FDA Update: Good News for Multiple Myeloma?

Unanimous vote by Food & Drug Administration (FDA) Committee Leads to New Endpoint for Multiple Myeloma Clinical Trials – And Could Lead to Changes for Other Cancers

Published April 13, 2024

Editor’s Note: This is not medical advice. Please consult your healthcare team for your care decisions.

The rapid shift in treatments and clinical trials available in multiple myeloma has led to more options for patients. But in the process, top myeloma experts and patient advocates have said the old standard to get new treatments approved out of clinical trials has become complicated and burdensome, leading to longer times to new approvals. An FDA committee decision Friday, April 12, may lead to even more treatment options on the table for what’s largely described as an incurable but highly treatable disease.

One of the leading advocates for the change and Director of the Myeloma Research Institute at University of Miami’s cancer center, C. Ola Landgren, MD PhD, says this new endpoint may also benefit other cancer spaces soon.

Good news for multiple myeloma? The big topic of the day in multiple myeloma for many patient advocates and physicians came on April 12, when the FDA’s Oncologic Drugs Advisory Committee (ODAC) unanimously approved (12 to zero vote) using minimal residual disease or MRD a new endpoint in multiple myeloma clinical trials. The decision means the group agreed it was time for a change that would benefit multiple myeloma patients.

WATCH: ODAC meeting on YouTube

General summary: What does this mean for multiple myeloma patients?

This may lead to the FDA moving more multiple myeloma therapies in clinical research through accelerated approval. Clinical trials are how new drugs and therapies are given the green light for commercial use, which means they become available as another tool in treating patients.

Leading voices in multiple myeloma, like  C. Ola Landgren, M.D., Ph.D., director of the Myeloma Research Institute at Sylvester Comprehensive Cancer Center in Miami, Florida, have been arguing that a better endpoint is minimal residual disease or MRD. He described the move as “fantastic news” for multiple myeloma patients.

“With MRD as a new endpoint, it will give patients access to new therapies much faster! This is exactly what patients need and want,” said Dr. Landgren in a University of Miami news update. “It is a milestone in the field of multiple myeloma and will set the stage for the development of novel therapies for other cancers.”

Myeloma patient advocates like Jenny Ahlstrom have said this is important because the disease, while highly treatable, is still not officially “curable,” and patients having to wait longer for a new treatment option may run out of time.

“With the acceleration of drug approvals, many patients are living 10, 15 years instead of three to five years although we still know that 40% of patients are still dying under 5 years,” said Ahlstrom, founder of Healthtree Foundation, during the nearly seven-hour long ODAC meeting. “There is still no known cure, so the innovation needs to continue and we still have an urgent need.”

Why should I care about a clinical trial endpoint?

In generic terms, the way the FDA decides whether to approve any clinical trial is based on a set of information, a lot of it comparing the study drug to the existing standard of care. The questions boils down to:

• Is this better for patients than what’s available now?
• How and why?

An endpoint is a measurement that’s used to help answer those questions. If a clinical trial does not meet the given endpoint, the FDA does not approve it.

What is the change in endpoints from the ODAC decision?

Old endpoint: Overall survival (OS)

Let’s set the stage for what has historically been used as the standard against which the FDA would decide whether to approve a study drug.

The endpoint used in many FDA clinical trial decisions, including in multiple myeloma, is something called overall survival (OS), defined as “the average length of time patients are alive after the start of treatment.”

According to the FDA’s own reporting, there are “advantages” and “disadvantages” to using overall survival as an endpoint:

• Advantages
  • Easily and precisely measured
  • Generally based on objective and
    quantitative assessment
• Disadvantages
  • May be affected by switch-over of
    control to treatment or subsequent
    therapies
  • Needs longer follow-up
  • Includes noncancer deaths

New endpoint: Minimal residual disease (MRD)

So what is minimal residual disease (MRD)? It is a measurement that indicates how many myeloma cells are still present after someone goes through treatment. Typically, the number of cells is so small that the disease is undetectable if using standard testing. The tests for MRD are much more sensitive, and can help patients and their physicians make a decision on whether they need to go for more treatment.

Ahlstrom, a long-time multiple myeloma patient advocate, shared with The Patient Story in 2023 how MRD testing helped her personally lead to treatment decisions.

“In myeloma, an official relapse is a monoclonal protein of 0.5. We were watching that number and it was 0.13. I first saw it on an MRD test that I did so it was very sensitive and started seeing some early indication. I had this faint band that wasn’t measurable for a really long time, for almost two years, so you couldn’t even quantify it,” shared Ahlstrom.

“But then the numbers started growing and once it got to 0.5, I started having conversations with multiple experts. ‘What would you do? How would you do it? What’s your strategy?’

“It took a couple of years and then once it hit 1, my doctor said, ‘Now it is time to do something.’ I got multiple opinions and I had a lot of knowledge about what my options were so I was able to weigh those in my mind.”

In a program hosted by The Patient Story in October 2023, Dr. Landgren shared the importance of MRD.

“The field has evolved. We see many patients achieving remissions and minimal residual disease (MRD) negativity so there is no detectable disease. When you don’t have those perspectives, it’s not really something brought up frequently with cure. With more and more of these drugs delivering great, deep, durable responses, it’s natural for the field to talk about it.”

Will this lead to changes in other cancer clinical trials?

Dr. Landgren says it’s very possible the move could “open the field for other diseases to move in the same direction, and it could give cancer patients access to therapy many years sooner, in a much broader context beyond myeloma.”

“It is a milestone in the field of multiple myeloma and will set the stage for the development of novel therapies for other cancers,” he added.

Stay tuned for more updates in multiple myeloma

The Patient Story is slated to talk more with top myeloma specialists on the implications of this decision for multiple myeloma patients and their families.

In the meantime, check below for our library of myeloma stories to hear from patients on their experiences after getting a diagnosis.

If you want to get first access to attend our educational programs featuring top myeloma experts and advocates and/or get an alert when a new myeloma patient story is up, please join our community here.

Relapsed/Refractory Myeloma Stories

Theresa T.



Diagnosis: Multiple myeloma, relapsed/refractory

Subtype: IgG kappa Light Chain

Initial Symptom: Extreme pain in right hip

Treatment: Chemotherapy, CAR T-cell therapy, stem cell transplant, radiation

Laura E.



Symptom: Increasing back pain
Treatments: Chemotherapy, stem cell transplant, bispecific antibodies

Donna K.



Diagnosis: Multiple myeloma, refractory
1st Symptoms: None, found by blood tests
Treatment: Total Therapy Four, carfilzomib + pomalidomide, daratumumab + lenalidomide, CAR T, selinexor-carfilzomib

Connie H.



Diagnosis: Multiple myeloma, relapsed refractory
1st Symptoms: Chronic bone pain
Treatment: IV Chemotherapy, CAR T cell therapy

Elise D.



Diagnosis: Multiple myeloma, refractory
1st Symptoms: Lower back pain, fractured sacrum
Treatment: CyBorD, Clinical trial of Xpovio (selinexor)+ Kyprolis (carfilzomib) + dexamethasone

Beth A.



Diagnosis: Multiple myeloma, relapsed/refractory
Subtype: Non-secretory (1-5% of myelomas)
1st Symptoms: Extreme pain between shoulder blades, sternum, head, burning sensation
1st Line Treatment: VAD chemo, radiation, stem cell transplant
RR Treatment: 8 chemo regimens, successful combo→selinexor+bortezomib+dexamethasone

Scott C.



Diagnosis: Multiple myeloma, relapsed/refractorySubtype: IgG lambda (majorityof myelomas)
1st Symptoms: Pain in hips and ribs, night sweats, weight loss, nausea
Treatment: Clinical trial, chemo, kyphoplasty, stem cell transplant