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
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
protein kinase fusions characterizing pediatric astrocytomas,"
Proceedings of the National Academy of Sciences,
Online Early Edition,
March 26, 2013
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: The Children's Hospital of
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