Remission of refractory necrotizing myopathy after 18 months of CAR-T cell therapy

After good results in lupus, myasthenia and anti-synthetase syndrome, a new publication reports the successful treatment of another autoimmune disease in China with CAR-T cells directed against the B lymphocyte maturation antigen (or BCMA) developed by Iaso biotherapeutics, CT103A :

  • the 25-year-old patient had lost his ability to walk due to refractory autoimmune necrotizing myopathy, which had appeared seven years previously,
  • at nine months after CAR-T cell infusion, his clinical examination was almost normal, with only persistent mild weakness of the proximal muscles of the lower limbs, but the patient was able to walk unaided, and his anti-SRP autoantibody level had fallen,
  • at 18 months, and without further immune-mediated treatment, these improvements persist, CKemia is virtually normalized and muscle lesions are fewer on MRI,
  • Sjögren’s syndrome, which also affected the young man, showed an equally favourable serological and clinical course.

The authors point out that these results have yet to be confirmed by large-scale studies, and that the efficacy of CAR-T cells in autoimmune diseases still needs to be improved, particularly in view of their limited survival (two months or even less) after infusion.

 

Single-cell analysis of refractory anti-SRP necrotizing myopathy treated with anti-BCMA CAR-T cell therapy. Qin C, Dong MH, Zhou LQ et al. Proc Natl Acad Sci U S A. 2024 Feb 6;121(6):e2315990121