FDA Designation for Kymriah CAR-T

I'm catching up on this:

A couple of weeks ago, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation for Kymriah (also known as tisagenlecleucel), a type of CAR-T therapy. This would be the first CAR-T for Relapsed or Refractory Follicular Lymphoma, if it is approved.

Let's break all of that down.

First, as most of you probably know, CAR-T stands for Chimeric Antigen Receptor (CAR) T-cell therapy. T cells are a kind of immune cell that do an excellent job of attacking invaders in the body. Cancer cells, however, are not invaders; they are our own cells that have been messed up so they don't know how to die like a normal cell. So T cells leave them alone. With CAR-T treatments, some of a patient's T cells are removed, taken to a lab so they can be changed to recognize cancer cells, and then put back into the patient, so they attack the cancer cells. Every treatment is made individually for a specific patient, so it's kind of expensive. Early CAR-T treatments work really well for some patients, pretty well for others, and not at all for some. But it shows huge promise.

(If you want to learn more about CAR-T, go to CAR-T and Follicular Non-Hodgkin's Lymphoma, a blog run by a CAR-T patient and a CAR-T caregiver. Good stuff.)

Right now, there are two CAR-T treatments that have received any kind of FDA approvals. The first is called Yescarta (also known as axicabtagene ciloleucel). It has been approved for some aggressive B cell lymphomas, including Transformed Follicular Lymphoma.

The second is called Kymriah. It has been approved for certain aggressive lymphomas and leukemias. But not Follicular Lymphoma.

The makers of Kymriah have applied for FDA approval, based on a Stage II clinical trial called ELARA. The FDA has granted a particular type of designation called Regenerative Medicine Advanced Therapy, or RMAT. This basically means that it is a type of therapy that is created from cells (like T cells), changes cells, or changes genes within cells. It was created to recognize that something like CAR-T is different from other types of treatments like traditional chemotherapy. the designation is only a few years old, but over 40 treatments have applied for it, which says something about where medicine is heading.

One last thing -- "Relapsed or Refractory" means the disease has stopped responding to the last treatment it received, or the last treatment just didn't work at all. This, to me, is the most important part of this approval. It means that anyone who has received treatment for Follicular Lymphoma would potentially be eligible to receive this CAR-T treatment, if it is approved. Right now, CAR-T is only available (outside of a clinical trial) to patients whose FL has transformed into a more aggressive lymphoma.

Now, a word of caution here. When results of CAR-T trials get announced, I see lots of people in online groups who say "I want this treatment! It looks great!" And they are right -- it does look great.

But it is expensive (roughly $400,000, from the numbers I have seen), since it needs to be created for each individual who gets it. It also can have some pretty rough side effects for some patients (though doctors have gotten better about anticipating them). And early trials found long-lasting effects for about 1/3 of patients, short-term effects (less than a year) for about 1/3, and no effects for the last 1/3. Those numbers seem to be getting better as researchers learn more about how CAR-T works.

But the point is, as with most FL news, there is a lot of great stuff in this, and it should give us a lot of hope.  But so far, there is no magic formula for FL treatment, and CAR-T doesn't seem to be. Keep that in mind in a few months when you hear about this being approved (assuming it happens).

But that doesn't mean this isn't worth being happy about.  Every arrow in the quiver is a victory for us. So I have hopes this this one will end up helping a lot of people, too.