New Intelligence on Pediatric Brain Tumors

Diffuse intrinsic pontine gliomas have unique genetic mistakes

/ Author:  / Reviewed by: Joseph V. Madia, MD

(RxWiki News) The race is on to find cures for cancers that take the lives of those who haven't yet lived a full life. And progress is being made against one particularly aggressive childhood brain tumor.

Medical scientists have discovered that nearly 80 percent of a rare, lethal childhood brain tumor has mutations in genes that have not been tied to cancer before. These findings open the way for developing drugs that could target these genes.

The tumor called diffuse intrinsic pontine glioma (DIPG) is aggressive, and most (90 percent) children just can't beat it.

"If you notice a change in your child's breathing, call your pediatrician."

The St. Jude Children's Research Hospital – Washington University Pediatric Cancer Genome Project (PCGP) uncovered that the mutant genes found in DIPG are involved in how DNA is organized.

"We are hopeful that identifying these mutations will lead us to new selective therapeutic targets, which are particularly important since this tumor cannot be treated surgically and still lacks effective therapies," said Suzanne Baker, Ph.D., co-leader of the St. Jude Neurobiology and Brain Tumor Program and a member of the St. Jude Department of Developmental Neurobiology. She is a corresponding author of the study published in the

For this study, researchers sequenced the complete normal and cancer genomes of seven children with DIPG.

Investigators found that 78 percent of DIPG tumors have rearrangements in one of two genes that  belong to the histone H3 family of proteins. These genes have to do with the packaging of DNA.

According to researchers these mutations are apparently seen only in aggressive childhood brain tumors.

"It is amazing to see that this particular tumor type appears to be characterized by a molecular 'smoking gun' and that these mutations are unique to fast-growing pediatric cancers in the brain," said Richard K. Wilson, Ph.D., director of The Genome Institute at Washington University School of Medicine in St. Louis and one of the study's corresponding authors.

These are the latest findings of the Washington University Pediatric Cancer Genome Project (PCGP), an ambitious three-year undertaking to sequence the complete normal and cancer genomes of 600 children with some of the most aggressive and poorly understood childhood cancers.

The goal of this work is to find the differences between normal and cancer genomes and explain why cancer develops, spreads and kills. Armed with this new understanding, researchers believe they can then develop new tools to diagnose, treat and possibly even prevent cancer.

This research appears in the January 29 online edition of the scientific journal Nature Genetics.

The research was funded in part by the PCGP, including Kay Jewelers, the National Institutes of Health, the Sydney Schlobohm Chair of Research from the National Brain Tumor Society; the Cure Starts Now Foundation, Smile for Sophie Forever Foundation, Tyler's Treehouse Foundation, Musicians Against Childhood Cancer, the Noyes Brain Tumor Foundation and ALSAC.

Reviewed by: 
Review Date: 
January 29, 2012
Last Updated:
January 30, 2012