Northwestern Study Discovers Treatment For Pediatric Gliomas

The study, published in Nature Medicine, identified a small molecule inhibitor that increases histone methylation, compensating for the reduced methylation caused by mutation in the H3F3A gene, resulting in gliomas.

Researchers at Northwestern Medicine have discovered a new potential drug that shows promise in treating a rare and incurable form of brain tumor in children. When the researchers used an experimental drug called GSKJ4, it inhibited the tumor-forming ability of a specific genetic mutation and prolonged survival in mice models.

Pediatric brainstem glioma, also known as diffuse intrinsic pontine glioma (DIPG), is a type of brain tumor that grows in the brainstem, which controls many of the critical body functions. Due to their location inside the brainstem, these tumors cannot be removed by surgery, nor targeted effectively with radiation or chemotherapy. The disease is thus incurable, and patients survive less than one year with this condition.

"No significant advances in the survival of DIPG patients have been made over the last few decades, and new therapeutic approaches are desperately needed," said first author of the study, Rintaro Hashizume, MD, PhD, assistant professor in neurological surgery at Northwestern University Feinberg School of Medicine.

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