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ASH 2017: CAR T-Cell Therapy in Heavily Pretreated Patients With Myeloma

By: Susan Reckling
Posted: Monday, December 11, 2017

Chimeric antigen receptor (CAR) T-cell therapy that targets a protein seems to be clinically active in heavily pretreated patients with relapsed or refractory multiple myeloma, according to the preliminary results of a phase I study presented at the 2017 American Society of Hematology (ASH) Annual Meeting and Exposition (Abstract 740). In fact, the overall response rate in 21 patients was 86%, with an increase to 94% in the 18 patients who received higher doses of infused CAR T cells.

“CAR T-cell therapy is completely different from other available treatments for multiple myeloma,” shared senior study author James N. Kochenderfer, MD, of the Center for Cancer Research at the National Cancer Institute, in a press briefing prior to his presentation at ASH. “We have patients who have a sustained response and have been able to go for over a year with no additional myeloma therapy and tolerable adverse events,” he added.

The second-generation CAR T-cell therapy studied—bb2121—targets BCMA, a member of the tumor necrosis factor superfamily that is expressed primarily by malignant myeloma, plasma, and some mature B cells. In this dose-escalation trial, the investigators reported efficacy at dose levels above 50 x 106 CAR-positive T cells, with manageable cytokine-release syndrome and no dose-limiting toxicities.

This study is the first U.S.-based multicenter trial of CAR T-cell therapy engineered to target BCMA. Twenty-one patients were enrolled in the dose-escalation phase of the trial. Of the 18 patients who received higher doses of the therapy, 10 had a complete response, and 9 of the 10 had a minimal residual disease–negative response.