Results from a recent analysis in the New England Journal of Medicine suggested a potentially effective approach for treating autoimmune diseases like systemic lupus erythematosus (SLE), idiopathic inflammatory myositis, and systemic sclerosis.

The case series analysis, which included 15 patients with severe forms of these conditions, evaluated the use of CD19 chimeric antigen receptor (CAR) T cells after preconditioning with specific drugs. The patients (8 with SLE, 3 with idiopathic inflammatory myositis, and 4 with systemic sclerosis) received a single infusion of CD19 CAR T cells following preconditioning with fludarabine and cyclophosphamide. The researchers assessed up to 2 years post-infusion using various criteria including Definition of Remission in SLE (DORIS) remission criteria, American College of Rheumatology–European League against Rheumatism (ACR–EULAR) major clinical response, and the European Scleroderma Trials and Research Group (EUSTAR) activity index.

According to the analysis results, all patients with SLE achieved DORIS remission, those with idiopathic inflammatory myositis experienced an ACR–EULAR major clinical response, and those with systemic sclerosis saw a decrease in the EUSTAR activity index. All patients were able to discontinue immunosuppressive therapy following treatment. The safety profile of the treatment was also assessed, with the most common adverse event being grade 1 cytokine release syndrome, occurring in 10 patients. Other adverse events included grade 2 cytokine release syndrome, grade 1 immune effector cell–associated neurotoxicity syndrome, and pneumonia requiring hospitalization (in one patient each).

"In this case series, CD19 CAR T-cell transfer appeared to be feasible, safe, and efficacious in three different autoimmune diseases, providing rationale for further controlled clinical trials," the authors concluded.

Source: Muller F, Taubmann J, Bucci L, et al. CD19 CAR T-cell therapy in autoimmune disease: A case series with follow-up. N Engl J Med. 2024;390:687-700.