108th Congress

Disease-Specfic Research

Issue

There is continuing pressure from Congress and patient advocates to devote significant National Institutes of Health (NIH) resources to research related to specific diseases such as muscular dystrophy and autism. Since it is anticipated that NIH’s budget will increase at a slower rate in the future, competition for research dollars will escalate, and both Members of Congress and NIH will be forced to choose among diseases and programs.

Background

Advocates and their congressional supporters have long argued for increased funding, the creation of specialized research centers, and visibility for specific diseases, and the many disease-oriented NIH Institutes exemplify the power of these interests. During the past 20 years, AIDS advocates and breast cancer advocates have significantly increased funding for these diseases through congressional action. Members have commented that one of their most difficult tasks is responding to advocates with a devastating disease, since they want to be responsive but also want science to influence decisions, not politics. Several acts of specific interest to NIH have recently been passed to support research on these diseases.

In 1997, Congress enacted the Morris K. Udall Parkinson’s Disease Research Act, followed in fiscal year (FY) 2000 by a requirement for the development of a Parkinson’s Disease Research Agenda, including professional judgment funding projections. Despite significant increases to the NIH budget during these years, funding levels could not keep pace with the expectations of the Agenda, and FY 2004 appropriations committee report language noted that “the NIH’s projected Parkinson’s funding of $242,000,000 for fiscal year 2004 again falls substantially short of the $400,000,000 professional judgment budget estimate cited by the Research Agenda for that year.” Advocacy for this and other diseases, such as muscular dystrophy and autism, is reflected in hearings and appropriations report language from the 108th Congress.

The Children’s Health Act of 2000 (P.L. 106-310), signed into law on October 17, 2000, established the Pediatric Research Initiative and highlighted the need for autism centers to address diagnosis and treatment. The relationship between thimerosal in vaccines and the increased incidence of autism is a longstanding issue for Representative Dave Weldon (R-FL) and was the subject of two hearings held during the 108th Congress. FY 2005 House appropriations report language encourages NIH to pursue expanded research on autism spectrum disorders and the toxic effects of inorganic mercury and organic mercury compounds on young children, adults, and the elderly.

Overwhelming support for muscular dystrophy research resulted in the enactment of the Muscular Dystrophy Community Assistance, Research and Education Amendments of 2001 (P.L. 107-84), sponsored by Representative Roger Wicker (R-MS) and the late Senator Paul David Wellstone (D-MN). Since the passage of the Act, other Members have taken a personal interest in muscular dystrophy, and FY 2005 appropriations report language encourages NIH to increase its commitment to Duchenne muscular dystrophy translational research and clinical trials, develop opportunities for clinical trials, and help make available all existing and emerging patient care options, above and beyond current levels of research.

Current Status

NIH funded three Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers in October 2003. In March 2004, NIH reissued the request for applications for the Wellstone Centers and plans to make awards for up to three additional centers in FY 2005. In addition to ongoing extramural and intramural research projects, NIH is supporting the National Registry of Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Patients and Family Members, which collects and characterizes information from patients and their families.

NIH also funded the 8 autism centers that make up the Studies to Advance Autism Research and Treatment Network, which complement the 10 Collaborative Programs of Excellence in Autism centers and 2 Children’s Environmental Health Centers that focus on autism. These Centers conduct basic and clinical research, including investigations into the causes, diagnosis, early detection, prevention, and treatment of autism. In addition, the National Institute of Mental Health (NIMH), on behalf of U.S. Department of Health and Human Services, convenes the Interagency Autism Coordinating Committee, which serves as a forum for Federal agencies and members of the public to share information about their autism-related activities. NIH currently spends $40 million on muscular dystrophy and $96 million on autism.

Future Action

The Muscular Dystrophy Coordinating Committee has developed a Muscular Dystrophy Research and Education Plan for NIH, which was submitted to Congress in August 2004. The Committee’s next steps include analyzing existing activities within the muscular dystrophy community that relate to specific goals of the plan and refining the plan to identify specific research gaps and needs. In addition to the expansion of the Wellstone Centers, NIH is preparing to release a program announcement entitled “Muscular Dystrophy: Pathogenesis and Therapies” to encourage investigator-initiated research grant applications in basic, translational, and clinical muscular dystrophy research. A goal of this initiative is to promote research that will lead to better treatment for all types of muscular dystrophy. NIH is also preparing to release the solicitation entitled “Muscle Disease Research Career Development and Mentoring Awards,” a program announcement for mentored career development awards for research or clinical scientists engaged in laboratory, patient-oriented, or quantitative research; midcareer investigators in patient-oriented research; and newly independent scientists interested in expanding their programs in muscle disease research.

NIH Autism Coordinating Committee is developing new initiatives and priorities, including reissuing a program announcement to potential grant applicants entitled “Research on Autism and Autism Spectrum Disorders.” Other major initiatives include expanding a repository at the NIMH Center for Genetic Studies, which establishes resources for genetic studies and enhances data sharing; holding two NIH-sponsored workshops on confronting methodological challenges in research on interventions; and establishing the National Autism Brain Bank, which will create an infrastructure for enhanced brain acquisition for the purpose of neuropathological investigations to characterize the morphological aspects of the pathophysiology of autism.