GD2-Targeting CAR T-cell Therapy for Patients with GD2+ Medulloblastoma.

ABSTRACT:

Purpose

Medulloblastoma (MB), the most common childhood malignant brain tumor, has a poor prognosis in about 30% of patients. The current standard of care, which includes surgery, radiation, and chemotherapy, is often responsible for cognitive, neurologic, and endocrine side effects. We investigated whether chimeric antigen receptor (CAR) T cells directed toward the disialoganglioside GD2 can represent a potentially more effective treatment with reduced long-term side effects.

Experimental design

GD2 expression was evaluated on primary tumor biopsies of MB children by flow cytometry. GD2 expression in MB cells was also evaluated in response to an EZH2 inhibitor (tazemetostat). In in vitro and in vivo models, GD2+ MB cells were targeted by a CAR-GD2.CD28.4-1BBζ (CAR.GD2)-T co

SUBMITTER: Ciccone R 

PROVIDER: S-EPMC11145172 | biostudies-literature | 2024 Jun

REPOSITORIES: biostudies-literature