Dr. Perl on CAR T Cells in Pediatric Leukemia

Alexander E. Perl, MD, associate professor of medicine, University of Pennsylvania, Perelman School of Medicine, discusses the promise of using chimeric antigen receptor (CAR) T cells for the treatment of patients with pediatric leukemia.

CAR T-cell therapy has made a significant impact in pediatric acute lymphoblastic leukemia (ALL), and Perl hopes that similar success will be seen with this approach in pediatric acute myeloid leukemia (AML). Although this therapy has demonstrated significant activity, it also has substantial toxicity that needs to be mitigated, Perl adds. Therefore, it is important to understand the toxicity profile of CAR T cells and to develop a plan to deal with adverse events. The biggest challenge with manufacturing an effective CAR T cell in AML is the fact that AML blasts are very similar to normal elements of the bone marrow that are critical to hematopoiesis. However, investigators are hopeful that this challenge can be overcome in time.

At the 2018 ASH Annual Meeting, data were presented demonstrating the sustained efficacy of this therapy in pediatric ALL. For example, after a long-term follow-up, the CD19-directed CAR T-cell therapy tisagenlecleucel (Kymriah) showed durable rates of relapse-free survival and overall survival at 24 and 18 months, respectively, for pediatric and young adult patients with relapsed or refractory ALL.