ONC201 is a new cancer drug that is now being tested in clinical trials. It is taken orally in capsule form and has been well-tolerated by patients.
Early results in brain cancer clinical trials suggest that this drug can kill cancer cells but not normal cells and may be effective in patients with a specific genetic mutation in their tumor: the H3 K27M mutation. This mutation primarily occurs in tumors that occur in the midline region of the brain (thalamus, pons, spinal cord). About 50-60% of children with high-grade glioma (and 70-90% of children with Diffuse Intrinsic Pontine Gliomas, or DIPG) have the H3 K27M mutation. About 10% of adult gliomas have the H3 K27M mutation.
Ask your doctor to have you tested, since there is a chance that you could have H3 K27M-mutant glioma and be eligible for one of our clinical trials.
The test for the H3 K27M mutation is readily available at most hospitals. The test involves taking a biopsy of the tumor and either staining (also referred to as immunohistochemistry (IHC) testing) or sequencing the tumor tissue.
If you have brain cancer that does not have the H3 K27M mutation or you have not had your tumor biopsied, ask your doctor to check your eligibility for this ONC201 clinical trial: NCT02525692.