Neuro-oncology covers all tumors of the brain and spinal cord, including, astrocytoma, glioma, glioblastoma, ependymoma, and others. These diseases are devastating to patients, and challenging to treat. Common treatments are radiation and, when accessible, surgery, but these tumors almost always recur.  Most therapeutic agents can not cross the blood brain barrier to access the tumor, and are therefore not useful for the treatment of these diseases.

Because ONC201 is able to cross the blood brain barrier and achieve high concentrations in the brain, and the fact that its mechanism of action relies on the dopamine receptor, which is highly expressed in the brain, it is uniquely suited to address these indications.

At an event sponsored by Oncoceutics, held in April, 2017, Dr. Minesh Mehta spoke about the challenges in treating patients with glioblastoma and other neuro-oncology indications. Additionally, he highlighted the opportunities presented by ONC201 and its unique ability to address GPCRs, specifically the dopamine receptor (DRD2) to treat patients with this devastating set of diseases.

Dr. Arrillaga is a neuro-oncologist at the Massachusetts General Hospital. She served as clinical director of the Pappas Center for Neuro-Oncology and most recently has become the dedicated CNS Phase 1 Oncologist for the group. Dr. Arrillaga is interested in early phase clinical trials for gliomas and serves as overall Principle Investigator (PI), site PI, and co-investigator, in several therapeutic and non-therapeutic clinical trials for patients with brain tumors including the Phase 2 trial of ONC201 in recurrent glioblastoma, which is currently accruing patients at MGH and DFCI.

She recently spoke about the early results seen in that study, specifically the results seen in a patient with a tumor mutation (H3.3 K27M) which may confer particular sensitivity to ONC201.