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Study Description

There are ~214 fully characterized patient cases with neuroblastoma (all tumor/normal pairs, 10 with relapse sample as well) that will make up the TARGET NBL dataset, along with some cell lines and xenografts. The dataset includes 24 4S cases as well. Each fully characterized case has gene expression, tumor and paired normal copy number analyses, methylation and comprehensive next-generation sequencing to include whole genome and/or whole exome sequencing. A majority of these cases will also have mRNA-seq and methylation data available as well. There are additionally a large number of cases, both low and high risk, with partial molecular characterization to include some next generation and targeted Sanger sequencing making this a large and informative genomic dataset. All cases can be sorted according to data type via the Case Matrix on the TARGET Data Matrix. Please visit the TARGET website, http://ocg.cancer.gov/programs/target, for additional information on this and other TARGET genomics projects. Please see the TARGET Publication Guidelines at the OCG website for updated details on sharing of any TARGET substudy data.

Authorized Access
Publicly Available Data
Study Inclusion/Exclusion Criteria

NBL patient samples were obtained from the Children's Oncology Group and chosen for inclusion in the TARGET project based on the following criteria: high risk group (<5 years at diagnosis, and 4S cases with available material were included as well), tumor samples contained >80% tumor nuclei and <30% necrosis, and there was an abundance of DNA and RNA for both tumor and paired normal available for use in expression, SNP and methylation arrays, and whole genome or exome sequencing.

Study History

Pediatric ALL and neuroblastoma were the first diseases to be piloted for the TARGET initiative, and ARRA funding allowed for its expansion to include pediatric AML, as well as osteosarcoma and kidney cancers. TARGET is jointly managed by the NCI Office of Cancer Genomics (OCG) and Cancer Therapy Evaluation Program (CTEP). With the success of the TARGET pilot studies, the initiative was expanded to include whole genome and exome sequencing for the neuroblastoma project. The neuroblastoma study is run as a cooperative collaboration between investigators and management staff at NCI, Children's Oncology Group, Children's Hospital of Philadelphia and Children's Hospital of Los Angeles.

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 pediatric ALL patients from the pilot phase of TARGET.
September, 2009 ARRA funding allows for expansion of TARGET pilot project to include additional cancers of study.
January, 2013 First TARGET NBL project publication highlighting whole exome sequencing analysis of high risk NBL patients.

Selected Publications
Diseases/Traits Related to Study (MeSH terms)
Authorized Data Access Requests
Study Attribution
  • Principal Investigator
    • John M. Maris, MD. Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Co-Principal Investigator
    • Robert Seeger, MD. Children's Hospital of Los Angeles, Los Angeles, CA, USA.
  • NCI Intramural Lead Investigator
    • Javed Khan, MD. National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.