Intravenous and intracranial GD2-CAR T cells for H3K27M⁺ diffuse midline gliomas.

ABSTRACT: H3K27M-mutant diffuse midline gliomas (DMGs) express high levels of the disialoganglioside GD2 (ref. ¹). Chimeric antigen receptor-modified T cells targeting GD2 (GD2-CART) eradicated DMGs in preclinical models¹. Arm A of Phase I trial no. NCT04196413 (ref. ²) administered one intravenous (IV) dose of autologous GD2-CART to patients with H3K27M-mutant pontine (DIPG) or spinal DMG (sDMG) at two dose levels (DL1, 1 × 10⁶ kg^-1; DL2, 3 × 10⁶ kg^-1) following lymphodepleting chemotherapy. Patients with clinical or imaging benefit were eligible for subsequent intracerebroventricular (ICV) intracranial infusions (10-30 × 10⁶ GD2-CART). Primary objectives were manufacturing feasibility, tolerability a

SUBMITTER: Monje M 

PROVIDER: S-EPMC11735388 | biostudies-literature | 2025 Jan

REPOSITORIES: biostudies-literature