CAR-T Given Priority Review for Follicular Lymphoma

Busy week last week, so I had a hard time posting. I was attending a (virtual) medical writing conference. Unlike something like ASCO, I don't have much to report, unless some of you want to know how to become a regulatory writer and create materials for clinical trials, or how to create infographics for drug pamphlets. Fascinating stuff, for me, anyway. 

The big news last week -- and it is pretty big news -- is that the FDA is granting Priority review for Tisagenlecleucel (also known as Kymriah), one of the different types of CAR-T treatment, for adult patients with relapsed or refractory Follicular Lymphoma who have already had at least two types of treatment that have stopped working.

This is major news. Right now, CAR-T treatments are approved for aggressive Follicular Lymphomas, like those that have transformed. There are several clinical trials looking at CAR-T and indolent FL, the slower-growing types that most of us have. An approval for Kymriah would mean a big expansion in who can receive the treatment.

The Priority Review is based on results of the phase 2 ELARA trial, which involves FL patients from 30 countries. The results were presented at ASCO last June, as I described here. There were 94 patients evaluated in the trial, with an 86% Overall Response, and a 66% Complete Response rate. Safety was also very good, with just under half having some kind of Cytokine Release Syndrome, but none having a serious CRS reaction.

Priority Review by the FDA means the goal is make a decision on approval within 6 months, rather than the usual 10 months. So if all goes well, this could be approved by next spring (though, of course, there is no guarantee that it will be approved). 

This is great news, and I'm sure it will potentially be an option for many of you. There's nothing great about having had at least two treatments for FL, and needing another, but in this case, perhaps something good is in the future.

I've written many times before about CAR-T. I know a lot of patients see it as The Answer for FL, and maybe some day it will be. For now, it's not perfect, with a very high cost, some potentially severe side effects, and good response that don't always last for very long. But from everything I've read, researchers are improving it all the time. I hope it ends up as a successful option for some of us some day.