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Study Description

The Therapeutically Applicable Research to Generate Effective Treatments (TARGET) Initiative seeks to accelerate research in novel marker and drug development, along with understanding the molecular basis of pediatric malignancy, through identification of 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) - Another type of blood cancer marked by too many myeloblasts, an alternate type of immature white blood cell, are found in the blood and bone marrow.
  • Neuroblastoma (NBL) - Cancer of cells of the sympathetic nervous system.
  • Osteosarcoma (OS) - A cancer of the bone that primarily affects children and adolescents.
  • Wilms' Tumor (WT) - A cancer of cells in the kidney that can spread to the liver, lung and lymph nodes.

Together these cancers account for the majority of the more than 10,000 childhood cancer cases diagnosed in the United States each year.

TARGET is employing a set of advanced and complementary genome analysis technologies, including large scale 2nd and 3rd generation genome sequencing, to strategically characterize alterations in both gene expression and in genomic structure (such as deletions and amplification) that are involved in childhood cancers. The goal of this coordinated effort is a comprehensive genomic and transcriptomic profile of each cancer. Integrated analysis of the TARGET data will identify those genes that are either altered in their expression level or mapped to the chromosome regions of deletion/amplification/translocation, as these genes represent strong candidates for therapeutic targeting. To learn more about the TARGET project, visit the website at

*Additional renal tumors are being added to the TARGET Initiative (clear cell sarcoma of the kidney - CCSK, and rhabdoid tumor - RT), along with some sequencing of cell lines and xenografts in conjunction with the NCI PPTP project. More information can be found about each TARGET subproject by following the links on the righthand side of this page.

TARGET primary genomic sequencing datasets (controlled-access) and limited phenotype data (open-access) are available from this site. TARGET characterization data will be deposited into the TARGET Data Coordinating Center (DCC) database, while the sequence data is deposited either into the NCBI's trace repository or the sequence read archive (SRA). Comprehensive access to TARGET datasets, including molecular characterization (e.g. gene expression, copy number variation and epigenetics), fully annotated clinical information, and targeted sequencing linking tables, is available via the TARGET Data Matrix.

  • Study Weblink: TARGET Data Matrix
  • Study Type: Cohort
  • Number of study subjects that have individual level data available through Authorized Access: 2701

Authorized Access
Publicly Available Data (Public ftp)

Connect to the public download site. The site contains release notes and manifests. If available, the site also contains data dictionaries, variable summaries, documents, and truncated analyses.

More information on TARGET data is available on the TARGET Data Matrix.

Study Inclusion/Exclusion Criteria

TARGET employs stringent criteria for inclusion while focusing on primary untreated pediatric tumors. All cases included for TARGET must include a matched normal sample with a tumor specimen from the same patient. All samples used within TARGET are reviewed by a pathologist who confirms the accuracy of cancer diagnosis, as well as the presence of at least 70-80% tumor nuclei with no more than 20-30% necrotic tissue.

Each cancer being studied has disease-specific inclusion and exclusion criteria for the cohorts being characterized and sequenced. Additional details can be found on the TARGET website, particularly within the TARGET Data Matrix (

Study History

Pediatric ALL was the first disease to be piloted for the TARGET initiative, which is jointly managed by the NCI Office of Cancer Genomics (OCG) and Cancer Therapy Evaluation Program (CTEP). The study is run as a cooperative collaboration between investigators and management staff at NCI, Children's Oncology Group, University of Colorado Cancer Center, University of New Mexico Cancer Center, and St. Jude Children's Research Hospital.

May, 2005 NCI and the American Cancer Society co-sponsor "Childhood Cancer Targeted Therapeutics Workshop" that ultimately served as a basis for the conception of TARGET.
October, 2006 TARGET project officially initiated.
January, 2009 First publication of characterization and analysis of 221 ALL patients.
May, 2009 Follow-up publication of additional genes involved in ALL treatment response.
September, 2009 ARRA funding allows for expansion of TARGET pilot project to include three additional cancers.
October, 2009
November, 2009
February, 2010
July, 2010
December, 2010
June, 2011
August, 2012
December, 2012
January, 2013
Follow-up publications of additional genes involved in poor outcome in pediatric ALL.

Selected publications
Diseases/Traits Related to Study (MESH terms)
Authorized Data Access Requests
Study Attribution