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A new CAR-T cell therapy targeting both BCMA and GPRC5D delivered impressive results in patients with relapsed/refractory multiple myeloma who also had extraosseous extramedullary disease — one of the toughest presentations to treat. In a phase 2 trial of 37 patients, the overall response rate hit 97%, with nearly all patients achieving MRD negativity. Side effects were manageable, with no severe cytokine release syndrome reported.
A phase 2 trial published in Blood is offering new hope for patients with one of the most challenging forms of multiple myeloma. Researchers tested an anti-BCMA/GPRC5D CAR-T cell therapy in 37 patients with relapsed/refractory multiple myeloma (RRMM) who also had extraosseous extramedullary disease (EMD) — a condition where myeloma spreads outside the bone marrow into soft tissues, which typically signals a poor prognosis. The patient population was heavily pretreated, with a median of 5 prior lines of therapy and nearly 70% being triple-class refractory.
The results were striking: a 97% overall response rate, with 43% of patients achieving a stringent complete response. Almost all patients reached MRD negativity, and over 95% did so within just one month of treatment.
By the Numbers:
Why it matters: Extraosseous EMD in myeloma is notoriously difficult to treat and associated with poor outcomes. This dual-target CAR-T approach shows that even in this high-risk group, deep and rapid responses are achievable with a manageable safety profile — a meaningful step forward for patients with limited options.