DoD Duchenne Muscular Dystrophy Therapeutic Idea Award

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The DMDRP Therapeutic Idea Award mechanism was offered in FY12 and FY14, and is being offered again in FY15. It is designed to promote new ideas that are still in the early stages of development with the potential to yield high-impact data and new avenues of investigation for novel therapeutics for DMD treatment. This award mechanism supports conceptually innovative, high-risk/high-reward research that could ultimately lead to critical discoveries or major advancement in DMD therapeutics. Research projects should include a well-formulated, testable hypothesis based on strong scientific rationale. Presentation of preliminary data is not consistent with the intent of the Therapeutic Idea Award mechanism. While the inclusion of preliminary data is not prohibited, the strength of the application should not rely on preliminary data. Innovation and Impact are the most important aspects of the Therapeutic Idea Award. Innovation: Research deemed innovative may introduce a new paradigm, challenge current paradigms, introduce novel concepts or agents, or exhibit other uniquely creative qualities that may lead to potential therapies for DMD (e.g., drug repurposing studies). Innovative research may include high-risk approaches to DMD research. Research that is investigating the next logical step or is an incremental advance upon published data is not considered innovative. Impact: Research that has high potential to significantly impact development of therapeutics for DMD. 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 The DMDRP encourages the discovery and 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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