What is ONC201?
ONC201 is the latest treatment for cancer cells that have been shown to kill most cancer cells without harming healthy cells in the body. This drug is being tested in a clinical trial for patients with recurrent glioblastoma or WHO Grade IV gliomas with the H3 K27M mutation.
ONC201 is an orally-active small molecule that targets the DRD2 receptor and kills cancer cells. For patients with newly-identified diffuse intrinsic pontine glioma, which is a type of brain cancer, the drug is eligible without histologic confirmation.
ONC201 targets the DRD2 receptor, an overexpressed protein in the brain known to promote the growth of cancer cells. Its goal is to stop the molecular death that can occur when these pathways are disrupted.