The FDA announced today that it has approved the CAR T cell therapy Yescarta™ (axicabtagene ciloleucel, Kite/Gilead), for the treatment of adult patients with relapsed or refractory large B cell lymphoma after two or more lines of systemic therapy. It is the first CAR T therapy approved for lymphoma patients, and comes on the heels of a recent landmark approval of another CAR T therapy, Kymriah™ (tisagenlecleucel, Novartis) for the treatment of leukemia less than two months ago.
Yescarta is a personalized immunotherapy that engineers a patient’s “killer” T cells—the foot soldiers of our immune system—to target the CD19 protein that is expressed by cancerous B cells, marking them for elimination.
Today’s approval was based on results from the phase II ZUMA-1 clinical trial, in which 101 patients with non-Hodgkin lymphoma were treated. Overall, 82% of the patients responded to treatment, and more than half (54%) had their tumors disappear completely after treatment. In some patients, side effects included potentially fatal cytokine release syndrome (CRS), which can be reversed with treatment when recognized early, as well as neurologic toxicities and anemia.
Diffuse large B cell lymphoma (DLBCL) is the most common aggressive non-Hodgkin lymphoma, accounting for three out of every five cases. Approximately 30,000 people in the United States are diagnosed with DLBCL each year. Currently, patients with large B cell lymphoma in second or later lines of therapy have poor outcomes and greater unmet need, since nearly half of them either do not respond or relapse shortly after transplant. The approval of Yescarta provides these patients with a much-needed new treatment option.
CRI scientists such as Carl H. June, M.D., Stanley R. Riddell, M.D., and Michel Sadelain, M.D.,Ph.D., have been instrumental in the development and refinement of CAR T cell technologies. While the efforts of these doctors and others have already contributed to clinical successes in lymphoma and leukemia, as evidenced by these two recent approvals, more work needs to be done to make CAR T cell immunotherapies effective against more common solid tumor types.
Read more about the approval in the press release.