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 initially investigating the genome-related changes associated with acute lymphoblastic leukemia (ALL) and neuroblastoma, two common types of childhood cancer. More than 3,000 children in the United States are diagnosed with either ALL or neuroblastoma 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 (using high-resolution array-based methods to determine differences in the patterns of gene expression in cancer samples and non-cancerous samples) and genome structure studies (using high-resolution array-based methods to characterize genomic structural changes that correlate with each cancer, such as chromosome region gains and losses) are integrated to provide a complete genomic "overview" of each cancer.

• Gene Resequencing: Up-to-date genetic sequencing techniques 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 resequencing efforts.