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This is an autologous glypican-2 (GPC2)-targeted chimeric antigen receptor (CAR) T-cell therapy developed by Stanford University for the treatment of pediatric central nervous system (CNS) embryonal tumors. The therapy involves genetically engineering a patient's own T cells to express a second-generation CAR that recognizes GPC2, a cell-surface proteoglycan and oncofetal antigen highly expressed in various aggressive pediatric brain tumors. Administered via intracerebroventricular (ICV) infusion directly into the cerebrospinal fluid, these CAR T cells are designed to induce tumor regression while minimizing systemic toxicity. It is currently being evaluated in a Phase 1 clinical trial for relapsed or refractory medulloblastoma and other CNS embryonal tumors, including atypical teratoid/rhabdoid tumors (ATRT), pineoblastoma, and embryonal tumor with multilayered rosettes (ETMR).
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