Phase 2 Trial Results for Liso-Cel CAR-T

I write about CAR-T treatments a lot, and it's easy to forget that CAR-T is not just one treatment. Instead, it's a general name for a bunch of different treatments. They all work in the same way, but they are different, made by different companies, with slightly different effectiveness and safety. It's kind of like using "chemotherapy" as a general term, though there are probably 10 different chemo combinations that have been used for Follicular Lymphoma over the years.

At the moment, there are two different CAR-T treatments approved for FL patients.  The first is Tisagenlecleucel, also known as Kymriah. The other is Axicabtagene Ciloleucel, also known as Yescarta. They are both approved for Relapsed/Refractory FL. There are many others in development (as I wrote about in February).

One of the CAR-Ts that already has some approvals is Lisocabtagene Maraleucel, also known as Liso-Cel or Bryanzi. This version of CAR-T has been approved in the U.S. and Europe for more aggressive lymphomas like DLBCL, but also for grade 3b Follicular Lymphoma, which is sometimes described as being very like DLBCL. But for the great majority of FL patients, Liso-Cel is not yet available.

But like many treatments that are approved for one blood cancer, Liso-Cel is in clinical trials for others, including Relapsed/Refractory FL. It's currently in a phase 2 clinical trial called TRANSCEND FL. The results were reported at the International Conference on Malignant Lymphoma in June. 

OncLive published the results. The patients included some with more than three previous treatments, and some that were POD24. Patients first received lympho-depleting chemotherapy (a round of chemo to help reduce the number of cancer cells). 

As of January 27, 2023 (the cut off for the data to be analyzed), 130 patients received Liso-Cel and 124 were able to be evaluated for the study. The Complete Response Rate was 94.1%. After 16.6 months, the median Duration of Response had not been reached, and neither had the median Progression Free Survival after 17.5 months. (In other words, fewer than half of the patients in the study had their disease return by that point.)

For safety, the most serious side effects were related to blood levels. The most common was neutropenia (low white blood cells), with 65% of patients. Cytokine release syndrome (CRS) happened in 58% of patients. There were nerve issues in 15% of patients. There was one death in the group.

As with other CAR-T treatments, Liso-cel looks safe and effective, and may very well end up as another CAR-T option for FL patients. That may take a few more years as trials continue. Another one to keep an eye on.