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sector_ico_Health_trans Human Health

Stratifying and Targeting Pediatric Medulloblastoma Through Genomics

  • Project Leaders: Michael Taylor, David Malkin, Marco Marra
  • Institutions: BC Cancer (Previously BC Cancer Agency (BCCA))
  • Budget: $9856814
  • Program/Competition: Large Scale Applied Research Programs
  • Genome Centre(s): Genome Canada
  • Fiscal Year: 2011
  • Status: Closed

Cancer is the leading cause of non-accidental death in Canadian children, and brain tumors (medulloblastoma) are the leading cause of pediatric cancer deaths. The aggressive treatment needed has a significant impact on the quality of life of surviving children. Researchers in the Medulloblastoma Advanced Genomics International Consortium (MAGIC) have sequenced DNA transcriptomes and exomes of the cancer cells from over 1000 medulloblastomas gathered from around the world to discover subgroups of medulloblastoma that are genetically similar or identical, and develop specific markers that can then be used to stratify patients for treatment in clinical trials.  The project evaluated the level of risk that families are willing to take in reducing radiation therapy in order to increase the quality of life with tumours classified as “good prognosis”. The findings are of critical value to both the research community and practicing physicians, as the results revealed a simple, robust, and remediable explanation for the failure of prior clinical trials of targeted therapy, namely the absence of the target in recurrent disease. The research also demonstrated that less radiation – while still therapeutically viable – results in less brain damage and limits intellectual impairment. The integration of two technologies developed by the project will give clinicians confidence that subgroup assignments are robust, and clinically useful. The validation study for the subgroup-specific “nanoString assay” has been completed and the assay transferred into the CLIA lab at the Hospital for Sick Children, and other global sites.