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.
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 or diffuse intrinsic pontine glioma , compared to previous patients.
"It's an incredibly difficult tumor to treat," said senior author Carl Koschmann, M.D., associate professor of pediatric neuro-oncology and clinical scientific director of the Chad Carr Pediatric Brain Tumor Center at Michigan Medicine."Prior to this study, there have been more than 250 clinical trials that have not been able to improve outcomes. This is a major crack in the armor.
Through the trial, they collected cerebrospinal fluid from patients. They used this fluid to analyze metabolic changes and found ONC201 got into the tumor cells and affected mitochondria. Patients who responded to the drug had an increase in a metabolite called L-2HG produced by tumor cells. Additional clinical trials are currently underway, including testing ONC201 in combination with other therapies. Researchers at U-M's Chad Carr Pediatric Brain Tumor Center are also continuing to look at ways to overcome resistance to ONC201 by using drug combinations.
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