I went.
Wife: You had to go. [Laughs] However, I think that men as a whole are afraid to find out the true diagnosis and how it would affect them.
Joe: But, yeah, they don’t go to the doctor.
Wife: As you get older, it’s important that you take care of yourself because the key is in early detection. If you see something going on, you have to get it evaluated.
Becoming More Health-Aware After Dad’s Diagnosis
Jamel: Most men don’t think about going to the doctor. They don’t want to go. It made me aware of my health by getting checkups on my heart, my prostate, and everything. It made me aware of how cancer can hit you at any age and how it can hit anybody. It doesn’t discriminate on race, religion, or anything.
I think the stigma is finding out the worst that can be going on with you. It’s scary because we’re not taught about those things health-wise. It’s not talked about. When you’re thinking about having something going on, you don’t know how to react to it. The unknown scares you. You’re scared of what can happen and what’s going on that you don’t want to find out. But that’s not the case. You need to find out so that you can hurry up, jump on it, and attack it.
A study of cancer caregivers showed that they deal with a multitude of different stressors. 50% reported high emotional stress connected to the caregiving role, 25% reported high financial strain, and 72% helped with medical-related tasks, all for an average of two years of caregiving. (Source: National Alliance for Caregiving)
Jamel: I can relate to that statistic, especially emotional stress. It was a very emotional time because I didn’t know what was expected and what was happening.
When you see him, you can’t tell. He’s a happy-go-lucky guy, so he’s always smiling and always happy. You can never tell what he’s going through on the inside. But I’m a bit more emotional. I still hide it, but it’ll come out now and then, which was what I had to deal with.
How Cancer Affected Their Marriage and Intimacy
Wife: I was concerned about how you would heal or what the result would be as far as intimacy, which was somewhat affected, but we worked through that as well. A relationship or a marriage is not entirely based on intimacy. You still love each other, and if true love is there, the relationship will remain.
Joe: I agree.
Wife: You’re a good support person for me. We support each other.
Joe: I was about to say. We do that for each other, whatever the case may be.
Wife: We have our differences. We don’t always agree.
Joe: No, we don’t.
Wife: It’s better to express your feelings than to assume what someone is thinking.
Joe: True.
The Power of Communication and Emotional Support in Caregiving
Jamel: Being able to communicate and talk with them about anything they want to talk about during that time is best to keep them emotionally sound.
Take your time. Be patient with the loved one that you are caregiving for and help them embrace life. Lift them up. Bring joy and peace back into their life. They’re going through a lot during this time and a lot of things can be very emotional for them.
It’s a hard time to go through because they don’t know what’s going on. They don’t understand what’s going on with their bodies. They know it’s different. It feels different. But they don’t know what to do about it at the time. They may want to simply vent. Let them vent. Let them talk to you. Hold their hand while they’re talking to you. Let them know that it’s okay to cry. They can cry. Give them a shoulder to lean on. That’s what I give them. Be there for them. Be a listening ear. It helps.
In the previous episode, Joseph shared how he navigated his diagnosis, chose robotic surgery, and learned to open up to his loved ones about his health.
Thank you to Pfizer for supporting our independent patient education content. The Patient Story retains full editorial control.
This interview has been edited for clarity and length. This is not medical advice. Please consult with your healthcare provider to make informed treatment decisions.
The views and opinions expressed in this interview do not necessarily reflect those of The Patient Story.
“Go Get a Checkup.” – Joseph’s Prostate Cancer Advice
Joseph Martin was living a full life surrounded by family when he was diagnosed with prostate cancer. The news came as a shock and forced him to face questions about his health, future, and faith.
In this cancer vlog, Joseph shares how he navigated his diagnosis, chose robotic surgery, and learned to open up to his loved ones about his health. With his wife, a nurse, by his side, and his children offering support, Joseph found strength in family conversations and regular checkups. His message encourages others to take care of themselves, seek medical guidance, and cherish every moment with family.
Thank you to Pfizer for supporting our independent patient education content. The Patient Story retains full editorial control.
This interview has been edited for clarity and length. This is not medical advice. Please consult with your healthcare provider to make informed treatment decisions.
The views and opinions expressed in this interview do not necessarily reflect those of The Patient Story.
Every 13 minutes, a Black man is diagnosed with prostate cancer. (American Cancer Society, 2022)
Meet Joseph
My name is Joseph Martin. I’m a survivor of prostate cancer.
Learning He Has Prostate Cancer
I didn’t know that much about it. I just heard of it. I didn’t ever think that I would have cancer, but you never know what life is going to bring you.
Black and African American men are much more likely to develop prostate cancer. 1 in 6 Black men will develop prostate cancer in their lifetime — compared to 1 in 8 men overall. (American Cancer Society, 2022)
Talking with His Wife and Doctors
When you hear something that you don’t know whether you can live with or without, it makes you think. I talked with the urologists, the doctors, and my wife, who was a nurse. We sat down and talked about the possibilities of what could be and what could not be. Once I decided that I wanted to do the robotic surgery, it was a challenge and something to get used to, but we overcame it, and I’ve been doing great.
Today, there are over 3 million prostate cancer survivors in the United States. Black men in the U.S. and Caribbean have the highest documented prostate cancer incidence rates in the world. (American Cancer Society, 2022)
Sharing Everything with His Family
Whenever something goes on with me, I let my wife, my son, and my daughter know. I used to hold back from them to see first what’s going to happen. But now, when I go to the doctor and find out something serious, I tell them that we have to talk.
The National Comprehensive Cancer Network (NCCN) recommends that Black men and those with a family history should begin talking to their doctor about screening as early as age 40.
Importance of Regular Checkups and Why Early Detection Matters
You don’t have to be sick to go to the doctor. Get a checkup every six months, every three months. Do it on a regular basis. That way, if anything goes wrong, you will find out before it’s too late. Some people don’t want to do anything but work and then die. I had more life in me. I wanted to retire and do anything that I wanted to do afterward.
Go to the doctor. Sit down and talk with your family. Let them know what’s going on in your life, whether it’s good or bad. Do what it takes and what’s necessary. Live a productive life. See your grandkids grow up. See your grandkids’ kids, if you can. I would love to see my grandkids grow up and, if possible, their kids. I have two great-grandkids now. Most people never get to see great-grandkids at my age. Do the right thing and go to the doctor.
In our next episode, we introduce you to Joe’s son, Jamel, who has played an important role in his father’s cancer journey.
Thank you to Pfizer for supporting our independent patient education content. The Patient Story retains full editorial control.
This interview has been edited for clarity and length. This is not medical advice. Please consult with your healthcare provider to make informed treatment decisions.
The views and opinions expressed in this interview do not necessarily reflect those of The Patient Story.
When Joseph was diagnosed with prostate cancer, the news came as a shock and forced him to face questions about his health, future, and faith. He shares how he navigated his diagnosis, chose robotic surgery, and learned to open up to his loved ones about his health.
Bladder cancer care is evolving rapidly, with new treatments offering the potential for longer survival and better quality of life—including the possibility of preserving the bladder.
In this expert-led discussion, MD Anderson’s Dr. Ashish Kamat breaks down the latest in research, emerging therapies, and what patients and care partners need to know now to make informed treatment decisions.
This webinar has been completed!
This webinar has not yet been completed.
Bladder Cancer Breakthroughs 2025: New Treatments & Bladder-Sparing Advances
Hosted by The Patient Story Team | 56m 1s
Bladder cancer care is evolving rapidly, with new treatments offering the potential for longer survival and better quality of life—including the possibility of preserving the bladder. In this expert-led discussion, MD Anderson’s Dr. Ashish Kamat breaks down the latest in research, emerging therapies, and what patients and care partners need to know now to make informed treatment decisions.
Webinars in this series must be completed in order.
Pick up where you left off →
We would like to thank our promotional parter, The World Bladder Cancer Patient Coalition (WBCPC), which brings together bladder cancer patient organisations from around the globe to improve the lives of people affected by bladder cancer. They are committed to raising awareness, providing trusted patient information, and ensuring the patient voice is heard in research, policy, and care.
Stephanie Chuang: My name is Stephanie Chuang. I’m a cancer survivor, patient advocate, and founder of The Patient Story. The mission came from my experience of trying to deal with a diagnosis and navigating through all of the questions and not knowing.
Here at The Patient Story, we try to humanize this information for you and part of that is to provide access to topic-specific cancer experts, such as Dr. Ashish Kamat from MD Anderson, so that you can learn more and empower yourself or your loved one in your care. We’ve had more than 100 million views of our in-depth story videos that mostly feature patients, sometimes caregivers, care partners, and doctors. Our goal is to help promote self-advocacy and connection.
While we hope this is helpful, this is not a substitute for medical advice. Please consult with your healthcare team when making treatment decisions.
We’re talking about bladder cancer treatments in 2025 and beyond. This follows big meetings, including the 2025 American Urological Association (AUA) annual meeting, where top researchers gather to learn and discuss the very latest. Our focus is on what’s new and promising, all through the lens of what patients and care partners need and want to know about to stay informed and empowered.
Dr. Kamat, I already told you, I’ve got to pause after that. You’ve got so many things and accolades, but what I want to say is thank you for joining us.
What Inspires Dr. Kamat to Work with Bladder Cancer Patients?
Stephanie: I’ve seen your name in so many patient-facing efforts and discussions and I’d love to ask you. What drives you to go beyond the clinic, to try and get as much information and awareness to as many patients and care partners as possible, even those who are not in your direct care?
Dr. Ashish Kamat: First, thank you so much for having me. As you said, it’s very important to reach out to patients, not just because that is what we do, but if we don’t do that and we don’t get patients and their carers to be educated, involved, and understand the disease process, then we can’t give them the best chance of a cure. It’s not just surgery, medication, or radiation, but it’s a true partnership. In some ways, it’s a selfish motive to get patients more educated because it helps me give them the best results, which in turn helps both of us.
Stephanie: I love that. In the spirit of this conversation, I read one of your quotes, which was, “Nothing is more rewarding than hearing a patient say, ‘We made the decision together,’” and I loved reading that. Thank you so much for all that you do.
Categories of Bladder Cancer Patients
Stephanie: Let’s start this conversation by addressing some of the basics of bladder cancer for those who are newer to the diagnosis. Of course, this conversation is not individualized, so we’re not able to address every single potential situation on diagnosis. But in broad strokes, how do you categorize patients in determining what treatment options you might recommend? Would that be muscle invasive or not, high- or low-grade, and staging? What goes into your ultimate recommendations when you’re talking to patients?
Dr. Kamat: The first thing I always want a patient to understand is what disease state they have. From that perspective, I always try to have the patient think of their bladder as a balloon and the muscle layer as the rubber of the balloon. Just like a balloon, when the bladder’s empty, it’s collapsed and crumpled. When it fills up with urine, it gets larger and thinner.
It’s important for the patient to understand that just because it’s not muscle invasive, if it’s high-grade, it can still be a threat to life.
Dr. Ashish Kamat
With that analogy, think of the muscle layer being the rubber of the balloon — the two layers on the inside and the muscle layer on the outside. When these tumors start, they start on the inner surface of the bladder, but they set their roots down towards the muscle of the bladder. That muscle of the bladder getting involved — or the rubber of the balloon, so to speak — is the key determining step as to whether it’s safe to save the bladder or not. That’s the first thing that I like patients to understand.
Do you have muscle invasive disease, where our focus shifts to whether we can save the bladder and, if not, do we need to do radical surgery? Do we need chemotherapy? Do we need to do trimodal therapy with radiation? Or is the tumor not even in the muscle yet? In that case, we’ll aim to save the bladder, but then what do we need to do?
Once I go through that broad categorization, then I help the patient understand: is it a low-grade disease or a high-grade disease? When the tumor is noninvasive, you can have a low-grade disease, which again is still cancer, but for most practical purposes, you can almost treat them like warts. They’re nuisance factors and require intervention, but they’re not going to threaten our patients’ lives.
On the other hand, you have high-grade tumors, which may be small — smaller than just a point of a pencil — but they can kill the patient. It’s important for the patient to understand that just because it’s not muscle invasive, if it’s high-grade, it can still be a threat to life. That’s the majority of patients that we see.
Of course, there is a small percentage of patients who present with novel metastatic disease, where the cancer’s already spread outside the bladder to other parts of the body. When that happens, I try to help the patient focus our discussion. If the tumor’s left the bladder, we focus on how we have to treat the rest of the body first and then bring our attention to the bladder to see if, at some point, we still need to address what happened in the bladder to begin with.
How Common are the Different Types of Bladder Cancer?
Stephanie: How many patients typically land in these categories?
Dr. Kamat: The most common presentation for a patient is non-muscle invasive disease. Usually, about 70 to 75% of patients will present with noninvasive disease. Roughly 20 to 25% patients will present with muscle invasive disease, where it’s already in the muscle of the bladder. Fortunately, only about 5% of patients present with metastatic disease because that’s where a cure is hard.
If you see blood in the urine, don’t ignore it. Let someone know.
Dr. Ashish Kamat
Unfortunately, it is gender-differentiated. Women tend to have a higher risk or percentage of presenting with more advanced stages of disease. Partly because when the most common presenting symptom of bladder cancer is blood in the urine, you can imagine many women, especially young women, get told, “It’s not anything to worry about. It’s probably that time of the month. It’s probably a contaminated sample.” It’s important for primary care physicians and females to know that if you see blood in the urine, don’t ignore it. Let someone know.
Stephanie: I appreciate that. We’ve certainly done lots of these stories and that’s something we hear commonly. Especially as a woman, it’s easy to dismiss or attribute it to something else. Always, if you don’t feel well, if something’s not right, or if something’s concerning, you’re saying to advocate for yourself. Find someone who’s going to address that. Is that right?
Dr. Kamat: Absolutely. The best advocate a patient can have is themselves and their family, and, of course, us as partners, but they have to find the right advocate. Before they find someone to advocate for them, I want patients to empower themselves to say, “I’m not willing to take this response that you’re giving me, saying it’s nothing to worry about. I hope it’s nothing to worry about, but tell me why it’s nothing to worry about.”
Getting Patients and Care Partners Involved in Treatment Decisions
Stephanie: I also read that non-muscle invasive bladder cancer, which, as you said, most people are diagnosed with, behaves more like a chronic condition that requires ongoing care, but it does require that because of high recurrence rates. Can you talk more about how that informs your approach with patients?
Dr. Kamat: Stephanie, that’s where it’s important to get the patient and their family involved early to set expectations a little bit. But also, I like to ask patients, “Tell me what’s important to you.” Non-muscle invasive bladder cancer is a chronic condition and we want to make it a chronic condition. We don’t want to make it such that a patient is diagnosed and then they die from their cancer. If you do the right thing and we treat the patients correctly, we are saving their bladder, but then the bladder is at risk of the tumor recurring.
In some ways, I allude to this with patients to make it easy to understand. Think of your backyard. You have a couple of weeds. You see them and you want to treat them, but then you still have the backyard in place, right? And unless you remove the entire sod, you’re going to have weeds pop up.
Some patients will say, “You know what? I can’t keep coming to the doctor’s office every three to six months. I live too far away. I had to have my grandson or granddaughter take time off to bring me there. Can you give me one-and-done treatment?” In that situation, sometimes even for a noninvasive disease, we will do a radical cystectomy, which is to remove the entire bladder. Yes, that is a drastic step, but for particular patients — unfortunately, a small number of patients — that is something that they want because it’s one and done.
I don’t encourage that, but since you asked, it’s important for me to know from a patient their desire for their treatment paradigm. If a patient tells me that they want a one-shot treatment, then we have to take a drastic step. But for most patients, it involves regular checkups, regular treatments, evaluation of the bladder, cystoscopy, cytology, imaging, etc.
How Important are Biomarkers in Making Treatment Decisions?
Stephanie: For a lot of people, their decision-making is largely based on efficacy, like how well this will treat the disease, but also the impact on quality of life, which is something we talk about across all kinds of cancers. Is there anything else upfront that you want to make sure that patients and their families understand? Are there other factors, for instance, that might inform a different way of treating them? We hear a lot about biomarkers in the space of solid tumors, in particular.
Dr. Kamat: Biomarkers is a field where we and most of us in this oncology space are very interested in. They help us understand the biology and differentiation of bladder cancer treatments, paradigms, and development, etc. But in some ways, we’re fortunate in the sense that the best treatment for non-muscle invasive bladder cancer is biomarker-agnostic. It’s an immune therapy called intravesical BCG, which works so well that we haven’t needed to use biomarkers to change or inform the treatment paradigm.
Don’t spend your own money doing all of this. It’s not going to help you or us upfront.
Dr. Ashish Kamat
Sometimes I see patients walk in with sheets of paper where they’ve paid out of pocket to get all this testing done. It’s not helpful, other than as a research or educational tool. If a patient wants to understand, “Is my cancer being driven by FGFR3, PD-1, or something else?” That’s great for education, but it doesn’t help us in today’s day and age with the upfront treatment.
Now, if the tumor doesn’t respond to first-line therapy, then we will want to do biomarker analyses because there are FGFR-directed therapies and specific Rb-directed therapies. But for that, we want to look at the tumor that develops after the treatment hasn’t worked. Looking at the first tumor itself is not cost-effective for the patient. From a research perspective, we do biomarker analyses all the time. This is more for patients. Don’t spend your own money doing all of this. It’s not going to help you or us upfront.
Stephanie: Thank you. That’s important because we hear things in the world of science and major news publications, and it’s hard to understand what applies to our specific situation and cancer.
Treatment for Low-Grade vs. High-Grade Bladder Cancer
Stephanie: Before we dive into more of the latest and promising therapies and options, what typically is the treatment decision-making path or the options given based on these larger categories of patients? If you’re talking about the non-muscle invasive bladder cancer and then high-grade versus low-grade, here’s how we usually do this for low-grade and here’s what we normally recommend for high-grade. Could you provide a more basic picture of what that landscape is?
Dr. Kamat: So let’s assume a patient is sitting in front of me and he or she has low-grade, non-muscle invasive bladder cancer. The first thing is to explain to the patient that that’s great news. If you’re going to have any kind of bladder cancer, this is the one to have. Yes, it is cancer, but it’s extremely treatable and even though it might recur at some point in your life, the recurrence is not a problem
For the treatment of low-grade, non-muscle invasive bladder cancer, the treatment is removing the tumor, which is usually done with the transurethral resection (TURBT). We look in the bladder with a scope. Currently, we use electricity to remove the tumor. Some places use lasers, but there’s no real benefit to one over the other. Then we give a single shot of chemotherapy into the bladder. That’s what the majority of patients need and what the majority of patients respond to.
Now, some patients have a recurrence of the tumor and as long as the tumor stays low-grade, that patient then transitions into intermediate-risk. It’s not high-risk. The only reason we call it intermediate-risk is because we want people, physicians especially, to understand that it’s gone from low-risk to intermediate-risk, so we might need something more. What is that something more? Intravesical therapy, either in the form of chemotherapy maintenance — so once a week for six weeks and then monthly chemotherapy maintenance — or intravesical BCG.
Now there’s a shortage of BCG across the globe. In today’s day and age, we don’t tend to recommend BCG for patients with low-grade disease, not because it doesn’t work well, but because there’s not enough BCG and it is something we need to reserve for our high-risk patients. For the low-grade, intermediate-risk patient category, the treatment is still intravesical chemotherapy.
Now there are paradigms for patients who are too sick to go to the operating room or who’ve had a recent myocardial infarction (MI) or heart attack and they’re on blood thinners, an antiplatelet drug like clopidogrel (Plavix), or aspirin, and we want to avoid a procedure. We can ablate these tumors with chemotherapy upfront. There are newly-approved agents, like the recently FDA-approved MitoGel (now known as Jelmyto), which is mitomycin in a gel format, where you can ablate the tumor. But that’s, in some ways, a select group of patients who are trying to avoid resection of the tumor or we are trying to buy some time before we can take them to the operating room.
Now, moving from the low-grade patient with low-risk or intermediate-risk category, the next category is the high-risk patient. Any patient with high-grade tumors is considered high-risk, which includes carcinoma in situ (CIS) or T1 disease. There are a lot of factors that come into play with regard to treatment recommendations.
Patients who have high-risk tumors can be further categorized according to their actual risk. If they have certain parameters that make them extremely high-risk — and I’m talking about risk of progression and metastatic disease — I might recommend to a patient, saying, “Your tumor is non-muscle invasive, but it’s getting there. It’s getting to become muscle invasive, so you might still want to consider radical treatment upfront.” Fortunately, that’s a small percentage of patients. For the majority of patients, we can still recommend treatments that allow us to spare their bladders.
The gold standard for many years — for the last 40-plus years and it’s not been dethroned yet — is intravesical immunotherapy with BCG. BCG (Bacillus Calmette-Guérin) is developed from the tuberculosis vaccine. It’s been used for other cancers in the past, like melanoma and leukemia, but it was toxic because you had to give it in the blood. With bladder cancer, we can put it in the bladder.
Early data suggested that it was fairly toxic to patients, which is because people didn’t understand how to use it. If it’s used appropriately with appropriate pre-medication or interval spacing, etc., 90% of patients can finish the whole three-year prescribed course. If you use adequate BCG, the recurrence rates are in the teens and the progression rates are in single digits. An efficacy rate of 90% when it comes to preventing progression of disease of high-grade bladder cancer allows 90% of patients to spare their bladder.
Again, nothing’s 100%. If it doesn’t work or patients can’t tolerate it, we have to use other treatments and that’s where you’ve seen this explosion of drugs coming up recently. There’s gene therapy with nadofaragene firadenovec-vncg (Adstiladrin). There’s cretostimogene grenadenorepvec. There’s an intravesical pretzel device. There is nogapendekin alfa inbakicept (Anktiva), which is the bioshield that Dr. Soon-Shiong has been talking about all over the world recently. All of those come in after BCG has not worked for a particular patient.
Stephanie: Thank you. I know it’s a lot to talk through. We will be talking about all this, but, of course, the explosion, so that people understand what they can consider and weigh might be good for them personally.
The Role of Biomarkers in Muscle Invasive Bladder Cancer
Stephanie: Biomarkers haven’t been a thing yet in bladder cancer. That’s largely a good thing, in that most people don’t require that. The standard of care has worked well. But as you have mentioned, there are meetings and gatherings where people are trying to figure out more about how they may play a role in bladder cancer, is that right?
Dr. Kamat: Absolutely, Stephanie. We were talking about noninvasive disease. If we move into the muscle invasive space, there, the biomarkers are showing more promise and slightly more utility. They’re still not used in the clinic. They still don’t inform us of how we counsel a patient, but they help us inform the patient about their prognosis. For patients who have muscle invasive disease, the discussion becomes, “How can we get you the best chance of long-term cure?” And that usually involves some form of bladder removal.
There is trimodal therapy, which, in selected patients, can offer the option of sparing the bladder and providing long-term control, but that’s the minority of patients. The majority of patients do require radical cystectomy, unfortunately, to remove the bladder. And then to give them the best chance of long-term cure, there is neoadjuvant therapy and with the new paradigm, sandwich therapy that comes into play.
Neoadjuvant therapy essentially means getting treatment before the surgery. Traditionally, it involves cisplatin-based chemotherapy, which can be fairly toxic. Roughly 40% of patients can’t even get cisplatin because of cardiac disease, renal dysfunction, age, neuropathy, or something else, so other drugs have been studied and are being studied to see how they can help us give patients who are not as cisplatin candidates some treatment.
In more recent times, the cisplatin combination has been combined with immunotherapy, immune checkpoint inhibitors, and the NIAGARA protocol, which is the most recent development in the space. This protocol essentially suggests that combining combination chemotherapy with a checkpoint inhibitor before surgery and then continuing the checkpoint inhibitor after surgery can offer the patients the best long-term chance of cure in the muscle invasive space.
This is where the biomarkers have come in handy. There are biomarkers that people have developed, trying to help us understand which patients may respond so well to systemic therapy that we don’t have to take the bladder out. Again, this is being studied. It’s not something that I can say a patient should go to their physician and say, “I have this biomarker. I don’t want my bladder out.” No, it’s not ready for primetime, but we’re looking at those. There are DNA repair genes. There are other agents and other markers we’re looking at.
The biggest buzz is around circulating tumor DNA (ctDNA) because data and evidence have come out that suggest that in patients who have detectable levels of circulating tumor DNA, it puts them in a category for prognosis that is not as good. And if they don’t have circulating tumor DNA, then it gives them a better prognosis. Again, this is a research field.
But it’s been studied in the sense that if a patient has elevated ctDNA, do we need to escalate their treatment? If they don’t have circulating tumor DNA, can we de-escalate that treatment? In other words, less toxicity but the same efficacy. A trade-off, of course, is that we need to offer patients shared decision-making because, like I said, it’s currently not standard of care. It still is a research question.
Everything is crucial because bladder cancer is not one of those diseases that you get second chances with, so you want to get the best treatment right up front. The whole team effort is very important.
Dr. Ashish Kamat
Your Bladder Cancer Team: Who’s Involved?
Stephanie: Before we dive even deeper into the details of what’s the latest, typically — and I know it depends on whether someone’s getting care at an MD Anderson versus a community provider or a different practice — what is the makeup of the bladder cancer care team and does that impact what you recommend to people?
Dr. Kamat: For all the time I’ve been doing this and in many parts of the world when I travel and help set up their cancer programs, one of the things I’ve been a champion of is to emphasize that bladder cancer is a multidisciplinary cancer. In order to give our patients the best chance of a cure, it has to be such that everything is considered. Yes, I’m a urologic oncologist on the surgical side, but bladder cancer is not a surgical disease, a medical disease, or a radiation disease. We have to look at the patient holistically.
That’s why whenever we’re guiding patients as to where to get their care, I can’t have everybody flying to Houston to be treated here at MD Anderson, but I tell them, “Wherever you seek your care, make sure that the care team over there is not just one person. That it involves not just the surgeon, but also the medical oncologist, the radiation oncologist, the nursing team, and the support staff.” Everything is crucial because bladder cancer is not one of those diseases that you get second chances with, so you want to get the best treatment right up front. The whole team effort is very important.
Concerns from Patients About Their Bladder Cancer Treatment
Stephanie: I’m sure a lot of people have traveled to see you, given your expertise. Are there common situations you’re hearing from them? Like, “Hey, I saw such and such. They’re not bladder cancer specialist and they wanted to immediately go to this step.” Or maybe things that you’re seeing at different levels that might make it harder for patients to understand what’s good for them?
Dr. Kamat: Yeah, and that’s why organizations like The Patient Story are very important because I see that very often. Patients will come in and say, “This is what I was told. I have no choice. I have to do this.”
First off, every patient has a choice, so when I hear that, it makes me not very happy because patients always have a choice. Our role is to guide them to make what I think is the best choice for them, but it’s very patient-specific. Like I said earlier, what may be right for one patient, like surgery, may not be right for someone else and it might be purely based on their beliefs or their support structure.
Patients always have a choice. Our role is to guide them to make what I think is the best choice for them, but it’s very patient-specific.
Dr. Ashish Kamat
But the other thing that often gets us is that if a patient goes to see someone who doesn’t do bladder cancer all the time, it’s not their fault. It’s not the physician’s fault. The physician is trying to do the best they can, but if they’re treating 10 other cancers or 10 other problems, I can’t expect them to be at the forefront of the latest in that arena. So they’re now offering the patient what is the best treatment in their honest opinion, which might not be the latest cutting-edge.
I always recommend that patients get a second opinion. Even if they come to see me and they want a second opinion from somewhere else, go get it from somewhere else. It’s good for patients to hear, from at least two separate people, if that treatment truly is the best treatment for them and then make the decision.
Now, of course, when patients have to get treated — like with chemotherapy, for example — I often tell them, “You don’t have to be stuck in Houston for the entire duration. Go back home. Your physicians are extremely qualified.” Everybody can give chemotherapy. As long as they’re willing to follow our protocol or our recommendations, get the chemo where it’s easier for the patient.
When it comes to surgery, it’s slightly different because surgery for bladder cancer is extremely specialized; it’s not something that most people can do unless they’re highly trained and it’s not something that I recommend patients go to someone who does it once in a while. It’s very important to go to someone who does it all the time, has a team to support them, not just the surgeon, but the nursing staff, the ICU staff, the anesthesia, the stoma nurses, etc.
Surgery for bladder cancer is extremely specialized; it’s not something that most people can do unless they’re highly trained and it’s not something that I recommend patients go to someone who does it once in a while.
Dr. Ashish Kamat
Ironically, in many ways, people forget that that’s just as important when it comes to radiation therapy. Even in radiation therapy, it’s not the machine that’s necessarily important. There might be multiple places in the country that have a good machine, but it’s the radiation technologist and the radiation oncologist that’s planning the treatment paradigm who can make a difference between radiation that helps save the patient’s bladder versus radiation that causes so many side effects that the patient then loses their bladder and their rectum. That is very important.
How to Find the Right Provider
Stephanie: Where can people go to figure that out? MD Anderson is world-class and people know about it. But in terms of figuring out if someone is the right surgeon or the right institution with people who can do radiation therapy with the precision that we need, how would people go about finding that?
Dr. Kamat: There’s a lot of junk online, but there are some good resources. Your institution, of course. The Bladder Cancer Advocacy Network (BCAN) is a very good, reliable place where patients can go because it’s all for patients and by patients. I recommend that patients make that their first stop. On a global scale, the World Bladder Cancer Patient Coalition has good resources.
It’s the radiation technologist and the radiation oncologist that’s planning the treatment paradigm who can make a difference.
Dr. Ashish Kamat
But I always tell patients to ask their family doctor and local physicians. “Who do you trust here? Who would a patient’s family doctor send their own family to?” Because that’s important as well. Not everybody who does bladder cancer makes it onto these resource sites. Just because I haven’t trained someone or haven’t heard of them, but they’re in Boise, Idaho, doesn’t mean they’re not doing excellent work. The local community of physicians often knows who is good. Conversely, often they know who may have published 5,000 papers, but is horrible. It’s not just academia. It is taking care of a patient that’s important.
Stephanie: I appreciate a lot of what you said. I also want to go back to what you said earlier, which is that it’s not that the practicing physicians aren’t smart; it’s that they are generalists. There’s no way, with all the advancements happening at the pace they’re happening at, that they could keep up with all of the things that are happening in the research. Thank you for that.
Exciting New Cancer Treatments on the Horizon
Stephanie: Let’s dive in more. You already talked about some of the explosion of options that have come up recently. You talked about gene therapy, pretzel devices, and BioShield coming in after BCG hadn’t worked. Can we go through some of the ones that you’re most excited by? And again, in the spirit of what patient group would be most interested in each of these options?
Dr. Kamat: The biggest explosion of data has been in two separate spaces. The first one is for the patient who has high-risk bladder cancer, has tried BCG and it hasn’t worked, and is now faced with a conundrum as to what to do next. The standard treatment for many years was to remove the bladder because there were no real drugs approved and there was nothing available. Unfortunately, when drugs were approved, they had a 4 to 5% success rate at two years. No patient wants to hear, “I’m going to try something with a 4 or 5% success rate.”
Because of that, different organizations came together and the FDA partnered with a lot of us and developed this paradigm of BCG-unresponsive disease. As many patients might know, there was an explosion of drugs studied in that space and currently has the most activity. If a patient has tried BCG and still has recurrent high-grade disease in the bladder, what are the options? That’s where the drugs have been approved, that’s where drugs are being studied, and that’s where some of the agents that I talked about come into play.
The first drug that was approved in that space is pembrolizumab (Keytruda), which is a systemic immuno-oncology (IO) therapy and the results were very exciting at that time, but there were some toxicities. Not many patients today will get single-agent pembrolizumab (Keytruda) for BCG-unresponsive disease, but there are studies using pembrolizumab (Keytruda) with other agents that I think will offer patients the opportunity to get that systemic therapy backbone plus something else.
The next agent that was approved in that space was intravesical gene therapy, nadofaragene firadenovec-vncg (Adstiladrin). Now this is a gene therapy that allows the patient’s bladder to, in some ways, become a bioreactor because it transfects the bladder cells with an adenovirus that then helps with the production of local interferon-alfa 2b (IFNα2b).
That has the advantage in the sense that it’s given in the bladder once every three months, so the patient doesn’t have to come to the clinic every week or every other week. It’s once every three months. The efficacy number, roughly at about 12 months, is that 25% of patients will have a positive result and be in disease remission. And that’s great, right? Because we can tell the patients, “It’s approved for BCG-unresponsive CIS. Let’s try it. If it doesn’t work, we’ll know and we can switch to something else. But if it works, it’s great because it’s once every three months and you might have at least a 25% chance of saving your bladder just with this treatment.”
The third drug that was approved in this arena is the IL-15 superagonist, which I’ve seen a lot of buzz about. It’s been called the “BioShield” by some people because there is some evidence that IL-15 might have efficacy across different cancers, but that’s a different topic. For bladder cancer, it’s given in combination with BCG and helps boost the patient’s bladder’s immune response to BCG itself.
That has excellent results in the sense that more than 50% of patients have a response in a year. The downside of that is that it has to be given in combination with BCG. Again, kudos to the company because they have gotten the FDA to agree to allow non-US BCG to be brought into the country and used in combination with this agent, especially in places where there is no BCG.
Those are the approved drugs. The same space now has multiple different drugs that are being studied. One is cretostimogene grenadenorepvec, which is another gene therapy that focuses on the retinoblastoma (Rb) pathway. It’s an oncolytic virus. It has excellent results. It’s not approved yet, but many of us expect that it will be approved.
Then there’s the intravesical pretzel device, which is where chemotherapy — in this case, gemcitabine (Inlexzo) — is put in a little silicone device that looks like a tiny pretzel. The company doesn’t like us calling it the pretzel, but it’s what helps patients understand. It’s put in the bladder and releases the drug at a slow rate. It’s great for patients because you put the device in once and then the patient just has to come in after three weeks or 12 weeks, depending on what stage they’re at, and have the device removed, but it’s constantly releasing gemcitabine (Inlexzo). Again, it’s not approved, but the data is looking promising. Everything that was presented at AUA, American Society of Clinical Oncology (ASCO), and elsewhere looks like this is something that’s going to be beneficial to our patients.
Then there are other agents. There is non-viral gene therapy, such as enGene, which allows patients to get gene therapy without using a virus. Some patients are afraid of the whole viral paradigm when it comes to gene therapy. Some hospitals in smaller communities can’t get certification to use gene therapy that’s viral-based. We don’t know yet, but if it’s approved, it’ll allow patients in smaller centers and smaller communities to get this drug.
Beyond this, I could go on for two hours, but there are so many more drugs being looked at, like laser therapy, etc., in this space. If you’re going to have bladder cancer, this is a great time because there are potentially many options that are going to be available.
Stephanie: I know that we could spend much more time going through every single amazing potential development and promising research happening, but let’s dive into some of the ones that you already talked about and then I have follow-up questions about how you would offer what therapy at what time. In any space, there are questions about whether you use a lot upfront first and try to hit hard in certain diagnoses or not.
First, you talked about gene therapy. Can you say the name of the therapy and share a little bit more about how close we might be? I know you don’t have a crystal ball, but how close are we to seeing this in a clinic? You can’t tell if the FDA is going to approve something, but if it’s promising and it does get approved, in six months, could it be something off of clinical trial, approved, and available?
Dr. Kamat: To clarify, the gene therapy nadofaragene firadenovec-vncg (Adstiladrin) is already approved. That’s interferon-alfa therapy. It’s already approved and patients are using it, and we can actually offer it to patients.
The other gene therapy is cretostimogene and that’s the gene therapy with CG Oncology. That’s not approved yet. We think that they will go in front of the FDA within 12 months, and hopefully, it’ll be approved and used for patients at that time. The results look great, but it’s not approved, so we can’t recommend it at this point.
There’s not one clear drug that’s better for every patient. It’s a personalized decision.
Dr. Ashish Kamat
Current Clinical Trials
Stephanie: We will also talk about clinical trials before the conversation is over and how you explain them to your patients. The term itself is not very friendly, I think. “Clinical trial” is pretty daunting for people, so we’ll humanize it.
There’s already gene therapy approved, like you mentioned, but cretostimogene is still in research. When you’re thinking about gene therapy for patients, let’s say that someone is able to go on to a clinical trial if it was recruiting or let’s say that it was already approved, why is this one better for certain patients than the one that’s already approved?
Dr. Kamat: There’s no one particular drug that’s better for a patient. In fact, when it comes to sequencing the right drug with the right patient, this is a very complex problem. The international bladder cancer group that I lead convened a think tank and spent months thinking about this. We had a hundred faculty members from all over the world go to Houston, sit in one room, and hash this out to try to figure out how to best sequence these drugs for the patients.
The short answer is that there’s not one clear drug that’s better for every patient. It’s a personalized decision. Some patients might benefit from treatment that’s given once every three months and, in which case, it’s obviously the nadofaragene firadenovec-vncg (Adstiladrin).
If the CG Oncology’s cretostimogene is approved and it’s available, that’s every week for six weeks and then there’s maintenance that goes with it. But the efficacy numbers there are higher than with the published data with nadofaragene firadenovec-vncg (Adstiladrin), so a patient might say, “I want to try this. I don’t mind coming every so often. I don’t mind the side effects of it. I would like this drug.” That becomes a discussion with the patient.
It’s not just these two gene therapies. Other treatment options are available as well. For example, the IL-15 NAI (nogapendekin alfa inbakicept), patients might like the fact that it’s given in combination with BCG and say, “BCG worked for me. It just stopped working after some time. I would rather get that treatment.”
Then some patients might say, “I don’t want any of this intravesical therapy. I want to get the device that you can put in my bladder because I like the fact that you can put it in, I don’t have to come back for X number of weeks, it’s constantly releasing the chemotherapy in my bladder, and I’m having good results with it.”
I don’t mean to dodge your question, but the real answer is there’s not one treatment that’s the best treatment for everybody.
Stephanie: I appreciate that and I probably should have said it differently. That’s exactly what I think the question is. We’re getting to a space of personalized treatment, not just in terms of the disease, but what a patient prioritizes for his or her life. Again, I appreciate that that’s how you approach your patients and how you bring this to light and at the forefront of these discussions, because it is different. People are optimizing for different things in their lives.
I know you said that they don’t like the pretzel name, but can you talk about the device and whether it’s in clinical trials? We’ll also be putting up the clinical trial names for people in case they are interested in asking their doctors. Do you have the clinical trial name for the gene therapy from CG Oncology?
Dr. Kamat: That’s the BOND-003 study. The pretzel is TAR-200 and TAR-210, and that’s Johnson & Johnson’s MoonRISe and SunRISe paradigms, and there are five SunRISe studies. Full disclosure: I’m part of advising pretty much every company in the bladder cancer space, so I help advise all these companies as well. The SunRISe paradigm is SunRISe-1, SunRISe-2, SunRISe-3, SunRISe-4, and SunRISe-5, and then MoonRISe, which is using TAR-210.
We don’t know what the company is going to call this when it actually gets approved in the clinic. There are a lot of names being thrown around and I can’t reveal that anyway, but we don’t know what the final name is going to be. That’s all in the noninvasive space.
I also want to enlighten the patients on the muscle invasive disease and the metastatic disease. In the metastatic space, this is not brand new, but using enfortumab vedotin with pembrolizumab (EV+P) clearly has changed the treatment paradigm. About eight to 10 months ago, we would tell patients with metastatic disease that the median survival is 14 months, which means half the men and women will not live 14 months.
Once EV+P was studied and presented by Dr. Tom Powles at ASCO, he got a standing ovation, as many of you know, because it changed the paradigm for patients. Now we can tell patients who have metastatic disease that the median survival has jumped from 14 months to 30 months, so half the men and women will live 30 months and that’s great.
In the metastatic space, a lot of second-line and third-line therapies are being studied. That’s where you have FGFR-directed therapies, platinum-based combination therapies (cisplatin or carboplatin), and other antibody-drug conjugates (ADCs) that have been developed for patients with biomarkers, etc There’s still a lot of work that needs to be done. It’s not as though EV+P is a full stop. We still need to get patients to live longer than a median survival of 30 months.
Traditionally, what’s happened is if you don’t take the patient’s bladder out, these tumors recur and when they do, they recur with a vengeance
Dr. Ashish Kamat
In the muscle invasive space, where patients have muscle invasive bladder cancer, but they’re trying to save their bladder, a lot of research is being done on whether we can avoid a radical cystectomy. The treatment paradigm there is to give neoadjuvant therapy, whether it’s platinum-based with gemcitabine-cisplatin plus X, and then get the patient to where there is no tumor left in the bladder.
Traditionally, what’s happened is if you don’t take the patient’s bladder out, these tumors recur and when they do, they recur with a vengeance and get worse and then you’ve missed that window of opportunity. Now, with the improved tools that we have where we can use good cystoscopy bladder MRI, using circulating tumor DNA, there is data emerging — and it’s still being studied in a clinical paradigm though — that we might be able to select a subgroup of patients where it might be safe for them to get systemic therapy and not necessarily have the bladder removed. But that, again, to caution people, is a clinical trial paradigm.
One of the trials being done in that space is the MODERN study. Dr. Matt Galsky is the principal investigator for that. It’s great because that gets patients to see, “I’ve achieved this particular endpoint. Now, on a clinical trial, can I continue on and not have my bladder removed?” That’s the clinical trial part of it.
But in the actual clinic, we are looking to improve results for patients who want to have their bladder taken out. That’s where the NIAGARA protocol, which is using gemcitabine and cisplatin, which used to be used alone as a combination, but now you can combine this with durvalumab (Imfinzi), which is an IO (immuno-oncology). They give this to patients upfront, they undergo radical cystectomy, and you continue the durvalumab (Imfinzi) after the surgery.
This has improved their survival and it’s the first time that the overall survival has shown an improvement in patients who are undergoing radical cystectomy. For many years, we’ve used neoadjuvant therapy and the benefit to patients is about 5%. Nothing has improved upon that. People have tried. They’ve used different types of chemotherapy regimens, dose-dense MVAC (ddMVAC or methotrexate, vinblastine, doxorubicin, and cisplatin), spacing out chemotherapy, etc. This is the first time that this combination therapy has been shown in a randomized phase 3 study to improve overall survival for these patients. Clearly, it changes the treatment paradigm.
Again, in the interest of time, I’m not going to go into too much detail, but there’s so much in between that is being developed and has been developed. I encourage patients who are faced with the conundrum of having muscle invasive disease to ask their physicians. “I know that this is standard of care for me, but if it doesn’t work, what else do you have available?” Because there’s a lot of stuff that’s available.
Stephanie: Thank you. I’m glad you explained it all in a very concise way, given the time crunch. I do want to go back to some of these because when people are hearing about it, it’s great that they have the information, but there are lots of questions about efficacy and impact on quality of life. You’ve talked a lot about bladder sparing, which is a huge part of what we’ve heard in terms of questions from patients as well, since there is such a high degree of impact on them.
There doesn’t appear to be any major difference between the side effect profiles of one over the other.
Dr. Ashish Kamat
Are New Bladder Cancer Treatments Decreasing Side Effects?
Stephanie: Going back to non-muscle invasive, with gene therapy, you have different considerations. One of them is once every three months and the other is once every week for six weeks. What about side effect profiles? Are you seeing something much better in the one that’s in clinical trial now, for instance, for the quality of life for people?
Dr. Kamat: Again, it’s not as though the side effect profile is majorly different or better or worse in some ways. Patients who will get side effects, you’ll see that upfront. Most patients will have some localized side effects in the bladder, but they’re very manageable and very tolerable, especially since we can give patients antispasmodics, etc. But there doesn’t appear to be any major difference between the side effect profiles of one over the other.
Understanding TAR-200 (“Pretzel” Device)
Stephanie: The pretzel device is novel and people will have questions about how this works. Do people go to the doctor or to the hospital and then it’s put in for them and it automatically releases the treatment? How often are you going back in? Is that indefinite or is there a particular amount of time that you’re supposed to have that pretzel device in?
Dr. Kamat: The way the clinical trials are designed is essentially the TAR-200 device, which we colloquially call the pretzel, is put in the bladder with a device that is like a catheter, so it’s not a major surgical procedure. It’s done under local anesthesia. You put it in the bladder and the patient keeps that in place.
Most patients will initially feel like there’s a little bit of a foreign body in there, but patients are, in some ways, used to it because they’ve had prior treatment and it’s something that you can easily treat with an antispasmodic. After a while, patients sometimes forget that they have this in there.
The TAR-200 device, which we colloquially call the pretzel, is put in the bladder with a device that is like a catheter, so it’s not a major surgical procedure. It’s done under local anesthesia.
Dr. Ashish Kamat
Now, in the early stages of treatment, the device has to be changed more often. They come in every three weeks and get the device changed. In the maintenance phase, it has to be changed roughly every three months, so they can leave it in there for that long.
It doesn’t bother most people. Sometimes we have to remind patients that they have a stent in place. We don’t want them to forget that they have it and never come back. Hopefully, that’s a good problem to have, but we don’t want them forgetting that they have it in there.
Like I said earlier, that’s where the discussion on personalized treatment with the patient comes in. Some patients are more than happy to come into the office every so often, while some don’t and prefer to come in once every three months. We’ll have different things to offer to patients, but if we look at the efficacy numbers, it looks like one of these newer agents that are not approved might end up with the best efficacy numbers. We don’t know that for sure yet.
Stephanie: We’re still waiting for that actual data.
Understanding Treatment with EV + Pembrolizumab (EV+P)
Stephanie: Lastly, as we’re wrapping this, the EV+P, which you’re talking about, the ASCO and the standing ovation, can you put into perspective what this means for patients who typically were looking at pretty rough numbers before?
Dr. Kamat: Traditional first-line chemotherapy for patients was cisplatin-based chemotherapy. Many patients could not get cisplatin because of the various comorbidities that our older patients tend to have, especially those with bladder cancer, with prior smoking history, cardiac disease, etc., and they had to get non-cisplatin-based therapy. There was a lot of push to study IO therapies, like pembrolizumab (Keytruda), atezolizumab (Tecentriq), etc. Then the antibody-drug conjugate, which is enfortumab vedotin (Padcev), targets nectin-4.
Long and short, this combination was studied in the patient population and compared head-to-head with standard chemotherapy, and clearly improved survival over standard chemotherapy. Now, it’s become the de facto first-line therapy for patients with metastatic disease.
But a caveat. It’s not available everywhere in the world. It’s expensive. There are many places in the world where patients still don’t have access to EV+P, or if they have access, it is something that can break the bank. Again, that’s a practical problem that we all face: resource allocation. Who gets the treatment? Who can get the treatment? Who can afford to get the treatment? But as long as the patient has access to it and it’s available and affordable, EV+P has become the de facto standard of care.
Humanizing Clinical Trials
Stephanie: We’ve talked about clinical trials. Without going into the details of these specific trials, they’re something that a lot of people are not familiar with. What tends to be the way that you have found most effective in describing what a clinical trial is and why patients whom you bring this as an option to might want to consider it? How do you humanize the concept of a clinical trial to them?
Dr. Kamat: That’s a very important question. Patients need to understand that a clinical trial does not mean that they are being treated like guinea pigs. That’s the furthest from the truth. There are some patients who, unfortunately, don’t have any options because they haven’t responded to anything; at that point, it’s a matter of life or death. It’s either a clinical trial or nothing. But for most patients, that’s not the situation.
For patients in front of me who have different options available, but there’s a clinical trial in the space, what I tell them oftentimes is, “This particular agent that’s being studied in the clinical trial is something that we believe in. We know from early data in the preclinical space that it has good mechanistic reasons to maybe be better than the standard of care at some point. We don’t know that yet. We have to study it.”
The advantage of taking part in the clinical trial for every patient is that they are at the forefront of the research, but they also get better care. If a patient is on a clinical trial, by definition, they are being followed very closely. There’s usually a nurse that’s assigned to them. Everybody’s looking at the pathology very carefully because the company doesn’t want any mistakes. We, as investigators, want to make sure patients are getting the best care across the board, but especially on a clinical trial because there’s that much scrutiny.
Patients need to understand that a clinical trial does not mean that they are being treated like guinea pigs. That’s the furthest from the truth.
Dr. Ashish Kamat
In some ways, the patients are most catered to in a clinical trial. There’s very little room for error. The pathology is often double-checked at a central pathology. Everything’s done on a well-defined protocol and schedule, and it’s made patient-friendly. I often tell patients, “If you take part in the clinical trial, you will be getting the highest standard of care of any patient that’s getting treated for a particular disease. And you’ll not only be helping yourself, but hopefully you’ll be helping inform the field and help other patients as well.”
But we have to be practical. We always tell patients, “Clinical trials are a commitment. It’s not as though you can skip appointments. It’s not as though you can not take part in every part of it. If it’s too much of a social or financial burden, let us know upfront because there’s no obligation.”
I never want patients to feel that if they say no to a clinical trial that I’m recommending, I’m going to take it personally. No, not at all. It’s not a personal thing. It’s me trying to get them to take part in something that I think will help them. But if they can’t participate, they’re not going to hurt my feelings at all and they still will get treated.
Conclusion
Stephanie: Dr. Kamat, thank you for spending time today with us and for everything you do for patients and care partners in this space. We appreciate it.
Dr. Kamat: It was my pleasure. Thank you so much.
Stephanie: I hope this conversation with Dr. Kamat helped you. Please feel free to share this discussion with others in your community.
While we hope that this was helpful and that you walk away with questions to ask your healthcare team, this is not a substitute for medical advice.
Visit ThePatientStory.com if you want an entire library of other patient stories. I’m so glad you could join us and I hope to see you again soon. Take good care.
We would like to thank our promotional parter, The World Bladder Cancer Patient Coalition (WBCPC), which brings together bladder cancer patient organisations from around the globe to improve the lives of people affected by bladder cancer. They are committed to raising awareness, providing trusted patient information, and ensuring the patient voice is heard in research, policy, and care.
Bladder cancer patients Ebony & LaSonya talk about their cancer journey, including their first symptoms, how they processed their diagnosis, treatment options, and how they found support. Dr. Samuel Washington, a urologic surgeon, also gives an overview of bladder cancer and its treatments.
Bladder cancer patients Ebony & LaSonya talk about their cancer journey, including their first symptoms, how they processed their diagnosis, treatment options, and how they found support. Dr. Samuel Washington, a urologic surgeon, also gives an overview of bladder cancer and its treatments.
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Living with an MPN (myeloproliferative neoplasm) brings challenges that aren’t always visible—but tracking your symptoms can make a powerful difference.
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Take Charge of Your MPN: Tracking Symptoms & Advocating for Better Care
Hosted by The Patient Story Team | 44m 55s
Living with an MPN (myeloproliferative neoplasm) brings challenges that aren’t always visible and tracking your symptoms can make a powerful difference. In this free patient-led discussion, MPN advocates Ruth, Nick, and Demetria open up about what it means to live proactively with ET, PV, or MF.
Webinars in this series must be completed in order.
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Thank you to Incyte and Karyopharm Therapeutics for supporting our patient education program. The Patient Story retains full editorial control over all content.
Ruth Fein: This conversation is focused on living with myeloproliferative neoplasms, or MPNs, and how tracking symptoms can empower you to advocate for the care that you need or the care of a loved one.
My story spans over 30 years. I was initially diagnosed with essential thrombocythemia (ET), which transformed into polycythemia vera (PV), and then eventually became myelofibrosis (MF), which is what I have now. I’ve had several symptoms and I’ve been on a few different drug therapies along the way. I’ve had life-threatening clots and life-threatening bleeds, which, of course, conflict with each other.
Now, I’m on a clinical trial and doing great. They call me the poster child for this clinical trial and it’s been five and a half years. I’m always proud and happy to speak to other people living with MPNs, particularly since I didn’t know anyone else living with an MPN for the first 20 years that I had this disease.
This discussion is brought to you by The Patient Story, where our mission is to humanize cancer. We’ve had more than 100 million views of our in-depth story videos that mostly feature patients, sometimes caregivers, care partners, and, of course, physicians, clinicians, and other care professionals. Our goal is to help promote self-advocacy and connection.
We want to thank our sponsors, Incyte and Karyopharm, for their support of our independent educational program. This allows us to create more content like this for free. The Patient Story retains full editorial control. While we hope this is helpful, this is not a substitute for medical advice. Please still consult your healthcare team when making treatment decisions.
Meet Our Panel
Ruth: I’m joined by two other amazing people who also advocate for those living with MPNs.
Nick Napolitano: Hi, Ruth. My name is Nick. I’m a polycythemia vera patient. I was diagnosed in 2016, so I’m approaching the 10-year mark, which is a big deal. Like you, Ruth, I’ve experienced a range of symptoms throughout my journey — everything from itching to bone pain. I have experienced different treatment options and am currently on a drug, doing very well.
I’m a husband to my beautiful wife, Kara. I have two boys, Jake and Nick. They keep me very active.
Demetria J: I’m a wife, a mother, a mother, a business owner, and a coach and mentor. I’ve been a business owner for nearly 20 years. I initially started in the beauty industry and owned a nail salon. Then in 2021, I pivoted to salon suites and an event space. And then life happened.
I received a diagnosis of ET in 2018. I went through a series of different doctors and ended up at Emory in Atlanta, where the oncologist put me on a new medication that had just come out of clinical trials and was showing positive results. He believed that, at that point, I would benefit better from that.
Eventually, my levels began to level out. My platelet counts went down tremendously, so I thought we were in the clear. Then in 2023, I found out it had progressed to myelofibrosis. I was experiencing different symptoms, such as fatigue and stomach pain. I went to the emergency room, was given some pints of blood, and a diagnosis of myelofibrosis after having a bone marrow biopsy.
Ruth: Thank you both for being here. Let’s dive in.
What Were the First Symptoms of Your MPN?
Ruth: We’re talking about understanding symptoms and what to track. One of the main challenges with MPNs is that the symptoms are often subtle and can easily be mistaken for normal aging or other conditions, like menopause. Nick, what symptoms did you notice first? You alluded to a few, but what was the first symptom that prompted you to start tracking?
Nick: The itching, for sure. Like you mentioned, a lot of symptoms can be dismissed, like headaches and fatigue, but the itching stood out. It wasn’t something I experienced before, so that raised the red flag and made me start tracking and monitoring the details around when it would happen.
Demetria: Probably the fatigue. I have a high pain tolerance level and the fatigue that I experienced would probably get to most people, but it wasn’t getting to me. My adrenaline was running, being a mom, a wife, and a business owner. I had a very demanding life, so I was going and going, and not paying attention to my body as much as I probably should have been.
It wasn’t until the day before I went to urgent care. I was having bad stomach pain, which I now know was because of my enlarged spleen. My doctor informed me that when your bone marrow is not making enough white blood cells that the spleen will kick in and try to do the job of the bone marrow and that’s not its job. In the process, your spleen gets enlarged because it’s doing something that it is not designed to do.
That day, I also had some shortness of breath, which was very unusual for me because I usually don’t get out of breath. My body was sending me signs and I ignored them until it gave me a dire warning. “We can’t go anymore. Something needs attention.”
I want to know every little detail about what I’m feeling and then I use that as part of my conversation with my doctor.
Nick Napolitano, Polycythemia Vera Patient & MPN Patient Advocate
Tracking Your MPN Symptoms
Ruth: What kinds of things do each of you track now to manage your MPN and how has that been helpful?
Nick: I track everything, but that’s me personally. I want to know every little detail about what I’m feeling and then use that in conversation with my doctor. I let my doctor tell me whether I’m crazy or not with respect to the symptoms I’m feeling. But I think it’s important to track everything.
So much of our disease is a journey over time. Things pop up and go away. I think you need to track everything and have the details around it. Make sure you’re communicating with your doctor because you never know what part of the journey or what symptoms you’re experiencing at any given time actually matter until you look at everything.
Demetria: I didn’t track symptoms until towards the end, maybe that last week before I received my myelofibrosis diagnosis. Each day, I was saying, “Wait a minute. I am really, really tired.” I would go to the grocery store, come back home, lie down, and would need to sleep for like an hour and a half.
The other thing I dismissed was a little bit of dizziness because I had just had a sinus infection, so they were thinking it was vertigo from the sinus infection. I thought everything was isolated and not related to one diagnosis. I thought they were all individual symptoms of something else.
The last week before receiving my diagnosis, I was noticing a little bit of progression. The fatigue was feeling a little bit more intense each day until that last day when I had severe stomach pain.
Ruth: How do you track symptoms? Is it through an electronic app or do you use a notebook?
Nick: It’s a combination of an app and a spreadsheet. I would say the most basic and useful tool for me is the notes app on my phone. I open it up almost daily and log stuff there. Then I use that as the basis for my conversations with my doctor. Everything is there between visits, so I can go over questions and things I notice.
It’s the fear of missing something that may play a role in changing the course of my disease or progression.
Nick Napolitano, Polycythemia Vera Patient & MPN Patient Advocate
What Motivates You to Track Your Symptoms?
Ruth: Nick, what’s helped you be consistent with your tracking? What’s made it easier and what helps you remember to keep it up?
Nick: It’s fear, to be honest with you. It’s the fear of missing something that may play a role in changing the course of my disease or progression. I’m very fearful of not tracking something, going to a doctor’s appointment, and being asked, “Have you experienced this before?” And then having to say, “I don’t know. I haven’t been tracking it.” Then, realizing that it’s too late. Quite literally, it’s fear.
Ruth: That’s real.
Noticing Your Symptom Patterns
Ruth: Let’s talk about symptom patterns. Do you ever notice your symptoms come and go, get worse at certain times, or come in waves?
Demetria: I don’t know that I necessarily noticed any patterns. It was an everyday thing. I was starting to notice that the fatigue was increasing every day. I don’t necessarily know the time of day, whether it was in the morning or the evening. I just knew that when I woke up, I was fatigued. When I went to bed, I was fatigued. Throughout the day, I was fatigued.
It’s unpredictable… You don’t know what’s going to happen from one day to the next when it comes to symptoms.
Nick Napolitano, Polycythemia Vera Patient & MPN Patient Advocate
Ruth: What about you, Nick? I assume that’s why you started tracking, because our symptoms do come and go. Do you ever notice that they get worse at certain times of the day or on certain days? What have you found in your tracking?
Nick: Yeah, I think that’s probably the most important part of tracking. I’ll give an example with itching. Itching was dormant for a while and then it popped up over the last couple of years. It’s not just random — it happens at certain times of the day. It pops up in the morning and at night, and then it goes away in between. When I take a shower, whether hot or cold, it pops up. When I roughhouse with my kids, the rubbing of my skin activates it. I used to have a lot of bone pain, but that’s gone.
One of the challenging parts of the disease is that sometimes it’s unpredictable. Things come and go. You don’t know what’s going to happen from one day to the next when it comes to symptoms.
Now I feel better prepared because I understand how important it is to pay attention to everything concerning your body.
Ruth: I think that uncertainty is palpable. A lot of us experience anxiety and stress related to that. Does tracking ease some of that anxiety because it helps make sense of things, or is it the opposite? Do you see those ups and downs and worry more because of it?
Nick: That’s a great question. It helps me because I feel like I’m educated and prepared when I go to my meetings. I call them meetings with my doctor and I treat it very much like a work meeting. I want to have all the information I possibly can when walking into that meeting. I feel a little more prepared. Having the knowledge of the ups and downs, and maybe the reasons why, is very, very important for me.
Demetria: Yeah, absolutely. One of the things my transplant surgeon pushed for was for me to be very observant of my body, like skin changes, because not only in terms of cancer coming back, but also graft versus host disease with having the transplant. Sometimes your body can reject the transplant. They were huge proponents of me making sure that I am paying attention to everything — not just how I’m feeling, but any changes in my skin, my eyes, my throat, my ears, or my range of motion.
Now I feel better prepared because I understand how important it is to pay attention to everything concerning your body and to make sure that I am letting my care team know, so that we can be ahead of it to be more proactive instead of reactive. Sometimes those subtle things that we don’t pay attention to could absolutely be the start of something. If you catch it early enough, you can stay ahead of it.
Don’t think, ‘Oh, this is probably not related to this.’ Express everything that’s going on in your body.
How Tracking Symptoms Can Inform MPN Treatment Options
Ruth Fein: Now we’re tracking our symptoms and thinking about when it’s important to share with our care teams. Has that directly led to any action or changes in your treatment? I’d love to hear any examples. We’re not talking about specific drugs, but has it led to a change in your treatment or your activities, Nick?
Nick: Yeah, for sure. I talked about the itching and brought that up with my doctor. We talked through the details of when it happens and when it doesn’t. He recommended a couple of different options and one in particular that has worked. I very much appreciated the dialogue and brainstorming. That’s one of the aspects I want from my doctor is brainstorming and idea generation. That’s been a life changer, to be honest with you.
Ruth: Tracking your symptoms and bringing that information to your health team can make a big difference. What tips do you have for talking openly, honestly, and clearly with your doctor and your entire care team about how you’re feeling? Do you have any specific tips? What helps you maintain an open conversation with your care team?
Demetria: One of the things, looking back, is I wish I hadn’t seen my symptoms as isolated incidents. I wish I had known to say to somebody, “I’m having these symptoms,” and list them all out, instead of thinking, “This may be related to the sinus infection. Maybe this is related to this.”
I wasn’t sharing with my oncologist or different people early on about other symptoms that seemed random. I didn’t mention those things when they could have been related to the progression of the disease. When working with your care team, it’s important to tell them everything that you’re feeling. Don’t think, “Oh, this is probably not related to this.” Express everything that’s going on in your body.
Ruth: Nick, do you have anything to add or something that specifically works for you to have an open and honest conversation with your practitioners?
Nick: I make sure that at the beginning of the appointment, we talk about how I’m feeling — mentally, physically, and symptom-wise. I try to talk about how I’m feeling in between visits. I’ve learned that doctors and caregivers want details. They’ll ask about what you’re experiencing and how you’re experiencing it. Coming prepared to the conversation right up front to talk about some of the details of what you’re feeling creates a nice dialogue with your doctor.
Ruth: It sounds like you have a perfect doctor because we’re always talking about how to have productive conversations with your doctor and care team. You’ve got it down and I’m sure our audience will learn something from that.
Nick: Ruth, I learned that over time. Believe me, I learned that over time. It’s not always perfect.
You can learn so much from other people’s experiences and gain real courage and support that way.
Nick Napolitano, Polycythemia Vera Patient & MPN Patient Advocate
Ruth: Same here. And it’s not easy for everyone. You’re communicative, I’m communicative, but some people aren’t. We always encourage bringing a care partner, someone who can interact on their behalf or listen closely, especially if they might miss something the doctor says. For those who aren’t as outgoing or find it hard to share how they’re feeling, that care partner can step in and say, “Remember last week, when…” or add important details. All of that helps. Thanks for sharing that!
Nick: I totally agree. That’s exactly why programs like this are so important. Connecting with the patient community makes such a difference. You can learn so much from other people’s experiences and gain real courage and support that way.
How Has Tracking Symptoms Changed Your Treatment?
Ruth: As one more follow-up to that, has any of your tracking changed not just your treatment, but raised new concerns that you then brought to your doctor? Questions and answers that perhaps would not have come up before?
Nick: It was the tracking of phlebotomies when I was being treated solely with phlebotomies and the impact that was having on me physically. For a while, I was able to deal with them well, but over time, they affected my overall health and well-being. I wasn’t tolerating them as well as I had in the past, so we started talking about different options. Not only were they not making me feel well, but I was also getting them too often, which is usually a red flag with MPNs. That changed the conversation toward exploring different treatment options for my care.
Ruth: That speaks to how important it is, as a patient or patient advocate. Sometimes doctors ask us yes-or-no questions, like, “Are you doing well with your phlebotomies?” and it’s either yes or no. But if you expand on that and say, “Yes, I’m going, and yes, they’re helping my blood counts, but I’ve been experiencing this or that.” If a person living with MPN or their care partner doesn’t step in to add to that yes-or-no question, we don’t progress in our knowledge and, therefore, better care. Thanks for going there. It’s important for others living with MPNs to hear different experiences.
I’m a woman of faith, so I rely heavily on my belief in God. It has anchored me throughout my journey.
Dealing with Fears About Your MPN Symptoms Returning
Ruth: Now, let’s talk about progression. It’s a topic nobody wants to face. Or maybe we all want to talk about it, but we don’t want to go there. We know MPNs can progress over time — for example, ET to PV, or for me, PV to MF, and MF can progress to acute myeloid leukemia (AML). That can bring up a lot of emotions, stress, and sleepless nights.
Sometimes we hear that sleeplessness is a side effect of living with an MPN, but I believe it’s not always a biological or molecular response from the disease itself. A lot of it is the stress. Personally, I don’t think about these things during the day, unless I’m doing a webinar like this. But when I close my eyes at night, that’s when everything starts swirling and it keeps me from sleeping.
It’s important to talk about the balance between facing reality, staying present, and being grounded in daily life. We don’t want anxiety or stress to take over, but it’s also important to acknowledge that it’s real. I believe it’s equally important for others to acknowledge that it’s real because sometimes we feel misunderstood. Demetria, are you worried about MPN symptoms coming back?
Demetria: I’m a woman of faith, so I rely heavily on my belief in God. It has anchored me throughout my journey. I don’t believe in crossing over into fear. It’s healthy to have concern and caution about things, but I try not to go too far into the future because that’s when anxiety sometimes can arise, because you’re thinking about the what-ifs. And sometimes the what-ifs never happen.
For me, I rely on my track record that God has always pulled me through. I believe that if something else were to arise, because I’m anchored in that belief that I’m taking care of spiritually, that it’s going to be okay. Sometimes we do have to go through journeys and valleys, but it’s how you respond to those valleys that makes the difference.
Ruth: Nick, what about you? How do you balance living in the moment and not worrying about progression? I also wonder: have you talked with your doctors about this? Because some doctors don’t always ask about mental health or make that connection, while others do.
Nick: I’ve been very open and public about some of the mental health struggles that I went through, especially early on. It was a very shocking and confusing diagnosis. I kept everything inside. I didn’t communicate at all with my wife or my family, and it was deteriorating my mental health.
What helped me was communicating. I communicated a little bit better with my wife. I did that through a documentary that I did, which was life-changing, to be quite honest with you. It wasn’t necessarily about the documentary, but it was about getting involved in the cause. It made me feel like I was doing something to help. You quickly realize that it’s not about you. There’s a greater cause out there. There are other people that you can help by sharing your story and communicating, and that was the turning point for me.
Connect with other patients. It’s so life-changing to speak with someone who is going through something similar and may have a different perspective.
Nick Napolitano, Polycythemia Vera Patient & MPN Patient Advocate
I did seek professional help. I remember going to the emergency room a couple of times because of the anxiety pains that I had, which I thought then was a heart attack. At that point, everything was all bottled up inside. Again, that was another pivot point to seek professional help.
Both of those things were life-changing. I talk about this all the time: don’t be afraid to communicate. You have to communicate because it is a release point. Get help and also connect with other patients. It’s so life-changing to speak with someone who is going through something similar and may have a different perspective. It is valuable.
Ruth: All of that is thanks to the internet. Obviously, when I was diagnosed 30 years ago, the internet was in its infancy. I didn’t have resources like The Patient Story, which are invaluable for those of us living with any chronic disease, particularly chronic cancer and an MPN.
Primarily my husband and then my mother… With both of them on my team, things that I didn’t say, didn’t know to say, or forgot to say, they would jump right in to say those things for me.
Ruth: Let’s move on to the role of care partners in symptom tracking. Do either of you have someone helping you track symptoms or notice changes that you might miss?
Demetria: Primarily my husband and then my mother. My mother has a background in the medical field. Sometimes she would accompany me on my visits and would ask questions that I may not have known to ask because of her background.
When I had my bone marrow transplant, I had to have a caregiver with me. My mother and my husband alternated because I had to go to another city to have the bone marrow transplant, which was two hours away from where we lived. My daughter at the time was 10, so he had to navigate caring for her as well as caring for me.
They were some of my biggest supporters. I remember one doctor visit. I was starting to itch a little bit. I hadn’t said anything to my oncologist about it and he said, “You need to tell him about the itching.” The doctor asks, “What about the itching?” I said, “Yeah, I’m having a little bit of itching.” With both of them on my team, things that I didn’t say, didn’t know to say, or forgot to say, they would jump right in to say those things for me.
Kara, my wife, is my caregiver and I don’t know where I’d be without her… She’ll ask me questions to make sure that I’m being honest about what I’m going to talk to the doctor about.
Nick Napolitano, Polycythemia Vera Patient & MPN Patient Advocate
Ruth: I know a lot of times people will go to their doctor and say, “No, I’ve been fine.” “Are you fatigued?” “No.” “Have you been tired?” “No.” Then the care partner says, “Well, we’ve had to rest a lot when we’ve gone on our two-mile walks every morning or when going up the stairs.” People in our lives play a role that I think is important. Have there been times when your caregiver noticed something you hadn’t picked up on at all yet?
Nick: Kara, my wife, is my caregiver and I don’t know where I’d be without her. We do a lot of communicating now. I’ve mentioned that I treat the doctor appointments like business meetings, so I will prep with her by going over my notes and what I’m feeling. She’ll ask me questions to make sure that I’m being honest about what I’m going to talk to the doctor about. Then we’ll do a debrief about what the doctor said, what the numbers looked like, and everything in between.
I give her a lot of credit for this. I was having trouble with my vision. I’ve had blurry vision before, but all of a sudden, I was having issues seeing. Everything was coming up blurry and she says, “You’ve been on a certain drug for a while. Do you think that has any effect? Why don’t you bring it up to your doctor?” Lo and behold, it does. There is an impact taking certain drugs with your vision. She’s fantastic. She’s a lot smarter than I am, so I rely on her a lot.
His presence and being willing to sacrifice to go through the journey with me were enough for me.
Ruth: Demetria, how does your care partner support you emotionally, particularly when you’re dealing with anxiety?
Demetria: Surprisingly, my husband and I have had these kinds of conversations, and he said that the way that I was so calm and remained calm through all of this helped him to stay calm. People look at the husband as the protector, as the one to shoulder the emotional burden to keep the household calm. But he shared that he watched me and how calm I was, which helped him. He felt as if there was nothing he could do to help me in terms of getting through this. We had to work through the process.
There is an emotional and mental component to someone simply being there. It makes you feel confident. It feels like you are seen. He sees me. He sees that I’m going through this. I feel secure because he’s here. He’s not leaving me by myself to go through this journey. Even though he may not have offered any specific emotional or mental help in the traditional sense, his presence and being willing to sacrifice to go through the journey with me were enough for me.
I heard from an MPN doctor that if we ask every person living with MPN if they were willing to participate in a study, 90-something percent would probably say yes.
Ruth: We’re moving on to clinical trials and looking ahead, which is important because some of the best drugs for MPNs are still in clinical trials. I like to say that clinical trials are no longer a last resort and I’m a perfect example of that. I’ve been in one for five and a half years, and I’m doing extraordinarily well on that trial.
There are challenges, however. I have to go back and forth 3.5 hours each way to the major academic center where I’m being seen, but I’m having a life-changing result, and to me, that’s worth it. By the same token, not everyone has access to clinical trials, which is difficult, and we acknowledge that.
Has your doctor ever brought up the idea of clinical trials as part of your treatment plan? I heard from an MPN doctor that if we ask every person living with MPN if they were willing to participate in a study, 90-something percent would probably say yes. The only obstacle is that they’re not asked. What about you, Demetria? Have you ever been offered a clinical trial?
Demetria: I haven’t ever been asked. But I have been told about them, especially when I was at Emory, because they do participate in a lot of clinical trials. The conversation was, “If this medication does not work well for you, then we could look at putting you in a clinical trial.”
He heard of a diet study that Dr. Angela Fleischman was doing to take a look at the Mediterranean diet and the impact that it has on MPNs. He thought that would be of interest to me.
Nick Napolitano, Polycythemia Vera Patient & MPN Patient Advocate
Ruth: What about you, Nick? Is that something you’ve been asked about?
Nick: I will answer this a little bit differently. When I first got diagnosed, I focused on my diet. I tried to take out a lot of the inflammatory foods I was eating and made that a focus of my overall care. My doctor knew that at the time because I talked about that openly with him.
He heard of a diet study that Dr. Angela Fleischman was doing to take a look at the Mediterranean diet and the impact that it has on MPNs. He thought that would be of interest to me, so I participated in it and it was nothing short of fantastic. It validated some of the things that I was doing were correct, but also exposed some of the things that I wasn’t doing, and some of the nutrients and proteins that I wasn’t getting based on my diet, and iron. We don’t need too much iron as MPN patients, obviously, but we need a base level of iron.
It was great and it was educational for me. It’s important for people to know that those kinds of studies are out there, outside of the drug clinical trials that we typically see.
Ruth: Thanks for mentioning that. Dr. Fleischman’s studies are interesting. We can learn a lot, not just from the Mediterranean diet, but from the Mediterranean diet’s influence, if you will — picking and choosing what works for us, but understanding inflammation’s effect on MPNs.
Have an open dialogue throughout your care with your doctor. I never want to feel complacent with where I’m at in my care.
Nick Napolitano, Polycythemia Vera Patient & MPN Patient Advocate
Considering Clinical Trials in the Future
Ruth: If you were asked, whether it’s today or when things change, what would your care team be able to do to make you feel comfortable exploring a clinical trial?
Nick: For me, it starts before talking about the clinical trial. I feel like you need to have an open dialogue throughout your care with your doctor. I never want to feel complacent with where I’m at in my care, so I’m constantly challenging my doctor. What’s out there? What can we be doing differently, if anything? What’s on the horizon? What if this happens, what do we do? Those are the type of questions that I ask my doctor, maybe every other appointment, and brainstorm.
If you’re having those discussions, it makes that conversation about clinical trials a little bit easier and more comfortable because you’ve already talked about it. Starting with having a regular cadence of talking about the what-ifs and what we could do if things change will help with the conversation about clinical trials and ultimately participating in them.
Ruth: Totally agree. Sometimes we have that conversation long enough that the clinical trial becomes an approved drug, and we don’t have to have that conversation anymore.
If there were newer things out there or that are coming, I want to be in the know and figure out how I can benefit.
Ruth: Have either of you ever come across a clinical trial in your own research that seemed interesting, but you didn’t know how to move forward or how to talk to your doctors about it?
Nick: I would say I think that’s where the communication part comes in with the patient community. Also, with organizations like The Patient Story, they can get you resources and someone to talk to to further vet that out, and then ultimately maybe find an MPN expert to talk through that in a little bit more detail.
Demetria: Initially, when I was diagnosed with ET, I was very young. I was about 34 when I was diagnosed. I did some research on the medication that she put me on and said to her, “My husband and I may still want to have children, and this medication is not going to fare well if that is the case.”
I researched some new medications and clinical trials that were out. There was one particular doctor whom I mentioned to her that I saw was making headway and introducing new concepts and treatment plans. I asked her, “Is there a way that you can see if these things would work for me?” I didn’t feel like she was willing to do anything other than what she suggested, so that’s how I ended up at Emory.
This is my life. I have to be an advocate for my life. I have a husband and a daughter. I want to live. And if there were newer things out there or that are coming, I want to be in the know and figure out how I can benefit from the newer things that could be better than what she was offering.
All options are on the table. I want to live as long as I possibly can and I will do whatever is necessary to make that happen.
Nick Napolitano, Polycythemia Vera Patient & MPN Patient Advocate
Ruth: If things change, Nick, are you open to participating in a drug clinical trial, if at some point that becomes the right choice for you?
Nick: Of course. The way I look at my disease and the care is that all options are on the table. I want to live as long as I possibly can and I will do whatever is necessary to make that happen.
Ruth: Thank you for sharing that. I will just add that science is advancing at unprecedented speeds. Thirty years ago, things were so different. Twenty years ago, ten years ago, even five years ago, for that matter. Within the last two or three years, our options have expanded exponentially. The longer we’re well enough to live a good quality of life while we wait for new and better treatments to come along, those decades add up, and that’s a good thing.
Do not think that any symptom is too insignificant. Track everything and let your doctor tell you whether you have to worry about a particular symptom or not.
Nick Napolitano, Polycythemia Vera Patient & MPN Patient Advocate
Final Words of Advice
Ruth: To move on to final reflections, what advice would you give to someone who was just diagnosed with an MPN about tracking symptoms specifically? Is there anything you’ve learned over time about how to talk to your doctors so they hear what’s going on and see that you’re a knowledgeable patient?
Nick: I would say track it all. Do not think that any symptom is too insignificant. Track everything and let your doctor tell you whether you have to worry about a particular symptom or not.
Be prepared for your doctor’s appointments. Do the research. Communicate how you’re feeling. Be open-minded with treatment options. Be open-minded in getting the care that you need, whether it’s an MPN expert or a mental health expert, if you’re struggling with that. But be open-minded, please.
Demetria: I would say to pay attention. Life is fast. I call this the microwave generation. We do everything fast. We want everything fast. And sometimes when you’re moving as fast as I was, you don’t get a chance to pay attention to what’s going on inside of you. Sometimes when you’re driving on the highway, you’re going so fast that you can’t see anything. But if you’re on the back roads, you have to slow down. You can’t go fast, but you’re able to see the scenery. When you slow down, you can pay attention to see what’s going on.
Sometimes, we need to slow down to pay attention and be intentional about looking at ourselves, looking at our bodies, and evaluating how we feel. How long have I been feeling this way? Don’t discount it as a fluke or an isolated incident. Take it seriously. Take it to your doctors and find out what’s going on so you can live.
Sometimes, we need to slow down to pay attention and be intentional about looking at ourselves, looking at our bodies, and evaluating how we feel.
Ruth: Thank you so much, Nick and Demetria, for sharing so openly.Your stories are powerful reminders of how even small steps, like tracking symptoms and writing them down, can have a big impact and make a real difference in managing your health.
We’d like to also thank our audience. If you’re living with MPN or caring for someone who is, we hope that this discussion has left you feeling more informed, supported, and empowered, and a little less invisible and misunderstood.
We want to thank our sponsors, Incyte and Karyopharm, for their support of our independent educational program. This allows us to be able to do more content like this one and at no cost to you. The Patient Story always retains full editorial control. While we hope that this is helpful, this is not a substitute for medical advice. Please still consult your own healthcare teams when making your healthcare decisions.
We want to hear from you. Please share your feedback on this discussion and also what you want us to cover next. Thanks so much from The Patient Story.
Thank you to Incyte and Karyopharm Therapeutics for supporting our patient education program. The Patient Story retains full editorial control over all content.
From Confusing Symptoms to Motherhood and Advocacy: Taja’s Polycythemia Vera Story
Living with polycythemia vera, a rare myeloproliferative neoplasm, changed Taja’s path entirely. She was diagnosed in 2015 after fainting spells and abnormal lab results. For months, doctors dismissed her concerns, telling her she was too young for cancer. By tracking her own labs and bringing them to a clinical director, she finally received the correct diagnosis.
At the same time, Taja was caring for her grandmother with pancreatic cancer. Experiencing illness both as a patient and a caregiver shaped her belief that gratitude reveals hidden beauty, even during pain and uncertainty.
When told she could not safely become pregnant, Taja and her husband sought specialists and pursued IVF (in vitro fertilization) during the COVID pandemic. Their efforts succeeded and they welcomed their daughter, Miracle. Afterward, her disease accelerated and she underwent a bone marrow transplant with her father as the donor.
Though the transplant caused ongoing side effects, Taja chooses to use her voice through Miracle Circle Hands, an advocacy group that supports people with invisible illnesses. Her hope is to show that even in hardship, life can still offer light and connection.
Key Story Takeaways
Self-advocacy can be life-saving when symptoms are overlooked or dismissed.
Gratitude became Taja’s core practice, helping her find meaning through illness.
IVF gave her and her husband the chance to welcome their daughter, Miracle, despite doubts from doctors.
The bone marrow transplant brought difficult side effects but also a path forward.
Taja transformed her experience into advocacy, creating support networks for others with invisible illnesses.
Name: Taja S.
Age at Diagnosis:
23
Diagnosis:
Polycythemia Vera (PV)
Symptoms:
Chronic fatigue
Fainting
Stroke-like episodes
Elevated hemoglobin, hematocrit, and platelet count
Treatments:
Emergency surgery for ruptured cyst & bowel obstruction
Chemotherapy
Radiation
Bone marrow transplant
Thank you to PharmaEssentia for supporting our independent 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 to make informed treatment decisions.
The views and opinions expressed in this interview do not necessarily reflect those of The Patient Story.
Friends and family describe me as compassionate, caring, and someone with a giving heart. I am very concerned about others, often less about myself.
When My First Symptoms Started
During my junior year of college, I was working two jobs. I was feeling off and not feeling like myself. I had multiple visits to emergency room doctors, primary care doctors, and a GI specialist (gastroenterologist).
The GI doctor wasn’t concerned about any gut issues, but he pulled up some lab work and he was extremely concerned about my red blood cells, my hemoglobin, my hematocrit, and my platelet count. At that time, I was working for the bone marrow transplant unit at a children’s hospital, which was pretty amazing and is why I’m where I am today.
My numbers were extremely high and very alarming, so I went to my primary care doctor and said, “I went to my GI doctor and he was extremely concerned.” But she blew me off, said that I was too young, and that there was nothing to worry about. She said it was probably an infection, so she would put me on antibiotics and I should be fine, and everything should level out. Afterward, I went back to my daily routine of going to school and work.
Then I had a car accident where I blacked out, which was alarming and extremely concerning. It wasn’t like I was deprived of sleep or it wasn’t my routine. At that point, I took it upon myself to do some research within my chart. I pulled some labs and got a visual of exactly what I was dealing with.
I was a health unit coordinator and doing a clinical rotation for social work. I brought my labs to the doctor, and then she looked at me and said, “You need to leave right now. You need to go see my husband,” who was also a hematologist. One of my coworkers took me straight to see him. He looked over my labs and gave me an idea that something was definitely going on, but he wasn’t quite sure what. But he drew some labs, did a full work-up and a week later, on February 14, 2015, I was diagnosed with polycythemia vera.
When Life Throws Everything at You at Once
My grandmother was diagnosed with pancreatic cancer around the time that I was diagnosed. At that time, her diagnosis was considered to be a death sentence. They told her that she had six months to live. But I took it upon myself to be there for her as much as I could, even while being in my last year of college and trying to be there for my family.
I was experiencing headaches and feeling fatigued. I couldn’t figure out what was wrong with me. I felt completely off. I was having fevers here and there. I was also having itchy skin and redness. Showers would bother me. I was getting extremely frustrated. But outside of that, I was so concerned about my grandmother and being there for her. I didn’t feel as if my family was on the same page, so I found myself overextending myself to make sure that I was there for my grandmother.
She was planning on retiring soon. One day, I noticed her eyes were yellow, so I asked her, “Why are your eyes yellow? Have you been at work all day and no one told you your eyes were yellow?” We were supposed to go to a family outing and I said, “No, we’re going to the emergency room.” Within that hour or two of being in the emergency room, she was diagnosed with pancreatic cancer.
Could I Start My Own Family?
I was told that pregnancy wasn’t an option and that I shouldn’t get pregnant because it wasn’t safe for me. At the time, research studies were very limited, so I didn’t have much information on what would happen if I were to bear a child. I got a second opinion at the hospital where I was at the time and they told me the same thing.
I went to my hematologist at the time because my fiancé and I were discussing it with her. After all, we were concerned about having children and we both wanted to have children, but she was totally against it. She said, “No, I don’t advise it.” She was so concerned.
I don’t think there’s enough conversation around reproductive health when it comes to women who have either had cancer or have been through cancer treatment. There are options.
My husband and I are faith-based people and understand that God has the last say. We went and did our due diligence and research, so we went to a reproductive specialist to get a second opinion. They did share some concerns with me about having children, but it wasn’t impossible, so we went through the IVF process. They told us, “This usually doesn’t happen on the first try, so don’t be upset if it doesn’t happen for you,” and put an emphasis on that. Knowing who God is and my relationship with God and how I’ve always tried to align who I am with God’s purpose, I put it in His hands.
We started the IVF process and did it during the COVID-19 pandemic, so a lot of the things were done at home. My husband and I did the process together, which was pretty amazing. He did all my shots and went to all the appointments.
After they did the implantation of the embryo, two weeks later, I found out I was pregnant.
Navigating My Pregnancy While Having Cancer
What’s funny, which I thought was eye-opening, was that during my pregnancy, my condition became dormant. I had no symptoms of polycythemia vera. All my numbers were normalized, which was pretty cool. I would go in weekly for labs and everything was normal throughout my pregnancy. No issues at all. No signs of stroke and no headaches. All I had were the typical pregnancy symptoms.
First, they told me that I couldn’t have children. Then, I had her, but I went through a traumatic experience of having a cyst rupture while six weeks pregnant and in the midst of COVID. Then they told me that our child may not live through the procedure. Her making it to this world was the main reason we call her Miracle. And she’s every bit of her name.
My Disease Progressed After Having Our Baby
Right after having our daughter, Miracle, my disease went haywire. Weeks after giving birth, my numbers skyrocketed. My platelet count was in the 2 million range weekly. They were also doing plateletpheresis (a procedure where platelets are separated from whole blood and collected) weekly. At the start of the week, I would have plateletpheresis and I would go down to a normal range, but by the end of the week, my platelet count was back to 2 million.
At that point, life-threatening things could occur. My hematologist said, “There’s no other option outside of having a bone marrow transplant.” He wanted me to understand exactly what would happen.
They started the process of preparing me for transplant, but mentally, I thought that we should probably speed it up. They felt that as well because my platelets continued to be in the 2 million range and a high number of platelets can cause clotting. They said that my blood was like syrup. Whenever they would draw blood for my labs, it would clot in the tube, which created an issue for them as they needed to do my labs weekly and had to repeat draws over and over.
Making the Decision to Do a Stem Cell Transplant
I had no options other than a stem cell transplant. I wouldn’t wish it on anyone. Being a person of color, I had no donors. There were no matches.
I had my bone marrow transplant at 29 years old. Having a transplant at 29 came with many uncertainties, especially not knowing whether I would ever be able to have children. Thankfully, because we decided to do IVF, my eggs had already been retrieved and frozen. However, for many young women who undergo a bone marrow transplant, things move so quickly that there’s often no time to consider fertility preservation.
In my case, if I hadn’t had Miracle when I did, I wouldn’t have had the chance to freeze my eggs at all. My only choices were to proceed with the transplant or not survive. My experience taught me how crucial it is for women to take charge of their reproductive health early in life, because without that foresight, I may never have had the option to have children.
After I had my transplant, my body was thrown into menopause. My hormones are completely shot. I don’t have a sex drive whatsoever and I’m only 34, which is hard being married. Being a woman, it’s hard for your hormones to be off because you experience moodiness, hot flashes, and irregularities within your body. It’s extremely difficult. I’ve spent a lot of time researching different options, trying to understand what might work best or be most helpful for me.
I will be forever grateful that I was able to have the transplant and that I’m here, but it’s definitely stressful to be in this space now. The uncertainty I faced before the transplant — wondering whether I would survive or not — still weighs more heavily on me than where I am today. I’m extremely grateful that I did the transplant, but it has taken a toll on my mental health. I now try to raise awareness and help others understand the challenges that come with the experience.
Life After a Stem Cell Transplant
During my transplant, I had to go on disability. After a year, like many corporate organizations, they put someone else in my role. I eventually went on full disability and haven’t been able to return to work, as it’s been one challenge after another. However, following my heart and pursuing what I believe I was meant to do led me to create the organizations I now run, allowing me to give back to the community and find purpose again. While I’m a mother and a caregiver, I still feel a deep desire to do more because I believe my voice and experiences have meaning and need to be heard.
In my advocacy work, I focus on starting those uncomfortable but necessary conversations. Yes, the main goal is to save your life, but what happens after the transplant? What does life look like then? I believe those are the kinds of questions that need to be discussed from the very beginning.
From the research I’ve done, I’ve learned that many marriages end either during or after a transplant, which is partly because families and loved ones aren’t given enough information upfront about what to expect. Mental health support is so important from the very start. I truly believe it’s essential to find a therapist, counselor, or someone who can help you navigate those challenges — whether that means starting antidepressants or just having someone ready to talk about it.
During my transplant, I was on antidepressants and even then, it was very lonely. No one really prepares you for that part. You feel completely isolated because no one can truly understand what you’re going through except for another patient. But since you’re immunocompromised, you can’t even connect with them in person. I lost a lot of friends during that time; they just disappeared and no one warned me that might happen. Honestly, there should be a survey asking, “After your transplant, what did you go through? How did you feel? What could we do differently?”
Right now, I’m focusing on helping others in the community, not just myself. I previously had an organization called “Invisible to the Eye,” but I’m now in the process of creating “Miracles Circle Hands.” This new organization is all about building advocacy in the community, not only for those living with invisible illnesses, but also for seniors. I’m dedicated to speaking up for those who can’t advocate for themselves.
I named the organization after my daughter because she’s truly been our miracle. The “Circle Hands” part represents the idea of coming together — when you’re advocating for people, you’re out in the community connecting resources and joining hands to make sure patients, loved ones, or anyone in need gets the support they deserve. To make that possible, we have to create a safe space where people feel comfortable being vulnerable and sharing what they’re really going through. Unfortunately, many individuals are taken advantage of — whether because of illness or age — and I want to be someone who steps in to help. My goal is to show families, loved ones, and people in the community that they’re not alone, that someone truly cares, and that there are resources available to help them find their way.
My Message of Hope
My hope is simply to wake up each day. I hope to pour back into others and to do what My Father did for me: give someone else another chance at life, something I’ll always be grateful for. I remind myself every day that the more grateful your heart, the more beauty you see, and I truly live by that. Even though I face challenges, I’m thankful to still be here. The more gratitude I have, the bigger my heart feels, and the more I can give back to my community and serve others. I also want people to know that there is light at the end of the tunnel. Yes, there are ups and downs, but that’s part of life. And when you finally reach that mountaintop, you realize you’ve triumphed. I was told many times that I wouldn’t still be here, but I am.
Thank you to PharmaEssentia for supporting our independent 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 to make informed treatment decisions.
The views and opinions expressed in this interview do not necessarily reflect those of The Patient Story.
How Tabbie Faces Stage 4 Neuroendocrine Pancreatic Cancer at 25
When Tabbie first heard the words “It’s cancer,” she was just 25 years old, full of energy, and living her life in the Bay Area while working at a hospital. She didn’t think she had symptoms. After all, who wouldn’t feel tired after working long night shifts and driving an hour and a half home? But after a sudden, sharp abdominal pain that grew worse by the day, she decided to seek medical care, a decision that changed everything.
At urgent care, the pain was so severe that even a gentle touch was unbearable. After multiple scans, doctors discovered tumors in Tabbie’s pancreas and liver. Soon, the diagnosis became clear: stage 4 neuroendocrine pancreatic cancer. Hearing that news was devastating, especially since a close friend had recently died of cancer. Tabbie’s first thought was, “I’m going to die,” but she quickly realized that she didn’t want this diagnosis to define her.
What makes Tabbie’s story especially powerful is the way she leaned into humor and positivity, even in the darkest moments. She cracked jokes with her nurses, tried on wigs for fun before chemo, and kept her spirit alive by staying connected with friends and support groups.
Still, the weight of decisions was heavy, especially when her medical team talked about freezing her eggs. At 25, Tabbie never thought she’d be injecting herself with hormones to preserve her fertility, but she chose to embrace the process and hold onto hope for the future.
Preparing for her first chemotherapy treatments was overwhelming. Tabbie started with oral chemo, hoping for a gentler option, but when it didn’t work, she moved to IV chemo and eventually underwent a Whipple procedure, also known as pancreaticoduodenectomy, along with a liver resection. (Editor’s Note: A pancreaticoduodenectomy, commonly known as the Whipple procedure, is a complex surgery to remove cancerous tumors from the pancreas. It involves removing the head of the pancreas, the duodenum, part of the bile duct, and the gallbladder.)
Surgery was frightening, but she found strength in the fact that her care team was made up of trusted colleagues from her own hospital. That trust, along with support from social workers, nurses, and online communities, became her anchor.
Even after surgery, Tabbie faced challenges like digestive issues, dietary restrictions, and the emotional impact of feeling “different” from others her age who were building families or careers. Despite all this, she continues to choose joy, reminding herself and others to love their bodies, ask for support, and not let negative thoughts take control. She openly shares her experience on social media and in support groups, offering advice to others preparing for Whipple surgery or chemotherapy.
Tabbie’s story highlights not just the medical side of neuroendocrine pancreatic cancer, but the deep mental health impact of survivorship. By speaking candidly about her fears, choices, and resilience, she empowers others to take control of their care, honor their feelings, and know they’re not alone.
Watch Tabbie’s interview or read the full transcript below to find out more about her story:
How Tabbie turned fear into laughter while preparing for chemo
The surprising age when she was told to freeze her eggs
Why she trusted her medical team like family
The hardest part of recovery after her Whipple procedure
How support groups and social media gave her strength on tough days
Name: Tabbie V.
Age at Diagnosis:
25
Diagnosis:
Pancreatic Neuroendocrine Tumor (pNET)
Staging:
Stage 4
Symptoms:
Abdominal pain
Unusual organ “inflammation” feeling when walking
Fatigue
Treatments:
Chemotherapy: oral and IV
Surgeries: Whipple procedure (pancreaticoduodenectomy) and liver resection (partial hepatectomy)
This interview has been edited for clarity and length. This is not medical advice. Please consult with your healthcare provider to make informed treatment decisions.
The views and opinions expressed in this interview do not necessarily reflect those of The Patient Story.
I’m a very loving and fun person. You’ll never see me upset because I don’t have that mentality where I need to let people know that something’s bothering me. I’m usually cracking jokes.
About four years ago, I moved to the Bay Area in California and I’ve been working at a hospital ever since.
I didn’t think I had any symptoms
I didn’t have any symptoms, or at least I didn’t think I had any. I work swings, so I go into work at 3 p.m. and get out by 1:30 in the morning. At the time, I lived far from work, about an hour and a half away, so the drive was exhausting and I assumed that’s why I was tired.
But one Monday, I was on a caregiving job in LA for an older woman, which involved lifting her off the chair and putting her anywhere she wanted. She was petite, but I’m also petite. I’m only 4’11. The following day, I called in sick because I had abdominal pain. I didn’t know if I was sore or if it was actual pain, so I ignored it. It was something that I hadn’t experienced before, but I let it go until Wednesday.
I had to fly back to the Bay Area for work and my abdomen hurt a little more, but it was a weird pain because every step I took, I felt my organs were inflamed. When I arrived, I texted my boss and asked if it was okay if I went to urgent care once the following person came in and she told me to go immediately.
I decided to go to urgent care
I was physically at the hospital, but I didn’t want to go to the emergency room, so I went to urgent care. The pain was intense. When the doctor palpated my stomach, he couldn’t even touch me; that’s how painful it was. I thought the worst, like twisted or dead bowels.
He said, “You need to go to the emergency room.” I said, “Do you think it has to do with my bowels being twisted?” He said, “I don’t know, but you need to get a scan done.” The hospital was a five-minute drive, so I went back to where I worked, but to the emergency room. I felt weird because I was in my scrubs, sitting in the waiting room, so people were looking at me.
When they finally took me in, they did a series of scans and asked me questions. I didn’t have a fever, diarrhea, nor was I throwing up. They asked me if I was around livestock or if I had left the country. They were wondering what could possibly be causing my symptoms. They did an ultrasound and that’s when they saw that I had cysts. Then they asked if I had been around a sick dog. My dog has been throwing up for a day, but nothing horrible. They thought that’s what it was and that I had a tapeworm. They decided to do a scan and saw it in my pancreas and liver.
They didn’t tell me that they still thought it was a tapeworm as I got admitted into the hospital. I was speaking back and forth to a bunch of doctors and eventually, one of them came in and said, “It’s cancer.” I didn’t know what to think. It was like how you always wondered. Nonetheless, my mom and my boyfriend were there.
Thankfully, with the type of cancer that I have, I know a surgeon whom I work with and who specializes in Whipple procedures, pancreatic damage, and liver surgeries. I contacted one of the nurses whom I work with. She’s my charge nurse. She said, “This is the doctor you’re going to see.” It worked out for me in the end because I had a care team that knew me and I was able to pick out my team because I worked alongside them.
The moment everything changed
As soon as they diagnosed me and told me that I had cancer, I started crying. That’s a normal reaction for everyone. The first thing that popped into my head was, “I’m going to die.”
It’s still hard to talk about it. How do I explain it? A year before I got diagnosed, I had a friend who passed away from cancer, so that’s the first thing that popped into my head. Obviously, no one should think that way, but, unfortunately, that’s the reality of things. That’s the first thought, “I’m going to pass away.” As time went by, after I cried it out, I realized that I can’t let cancer determine who I am.
Another thing that popped into my head was the thought of losing my hair. Immediately, I got on Amazon for wigs. My boyfriend’s sister loves to wear wigs to style them, so one day, I came over to her house and she said, “This wig would look good on you.”
A whole bunch of negative thoughts popped into my head and I realized: what good is that going to do for me? I’m not that person. I’m not a sad person. I’m always the life of the party. I always like making people laugh in the most uncomfortable situations.
That’s what I did when I was in the hospital. I was waiting for a biopsy on my pancreas and they didn’t let me eat for close to 34 hours. I made a joke to the nurses, which I probably shouldn’t have in that moment. I said, “I guess hunger is going to kill me before the cancer does.” They all looked at me like, “Are you okay?” It’s just a joke. That’s how I cope.
The cancer had spread
I was still admitted to the hospital during all this. On the first night, I had CT scans, an MRI, and ultrasounds. I was in the hospital for three days. They wanted to do a biopsy on my liver, but based on the scans, they saw that the tumors were fluid-filled, so they decided not to mess with them. They went to the pancreas to look at the two tumors there.
I was waiting and wasn’t allowed anything by mouth for the whole day. Finally, they brought me down to get an EGD (esophagogastroduodenoscopy) done, where they put me to sleep, so they could put a tube down my throat and biopsy my pancreas. I got the results the following day. I don’t remember exactly what test they wanted to do, but I knew that the section where they wanted to do the test didn’t run on weekends. I said, “I work at the hospital. It’s not like I’m going anywhere. Can I just go home and be scheduled for whenever I need to do that?” They agreed and were able to discharge me.
I followed up the next week to do the test to confirm the type of cancer it was, which was neuroendocrine. But it was a series of waiting games because initially, they told me it was stage 1. Unfortunately, they could not test the liver due to the fluid-filled sacs. I remember getting that phone call and they said it was stage 4 because it metastasized to my liver. From what I remember, I had two large tumors on my pancreas and about 17 on my liver.
When I heard it was stage 4, it was a little more serious for me because they hadn’t given me any information while I was in the hospital. The most that I could do was cry because I was sick.
Immediately, I ran to Doctor Google. I searched “pancreatic cancer stage 4” and I read that pancreatic cancer is the deadliest and that people don’t find out about it or get symptoms from it until they’re at the end stage, so that scared me even more. Why am I on Google? Let me get off of this because it’s not doing me any good.
I cried more when I heard it was stage 4 than I did when I was in the hospital, mostly because I was by myself. I didn’t have a circle of doctors where I was trying to hold back my tears to be able to talk to them. But it was scary because it was just me and my best friend in the house, but she was sleeping because she had just gotten home from a shift, so I didn’t know what to do. I was going crazy. I needed to talk to someone about this and everyone’s at work. I ended up waiting until she woke up, walked into the room, and dropped the news on her.
At this time, I was doing my prerequisites to apply to the nursing program. I was also in the process of moving out of the Bay Area and into LA with my boyfriend. Everything had to be put on hold. Because my care team was in the Bay Area, I decided to stay at my job because I have my doctors right there and for insurance reasons. If I moved to LA, I didn’t know if I would find a good job that would provide good insurance. I had to put school on hold. I had to focus on myself.
There are a lot of things you don’t realize that people go through before they start chemotherapy. I was only 25 at the time. They told me about freezing my eggs. I was injecting myself three times a day. Sometimes I’d be too scared because I don’t like needles, so I would ask one of the nurses to inject me. I was constantly going back and forth between the Bay Area and LA until I started chemo. That’s when it got a little harder because I couldn’t travel as much. I felt like I was getting sicker.
Working in a hospital, I had direct contact with a great medical team
I was lucky enough not to run into any issues because I had resources. It all unfolded. I had a lot of people by my side helping me and making sure that I was on the right path. People around me in the hospital didn’t know that I had cancer. Everyone was in the dark but we made it work.
I had contact with my surgeon. He provided his cell phone number and told me that if I had any questions, I could text him. I had a social worker who gave me all the information, sent me everything through my health portal, and said, “If you have any questions, reach out to this person. If you want to know more information, here’s a link to information from different sites.”
My surgeon initially told me that the end goal would be to go through a Whipple procedure (pancreaticoduodenectomy), but he would like me to have chemotherapy first. He wanted to shrink the tumors as much as possible, so it’d be a lot easier to go in there.
But he did give me some treatment options. The first one was a clinical trial. I don’t remember if it was radiation, but he told me that one of the risks would be potentially having a different type of cancer, potentially a blood cancer, down the line. I said, “Why would I want to treat this cancer and then risk getting a different type of cancer in the future?” Then he gave me another option, which was oral chemotherapy, and see how that plays out. If that worked out, we would continue with that and if it didn’t, we would start IV infusions.
I never imagined I would have to worry about my fertility at 25 years old
When they told me that I should potentially freeze my eggs, I said, “Let’s do it.” I’m 25. I don’t have any kids. I would like to have the opportunity to have kids. I didn’t know what I was getting myself into. I didn’t know what the process was.
I had my first consultation and they explained it to me. They said I would be injecting myself. I would be coming in every other day to get a transvaginal ultrasound. They’re going to get bloodwork done every day to monitor, which was painful in itself. But the silver lining of that was when I would get my ultrasound done, the doctor would say I was very fertile. I always thought that I was infertile. I don’t know why that was in the back of my head, so hearing that made me feel a little better. I now know that I will be able to have kids in the future.
The treatment plan I chose
I chose to do oral chemo, which, when I researched, wasn’t as aggressive, so I thought I could manage it. I was on chemo for two weeks and then I had a break for a week before I started again.
Luckily enough, my boss worked with me so that I could do a hybrid schedule. I could work from home while I was on chemo and then when I was off, I could go to work. For the two weeks that I was on the oral chemo, I would work from home and then during the one-week break, I would go back to work. It was a constant back and forth, which I did for about three cycles.
After three cycles, we found that it wasn’t working. The tumors weren’t shrinking, so I got put on IV chemo for another four rounds. Again, that still wasn’t working, so that resulted in immediately going into the Whipple because they wanted to deal with the source before it got worse.
I was terrified. I’ve never had surgery. I’ve never broken a bone, sprained an ankle or wrist, nothing. I was never in the hospital until now. The idea of surgery terrified me because I didn’t know what to expect.
They told me that I would need an epidural and, obviously, the first thing you think of is a needle in your back. I didn’t want to do that, but I had no choice. I needed to choose life over whatever fear I had in my head.
He explained to me that with the Whipple, they would make an incision from the middle of my chest to my belly button. He said, “Essentially, it’s going to be like the size of your iPhone.” I thought it wasn’t so bad. I didn’t realize that on your abdomen, it’s pretty big.
He explained that they would take out my gallbladder completely, a portion of my stomach, my pancreas, and my small intestine, and group them together to make a new digestive system. Since I had tumors on my liver, he did a resection of the left side of my liver because that organ is able to regenerate after a good amount of time.
The procedure doesn’t sound fun. This is the worst surgery you can think of. He said, “It’s a very intense surgery that can take up to nine hours.” I’ve seen him do these surgeries so many times that I thought it should be a piece of cake for him. He cares about me. He trusts me and I trust him. I put myself a little more at ease.
I’ve had conversations with nurses who have also been in the operating room with him and they said he’s great and good at what he does. It put me more at ease knowing that I had my team of people who I knew cared about me and I trusted to take care of me.
I’m taking control of my treatment moving forward
After the surgery, they told me that they were going to monitor me every three months with a CT scan. Because the previous chemotherapy treatment didn’t work, there’s no point in putting me back on a treatment that is potentially not going to work.
They did want to put me on hormonal injections every month, but I opted out of that. I said, “Listen. I’m still healing from my surgery. I’m still going through the motions of finding my new normal. I also don’t want to be miserable and get an injection that you can’t prove will work. Why would I put myself through that? I want to heal on my own. I want to eat healthier rather than put myself through more pain and misery.”
I still have about 14 to 15 tumors in my liver, but I’ve been getting a scan every three months. Thankfully, the tumors have been shrinking on their own without any treatment. Essentially, he wanted to do another liver resection to try to do the right side of the liver. I asked if there’s a need to do surgery right now or if we can continue monitoring every three months and if they continue shrinking, if we could push the surgery as much as possible. He said it wasn’t a bad idea. I seem to be stable, so we’ll keep doing scans and blood work every three months, and go from there.
I’ve had to adjust my diet and workouts
Since my Whipple, there are a lot of things that I have to learn not to eat, like broccoli. I love broccoli, but I can’t eat it because, unfortunately, it makes me gassy and the gas hurts. I get bloated like a balloon quickly. I can’t eat a lot of dairy products. I used to drink milk, but I can’t do that anymore.
The other difficult part is when I wake up in the morning and I drink a cup of water, I have to run instantly to the bathroom. I have something called dumping syndrome, where anything I eat or drink, I go straight to the bathroom and it all comes out.
Since the surgery, I’ve lost 20 pounds and I haven’t gained it back. I haven’t lost more weight because everything I eat just comes out and it sucks. I like to go on vacations with my friends and I worry about going out to eat because I need to have a bathroom within two feet because I’m constantly going. It sucks. No one wants to live like that.
Aside from that, I haven’t had any moments in my life that changed drastically. I coped with the chemotherapy and didn’t let it take over me. I still functioned as normally as I could. I still lived my life. I still went back home to New York and saw my friends. I still went on trips. I went to Puerto Rico and Hawaii. I went swimming with the sharks. It was only the Whipple that made such a change in my life. But even then, I’m still very hard-headed and eat food that I’m not supposed to eat, then pay the price later, but only when I’m home. If I’m outside, I won’t do that. I’ve been eating a lot of fruits I stayed away from. Fast food doesn’t do any good and I feel disgusting after.
I feel like I haven’t had the normal lifestyle of a 25- to 27-year-old because people around me are getting married or having kids. Having to put my prerequisites on hold sucked because I was in the zone. I feel like I only had three prerequisites left until I could apply for the nursing program, but chemo got in the way. I could not stay awake long, so I unfortunately had to quit until my situation was resolved, especially going back and forth from the Bay Area to LA.
There are certain things that I can’t do. I see people doing Pilates. I have no core strength anymore and if I try, it’ll hurt me and I could possibly get a hernia. I can’t participate in that and it sucks. Yes, I can do a class for beginners, so it’s easier, but I would want to experience that. I have to be a lot more cautious than other people.
Where I find support
I love to go on TikTok. I love to do my own research. I found a support group on Facebook. While going through healing from the Whipple, that page helped a lot because if I had questions, I would pop it in there and a ton of people would give me their response, what they went through, and what worked for them. I was able to cope with that.
If I felt sad or in the dark, I’d pop a message on there because they can relate. Yes, I can run to my boyfriend and tell him I feel this, this, and this, but he wouldn’t understand. It felt a lot easier for me to speak to strangers, but they weren’t strangers to what I was going through, so that was very helpful.
Until this day, a lot of people, especially on TikTok, where I post about my experience, would message me and say, “Hey, I’m going to go through a Whipple. What do you suggest?” I’ll give them my suggestions. But I also refer them to the Facebook support group so that they can see what everyone’s going through and ask their questions.
What I want others to know
Be kind to your body. You have to love yourself. Understand and accept the fact that you’re sick and go from there. Everything is a mental game. If you constantly think the worst, you’re going to put yourself in that mental state and feel sicker because now you’re feeling depressed.
I forced myself to get up from bed every day. I forced myself to talk about it because it’s one thing to have those thoughts in your head and it’s another thing to talk about it out loud. It makes you feel better. When you let it out, you’re hearing what other people have to say. Positive feedback helps a lot.
I love the support group for Whipple Warriors. I gave an eighth-month post-Whipple update and people were saying, “Stories like yours inspire me. You’re doing great. I’m so happy to see that you’re doing amazing. Keep going. Keep fighting. You’re a warrior.” Those are better compliments than being told you’re pretty or you make it look so good because they’re coming from people who are also going through the same thing as you. I look up to older people. It’s like a warm hug and you get that warm feeling.
Symptom: None; found the cancers during CAT scans for internal bleeding due to ulcers Treatments: Chemotherapy (capecitabine + temozolomide), surgery (distal pancreatectomy, to be scheduled)
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Emily Breaks Stigmas and Builds Awareness Around Stage 4 EGFR+ Lung Cancer
Emily never imagined that a lingering cough would lead to a diagnosis of stage 4 EGFR+ lung cancer in September 2024. As someone who is active and outdoorsy, she initially chalked her symptoms up to Austin’s notorious allergy season. But when she struggled to breathe during a familiar hike and noticed her voice changing, she knew something was off. Even then, cancer wasn’t on her radar until scans confirmed the diagnosis.
At first, Emily felt overwhelmed, thinking her life expectancy had suddenly been cut short. However, her oncologist reassured her that while stage 4 EGFR+ lung cancer is incurable, it’s treatable and treatments have come a long way. That perspective helped her move from despair to action. Emily chose both chemotherapy and targeted therapy, later adding radiation, and now she’s stable with minimal side effects. She enjoys being active again and even swims with her neighborhood team.
Throughout her experience, Emily has leaned heavily on her friends, family, and wider community. From organizing rides to appointments to surprising her with a fully cleaned and organized house, her circle showed up in ways she never expected. She emphasizes that asking for help is not a weakness; it’s a gift that allows others to love and support you.
Emily also became vocal about advocacy, especially within the Asian community, where awareness about stage 4 EGFR+ lung cancer is still limited. She shares her story and reminds others not to dismiss persistent coughs or shortness of breath. She hopes that by speaking up, more people will push for answers earlier and potentially catch cancer sooner.
Another pillar of her healing has been focusing on mental health. Emily admits that when treatment began, she was too overwhelmed to join support groups, but therapy later helped her process the uncertainty of living with advanced cancer. Practicing mindfulness and giving herself grace has been essential. She’s learning to appreciate everyday moments, like petting her dogs, spending time with her kids, and cherishing the present, without constantly worrying about the future.
Stage 4 EGFR+ lung cancer does not mean life is over. With evolving treatments and community support, it’s possible to live fully and meaningfully. Emily’s story reminds us that resilience often looks like honesty, vulnerability, and the courage to ask for help.
Hear directly from Emily in her video or keep scrolling to read the full interview:
How a “simple cough” turned into a diagnosis of stage 4 EGFR+ lung cancer
The powerful ways Emily’s friends and family rallied around her
Why Emily is raising awareness about lung cancer, specifically within the Asian community
How therapy, mindfulness, and giving herself grace helped Emily find peace
The hopeful perspective her oncologist shared that changed everything
Name: Emily N.
Age of Diagnosis:
46
Diagnosis:
Non-Small Cell Lung Cancer (NSCLC)
Staging:
Stage 4
Mutation:
EGFR
Symptoms:
Chronic cough
Persistent post-nasal drip
Shortness of breath while doing simple activities
Changes in voice
Rib pain
Treatments:
Chemotherapy
Targeted therapy: tyrosine kinase inhibitor (TKI)
Radiation therapy
This interview has been edited for clarity and length. This is not medical advice. Please consult with your healthcare provider to make informed treatment decisions.
The views and opinions expressed in this interview do not necessarily reflect those of The Patient Story.
I live in Austin, Texas, and I was diagnosed with stage 4 lung cancer with an EGFR mutation in September 2024.
I would say that I’m an ambivert, a little bit quirky, and pretty dorky. I love nature and animals. I try to love my plants, but they don’t always love me back. I love to read, swim, and trail run. I have a very large support network that I have been very blessed with.
When I first felt something was wrong
In the fall of 2023, I started developing a cough, which I didn’t think much of. I live in Austin. The allergies are insane. They’re tough, so I didn’t think much of it.
When the cough persisted into early 2024 again, I didn’t think much of it because now we’re into cedar season. There was always something on top of it. I’m pretty active and healthy, so I never considered that anything was wrong.
Around May 2024, I went to a doctor because I could feel that I had a persistent post-nasal drip, which he confirmed. I mentioned a cough, but he didn’t seem too concerned about it. He prescribed some nasal sprays and sent me on my way.
It was on August 31st when I realized that something was wrong. I went for a hike with my partner and it was hot. But as we were finishing the hike, which had a decent uphill grade, I couldn’t breathe. I was doubled over and couldn’t catch my breath. This is a hike I’ve done many times, so I was a little bit surprised, as was he. I chalked it up to the heat.
But by then, my voice had already started to change. I had no projection in my voice. It was scratchy. Sometimes it would get high-pitched; other times, it would get a little deeper. I didn’t think much of it because I thought it was because I was coughing a lot.
But when I realized I was having some difficulty breathing on a hike that I did all the time, it became my first red flag. Then I started noticing little things like getting winded going up stairs or doing a trail run and having trouble breathing. That’s when I freaked out and realized I needed to see a doctor about this.
The moment everything changed
When I went to a new primary care provider (PCP), I thought she was going to say I have asthma or bronchitis. Never in the realm of possibility did I think I was going to have lung cancer. But she took everything very seriously. She sent me to get a chest X-ray, referred me to an ENT, and ordered blood tests.
I got a chest X-ray that same week and then she called me on the day I was supposed to go with my best friends on a girls’ trip to celebrate a birthday. She said that there were concerns with my chest X-ray. There were nodules and she wanted me to get a CT scan when I returned. She mentioned the possibility of cancer, but she said it also could be nothing. So when my best friend walked in the door to pick me up to go to the airport, the first words out of my mouth were, “I might have cancer,” and we started freaking out. This is a great start to a girls’ trip.
When I got back, I got my CT scan done on a Friday and the physician assistant (PA) called me that Sunday, and it’s never good news when your PA calls on a weekend. I was out and almost didn’t pick up the call because I didn’t know who it was, but I’m glad I did.
She introduced herself and I asked, “Do I need to sit down?” She said yes. She explained that the nodules were cancerous. I had a bajillion questions and rightfully, she said, “I’m not an oncologist. I can’t talk about staging.” She did give me her personal cell phone number for support and then said, “I’m referring you out to Texas Oncology.”
At that time, I hadn’t told anybody other than my two best friends with whom I went on the girls’ trip. I called my sister on my way home, went straight to another best friend’s house, and banged on her door and said, “Let me in. I need you.” We held each other and sobbed. That was a rough day.
The cancer spread
That same best friend took me to my appointment and my oncologist explained that this was stage 4. It metastasized to other parts of my body. I found out later exactly where it all went. There were quite a few places.
One thing she indicated was that I had a spot on my eighth rib bone on my right side, which explained why, over the summer, I apparently had fractured it and had no idea. I thought it was an intercostal rib strain. I would have no reason to think I would have fractured a rib, but she explained why I was in so much pain there.
I went into the appointment saying, “How can I have lung cancer? I’m a nonsmoker. I’m 45 years old. I’m active. I’m healthy.” She explained that I perfectly fit the demographic of EGFR lung cancer. At that point, it was the end of September. I still hadn’t had any biopsies, so she had me admitted to the hospital. I was there for about five days for a bunch of tests. They did a lung biopsy, checked out my pancreas, and did another MRI. That week was a blur.
My oncologist is very well regarded within the community here. Every time I mention her name, other patients would say she’s the best. Other medical professionals I went to also say she’s the best. At one point, she explained that she was considering working at MD Anderson and decided not to. My other thought was to go to MD Anderson, but that would have been logistically very difficult. I completely trusted her judgment and my case seemed like an open-and-shut case.
How I educated myself about my diagnosis
I knew absolutely nothing because in my head, lung cancer was for smokers. When she mentioned that, of course, I started Googling everything I could about EGFR. It came down to the question: Why me? What could I have done differently? It just came down to bad luck. Had I known that a chronic cough was a symptom, I would have gone to the doctor immediately. That’s when I started speaking about this type of lung cancer because I got a sense that it wasn’t well-known, especially in the Asian community.
At the time, I didn’t join any support groups. I thought it would be too overwhelming because initially, it was all too much. A lot of the information, especially before my first appointment with my oncologist, was very grim. There was a two-week period when I had no idea what to do. I just knew I had stage 4 cancer.
At the time, my boys were 12 and 14. I wanted to be as open and honest with my kids as possible. I didn’t want to hide anything from them, so I was very real and honest. I said, “This is scary. I don’t know what’s going to happen. The worst thing that could happen is something bad happening to you guys. The second worst thing is me leaving you too soon.”
I was worried that I wouldn’t see my youngest son graduate from high school. They’re still babies. They’re not full-fledged adults. I wasn’t ready to leave them. I needed to process all the fears and worries by myself. I thought that if I joined a support group, I would spiral deeper.
For my kids, we had them go to Wonders & Worries in Austin. They went through a six-week program, which I think was helpful for them. Once I got through my hospital stay, process, and understood that this wasn’t necessarily a death sentence and it was treatable, my mindset changed. I didn’t need therapy at that time because I needed to survive. I needed to process everything. I’m in therapy now, but at that time, I was in my head too much to even talk about it over and over again.
It was absolutely sobering. My immediate reaction was I’m screwed. I’m dead in a few years. Of course, this was before my oncologist said anything. I thought, “This is it for me.” It’s probably the scariest news someone can get about their health, especially having no idea that you were sick to begin with.
The primary was my left lung. I had innumerable small nodules in my right lung. It had also spread to the rib, sacrum, and pancreas, and I also had a tiny spot on my brain.
I was given options for treatment
She mentioned targeted therapy and a particular drug that worked very well with this type of cancer. However, it’s a little bit slow-acting. She mentioned that if I wanted to be more aggressive, I could do chemotherapy. We discussed the pros and cons of both treatment options. I want to fight this as hard as I can, so I decided to take the aggressive approach and start chemotherapy, which was in November. She agreed with it. She let me drive that discussion and the decision. Afterward, she said, “That’s what I would have chosen for you as well.”
I started the targeted therapy drug and chemotherapy on the same day. I had four rounds of chemo between November 2024 and January 2025. After my PET scan in January, I received good news — she told me I wouldn’t need chemo anymore.
I went back to work and that same week, I received word that after discussing with a radiation oncologist, the two of them decided that it would be beneficial for me to also do a couple of rounds of radiation.
What life is like on targeted therapy
This particular drug that I’m on, I need to take at the same time every day. I take it between 10:00 and 10:30 every morning. I have been very lucky that I haven’t had a lot of side effects with the drug itself. I definitely felt them with chemo. The drug itself has been fine. I don’t feel much of a difference. I feel great right now. I wake up, hang out, take my drug, and go about my day. No major side effects.
The big side effect I had was joint pain. I felt it mostly in my fingers and knees, and occasionally on my hips. It varied from time to time, but mostly my knees and my fingers, which I also wondered about. Coupled with perimenopause, it was a double whammy. I talked to my oncologist about it and she recommended that I go on a supplement. Ever since then, I’ve felt great.
How I feel about being on treatment indefinitely
My oncologist said she would be open to clinical trials for me. It would just have to be the right one. We haven’t discussed it since then, which tells me she has not seen the right one come through. She would want something that has gone through a round or two before getting me in because she does not want me to be one of the first-round folks, which I appreciate.
Part of why I’m in therapy now is that the closer I got to feeling better, the more anxious I became. I’m stage 4 and I know that I’m on this drug until it stops working. Being stage 4, I expect it to come back. I’m in a very good place right now. I’m as healthy as I can be. But it will come back. It’s just a matter of time. Waiting for the other shoe to drop brought me so much anxiety.
I feel great right now. How long am I going to feel great before it creeps up again or before the drug stops working? Is it five years? Two years? 20 years? Having that loom over my head is what was so unnerving. I keep practicing mindfulness in that regard. I talk to my therapist about it and try to stay in the moment. I also tell myself every single day that tomorrow is not guaranteed for anybody, regardless of how healthy they are.
My life will never be the same again
My fitness level completely decreased, especially while going through chemotherapy and radiation. Fall is a great time in Austin to be outside, hiking, and enjoying the weather, and I couldn’t do any of that. I couldn’t trail run anymore. I was told not to hike. I was told to walk around my neighborhood, but walking on sidewalks is not as fun as walking on dirt for me, so that took a hit in terms of my physical and mental well-being.
Once the weather started getting warmer, I also found that I couldn’t be back in the pool immediately. Any sort of exercise was basically gone, other than a nice, leisurely walk, which, for someone who is a pseudo-athlete, is very difficult. Constantly going places, whether it’s the grocery store, felt like the COVID pandemic again, where I had to make sure that I did not get any illness whatsoever from anybody. Even my friends would say, “I’m not feeling well. I need to cancel plans with you.” It’s fine and I thank them, but not having the lung capacity to do basic happy things was tough.
I went through life imagining this beautiful future, growing old with my partner, and becoming a grandma, hopefully. I had to come to grips with the idea that I could get in a car crash the next day, but what I had planned for my future may not happen. I have come to accept that if I get to be 65 years old, I will be lucky. I will be lucky if I live long enough to become a grandmother because that’s all I want right now. I want to get promoted to grandma.
Before, it felt automatic that I’d be here until my kids grow old and then I’m going to help take care of their kids. I get to love my future grandchildren. I realize that may not happen for me, so I appreciate what I have now, as cheesy as it sounds, and enjoy the moment. I appreciate every single day for what it is.
I have been through other hardships recently, but being able to travel and see old friends is a blessing, even if the reason isn’t great. Being able to pet my dogs and waking up in bed are simple things that I’m still able to do. On the flip side, life is too short to put up with a bunch of crap. It’s also important to know when to say you’re not going to deal with certain things or not live in that space and let things go.
I was able to go back to work for a few months. I was laid off recently, so that’s one of the hardships I mentioned. But being on the drug has improved my life so much. I’m back in the pool. We even have a neighborhood swim team and that’s been fun. I look forward to our weekly swim practices and hanging out with the team.
When it’s not a thousand degrees here, I will go back to the trails and do some trail running. I have a sense of normalcy now and it feels different than taking the drug every day. My life feels like it was before I got diagnosed.
Those who were closest to me, my inner circles, were aware. After diagnosis, I told my immediate family, my best friends, and two other close friends, and that was it. Then it became a need-to-know basis. If I went to a party and someone asked why I was losing my voice, if it was someone I knew well enough, I would tell them.
My story slowly came out, but I didn’t go public with it until around March. I realized that I fit the demographic. Had I known that my cough was an indicator of cancer, I would have gone to the doctor immediately and said, “Tell me about lung cancer. I think I’m at risk of this. Please run all the tests.” I would have advocated for myself.
I figured there are plenty of other people like me, so that’s when I started talking about it at work. I posted about it to our Asian network and our women’s network. I posted about it on our neighborhood Facebook page. It was not just the Asian community specifically. My non-Asian friends might know somebody. I’m trying to get the word out there that this was a surprise.
The more I looked into it, the more I realized there are so many people like me who get diagnosed at a late stage because they had no idea. I slowly started putting myself out there. I joined that Facebook group to hopefully have that support system. Now, I feel better, but I’m also reaching out to as many places as possible. Over the course of a few months, I had 4 or 5 people say they had a cough or knew someone who had a cough, and someone else was diagnosed. If you have a cough, go and make that appointment.
I’m so grateful for my support system
If there’s one thing my friends know about me, it’s that I’m not shy about asking for help. My best friend Jenny created a Facebook group for me so we could keep friends and family updated in terms of my appointments, test results, etc. “I’ve got an appointment on this day. Who can take me?” “I’m coming back from chemo. Who wants to bring me food?” I’ve had no shortage of help in that regard. It was one of the greatest gifts I had during that time.
Before I started treatment, my sister took me on a vacation because I needed it. But while I was gone, one of my best friends got an army together to help clean my house. I had previously told her, “Oh, man. My parents are coming in with a one-way ticket. The one thing I’m not looking forward to is cleaning the guest room, organizing it, and organizing the garage.” I, like many people, use the guest room as a storage space. She jumped on it and said, “Oh, absolutely, I will do that for you.”
She did it while I was gone and not only that, she enlisted the help of so many other friends and they did the entire house. When I came back, I thought she was only doing the two spaces. A big group of friends, my partner, my ex-husband, and the kids all contributed to this huge gift, which she still claims is not that big of a deal. It’s a big deal to me.
What I want others to know
My oncologist told me at my first appointment that stage 4, especially with EGFR-positive lung cancer, is not an absolute death sentence. She told me repeatedly that it is incurable, but it is treatable. I do feel like I’m living proof of that. I did not expect to be in such a good place in such a short time. I thought I would still be struggling with this diagnosis, even with my treatments. I was diagnosed in September and here we are, less than a year later, and I’m basically in remission.
It’s a tough pill to swallow, but there’s hope. I was also told repeatedly that lung cancer research is always evolving. It’s a great time right now. We just need to make it to the next breakthrough. Even the drug that I’m on now didn’t exist years ago. Keep making it to the next one.
As cheesy as it sounds, try to stay positive because that mindset is everything. Could we lie in bed and cry, and feel sorry for ourselves? Absolutely. But that doesn’t help the body or the mind. Try to stay positive, enjoy the life we have now, and know that there’s still hope for a life post-diagnosis.
My life expectancy is much shorter. I thought I was only in my midlife at this point. I’m turning 47 soon. I envisioned life until I was 80-something. It undoubtedly will be cut short, but that’s okay because when I think about perspective, hopefully I can see my kids into adulthood, which not everybody can. I can enjoy the life that I have now as much as possible.
Asking for help is not a weakness. People want to do things on their own. They think they can manage or don’t want to bother family and friends. They don’t want to bother people by asking for help. What I’ve learned is your people, your tribe, want to help you. They want to be there for you in times of crisis. Why else would they be your friends? Don’t be afraid to ask for help and support.
