ONC201 is a new cancer drug that is now being tested in clinical trials. A list of all clinical trials with ONC201 can be found here.

A list of all ongoing trials for patients with gliomas, including H3 K27M mutant gliomas and DIPG can be found here.

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 a midline glioma 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.


ONC201 is being evaluated for efficacy in several cancer types in clinical trials across the United States. These trials include treatment for multiple myeloma, leukemia, lymphoma, neuroendocrine tumors (e.g. PCPG, DSRCT, adrenal tumors), endometrial cancer and other solid tumors.



Cancer Commons is a patient-centric 501(C)3 nonprofit network of patients, physicians, and scientists that helps identify the best options for treating an individual’s cancer. Members contribute to and benefit from a uniquely dynamic knowledge base of case histories, treatment insights and rationales, clinical trials, and patient outcomes and experiences.

The Musella Foundation For Brain Tumor Research & Information, Inc. is a 501(C)3 nonprofit organization dedicated to helping brain tumor patients by providing emotional and financial support, education and advocacy, and raising money for brain tumor research. The Musella Foundation has awarded over $5 million in direct patient assistance and has raised millions of dollars for brain cancer research. The Musella Foundation website serves as a rich information and educational resource for patients.