Skip Navigation
NATIONAL CANCER INSTITUTE NATIONAL CANCER INSTITUTE
Therapeutically Applicable Research to Generate Effective Treatments
Go Go
 

Areas of Research

The TARGET Initiative, led by the National Cancer Institute in collaboration with several world-class institutions, seeks to identify valid therapeutic targets so that new, more effective treatments can be developed for children with cancer.

The TARGET Initiative is investigating the genome-related changes associated with acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), neuroblastoma, osteosarcoma, and high-risk Wilms tumor, five common types of childhood cancer. These tumors account for a majority of the more than 10,000 children in the United States diagnosed with cancer each year. Although improvements in survival for childhood cancers have been observed over the last several decades, the initial fast pace of these improvements has slowed, suggesting that we have reached a plateau in our ability to achieve better outcomes for patients and their families.

The research conducted by TARGET is divided into three distinct yet tightly integrated components that together form a system for selecting new molecular targets for the development of novel therapies for these childhood cancers:

  • Genomic Characterization: Gene expression studies and genome structure studies are integrated to provide a complete genomic "overview" of each cancer. High-resolution array-based methods determine differences in the patterns of gene expression in both malignant and non-cancerous samples, and further characterize genomic structural changes that correlate with each cancer, such as chromosome region gains and losses, loss of heterozygosity, and epigenetic changes in DNA methylation.

  • Gene Sequencing: Second generation sequencing techniques using whole exome, transcriptome or genome are used to "read" genes that have been identified to have altered expression and/or structural alterations to identify the specific cancer-related mutations in the DNA sequence.

  • Identification of Therapeutic Targets: RNA interference (RNAi) is used to identify and initially validate potential targets identified from the genomic characterization and sequencing efforts.