Reengineering Translational Science: The Time Is Right

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Science Translational Medicine  06 Jul 2011:
Vol. 3, Issue 90, pp. 90cm17
DOI: 10.1126/scitranslmed.3002747


  • Table 1. The GWAS potential.

    GWAS* can reveal new therapeutic targets for complex diseases (8, 56, 57).

    DiseaseTotal GWAS hitsGWAS hits associated with marketed drugsGWAS hits associated with drug effects§
    Type 2 diabetes4468
    Multiple sclerosis3652

    *Genome-wide association studies (GWAS) assume no knowledge of disease pathogenesis and provide a comprehensive approach to the discovery of common genetic risk factors. Many known drug targets and associated pathways appear on the list of GWAS hits for common diseases, suggesting that other GWAS hits likely represent “druggable” targets worthy of further investigation.Genetic variants strongly linked to disease susceptibility.Genetic variants that are primary targets of drugs currently marketed for the listed indication. §Genetic variants associated with cellular, pharmacokinetic, pharmacodynamic, or clinical variations in response to one or more drugs currently marketed for the listed indication.

    • Table 2. NCATS components.

      Programs that will be incorporated into or managed by NCATS (excepting CAN, which has not yet been funded) together represent ~$720 million annually in research support.

      ProgramDescriptionContributions or expertises
      CTSA program (48)Infrastructure grants awarded to academic medical institutions to facilitate translational researchNetwork of 60 U.S. centers with expertise in preclinical science, clinical trials, comparative effectiveness research, training, and community engagement
      Components of the Molecular Libraries Program (58)Supports centers that provide access to large-scale screening, medicinal chemistry, and informatics for the identification of therapeutic and experimental chemical entitiesAssays development, high-throughput screening, medicinal chemistry, and compound databases
      Therapeutics for Rare and Neglected Diseases (TRND) (59)A drug-development pipeline within the NIH used for research collaborations with academic scientists, nonprofit organizations, and companies working on rare and neglected illnessesPreclinical development of promising compounds
      Rapid Access to Interventional Development (RAID) (60)A competitive granting program that provides resources for the development of new therapeutic agentsAccess to resources for preclinical development, production, bulk supply, GMP manufacturing, formulation, development of an assay suitable for pharmacokinetic testing, and animal toxicity
      Office of Rare Diseases Research (61)A multifunctional NIH office that serves as a focal point for rare diseasesCoordination and support of research on rare diseases
      NIH-FDA Regulatory Science Initiative (45, 46)A competitive grant program that funds regulatory scienceSupport of research on applicability of novel technologies and approaches to regulatory review of drugs, biologics, and devices
      Cures Acceleration Network (CAN) (62)A competitive grant program to fund translational solutions to high-need medical problems; awaits appropriationSupport of translational research with greater flexibility to NIH to fund innovative research in therapeutic development

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