Locoregionally administered B7-H3-targeted CAR T cells for treatment of atypical teratoid/rhabdoid tumors

Nat Med. 2020 May;26(5):712-719. doi: 10.1038/s41591-020-0821-8. Epub 2020 Apr 27.

Abstract

Atypical teratoid/rhabdoid tumors (ATRTs) typically arise in the central nervous system (CNS) of children under 3 years of age. Despite intensive multimodal therapy (surgery, chemotherapy and, if age permits, radiotherapy), median survival is 17 months1,2. We show that ATRTs robustly express B7-H3/CD276 that does not result from the inactivating mutations in SMARCB1 (refs. 3,4), which drive oncogenesis in ATRT, but requires residual SWItch/Sucrose Non-Fermentable (SWI/SNF) activity mediated by BRG1/SMARCA4. Consistent with the embryonic origin of ATRT5,6, B7-H3 is highly expressed on the prenatal, but not postnatal, brain. B7-H3.BB.z-chimeric antigen receptor (CAR) T cells administered intracerebroventricularly or intratumorally mediate potent antitumor effects against cerebral ATRT xenografts in mice, with faster kinetics, greater potency and reduced systemic levels of inflammatory cytokines compared to CAR T cells administered intravenously. CAR T cells administered ICV also traffic from the CNS into the periphery; following clearance of ATRT xenografts, B7-H3.BB.z-CAR T cells administered intracerebroventricularly or intravenously mediate antigen-specific protection from tumor rechallenge, both in the brain and periphery. These results identify B7-H3 as a compelling therapeutic target for this largely incurable pediatric tumor and demonstrate important advantages of locoregional compared to systemic delivery of CAR T cells for the treatment of CNS malignancies.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Animals
  • B7 Antigens / immunology*
  • Brain / drug effects
  • Brain / immunology
  • Brain / pathology
  • Brain Neoplasms / immunology
  • Brain Neoplasms / pathology
  • Brain Neoplasms / therapy*
  • Cancer Vaccines / administration & dosage*
  • Cells, Cultured
  • Child, Preschool
  • Female
  • Fetus / pathology
  • Humans
  • Immunotherapy, Adoptive / methods*
  • Infant
  • Injections, Intraventricular
  • Mice
  • Mice, Inbred NOD
  • Mice, SCID
  • Receptors, Chimeric Antigen / administration & dosage
  • Receptors, Chimeric Antigen / genetics
  • Receptors, Chimeric Antigen / immunology
  • Rhabdoid Tumor / immunology
  • Rhabdoid Tumor / pathology
  • Rhabdoid Tumor / therapy*
  • T-Lymphocytes / immunology
  • T-Lymphocytes / metabolism
  • T-Lymphocytes / transplantation
  • Teratoma / immunology
  • Teratoma / pathology
  • Teratoma / therapy*
  • Xenograft Model Antitumor Assays

Substances

  • B7 Antigens
  • CD276 protein, human
  • Cancer Vaccines
  • Receptors, Chimeric Antigen

Supplementary concepts

  • Teratoid Tumor, Atypical