Following the publication of initial success in 2024, a Chinese team has published the results of a multicentre phase I trial that evaluated three different doses of autologous anti-BCMA and anti-CD19 CAR-T cells preceded by lymphodepletion in 18 adults with refractory autoimmune myasthenia gravis:
- myasthenia had previously been resistant to IV immunoglobulins (67% of participants), neonatal Fc receptor inhibitors (44%) and complement inhibitors (11%) in particular;
- the most common grade 3 or higher side effect within 28 days of CAR-T cell treatment was transient and resolvable haematological toxicity;
- all patients experienced significant clinical improvement, which was sustained in the 17 participants followed for 180 days;
- at this point, 82% of these patients had achieved minimal manifestation status, 47% had negative anti-RACh autoantibodies, 88% were able to stop corticosteroids, and no participants were taking non-steroidal immunosuppressants.
Notably, around 20 other trials evaluating CAR-T cells are currently underway for the same indication, including two in France.
