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Drug Repurposing and Repositioning: Workshop Summary (2014)

Chapter: Appendix B: Speaker Biographical Sketches

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Suggested Citation:"Appendix B: Speaker Biographical Sketches." Institute of Medicine. 2014. Drug Repurposing and Repositioning: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18731.
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B

Speaker Biographical Sketches

Ronald J. Bartek, M.A., is co-founder and president at Friedreich’s Ataxia Research Alliance; chairman of the board of the National Organization for Rare Disorders; 4-year member of the National Advisory Neurological Disorders and Stroke Council at the National Institutes of Health; and former partner and president of a business and technology development, consulting, and government affairs firm. Mr. Bartek’s professional experience also includes 20 years of federal executive branch and legislative branch service in defense, foreign policy, and intelligence, including 6 years on the policy staff of the House Armed Services Committee; 4 years at the State Department’s Bureau of Politico-Military Affairs, including a year as a negotiator on the U.S. Delegation to the Intermediate-Range Nuclear Forces Treaty talks in Geneva; 6 years as a Central Intelligence Agency analyst of political–military aspects of the East–West balance, including 1 year as an intelligence community representative to the interagency groups charged with U.S. arms control policy; and former director of the American Friends of the Czech Republic. Following graduation from the U.S. Military Academy at West Point, Mr. Bartek spent 4 years as an Army officer, serving as a company commander in Korea and an infantry and military intelligence officer in Vietnam. He has a master’s degree in Russian area studies from Georgetown University.

Atul Butte, M.D., Ph.D., is chief of the Division of Systems Medicine in the Department of Pediatrics and an associate professor in pediatrics, medicine, and, by courtesy, computer science at Stanford University and the Lucile Packard Children’s Hospital, and he is a pediatric endocrinologist. Dr. Butte received his undergraduate degree in computer science

Suggested Citation:"Appendix B: Speaker Biographical Sketches." Institute of Medicine. 2014. Drug Repurposing and Repositioning: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18731.
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from Brown University, and he worked in several stints as a software engineer at Apple Computer (on the System 7 team) and Microsoft Corporation (on the Excel team). He graduated from the Brown University School of Medicine. He completed his residency in pediatrics and his fellowship in pediatric endocrinology, both at Children’s Hospital, Boston. Dr. Butte received a Ph.D. in health sciences and technology from the Medical Engineering/Medical Physics Program in the Division of Health Sciences and Technology at Harvard Medical School and the Massachusetts Institute of Technology. The Butte Laboratory builds and applies tools that convert more than 300 billion points of molecular, clinical, and epidemiological data—measured by researchers and clinicians over the past decade—into diagnostics, therapeutics, and new insights into disease. Examples of this method includes work on cancer drug discovery published in the Proceedings of the National Academy of Sciences of the United States of America (2000), on type 2 diabetes published in the Proceedings of the National Academy of Sciences of the United States of America (2003), on fat cell formation published in Nature Cell Biology (2005), on obesity in Bioinformatics (2007), and in transplantation published in Proceedings of the National Academy of Sciences of the United States of America (2009). To facilitate this, the Butte Lab has developed tools to automatically index and find genomic datasets based on the phenotypic and contextual details of each experiment, published in Nature Biotechnology (2006); to re-map microarray data, published in Nature Methods (2007); to deconvolve multi-cellular samples, published in Nature Methods (2010); and to perform these calculations on the Internet “cloud,” as published in Nature Biotechnology (2010). The Butte Lab has also been developing novel methods for comparing clinical data from electronic health record systems with gene expression data, as described in Science (2008), and it was part of the team performing the first clinical annotation of a patient presenting with a whole genome, as described in Lancet (2010). The Butte Laboratory currently has been funded by the Howard Hughes Medical Institute and the National Institutes of Health.

Lon Cardon, Ph.D., joined GlaxoSmithKline (GSK) in 2008, initially as head of genetics, and now holds the position of head of alternative discovery and development, a pan-therapeutic division focused on nontraditional approaches for drug discovery and development. The unit takes on new diseases for GSK, such as ophthalmology and rare diseases; new modalities, including gene therapy, stem cells, and oligonucleotides; and bespoke academic and biotechnology partnership models. Prior to join-

Suggested Citation:"Appendix B: Speaker Biographical Sketches." Institute of Medicine. 2014. Drug Repurposing and Repositioning: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18731.
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ing GSK, Dr. Cardon was a senior academic in the United Kingdom and the United States, as professor of bioinformatics at the University of Oxford until 2006 and then as professor of biostatistics at the University of Washington and Fred Hutchinson Cancer Research Center in Seattle. He received his Ph.D. training from the Institute for Behavioral Genetics at the University of Colorado and conducted his postdoctoral research in the Department of Mathematics at Stanford University. He has received a number of scientific awards, including election to the United Kingdom’s Academy of Medical Sciences in 2005. He has authored more than 200 scientific publications and 15 books and chapters.

Christine Colvis, Ph.D., joined the National Center for Advanced Translational Sciences (NCATS) in June 2012 to lead the National Institutes of Health (NIH)-Industry Pilot Program: Discovering New Therapeutic Uses for Existing Molecules, which tests a new model for public–private partnership collaborations, including template agreements to shorten the time it takes to establish collaborations between an academic institution and a pharmaceutical company and move more rapidly into the actual research. The pilot involves eight pharmaceutical companies that made 58 assets available for repurposing by the broader research community. Collaborations established between academic institutions and the company will test ideas for new therapeutic uses, with the ultimate goal of identifying promising new treatments for patients. Before joining NCATS, Dr. Colvis had been a program director at the National Institute on Drug Abuse (NIDA) beginning in 2001 and later its director of program integration. She led NIDA’s management of the American Recovery and Reinvestment Act, which resulted in commitments of more than $300 million made by the Institute in 7 months. She has also been a leader and advisor for complex NIH programs, such as the molecular libraries and the NIH epigenomics programs. Before becoming a program director, Dr. Colvis had done a short postdoc at the National Eye Institute in its intramural program after receiving her Ph.D. from Oregon Health Sciences University in Portland.

Thomas O. Daniel, M.D., is executive vice president and president of research and early development at Celgene Corporation. Dr. Daniel has more than 2 decades of medical and pharmaceutical research leadership experience. He served as chief scientific officer and director at Ambrx Inc., a biotechnology company focused on discovering and developing protein-based therapeutics. As vice president of research at Amgen Inc., he was

Suggested Citation:"Appendix B: Speaker Biographical Sketches." Institute of Medicine. 2014. Drug Repurposing and Repositioning: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18731.
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research site head of AmgenWashington and therapeutic area head of inflammation. Prior to Amgen’s acquisition of Immunex, Dr. Daniel served as senior vice president of discovery research. At Immunex, he consolidated and built programs in oncology and vascular biology, advanced candidate therapeutics in those areas, and forged programmatic emphasis on antibody therapeutics. Dr. Daniel is a member of the Therapeutic Advisory Board of aTyr Pharma Inc. and is a director of Epizyme and Ferrumax. A nephrologist and former academic investigator, Dr. Daniel was previously the K.M. Hakim Professor of Medicine and Cell Biology at Vanderbilt University and the director of the Vanderbilt Center for Vascular Biology. He conducted research in the Howard Hughes Medical Institute at the University of California, San Francisco. He earned an M.D. from University of Texas, Southwestern, and completed a medical residency at Massachusetts General Hospital.

Harry (Hal) C. Dietz, III, M.D., is Victor A. McKusick Professor of Pediatrics, Medicine, and Molecular Biology and Genetics in the Institute of Genetic Medicine at the Johns Hopkins University School of Medicine. He is also an investigator in the Howard Hughes Medical Institute. His undergraduate training in biomedical engineering was performed at Duke University, and his M.D. degree was received from the Health Sciences University of Syracuse. Clinical and research training in pediatrics, pediatric cardiology, and genetics occurred at Johns Hopkins University School of Medicine. Dr. Dietz heads a multidisciplinary clinic for the diagnosis and management of individuals with heritable forms of cardiovascular disease, with a special emphasis on Marfan syndrome and related connective tissue disorders. He is director of the William S. Smilow Center for Marfan Research, a group of dedicated molecular biologists focused on improvement of the lives of individuals with Marfan syndrome and related disorders through the development of novel diagnostic and treatment strategies. Dr. Dietz has received multiple prestigious awards, including the Curt Stern Award from the American Society of Human Genetics and the Taubman Prize for excellence in translational medical science. He is an inductee of the American Society for Clinical Investigation, the American Association for the Advancement of Science, the Institute of Medicine, the Association of American Physicians, and the National Academy of Sciences.

Don Frail, Ph.D., is the vice president of science with AstraZeneca (AZ). He leads the science group in the New Opportunities iMed, which

Suggested Citation:"Appendix B: Speaker Biographical Sketches." Institute of Medicine. 2014. Drug Repurposing and Repositioning: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18731.
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seeks new opportunities complementary to AZ core areas through drug repositioning and open innovation partnerships. Recently, the team implemented groundbreaking partnerships with the United Kingdom’s Medical Research Council and the National Institutes of Health to collaborate with investigators to explore the use of AZ development compounds in new indications. Don recently coauthored the book Drug Repositioning: Bringing New Life to Shelved Assets and Existing Drugs. Prior to joining AZ, Dr. Frail held several leadership positions, including founder and chief scientific officer of the Indications Discovery Unit at Pfizer, head of Pfizer’s St. Louis research and development site, vice president of biology for the St. Louis site, and head of discovery neurosciences in Pharmacia.

Geoffrey Ginsburg, M.D., Ph.D., is the founding director for genomic medicine at Duke University and assumed his current position in the Duke Institute for Genome Sciences & Policy in 2004. He is also the founding executive director of the Center for Personalized Medicine established in the Duke University Health System in 2010. He is currently professor of medicine and pathology at Duke University Medical Center.

While at Duke, Dr. Ginsburg has pioneered translational genomics, initiating programs in genome-enabled biomarker discovery, longitudinal registries with linked molecular and clinical data, biomarker-informed clinical trials, and the development of novel practice models and implementation research for the integration of genomic tools in heath care systems. With a strong commitment to interdisciplinary science, he has led projects to develop predictive models for common complex diseases using high-dimensional genomic data as well as collaborations with engineering groups to develop novel point of care sensors. His work spans oncology, infectious diseases, cardiovascular disease, and metabolic disorders, and his research is addressing the challenges for translating genomic information into medical practice using new and innovative paradigms and the integration of personalized medicine into health care. He is an internationally recognized expert in genomics and personalized medicine with more than 200 published papers and funding from the National Institutes of Health, the Department of Defense, the Defense Advanced Research Projects Agency, the Gates Foundation, and industry.

In 1990 he joined the faculty of Harvard Medical School, where he was director of preventive cardiology at Beth Israel Hospital and led a laboratory in applied genetics of cardiovascular disease at Children’s Hospital. In 1997 he joined Millennium Pharmaceuticals Inc. as senior

Suggested Citation:"Appendix B: Speaker Biographical Sketches." Institute of Medicine. 2014. Drug Repurposing and Repositioning: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18731.
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program director for cardiovascular diseases, and he was eventually appointed vice president of molecular and personalized medicine, where he was responsible for developing pharmacogenomic strategies for therapeutics as well as biomarkers for disease and the implementation of those biomarkers in the drug development process.

He has received a number of awards for his research accomplishments, including the Innovator in Medicine Award from Millennium in 2004 and the Basic Research Achievement Award in Cardiovascular Medicine from Duke in 2005. He is a founding member and former board member of the Personalized Medicine Coalition, a senior consulting editor for the Journal of the American College of Cardiology, an editor for the HUGO Journal, and an editorial advisor for Science Translational Medicine. In addition he is the editor of Genomic and Personalized Medicine (Elsevier), the first edition of which was published in 2009.

He has been a member of the Secretary of Veterans Affairs Advisory Council on Genomic Medicine and the National Advisory Council for Human Genome Research at the National Institutes of Health (NIH). He is currently an international expert panel member for Genome Canada; a member of the board of external experts for the National Heart, Lung, and Blood Institute; a member of the Institute of Medicine’s Roundtable on Translating Genomic-Based Research for Health; and a member of the external scientific panel for the Pharmacogenomics Research Network. He has recently been appointed to the advisory council for the newly established National Center for Advancing Translational Sciences at NIH. He has recently been nominated to serve on the World Economics Forum’s Global Agenda Council on Personalized and Precision Medicine.

He received his M.D. and a Ph.D. in biophysics from Boston University and completed an internal medicine residency at Beth Israel Hospital in Boston, Massachusetts. Subsequently, he pursued postdoctoral training in clinical cardiovascular medicine at Beth Israel Hospital and in molecular biology at Children’s Hospital as a Bugher Foundation Fellow of the American Heart Association.

Petra Kaufmann, M.D., M.Sc., is director of the Office of Clinical Research (OCR) at the National Institute of Neurological Disorders and Stroke (NINDS). In this capacity she oversees the clinical research programs funded by NINDS. The OCR fosters clinical research that increases our understanding of the cause, diagnosis, treatment, and prevention

Suggested Citation:"Appendix B: Speaker Biographical Sketches." Institute of Medicine. 2014. Drug Repurposing and Repositioning: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18731.
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of neurological diseases and translates scientific discoveries into improved therapies for people living with neurological diseases worldwide.

Prior to joining NINDS, Dr. Kaufmann was a tenured associate professor of neurology at Columbia University in New York City. She earned her medical degree from the University of Bonn, Germany, and a master of science degree in biostatistics from Columbia’s Mailman School of Public Health. She completed an internship in medicine at St. Luke’s/Roosevelt Hospital in New York City and trained in neurology and clinical neurophysiology at Columbia University. She did a postdoctoral fellowship in molecular biology of mitochondrial diseases at Columbia’s H. Houston Merritt Center for Muscular Dystrophies and Related Diseases. While on the faculty of Columbia University, she worked clinically in the neuromuscular division, the electromyography laboratories, and the pediatric neuromuscular clinic. Her research focused on the clinical investigation of spinal muscular atrophy, amyotrophic lateral sclerosis, and mitochondrial diseases.

Gabriela Lavezzari, Ph.D., M.B.A., joined PhRMA in July 2012 as assistant vice president, scientific affairs. In this role Dr. Lavezzari is the primary staff lead for a variety of strategic initiatives aimed at establishing PhRMA as a valuable source of scientific expertise in innovative biopharmaceutical research and development within the Scientific & Regulatory Affairs (S&RA) division of PhRMA. Dr. Lavezzari brings to PhRMA more than 10 years of combined research experience in the government and industry, with multidisciplinary expertise in personalized medicine.

Prior to joining PhRMA, Dr. Lavezzari served as director of extramural development at the Medco Research Institute, a subsidiary of Medco Health Solutions, where she led clinical utility and cost-effectiveness research to create value-based reimbursement decisions in a variety of different therapeutic areas. Prior to Medco, Dr. Lavezzari spent a few years at Theranostics Health, a proteomic-based diagnostics company where she led the laboratory operations and the oncology product development. Prior to Theranostics, Dr. Lavezzari worked at Social Scientific Systems, where she provided scientific support to and managed multiple AIDS clinical trials group, laboratory science, laboratory technical, and specialty laboratory committees, subcommittees, and working groups.

Suggested Citation:"Appendix B: Speaker Biographical Sketches." Institute of Medicine. 2014. Drug Repurposing and Repositioning: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18731.
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In addition to her experience in industry, Dr. Lavezzari spent almost 6 years in research at the National Institutes of Health and at Georgetown University, where she completed her postdoctoral training.

Dr. Lavezzari received her Ph.D. in biological sciences from the University of Milano (Italy) and received her M.B.A. from the New York Institute of Technology.

John C. McKew, Ph.D., has been branch chief of the Therapeutic Development Branch at the National Institutes of Health (NIH) Center for Translational Therapeutics (NCTT) and the director of chemistry for the NCTT. His responsibilities include developing the Therapeutics for Rare and Neglected Disease Program and the Bridging Interventional Development Gaps Program (formerly the NIH-RAID Program). Both of these programs focus on novel public–private partnerships to advance collaborative drug discovery projects through pre-clinical development into early clinical development. Prior to joining NIH, Dr. McKew held a director-level position at Wyeth Research, and he began his career at Genetics Institute in Cambridge, Massachusetts, spending a total of 17 years between the two. At Wyeth he led a chemistry group to identify promising compounds for cardiovascular, musculoskeletal, and metabolic disease therapeutic areas. Prior to that, Dr. McKew spent 10 years working in the inflammation therapeutic area, with his work resulting in multiple compounds entering clinical evaluation. His research interests include rare and neglected diseases, medicinal chemistry, synthetic methodology, and tool compounds to probe biology. These interests have resulted in more than 20 publications, 10 granted U.S. patents, and multiple podium presentations. Dr. McKew also enjoys sharing his passion for science with others. This has prompted him to become course director and lecturer in GMS PM 881, Drug Discovery and Development, a graduate-level course in the Department of Pharmacology and Experimental Therapeutics, which resulted in his appointment as an adjunct associate professor at the Boston University School of Medicine. He has also taken an active role in the Northeastern Section of the American Chemical Society and has served as the chair-elect, chair, and the immediate past chair. Dr. McKew graduated from the State University of New York at Stony Brook with B.S. degrees in chemistry and biochemistry. He completed his Ph.D. in organic chemistry at the University of California, Davis, and held postdoctoral research positions at the University of Geneva and Firmenich, S.A.

Suggested Citation:"Appendix B: Speaker Biographical Sketches." Institute of Medicine. 2014. Drug Repurposing and Repositioning: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18731.
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Michael A. Pacanowski, Pharm.D., M.P.H., is the acting associate director for genomics in the Office of Clinical Pharmacology at the U.S. Food and Drug Administration (FDA). Dr. Pacanowski received his Pharm.D. from the Philadelphia College of Pharmacy. He then completed clinical training at Bassett Healthcare in Cooperstown, New York, and a clinical research fellowship in cardiovascular pharmacogenomics at the University of Florida, where he also received his M.P.H. Dr. Pacanowski’s expertise is in the area of genetic epidemiology and public health genomics, specifically as related to pharmacogenomic strategies in drug development and utilization. At FDA, he oversees review of investigational and new drug applications, contributes to regulatory policy development, and conducts research that supports FDA’s core public health mission.

Aidan Power, M.B., B.Ch., M.Sc., M.R.C.Psych., has been vice president and head of PharmaTx Precision Medicine since January 2008. Precision medicine represents a synthesis of all the emerging technologies and operations (computational science, imaging, pharmacogenomics, metabolomics, proteomics, physiological measurements, and diagnostics) that form the scientific basis of emerging approaches to the development of personalized medicine. Graduating in medicine from University College Cork, Ireland, Dr. Power trained as a psychiatrist in England and joined Pfizer in the United Kingdom in 1993, working on the antidepressant sertraline and the antipsychotic ziprasidone. In 2002 Dr. Power relocated to Pfizer’s global research and development headquarters in New London, Connecticut, where he headed clinical pharmacogenomics. For the past 3 years he has headed up molecular medicine (now PharmaTx Precision Medicine), which has been integrating molecular studies across disease areas as well as developing diagnostics for critical programs in the Pfizer product pipeline.

Arti Rai, J.D., the Elvin R. Latty Professor of Law, is an internationally recognized expert in intellectual property (IP) law, administrative law, and health policy. Ms. Rai has also taught at Harvard, Yale, and the University of Pennsylvania law schools. Her research on IP law and policy in biotechnology, pharmaceuticals, and software has been funded by the National Institutes of Health and the Kauffman Foundation. She has published more than 50 articles, essays, and book chapters on IP law, administrative law, and health policy. Her publications have appeared in both peer-reviewed journals and law reviews, including the New England

Suggested Citation:"Appendix B: Speaker Biographical Sketches." Institute of Medicine. 2014. Drug Repurposing and Repositioning: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18731.
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Journal of Medicine, the Journal of Legal Studies, Nature Biotechnology, and the Columbia, Georgetown, and Northwestern law reviews. She is the editor of Intellectual Property Law and Biotechnology: Critical Concepts (Edward Elgar, 2011), the coauthor of a 2012 Kauffman Foundation monograph on cost-effective health care innovation, and the coauthor of a casebook on law and the mental health system.

From 2009 to 2010 she served as the administrator of the Office of External Affairs at the U.S. Patent and Trademark Office (USPTO). As external affairs administrator, Ms. Rai led policy analysis of the patent reform legislation that ultimately became the America Invents Act and worked to establish the USPTO’s Office of the Chief Economist. Prior to that time, she had served on President-Elect Obama’s transition team reviewing the USPTO. Prior to entering academia, Ms. Rai clerked for the Honorable Marilyn Hall Patel of the U.S. District Court for the Northern District of California, was a litigation associate at Jenner & Block (doing patent litigation as well as other litigation), and was a litigator at the Federal Programs Branch of the U.S. Department of Justice’s Civil Division.

Ms. Rai regularly testifies before Congress and relevant administrative bodies on IP law and policy issues and regularly advises federal agencies on IP policy issues raised by the research that they fund. Recently her work has focused on advising the Defense Advanced Research Projects Agency and the National Human Genome Research Institute. She is currently co-chair of the IP Committee of the Administrative Law Section of the American Bar Assocation. She also serves as a member of the Institute of Medicine’s Committee on Understanding the Global Public Health Implications of Substandard, Falsified, and Counterfeit Medical Products. In 2011 Ms. Rai won the World Technology Network Award for Law.

Ms. Rai graduated from Harvard College magna cum laude with a B.A. in biochemistry and history (history and science), she attended Harvard Medical School for the 1987–1988 academic year, and she received her J.D. cum laude from Harvard Law School in 1991. Ms. Rai’s moot court team at Harvard Law School won best brief and team honors at the school’s prestigious Ames Moot Court Competition.

Michael Ringel, Ph.D., is a partner and managing director in the Boston office of the Boston Consulting Group (BCG) and is BCG’s global topic leader on research and development (R&D) productivity in biopharmaceuticals. Dr. Ringel has worked for a range of biotech and pharmaceutical

Suggested Citation:"Appendix B: Speaker Biographical Sketches." Institute of Medicine. 2014. Drug Repurposing and Repositioning: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18731.
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clients, with the bulk of his work focused on R&D strategy, operations, and organization. He has worked on topics such as disease area strategy, transformation and process optimization, sourcing/partnerships, and organizational design as well as mergers and acquisitions/licensing strategy, valuation, and post-merger integration. Prior to joining the firm, Dr. Ringel worked in academia, pursuing research in theoretical population dynamics and conducting field experiments in the Amazon basin near Manaus, Brazil.

Dr. Ringel has a B.A. summa cum laude in biology from Princeton University, a Ph.D. in biology from Imperial College and a J.D. cum laude from Harvard Law School. He sits on the Board of The Nature Conservancy in Massachusetts.

Allen D. Roses, M.D., is president and chief executive officer of Cabernet, Shiraz, and Zinfandel Pharmaceutical Companies. Dr. Roses has established an international reputation for his work in pharmacogenetics, exploratory drug discovery, and clinical neuroscience. He founded Cabernet Pharmaceuticals in 2008 to provide pharmacogenetics (PGx) and project-management services to pharmaceutical and biotechnology companies, clinical research, and managed health care organizations and to academic institutions. He has formed a team of consultants with deep experience in the practical application of PGx to drug development.

Dr. Roses also serves in several capacities at Duke University: as Jefferson–Pilot Professor of Neurobiology and Genetics, as professor of medicine (neurology), as director of the Deane Drug Discovery Institute, and as senior scholar at the Fuqua School of Business. He recently returned to Duke after a decade-long career as a senior vice president at GlaxoSmithKline (GSK) and its corporate predecessor GlaxoWellcome (GW). Upon joining GW in 1997, he organized genetic strategies for susceptibility-gene discovery, pharmacogenetics strategy and implementation, and integration of genetics into medicine discovery and development. Subsequently at GSK, he headed research in genetics, genomics, proteomics, and bioinformatics in support of the entire research and development pipeline. Among the specific activities of his group at GSK were proof-of-principle experiments using linkage-disequilibrium mapping to identify susceptibility loci for drug-associated adverse events. With respect to hypersensitivity to the HIV/AIDS drug abacavir, for example, GSK identified the HLA-*B5701 locus with candidate-gene analyses and then prospectively established the sensitivity (97 percent) and specificity (>99 percent) of this genetic risk marker.

Suggested Citation:"Appendix B: Speaker Biographical Sketches." Institute of Medicine. 2014. Drug Repurposing and Repositioning: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18731.
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During his previous tenure at Duke, Dr. Roses was Jefferson–Pilot Professor of Neurobiology and Neurology, founding director of the Joseph and Kathleen Bryan Alzheimer’s Disease Research Center, chief of the Division of Neurology, and director of the Center for Human Genetics. He was one of the first clinical neurologists to apply molecular genetic strategies to neurological diseases. His laboratory reported the chromosomal location for more than 15 diseases, including several muscular dystrophies and Lou Gehrig’s disease. He led the team that in 1992 identified Apolipoprotein E (APOE) as a major, widely confirmed susceptibility gene in common late-onset Alzheimer’s disease. Translation of these findings to metabolic-pathway analyses and drug discovery and development continued in GSK, leading to Phase 3 trials now under way to evaluate the drug rosiglitazone for the treatment of Alzheimer’s disease.

Dr. Roses was a member of the science board of the U.S. Food and Drug Administration (FDA) between 2003 and 2007. He was a member of the board’s Subcommittee on Science and Technology that in 2007 authored the report FDA Science and Mission at Risk. He continues to consult with FDA and other regulatory agencies in the field of pharmacogenetics and companion diagnostics.

Recently Dr. Roses described the association of a variable polyT repeat [rs10524523] with the age of onset distribution of Alzheimer’s disease. The data enhanced the accuracy of the prior age of onset curves based solely on APOE genotypes (also developed by Dr. Roses at Duke in 1992). Shiraz Pharma was founded in 2009 to commercialize the intellectual property from this new discovery. Zinfandel Pharma was also founded in 2009 to plan and execute a prospective validation of the “523” diagnostic for predicting risk of Alzheimer’s disease onset in the next 5 to 7 years for individuals aged 60 to 87 years. A combination diagnostic validation study and prevention (delay of age of onset) clinical trial in epidemiologic-selected populations is currently in progress, having completed a voluntary genomic data submission discussion with FDA regarding the design of the clinical trial. Five epidemiology-based recruitment sites for Caucasians without cognitive impairment have been organized and are piloting subject recruitment in order to decrease the recruitment time once the trial commences.

Larry A. Sklar, Ph.D., is principal investigator and director of the University of New Mexico (UNM) Center for Molecular Discovery for the Roadmap Molecular Libraries Initiative of the National Institutes of Health.

Suggested Citation:"Appendix B: Speaker Biographical Sketches." Institute of Medicine. 2014. Drug Repurposing and Repositioning: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18731.
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He is Regents Professor of Pathology, Distinguished University Professor, Maralyn S. Budke Endowed Chair in Cancer Drug Discovery in the National Cancer Institute–designated UNM Cancer Center, and co-director of translational technology in the UNM Clinical and Translational Science Center. He has more than 360 publications and patents in leukocyte biology, molecular assembly in signal transduction, and cell adhesion as well as high-throughput flow cytometry for drug discovery and repurposing. He is co-inventor of the HyperCyt high-throughput flow cytometry platform and co-founder of IntelliCyt. Dr. Sklar received his Ph.D. in physical chemistry from Stanford University.

Simeon Taylor, M.D., Ph.D., received his education at Harvard University, from which he received a B.A. in chemistry, Ph.D. in biological chemistry, and an M.D. degree. He completed clinical training at Massachusetts General Hospital with a specialty in internal medicine and a subspecialty in endocrinology and metabolism. He joined the Diabetes Branch in the Division of Intramural Research at the National Institutes of Health (NIH) in 1979, where he rose to the position of branch chief, a position he held for 11 years (1989–2000). In addition, he served as director of the NIH Inter-Institute Clinical Training Program in Endocrinology and Metabolism (1995–1998). During his time at NIH, Dr. Taylor’s research was directed toward elucidating the molecular mechanisms of insulin action and also toward understanding the causes of insulin resistance in human diseases such as diabetes and obesity. This work resulted in more than 200 publications in the scientific literature. Dr. Taylor’s contributions have been recognized by several awards, including the Outstanding Service Award of the U.S. Public Health Service (1990) and the American Diabetes Association’s Outstanding Scientific Achievement Award (“Lilly Award”) (1992). In addition, he has served on the editorial boards of numerous journals, including the Journal of Clinical Investigation; Journal of Clinical Endocrinology and Metabolism; Journal of Biological Chemistry, Endocrinology, Molecular Endocrinology; and Endocrine Reviews. After 21 years at NIH, Dr. Taylor moved to Eli Lilly and Co., where he was a Lilly Research Fellow in the Department of Endocrine Research (2000–2002). In 2002 Dr. Taylor was appointed as vice president, discovery biology at the Hopewell, New Jersey, site of the Bristol-Myers Squibb Company, where he led drug discovery biology in cardiovascular and metabolic diseases (2002–2010). In addition, he served as co-chair of the cardiovascular and metabolic dis-

Suggested Citation:"Appendix B: Speaker Biographical Sketches." Institute of Medicine. 2014. Drug Repurposing and Repositioning: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18731.
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ease strategy team (2002–2008) and currently serves as co-chair of the cardiovascular disease early asset team.

Sharon Terry, M.A., is president and chief executive officer of the Genetic Alliance, a network of more than 10,000 organizations, 1,200 of which are disease advocacy organizations. Genetic Alliance improves health through the authentic engagement of communities and individuals. It develops innovative solutions through novel partnerships, connecting consumers to smart services. Ms. Terry is also the founding chief executive officer of PXE International, a research advocacy organization for the genetic condition pseudoxanthoma elasticum (PXE). As co-discoverer of the gene associated with PXE, she holds the patent for ABCC6 and has assigned her rights to the foundation. She developed a diagnostic test and is conducting clinical trials. Ms. Terry is also a co-founder of the Genetic Alliance Registry and Biobank. She is the author of more than 90 peer-reviewed articles. In her focus at the forefront of consumer participation in genetics research, services, and policy, she serves in a leadership role on many of the major international and national organizations, including the Institute of Medicine (IOM) Health Sciences Policy Board, the National Coalition for Health Professional Education in Genetics board, and the International Rare Disease Research Consortium Interim Executive Committee, and she is a member of the IOM Roundtable on Translating Genomic-Based Research for Health. She is on the editorial boards of several journals. She was instrumental in the passage of the Genetic Information Nondiscrimination Act. In 2005 she received an honorary doctorate from Iona College for her work in community engagement; the first Patient Service Award from the University of North Carolina Institute for Pharmacogenomics and Individualized Therapy in 2007, the Research!America Distinguished Organization Advocacy Award in 2009, and the Clinical Research Forum and Foundation’s Annual Award for Leadership in Public Advocacy in 2011. She is an Ashoka Fellow.

Weida Tong, Ph.D., is director of the Division of Bioinformatics and Biostatistics at the U.S. Food and Drug Administration’s National Center for Toxicological Research. He also holds several adjunct positions at universities, including that of associate professor at the University of Medicine and Dentistry of New Jersey. His division at the Food and Drug Administration (FDA) develops bioinformatic methodologies and standards to support FDA research and regulation and advances

Suggested Citation:"Appendix B: Speaker Biographical Sketches." Institute of Medicine. 2014. Drug Repurposing and Repositioning: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18731.
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regulatory science and personalized medicine. The most visible projects from his group are (1) development of the FDA bioinformatics system, ArrayTrack™ suite, to support FDA review and research on pharmacogenomics; (2) leading the effort on the Microarray Quality Control Consortium to develop standards for translational science and personalized medicine; (3) development of liver toxicity knowledge base for drug safety; and (4) in silico drug repositioning. In addition, his group also specializes in molecular modeling and quantitative structure–activity relationships with specific interest in estrogen, androgen, and endocrine disruptor. Dr. Tong has published more than 180 papers and book chapters.

Christopher S. Watkins, Ph.D., is director of translational research and industry at the Medical Research Council (MRC) in the United Kingdom. The MRC is the largest public funder of biomedical research in the United Kingdom, with an annual budget of more than £750 million. The MRC supports and advances medical research in three main ways: by providing research grants and career awards to scientists in UK universities and hospitals, by funding research centers in partnership with universities, and through its own research facilities. Dr. Watkins’ role is leading the development and implementation of the MRC’s translational research and industry liaison strategies, which include activities such as the MRC/TSB Biomedical Catalyst, the multiple partner consortia in stratified medicine, and the recent MRC/AstraZeneca compound access initiative. After undergraduate and postgraduate studies at Imperial College, Dr. Watkins undertook postdoctoral research at the Royal Free Hospital, London, and the MRC National Institute for Medical Research. He has been engaged in research administration at the MRC head office since 1999, with previous roles including responsibility for the MRC’s clinical trials portfolio and the MRC’s Health Services and Public Health Research Board, before leading its translational research activities beginning in 2008.

Suggested Citation:"Appendix B: Speaker Biographical Sketches." Institute of Medicine. 2014. Drug Repurposing and Repositioning: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18731.
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Next: Appendix C: Statement of Task »
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 Drug Repurposing and Repositioning: Workshop Summary
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Drug development can be time-consuming and expensive. Recent estimates suggest that, on average, it takes 10 years and at least $1 billion to bring a drug to market. Given the time and expense of developing drugs de novo, pharmaceutical companies have become increasingly interested in finding new uses for existing drugs - a process referred to as drug repurposing or repositioning.

Historically, drug repurposing has been largely an unintentional, serendipitous process that took place when a drug was found to have an offtarget effect or a previously unrecognized on-target effect that could be used for identifying a new indication. Perhaps the most recognizable example of such a successful repositioning effort is sildenafil. Originally developed as an anti-hypertensive, sildenafil, marketed as Viagra and under other trade names, has been repurposed for the treatment of erectile dysfunction and pulmonary arterial hypertension. Viagra generated more than $2 billion worldwide in 2012 and has recently been studied for the treatment of heart failure.

Given the widespread interest in drug repurposing, the Roundtable on Translating Genomic-Based Research for Health of the Institute of Medicine hosted a workshop on June 24, 2013, in Washington, DC, to assess the current landscape of drug repurposing activities in industry, academia, and government. Stakeholders, including government officials, pharmaceutical company representatives, academic researchers, regulators, funders, and patients, were invited to present their perspectives and to participate in workshop discussions. Drug Repurposing and Repositioning is the summary of that workshop. This report examines enabling tools and technology for drug repurposing; evaluates the business models and economic incentives for pursuing a repurposing approach; and discusses how genomic and genetic research could be positioned to better enable a drug repurposing paradigm.

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