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Details Of Gene Pathways Suggest Fine-Tuning Drugs For Child Brain Tumors

The direction of brain tumor research over the past several years reflects some of those data-driven advances, says Adam C. Resnick, Ph.D., the senior author of the current paper and principal investigator of the astrocytoma research team in the Division of Neurosurgery at Children's Hospital. "For years, astrocytomas have been lumped together based on similar appearance to pathologists studying their structure, cell shape and other factors," said Resnick. "But our current discoveries show that the genetic and molecular structure of tumors provides more specific information in guiding oncologists toward customized treatments."

Earlier this year, Children's Hospital announced its collaboration with the gene-sequencing organization BGI-Shenzhen in performing next-generation sequencing of pediatric brain tumors at the Joint Genome Center, BGI@CHOP. The center's sophisticated, high-throughput sequencing technology will greatly speed the discovery of specific gene alterations involved in childhood brain cancers.

This genomic discovery program dovetails with the work of the Childhood Brain Tumor Tissue Consortium, a multi-institutional collaboration recently launched by CHOP, with support from the Children's Brain Tissue Foundation. Because even large research centers may not hold enough tumor tissue specimens to power certain research, the consortium pools samples from a group of institutions, providing an important scientific resource for cooperative studies.

"The better we understand the mutational landscape of tumors, the closer we'll be to defining therapies tailored to a patient's specific subtype of cancer," added Resnick.

Funding from the National Institutes of Health (grants CA076931 and 5T32043126-09), the Matthew Larson Pediatric Brain Tumor Foundation, Alex's Lemonade Stand Foundation, the Rally Foundation for Childhood Cancer Research, the Bear Necessities Pediatric Cancer Foundation and A Kid's Brain Tumor Cure/PLGA Foundation supported this study. Plexxikon, Inc., provided the BRAF inhibitors used in the study. Sievert, Resnick and Storm are all on the faculty of the Perelman School of Medicine at the University of Pennsylvania in addition to their positions at Children's Hospital.

"Paradoxical activation and RAF inhibitor resistance of BRAF protein kinase fusions characterizing pediatric astrocytomas," Proceedings of the National Academy of Sciences, Online Early Edition, March 26, 2013. www.pnas.org/cgi/doi/10.1073/pnas.1219232110

About The Children's Hospital of Philadelphia: The Children's Hospital of Philadelphia was founded in 1855 as the nation's first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals and pioneering major research initiatives, Children's Hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country, ranking third in National Institutes of Health funding. In addition, its unique family-centered care and public service programs have brought the 516-bed hospital recognition as a leading advocate for children and adolescents. For more information, visit http://www.chop.edu.

Contact: John AscenziThe Children's Hospital of PhiladelphiaPhone: (267) 426-6055 Ascenzi@email.chop.edu

SOURCE The Children's Hospital of Philadelphia

Copyright 2011 PR Newswire. All rights reserved. This material may not be published, broadcast, rewritten or redistributed.
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