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Mutations Synergize in Pediatric Brain Cancer

 8 months ago       82 Views

A new Northwestern Medicine study finds that a combination of two mutations cause a form of pediatric brain tumors to become more deadly, according to findings published in Nature Communications.

An experimental drug targeting one mutation prolonged survival in models of diffuse intrinsic pontine glioma (DIPG), but questions remain about the drug’s efficacy, according to Oren Becher, MD, associate professor of Pediatrics in the Division of Hematology, Oncology and Stem Cell Transplantation, and senior author of the study. Nevertheless, both mutations are promising targets for future therapies, Becher said.

“About 25 percent of kids with DIPG have these mutations — the mutations must be important for the biology of the disease,” said Becher, who is also an associate professor of Biochemistry and Molecular Genetics and a member of the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. “The first generation inhibitors need to be fine-tuned, but the results of this study show they have the potential to treat this disease in the future.”

Scientists have known about ACVR1 mutations’ role in non-cancerous disease for some time — specifically, in a rare condition called fibrodysplasia ossificans progressiva (FOP). In FOP, muscle and connective tissue such as ligaments and tendons are gradually replaced by bone, causing a progressive loss of mobility for afflicted patients.

Author: @DailyCupofYoga


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