Phase 1 trial of GPC2 CAR-T cells targeting GPC2 in pediatric CNS embryonal tumors

This single-site, open-label Phase 1 trial at Stanford University evaluates feasibility, safety, and preliminary activity of autologous GPC2 CAR-T cells delivered via intracerebroventricular infusions in children and young adults with relapsed or refractory medulloblastoma and other CNS embryonal tumors. Participants undergo leukapheresis for T cell collection, followed by lymphodepleting chemotherapy with fludarabine (DNA synthesis inhibitor) and cyclophosphamide (alkylating agent) before dose-escalated CAR-T administration, representing a first-in-human exploration of this novel intracerebroventricular delivery approach.