DoD Duchenne Muscular Dystrophy Translational Leverage Award

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The DMDRP Translational Leverage Award (TLA) mechanism is being offered for the first time in FY15. The TLA supports leveraging existing human-based resources in translational research to address high-impact research ideas or unmet needs in at least one FY15 DMDRP Focus Area. Often, resources are not leveraged for maximum use beyond the original source, and as a result, investigators expend time and money to duplicate those resources. This award supports translational research projects leveraging and/or querying existing repositories, registries, and/or clinical trials or clinical studies in order to conduct research in the FY15 DMDRP Focus Areas.Projects may include, but are not limited to, drug and/or device development, novel interventions, resource refinement, database mining, and correlative studies.Leveraging: For the FY15 DMDRP, leveraging involves addressing research questions using existing resources to amplify potential gains in knowledge in the field of DMD, and subsequently making the results or outcomes available for use by others. Outcomes from research funded by this award are expected to provide data and scientific rationale for future research that will impact DMD clinical care.• Human-Based Resources: Examples of human-based resources include, but are not limited to, biorepositories of clinical specimens, imaging repositories, epidemiological datasets, clinical databases, and databases of clinical data and/or metadata.• Biomarker Studies: For projects addressing the FY15 DMDRP Focus Area of “discovery and qualification of pharmacodynamic, prognostic, and predictive biomarkers,” a biological marker, or biomarker, is defined as a characteristic that is objectively measured and evaluated as an indicator of normal biologic processes, pathogenic processes, or biological responses to a therapeutic intervention.1 For the purpose of this award, biomarker qualification is defined as the evidentiary fit-for-purpose process of correlating a biomarker with the effects of an agent on biological processes and clinical endpoints.2 Alternatively, biomarker validation refers to the process of ensuring that a biomarker or technology (e.g., imaging) will be accurately and reliably measured through the performance characteristics of a biomarker assay.3 The DMDRP encourages the study of biomarkers that can be detected through minimally invasive procedures (e.g., blood, urine, tissue, imaging, etc.). Examples of biomarkers may include signatures of genetic or epigenetic changes, specifically expressed genes, proteins, or metabolites, and molecular, physiological, and/or imaging entities, among others.Clinical trials are not supported by this award mechanism.

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