Scientists from the University of Michigan’s Center for Public Health and the Chad Carr Children’s Brain Tumor have found that a new anti-cancer drug can nearly double the survival rate of children with aggressive brain tumors. Research published in the journal Cancer Discovery.
The scientists tested a compound called ONC201 in children with diffuse midline glioma with the H3 K27M mutation. A total of 71 patients participated in the two clinical trials. Almost one-third of patients lived longer than two years, although survival is usually 11-15 months.
Diffuse median gliomas are most common in children and young adults. The only treatment available is radiation, and it is not always effective as it can damage areas of the brain that have important functions.
Analysis of the patients’ cerebrospinal fluid showed that the drug ONC201 penetrated the tumor cells and affected the mitochondria, the structures responsible for cellular energy production. In patients who responded to the treatment, there was an increase in the substance called L-2HG, which is secreted by cancer cells and slows down their division. In fact, the drug neutralized the effect of the H3K27M mutation.
“This is an incredibly difficult tumor to treat. Prior to this study, there were more than 250 clinical trials that failed to improve outcomes,” said senior author Carl Koschmann, Associate Professor of Pediatric Neuro-Oncology and Clinical Scientific Director of the Chad Carr Child Brain Tumor Center.
He also noted that for families of patients with such a diagnosis, even an almost double increase in survival is not enough, as the tumor remains so deadly. But he hopes this first step will lead to greater success in the future.
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