CAR-T cells for childhood and adult Acute Lymphoblastic Leukemia

CAR-T cells are T lymphocytes expressing a chimeric receptor targeting a specific antigen combined with an intracellular signaling domain. CAR-T cells CD19 target CD19 antigen, marker on B acute lymphoblastic leukemia (ALL). Five-year overall survival after acute lymphoblastic leukemia B (B-ALL) in children is greater than 90 % but these leukemia remains a major cause of death from cancer in children. Overall survival in adults is not greater than 60 %. Adults with relapsed ALL obtain a median overall survival of 3 to 9 months after standard chemotherapy. A new class of immuno therapy based on a chimeric antigen receptor "CAR " targeting the CD19 on the B leukemic cells will allow to transform the prognosis of refractory or relapsed B-ALL. Overall response rates range from 60 to 90 % in phase I-II studies of CAR-T cells in patients with second relapse or more or with refractory disease. Persistent remissions and even cures have been observed. This "living drug ", manufactured for each patient, has obtained a marketing authorization in the USA in 2017 and in the EU in 2018. Preventing clonal evolution, improving the production of these cells and managing their short and long-term side effects, controlling their expansion and persistence in the body remain major issues before we can extend the indications of this innovative therapy.