This review examines the emerging application of chimeric antigen receptor (CAR) T-cell therapy in myasthenia gravis (MG), with emphasis on safety, efficacy signals, and future therapeutic potential in treatment-refractory disease. A comprehensive literature search was conducted across PubMed, medRxiv, bioRxiv, and Google Scholar for studies published between January 1, 2010 and July 1, 2025, using the terms "CAR T-cell," "chimeric antigen receptor," and "myasthenia gravis." Eligible reports included clinical trials, case reports, case series, and mechanistic studies describing CAR T-cell therapy outcomes in MG. Across five independent case series, seven patients receiving CAR T-cells targeting CD19, BCMA, or bispecific antigens showed consistent clinical improvement. Peak expansion occurred 8-22 days postinfusion, with persistence of 28 to ~180 days. Despite small cohorts, safety in CAR T-cell therapy was favorable versus oncology, with mainly Grade 1-2 cytokine release syndrome, rare immune effector cell-associated neurotoxicity, and no severe or life-threatening events. Seven registered trials (> 260 patients) are expected to conclude between 2026 and 2029. Overall, CAR T-cell therapy shows substantial promise for treatment-refractory MG, potentially offering greater durability than existing therapies with an acceptable safety profile. Nevertheless, current evidence is restricted to small, uncontrolled studies, and long-term efficacy, durability, and optimal patient selection require validation in larger controlled trials.
Hegelmaier et al. (Fri,) studied this question.