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Therapeutically Applicable Research to Generate Effective Treatments
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Promoting Discoveries

Leading to Cures

What is TARGET? The Therapeutically Applicable Research to Generate Effective Treatments (TARGET) Initiative seeks to harness the power of modern genomics technologies to rapidly identify valid therapeutic targets in childhood cancers so that new, more effective treatments can be developed and ultimately bring new hope to children and their families who face the devastating burden of these diseases.

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Cancer Genomics for Pediatric Cancers Video

Cancer Genomics for Pediatric Cancers
October 11, 2011

Javed Khan, M.D., a molecular biologist at the National Cancer Institute (NCI), discusses the approach and implications of cancer genome programs such as TARGET in this video.

Key pathways are frequently mutated in high risk childhood acute lymphoblastic leukemia: a report from the Children's Oncology Group
Blood
June 16, 2011

In the largest pediatric cancer genome sequencing effort reported to date, TARGET ALL researchers sequenced 120 candidate genes in 187 high-risk childhood B-precursor acute lymphoblastic leukemias (HR B-ALL). Integrated analysis combined validated somatic sequence mutations with results from genome-wide copy number alterations and gene expression profiles, and a high frequency of recurrent changes in key cancer signaling pathways was discovered. Further the frequency of mutations within the four major pathways varied greatly across genetic subtypes. These data highlight potential new therapeutic targets for in certain subsets of childhood ALL.

TARGET Sessions at the AACR 2011 Annual Meeting Highlighting Innovation in Pediatric Cancer Genomics

Ancestry and Pharmacogenomics of Relapse in Acute Lymphoblastic Leukemia
Nature Genetics
February 6, 2011

In a letter published in Nature Genetics, pediatric researchers found that Native American ancestry is genetically linked with an increased risk of relapse in childhood acute lymphoblastic leukemia (ALL), the most common cancer in children. Read more on these findings and how genomics studies can lead to modifications in therapy that will result in better outcomes for cancer patients.

For 5-year-old Hayley, NCI TARGET researchers are working hard to keep her neuroblastoma in remission through genetic targeting, with the goal of eliminating life-long side effects of traditional treatments. Recovery Act funds are making it possible. View the Video.

American Recovery Act Funding in Action at NCI:
TARGETing Genomic Mutations in Childhood Cancers to Make Therapeutic Advances

December 6, 2010

In this article, John Maris, M.D., Chief of the Division of Oncology at the Children’s Hospital of Philadelphia, explains how Recovery Act funds are enabling TARGET researchers to uncover the alterations in the neuroblastoma genome to develop new, more effective targeted therapies against this cancer. Read the full article.

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TARGET Podcast: NCI's Malcolm Smith, M.D., Ph.D., explains the impact of genomics research on the treatment of pediatric cancers.

ALL and Neuroblastoma data now accessible through new easy-to-use TARGET Data Matrix

Identification of Novel Cluster Groups in Pediatric High-Risk B-Precursor Acute Lymphoblastic Leukemia with Gene Expression Profiling
Blood
August 10, 2010

TARGET researchers have discovered eight unique subgroups among patients with pediatric high-risk B-precursor acute lymphoblastic leukemia (ALL). These subgroups can be distinguished by high-level expression of unique “outlier” genes, distinct DNA copy number abnormalities, specific clinical features and significantly different rates of relapse-free survival. Read more about the novel genes uncovered in this study.

Outcomes for Children and Adolescents With Cancer: Challenges for the Twenty-First Century
Journal of Clinical Oncology
May 20, 2010

Leaders from the NCI Cancer Therapy Evaluation Program (CTEP) and the Children’s Oncology Group provide an overview of the most current childhood cancer statistics, an analysis of the impact past research discoveries have had on survival and treatment outcomes, and essential information for prioritizing future treatment and research directions. Read more about the outcomes reported in this overview.

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Launch TARGET Data Matrix

The TARGET Initiative pursues three primary areas of research:

The TARGET Initiative is currently focused on identifying therapeutic targets in five childhood cancers:

  • Acute Lymphoblastic Leukemia (ALL)
  • Acute Myeloid Leukemia (AML)
  • Neuroblastoma
  • Osteosarcoma
  • Wilms Tumor

These cancers were the chosen for study because of their prevalence among children, the inadequacy of current treatment options and the availability of human tissue collections that met TARGET's strict scientific, technical, and ethical requirements. Learn more about the cancers selected for study by TARGET.

In the TARGET Initiative, the National Cancer Institute (NCI) actively collaborates with several world-class institutions to identify valid therapeutic targets in childhood cancers. Learn more