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Therapeutically Applicable Research to Generate Effective Treatments
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Kidney Tumors

Kidney tumors refer to a collection of various types of tumors that originate in the urinary system of the body. Kidney tumors are rare and generally curable in children, however, there are subsets of patients with poor prognoses that either do not respond to treatment or experience a relapse in their disease. Identifying genomic alterations in patients with a high risk (HR) for relapse and further targeting those lesions therapeutically can improve outcomes for these children.

TARGET Wilms Tumor (WT) Project

Wilms tumor (WT) is the most common type of kidney cancer seen in children, usually occurring before the age of five years. Although WT typically responds well to treatment, some patients relapse. In addition, WT treatment often includes toxic chemoradiotherapies that may leave surviving children with lifelong side effects. The discovery of novel therapeutic targets in WT could lead to more directed treatment regimens and reduce the sustained complications of harsh therapies.

Additional funding, largely through the American Recovery and Reinvestment Act (ARRA) of 2009, allowed for the expansion of the TARGET initiative to investigate additional pediatric cancers, including Wilms tumor. Following the success of the TARGET ALL pilot project, the kidney tumor project team employed similar comprehensive genomic approaches to identify new therapeutic targets for pediatric HR WT. Data were generated using clinically annotated, matched tumor and normal samples obtained from patients enrolled in various Children's Oncology Group (COG) biology and clinical trials.

Genome-wide analysis of matched tumor and normal samples (relapse sample when available)

  • Gene expression (Affymetrix U133+2.0 arrays)
  • Copy number analyses and loss of heterozygosity (Affymetrix SNP 6.0 arrays)
  • DNA Methylation (Illumina Infinium 27K methylation assay)
  • 2nd and 3rd Generation Sequencing
    • Whole Genome Sequencing
    • Whole Exome Sequencing

The types of data on more than 120 clinically annotated patient cases

  • 120+ fully characterized (gene expression, copy number analysis, and sequencing)
    • ~87 of those are relapse cases

Data generated for this project will be made available to the research community through the TARGET Data Matrix rapidly upon completion of validation.

Investigators

The TARGET kidney tumor project team consists of COG investigators at various institutions who work together with the scientists, analysts, project managers and technicians from the COG (Biorepository, Data and Statistics Core) and NCI offices (Office of Cancer Genomics, Clinical Therapy Evaluation Program, Center for Cancer Research and Center for Bioinformatics and Information Technology). This collaborative network is led by the following:

Principal Investigator

  • Elizabeth J. Perlman, MD. – Ann & Robert H. Lurie Children's Hospital of Chicago

NCI Bioinformatics Lead Investigator – National Cancer Institute, Center for Bioinformatics and Information Technology

  • Daoud Meerzaman, Ph.D.