ONC201 is the first of a new group of cancer therapies called imipridones that have a novel mechanism of action. Unlike other oncology drugs, ONC201 and other imipridones selectively target G protein-coupled receptors (GPCRs) that are dysregulated in cancers.
ONC201 is an orally active dopamine receptor D2 (DRD2) antagonist that is well-tolerated and has been shown in Phase I and II clinical trials to be active in specific advanced cancers.
ONC201 has shown efficacy in gliomas with the H3 K27M mutation. About 10% of adults with glioma have the H3 K27M mutation. About 50-60% of children with high-grade glioma (and 80-90% of children with Diffuse Intrinsic Pontine Gliomas, or DIPG) have the H3 K27M mutation.
Phase I/II clinical trials of ONC201 in glioma patients with the H3 K27M mutation are underway at several locations in the U.S.
Patients should be tested for the H3 K27M mutation and, if they meet the eligibility criteria, should be enrolled in these clinical trials.
The test is commercially available and easily accessible either through immunohistochemistry (IHC) staining or gene sequencing.
If your patient does not have the H3 K27M mutation or the tumor has not been biopsied, check the eligibility for this ONC201 clinical trial: NCT02525692.