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Abstract
Recent advancements in immunooncology 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.
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Acknowledgments
This work was supported by grants from Stand Up To Cancer St Baldrick’s Pediatric Dream Team Translational Research Grant SU2C-AACR-DT1113 (CMC), the NCI/NIH K08 CA174750 (CMC), NIH/NCI P30 CA014520 to the University of Wisconsin Carbone Cancer Center (KS and CMC), NSF CBET-1350178 (KS), and NSF CBET-1645123 (KS and CMC). Stand Up To Cancer is a program of the Entertainment Industry Foundation administered by the American Association for Cancer Research. The contents of this article neither necessarily reflect the views or policies of the Department of Health and Human Services nor do mention trade names, commercial products, or organizations imply endorsement by the US Government.
Disclosure
CMC received honorarium for serving on a one-time advisory board for Novartis. The authors report no other conflicts of interest in this work.