DoD Duchenne Muscular Dystrophy Investigator-Initiated Research Award

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The DMDRP Investigator-Initiated Research Award (IIRA) supports translational research that will accelerate the movement of promising ideas in DMD into clinical applications. Translational research may be defined as an integration of basic science and clinical observations with the specific goal of developing new therapies. While the ultimate goal of translational research is to move an observation forward into clinical application, translational research is most effective as a two-way continuum between the bench and the bedside. Within this continuum, the IIRA supports mid-stage or later translational research projects, including early-phase, proof-of-principle clinical trials and correlative studies to better inform the development of drugs, devices, and other interventions. Research projects may also include preclinical studies utilizing animal models, human subjects, or human anatomical substances. Studies proposed under this award should not include: • Target discovery • Drug screening • Mechanism of action studies • Hypothesis-driven pathophysiology studies 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.

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