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Promising Strategy to Fight Deadly Brain Tumor in Children

New research supported by Northwestern philanthropic partners opens the door to a promising new treatment strategy for diffuse intrinsic pontine glioma (DIPG)—the most deadly brain tumor in children.

The study, published in Nature Communications, found that an inhibitor of an enzyme called ACVR1 slows tumor growth and increases survival in an animal model of diffuse intrinsic DIPG. Currently, there are no approved drugs for treating DIPG. This research opens the door to a promising new treatment strategy.

“Our results are encouraging and suggest that it might be reasonable to test an inhibitor of this enzyme in a clinical trial,” says senior author Oren Becher, MD, associate professor of Pediatrics at Feinberg and a scientist at the Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago. “Prior to that, we need to evaluate different ACVR1 inhibitors in animal models to make sure we bring the most safe and effective agent to trials with children.”

In 2014, Dr. Becher’s lab co-discovered that ACVR1 mutations are found in approximately 25 percent of DIPGs, leading the enzyme to be overactive. In the current study, Dr. Becher and colleagues demonstrate for the first time in an animal model that this enzyme mutation cooperates with a histone mutation (H3.1 K27M) found in 20 percent of DIPGs. Together, these mutations are important in initiating tumor development.

Histone is a protein that acts like a spool for DNA, helping to package the six-foot long DNA strand into the tiny nucleus of every cell. Histones also help regulate which genes turn on and off, a process that goes awry when there is a histone mutation.

“Our future work will examine why and how the ACVR1 and histone mutations work together to trigger DIPG development,” says Dr. Becher, who also a professor of Biochemistry and Molecular Genetics and a member of the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. “Greater insight into this process will bring us closer to identifying a successful therapy for children with DIPG.”

The first author on the publication is Christine Hoeman, PhD, research assistant professor of Pediatrics at Feinberg. She worked for six years on this project in Dr. Becher’s lab.

This work was supported by the Damon Runyon Cancer Research Foundation, the Stewart Trust Foundation, National Institutes of Health grants R01 CA197313 and K02-NS086917, Madox’s Warriors, Fly the Kite Foundation, Cristian Rivera Foundation, John McNicholas Pediatric Brain Tumor Foundation, and the Cancer Smashers.

Make a gift to support the Malnati Brain Tumor Institute.

For more information about supporting brain tumor research or clinical care, please contact Elizabeth Gordon at elizabeth.gordon@northwestern.edu or (312) 503-0759.