Study identifies potential treatments for children's brain cancer

By Ryan Maass

SALT LAKE CITY, Oct. 27 (UPI) -- Researchers at the University of Utah Health Sciences have identified potential drugs for treating brain cancer in children.

In a study published in the journal Cell Reports, the research team says their potential new treatments reduce or eliminate a subgroup of childhood brain cancers while leaving brain tissue intact. The team conducted their research using a zebrafish animal model in combination with studies on human brain tissue.


"For many pediatric brain tumors no cell or animal model exist to test targeted, or personalized, medications that could significantly improve survival and alleviate the harmful side effects of conventional therapies," study author Rodney Stewart said in a press release. "Indeed, children with rare brain tumors have few options for life-saving treatment. Our hope is by creating this animal model we will be a step closer to finding effective therapies."

With the help of zebrafish, the research team examined primitive neuroectodermal tumors of the central nervous system, a highly aggressive pediatric tumor also known as CNS-PNET. Few animal or cell line models exist for this analysis, making the zebrafish ideal test subjects. Stewart and other investigators spent 7 years developing the experimental model.


The team tested several existing compounds to see if they could identify a targeted therapy for the oligoneural subgroup, and observed MEK inhibitors were capable of effectively reducing the tumors.

"When we treated the fish with MEK inhibitors - drugs that inhibit an enzyme - they exhibited a remarkable response," Stewart explained. "Not only was the tumor burden reduced, it completely eliminated the tumor in about 80% of the fish and those tumors have not come back. This is a durable response from a transient treatment. It's what we look for in cancer therapy, an effective drug that can be taken for a certain amount of time but, after the cancer is gone, patients can stop taking the drug and go on living their lives."

While the study's authors are pleased with their results, they stress there are important differences between brain tumors in fish and humans. They are calling for additional studies to determine if the same positive responses can be found in humans.

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