CAR T-cell therapy has emerged as a groundbreaking treatment for leukemia, lymphoma, and multiple myeloma, offering significant improvements and even cures for patients. While traditionally used in oncology, an early pilot study suggests promising results for myasthenia gravis (MG) patients, marking a potential breakthrough in neuro-immunologic conditions. The study investigates Descartes-08, a personalized RNA-engineered CAR-T therapy targeting B-cell maturation antigen (BCMA), a protein expressed in plasma cells. In the proof-of-concept trial, Descartes-08 demonstrated safety and significant clinical improvement in 14 MG patients with no observed cytokine release syndrome (CRS) or neurotoxicity. Patients experienced clinically meaningful decreases in MG severity scales, with some achieving sustained clinical remission and resolving dependence on intravenous immune globulin (IVIG). The promising results warrant further investigation in larger trials, potentially offering a feasible long-term treatment option for patients with MG, complementing the growing array of novel therapies in development for this neuro-immunologic disorder.

Reference: Shaw G Myasthenia Gravis Patients Benefit From Modified Form of CAR T Therapy. ​Neurology Today. Updated August 9, 2023. Accessed May 2, 2024. https://journals.lww.com/neurotodayonline/blog/breakingnews/pages/post.aspx?PostID=1380