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. Clinical trials of ONC201 in glioma patients with the H3 K27M mutation are underway at several locations in the U.S.
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. 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.
ONC201 is being evaluated for efficacy in several cancer types and in trials across the United States.
These trials include treatment for multiple myeloma, leukemia, lymphoma, neuroendocrine tumors (e.g. DSRCT, adrenal tumors), endometrial cancer and other solid tumors. Please refer to our clinical trial page for a detailed list of all ongoing trials with ONC201.