Pediatric cancer researchers at The Children's Hospital of Philadelphia contributed important expertise to a new landmark study of medulloblastoma, a type of brain tumor typically found in children. The large multicenter study defines the genetic landscape of this cancer, and holds intriguing clues to gene changes on signaling pathways that may become fruitful targets for future therapies.
The most common cancerous brain tumor in children, medulloblastoma is, fortunately, rare. However, it causes significant mortality, and survivors may suffer serious long-term side effects from treatment, so less toxic, more effective therapies are sorely needed.
The study appears online today in the journal Science. Collaborators from three countries and more than a dozen institutions co-authored the research, which was led by three scientists from Johns Hopkins University and the Howard Hughes Medical Institute: Bert Vogelstein, M.D., Kenneth Kinzler, Ph.D., and Victor Velculescu, M.D., Ph.D.
The researchers used recent innovations in gene-sequencing and informatics technology to analyze the whole exome (all the genes known to code for proteins) of medulloblastoma tumors. "This is the first application of whole-exome DNA sequencing analysis to a solid pediatric tumor, and there were encouraging results," said co-author Tom Curran, Ph.D., whose laboratory at The Children's Hospital of Philadelphia focuses on finding new treatments for medulloblastoma.
Curran added, "The study team found that the number of mutations in pediatric medulloblastoma tumors is five to ten times fewer than in adult medulloblastoma tumors. This suggests that, compared to adult tumors, pediatric tumors may respond better to drugs that target the genes and pathways altered by mutations that drive cancer progression. This is a hopeful finding."
In addition to detecting gene alterations previously found in medulloblastoma, such as mutations in the Hed
|Contact: John Ascenzi|
Children's Hospital of Philadelphia