CAR-T lymphocytes helped in three autoimmune diseases

German, Italian and French scientists have reported the success of small pilot trials of CAR-T-lymphocytes in three different severe autoimmune diseases. A publication about it appeared in The New England Journal of Medicine. The work by Georg Schett of the Friedrich-Alexander University of Erlangen and Nuremberg and colleagues from Germany, Italy and France involved 15 patients with an unsatisfactory response to previous immunosuppressive therapy. Eight of them had severe systemic lupus erythematosus, three had idiopathic inflammatory myopathy and four had systemic scleroderma. All of them, after lymphodepletion with fludarabine and cyclophosphamide, received a single infusion of the experimental drug MB-CART19.1, which is autologous T cells with a second-generation chimeric antigenic receptor (CAR) to CD19 (a B-lymphocyte antigen). The follow-up period for participants ranged from 4 to 29 (median 15) months.

The median duration of B-cell aplasia was 112 (standard deviation ±47) days. All patients showed marked clinical remission and reduction of disease activity (in systemic lupus erythematosus as defined by DORIS, in idiopathic inflammatory myopathy according to ACR/EULAR criteria, in systemic scleroderma according to EUSTAR activity index). Immunosuppressive therapy was discontinued in all cases. Tolerability of treatment was generally satisfactory. First (most mild) degree cytokine release syndrome (CRS) developed in 10 participants; one case of second-degree CRS, ICAN syndrome, and hospital-acquired pneumonia was also reported. All these conditions were managed. According to the authors, the results obtained allow us to proceed to controlled clinical trials of CAR-T-lymphocytes in autoimmune diseases.