The purpose of this Funding Opportunity Announcement (FOA) is to publicize a competition for Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Centers (MDSRCs). These Centers promote collaborative basic, translational, and clinical research and provide important resources that can be used by the national muscular dystrophy research communities. The Centers also provide outstanding environments for the training of new researchers capable of addressing high priority objectives in muscular dystrophy research. Center investigators are expected to participate in important community outreach efforts to increase awareness of their research activities in the patient and advocacy communities and to incorporate community perspectives into the conduct of patient-centered research.
The Muscular Dystrophy Community Assistance, Research, and Education Amendments of 2001 (the MD-CARE Act, Public Law 107-84) specified 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 centers program was subsequently developed in honor of Senator Paul D. Wellstone, a champion of muscular dystrophy research. Through open competitions and peer review of applications for awards, the participating NIH institutes established three Centers in 2003, three more in 2005 and have since maintained six active centers.
The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), The Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), the National Heart, Lung and Blood Institute (NHLBI) and the National Institute of Neurological Disorders and Stroke (NINDS) are committed to continuing and enhancing the tradition of scientific excellence that has been fostered in this centers program.
This Wellstone Centers program was formally evaluated in 2018 by an external Working Group of the NIAMS Advisory Council, facilitated by NIH staff. The report from the Working Group was approved by the Council on February 5, 2019, and the Executive Summary is available at https://www.wellstonemdcenters.nih.gov/sites/wellstone/files/WellstoneCenterEvalRptExecSumm-508.pdf . Recommendations described in the report were incorporated into this funding opportunity announcement and informed other aspects of the program.
Muscular dystrophy refers to a group of hereditary, progressive degenerative disorders causing weakness of the skeletal or voluntary muscles. There are many different forms of muscular dystrophy, which differ in their age of onset, severity, and pattern of muscles affected. Most types of muscular dystrophy are not simply muscle disorders, but rather multi-system disorders with manifestations in a variety of body systems, including the heart, respiratory system, gastrointestinal system, endocrine glands, skin, eyes, brain, and other organ systems. The major forms of muscular dystrophy include congenital, Duchenne/Becker, Emery-Dreifuss, facioscapulohumeral, limb-girdle, myotonic, and oculopharyngeal. Although some forms first become apparent in early childhood, others may not appear until middle age or later, but all have a significant clinical, economic, and psychosocial consequences.
Studies that are responsive to this FOA must be focused on one or more type of muscular dystrophy including those conditions listed above or other inherited condition(s) that directly affect muscle, lead to progressive weakness and muscle degeneration and have pathophysiological mechanisms and clinical manifestations similar to other muscular dystrophies. Potential applicants are strongly encouraged to contact NIH program staff listed under Scientific Contacts in section VII. below prior to developing an application if it is on a disease/condition not listed above, or risk having the application withdrawn as non-responsive.
Different muscular dystrophies are distributed across the progression from disease characterization to therapy development, to conducting clinical trials, to incorporating approved therapies into the standard of care. Currently, therapeutic options to treat any of the muscular dystrophies are limited. For some muscular dystrophies there remains a need for cohort studies to identify genetic mutations and characterize disease progression, as well as studies in cell and animal models to understand disease mechanisms. For other dystrophies there have been significant advances in the development of candidate therapeutics that address specific disease mechanisms, and several candidates are currently being tested in clinical trials. In addition to the development of therapies that address “root” causes, research in optimizing symptomatic treatment such as respiratory support, nutrition and physical therapy are critically important to improve quality of life and decrease associated morbidity. In addition to the need for new disease- or symptom-modifying therapies, there are gaps in understanding how the muscular dystrophies affect the lives of patients and their families and interfere with social interactions, education and workforce integration of those living with muscular dystrophies. There may also be missed opportunities for optimizing health care and improving accesses to care and services.
The Action Plan for the Muscular Dystrophies (https://mdcc.nih.gov/action_plan/2015-action-Plan-to-MDCC-508comp.pdf), updated and approved in November 2015 by the interagency Muscular Dystrophy Coordinating Committee (MDCC) (https://mdcc.nih.gov/index.htm), is a consensus document of high priority research objectives and other strategies to decrease the consequences of these diseases. This document was developed with input from patients, advocacy groups, Federal agencies and basic-, translational- and clinical-scientists to guide the muscular dystrophy research field for years to decades. Applicants to this FOA are encouraged to consider this Action Plan when selecting research projects for their proposed Center.
Cardiomyopathy and respiratory dysfunction in muscular dystrophies are clinically important but understudied. Patients often experience sleep disruptions due to decreased respiratory muscle function or other respiratory complications. High priority research objectives related to cardiomyopathy, respiratory dysfunction and disrupted sleep are included in the Action Plan, and applicants to this FOA are encouraged to consider proposing projects that address these and other objectives.
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Filed Under: Funding Opportunities