BCMA-directed mRNA CAR T cell therapy for myasthenia gravis: a randomized, double-blind, placebo-controlled phase 2b trial

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Source: Nature Medicine

Original: https://www.nature.com/articles/s41591-025-04171-y...

Published: 2026-01-09

A randomized, double-blind, placebo-controlled phase 2b study evaluated autologous BCMA-directed mRNA CAR T-cell therapy in patients with generalized myasthenia gravis. Patients were randomized to Descartes-08 or placebo, all receiving a series of infusions according to the study protocol.[5] Change in symptom severity was assessed using standardized scales, particularly MG-ADL and MGC, after the end of treatment.[5] However, in the results presented, a significantly higher percentage of patients in the placebo arm achieved a reduction in disease activity compared to patients treated with BCMA-mRNA CAR T cells (as described in the assignment). The study was designed to assess both efficacy and safety; adverse effects were noted, but the entry does not indicate any specific safety signals or serious toxic manifestations. The main finding of the typed text is that the primary efficacy objective was not met in this phase 2b trial, as the placebo group had a better outcome than the actively treated group of patients.