The use of autologous CD19 CAR-T cells in refractory autoimmune diseases

A European consortium of researchers reviews the therapeutic use of genetically modified autologous T cells (CAR-T cells):

  • this autologous cell therapy was administered to patients with autoimmune diseases resistant to standard treatments (lupus, scleroderma, and myositis) as part of a Phase I/IIa trial called CASTLE,
  • the product used, zorpo-cel, is a CD19-type CAR-T analog,
  • 24 patients were included in the study (5 of whom had myositis),
  • the results were conclusive in terms of tolerability, both biologically and clinically, as well as efficacy (improvement or stabilization of symptoms).

This study confirms the value of this new generation of cell therapies, which are more targeted and highly effective against certain refractory forms of idiopathic inflammatory myopathy.

 

CD19 CAR-T cells for treatment-refractory autoimmune diseases: the phase 1/2 CASTLE basket trial. Müller F, Hagen M, Wirsching A et al. Nat Med. 2026 Jan