Science Highlights
Published on November 26, 2025
Targeting GPC2 with CAR T cells in neuroblastoma
by Clinical Cancer Research
Giudice AM, Matlaga S, Roth SL, et al. D3-GPC2-Directed CAR T Cells Are Safe and Efficacious in Preclinical Models of Neuroblastoma and Small Cell Lung Cancer. Clinical Cancer Research. 2025; (doi: 10.1158/1078-0432.CCR-25-0089).
Data gleaned from preclinical models support glypican 2 (GPC2) as a viable target for chimeric antigen receptor (CAR) T-cell therapy in the setting of neuroblastoma and small cell lung cancer. Building on promising results with their previously developed D3-GPC2 antibody, researchers engineered second-generation CAR T cells aimed at GPC2, which is highly expressed on human neuroblastomas and on cellular models of the disease. In a series of in vitro co-incubation studies, the D3-GPC2 CAR T cells induced robust neuroblastoma cell cytotoxicity and mediated substantial regression of GPC2-expressing neuroblastoma xenografts. No toxicities related to GPC2 CAR were documented. Additional safety and efficacy data are now being collected as part of a first-in-human Phase I clinical trial of relapsed or refractory neuroblastoma in pediatric patients. The study authors believe D3-GPC2 CART T cells also have potential for other malignancies, based on results from preclinical models of GPC2-expressing small cell lung cancers.
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