BCMA CAR T cells: the winding path to success.

Chimeric antigen receptor (CAR) T cells have been shown to successfully treat some hematopoietic malignancies. Recognition of a relevant target on malignant cells and the proper costimulatory molecule are essential for CAR T cell efficacy. In this issue of the JCI, Cohen et al. conducted an early phase trial to evaluate B cell maturation antigen-targeting (BCMA-targeting) CAR T cells in patients with refractory multiple myeloma. Patients who received the highest dose of BCMA-targeting CAR T cells in combination with lymphodepletion had the greatest response. The results of the study provide further support for the use of BCMA-targeting CAR T cells for myeloma, and reiterate the importance of space and cell dose for CAR T cell success.