GPC2-CAR T cells have potent preclinical activity against orthotopic medulloblastoma xenografts.

ABSTRACT: Medulloblastoma (MB) is the most common malignant brain tumor in children. Patients with group 3 and 4 MB have poor clinical outcomes, underscoring the urgent need for new therapies. Glypican-2 (GPC2) is a recently discovered oncofetal antigen. Given its expression in brain tumors, we evaluated the preclinical activity of our GPC2-chimeric antigen receptor (CAR) against MB and compared it to two existing CARs targeting GD2 and B7-H3. Gene expression analysis of publicly available datasets was performed and validated with immunohistochemistry staining of patient samples. The MB cell line D283 and a newly generated cell line patient-derived xenograft, MAF1433, were used. Cytokine bead assays and single-cell RNA sequencing (scRNA-seq) were used for mechanistic studies. MB patient samples expr

SUBMITTER: Okada R 

PROVIDER: S-EPMC12554907 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature