CAR-T cells have led to a remission lasting at least 19 months in three patients with myasthenia gravis

The anti-CD19 CAR-T cells KYV-101 are being evaluated in a Phase II/IIIUn essai de phase II/III permet de tester l’efficacité d’un traitement potentiel et son dosage en une seule étape : les phases II (dose et mode d’administration) et III (efficacité) de l’essai clinique sont regroupées en une seule. Au terme de l’essai, si les résultats sont positifs, une autorisation de mise sur le marché (AMM) peut être donnée. trial involving 66 patients with refractory generalised autoimmune myasthenia gravis. Initial results for three participants aged 33, 37 and 76 show:

• clinical remission without treatment, which persisted at the last follow-up visit—19 months after the CAR-T cell infusion for two of the participants, and 24 months for the third,
• no correlation between clinical improvement and anti-RACh autoantibody levels, which did not normalise,
• transient adverse effects such as neutropenia, elevated liver enzymes and respiratory infections, but no neurotoxicity or opportunistic infections.

It should be noted that one of the participants also had rheumatoid arthritis, which relapsed one year after CAR-T cell treatment.