Big announcement yesterday for Follicular Lymphoma -- the FDA approved Tisagenlecleucel (also known as Kymriah), a type of CAR-T, for Follicular Lymphoma patients who are relapsed or refractory to treatment, and who have received at least two prior treatments. Earlier this month, the European Commission gave the same approval.
It's a big deal for FL patients, because it greatly increases the number of people eligible for the treatment, in the US and in Europe. Up until now, CAR-T treatments (there are several of them) were only approved for FL patients with transformed or more aggressive types of FL. It's still not approved as a first-line treatment, but that may come with time.
The announcement is very new, so there isn't a whole lot of commentary on it. I think that will happen in the next few weeks, especially as experts give their opinion during and after the ASCO conference.
The approval came based on results from the phase 2 trial called ELARA. This involved fewer than 100 patients, and like all accelerated approvals, there will need to be a phase 3 trial with more patients to confirm that it is as effective and as safe as it seems to be. (This doesn't always result in permanent approval, as we saw recently with PI3K inhibitors.)
The numbers, at least from what is being presented right now, look very good. One of the negatives about early CAR-T was that it was very effective for some, sort of effective for others, and not effective for the rest. I remember my oncologist kind of summing it up as, 1/3 of patients had a long-lasting response, 1/3 had a response that lasted a year, and 1/3 didn't respond.
With multiple different version of CAR-T, there have been lots of opportunities for researchers to figure out how to improve the process, and the ELARA trial seems to show that they have been able to. With a follow-up of 17 months, 86% of patients had a response, including 68% who had a Complete Response. Of those with a CR, 85% of them still had a response after 12 months. So that's an improvement over that one-third/one-third/one-third split. And it was effective even in patients who sometimes have trouble responding to treatment, like those with POD24 or who have had many treatments that stopped working.
As for safety, the press release from the maker calls the results "remarkable." While 53% of patients in the trial had Cytokine Release Syndrome (CRS), none of them had a high-grade (more dangerous) type of reaction. Also, 43% of patients had nerve-related side effects, but only 6% had high-grade. The troubling safety statistic was that 3 patients died during the trial. This is one of those things that I would like to see more commentary on. I remember reading that some patients in early CAR-T trials had died from CRS, but that researchers quickly realized how to look for it and control it very early on. I don't know if that's what happened here, and the otherwise excellent safety statistics reflect that. I'll keep an eye out for more on what happened there. But if regulators in both Europe and the US have found the treatment to be safe, there has to be some explanation for those numbers.
Effectiveness and safety look good, so the next issue to consider is cost. CAR-T is still a fairly expensive treatment. But if it turns out that patients only need one very expensive that lasts a long time, instead of many moderately expensive treatments, that might make a difference.
As I said, I'll keep an eye out for more information and expert commentary about this, but for now, it certainly seems like CAR-T is going to be a part of more FL patients' lives from here on. I hope it proves to be as effective and safe for some of you as the trials would suggest.
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