A groundbreaking clinical trial featured in Nature Medicine’s “Eleven Clinical Trials That Will Shape Medicine in 2026” is redefining the future of autoimmune disease treatment. The study, led by Dr. James Howard and Cartesian Therapeutics, explores the use of mRNA-based CAR T cell therapy for myasthenia gravis, a chronic autoimmune disorder that disrupts communication between nerves and muscles, causing debilitating weakness and fatigue.
Unlike traditional CAR T therapies used in cancer, which involve permanent DNA edits, this innovative approach uses mRNA to temporarily program T cells, reducing long-term risks such as cytokine-release syndrome (CRS) and Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS). The therapy targets BCMA-expressing plasma cells, producers of harmful antibodies, while sparing the broader immune system—avoiding the side effects of conventional immunosuppressants.
Early results from the phase 2b trial are promising:
- 57% of patients achieved minimal symptom expression by month 6, maintaining remission through month 12.
- Treatment consists of six short weekly infusions, and some participants have remained symptom-free for over a year.
If confirmed in the ongoing phase 3 trial, this therapy could become a first-line outpatient treatment, offering durable relief without the burden of chronic immunosuppression. Beyond myasthenia gravis, researchers believe this mRNA CAR T platform could extend to other autoimmune diseases, including lupus and rheumatoid arthritis and multiple sclerosis.