Study Unveils a Blueprint for Treating a Deadly Brain Tumor

In a study of mice and human brain tumors, researchers at the University of Michigan searched for new treatments by exploring the reasons why some patients with gliomas live remarkably longer than others. The results suggested that certain patients’ tumor cells are less aggressive and much better at repairing DNA than others but are difficult to kill with radiation. The researchers then showed that combining radiation therapy with cancer drugs designed to block DNA repair may be an effective treatment strategy. The study was funded by NIH.

The researchers focused on low-grade gliomas that carry a disease-causing mutation in a gene called isocitrate dehydrogenase 1 (IDH1), which encodes a protein known to help cells produce energy. This mutation is found in about 50 percent of cases of primary low-grade gliomas, a common and lethal form of brain tumor. Glioma patients whose tumors have mutations in IDH1 are typically younger and live longer than those whose tumors have the normal gene. These tumors also often have mutations in genes called TP53 (a tumor suppressor gene) and ATRX (a DNA-protein complex remodeling gene).

“Every year thousands of people are diagnosed with brain cancer and have little hope for long-term survival,” said Dr. Maria Castro, professor of neurosurgery at Michigan Medicine and a senior author of the paper published in Science Translational Medicine. “Our team’s mission is to find life-saving treatments for these patients. The results from this study could be a blueprint for extending, if not saving, the lives of many patients.”

“These findings have the potential to impact many younger glioma patients with low-grade tumors by either ‘curing’ them or extending their lives,” said Dr. Jane Fountain, an NINDS program director. “The preclinical model Dr. Castro’s team developed will be extremely valuable to cancer researchers. It closely mirrors the human disease.”