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Genetically InFormed Therapies—A “GIFT” for Children with Cancer

The national investment that was made in oncology research with the passage of the National Cancer Act in 1971 is now coming to fruition. Nowhere is this more apparent than in the exciting prospects for genetically informed precision medicine as applied to the treatment of children with cancer. The wealth of information gleaned from intensive genetic analyses and NexGen sequencing studies has identified a number of viable targets in leukemias and solid tumors. Our rapidly evolving understanding of the enzymatic controls that regulate chromatin dynamics during normal differentiation of stem cells and their mutation or dysregulation in tumor cells is leading to a new library of therapeutically tractable tumor targets. The recent identification of germline variants associated with toxicity and/or response to therapy has further enhanced our ability to deliver individualized treatments for pediatric cancer patients. Our challenge today is to determine how best to use genomic data and integrate it into evolving clinical protocols to provide more efficacious therapies and a better quality of life for children with cancer. Clin Cancer Res; 18(10); 2735–9. ©2012 AACR.