STRENGTHENING the global effort to CURE neuromuscular disease

Tuan Vu, MD, has been recognized with the 2025 Best Abstract Runner-Up Award from the American Neuromuscular Foundation for his abstract, Efficacy and Safety of Autologous BCMA Directed CAR T-Cell Therapy in Generalized Myasthenia Gravis: 12-Month Follow-Up of a Phase 2B Randomized Placebo-Controlled Trial. The study evaluates Descartes-08, a personalized CAR-T cell therapy using mRNA to transiently program a patient’s T cells to express a receptor targeting BCMA—a protein tied to autoantibody production in generalized myasthenia gravis (gMG). Delivered as 6 weekly outpatient infusions without preconditioning chemotherapy, Descartes-08 showed “deep and sustained improvements in MG symptoms through 12 months, with a favorable safety profile.” 

The trial was driven by “the significant unmet need for more effective, durable, and well tolerated treatment options.” Current therapies often rely on long-term immunosuppression and may still leave patients symptomatic. Descartes-08 aims to change that by targeting the long-lived plasma cells behind the disease. A key outcome was “the durability of clinical benefit following just six once-weekly infusions,” with marked and lasting reductions in MG-ADL scores, especially in patients not previously treated with complement inhibitors or FcRn antagonists. 

Dr. Vu sees this as a step toward “a more personalized and durable approach to treating gMG,” potentially shifting the treatment model away from chronic regimens. “For patients, Descartes-08 could offer a personalized approach to treating gMG by using a patient’s cells to target the underlying cause of disease.” With a Phase 3 trial underway, this therapy could mark a major advancement in gMG care. 

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