This BSA Ad hoc subcommittee provides advice and oversight of the proactive NCI efforts to systematically apply a set of state-of-the-art methods to childhood cancer tumor specimens with the focused objective of identifying new therapeutic targets that can be exploited to improve outcome for children with cancer.

A public-private partnership agreement (Memorandum of Understanding) between the Foundation for the National Institutes of Health, Inc. and the National Cancer Institute shall be used to support the identification and validation of therapeutic targets for childhood cancers. This initiative is important because current treatment approaches have reached a plateau in improving outcome for children with cancer, and the introduction of targeted agents in the childhood cancer setting will benefit children only if they are utilized with a sound knowledge of relevant therapeutic targets for specific pediatric cancers.

The three initial areas of research focus are:

1. Application of high-throughput, cost-effective technologies to map and characterize comprehensively genomic and/or transcriptomic and/or epigenomic alterations.
2. Application of targeted high-throughput resequencing to identify genes or chromosome regions that are consistently mutated in specific childhood cancers. Genes or chromosome regions will be selected for resequencing based in large measure on the data generated through the genomic characterization research described above.
3. Application of high-throughput RNAi and small molecular screening methods to identify and validate therapeutic targets.