Therapeutically Applicable Research to Generate Effective Treatments (TARGET)

The Therapeutically Applicable Research to Generate Effective Treatments (TARGET) Initiative is committed to focusing genomics tools to rapidly identify potential therapeutic targets in childhood cancers so that new, more effective treatments can be developed in shorter time and ultimately bring new hope to children and their families who face the devastating burden of these diseases.

The TARGET Initiative seeks to identify the genomic changes associated with the following childhood cancers:

• Acute Lymphoblastic Leukemia (ALL)

  
  A fast-growing type of blood cancer in which too many immature white blood cells are found in the blood and bone marrow.

• Acute Myeloid Leukemia (AML)

  
  A cancer marked by too many myeloblasts (as opposed to lymphoblasts).

• Neuroblastoma

  
  A cancer of cells of the sympathetic nervous system.

• Osteosarcoma

  
  A cancer of cells in the bone that most frequently occurs in adolescents.

• Wilms Tumor

  
  A cancer often found in young children that begins with malignant cells in the kidney that can spread to the liver, lung and lymph nodes.

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 resequencing efforts.

The TARGET Initiative is a collaboration between the NCI Office of Cancer Genomics and the Cancer Therapy Evaluation Program (CTEP). To learn more about the Therapeutically Applicable Research to Generate Effective Treatments (TARGET) Initiative, visit http://target.cancer.gov/.

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