Clinical studies reveal extended survival in diffuse midline glioma patients receiving ONC201 treatment; research also explains the underlying mechanism of the drug’s success. For the first time, a potential drug candidate has been identified by researchers that show promise in improving outcomes f
Researchers have discovered a drug candidate, ONC201, that nearly doubled the survival rate for patients with aggressive childhood brain tumors known as diffuse midline gliomas or diffuse intrinsic pontine glioma .
In addition to reporting on the results of two early-stage clinical trials, the paper reveals the underlying mechanisms behind the compound’s success in these tumors. The paper is published inDiffuse midline gliomas including DIPG with a mutation called H3K27M are particularly aggressive, with an overall survival rate of 11-15 months. These tumors are most frequently found in children and young adults.
Meanwhile, Koschmann and co-author Sriram Venneti, M.D., Ph.D., were trying to figure out what was happening in these tumor cells. “This could explain why this patient population was responding so well to the drug because it had this specific epigenetic abnormality that could be turned off by ONC201. The tumors have an epigenetic change caused by the H3K27M mutation and ONC201 metabolically undoes that change,” said Venneti, associate professor of pathology and pediatrics and scientific research director of the Chad Carr Pediatric Brain Tumor Center at Michigan Medicine.
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