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Fiscal Year 2009 Budget Request before the House Subcommittee on Labor-HHS-Education Appropriations


House Subcommittee on Labor-HHS-Education Appropriations
Statement by Story C. Landis, Ph.D., Director, National Institute of Neurological Disorders and Stroke

House Date: March 5, 2008

Mr. Chairman and Members of the Committee:

I am pleased to present the President's budget request for the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH). The Fiscal Year (FY) 2009 budget of $1,545,397,000 includes an increase of $1,496,000 over the FY 2008 appropriated level of $1,543,901,000.

The mission of NINDS is to reduce the burden of neurological disorders by developing ways to prevent or to treat these diseases. Traumatic brain injury, stroke, chronic pain, dementias, epilepsy, and neuropathies are among the diseases that affect millions of Americans, and hundreds of less common neurological diseases together impose an enormous burden on people of all ages. To better accomplish our mission, NINDS is working with the research community, patient groups, and the public in a strategic planning process. Although the emphasis is on practical steps to enhance the productivity of researchers throughout the U.S., "blue sky" discussions are painting a vision for the future to which we should aspire.

Stroke

Stroke illustrates the progress, promise, and challenges that neurological disorders present. Because stroke is the third leading cause of death in the U.S. and a major cause of long term disability, even small improvements in prevention and care have a major impact on public health. According the latest figures from the Centers for Disease Control and Prevention, the age adjusted stroke death rate is continuing to decline, down 24.4% since 1999. Yet, the risk of stroke increases steeply with age, as does the risk for dementias, Parkinson's, and many other neurological disorders. So, speeding the pace of research is essential to avoid a crisis of neurological disability, as a higher proportion of our population is older in coming years.

No single advance drove the progress against stroke. Cumulative results of many studies, from the NIH and others, inform a personalized choice of the best stroke prevention plan for a given individual. Study results, for example, help predict who is most at risk for stroke, who will do best on which drug, and whether surgery to remove deposits of atherosclerosis from a cerebral artery or to repair an aneurysm leads to net benefit given the risk of the procedure for a particular patient. This year researchers developed a computational tool to optimize treatment for people who have transient ischemic attacks, a "temporary stroke" which warns that a major stroke may be imminent; other investigators found preliminary evidence that people who take a certain combination of three preventive therapies have less severe strokes when they occur; and the continuing REGARDS (REasons for Geographic And Racial Differences in Stroke) study found that "silent strokes," whose damage brain imaging reveals, are surprisingly common and could provide a warning of a more serious stroke. This is a small sampling of continuing efforts to improve stroke prevention, step by step.

A decade ago an NINDS clinical trial showed that the clot busting drug tPA was the first emergency treatment that could improve the outcome from stroke. The reduction in nursing home and rehabilitation costs far outweigh the cost of the therapy [Neurology 50:883-90 1998]. The success of tPA engaged the community in stroke education, stimulated the organization of more than 250 primary stroke centers nationally, and energized researchers to develop even better emergency care. Clinical trials are now testing whether ultrasound can improve the ability of tPA to break up clots in large brain arteries. Others test whether direct injection of tPA through a catheter threaded into the blocked brain artery or the use of clot retrieval devices help patients with clots too difficult to clear with tPA given by vein. The seven NINDS SPOTRIAS (Specialized Program of Translational Research in Acute Stroke) centers across the U.S. are developing new acute stroke treatments and expanding access to treatment through education and telemedicine links to community hospitals. The FAST-MAG trial is pushing stroke response even further by pioneering the ultra-rapid delivery of a therapy that may protect nerve cells by emergency personnel in the field prior to even reaching the hospital. All of this depends on educating the public to recognize strokes and act quickly, which is a focus of NINDS community outreach programs, working with the Brain Attack Coalition.

Brain Imaging and Biomarkers

Advances in brain imaging have transformed medical care for stroke and many other neurological disorders, and the pace of progress shows no sign of slowing. This year researchers reported on new imaging methods to non-invasively monitor neuroprogenitor cells, which give rise to new brain cells. Imaging tools are critical to enable study of how neuroprogenitor cells contribute to normal brain development, adult brain function, and repair. In other work, an NINDS intramural team developed an imaging technique that uses a high powered research MRI scanner to produce extraordinarily detailed images, revealing structures never before seen in the living human brain. Better imaging may allow future physicians to diagnose brain disease before irreversible brain damage has occurred, opening the door to prevent or slow disease onset-a goal that has so far eluded us. Imaging might reveal quickly and at substantially less cost whether experimental protective treatments are working, as well as yielding insights into the normal brain development and what causes diseases.

Imaging is one approach to the development of biomarkers, which are measurable indicators of disease risk, onset, progression, and response to therapy. The potential of biomarkers to guide early neuroprotective intervention and to expedite therapy development is a recurring theme of "blue sky" discussions of the future, and companies tell us validated biomarkers would leverage more investment from them to develop therapies for neurological disorders. NINDS supported researchers are developing biomarkers for neurological disorders using imaging, genetic technologies, proteomics, and electronic devices. We cannot predict when we might have a blood test for stroke or an early read-out of neurodegeneration, but biomarkers promise to revolutionize therapy development and preventive care in the future.

Genes

For some diseases, one type of biomarker has arrived-genes. Twenty years ago we had identified the gene defects responsible for a few neurogenetic disorders. Now, we know hundreds of them. For many families, diagnostic testing based on gene findings eliminates "diagnostic odysseys" that cost thousands of dollars and years of uncertainty. The identification of gene defects responsible for diseases such as Batten disease, Huntington's disease, muscular dystrophies, Rett syndrome, neurofibromatosis, and inherited subtypes of more common diseases like Parkinson's, has led to animal models that mimic the human diseases and to rational strategies for therapy via drugs, gene transfer, turning off harmful genes, and other approaches. For example, the NINDS SMA Project devised a drug development plan for spinal muscular atrophy based on insights from gene findings, then tested hundreds of potential drugs, and crafted drug candidates that are now in preclinical safety testing and may enter clinical trials within a year or two. For 2009, NINDS will renew initiatives that support translation of basic science insights for any neurological disease, whether from genes or other advances, to treatments ready for testing in people.

Most of the hundreds of diseases caused by defects in single genes are relatively uncommon, but combined variations in multiple genes, often operating in concert with environmental influences, contribute to many common neurological disorders and to differences in how people respond to treatment. Researchers have developed new tools to uncover these genetic influences. This year investigators identified the first two new genes linked to multiple sclerosis in more than 30 years, reinforcing the rationale for a therapy that is already in clinical trials. Because these studies often require participation of thousands of people, sharing data among researchers is essential. Last year, an NIH Parkinson's disease study set a standard in rapid data sharing. The NINDS Human Genetics Repository and other Institute efforts will continue that theme.

Epilepsy is another disease in which genes often play an important role. In 2000, NINDS and non-governmental organizations convened a conference that set the goal of "no seizures, no side effects" and brought research and patient communities together to generate the Epilepsy Benchmarks as guideposts toward that goal. A follow up conference in 2007 found encouraging progress and launched a successful effort to update the benchmarks. Catalyzed by the longstanding NINDS Anticonvulsant Screening Program, more drugs are available to treat epilepsy. Yet, finding the right drugs for each person is still requires trial and error, and for far too many people, the available drugs provide inadequate treatment. Finding rational ways to tailor therapy for each person, address intractable epilepsy, and prevent epilepsy from developing are ambitious goals for the future. Emerging strategies include implantable devices that analyze brain activity, detect incipient seizures, and stop them with precise electrical stimulation or drug delivery before they take hold. The meeting put a new emphasis on co-morbidities-stopping seizures is not enough without addressing the other problems that accompany epilepsy. Even in the absence of overt seizures, brain activity may be abnormal, with important consequences, especially for the developing brain.

A Healthy Brain

Similarly, promoting a healthy brain, rather than just preventing specific diseases, emerged as a theme of the NINDS blue sky discussions. Basic science has found that the brain generates new nerve cells and connections among brain cells change throughout life. "Brain plasticity" has already been harnessed to rehabilitate after stroke, brain injury, and other disorders-this is a focus of an NINDS initiative for the coming year. For the future, we must learn what each of us can do to foster a healthy brain throughout life, perhaps through even simple changes in diet and exercise.

Basic research on the normal brain must continue to be a major aspect of NINDS research because it provides the foundation for clinical advances by the private sector, as well as by NIH researchers. Drugs to treat epilepsy and pain, neuroprosthetic devices, and cell and gene therapies now available or in testing are recent examples. To cite one finding this year, basic scientists studying ion channels, which are proteins that control nerve cell electrical activity, have developed a method to silence chronic pain signals in rats without blocking normal sensations. If past is prelude to the future, basic research will transform medicine in ways we cannot anticipate.

Finally, progress depends on the quality of the research workforce. Not only is our population aging, but also our researchers. If NIH and private sector research are to continue their vigor, we must make research an attractive career for young people who are innovative and intelligent, dedicated and diverse.

Thank you, Mr. Chairman. I would be pleased answer questions from the Committee.

Last Modified March 14, 2012