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FDA Approves Liso-cel, the First and Only CAR-T for CLL / SLL

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Authored by Dr. Brian Koffman

3/14/24: FDA approved lisocabtagene maraleucel, the first CAR-T for relapsed / refractory chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL).

Brand-named Breyanzi and commonly known as liso-cel, it is the first and only chimeric antigen receptor (CAR) T or CAR-T cell therapy available for CLL / SLL patients. Its accelerated approval is for patients who have been previously exposed to a BTK inhibitor (ibrutinib, acalabrutinib, zanubrutinib, or pirtobrutinib) and a BCL-2 inhibitor (venetoclax).

It is a potent cellular immunotherapy made from the patient’s own T cells, which are genetically engineered ex-vivo (outside the body) to recognize and attack CD19, a surface marker seen on all mature B cells, including malignant CLL cells. To understand more about CAR-T cell therapy, visit the CAR-T section on our website and read CLL Society’s CAR-T brochure.

The approval was based on data from the Transcend CLL 004 trial, where a group of difficult-to-treat relapsed / refractory CLL and SLL patients with few remaining treatment options saw a 45% overall response rate and 20% complete response rate. For those 45% who did respond, the median duration was almost three years, and for the lucky one in five who had complete response, none had progressed at the time of the data cut.

CAR-T therapy does come with some well-known serious side effects, usually in the first few weeks, with 83% and 46% having some grade of Cytokine Release Syndrome (CRS) and neurotoxicity, respectively. Still, there were no fatalities, and nearly all patients fully recovered within approximately a week of the onset of symptoms. Scary and unpleasant, but usually quick and manageable.

Longer term, some low blood counts were seen, and the therapy suppresses immunity with lowered immunoglobulin levels and depletion of both healthy and cancerous B lymphocytes.

CLL / SLL is where much of the original excitement for cellular cancer therapy began over a decade ago. Still, it was late in the game to have a commercial product, but as of March 14, 2024, patients who are beyond the usual therapies will have another strong option to consider.

There will be a single-arm phase 3 confirmatory clinical trial of liso-cel coming soon that is needed for final FDA approval.

3/14 can now be celebrated not only as Pi (π or 3.14) Day but also as the day CLL / SLL patients finally had access to cellular CAR-T therapy outside of a clinical trial.

We invite you to read the official press release from BMS.