BCMA CAR Ts: Are short-term risks worth long-term gains?

FDA is reviewing two BCMA-directed CAR T cell therapies for use earlier in the course of multiple myeloma treatment, and there’s a trade-off between higher upfront risks and greater long-term benefits. How FDA and ODAC weigh the risks and benefits will help define expectations for future CAR T cell applications, and possibly cell and gene therapies more broadly.

For both products, the dilemma is that patients treated with the CAR T cells are more likely to die in the first months after randomization than those on standard of care, but those who survive the waiting period while the products are being manufactured, and the therapies’ acute toxicities, are more likely to achieve long-term remission...