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Therapeutically Applicable Research to Generate Effective Treatments
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Acute Myeloid Leukemia

Acute myeloid leukemia (AML) is a cancer that begins in the bone marrow in immature white blood cells called myeloblasts. Although a majority of childhood AML cases are successfully treated, there are a significant number of cases that relapse. Accurately diagnosing patients who are high-risk for relapse and treating them upfront with more targeted therapies can greatly enhance their outcomes.

TARGET AML Project

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 AML. Following the success of the TARGET ALL pilot project, the AML project team employed similar comprehensive genomic approaches to identify new therapeutic targets for pediatric HR AML. 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. Up to 50% of the cases studied in the TARGET AML project will include analysis of a relapse sample to produce a highly informative dataset.

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

  • Gene expression (Affymetrix Gene ST platform)
  • 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
    • Transcriptome Sequencing (mRNA-seq & miRNA-seq)

The types of data on more than 300 clinically annotated patient cases

  • 200 fully characterized (gene expression, copy number analysis, and sequencing)
    • 100 of those relapse cases
  • 100+ partially characterized

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 AML project team consists of multiple 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 lead by the following:

Principal Investigators

  • Soheil Meshinchi, MD., Ph.D. – Fred Hutchinson Cancer Research Center
  • Robert Arceci, MD., Ph.D. – Sidney Kimmel Comprehensive Cancer Center