110th Congress

Paul D. Wellstone Muscular Dystrophy Community Assistance, Research, and Education Amendments Act, 2008

H.R. 5265, S. 2618

Background

Muscular dystrophy (MD) refers to a group of more than 30 genetic diseases that cause progressive weakness and degeneration of skeletal muscles used during voluntary movement. These disorders vary in age of onset, severity, and pattern of affected muscles. All forms of MD grow worse as muscles progressively degenerate and weaken, and the majority of those affected by the disease eventually lose the ability to walk. Some types of MD also affect the heart, gastrointestinal system, endocrine glands, spine, eyes, brain, and other organs. Respiratory and cardiac diseases are common, and some patients with MD may develop a swallowing disorder. MD is not contagious and cannot be brought on by injury or activity.

The National Institute of Neurological Disorders and Stroke (NINDS), National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), and Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) support the majority of research studies on MD at the National Institutes of Health (NIH). The goals of these studies are to increase understanding of MD and its causes, develop better therapies, and ultimately find ways to prevent and cure it. The National Heart, Lung, and Blood Institute (NHLBI) also supports a number of research grants focused on ultimately improving heart and lung health for children and adults with MD.

On December 18, 2001, the President signed into law the Muscular Dystrophy Community Assistance, Research and Education (MD-CARE) Amendments of 2001 (P.L. 107-84). The NIH-related provisions in the MD-CARE Act amended Title IV of the Public Health Service Act to require the Director of NIH, in coordination with the Directors of NINDS, NIAMS, NICHD, and other national research institutes, as appropriate, to expand and intensify programs with respect to research and related activities concerning Duchenne, myotonic, facioscapulohumeral, and other forms of MD. The MD-CARE Act also required the establishment of MD Centers of Excellence and the Muscular Dystrophy Coordinating Committee (MDCC), and authorized such sums as may be necessary (later eliminated by P.L. 109-482, the NIH Reform Act of 2006) for activities required by the statute.

NIH currently funds six Paul D. Wellstone Centers supported by NINDS, NIAMS, and NICHD. The Wellstone Centers program has built-in set-aside funds to promote new collaborations, and a total of 14 projects have been approved for funding using these collaborative funds, 10 of which were approved in 2007. NHLBI recently joined NINDS, NIAMS, and NICHD in reissuing a request for applications for the selection of the next set of Wellstone Centers. These new Centers will each contain a minimum of one core facility providing services, data, or biological specimens that will be available for use by other Centers and the broader MD research community, and another core facility focused on training new investigators and engaging patients in research and education activities. To further promote and disseminate information related to the activities of the Wellstone Centers, a new Web site (http://www.wellstonemdcenters.nih.gov/index.htm) is available to investigators and patients interested in MD research, which includes new NIH funding opportunities. This Web site also provides information for MD investigators interested in using resources, facilities, and reagents that the Centers share with the research community.

MDCC coordinates activities related to the various forms of MD across NIH and with other Federal health programs. The MD-CARE Act directed MDCC to develop a plan for conducting and supporting research and education on MD through the various national research institutes. The MDCC Action Plan for the Muscular Dystrophies, approved by MDCC in December 2005, contains specific research objectives that are appropriate to the missions of all MDCC member agencies and organizations and thus serves as a central focus for the coordination of MD research.

NHLBI was recently invited to join MDCC. Although representatives from NHLBI have been actively participating in committee activities, the addition of an NHLBI representative to MDCC further emphasizes the importance of NIH-supported research on the cardiopulmonary complications associated with MD and of greater collaboration between NIH Institutes.

Provisions of the Legislation/Impact on NIH

The legislation would reinsert an authorization of appropriations section in the Public Health Service Act for such sums as may be necessary for fiscal years 2008–2012, name in statute the MD centers of excellence as the Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers, specifically name NHLBI as a member of MDCC, and require MDCC to give special consideration to enhancing the clinical research infrastructure to test emerging therapies for the various forms of MD.

The Centers for Disease Control and Prevention, in carrying out the MD information and education program required by the MD-CARE Act, would be required to partner with leaders in the MD patient community and widely disseminate the Duchenne-Becker MD care considerations.

The Agency for Healthcare Research and Quality (AHRQ) would be required to evaluate the available scientific evidence for the appropriate medical or patient organizations to develop and issue an initial set of care considerations for Duchenne-Becker MD and provide ongoing review and updates where appropriate. AHRQ would also be required to replicate the same systematic review methodology used to develop the Duchenne-Becker MD care considerations as a model for other types of MD.

Status and Outlook

H.R. 5265 was introduced by Representative Eliot Engel (D-NY) on February 7, 2008, and was referred to the House Committee on Energy and Commerce. No further action has occurred on this legislation.

S. 2618 was introduced by Senator Amy Klobuchar (D-MN) on February 8, 2008, and was referred to the Senate Committee on Health, Education, Labor and Pensions. No further action has occurred on this legislation.

March 11, 2008