CAR-T Approval by FDA

Last week, the FDA granted accelerated approval to Breyanzi, also known as lisocabtagene maraleucel or Liso-cel, to patients with relapsed or refractory Follicular Lymphoma who have received two or more prior systemic treatments. Liso-cel is a type of CAR-T. This is great news for many of us, since it makes the treatment available to lots of patients.

Some background first. CAR-T is not a single treatment (even though I know I am guilty of writing about it as if it were). It' a broader category, like chemotherapy or immunotherapy, with lots of different options under that umbrella. So there are a few different versions of CAR-T that have been approved for different cancers, and many more in different stages of development. But they all work in basically the same way -- T cells, a type of immune cell, are removed from the patient's body, manipulated in a lab so they recognize cancer cells, and are then put back into the patient to do their job. 

Liso-cel had already been approved for some aggressive blood cancers, including FL grade 3b, which is an especially aggressive type. But now it is approved for more FL patients, those with r/r disease (their last treatment stopped working, r didn't work at all) who have had at least two other treatments. 

The approval is based on a phase 2 clinical trial called TRANSCEND-FL. Like other accelerated approvals, this one is not based on a large phase 3 trial, but rather the smaller phase 2. Despite that, there seem to be fewer concerns about safety than with other trials, since Liso-cel has already been approved for other cancers, and safety issues are fairly well-known by now,

The phase 2 trial was very successful -- the Overall Response Rate was 95.7% in 94 patients, and after a median follow-up of 16.8 months, the median durability of response hadn't been reached (meaning more than half of the patients still had a response to the treatment). 

Safety was not a concern, in that there were not any new side effects. The most common were cytokine release syndrome (CRS), headache, musculoskeletal pain, fatigue, constipation, and fever. The approval included a "Risk Evaluation and Mitigation Strategy" for Liso-cel, meaning they recognized the potential seriousness of CRS and nerve problems, and made sure there was a plan to deal with any patients who showed signs of them before they became serious. And that includes the "black box warning" that has already been required. 

So, as I said, all of this is great news for FL patients. Even for those of us who do not qualify (including me), it shows that CAR-T is effective for more and more of us. We're waiting on more trial results for CAR-T treatments for other FL populations, including those who need a first-line treatment. Maybe we'll get an update in a few weeks at ASCO. 

Speaking of ASCO -- for those who don't know, this is the American Society for Clinical Oncology, and their annual meeting happens in early June -- I'm still waiting for them to release their abstracts so I can see what kind of research will be presented. I was once again allowed to register for free as an independent cancer advocate, which means I'll have access to more information that usual. Not everything, but lots of things. I'm looking forward to sharing what I learn. And a big THANK YOU to ASCO for allowing independent advocates like me to participate in this way. Not every medical organization is so generous.

More reasons for hope, coming soon.