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Home arrow Grants and Funding News arrow NIH - Muscular Dystrophy Cooperative Research Centers Program (U54) [11/19/07]
NIH - Muscular Dystrophy Cooperative Research Centers Program (U54) [11/19/07] PDF Print E-mail

http://grants1.nih.gov/grants/guide/rfa-files/RFA-NS-08-002.html 

The Muscular Dystrophy Community Assistance, Research, and Education Amendments of 2001 (the MD-CARE Act, Public Law 107-84) specified a number of provisions for expanding and intensifying research on muscular dystrophy. One provision of the MD-CARE act was that the NIH establish centers of excellence for research on muscular dystrophy. The Muscular Dystrophy Cooperative Research Centers (MDCRCs) program was subsequently developed in honor of Senator Paul D. Wellstone, a champion of muscular dystrophy research.

The National Institute of Neurological Disorders and Stroke (NINDS), the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), the National Institute of Child Health and Human Development (NICHD), and the National Heart, Lung, and Blood Institute (NHLBI) are now committed to continuing and enhancing the tradition of scientific excellence that has been fostered in the MDCRC program. While each center may contain a mixture of basic, translational, and clinical studies, the overall focus of this continuation of the MDCRC program is directly upon tightly integrated activities that foster the development of new therapies for the muscular dystrophies. Major review criteria for the MDCRC program include the degree to which an applicant demonstrates the potential to attack key problems in muscular dystrophy that require substantive collaborative efforts to solve and the ability of the applicant group to serve as a national infrastructure and training resource.

NINDS, NIAMS, NICHD, and NHLBI seek to further develop the MDCRC program to foster the translation of new scientific findings and technological developments into the clinical research setting. This FOA solicits both new (type 1) and competitive renewal (type 2) applications for MDCRCs. Under the FOA, each center may contain a mixture of basic, translational, or clinical research, as long as efforts are directed toward the steps required for therapeutic development. These may include, but are not limited to
- therapeutic target identification;
- characterization;
- validation;
- development of diagnostics and biomarkers to characterize or stratify patient populations;
- in vitro assay development;
- animal model development;
- candidate therapeutic efficacy screening;
- preclinical therapeutic optimization and FDA-required activities leading to an investigational new drug (IND) application;
- clinical research infrastructure;
- patient-oriented natural history studies;
- clinical outcome measure validation;
- cohort characterization;
- other studies in support of clinical trials; and
- early stage clinical trials for one or more types of muscular dystrophy. The proposed research, including a consideration of the balance between basic, translational, and clinical research, should be feasible within the budget limits described elsewhere in this FOA. Applicants should emphasize multi-disciplinary and collaborative studies that address one or more gaps in the therapeutic development pipeline. In addition, research problems should require substantial collaborative efforts to solve, and thus are best carried out in a center setting. Each MDCRC should involve clinical research and should engage muscular dystrophy patients and patient advocates in educational programs or as advisors.

Collectively and in cooperation with the NIH, the MDCRCs form part of a coordinated national program. Applicants are expected to emphasize new ideas, novel approaches, and state-of-the-art technologies to address any or all of the steps in the pipeline from identification of mechanistic targets for therapeutic development to translation of that knowledge into clinical interventions for the prevention or treatment of muscular dystrophy. Multidisciplinary collaborative efforts, in particular those involving basic scientists and clinicians with appropriate expertise, are expected for the components of an MDCRC. MDCRC applicants should also propose resource core facilities and training activities that will have national impact upon research in muscular dystrophy.