Researchers at the University of Michigan’s Center for Public Health and the Chad Carr Children’s Brain Tumor Center report that a new anti-cancer drug may nearly double survival in children with aggressive brain tumors. The findings appear in Cancer Discovery.
The study tested a compound named ONC201 in children diagnosed with diffuse midline glioma carrying the H3 K27M mutation. A total of 71 patients took part across two clinical trials. Roughly one-third lived beyond two years, compared with typical survival of 11–15 months for this condition.
Diffuse midline gliomas are most common among children and young adults. Standard treatment options have relied on radiation, which can be effective but often damages brain regions essential for function, limiting overall benefit.
Analysis of cerebrospinal fluid from patients indicated that ONC201 penetrated tumor cells and targeted mitochondrial processes, the energy hubs of cells. In responders, researchers noted an increase in L-2HG, a molecule released by cancer cells that slows their division. The drug appears to counteract the impact of the H3K27M mutation in these cases.
“This tumor is notoriously hard to treat. Before this study, more than 250 trials had failed to improve outcomes,” stated the senior author, a pediatric neuro-oncology expert and clinical scientific director at the Chad Carr Center.
The investigator added that even a substantial extension in survival can feel small to families facing a diagnosis of this relentless cancer. Still, the work marks an important step forward and may guide future advances in therapy.
In related context, ongoing research continues to explore how early interventions and combination strategies could enhance treatment response and quality of life for affected children and their families.
Additional laboratory findings suggest that targeted approaches affecting cancer metabolism and epigenetic regulation could complement existing therapies. Researchers emphasize the need for broader clinical testing and careful evaluation of long-term outcomes to determine the full potential of ONC201 in pediatric patients.