The potential of CAR T therapy for relapsed or refractory pediatric and young adult B-cell ALL

Matthew H Forsberg,1 Amritava Das,2,3 Krishanu Saha,2,4,5 Christian M Capitini,1,6 1Department of Pediatrics, University of Wisconsin School of Medicine and Public Health, Madison, WI, USA; 2Wisconsin Institute for Discovery, University of Wisconsin, Madison, WI, USA; 3Morgridge Institute for Research, University of Wisconsin, Madison, WI, USA; 4Department of Medical History & Bioethics, University of Wisconsin, Madison, WI, USA; 5Department of Biomedical Engineering, University of Wisconsin, Madison, WI, USA; 6Carbone Comprehensive Cancer Center, University of Wisconsin, Madison, WI, USA Abstract: Recent advancements in immuno-oncology have resulted in the generation of novel therapies such as chimeric antigen receptor (CAR) T cells, which have revolutionized the treatment of pediatric patients with relapsed or refractory B-cell acute lymphoblastic leukemia. The journey of tisagenlecleucel (formerly CTL019) from early preclinical success to the US Food and Drug Administration approval is summarized in this review. Strategies that are currently being investigated to improve the efficacy and safety profile of CAR T-cells are also explored, as well as the factors contributing to the present state of patient access to CAR T therapy. Keywords: CAR T-cells, tisagenlecleucel, acute lymphoblastic leukemia, CD19, cancer immunotherapy, chimeric antigen recepto