Locoregional CAR T cells for children with CNS tumors: Clinical procedure and catheter safety

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Nicholas A. Vitanza, Rebecca Ronsley, Michelle Choe, Casey Henson, Mandy Breedt, Adriel Barrios-Anderson, Amy Wein, Christopher Brown, Adam Beebe, Ada Kong, Danielle Kirkey, Brittany M. Lee, Sarah E.S. Leary, Erin E. Crotty, Corrine Hoeppner, Susan Holtzclaw, Ashley L. Wilson, Joshua A. Gustafson, Jessica B. Foster, Jeffrey J. Iliff, Julie R. Park
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This study discusses the use of chimeric antigen receptor (CAR) T cell therapy to treat pediatric central nervous system (CNS) tumors. These tumors are the most common solid malignancy in children, and CAR T cell therapy has shown success in treating childhood leukemia and preclinical efficacy against pediatric CNS tumors. However, delivering CAR T cells to tumors in the brain and spine is challenging, as T cells have difficulty reaching these sites. To address this challenge, locoregional CAR T cell delivery via infusion through an intracranial catheter is being studied in early phase clinical trials. Here, we describe Seattle Children's experience in preparing for and delivering intracranial T cell infusions, and the safety of the procedure based on 307 intracranial CAR T cell doses. This research offers hope for improving outcomes for recurrent CNS tumors and universally fatal diseases like diffuse intrinsic pontine glioma (DIPG).