‘Universal’ CAR-T cells in autoimmune necrotising myopathy (and scleroderma)

In February 2024, an initial Chinese team published the case of a patient suffering from a refractory form of autoimmune necrotising myopathy, who went into remission following an infusion of CAR-T cells directed against B lymphocyte maturation antigen (BCMA).

Six months later, a second publication, this time from a clinical trial, reported another success in China in the same indication:

  • the 42-year-old patient suffers from autoimmune necrotising myopathy with anti-SRP, diagnosed in 2013 and highly progressive despite extensive immune therapy,
  • she received TyU19 CAR-T cells (or BRL-301) directed against CD19 and derived from T lymphocytes of a healthy donor (allogeneic transplant of ‘universal’ CAR-T cells),
  • this treatment led to a major clinical improvement (ITS score) at M1, before complete remission at M2, which was still the case at M6,
  • CPK levels gradually returned to normal and anti-SRP autoantibodies became undetectable from M1,
  • MRI and muscle biopsy also improved over the 6 months of post-injection follow-up.

Comparable benefits were recorded in two other participants in the same clinical trial, suffering from refractory scleroderma.

 

Allogeneic CD19-targeted CAR-T therapy in patients with severe myositis and systemic sclerosis. Wang X, Wu X, Tan B et al. Cell. 2024 Jul 9:S0092-8674(24)00701-3.