Promising results from a CAR-T cell trial in refractory myasthenia gravis

Following the publication of initial success in 2024, a Chinese team has published the results of a multicentre phase IAu cours d’un essai clinique de phase I un médicament dont l’intérêt thérapeutique a été montré sur des modèles animaux et/ou cellulaires (essais précliniques) est administré pour la première fois à un petit groupe de volontaires sains, plus rarement à des malades, afin d’évaluer leur tolérance à la substance en fonction de la dose (Comment le futur traitement est-il absorbé et éliminé ? Comment se fait sa répartition dans les organes ? Est-il toxique et à quelles doses ? Existe-t-il des effets secondaires ?). 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.

Anti-BCMA/CD19 CAR T cell therapy in patients with refractory generalized myasthenia gravis: a single-arm, phase 1 trial. Zhang Y, Liu D, Zhang Z et al. EClinicalMedicine. 2025 Oct 31;90:103621.