For the first time, researchers have found a potential drug candidate that improved outcomes for patients with a type of childhood brain tumor for which there are no effective treatments. The compound, called ONC201, nearly doubled survival for patients with diffuse midline glioma (DMG) or diffuse intrinsic pontine glioma (DIPG), compared to previous patients.
The findings are reported by an international team of researchers led by the University of Michigan Health Rogel Cancer Center and the Chad Carr Pediatric Brain Tumor Center., 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.
In two clinical trials testing ONC201 in a total of 71 patients with H3K27M-mutated diffuse midline gliomas, thewas nearly 22 months for tumors that had not recurred at the time of enrollment. Almost a third of the patients lived longer than two years. ONC201 took an unusual path to a clinical trial. Initially designed to target dopamine receptors, which are upregulated in many different tumors, researchers saw that the drug passes the, one of the biggest challenges to designing drugs for brain tumors. Initial trials in glioblastoma were not successful, but a small number of patients with DMG that carried the H3K27M mutation had more promising results.
Meanwhile, Koschmann and co-author Sriram Venneti, M.D., Ph.D., were trying to figure out what was happening in these tumor cells.
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