Highlighting NINDS contributions to treatments for neurological disorders

Friday, February 9, 2018

The toll that neurological disorders exact on patients and their families is truly tragic. Yet, looking forward there is cause for real optimism for some conditions. I see the promise of an ever-growing range of new technologies and approaches applied to neuroscience research, and looking back I see that step by step, research does lead to successful new treatments.

NINDS now proudly unveils a new segment of our website dedicated to highlighting some of these successes, both past and recent. The site, “NINDS Contributions to Approved Therapies,” provides timelines and narratives documenting the development of treatments for neurological disorders for which NINDS played a critical role. The first two posts focus on tissue plasminogen activator (tPA) for acute ischemic stroke (approved by the FDA in 1996), and nusinersen for spinal muscular atrophy (approved in 2016). These two examples illustrate the many ways NINDS and the scientists we support contribute along the spectrum of therapy development – from basic science to understand disease mechanisms, to translational research to turn knowledge into new medicines and devices, and clinical trials to assess these therapies in people.   

The development of tPA revolutionized acute stroke care and is estimated to save $4 million for every 1,000 patients treated, due to improved outcomes. NINDS funded early studies that bolstered the rationale behind using tPA for acute ischemic stroke, led pivotal clinical trials that treated stroke patients with unprecedented speed, and even promoted public awareness of stroke and the benefits of timely treatment. The development of tPA also set the stage for further advances. Earlier this month, results from an NINDS-funded clinical trial showed that the time window for removing clots in acute ischemic stroke can be extended dramatically through the use of brain imaging to identify patients with salvageable brain tissue. As a more recent example, nusinersen is the first treatment approved for SMA, a leading genetic cause of death in infants and toddlers. NINDS and other NIH institutes supported research to uncover SMA’s genetic mechanisms, identify a treatment strategy, and facilitate later stage translational and clinical research. The history of nusinersen also highlights the central role of patients and non-profit organizations as partners in our shared mission to reduce the burden of neurological disorders.

I invite you to learn more about the development of these two treatments and to check back throughout the year as we add new success stories to this site.

Related Link:

NINDS Contributions to Approved Therapies

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NINDS: A Look Back at 2017

Wednesday, January 31, 2018

The beginning of a new year has a way of sneaking up on all of us, but we always gain helpful perspectives by stopping to reflect on the year that has passed. In 2017, NINDS made significant strides through funding awards, collaboration, new research initiatives, and leadership additions. We are grateful to our investigators, research subjects, and our partners representing those suffering from neurological disorders and stroke.

In 2017, NIH launched an Opioid Initiative to marshal our resources to address the public health crisis of opioid addiction, misuse and overdose. In June and July, the NIH hosted meetings, led by NIH Director Dr. Francis Collins, that brought together experts from government, industry, and academia. Emerging from those discussions, NIH is working with FDA and private sector experts to develop a public-private partnership to advance non-addictive, pharmacological treatments for pain and more effective ways to combat addiction and overdose deaths. NINDS leads the NIH Pain Consortium made up of Institutes and Centers all working to advance our knowledge of the biology of pain and find safe and effective treatments. In addition, NINDS led the development of the Federal Pain Research Strategy, a long-term strategic plan to advance the federal pain research agenda, which was released in September. Finally, in conjunction with the Office of the Assistant Secretary for Health in the Department of Health and Human Services, NIH hosted a public forum on implementation activities associated with the National Pain Strategy, a plan for improving pain management in the US (read the final report - pdf, 257 kb).

NINDS kicked off 2017 by announcing 30 inaugural recipients of the R35 Research Program Award (RPA). Unlike R01 awards, which provide support for up to five years for a specific set of experiments, the RPA funds an investigator’s laboratory for up to eight years, enabling the pursuit of long-range, innovative research. The awards cover a spectrum of topics, but tend to focus more on fundamental and disease-related basic research, such as elucidating the structure of the enigmatic fusion pore, and defining neural development processes that can be leveraged to identify genetic risk factors for neurological disorders.

In the fall of 2017, with guidance from a Trans-NIH Working Group, NIH invested over $7 million to launch a consortium of centers to conduct coordinated scientific research on Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS), a complex disease that has so far eluded understanding. The heterogeneous presentation of ME/CFS requires large-scale, trans-NIH efforts to provide an evidence base for effective therapy development. In addition to establishing the Consortium, the NIH awarded Administrative Supplements to expand ME/CFS research in current grants and initiated ME/CFS research at the NIH Clinical Center in Bethesda, Maryland, led by NINDS Clinical Director, Dr. Avi Nath.

Now in its fourth full year, the BRAIN Initiative® is paving a new course of fundamental neuroscience research. In 2017, the Initiative funded 110 new awards, bolstered by funds from the 21st Century Cures Act. These new awards support cross-disciplinary, team-based science and cutting-edge technology development to understand neural circuit function, as well the first set of awards for dedicated neuroethics research. As part of this investment, NIH made nine awards that together formed the BRAIN Initiative Cell Census Network, which will create a comprehensive 3D mouse brain cell atlas, generate reference brain cell atlases from postmortem healthy adult human and non-human primate samples, and build an integrative data center. To date, more than 275 publications have described new BRAIN Initiative-related advances and techniques for studying the brain in action. As the Initiative nears its halfway point, the NIH is planning to form an external working group to review progress to date and provide an updated scientific vision to guide the second half of the Initiative.

Congressional appropriations to the NIH in Fiscal Years 2016 and 2017 provided a generous boost in funding for Alzheimer's Disease (AD) and AD-related dementias (ADRD). As a lead NIH Institute for ADRD research, NINDS collaborates with the National Institute on Aging (NIA) to create new funding opportunity announcements (FOAs) and extend the payline for meritorious investigator-initiated projects on AD/ADRD. We urge the research community to join in our efforts to accelerate scientific progress toward reducing the enormous burden and cost of dementia. The National Advisory Neurological Disorders and Stroke Council (NANDSC), the Advisory Council to NINDS, approved several AD/ADRD research concepts to be further developed into new programs and initiatives, based on input from ADRD summits.

Separately, NINDS, Celgene, Verily, Pfizer, GlaxoSmithKline, Sanofi, the Michael J. Fox Foundation (MJFF), and the Foundation for NIH (FNIH) are launching the Accelerating Medicines Partnership for Parkinson’s Disease (AMP-PD). This new addition to the AMP program aims to identify and validate biomarkers and new therapeutic targets for PD, and it will leverage a treasure trove of data and resources supported over the past several years by NINDS and others.

As another example of collaboration, along with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), NINDS also released the 2017 Strategic Plan for Cerebral Palsy Research, which outlines a set of research priorities to advance our understanding and treatment of cerebral palsy, working together with our partners as we seek to make meaningful progress in cerebral palsy research.

Finally, and as a reminder of the promise of research supported by NINDS and others, 2017 brought new FDA-approved therapies for neurological disorders, including an enzyme replacement therapy for a form of Batten disease, the first treatments for tardive dyskinesia and chorea associated with Huntington’s disease, and new drugs for amyotrophic lateral sclerosis (ALS) and multiple sclerosis (MS). A number of promising new therapies, including several supported through NINDS translational research programs, have also entered clinical trials. Overall, NINDS extramural and intramural investigators reported more important advances across basic, translational, and clinical neuroscience than I can describe here, but I invite you to look back on a small selection of highlights below.

These efforts could not advance without leaders at the helm. In 2017, NINDS welcomed three individuals to leadership roles: Dr. Nina Schor as Deputy Director, Dr. Amir Tamiz as Director of Translational Research, and Dr. Clinton Wright as Director of Clinical Research. Moving into 2018, we look forward to appointing a new Scientific Director and Director of Neuroscience to the NINDS team. With these new leaders in place, we look forward to an even more productive 2018, as we continue our mission of seeking fundamental knowledge about the nervous system and using that knowledge to reduce the burden of neurological disease.

Selected 2017 NINDS Science Advances


  1. NINDS-funded research links midlife cardiovascular risk factors to dementia. With an aging population, dementia is a major health concern. A long-term study in over 15,000 participants found that middle aged Americans who have vascular risk factors, including diabetes, high blood pressure and smoking, have a greater chance of suffering from dementia later in life. The findings add to a growing body of evidence linking cardiovascular health to brain health and suggest that managing vascular risk factors in middle age may help prevent the development of dementia. See article. See NINDS Press Release. See more.
  2. Largest study to date documents chronic traumatic encephalopathy (CTE) in former American football players. CTE is a delayed neurodegenerative disorder that results from repetitive mild injury to the brain. Several individually small studies have described brain pathology consistent with CTE in postmortem brains from former athletes exposed to sports-related head trauma. This study provided alarming confirmation on a larger scale, with CTE found in 177 of 202 (87%) former American football players whose brains were donated for research, including 117 of 119 (98%) former professional players. See article.
  3. Silent seizures may contribute to cognitive decline in Alzheimer’s disease. An increased risk of seizures has been viewed as a late-stage phenomenon in Alzheimer’s disease (AD). In this study, researchers recorded brain activity in two patients with earlier-stage AD using electrodes inserted near the hippocampus, a region critical for memory that is affected in AD and commonly involved in epilepsy. The recordings revealed seizure activity without convulsions or other observable events, and these “silent” seizures occurred during sleep, when they could disrupt memory consolidation. While small, this study builds on prior research describing seizure-like activity in AD animal models and in patients, where it was associated with more rapid cognitive decline. Together, these studies raise intriguing questions about whether silent seizures contribute to AD progression and whether they may be a target for treatment. See article.


  1. Gene-editing therapies move from promise to reality. Antisense oligonucleotide (ASO) therapies employ sequences of nucleotides (the letters in the genetic code) designed to bind specific regions of a gene and modify its expression. This approach led to the FDA approval of an ASO therapy for spinal muscular atrophy (SMA) in late 2016. NINDS-funded investigator Don Cleveland, PhD, received a 2018 Breakthrough Prize, for his work to understand an inherited type of amyotrophic lateral sclerosis (ALS) and his role in developing ASO therapies now in clinical trials for ALS and Huntington’s disease. (See UCSD Press Release.) As additional examples, NINDS-funded researchers reported exciting results in animal models with an ASO for spinocerebellar ataxia (see article) that also affects disease mechanisms in ALS (see article and NINDS Press Release); and others are developing an ASO to reduce levels of tau proteins, which contribute to several neurodegenerative disorders (see article and NINDS Press Release).


  1. Noninvasive Deep Brain Stimulation via temporally interfering electric fields. Brain stimulation approaches are important in neuroscience research and for the treatment of disorders such as Parkinson’s disease, depression, and epilepsy. However, current methods for human brain stimulation present several limitations: invasive approaches use electrodes inserted into the brain, and non-invasive approaches fail to reach regions beyond the brain’s surface. Investigators supported by NINDS and others have described a new approach to non-invasive deep brain stimulation based on a concept known as beat frequency. When two oscillating electric fields coincide, they create a combined envelope that ‘beats’ at a frequency equal to the difference between the frequencies of the original fields. The investigators showed that they can can stimulate neurons in mice with this approach. Moreover, by manipulating the intensities and locations of applied fields, they could steer intersections with effective beat frequencies to different brain regions. The investigators hope further development will allow this approach to be tested in humans. Devising innovative new approaches to non-invasive, selective brain stimulation is also a goal of the NIH BRAIN initiative, and these results point to exciting progress on the horizon. See article.


  1. Reactive astrocytes can be deadly. Astrocytes are a type of non-neuronal glia cell in the central nervous system with important roles supporting neural and immune function. Brain injury and disease can cause astrocytes to enter a 'reactive' state, and researchers have debated the extent to which reactive astrocytes are helpful or harmful. In this study, NINDS-funded investigators describe a novel subtype of reactive astrocytes that lose supportive and survival-promoting functions and instead induce cell death. These newly characterized astrocytes were abundant in postmortem human brain samples from individuals with Alzheimer’s disease, Huntington’s disease, ALS, Parkinson's disease, and multiple sclerosis, suggesting their presence may contribute to neurodegenerative disease processes and opening new avenues in the search for neuroprotective interventions. The study’s last author, Ben A. Barres, Ph.D., sadly passed away at the end of 2017. His research transformed our understanding of glia cells, and his dedication to mentorship, equality, and diversity strengthened the neuroscience community in immeasurable ways.  See article.
  2. Novel mechanism in neuronal response to toxic stress. An NIH-funded research team has discovered a new process that helps neurons clear out toxic protein aggregates and organelles such as mitochondria that no longer function properly. Through studies in the round worm C. elegans, the researchers found that neurons extrude defective mitochondria and toxic clumps of protein by generating large vesicles they call exophers. Over time, exopher content appears to be degraded or engulfed by other cells. The researchers suggest that this process, when limited through aging or other factors, may contribute to neurodegenerative disease. See article.
  3. Possible cause discovered for mysterious pediatric epilepsy. Nodding syndrome is a devastating form of pediatric epilepsy found in areas of east Africa and characterized by head nodding, seizures, cognitive deterioration, and stunted growth. Many studies have associated Nodding syndrome with the parasitic worm Onchocerca volvulus, but whether and how the worm caused the disorder had remained a mystery. In this study, NINDS intramural investigator and Clinical Director Avindra Nath, M.D., and his colleagues compared serum samples from patients with Nodding syndrome and healthy controls from the same village in Uganda. In those with Nodding syndrome, they found antibodies to a protein normally expressed in the brain called leiomodin-1. The antibodies were toxic to neurons grown in the lab, and they cross-reacted with antigens from the O. volvulus worm. The results point to an autoimmune mechanism for the disease, whereby antibodies generated by the immune system to attack a harmful intruder inadvertently attack the body’s own proteins as well. This research may inform better ways to diagnose, treat, and prevent Nodding syndrome, and potentially other epilepsies with autoimmune causes. See article. See NINDS Press Release.
  4. Researchers identify gut chemosensors that couple to neural pathways. The lining of the gut is an important site for transmitting information about dietary, microbial, and inflammatory agents to the nervous system. Exactly how such signaling occurs has remained unclear, in part because cells suspected to play a major role (enterochromaffin (EC) cells) are sparse and hard for researchers to access. The authors of this study exploited a new technique to grow organoids in the lab, which allowed them to study EC cells directly. They found that EC cells express receptors that detect irritants, microbial metabolites, and stress-response hormones. EC activation by such agents triggers them to release the neurotransmitter serotonin, which in turn modulates sensory nerves through synaptic connections. The findings shed new light on how the gut conveys information to the nervous system, with implications for understanding bowel disorders and a wider range of processes influenced by the gut-brain axis. See article.
  5. Researchers connect brain blood vessel lesions to intestinal bacteria. Cerebral cavernous malformations (CCMs) are clusters of dilated, thin-walled blood vessels that can lead to seizures or stroke when blood leaks into surrounding brain tissue. Scientists studying the mechanisms that cause CCMs found an unexpected link to bacteria in the body. The researchers observed that mice with CCMs also had bacterial infections, and further study revealed that mice raised in a germ-free environment were protected from CCM development, as were mice treated with antibiotics to reset their internal bacteria. CCM formation was accelerated by inducing infection or activating a signaling pathway triggered by bacterial infection, and blocking this pathway prevented CCM formation. These findings may help explain the variability seen across people genetically predisposed to develop CCMs and could inform strategies for prevention. More broadly, they add to growing recognition of the many ways our microbiome – the community of bacteria residing in our bodies – contributes to health and disease. See article. See NINDS Press Release.

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Looking Forward to Serving as NINDS Deputy Director!

Thursday, January 18, 2018

By: Nina Schor, NINDS Deputy Director

Photo of Nina Schor, M.D., Ph.D.
Nina Schor, M.D., Ph.D., will be joining the NINDS as the Deputy Director. Photo courtesy Dr. Nina Schor

It is truly an honor and a pleasure, as I assume the position of Deputy Director of NINDS, to send my very first NIH web message to my new colleagues and friends! For me, joining the leadership team at NINDS is a bit like coming home. Over the past 11 years, I have been Chair of the Department of Pediatrics and Pediatrician-in-Chief at the University of Rochester. Like being a child neurologist, this role fit my passion for enriching the lives and well-being of children, but it often pulled me away from my neuroscience “family” and neuropharmacology roots. So, I am excited to be back in my topical home and to bring to NINDS all that I have learned through running a large department and children’s hospital, partnering with the local community, and supporting trainees, faculty, employees, and families in Rochester. What a privilege it will be to work to improve the lives of people with neurological disorders across a national stage!

I suspect that like many of my new colleagues, it was that potential to make a national impact that greatly fueled my interest in coming to NINDS. In my view, making that impact requires achieving three mission-critical goals.

First, we must ensure the robustness of investigator-initiated intramural and extramural neuroscience research programs in ways that foster creativity and intellectual rigor. Tomorrow’s progress in reducing the burden of neurological disorders depends upon the science we develop today. Unfettered, intellectual freedom in basic science research is essential to build knowledge and drive innovation, and strong bridges between the laboratory, the clinic, and the community are equally essential to translating the best new ideas into meaningful clinical advances and other applications. While knowledge without action may not be optimal, in medicine, action without knowledge and understanding is at best unwise and at worst, dangerous.

Second, we must consistently fuel the research pipeline with a diverse cadre of enthusiastic, promising, and innovative neuroscience students, trainees, and physician-scientists. For our patients and their families, this pipeline represents the promise of innovation in neurology and neuroscience, and – if we make it happen – it is key to their future.

Finally, we must communicate the role of neuroscience and a message of partnership and hope for understanding, cure, and prevention of disease to the public - it is their investment that makes our work possible.  I feel privileged to have witnessed and contributed to the vast evolution of neuroscience and neurology over the past several decades. In my career, I heard increasingly detailed descriptions that characterized more fully and sharply the mechanisms behind the syndromes we encounter in our clinics and hospitals. Recently, I and others have generated clinical applications that followed directly from these mechanisms. Our gross observations, microscopic depictions, electrophysiologic studies, and molecular discoveries are finally leading to strategies that connect back to patients and families to prevent and treat and, yes, even cure, diseases of the nervous system. This enormous success must not be our best-kept secret any more. No patient or family should leave our offices or our hospitals without knowing why our work is so important. No medical student should graduate without understanding the value of neuroscience and neurology research for their future patients. No legislator, financial manager, politician, or business person should go without knowing why this research is mission-critical for the health and well-being of their constituents today and for generations to come.

The time is now. The need is great. And the potential is limitless. Never before has the gap between biomedical research and clinical practice been so narrow. Never before have our creativity and collaborative spirit been so important as we understand previously cryptic mechanisms of neurological health and disease and forge health care solutions for people challenged by individual medical, psychological, and socioeconomic circumstances. NINDS is uniquely poised to ensure that the best of biomedical science is applied to make tomorrow’s state-of-the-art better than anything of which we dare to dream today. I am so very much looking forward to this journey with you!

Related links:

NINDS Names Dr. Nina Schor as Deputy Director

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Help us promote your stories of discovery

Wednesday, January 3, 2018

I’m a news junkie, and I love stories, especially those about science. You may not realize it, but every day as part of the NINDS research community, you are generating incredible news. There is so much amazing science being done in your labs, whether you are using state-of-the-art technology to image people’s brains or teasing apart the nervous system of a zebrafish. And although that may be routine for you, to the public that work is often fascinating. NINDS wants to help you share these stories.

You can help us do this by notifying your program directors of upcoming papers. NINDS’ Office of Communications and Public Liaison (OCPL) has a team of science writers who have backgrounds in neuroscience and are skilled in translating technical findings into accurate, plain language stories for the public in the form of press releases. To maximize the news impact and reach of your study, press releases should go out just before the study becomes available online. To make this happen, it is helpful to receive manuscripts as early as possible, ideally just after submission. The OCPL team has extensive experience coordinating with journals and understands the importance of adhering to their confidentiality rules.

NINDS news releases are picked up by the general media, and health and science reporters who adapt the scientific findings into articles for their unique audiences. In 2017, NINDS covered several stories that also appeared in popular outlets, including The New York Times, BBC World Service, NPR,  Reuters, The Atlantic, and Scientific American.

Many of you have media offices at your institutions, so you may already be somewhat familiar with this process. But, by including NINDS, we can help your exciting research findings reach an even larger audience. In this time of an ever-shifting digital landscape, news travels fast through a variety of channels and it can be hard to keep up. At NINDS, we use many strategies to get the word out about your research. We post the NINDS releases on our and the NIH website, which receives an average of 1.5 million page views per month. Information about NINDS-funded studies also gets posted to our Twitter accounts, which have thousands of followers, and to our Facebook pages as well as the Grantees in the News section of our website. And, sometimes, Dr. Collins, the NIH Director, includes them in his blog posts. By using a combination of traditional outreach strategies and current technology, while coordinating with your institution’s media office, we can get the news about your research in front of as many eyeballs as possible.

The public—the taxpayers who make NIH-funded research possible—want to know what their dollars are paying for. When thinking about topics with broad interest, I don’t know of anything with more universal appeal than the brain. Our healthy brains enable us to do extraordinary things and make us who we are as individuals. How it does that is the mystery that makes the brain so fascinating—and newsworthy.

So, help us to get the neuroscience conversation going — not just among your colleagues, but for everyone. Please remember to send recently submitted or accepted manuscripts to me, your program director or to the NINDS Press Team.  

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Tissue Plasminogen Activator for Acute Ischemic Stroke (Alteplase, Activase®)


A stroke occurs when the blood supply to brain tissue is blocked by a blood clot (ischemic stroke), or when a blood vessel in the brain ruptures (hemorrhagic stroke), causing brain cells to die and leading to functional impairments. Stroke is a leading cause of death and disability both globally and in the U.S., where approximately 800,000 people experience a stroke each year1.

Hand touching medical scans on board

Nusinersen (Spinraza™) – Spinal Muscular Atrophy (SMA)


Spinal muscular atrophy (SMA) refers to a group of inherited neurological disorders that begin in infancy or childhood and lead to the degeneration of spinal motor neurons, the neurons that control skeletal muscles. This degeneration results in weakness, muscle wasting, and in the most severe cases, paralysis and death before two years of age. SMA affects approximately 1 in 10,000 newborns and is a leading genetic cause of death in infants and toddlers. Nusinersen, marketed in the U.S. as Spinraza™ (Biogen) is the first therapy approved for the treatment of SMA.

Hand touching medical scans on board

Neuroscientists, Welcome to Washington!

Friday, November 10, 2017

This week, more than thirty thousand neuroscientists are coming to Washington, DC for the annual meeting of the Society for Neuroscience (SfN). The meeting is the largest scientific conference on brain and nervous system research, and I am looking forward to hearing about the latest science advances, speaking with NINDS grantees, catching up with colleagues and collaborators, and meeting investigators just entering the field. We are a growing field, fueled by heightened public interest, and I always leave the meeting feeling reenergized by the exciting science and individuals I meet.

At NINDS, we rely on this talented community to advance our mission; both researchers to drive the discoveries but also staff to lead and administer our programs. Growing a healthy neuroscience workforce is one of my top priorities. Among my focuses at SfN will be to share news about our new NINDS mentorship award, programs and policies at NINDS and NIH to help researchers balance family and personal needs with busy research careers, and to spread the word about important open positions within our own institute. For those attending the meeting, please come by booth 3404 to learn more.

As I’ve written about recently, NINDS is offering a new mentorship award, named after my predecessor, Dr. Story Landis. Nominations for the inaugural year of this award are due December 15 – please take a moment to consider nominating someone that has been an extraordinary mentor for you. This year, NINDS is seeking nominees for junior faculty members who have demonstrated dedication to training and ensuring that their trainees become exceptional scientists, I know how critical mentoring is, and we are hoping to reward and incentivize excellence in mentoring with the recognition and financial support that come with this award, which includes $100,000 in direct costs for an investigator to use to foster the career advancement for trainees. More information can be found here.

In concert with mentoring, we are working to enhance postdoctoral training by focusing the NINDS postdoctoral fellowship training (F32) on the beginning of the postdoctoral training period. By requiring an applicant to submit an application before the end of their first year in the new lab, and emphasizing the importance of an innovative, impactful project, and rigorous quantitative training, NINDS hopes to spur talented trainees to move successfully to the next phase of their careers more quickly. Indeed, our interest in bold new ideas is so strong that we hope that students will develop an idea with their future mentor before they start their postdoctoral training and apply while finishing up their predoctoral career. With the new NINDS F32, we actually don’t want preliminary data. We want great ideas to do important research. What NINDS does not want is to discourage researchers who have a child during this period from obtaining a fellowship  – extensions of the eligibility window are readily available for the F32 (and other training mechanisms, such as the K99/R00) for parental leave or other well-justified leave. Please reach out to your program director or the NINDS training director to learn more.

We also want the community to be aware that this FOA features new language for fellows who have children during their fellowship. Many early career investigators face a tough balancing act between work in the lab and starting families. Unfortunately, the evidence suggests that having children is a major reason for why young female scientists opt out the academic pipeline before obtaining their first academic research jobs. While addressing this challenge will ultimately require a sustained, multi-pronged approach, as a first step NINDS is encouraging Fellows who have a child during the period of their F32 award and serve as the primary caregiver to request a six-month paid extension of their fellowship. We are considering this a pilot program, and after evaluation, will consider expanding it to other training funding opportunities.

We believe these flexibilities will not only train fellows to be better scientists, but will enable fellows to balance research with new parental responsibility.

The NINDS model for the postdoctoral training fellowship has been adopted by the NIH BRAIN Initiative, which just released a new postdoctoral fellowship opportunity (F32) for fellows who have the potential to become productive investigators in research areas that will advance the goals of the BRAIN Initiative. A special focus in this initiative is encouraging scientists from other fields to collaborate with neuroscientists. We welcome scientists in the fields of statistics, physics, mathematics, engineering, and computer and information sciences to get involved in experimental neuroscience. As with the NINDS fellowship, we are emphasizing the importance of scientific skills and rigor in these applications. As I have stated before, a focus on robust, reproducible results is critical to our mission of understanding the brain and nervous system and reducing the burden of neurological disease. Further, with this fellowship, we are providing the opportunity to support postdoctoral research on the ethical implications of recent advancements in neurotechnology and brain science

Lastly, NINDS is hoping to utilize our robust neuroscience workforce to recruit a few motivated leaders to direct critical programs here in our Institute. We are currently searching for a Director for our newly-established Division of Neuroscience. This individual will lead the largest portfolio in our extramural research program and is critical to charting the future of a vibrant, growing neuroscience research community. From a trans-NIH perspective, we are also looking for an exceptional scientist to lead the NIH BRAIN Initiative, a complex program involving 10 NIH neuroscience Institutes and Center that is aimed at revolutionizing our understanding of the human brain. The NIH BRAIN Initiative Director will lead the innovative trans-NIH component of this collaborative, public-private research initiative and coordinate with the multiple federal and non-federal participants to ensure we collectively meet the initiatives bold, audacious goals. More information on these exciting, high profile positions can be found here.

To learn more about all of these opportunities, please come visit booth 3404 at the meeting or reach out to the NINDS program directors and staff who will be attending. I am excited to meet and interact with many of you and make the neuroscience field the best it can be. 

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Boosting Research on Alzheimer’s Disease and Related Dementias (AD/ADRD)

Monday, October 30, 2017

If you know anyone with dementia, you probably understand how devastating it is to those affected and their families. Alzheimer’s Disease (AD), the most common and widely-diagnosed neurodegenerative disorder, affects more than 5 million people in the United States. Other related forms of dementia also affect millions of Americans and commonly co-occur with typical AD, representing a significant and increasing burden on public health. Known as AD-related dementias (ADRD), these include Lewy body dementia (LBD), frontotemporal dementia (FTD), vascular cognitive impairment/dementia (VCI/D), and mixed dementias. Notably, cerebrovascular disease is exceedingly common in older populations who are diagnosed with AD and in persons with disabling age-related cognitive decline. 

Congressional appropriations to the NIH in Fiscal Years (FY) 2016 and 2017 provided a generous boost in funding for AD and ADRD research – an additional $350 million in FY2016 and an additional $400 million in FY2017. As a lead NIH Institute for research on ADRD, NINDS collaborates with the National Institute on Aging (NIA), the lead NIH Institute for AD research, to put these funds to work by creating new funding opportunity announcements (FOAs) and extending the payline for meritorious investigator-initiated projects on AD/ADRD. Going forward, NINDS and NIA will continue to partner in AD/ADRD research planning and implementation, and we urge the research community to join in our efforts to accelerate scientific progress toward reducing the enormous burden and cost of dementia.

The need for scientific effort to reduce the burden of Alzheimer’s Disease and Related Dementias

Currently, there are no disease-modifying treatments available for AD, LBD, FTD, vascular, and mixed dementias, and the lack of optimal clinical diagnostic tools for dementia makes it even more challenging to find effective treatments or guide patient care. We have previously noted the importance of controlling hypertension, the primary driver of cerebrovascular disease, for stroke prevention and healthy brain aging. A recent report from the National Academies of Sciences, Engineering, and Medicine, “Preventing Cognitive Decline and Dementia: A Way Forward,” concluded that the “evidence suggests that managing blood pressure for people with hypertension, particularly during midlife (ages 35 to 65 years), is supported by encouraging but inconclusive evidence for preventing, delaying, and slowing clinical Alzheimer’s-type dementia.” While promising, we still have much to learn about the interaction between vascular and AD pathology, how to leverage cardio- and cerebro-vascular health measures to prevent dementia, and moreover, how to prevent or halt other types of dementia.

Chart showing the change in NIH Research Funding for AD and ADRD.
Since 2011, the NIH budget for AD/ADRD research has more than doubled. The first largest increase occurred in FY2016, when NIH received an additional $350 million toward AD/ADRD research. (Based on data from the NIH RePORT Categorical Spending). In FY2017, NIH received an additional $400 million (not shown in graph)

As the U.S. population ages, we need to reduce the tremendous health and economic burdens that face individuals with dementia, their loved ones, caregivers, and society. Recognizing this urgent need, Congress passed and President Obama signed the National Alzheimer’s Project Act (NAPA) into law in 2011, as a coordinated national effort to fight AD/ADRD. Since then, the NIH budget for AD/ADRD research has more than doubled, including the significant increase in funds in FY2016 and FY2017. 

Extended Payline for AD/ADRD Investigator-initiated Research

Since FY2016, the NIA has utilized a higher pay line for AD/ADRD research to support investigator-initiated research grants, which it extends to NINDS and other Institutes and Centers for applications that meet this bar. This pay line is considerably higher than the pay line for other types of research in either NINDS or NIA. NINDS remains very interested in grant submissions focused on innovative ideas that could lead to prevention or effective treatment of AD/ADRD. 

Future Opportunities

NINDS is also actively developing concepts for new initiatives to address research priorities identified through periodic NIH-hosted ADRD Summits (ADRD 2013 & ADRD 2016), where researchers, clinicians, patients, caregivers, and advocates gather to assess scientific progress and generate research recommendations. Some of these prioritized scientific opportunities include improving understanding of disease mechanisms in ADRD, determining the presence and significance of co-morbid brain pathologies in individuals with AD/ADRD, and improving clinical detection and diagnoses of AD/ADRD. Last year, the National Advisory Neurological Disorders and Stroke Council (NANDSC), the Advisory Council to NINDS, approved several AD/ADRD research concepts to be further developed into new programs and initiatives as funds become available. Six examples are shown below.

      NINDS’s ADRD research concepts for FY2018

  • Center without Walls (CWOW) for ADRD Radioligand Development and Testing – This would support the development of imaging ligands for Tau, TDP43, synuclein, and synaptic and neuroinflammatory markers that will enable better patient stratification, diagnosis, and tracking of disease progression in FTD ,  LBD  and dementias with mixed etiologies. Current Tau PET ligands, which were identified by screening for binding in AD tissue, lack the specificity and sensitivity required for the tau-related ADRDs. The research focus could include screening of existing and derivative compounds against tau, as well as the other protein aggregates found in FTD, LBD, and Parkinson’s Disease human tissue. The Center could support the synthesis of ligands, analysis of ligands in human tissue, and first-in-human studies. 
  • Planning Grants for Clinical Trials to develop treatments for LBD – To address the lack of treatments for the disabling clinical features of LBD, this concept was developed to provide support to convene a clinical trial team that will develop the rationale and research design required for the submission of an NIH clinical trial grant application.   
  • Mechanisms of diffuse white matter disease and small vessel pathology in Vascular Contributions to Cognitive Impairment and Dementia (VCID) – This would support hypothesis-testing research to elucidate cellular and molecular mechanisms that underlie diffuse white matter disease and small vessel disease in VCID. The ultimate goal is to develop a rigorous mechanistic understanding that will enable future translational and clinical studies in VCI/D, AD, and other mixed etiology dementias that include diffuse white matter disease and small vessel pathology.
  • LBD Center without Walls – A collaborative, multidisciplinary and possibly multi-national research group would be developed to elucidate the neuropathological mechanisms that result in the clinical pathology characteristic of LBD.  The Centers will focus on understanding interactions between tau and alpha-synuclein and how these contribute to selective cell and circuit vulnerabilities.
  • Pathway and target discovery for ADRD – This would support the discovery and validation of new pathways, targets, and potential biomarkers related to the human biology of ADRD, and support hypothesis-driven research using enhanced bioinformatics for large scale data analysis and disease modeling. Researchers would be encouraged to use existing cohorts, data, and well-characterized biological resources.
  • Understanding the structural biology of ADRD-associated proteins – This would leverage recent advances in Cryo-electron microscopy (Cryo-EM) to identify structural forms of ADRD-associated proteins present in human brain tissue.  Understanding structural differences in strains or isoforms of proteins like tau, alpha-synuclein, and TPD43 will support and advance the basic biology underlying ADRD, as well as contribute to the development of better diagnostic tools and therapies for these diseases.

Now is truly an exciting time for dementia research, and our program staff is working hard to transform these concepts into real funding announcements. In the meantime, I strongly encourage the research community to develop ideas and be prepared to take advantage of upcoming, unprecedented opportunities to advance ADRD research. 

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