Forum on Neuroscience and Nervous System Disorders: 2012 Annual Report (2013)

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BOARD ON HEALTH SCIENCES POLICY Forum on Neuroscience and Nervous System Disorders 2012 Annual Report 

About the Neuroscience Forum The IOM in 2006 established the Forum on Neuroscience and Nervous System Disorders. The Forum is designed to provide its members with a neutral venue for exchanging information, shar- ing individual views, and allowing a structured opportunity for dialogue and discussion while scrutinizing critical and possibly contentious scientific and policy issues. Representatives from government, industry, academia, patient advocacy organizations, and other interested parties serve as Forum members. It convenes three times a year to confer on subject areas of mutual interest and concern. At its meetings, the Forum identifies and discusses emerging scientific and policy issues related to basic neuroscience and nervous system disorders, as well as effective clinical inter- ventions and policy options. The Forum also sponsors workshops (symposiums) as an additional mechanism for informing the mem- bership of the Forum, other relevant stakeholders, and the public. Information about past or upcoming meetings is available at the Forum’s website, www.iom.edu/neuroforum. Cover Image Credits Left: Greg Hood, John Burkardt, and Greg Foss, Pittsburgh Supercomputing Center Right: Paul Thompson and Arthur Toga, UCLA 

Message from the Chair The Institute of Medicine (IOM) Forum on Neuroscience and Nervous System Disorders brings together the private sector; federal agencies that serve as research sponsors and regulators; the nonprofit sector, including foundations and groups focused on nervous system disease; and the academic community to consider shared approaches to pressing issues. Through its quarterly meetings and public workshops, the Forum has provided a venue for its members, other neuroscience leaders, government officials, and members of the public to have rich and candid discussions about issues of mutual interest. In 2012, discussions included such diverse topics as possible mechanisms for sharing and analyzing clinical trials data from industry-led studies; the limita- tions of animal models in central nervous system drug discovery and development; workforce requirements for treating mental, neurological, and substance use disor- ders in sub-Saharan Africa and shared priority setting for neuroscience research. Looking ahead to 2013, the Forum plans an array of activities on topics such as: • Accelerating therapeutic development for nervous system disorders towards first-in-human trials • Improving access to essential medicines for mental, neurological and substance use disorders in sub-Saharan Africa • Data standards to enable analysis of large basic research datasets • Defining the precompetitive space in neuroscience • Clinical trials infrastructure • Gene therapy and stem cell transplantation for the eye and brain I look forward to another productive year for the Forum. Steve Hyman Chair 

Reflecting Back Forum Activities in 2012 Neurodegeneration: Opportunities for Collaboration Across Disease- Specific Research and Development Communities Neurodegenerative diseases are becoming increasingly prevalent in the United States. In 2000, approximately 4 million people had Alzheimer’s disease and another 1 million had Parkinson’s disease. Recent findings have revealed potential commonalities and parallels in genetic and cellular mecha- nisms across neurodegenerative diseases. The Forum hosted a workshop that explored these commonalities, focusing on Alzheimer’s, Parkinson’s, amyo- trophic lateral sclerosis, and frontotemporal dementia. Participants from dif- ferent research and development communities came together to identify and discuss commonalities related to genetic and cellular mechanisms, identify areas of fundamental science needed to facilitate therapeutic development, and explore areas of potential collaboration among the respective research communities and sponsors. Improving Translation of Animal Models for Nervous System Disorders Animal models of disease mechanisms have significantly increased our understanding of nervous system diseases and disorders. Yet, a large gap remains in treatment options that are high in efficacy, but low in side effects, for many diseases. Given the tremendous disease burden associated with nervous system diseases and disorders, the Forum hosted a workshop that brought key stakeholders together to discuss potential opportunities for maximizing the translation of effective therapies from animal models to clinical practice. Specifically, participants discussed key issues that contribute to poor translation and examined case studies that highlighted successes and failures. Workshop sessions focused on standardization of animal models, matching basic and clinical research endpoints, and meth- ods to increase bidirectional translation. In addition, participants worked to NEURO FORUM TIMELINE 2006 2007 2006 Oct 4–5 2007 1st meeting 

Reflecting Back: Forum Activities in 2012 identify next steps that will be critical for improvement of the development and testing of animal models and key infrastructure components required for implementation. Strengthening Human Resources Through Core Competency Development for Mental, Neurological, and Substance Use Disorders in Sub-Saharan Africa Sub-Saharan Africa (SSA) has one of the largest treatment gaps for mental, neurological and substance use (MNS) disorders in the world. It is estimated that four out of five people with serious mental disorders do not receive the mental health services they need. The ability to provide adequate human resources for delivery of essential interventions in MNS disorders has been identified as a critical barrier to bridging the treatment gap. The Forum held a workshop that brought together key stakeholders to examine human resource core competencies needed to improve care for MNS disorders. The workshop focused on the disorders with the greatest disease burdens: depression, psy- chosis, epilepsy, and alcohol use. In addition, participants discussed potential mechanisms for task shifting and task sharing within human resources and across treatment locations. Sharing Clinical Research Data Pharmaceutical companies, academic institutions, advocacy organizations, and government agencies such as the Food and Drug Administration and the National Institutes of Health have large quantities of clinical research data. Increased data sharing could facilitate scientific and public health advances, among other potential benefits to patients and society. Much of this infor- mation, however, is not transparent or available beyond the data owner. Feb 27 Apr 18 2nd meeting Autism & Environment Workshop Feb 26 Jul 30–31 Oct 24–25 2008 Biomarkers Workshop 3rd meeting 4th meeting 

Specifically, study results are not always published and, when results are published, they typically include only summary-level data; participant-level data is privately held and rarely shared or revealed publicly. In collaboration with the IOM Forum on Drug Discovery, Development, and Translation; the National Cancer Policy Forum; and the Roundtable on Translating Genomic-Based Research for Health, the Forum organized a workshop to explore benefits of and barriers to sharing clinical research data and strategies for enhancing sharing both within and across sectors. During the workshop, models and projects that involve sharing other types of data were highlighted to the extent that these models provide lessons and best practices applicable to sharing preplanned interventional clinical research data. Jun 25 Molecules to Mind: Oct 15–16 Grand Challenges Workshop 7th meeting 2008 2009 2008 Jan 30 Jun 24 Oct 3 2 5th meeting 6th meeting Venture Philanthropy Strategies Workshop 

Looking Forward Looking Forward: Forum Activities in 2013 Forum Activities in 2013 Accelerating Therapeutic Development for Nervous System Disorders Towards First-in-Human Trials This activity will build on a previous Forum workshop, Improving Translation of Animal Models for Nervous System Disorders, which explored strategies for maximizing the translation of effective therapies from animal models to clinical practice. Two themes that emerged from the workshop were that many have lost confidence in the ability of animal models to predict efficacy and that current ani- mal models may, in fact, be screening out potentially effective compounds. Another theme was the need to combine animal models with emerging translational tools and technologies in therapeutic development. Following on these themes, the Forum will host a workshop to explore opportunities to accelerate the pathway from discovery to approval of new therapeutics for nervous system disorders. The workshop will seek to identify avenues for moving directly from cellular models to human trials, minimizing the need for animal models to test efficacy. Participants will also explore the potential usefulness of new neuroscience technologies and techniques. In addition, the workshop will examine regulatory mechanisms that may facilitate faster entry of potential treatments into first-in-human trails and identify potential metrics for determining readiness for first-in-human trials. Improving Access to Essential Medicines for Mental, Neurological, and Substance Use Disorders in Sub-Saharan Africa Sub-Saharan Africa (SSA) has one of the world’s largest treatment gaps for men- tal, neurological and substance use (MNS) disorders, causing a substantial burden for the continent’s population. Improving access to essential medicines has been identified as a critical component of reducing the treatment gap for MNS disorders. In an effort to achieve the long-term and reliable availability of essential medicines, the Forum will host a meeting that will bring together key stakeholders to discuss Aug 4–5 Nov 9 Feb 25 Jun 17 Sub-Saharan Africa Animal Law 8th meeting 9th meeting Workshop Workshop 2009 Jun 16 Nov 3 Suicidality Workshop 10th meeting 

opportunities for providing access to essential medicines for MNS disorders. Specifically, participants will examine successful models in a variety of disease areas and in low- and middle-income countries outside of SSA. Because of the complexity and multi-faceted nature of this topic, the meeting will also focus on determining the specific steps and part- nerships needed to make drugs accessible. Jun 17 Jun 22 Jul 12 9th meeting Glutamate Workshop ADNI-2 Workshop 2010 2011 2010 Mar 8–9 Jun 23 Dec 10 2011 Sex Differences Workshop 11th meeting 12th meeting 

Working Groups Working Groups The Forum has created a series of working groups to provide an opportunity for subsets of the broader group to work together on selected topics. Workshop topics often result from these groups and are organized by an independently appointed workshop planning committee. Translational Neuroscience The translational neuroscience working group was established to identify areas of synergy where public- and private-sector stakeholders can work together to improve the efficiency and effectiveness of drug discovery and development for neurological and mental disor- ders. The group is currently addressing barriers to repurposing and reusing existing com- pounds. In addition, it is discussing strategies to improve the understanding between what pharmaceutical and diagnostic developers are looking for and how academic researchers can best contribute to the partnership in a meaningful way in order to improve the neuro- science translational research pipeline. Training Translational Neuroscientists The pace of new therapeutic discovery for central nervous system disorders has noticeably slowed during the past decade. Advancing translational neuroscience, the application of fundamental research to therapeutic development, will be critical in efforts to reverse this trend. There is an increasing need for investigators who are able to bridge the gap between basic and clinical neuroscience. A working group will be established to examine current practices of training scientists and identify opportunities for increasing and strengthening translational neuroscience programs. Mental Health, Neurological, and Substance Use Disorders in Sub-Saharan Africa In collaboration with the World Health Organization mental health Gap Action Programme (mhGAP), the Forum is continuing to explore additional opportunities to build on the 2009 Mental Health and Neurological Disorders in Sub-Saharan Africa work- shop. The working group is focused on identifying innovative solutions to enhance care for MNS disorders in SSA. Special attention is being paid to finding solutions to increase human and financial resources to support current and future efforts. Mar 2–3 Jul 26–27 Neuroscience and the Law Workshop Animal Regulations Workshop 2012 Feb 2 Jul 18 Aug 10 Nov 30 2012 13th meeting AAIC 2011 Session 14th meeting 15th meeting 

Forum Members (as of December 2012) Steven Hyman (Chair) Richard Hodes Todd Sherer Broad Institute of the Massachusetts National Institute on Aging Michael J. Fox Foundation for Institute of Technology and Harvard Stuart Hoffman Parkinson’s Research Susan Amara Department of Veteran Affairs Paul Sieving Society for Neuroscience Thomas Insel National Eye Institute Marc Barlow National Institute of Mental Health Judy Siuciak GE Healthcare Phillip Iredale Foundation for the National Institutes of Health Mark Bear Pfizer, Global Research and Marc Tessier-Lavigne Massachusetts Institute Development Rockefeller University of Technology Daniel Javitt William Thies Katja Brose New York University School Alzheimer’s Association Cell Press of Medicine Nora Volkow Daniel Burch Frances Jensen National Institute on Drug Abuse Pharmaceutical Product University of Pennsylvania Kenneth Warren Development, Inc. Health System National Institute on Alcohol Abuse C. Thomas Caskey Story Landis and Alcoholism Baylor College of Medicine National Institute of Neurological John Williams Timothy Coetzee Disorders and Stroke Wellcome Trust FastForward of the National Alan Leshner Stevin Zorn Multiple Sclerosis Society American Association for the Lundbeck USA Emmeline Edwards Advancement of Science Charles Zorumski National Center for Complementary Husseini Manji Washington University School of Medicine and Alternative Medicine Johnson & Johnson Pharmaceuticals Martha Farah David Michelson University of Pennsylvania Merck Research Laboratories Richard Frank Richard Mohs IOM Staff Bruce Altevogt, Ph.D. GE Healthcare, Inc. Eli Lilly and Company Project Director Daniel Geschwind Jonathan Moreno University of California, Los Angeles University of Pennsylvania Diana Pankevich, Ph.D. School of Medicine Program Officer Hank Greely Stanford University Atul Pande Andrew M. Pope, Ph.D. GlaxoSmithKline Director, IOM Board on Health Sciences Policy Myron Gutmann National Science Foundation Steven Paul Magali Haas Weill Cornell Medical College One Mind for Research Mar 28 Jul 11 Sep 4–5 Nov 28 Mar 5 16th meeting 17th meeting SSA Workshop 18th meeting 19th meeting 2013 2012 Mar 28–29 May 10–11 Oct 4–5 2013 Animal Models Workshop Neurodegeneration Workshop Sharing Clinical Research Data Workshop 

Sponsors Forum Members and Sponsorship (as of December 2012) Financial support for the Forum is derived from federal agencies, patient advocacy organizations, industry, and a nonprofit membership society. Alzheimer’s Association Nonprofit CeNeRx Biopharma Department of Veterans Affairs Eli Lilly and Company 10% Foundation for the National Institutes of Health GE Healthcare GlaxoSmithKline Federal Johnson & Johnson Pharmaceuticals 47% Lundbeck USA 43% Merck Research Laboratories Industry The Michael J. Fox Foundation for Parkinson’s Research National Eye Institute National Institute of Mental Health National Institute of Neurological Disorders and Stroke National Institute on Aging National Institute on Alcohol Abuse and Alcoholism National Institute on Drug Abuse National Institutes of Health Blueprint for Neuroscience Research National Multiple Sclerosis Society National Science Foundation One Mind for Research Pfizer, Global Research and Development Society for Neuroscience Wellcome Trust Apr 8–9 Accelerating Therapeutic Oct 8 Development Workshop 20th meeting 

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