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Compositions and Methods for Treatment of Cancers Harboring an H3K27M Mutation

Summary of Invention

This invention represents a novel approach to treat H3K27M gliomas, which are highly lethal and predominantly occur in children as midline brain tumors or spinal tumors. This approach uses T-cells engineered to express a chimeric antigen receptor (CAR) that targets the GD2 ganglioside, a cell surface disialoganglioside shown by the inventors to be overexpressed on midline gliomas. The treatment is designed to improve survival of patients with H3K27M gliomas while minimizing on-target, off-tumor activity of the CAR T-cells.

International Publication Number

WO 2019/014456 A1 (.pdf)

International Application Filing Date