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.