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 IAu cours d’un essai clinique de phase I un médicament dont l’intérêt thérapeutique a été montré sur des modèles animaux et/ou cellulaires (essais précliniques) est administré pour la première fois à un petit groupe de volontaires sains, plus rarement à des malades, afin d’évaluer leur tolérance à la substance en fonction de la dose (Comment le futur traitement est-il absorbé et éliminé ? Comment se fait sa répartition dans les organes ? Est-il toxique et à quelles doses ? Existe-t-il des effets secondaires ?)./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