A joint message from the NINDS Director and Deputy Director describing advances supported by NINDS and highlighting the new strategic framework for investing in the future of neuroscience research.
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Neurological disorders—that is, diseases of the brain, spinal cord, peripheral nerves, and neuromuscular system—inflict an enormous toll of lost life, disability, and suffering on people of all ages throughout the United States and the world [see Burden of Neurological Disorders below]. Furthermore, the need for progress is growing more urgent. Brain disorders are becoming more common with the aging of our population, the high prevalence of poorly treated chronic pain is a major factor in the opioid crisis, and health inequities persist despite encouraging overall progress against many diseases.
There are formidable barriers to progress. Foremost among them is our limited knowledge of how the normal brain, spinal cord and nerves develop, process information, become susceptible to aging processes, respond and recover after injury, and integrate into the workings of a host of other body systems. Despite the challenges, over the 70 years since Congress established the Institute, research has driven remarkable advances in fundamental knowledge of how the nervous system works and in the diagnosis, treatment, and prevention of neurological disorders and diseases. When the Institute was born in 1950, how neurons generated electrical impulses or communicated with one another was still a mystery. Scientists can now explain these phenomena with intricate detail, leading to many advances for people with neurological conditions. Then as now, millions suffered from an astonishing variety of perplexing, troublesome, and debilitating signs and symptoms. Neurologists and neurosurgeons provided a diagnosis based largely on clinical observation and too often had little to offer beyond a diagnosis and a grim prognosis. Now, we are on the cusp of mapping the human brain in its exquisite complexity and can monitor the electrical activity of a million neurons simultaneously in an awake, behaving mouse. Dramatic advances in brain imaging, the advent of genetic testing, and development of many diagnostic tools augment clinical observation, often eliminating the years-long diagnostic odysseys that families endured in the past. Moreover, research has brought effective options when before there were none for common diseases including multiple sclerosis, Parkinson’s disease, epilepsy, migraine, and acute stroke. Experimental treatments using combinations of devices, drugs, and rehabilitation, together with extraordinary persistence, have even enabled a few people paralyzed with spinal cord injuries to stand and take their first steps. Most recently a remarkable new approach for treating infantile spinal muscular atrophy, a fatal genetic disorder, has opened the door to an entirely new armamentarium to treat neurologic disorders. For stroke, breakthroughs in emergency treatments improve outcomes for many people, and, with major improvements in stroke prevention, the age adjusted stroke death rate has fallen by more than 70 percent since NINDS was established, according to the Centers for Disease Control and Prevention (CDC), saving millions of people from premature disability and death.
NINDS alone is not responsible for these advances. Many parts of the NIH, other government agencies, non-governmental organizations, and private sector companies in the United States and internationally have made substantial contributions to progress against neurological disorders. NINDS-funded research, however, has been pivotal, catalyzing progress across the public and private research ecosystem in many ways. Basic research on how the nervous system is organized and functions is funded predominantly by NIH and serves as the foundation from which almost all clinical advances spring. For some advances, NINDS led research from early laboratory studies in animals through definitive clinical trials. In other breakthroughs, the Institute supported proof-of-concept demonstrations in experimental animals that sufficiently “de-risked” innovative therapeutic development strategies to attract private sector investment. NINDS research also identifies risk factors that guide prevention through epidemiological studies, develops diagnostic tools and biomarkers for targeting of interventions to people who will benefit, creates and distributes research reagents and resources, and validates clinical outcome measures to evaluate the effectiveness of interventions. NINDS supports training of the nation’s neuroscience research workforce and disseminates timely and accurate information about neurological disorders to the research community, physicians, and the public. Most importantly, researchers in the public and private sector agree that NIH basic research on the nervous system in health and disease is the wellspring of progress.
Although we have come a long way, there is a long way to go. Drugs can treat the symptoms of Parkinson’s disease, but do not slow the inexorable death of brain cells, nor has any treatment been proven to halt the progression of amyotrophic lateral sclerosis (ALS), Huntington’s disease, or many types of dementia. No existing therapies are effective for about a third of people with epilepsy, prevention of epilepsy is still elusive, and all anti-seizure drugs carry troublesome side effects. Although advances in critical care have improved survival from severe traumatic brain injury (TBI), dozens of clinical trials of treatments have failed to improve long-term outcomes for those who survive, nor do we fully understand the long-term consequences of mild TBI (concussions). The lack of effective non-addictive treatments for acute and chronic pain affects millions of people and is a major driver of the opioid crisis. After decades of progress, stroke rates are now increasing for some segments of our population, and substantial disparities persist. For hundreds of genetic disorders, many of which affect infants and children, we now know the gene mutations responsible, but have no disease modifying therapy. The imperative for progress is evident every day to people affected by neurological disorders, their families, and the physicians who treat them.
In addition to priorities evident today, the NINDS Strategic Plan must prepare the Institute for tomorrow’s unanticipated public health challenges and scientific opportunities. Past strategic planning could not, for example, have anticipated the pain-opioid crisis nor the impact of the COVID-19 pandemic. The Institute is intensively engaged with its partners across the NIH in many aspects of COVID-19 research. Looking forward, the potential long-term sequalae of infection will be a major focus in the coming years. The Institute will also apply positive lessons from COVID-19 research programs to other diseases, including, perhaps, answering new questions by leveraging existing clinical cohorts and using telehealth to expand access to underserved communities. NINDS recognizes the significant impact the COVID-19 pandemic has had on the research enterprise and protecting early career and diverse investigators who are essential for future progress, but vulnerable to disruption of their research, is a high priority for the Institute. NINDS also plays a leadership role in the Helping to End Addiction Long termSM (HEAL) Initiative to advance non-addictive treatments for pain. Partnering with the National Institute of Aging and with special funding from Congress, NINDS has intensified efforts to understand and treat causes of dementia due to Alzheimer’s disease, cerebrovascular disease, frontotemporal degeneration, Lewy body dementias, and mixed dementias. Congress also enabled the development of transformative new research tools with the Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative® through the 21st Century Cures Act to map, monitor and modulate brain circuits. The Institute’s emphasis on foundational basic research, readily adaptable preclinical and clinical applied research programs, and sustaining a vigorous and diverse research workforce are among the overarching strategies that prepare NINDS for unexpected challenges and opportunities. NINDS is now poised to leverage powerful new genetic technologies, advances in data science and artificial intelligence (AI), and innovative research, diagnostic, and therapy device technologies that were all beyond the horizon during the previous strategic planning.
This plan presents an overarching strategic framework to accelerate science that will result in improvements in quality of life for all people with neurological disorders, and ultimately to prevent or cure these diseases. The Institute’s guiding vision is a world that is free from the burden of neurological disorders. Although the need is great, and the challenges daunting, we firmly believe real progress will most assuredly come from the dedicated scientists that NINDS funds. NINDS’s role is to optimize the taxpayers’ investment in neuroscience to maximize the impact and accelerate the pace of discovery.
To watch how this future unfolds, I invite you to monitor our website at https://www.ninds.nih.gov/. We will update this website as the Institute takes specific actions to implement the broad goals and strategies described in this plan.
Walter J. Koroshetz, M.D.
Director, National Institute of Neurological Disorders and Stroke
Nina Schor, M.D., Ph.D.
Deputy Director, National Institute of Neurological Disorders and Stroke
By some calculations of premature death, disability, and suffering, neurological disorders account for a greater burden than any other group of diseases (Nature Neurology 15:371-2, 2019). These diseases affect people of all ages—infants and children, young adults, and the elderly—and include some of the most common of all afflictions. For example:
Collectively rare disorders also take an enormous toll. According to the NIH Office of Rare Diseases, of 7,000 rare disorders overall, about 40 percent are neurological disorders and 90 percent of rare childhood disorders have major neurological effects.
The mission of the National Institute of Neurological Disorders and Stroke (NINDS) is to seek fundamental knowledge about the brain and nervous system and to use that knowledge to reduce the burden of neurological disease. NINDS supports research that advances the diagnosis, prevention, and treatment of neurological disorders, that is, diseases of the brain, spinal cord, nerves, and neuromuscular system. Basic research to understand the nervous system in health and disease, which provides the foundation for public and private sector progress, is at the core of this mission.
NINDS is one of 27 Institutes and Centers that make up the National Institutes of Health (NIH). The Institute has been a leader in the world of neuroscience for 70 years. Although the name of the Institute and the scope of its responsibilities have changed over time [see NINDS Legislative History box below], NINDS has maintained its fundamental principles and core strategies since its inception.
The Institute’s Extramural Research Program supports research at universities, medical centers, research institutions, and small businesses throughout the United States. The Extramural Research Program strongly emphasizes investigator-initiated research with rigorous peer review because of its long track record of driving progress. Augmenting investigator-initiated research, the Institute solicits research targeted to unmet public health needs, extraordinary scientific opportunities, and provision of resources that catalyze progress.
In fiscal year 2019, the NINDS budget of $2.274 billion supported 3,735 research grants and 657 research training grants across the United States. Research Project Grants, primarily investigator initiated, make up approximately 72 percent of the budget.
The NINDS Intramural Research Program at the NIH campus in Bethesda, Maryland, has unique capabilities that complement extramural research. These include the John Edward Porter Neuroscience Research Center, which integrates the extensive NIH neuroscience research community across Institutes, and the NIH Clinical Center, the largest hospital entirely dedicated to research in the world. The Intramural Research Program is about 9 percent of the budget, supporting 47 principal investigators. Throughout its history, in both extramural and intramural research, NINDS has emphasized basic research, while also supporting a full spectrum of innovative laboratory and clinical research to advance diagnosis, therapy, and prevention of neurological conditions. Support for training of graduate students and post-doctoral researchers and for early stage investigators has been, and continues to be, a long-standing priority for NINDS.
NINDS collaborates with all parts of the NIH. In addition to many joint activities with individual Institutes and Centers, NINDS is a leader in the NIH Blueprint for Neuroscience, which is a collaborative framework that includes the NIH Office of the Director and 14 Institutes and Centers that support research on the nervous system. In recent years, NINDS has taken on leadership for large-scale trans-NIH Initiatives to which Congress has directed funding. These include the Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative®, the Helping to End Addiction Long-termSM (HEAL) Initiative, and Alzheimer’s Disease and Alzheimer’s Disease Related Dementias (AD/ADRD) research. Beyond NIH, NINDS collaborates extensively with many other federal agencies, including the Departments of Defense and Veterans Affairs, the Centers for Disease Control and Prevention (CDC), the Food and Drug Administration (FDA), and the National Science Foundation. Similarly, NINDS interacts with the private sector via, for example, small business research (SBIR/STTR) programs, the Accelerating Medicines Partnership (AMP) for Parkinson’s disease, the NIH HEALSM Initiative, the Epilepsy Therapy Screening Program, and various clinical trials activities. These interactions are instrumental in advancing the NINDS mission.