House LHHS Appropriations Bill Report #108-88 FY04 NINDS

NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE

The Committee provides $1,468,926,000 for the National Institute of Neurological Disorders and Stroke (NINDS), which is $12,450,000 above the fiscal year 2003 comparable level and the same as the budget request.

Mission.–The NINDS supports and conducts basic and clinical neurological research and research training to increase understanding of the brain and improve the prevention and treatment of neurological and neuromuscular disorders. The NINDS mission encompasses over 600 disorders, including stroke; head and spinal cord injury; epilepsy; multiple sclerosis; and neurodegenerative disorders such as Parkinson’s disease.

Epilepsy.–Epilepsy remains a major, unsolved public health problem affecting the lives of millions of Americans and their families. The Committee seeks intensified efforts by the Institute in the prevention, treatment, and eventual cure of epilepsy. The Committee applauds the development of benchmarks for epilepsy research resulting from the `Curing Epilepsy: Focus on the Future’ conference held in March, 2000. The Committee encourages NINDS to address the research issues related to the impact of seizures on young women, children, the elderly and those with intractable or uncontrolled epilepsy. The Committee urges NINDS to develop specific research plans and goals for the anti-epileptic drug development program that has led to the discovery of many important anti-epileptic medications.

Alzheimer’s disease.–Research supported by NINDS continues to play an integral role in widening the scientific base of knowledge about Alzheimer’s disease. NINDS is working closely with the National Institute on Aging (NIA) in the area of immunotherapy for Alzheimer’s disease, which can involve the production of antibodies that reduce the cellular and behavioral effects of the disease. The Committee encourages NINDS to continue to assign a high priority to its Alzheimer’s research portfolio, and to continue to work closely with NIA and other institutes.

Amyotrophic lateral sclerosis (ALS).–ALS is one of a family of neurodegenerative diseases that plague millions of Americans. The Committee is pleased by the Institute’s recent efforts to intensify its research into ALS, and commends NINDS on its multiple initiatives involving high throughput screening and assay development to identify compounds with activity in neurodegenerative disorders, including ALS. The Committee understands that the Institute has worked with voluntary associations as co-sponsors of some of these activities, and encourages NINDS to continue such productive partnerships to understand and develop treatments for ALS. These efforts may lead to possible prevention and treatment interventions for other degenerative disorders.

The Committee is also pleased by the January 2003 scientific workshop on ALS that NINDS held with the Department of Veteran Affairs (VA), as well as other entities. The Committee encourages the Institute’s further collaboration with the VA in developing an initiative to address the scientific questions and gaps in the knowledge of ALS and motor neuron biology discussed at the workshop. The Committee also encourages the NINDS to coordinate, and collaborate on, ALS research with other appropriate NIH Institutes, particularly the NIEHS, and continue its partnership with other organizations, as appropriate, to advance ALS research.

Transmissible spongiform encephalopathies (TSE).–The Committee recognizes the efforts of NINDS, in collaboration with NHLBI, to fund contracts for the development of a biological assay for TSE. The Committee requests that the Director of the Institute to be prepared to provide a report on the progress made toward the development of a TSE bioassay at the fiscal year 2005 appropriations hearing. The Committee is particularly interested in the success in detecting disease-causing agents in blood, saliva, cerebrospinal fluid, and other bodily fluids, as well as lymphoid tissue, especially tonsils.

Therapies for multiple sclerosis and other immune system diseases.–The Committee encourages NINDS and NIAID to continue collaborative efforts to investigate the role of neutralizing antibodies as described in the FY 2003 conference report. The Committee further encourages NIH to use all available mechanisms, including conducting a scientific workshop, to investigate the use of existing, approved pharmaceuticals, biologicals, and other therapies as platforms for combination therapies to address diseases of the immune system, such as multiple sclerosis, and other diseases.

Dystonia.–The Committee continues to support the expansion of research on the neurological movement disorder dystonia, the third most common movement disorder. The Committee encourages NINDS to support additional research on both focal and generalized dystonia, and commends NINDS for its study of the DYT1 gene. The Committee encourages the Institute to continue its collaboration with the dystonia research community in supporting epidemiological studies on dystonia and in increasing public and professional awareness. The Committee commends NINDS for the recent release with other Institutes of the joint dystonia research program announcement. The Committee would like NINDS to be prepared to report on the dystonia research portfolio at the FY04 budget hearings.

Spina bifida.–Spina bifida is the leading, permanently disabling birth defect in the U.S. The Committee urges NINDS to allocate resources to and prioritize research on primary and secondary prevention for spina bifida.

Tuberous sclerosis.–Tuberous sclerosis complex (TSC) is a genetic disorder that affects many different organ systems. Genetic links provided by the TSC genes and research on TSC provide significant implications for scientific advancement benefiting other large patient populations suffering from epilepsy, lung disease, cancer, diabetes, autism, learning disabilities, and mental retardation. The Committee encourages NINDS to enhance research in this area, including consideration of the development of a comprehensive TSC patient registry, epidemiology studies on the natural history of TSC, and animal models/cell lines.

Juvenile diabetes.–The Committee commends NINDS for its efforts to prevent and treat hypoglycemia and neuropathy, both of which are serious complications of diabetes and demonstrate accelerated development in individuals with juvenile diabetes. The Committee encourages NINDS to expand its research in neuropathy by considering the establishment of centers specifically for detection and prevention of this dangerous complication of diabetes.

Neurofibromatosis (NF).–Advances in NF research have linked NF to cancer, brain tumors, learning disabilities and heart disease affecting over 150 million Americans. Because NF regulates both the RAS and cAMP pathways relating to cell growth and cognition, NF plays a pivotal role both in disorders of the brain and in cancer. The enormous promise of NF research is now reaching fruition in the testing of potential therapies. Therefore, the Committee encourages NINDS to enhance its NF clinical and basic research portfolios through clinical trials, RFAs, and other funding mechanisms to accelerate and exploit the substantial progress in NF research. The Committee commends NINDS for its leadership role in NF research and in coordinating efforts with other Institutes engaged in NF research.

Reflex sympathetic dystrophy syndrome (RSD).–The Committee recognizes the substantial burden RSD imposes upon people and the lack of understanding and adequate treatment for this disease. The Committee commends the NINDS for holding the December, 2001 state of the science meeting on RSD and encourages NINDS to follow through on the meeting’s recommendations, including efforts to develop standardized diagnostic criteria, to assess risk factors and incidence through epidemiology studies, to develop and validate animal models and to explore avenues for developing treatments.

Spinal muscular atrophy (SMA).–SMA is the most common genetic killer of infants and toddlers and is the most prevalent genetic motor neuron disease. While there is currently no cure for the disease, the research outlook is promising. Researchers have already identified the genes involved in SMA as well as compounds that may lead to potential treatments. The Committee understands that the severity of the disease, its relatively high incidence, and the possibility of imminent treatments have led the NINDS to select SMA as a model for a new approach to funding translational research. The Committee encourages NINDS to sponsor and convene a scientific and clinical workshop on SMA with a focus on implementing SMA translational research, and to employ existing mechanisms such as websites, publications, and participation in conferences to promote awareness of funding opportunities. The Director is also encouraged to work with other Institutes and Federal agencies to develop formal programs that increase public and professional awareness of the disease. The Committee requests NINDS to submit a report by March, 2004 on progress on all aspects of SSMA translational research.

Lupus.–Individuals with lupus commonly experience headaches, confusion, difficulty concentrating, and occasionally seizures, strokes, or other disorders of the nervous system. Stroke and seizures are a serious health effect of lupus. The Committee encourages NINDS to enhance efforts to study the neurological aspects of lupus.

Down syndrome.–Down syndrome is caused by extra genetic material on the 21st chromosome. It is the leading genetic cause of mental retardation in humans. The Committee encourages NINDS to enhance its efforts on Down syndrome, particularly as it relates to cognitive enhancement.
Mucolipidosis type IV (ML4).– Building on the identification of the gene that causes this debilitating genetic metabolic disorder, the Committee encourages NINDS to expand its efforts to support research leading to possible treatments and cures for those with ML4. In particular, NINDS is encouraged to support research involving other organisms which bear genes resembling the one whose mutation in humans causes ML4. This research should also offer insight into other genetic disorders.

Mucopolysaccharidosis (MPS).–The Committee is encouraged by NINDS sponsorship of a scientific conference focusing on central nervous system issues and the barriers to and development of effective therapies for MPS disorders. The Committee urges NINDS to solicit and provide for investigator proposals resulting from the findings of this conference. The Committee encourages NINDS, in collaboration with NIDDK and NICHD, to support current MPS research and study of the blood brain barrier as an impediment to treatment, and to use all available mechanisms to further stimulate and enhance efforts to better understand and treat MPS disorders.