CAR-T Approved for Lymphoma

More good news for us -- the FDA has approved a CAR-T treatment for some Follicular Lymphoma patients.

(As usual, when it comes to CAR-T news, Ben beat me to the news. I just learned that he is a fan of the New York Yankees -- rivals to my beloved Boston Red Sox. I still like him anyway, and I'll keep sending you to his blog for everything you want to know about CAR-T and Lymphoma.)

The treatment is called axicabtagene ciloleucel, or axi-cel, though it will probably known to patients as Yescarta (to help you remember: Yes! CAR-T! Ahhhhhh!). The approval came from the results of the ZUMA-1 clinical trial, which involved patients with refractory aggressive B cell lymphomas.

That's important to remember. For now, anyway, the approval is for a very specific group of Follicular Lymphoma patients -- those with Transformed FL. There are other types of aggressive B cell lymphomas, too, who meet the approval criteria. But it's a big step for all lymphomas.

As a reminder, CAR-T stands for Chimeric Antigen Receptor T cell therapy. In the procedure, a patient has T cells removed from her blood. T cells are a type of white blood cell, part of the immune system, whose job is to attack invaders like viruses and bacteria. But they don't attack cancer cells. With CAR-T, after the T cells are removed, they are genetically engineered so that, when they are put back into the patient, they do recognize the cancer cells as invaders, and they attack. T cells have a memory, so if the cancer cells come back, they should be able to find them and attack again.

The ZUMA-1 trial involved 101 patients with several types of aggressive B cells lymphomas, including Transformed FL. The results were great -- 82% had a Response, including 54% who had a Complete Response. Six months after treatment, 36% were still in remission.

There are some side effects, of course, though researchers are looking into ways to recognize the most dangerous ones early and control them.

But there may be some other problems with this treatment (which I have seen coming up since the approval news).

The first will be the cost. This is an expensive treatment -- about $373,000. Granted, it's a personalized treatment -- each patient will have her own cells removed, manipulated, and put back, so it's not like it can be manufactured in large quantities. But it's still a lot of money.

The second will be the limited number of patients who have conditions that it has been approved for. It's pretty clear that only certain patients are approved for this (as is the case with any FDA approval). But Dr. John Leonard had a good point on Twitter last night. He said "I expect MDs to spend much more time explaining to lymphoma patients why they are not CAR-T candidates than managing the very few who are." This is likely to get a lot of attention (which it deserves), but won't be able to help a lot of patients -- yet. The manufacturer is developing CAR-T treatments for other types of solid cancer. The ZUMA-2 trial is looking at this treatment in Mantle Cell Lymphoma patients. ZUMA-3 is for Acute Lymphoblastic Leukemia patients.

It keeps going. ZUMA-5 is the trial for indolent (slow-growing) lymphoma patients, like those who have Follicular Lymphoma. It will look at 50 patients with indolent lymphoma whose disease has relapsed within two years of initial treatment, or had a second (or greater) treatment stop working, or who relapsed after a transplant. The first patient to get treatment in that trial got it in August.

We have a lot to look forward to.

(And I don't just mean next spring, when baseball starts up again for some of us.)