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A phase 1 clinical trial led by investigators at Mass General Brigham Cancer Institute and the University of Chicago Medicine Comprehensive Cancer Center has found that an investigational CAR-T therapy can lead to deep and durable responses in people with relapsed or refractory multiple myeloma. Results are published in The New England Journal of Medicine.

Patients with relapsed or refractory multiple myeloma often run out of effective treatment options after multiple rounds of therapy. Some existing CAR-T cell therapies targeting BCMA, a protein expressed on the surface of myeloma cells, have also been linked to rare but serious neurological side effects.

The research team reported results from a phase 1 clinical trial of anitocabtagene autoleucel (anito-cel), an investigational BCMA-targeted CAR-T cell therapy being developed by Kite, a Gilead company.

All 38 treated participants responded to the therapy, and nearly 80% achieved a complete response. Many experienced long-lasting benefits, with more than half remaining progression-free two years later and 65% still alive after three years. Serious immune-related and neurological side effects were uncommon, and no delayed neurological complications were reported.

"These were great results for a phase 1 trial," said senior author Michael R. Bishop, MD, professor of medicine and director of the David and Etta Jonas Center for Cellular Therapy at UChicago Medicine.

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