What question did this study set out to answer?

Evaluate the feasibility, safety, and clinical activity of C7R-GD2.CAR T cell therapy in pediatric CNS tumors.

November 1, 2025Neuro-OncologyOpen Access

CTP-07. Phase I study of intravenous and intracerebroventricular C7R-GD2.CAR T cell therapy for pediatric central nervous system (CNS) tumors

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Evaluate the feasibility, safety, and clinical activity of C7R-GD2.CAR T cell therapy in pediatric CNS tumors.
Phase I clinical trial
Treatment with intravenous and intracerebroventricular delivery of C7R-GD2.CAR T cells
Lymphodepleting chemotherapy before administration
Patient population includes children with diffuse midline gliomas and medulloblastoma
Eight patients treated with IV-only regimen, showing no dose-limiting toxicities
One patient suffered transient grade 4 cytokine release syndrome
Six of seven patients with neurological deficits showed clinical improvement
Concurrent ICV-IV dosing led to increased neurotoxicity, whereas staggered dosing was better tolerated.

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Phase I report shows intravenous C7R-GD2.CAR T cells improved outcomes in CNS tumors, suggesting further investigation is warranted.

Mahdi et al. (2025) studied this question.

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