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Fiscal Year 2001 Budget Request

Senate Appropriations Subcommittee on Labor, Health and Human Services, Education and Related Agencies Statement of Gerald D. Fischbach, M.D. Director, National Institute of Neurological Disorders and Stroke

Mr. Chairman and Committee Members:

I am pleased to present the President's non-AIDS budget request for the NINDS for Fiscal Year 2001, a sum of $1,050,412,000, which reflects an increase of $54,327,000 over the comparable Fiscal Year 2000 appropriation. Including the estimated allocation for AIDS, total support requested for NINDS is $1,084,828,000, an increase of $55,085,000 over the Fiscal Year 2000 appropriation. Funds for the NINDS efforts in AIDS research are included within the Office of AIDS Research budget request.

I became Director of NINDS eighteen months ago with great enthusiasm about neuroscience research and the likelihood of significant advances in treating neurological disorders that were considered intractable only a few years ago. My enthusiasm has grown with time because new discoveries, generous public support and a widening sphere of collaborations within the NIH and with outside organizations have brought our mission of reducing the burden of neurological diseases into clearer focus.

We are now in the second year of a strategic planning process that has galvanized our research and patient communities as well as our own staff. Last year's planning document, "Neuroscience at the New Millennium," identified major targets of opportunity and laid out a strategy for approaching disease problems and for strengthening the capacity of the research community to continue the stunning advances of recent years. The momentum generated by this process, that engaged efforts of more than 100 distinguished extramural and intramural scientists, professional societies, and many patient advocates, resulted in many new initiatives. The Strategic Plan is based on the cross-cutting topics of neurodegeneration, neural repair, neurodevelopment, neurogenetics, synapses and circuits, cognition and behavior, and the neural environment. Our Plan is now in its second phase. Because the planning panels were so successful, we reorganized the extramural program staff into working clusters that track the major planning topics. This flexible, non-hierarchical structure has led to productive interactions among our program directors, senior staff, and external advisors in advancing our research agenda and in responding to the initiatives of investigators and to concerns of the lay members of the planning community.

Uses of FY 2000 Increase
The FY 2000 appropriation will allow NINDS to maintain and build on critical initiatives begun in FY 1999 and to take advantage of new, extraordinary opportunities, including support of 200 more project grants and 50 more scientists in training and career development. I am pleased to report that in FY 1999 we were able to fund eight new Morris K. Udall Centers of Excellence in Parkinson's Disease, instead of the five we had planned. Together with the three Centers funded in late 1998, we now have a national network of eleven Centers that includes a wide spectrum of basic and clinical research. Annual meetings of the Centers, along with ongoing informal interactions, will increase opportunities for collaboration and maximize this significant investment. Each Center has a training component, so new investigators will be introduced to Parkinson's disease and related disorders each year.

Another new initiative this year seeks to explore the promising new technology of deep brain stimulation in Parkinson's disease and other neurological disorders. Studies of electrode design, patterns of stimulation, and clinical trials will determine if DBS can halt the progress of neurodegeneration as well as reverse disabling symptoms.

Another solicitation is concerned with the safety of the blood supply. We seek a rapid and sensitive test for the infectious agent (prion) responsible for the new variant Creutzfeldt-Jakob Disease. The public must be confident in the safety of the blood supply.

During the current year, we will expand our efforts to apply sophisticated technology to map the location and timing of gene expression in the brain. This is an essential step in determining the function of normal and mutant, disease-causing genes. We will expand our successful neural prosthesis program, and we will develop innovative, high throughput screens for potential therapeutic agents. We intend to promote new approaches to spinal cord injury, and we plan a broad approach to analyze the efficacy of neural stem cells in repairing focal and generalized lesions. Building on one of our most successful innovations in FY 1999, we plan to expand our support for a full range of infrastructure needed for modern neuroscience research. Finally, we plan to increase our investment in training physician-scientists who are most likely to engage in translational research and patient oriented research. Looking to the future, I would like to tell you about just a few of our major initiatives and priorities for FY 2001.

A Healthy Brain for Life
We are concerned with neurological disorders over the entire lifespan. It is important to focus on developmental and degenerative disorders of children that can produce a lifetime of disability. Our efforts range from a new, exploratory grants program looking for new insights into common disorders such as autism and epilepsy, rare disorders such as Rett's Disease, Batten's Disease, and lipid storage diseases. We have emphasized gene discovery in epilepsy because it seems that even the most common forms such as febrile convulsions have a heritable component. At the same time we seek to promote better treatments, and even a cure, for the large number of people with "intractable" epilepsy. Many of these individuals are children, whose lives are disrupted by inadequately controlled seizures. Later this spring we will sponsor a White House-initiated conference, "Curing Epilepsy: Focus on the Future."

Halting the Process of Neurodegneration
As requested by the Appropriations Committees, NINDS is working on the first phase of an effort to develop a comprehensive research agenda for Parkinson's disease. We were joined in this effort by NIH Institutes and Centers with significant programs in Parkinson's disease, by patient advocacy groups, and by distinguished intramural and extramural scientists. We are confident that the proposed research agenda will advance the fight against Parkinson's disease and point the way for similar progress in other neurodegenerative disorders.

Neurodegeneration is more widespread than previously thought. In addition to classical adult neurodegenerative disorders such as Alzheimer's, Parkinson's, Huntington's and Lou Gehrig's diseases, neurodegenerative processes are at work in a number of serious disorders of childhood. Neurodegeneration also complicates conditions as disparate as stroke, spinal cord injury, epilepsy, multiple sclerosis, and depression. A cell death program, named apoptosis, appears to be a "final common pathway" in the process of neurodegeneration. Encouraging evidence indicates that inhibition of this pathway may be a useful therapeutic strategy, regardless of the initial causes of the degeneration.

We must not lose sight of our goal of cognitive and emotional health throughout life. The study of disease is teaching us that decline in cognitive and emotional health is not an inevitable consequence of aging. For many, perhaps most of us, a healthy brain is as realistic a goal as is a healthy heart. But we cannot achieve our goal without a much better understanding of disease, particularly the risk factors and early changes that point to possible preventive or corrective measures. A new patient registry for Parkinson's disease, to be followed by a larger population-based study, will point the way to further study of neurodegenerative diseases at every stage of life. Through collaboration with other Institutes, we will expand these studies to include cognitive and emotional disorders and to define and promote cognitive and emotional health across the life span.

Repairing the Injured Nervous System
Modern neuroscience is rewriting the textbooks that tell us that nerve cells cannot recover from deadly injury. Research on a number of fronts has produced tantalizing evidence that manipulating the cells' environment-by adding factors that promote growth or interrupting processes that disrupt it-will eventually redefine the future for those who have lost function due to injury. A recent initiative is seeking additional research on interneuronal circuits to restore lost function. As in so many other areas of neuroscience, the ability to manipulate and implant stem cells from a variety of sources is particularly promising for both acute and chronic injury. Complementing these efforts are advances in our ability to design neural prostheses- devices that connect with the patient's own nerves and muscles to restore or augment function.

Reducing Health Disparities
As we rejoice in the progress of modern medicine, we must not neglect those who, by virtue of biology or circumstance, bear a disproportionate share of the burden of disease. NINDS enthusiastically shares the commitment of NIH to reducing health disparities, and we will continue our leadership in this area. Stroke is a major health problem for the entire population but one that disproportionately affects minority citizens, particularly African-Americans. We support a broad program directed at the impact of stroke on minority populations, ranging from epidemiological and descriptive studies of disease patterns to specific therapies and educational approaches. The neurological complications of diabetes, another common disorder that particularly affects minority groups, is a major focus of interest.

Progress against health disparities also depends on building a diverse scientific workforce-a strategy that makes sense in general but is particularly important in working with minority populations. NINDS has a long history of leadership on this front. More recently, with support from the Office of Research on Minority Health, we initiated the first prototype of a Specialized Neuroscience Research Program (SNRP) at the Morehouse School of Medicine in Atlanta. A unique feature of the program is the establishment of collaborations and professional networks between investigators at minority institutions and those from more research intensive institutions and community-based organizations. Based on excellent results from the pilot program at Morehouse, and recognizing the work still to be done, the NINDS, in collaboration with NCRR, is now supporting additional SNRPs. This year we will expand the program to include a focus on HIV/AIDS, a particular problem in the nervous system, where the virus can cause dementia and neuropathy even when other manifestations of disease are well controlled.

Working to Fight Brain Disease
Neuroscience is recognized as one of a few great unifying themes in modern science. Nowhere is this more evident than at NIH, where almost every Institute and Center is involved to some extent in brain research. Here we have a unique opportunity to break down what is increasingly recognized as an artificial barrier between mind and brain, between neurology and psychiatry. Our goal is to develop a model for collaborative neuroscience with an emphasis on translational research. The National Neuroscience Research Center, for which start-up funds are requested in the Buildings and Facilities budget, will provide an environment that will promote modern neuroscience in the form of collaboration, communication, and shared resources. It will build on the impressive progress already made by intramural science leaders and on the example being set by the National Vaccine Research Program.

The emphasis on collaboration will, in my view, stand out as the distinguishing feature of NIH in our time. NINDS is actively working with one or more Institutes and Centers on diseases including autism, Duchenne and facioscapulohumeral dystrophy, and neurofibromatosis. Our efforts against neurodegenerative disease include collaborations with the National Institute of Aging on clinical trials for Alzheimer's disease and with the National Institute of Environmental Health Sciences on Parkinson's disease, as well as plans for an innovative public-private partnership to foster future research. Our collaboration with the National Cancer Institute to map the genes involved in a deadly form of brain tumor has blossomed into the formation of a joint Progress Review Group for brain tumor research, building on a planning technique that has been highly successful for research on other forms of cancer. In stroke, we have joined forces with the National Heart, Lung, and Blood Institute and with Suburban Hospital in Bethesda to improve rapid diagnosis and treatment of both stroke and heart disease. We continue to work with the Brain Attack Coalition to raise public and professional awareness of stroke as a preventable and treatable disease.

The NIH budget request includes the performance information required by the Government Performance and Results Act (GPRA) of 1993. Prominent in the performance data is NIH's first performance report, which compares our FY 1999 results to the goals in our FY 1999 performance plan. As our performance measures mature and performance trends emerge, the GPRA data will serve as indicators to support the identification of strategies and objectives to continuously improve programs across the NIH and the Department. For NINDS, this effort will be augmented by our strategic planning process, which provides an ongoing forum for assessing progress and setting priorities, and by our strong commitment to efficient and effective management of our resources.

Mr. Chairman, this concludes my prepared statement. I would be happy to answer questions you or the other Members may have.

Last Modified December 29, 2010