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Fiscal Year 2002 Budget Request

Senate Subcommittee on Labor-HHS-Education Appropriations
Statement by Audrey S. Penn, M.D. Acting Director, National Institute of Neurological Disorders and Stroke
Senate Date: May 23, 2001

Mr. Chairman and Members of the Committee:

I am Audrey Penn, Acting Director of the National Institute of Neurological Disorders and Stroke. I am pleased to present the President's budget request for NINDS for FY 2002, a sum of $1,316,448,000, which reflects an increase of $139,428,000 over the comparable Fiscal Year 2001 appropriation. 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 second annual performance report which compares our FY 2000 results to the goals in our FY 2000 performance plan. As performance trends on research outcomes emerge, the GPRA data will help NIH to identify strategies and objectives to continuously improve its programs.

The mission of NINDS is to reduce the burden of neurological disorders. Today I will speak briefly about that burden. I will also say a few words about the progress so far in treating these diseases and the remarkable opportunities presented by recent scientific advances. However, I will spend most of my limited time telling you what NINDS is doing to ensure that science is translated as quickly as possible into help for people with neurological disorders.

The Burden of Neurological Disorders

Disorders that affect the brain, spinal cord, nerves of the body, muscles and their control impose an enormous toll on society. Neurological disorders can compromise the complex thinking and emotions that make us human, the routine perception and movement that we take for granted, and even the control of bodily systems that are normally beneath our conscious awareness. Trauma, infections, toxic exposure, birth defects, degenerative diseases, tumors, gene mutations, systemic illness, vascular events, nutritional deficiencies, and adverse effects of essential treatments for diseases like AIDS and cancer can all disrupt the functions of the nervous system.

We often think first of neurological disorders that afflict older Americans- stroke, Alzheimer's, Parkinson's. But problems like multiple sclerosis, brain and spinal trauma usually strike young adults, and the list of childhood disorders is enormous-autism, cerebral palsy, Duchenne muscular dystrophy, Batten disease, Canavan disease, to name just a few. Disorders like epilepsy and brain tumors, which can strike at any age, also occur frequently.

Some neurological disorders are very common. NINDS and American Heart Association studies show that more than 700,000 Americans suffer strokes each year. The Centers for Disease Control and Prevention estimates that head trauma kills more than 50,000 people each year and more than five million survivors suffer disabilities. Physicians tell us that pain is the most common symptom that brings people to a doctor. A recent journal from the American Academy of Neurology, for example, suggests that migraine headaches affect about twenty percent of women. Diabetic neuropathy commonly accompanies diabetes. Epilepsy, autism, cerebral palsy, dystonia, the neurological aspects of AIDS and several other disorders also affect many people.

Collectively the hundreds of rare disorders of the nervous system also affect many people and their families. The history of medicine teaches us that studying rare diseases often has wide repercussions. Creutzfeldt-Jakob disease (CJD) is a terrible disorder that strikes about one in a million people. The long tradition of NINDS research on this formerly obscure disease is now coming to the forefront because of the public health concerns raised by the related bovine spongiform encephalopathy (BSE or "mad cow disease"). In the last year NINDS initiated a contract program to develop tests needed for confronting the public health and economic threats from BSE. Rare disorders often provide clues to more common diseases, and CJD is a good example here too. Abnormal aggregation of proteins called prions are at the crux of CJD. Abnormal clumps of other proteins have also been implicated in common diseases such as Parkinson's, Huntington's, and Alzheimer's.

Progress and Promise in Neurological Diseases

Perhaps because the brain is so complex and inaccessible, neurological disorders have always been among the most difficult to treat of all medical problems, but we are making progress. The American Heart Association estimates that the death rate from stroke went down by 14% from 1987 to 1997. NIH is continuing prevention trials that have contributed to that decline. In the 1990's NINDS clinical trials demonstrated the first acute treatment that improves outcome from stroke, the drug t-PA, and the first emergency drug treatment that can reduce the disability from spinal cord injury, high dose methyprednisolone. Neurosurgeons have developed guidelines that can improve outcome from head trauma. The first treatments that reduce symptoms and even slow progression of multiple sclerosis have emerged from studies of the nervous and immune systems. Several new drugs for epilepsy are now available, partly through efforts of the NINDS drug development programs. New surgical treatments, such as chronic deep brain stimulation, show promise for Parkinson's and other disorders. We have new genetic tests that help diagnose inherited neurological disorders. Clearly we are making progress, and much of that progress rests on the stream of advances from basic neuroscientists which is continuing. But each example represents only the first steps toward adequate treatments and prevention. We have a long way to go.

What is most encouraging is the range of new therapeutic strategies on the horizon. It may seem peculiar, but one important step in learning how to prevent or cure a disease is to first learn how to cause it. The striking progress in understanding the nervous system and its diseases at the level of genes, proteins, cells and brain circuits, is bringing us long sought after animal models of human disorders. Animal models enable scientists to follow the course of disease progression, refine understanding of causes, and develop therapies. Discovery of the genes responsible for inherited disorders such as Batten disease, ataxias, spinal muscular atrophy, and muscular dystrophy often leads to animal models of these disorders. Finding the genes responsible for uncommon inherited forms of Alzheimer's, Parkinson's and amyotrophic lateral sclerosis (ALS) has led to animal models that will foster progress against the more common non-inherited versions of these disorders. Genes are not the only route to developing animal models. This year brought new models for neurodegeneration in Parkinson's disease through use of a pesticide, rotenone, as well as through manipulation of genes; surgical techniques have always been important for developing animal models of stroke and trauma; and immunological approaches are important in diseases such as multiple sclerosis.

Using animal models, researchers are exploring the potential of cell transplantation, gene transfer, natural biochemicals, electrical stimulation, and new approaches to drug therapy for treating neurological disorders. Researchers using gene transfer vectors to deliver the natural neurotrophic (growth and survival) chemical GDNF were able to counter some Parkinson's-like effects in animals. Gene transfer also has shown promise in mice with the same gene defect as boys with Duchenne muscular dystrophy, and strategies to repair defective genes also now appear plausible for this disease. Cell transplantation in animals has helped repair damage from spinal cord injury, restore the myelin insulation of nerve fibers that is lost in multiple sclerosis, provide missing enzymes in inherited disorders like Tay-Sachs disease, and replenish the chemical dopamine in Parkinson's disease. Study of the steps in "cell suicide" that occurs as the simple nervous system of the worm develops led to the recognition that similar cell death mechanisms play out in several neurological disorders. Blocking steps in this cell death program has shown benefits in animal models of stroke, trauma, Huntington's disease and ALS. This is only a sampling, but shows that not far over the horizon are possibilities for treating many neurological disorders.

What is NINDS Doing to Move Toward Cures?

The genius of the NIH system is its power to engage the collective wisdom and ingenuity of the nation's scientific community. We must continue to nourish those ongoing efforts and position ourselves prudently to continue to support that base upon which all our efforts rest. At the same time, the scientific progress compels us to target efforts toward translating the scientific potential into real help for people as quickly as possible, and the recent funding increases empower us to do so. The key is again to rely upon the distributed insight of the medical and scientific community.

NINDS has a multi-tiered planning process to harness that collective wisdom to meet our mission. The planning process brings together scientists, physicians, the advocacy community, industry, and NIH staff in several complementary ways. We began, about two years ago, by convening more than 100 leaders from the scientific community, together with patient-advocates and NINDS staff, to assess needs, opportunities and priorities in several cross-cutting areas, each relevant to progress against many disorders. Seven panels focused on Neurogenetics; Neurodegeneration; Channels, Synapses, and Circuits; Cognition and Behavior; Neurodevelopment; Plasticity and Repair; the Neural Environment; and Experimental Therapeutics and Clinical Trials. NINDS posted draft panel reports on the internet and solicited comments from more than 250 patient advocacy groups and professional scientific organizations. This input helped shape the NINDS strategic plan "Neuroscience at the New Millenium" which serves as a foundation for all our planning efforts.

Building on the strategic goals, we have begun a series of disease specific planning efforts, beginning with Parkinson's disease. A January 2000 workshop brought together intramural, extramural, and industry scientists, Parkinson's disease advocates, and ethicists, forming the basis for the "NIH Parkinson's Disease Research Agenda," submitted to Congress in March of last year. NIH is vigorously implementing the Agenda. We held several workshops focused on specific aspects of Parkinson's disease research, such as drug therapies, gene therapies, cognitive and emotional aspects, and environmental influences. Including solicitations issued shortly prior to the Agenda or nearing release, NIH has developed more than a dozen requests for grants or contracts that target specific Agenda priorities. Targets of opportunity include deep brain stimulation, clinical trials for neuroprotective drugs, and proteins implicated in the disease. We have supplemented existing grants to expedite work on high throughput drug screening, bringing new investigators to the field, and genetics of Parkinson's in minority ethnic groups. Working groups or consortia have formed in critical areas, such as deep brain stimulation. NINDS is also continuing to support the eleven Udall Parkinson's disease research centers. The Institute will soon launch a website that will set the standard for informing the public about progress in implementing a disease specific research plan, and also serve as a resource for researchers and caregivers.

Several other disease specific planning efforts are also underway. As a joint undertaking NCI and NINDS brought together a Progress Review Group on Brain Tumors. More than 100 experts in several scientific and medical disciplines with a bearing on brain tumors presented assessments of current understanding and future needs in 14 critical areas. NINDS and NCI are working to implement these recommendations. A Progress Review Group in Stroke is following a similar process. Last spring's landmark conference "Curing Epilepsy: Focus on the Future" launched efforts that developed "benchmarks" for epilepsy research which are the first step towards a research agenda. This spring the Institute of Medicine of the National Academy of Sciences released an assessment of current status of research on multiple sclerosis with recommendations for future research. This effort, which included NIH researchers, will inform future efforts on this disease. Disease specific planning efforts dovetail with the NINDS strategic plan, revealing elements in common for many diseases and those unique to each disorder.

Health disparities has also been a focus of specific planning efforts at NINDS. Since FY99 NINDS, working together with NCRR, the ORMH and other Institutes, has expanded its original center at Morehouse School of Medicine to eight specialized neuroscience research programs at minority institutions and a network of research consortia at 28 leading neuroscience research programs. NINDS is expanding activities of this program with a parallel development of other research as an integral part of the Institute plan for addressing health disparities.

Another aspect of NINDS planning efforts is an active agenda of scientific workshops, often held in cooperation with private groups and with other components of NIH, such as the Office of Rare Diseases. NINDS holds about 40 of these meetings each year. Some focus on specific therapeutic strategies or technologies, such as neural prostheses, gene therapy, optical imaging, computational neuroscience and high throughput drug screening. Others target specific diseases. Recent workshops focused on Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, hereditary spastic paraplegias, spinal cord injury, ALS and spinal muscular atrophy, channelopathies, neurofibromatosis, and pediatric stroke. At these workshops, in addition to the scientific discussions, NINDS solicits advice on how to eliminate bottlenecks and encourage progress.

I could describe many other planned and ongoing activities, and certainly more exciting science, but I want to conclude with a simple message. Because NINDS has an "acting" director does not mean the Institute will be less active until a permanent director is appointed. Given the burden of neurological disorders, the scientific opportunities, and the favorable funding environment, it would be unconscionable for the Institute to become passive. I assure you we are doing everything we can to move aggressively toward better ways to treat and prevent neurological disorders.

Thank you. I would be happy to answer questions.

Last Modified February 3, 2011