articleNature MedicineJan 7, 2025HYBRID OA

Intracerebroventricular B7-H3-targeting CAR T cells for diffuse intrinsic pontine glioma: a phase 1 trial

Seattle Children's Hospital · University of Washington · +9 more institutions

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Abstract

Abstract Diffuse intrinsic pontine glioma (DIPG) is a fatal central nervous system (CNS) tumor that confers a median survival of 11 months. As B7-H3 is expressed on pediatric CNS tumors, we conducted BrainChild-03, a single-center, dose-escalation phase 1 clinical trial of repetitive intracerebroventricular (ICV) dosing of B7-H3-targeting chimeric antigen receptor T cells (B7-H3 CAR T cells) for children with recurrent or refractory CNS tumors and DIPG. Here we report results from Arm C, restricted to patients with DIPG. The primary objectives were to assess feasibility and tolerability, which were both met. Secondary objectives included assessments of CAR T cell distribution and survival. A total of 23…

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119
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Authors

42

Topics & keywords

Keywords
  • Medicine
  • Regimen
  • Tolerability
  • Adverse effect
  • Refractory (planetary science)
  • Single Center
  • Internal medicine
  • Oncology
UN Sustainable Development Goals
  • Good health and well-being
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