Fiscal Year 2015 Budget Request

Senate Subcommittee on Labor-HHS-Education Appropriations

Statement by Story C. Landis, Ph.D.

Director, National Institute of Neurological Disorders and Stroke

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) 2015 NINDS budget of $1,608,461,000 includes an increase of $22,664,000 over the comparable FY2014 level of $1,585,797,000.  NINDS supports research to reduce the burden of neurological disorders, from basic studies of the normal brain through clinical trials of prevention and treatment interventions.  Today, I will make four points:  1) the burden of neurological disorders is enormous; 2) past NINDS research has paid off; 3) opportunities for future progress are extraordinary; and 4) we have well informed plans to exploit these opportunities..

BURDEN OF NEUROLOGICAL DISORDERS

Nearly 800,000 Americans experience a stroke each year, and 15 to 30 percent of the 6.8 million stroke survivors alive today suffer permanent disability^[1]. Traumatic brain injury (TBI) is the leading cause of death and disability in children and young adults, common among the elderly, and a major concern for the military and veterans.  In the United States, 2.5 million people receive emergency care for a TBI each year, and millions more suffer mild TBI (concussions).  Epilepsy affects 2.3 million Americans, including 1 in 26 people at some time in their lives.  Alzheimer’s disease is receiving increasing attention, but most people are less aware that frontotemporal dementia (FTD) is the most common dementia in people under age 60, and vascular dementia, which affects blood vessels in the brain, is the second most common dementia overall and is so closely intertwined with Alzheimer’s disease that most dementia patients have a combination of the two.  Parkinson’s disease, spinal cord injury, cerebral palsy, multiple sclerosis, and hundreds of rare diseases that affect children and adults add to the immeasurable human and economic burden.

PROGRESS FOR PATIENTS AND FAMILIES

NINDS research drives progress directly, and indirectly catalyzes private sector advances.  NINDS studies on risk factors and prevention contributed to a decline in the age-adjusted stroke death rate by 35.8 percent from 2000 to 2010; the actual number of stroke deaths fell 22.8 percent. ^[2]  NINDS research developed the only approved emergency drug therapy that restores blood flow to the brain following stroke, increasing likelihood of recovery with little or no disability by 30 percent.  Research has also demonstrated, defying conventional wisdom, a wider window of opportunity for stroke rehabilitation—even patients who start rehabilitation as late as six months after a stroke can improve, and patients can continue to improve one year after a stroke.  For people with epilepsy, an implantable device approved this year senses impending seizures and delivers electrical pulses to stop them.  Long-term NINDS research provided the essential foundation for private sector development of this device.  Similarly, NINDS research directly and indirectly contributed to deep brain stimulation (DBS) therapies now in use for Parkinson’s, essential tremor, and dystonia and under clinical testing for many other disorders, and to development of drugs for multiple sclerosis—10 are now on the market, including the first oral drugs.  Overall, the private sector has nearly 450 medicines in development for neurological disorders, which would not be possible without the foundation of NIH research^[3]. 

EXTRAORDINARY OPPORTUNITIES

Science and technology are opening unprecedented opportunities for progress against neurological disorders.  Studies on the normal brain build the foundation.  Notable recent advances, for example, revealed how the brain clears out debris during sleep, how molecular structures called ion channels control electrical activity, and the first human “connectome” maps, providing astonishing views of the basic wiring diagram of living, thinking human brains.  Advances in stem cell biology now enable researchers to reproduce in cell culture key steps in amyotrophic lateral sclerosis (ALS) and other disorders using brain cells derived from patients’ own skin cells.  Basic science has led to new insights that explain how chronic pain is wired in the brain, what happens in the brain following a concussion, and how cell-to-cell propagation of abnormally folded proteins could drive progression of Parkinson’s, Alzheimer’s, and other neurodegenerative disorders.  New gene sequencing methods and high throughput gene silencing technologies have accelerated the discovery of genes that cause epilepsy and revealed potential new drug targets for Parkinson’s disease.  In a few dramatic cases, gene discoveries have led directly to treatments that help patients with rare disorders, including subtypes of dystonia and childhood neurodegenerative disease, but more often painstaking translational research is required to advance genetic and other discoveries toward therapies.  Among the many examples, promising reports in laboratory animals this year demonstrated a drug therapy that prevented the development of epilepsy, cell transplants that controlled seizures, natural growth factor rescue of neonatal brain injury, therapies that improved cognition in Down syndrome, and a hand neuroprosthesis that restored touch sensation as well as movement.

PROGRAMS AND PRIORITIES

NINDS relies heavily upon the wisdom and ingenuity of researchers throughout the United States to propose and evaluate the best scientific opportunities.  Complementing investigator-initiated programs, NINDS initiatives target unmet opportunities or public health needs.  Institute priorities reflect strategic and disease-specific planning that engages the scientific community and the public, and rigorous evaluation of programs, closing those that have met their goals or are no longer appropriate for today’s science.  Recent plans focused on stroke, epilepsy, Parkinson’s disease, and Alzheimer’s Disease-Related Dementias.  Among recent initiatives:

• the Stroke Trials Network will determine more quickly and at less cost what treatment, prevention, and rehabilitation strategies work best.
• new Epilepsy Centers without Walls will target Sudden Unexplained Death in Epilepsy (SUDEP) and disease modification or prevention.
• the Parkinson’s Disease Biomarkers Program is developing assessment tools that will overcome roadblocks to more effective clinical trials.
• the International TBI Research Initiative, coordinated with the European Union and the Canadian Institute of Health Research, will answer questions on care and classification of TBI that have confounded development of interventions.
• two major cooperative studies are investigating the long-term changes in the brain years after a single TBI or multiple concussions, coordinated via the Foundation for NIH’s Sports and Health Research Program, which was established with a donation from the National Football League.
• the NeuroBioBank, NINDS Human Genetics Repository, Federal Interagency TBI Research database, Common Data Elements Program, and an epilepsy clinical genetics data repository are examples of new and continuing resource initiatives that empower individual investigators and promote data sharing.

Finally, and most ambitiously, the President’s Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative will dramatically improve tools to understand heretofore unapproachable questions about how networks, or circuits, of brain cells enable us to perceive, think, and act.  As for basic research generally, there are excellent reasons for confidence that this basic research Initiative will ultimately advance progress against disease.  Autism, dystonia, and epilepsy, for example, are fundamentally disorders of brain circuitry, and stroke, Parkinson’s, and Alzheimer’s disease disrupt brain circuits as nerve cells die.  Even with our limited understanding of brain circuits and imprecise technologies for altering them, interventions that compensate for malfunctioning brain circuits already produce remarkable results.  For example, deep brain stimulation reverses symptoms for many people with Parkinson’s disease and dystonia, and paralyzed people have controlled a robotic arm by signals directly monitored from their brains’ movement control circuits.  It is perhaps obvious that better understanding of brain circuits and tools to influence their activity would greatly improve these interventions, but history teaches that the most important payoffs of the BRAIN Initiative, as for basic research generally, may be entirely unforeseen.

Story C. Landis, Ph.D.

Director, National Institute of Neurological Disorders and Stroke

Dr. Story C. Landis began her appointment as the Director of the National Institute of Neurological Disorders and Stroke (NINDS) on September 1, 2003. A native of New England, Dr. Landis was awarded her B.A. degree in Biology, with highest honors, from Wellesley College (1967), and her M.A. (1970) and Ph.D. (1973) degrees from Harvard University. After postdoctoral work at Harvard University studying transmitter plasticity in sympathetic neurons, she served on the faculty of the Harvard Medical School’s Department of Neurobiology.

In 1985, Dr. Landis joined the faculty of the Case Western Reserve University School (CWRU) of Medicine in Cleveland, Ohio, where she held many academic positions, including Professor and Director of the Center on Neurosciences, and Professor and Chairman of the Department of Neurosciences, a department that she was instrumental in establishing. Under her leadership, the CWRU Department of Neurosciences achieved worldwide acclaim and a reputation for excellence. In 1995, Dr. Landis was appointed as the NINDS Scientific Director, and was responsible for the direction and re-engineering of the Institute’s intramural research program. Beginning in 1999, in conjunction with the leadership of the National Institute of Mental Health (NIMH), she spearheaded a movement to bring a sense of unity and common purpose to the numerous laboratories, in multiple NIH Institutes that conduct leading edge clinical and basic neuroscience research. This increased research cooperation and collaboration, and resulted in the construction of the new NIH Neuroscience Research Center, Phase II, on the NIH campus. Since early 2007, Dr. Landis has also been Chair of the NIH Stem Cell Task Force.

Throughout her research career, Dr. Landis has made many fundamental contributions to understanding the developmental interactions required for synapse formation, and has garnered many honors and awards. Dr. Landis is an elected Fellow of the American Academy of Arts and Sciences and the American Association for the Advancement of Science, and an elected member of the American Neurological Association. In 2002, she was elected President of the Society for Neuroscience, and served as President-elect until her appointment as the NINDS Director in September 2003. In 2009, Dr. Landis was elected to the Institute of Medicine of the National Academy of Sciences.