‘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 lymphocytesC’est une catégorie de globules blancs. Ces petites cellules au noyau arrondi et volumineux sont impliquées dans les aspects spécifiques des réactions immunitaires. Il existe deux catégories de lymphocytes les lymphocytes B (qui secrètent les anticorps) et les lymphocytes T. 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,
• CPKEnzyme contenue dans les cellules musculaires, qui est libérée dans le sang en cas d’atteinte musculaire. 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.