BCMA targeted CAR-T therapy

BCMA targeted CAR-T therapy involves engineering patient-derived T cells to express a chimeric antigen receptor (CAR) specific for B-cell maturation antigen (BCMA, encoded by TNFRSF17), a protein highly expressed on malignant plasma cells in multiple myeloma (MM). The CAR typically includes a BCMA-binding domain (e.g., scFv or novel D-domain), hinge/transmembrane region, costimulatory domains (e.g., 4-1BB or CD28), and CD3ζ signaling domain, enabling T-cell activation, proliferation, and targeted cytotoxicity against BCMA-expressing tumor cells. Developed by companies including Bristol Myers Squibb (idecabtagene vicleucel/Abecma), Janssen/Legend Biotech (ciltacabtagene autoleucel/Carvykti), and others (e.g., Cartesian Therapeutics for allogeneic P-BCMA-ALLO1), it is primarily indicated for relapsed/refractory multiple myeloma (RRMM) after ≥4 prior lines, yielding high overall response rates (ORR ~80-98%), deep responses (CR/sCR ~40-80%), and median PFS of 8-12 months in pivotal trials (KarMMa, CARTITUDE-1), though relapses occur due to BCMA-low/negative clones, soluble BCMA, or T-cell exhaustion. Safety includes cytokine release syndrome (CRS, mostly low-grade), immune effector cell-associated neurotoxicity syndrome (ICANS), cytopenias, and infections; dual-targeting (e.g., BCMA/GPRC5D) and next-gen designs (allogeneic, bicistronic) aim to enhance durability.

Brand names

AbecmaCarvykti

Other names

BCMA CAR-Tanti-BCMA CAR-T cell therapy