ADVISORY COMMITTEES
A unique project Steering Committee with expertise in drug discovery and development has been a defining feature of this project. Representatives include industry-seasoned scientists, leaders in academic translational research, and ex officio members from the FDA and NIH. This committee established the overall strategic plan and meets regularly with project participants to oversee the progress of the discovery effort.
The SMA Project Lead Development Team (LDT) provides intensive, day-to-day direction to the multi-site SMA Project drug discovery operations. The LDT includes members with extensive experience in industry drug discovery who bring high-level, practical expertise to our discovery effort. The LDT communicates frequently, both in its entirety and in smaller subgroups directed at specific program areas such as medicinal chemistry, in vitro assays, and animal testing. Regular communications between the LDT and the research facilities that form the backbone of our virtual drug company-style effort provide the expert project management and direction that are required for successful drug discovery on an accelerated schedule.
STEERING COMMITTEE MEMBERSHIP
Robert Pacifici, Ph.D. – Steering Committee Chair
Site Director and Chief Scientific Officer for CHDI Management/CHDI Foundation; Adjunct Professor, Department of Molecular Pharmacology and Toxicology, University of Southern California (USC). Former Chief Scientific Officer and Site Director for Research Triangle Park Laboratories of Eli Lilly and Company; ; former Vice President, Discovery Technologies, Xencor; former Director of Research Information and Automation Technologies, Amgen; and member, Advisory Board, Business Institute for Science, University of North Carolina Kenan-Flagler Business School. Pioneered an academic/industrial Ph.D. program between Amgen and USC and holds two patents. Research interests: Oxidative stress, receptor protein tyrosine kinases, automation, informatics, and quantitative biology. Disease areas: Drug development for multiple disease areas.
Anthony Bannon, Ph.D.
Director of Pharmacology, Icagen; former Principal Scientist, Neuroscience, Amgen, Inc.; former Research Scientist, Pharmacology/Pathology, Amgen, Inc.; former Senior Research Scientist, Neuroscience, Abbott Laboratories. During his career, Dr. Bannon has led multidisciplinary teams involved in the drug discovery process including chemistry, formulations, high-throughput screening, and pharmacokinetics and drug metabolism. He has also participated in teams that have identified and advanced four compounds from discovery/research to clinical development. Research interests: Drug discovery including efficacy model development, structure-activity relationships, in vivo pharmacokinetics, and early non-GLP toxicology. Disease areas: Diverse areas within CNS research including learning and memory, pain, feeding, cognition, anxiety, and schizophrenia.
Diana M. Escolar, M.D.
Associate Professor of Neurology and Pediatrics, George Washington University; member, Association for Patient-Oriented Research and the Muscular Dystrophy Association’s Clinical Services Advisory Board; and Director, Cooperative International Neuromuscular Research Group. Member or former member of editorial boards, including Journal of Clinical Neuromuscular Disorders. Holds professional certifications from the American Boards of Psychiatry and Neurology (additional qualifications in clinical neurophysiology) and American Association of Electrodiagnostic Medicine. Research interests: Neuromuscular disorders of infancy, childhood, and adolescence, and neuromuscular function and disease. Disease areas: Duchenne muscular dystrophy, Andersen syndrome, and Brody disease.
Howard J. Federoff, M.D., Ph.D.
Director, Center for Aging and Developmental Biology, Aab Institute of Biomedical Sciences, University of Rochester; Senior Associate Dean for Basic Research and Professor of Oncology, Genetics, Neurology, Medicine, Microbiology, and Immunology, University of Rochester School of Medicine; and member of several professional societies, including the American Association for the Advancement of Science, the New York Academy of Science, the Society for Neuroscience, and the American Society for Gene Therapy. Member or former member of editorial boards, including Gene Therapy, Experimental Neurology, and Gene Therapy Reviews. Currently holds nine patents and consults for several biotechnology companies, including AmpliVex, which he founded in 2003. Research interests: Molecular mechanisms that regulate nervous system plasticity and therapeutic strategies for the repair of damaged nervous system components. Disease areas: Stroke, Parkinson’s disease, and Alzheimer’s disease.
Edward D. Hall, Ph.D.
Director, Spinal Cord and Brain Injury Research Center, and Professor of Anatomy and Neurobiology, Neurology, and Neurosurgery, University of Kentucky Chandler Medical Center; Vice President-Elect, National Neurotrauma Society; Chair, VA Merit Review Neurobiology C Panel; member, Paralyzed Veterans of America Spinal Cord Research Foundation Scientific Advisory Board; former Senior Director of CNS Pharmacology, Pfizer Global Research and Development; former Senior Research Fellow and Director, Neurology Section, Neuroscience Therapeutics, Warner-Lambert/Parke-Davis; and past Distinguished Scientist VI, CNS Diseases Research, Upjohn/Pharmacia and Upjohn, Inc. Member or former member of editorial boards, including Journal of Neurotrauma and NeuroRx and a member of several professional societies. Research interests: Drug discovery aimed at finding neuroprotective agents for acute and chronic neurodegenerative disorders, and oxidative neurodegenerative mechanisms and pharmacological treatment of acute spinal cord and brain injury. Disease areas: Alzheimer’s disease, amyotrophic lateral sclerosis, stroke, and spinal cord and brain injury.
Graham Johnson, Ph.D.
Vice President for Discovery, Rib-X Pharmaceuticals, New Haven, Connecticut. Subsequent to obtaining a B.Sc. in Chemistry and Ph.D. from Heriot-Watt University in Edinburgh, Scotland and postdoctoral research at M.I.T. (Cambridge, USA), Imperial College (London) and R.I.M.A.C (Cambridge, USA), Dr. Johnson has held a number of increasingly senior positions within both the UK and US pharmaceutical industry. These companies have included Hoechst UK Ltd, Parke-Davis, and most recently Bristol-Myers Squibb, where before joining Rib-X, he was Vice President for Discovery Chemistry for Connecticut and Canada. During his career, Dr. Johnson has developed expertise in such diverse areas as antibiotics, nuclear hormone receptors, neuroscience and genitourinary diseases as well as early stage drug discovery through early clinical development. In addition to authoring and co-authoring a range of original and review articles reflecting these research interests, he has served in an editorial capacity for several research and review journals. Dr. Johnson has also been an inventor on over 50 patents. He has participated as a speaker, session chair, and conference organizer in a number of national and international meetings, including American Chemical Society, Medicinal Chemistry of Neurodegenerative Diseases, and the Gordon Medicinal Chemistry Conference.
John M. McCall, Ph.D.
Senior Consultant, PharMac, LLC; former Vice President, Global Research and Development, Pfizer; former Vice President, Global Discovery Chemistry, Pharmacia. Member of the Scientific Advisory Board for the Department of Medicinal Chemistry and Pharmacognosy at the University of Illinois. Member of the Business and Finance Committee of the Biosciences Research and Commercialization Center at Western Michigan University. Member of Board of Directors of ADMETRx, Avaant, and the M-Tec Screening Center. Member or former member of editorial boards including Current Opinion in Investigational Drugs, Current Opinion in Drug Discovery and Development, Journal of Medicinal Chemistry, and Expert Opinion on Investigational Drugs. Recipient of the Upjohn Award, the Chemistry Symposium Award and the Upjohn Science and Medicine Award. Dr. McCall has been awarded over 50 patents for his work. Research Interests: Drug discovery. Disease Areas: Ischemic stroke, spinal cord injury, and neurodegenerative diseases.
Richard M. Ransohoff, M.D.
Professor, Department of Molecular Medicine, Cleveland Clinic Lerner College of Medicine, Case Western Reserve University; Staff Neurologist, Mellen Center for Multiple Sclerosis Treatment and Research, Cleveland Clinic Foundation; Staff Scientist, Department of Neuroscience, Lerner Research Institute, Cleveland Clinic Foundation; member, Scientific Advisory Board of Chemocentryx, and External Advisory Board, CNS HIV Anti-Retroviral Therapy Effects Research (CHARTER); and Co-Director, Marine Biological Laboratory Special Topics Course “Pathogenesis of Neuroimmunological Disease.” Section Editor for Journal of Immunology and member, Advisory Editorial Board for Trends in Immunology. Research interests: Neurobiology of chemokines and their receptors, gene regulation by interferons. Disease areas: Multiple sclerosis and other CNS demyelinating disorders.
Allan J. Tobin, Ph.D.
Managing Director, MRSSI Inc.; Senior Scientific Advisor, High Q Foundation; Professor Emeritus of Neurology and of Physiological Science, University of California, Los Angeles (UCLA); former Director, UCLA Brain Research Institute; former Director, NeuroEngineering Training Program, and Former Eleanor Leslie Chair in Neuroscience, UCLA; Scientific Director Emeritus, Hereditary Disease Foundation; and member, Scientific Advisory Board, Diamyd (formerly Synectics), Stockholm, Sweden. Chaired the NINDS Strategic Planning Panel on Neurodegeneration, which recommended new NINDS-sponsored programs for neurodegenerative disease research. Member or former member of editorial boards, including Journal of Biological Chemistry and Neurobiology of Disease. Research interests: Pathogenic mechanisms and interventions in Huntington’s disease. Disease areas: Huntington’s disease, Parkinson’s disease, and spinal cord injury.
Ex Officio Steering Committee Members:
Jill Heemskerk, Ph.D.
Program Director, Technology Development Program, former Program Director, Neurodegeneration Program, and Project Officer, Spinal Muscular Atrophy Project and NINDS High Throughput Drug Screening Facility for Neurodegeneration, NINDS; and Member, NIH Molecular Libraries and Imaging Roadmap Implementation Group. Established numerous NINDS initiatives with an emphasis on small molecule screening and translating basic research findings to the clinic. Established the FDA-approved Drug Screening Program for Neurodegeneration in partnership with the Huntington’s Disease Society of America, the Amyotrophic Lateral Sclerosis (ALS) Association, and the Hereditary Disease Foundation. Established the Comparative Drug Testing in Rodent Models of Neurodegeneration program in partnership with the Michael J. Fox Foundation. Research areas: Neurodegeneration, therapeutics development, and molecular screening. Disease areas: Neurological disorders.
Tan Nguyen, M.D., Ph.D.
Medical Officer and Project Manager, Office of Orphan Products Development, Food and Drug Administration; Medical Director, Diagnostic Services and Comparative Medicine Laboratory, and Associate Professor of Pathology, Uniformed Services University of the Health Sciences; Affiliate Staff Pathologist, Walter Reed Army Medical Center; and member, College of American Pathologists, American Society of Clinical Pathologists, Commissioned Officer’s Association for the U.S. Public Health Service, and Association of Military Surgeons of the United States. Has received numerous service awards from the Department of Defense and Public Health Service, including the Defense Meritorious Service Medal and Surgeon General’s Exemplary Service Medal. Research interests: Promoting the development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. Disease areas: Orphan disorders including leukodystrophy and spinal muscular atrophy.
Expert Advisor
Kenneth Fischbeck, M.D.
Chief, Neurogenetics Branch, NINDS; former Professor of Neurology, University of Pennsylvania; professional certification, American Board of Psychiatry and Neurology. Elected to the Institute of Medicine of the National Academy of Sciences. Member or former member of editorial boards, including Neurobiology of Disease and Advances in Neurology. Research interests: Elucidating the mechanisms of hereditary neurological and neuromuscular disorders, particularly the polyglutamine expansion neurodegenerative diseases. Disease areas: Huntington’s disease, Kennedy’s disease, spinocerebellar ataxia, spinal muscular atrophy, Charcot-Marie-Tooth disease, muscular dystrophy, hereditary motor neuron disease, and Friedreich’s ataxia.
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LEAD DEVELOPMENT TEAM (LDT) MEMBERSHIP
John M. McCall, Ph.D. – LDT Chair
Senior Consultant, PharMac, LLC; former Vice President, Global Research and Development, Pfizer; former Vice President, Global Discovery Chemistry, Pharmacia. Member of the Scientific Advisory Board for the Department of Medicinal Chemistry and Pharmacognosy at the University of Illinois. Member of the Business and Finance Committee of the Biosciences Research and Commercialization Center at Western Michigan University. Member of Board of Directors of ADMETRx, Avaant, and the M-Tec Screening Center. Member or former member of editorial boards including Current Opinion in Investigational Drugs, Current Opinion in Drug Discovery and Development, Journal of Medicinal Chemistry, and Expert Opinion on Investigational Drugs. Recipient of the Upjohn Award, the Chemistry Symposium Award and the Upjohn Science and Medicine Award. Dr. McCall has been awarded over 50 patents for his work. Research Interests: Drug discovery. Disease Areas: Ischemic stroke, spinal cord injury, and neurodegenerative diseases.
Jill Heemskerk, Ph.D.
Program Director, Technology Development Program, former Program Director, Neurodegeneration Program, and Project Officer, Spinal Muscular Atrophy Project and NINDS High Throughput Drug Screening Facility for Neurodegeneration, NINDS; and Member, NIH Molecular Libraries and Imaging Roadmap Implementation Group. Established numerous NINDS initiatives with an emphasis on small molecule screening and translating basic research findings to the clinic. Established the FDA-approved Drug Screening Program for Neurodegeneration in partnership with the Huntington’s Disease Society of America, the Amyotrophic Lateral Sclerosis (ALS) Association, and the Hereditary Disease Foundation. Established the Comparative Drug Testing in Rodent Models of Neurodegeneration program in partnership with the Michael J. Fox Foundation. Research areas: Neurodegeneration, therapeutics development, and molecular screening. Disease areas: Neurological disorders.
Keith A. Houck, Ph.D.
Independent consultant, Becton-Dickinson Technologies. After obtaining a Ph.D. from Duke University in pathology and toxicology and doing postdoctoral research on the molecular and cellular characterization of VEGF at Genentech, Inc., Dr. Houck held a number of progressively senior positions with Sphinx Pharmaceuticals and Eli Lilly & Co. As Research Advisor with Eli Lilly, Dr. Houck directed lead generation projects from high-throughput screening assay development through SAR support for a wide variety of disease indications. Research interests: Cellular imaging, nuclear receptors, and cell signaling pathways. Disease areas: Cancer, diabetes, and cardiovascular diseases.
Graham Johnson, Ph.D.
Vice President for Discovery, Rib-X Pharmaceuticals, New Haven, Connecticut. Subsequent to obtaining a B.Sc. in Chemistry and Ph.D. from Heriot-Watt University in Edinburgh, Scotland and postdoctoral research at M.I.T. (Cambridge, USA), Imperial College (London) and R.I.M.A.C (Cambridge, USA), Dr. Johnson has held a number of increasingly senior positions within both the UK and US pharmaceutical industry. These companies have included Hoechst UK Ltd, Parke-Davis, and most recently Bristol-Myers Squibb, where before joining Rib-X, he was Vice President for Discovery Chemistry for Connecticut and Canada. During his career, Dr. Johnson has developed expertise in such diverse areas as antibiotics, nuclear hormone receptors, neuroscience and genitourinary diseases as well as early stage drug discovery through early clinical development. In addition to authoring and co-authoring a range of original and review articles reflecting these research interests, he has served in an editorial capacity for several research and review journals. Dr. Johnson has also been an inventor on over 50 patents. He has participated as a speaker, session chair, and conference organizer in a number of national and international meetings, including American Chemical Society, Medicinal Chemistry of Neurodegenerative Diseases, and the Gordon Medicinal Chemistry Conference.
Sabina Robinson, Ph.D.
Senior Scientist, Science Applications International Corporation (SAIC). Dr. Robinson earned her Ph.D. in Pharmacology and Experimental Therapeutics from The Johns Hopkins University School of Medicine. Since joining SAIC in 1983, she has participated in start-up and management of basic, translational, and clinical research programs to support discovery, development, and selection of drugs and other medical products. Dr. Robinson is skilled in bioinformatics and medical decision support and was selected as a 1999 Computerworld-Smithsonian Innovation Laureate. Through contracts awarded to SAIC, Dr. Robinson has provided scientific, technical, and managerial expertise to agencies that include the National Cancer Institute, Agency for Healthcare Research and Quality, Department of Health and Human Services, Substance Abuse and Mental Health Services Administration, U.S. Army Medical Research and Materiel Command, Defense Threat Reduction Agency, and NATO.
Anthony Bannon, Ph.D.
Director of Pharmacology, Icagen; former Principal Scientist, Neuroscience, Amgen, Inc.; former Research Scientist, Pharmacology/Pathology, Amgen, Inc.; former Senior Research Scientist, Neuroscience, Abbott Laboratories. During his career, Dr. Bannon has led multidisciplinary teams involved in the drug discovery process including chemistry, formulations, high-throughput screening, and pharmacokinetics and drug metabolism. He has also participated in teams that have identified and advanced four compounds from discovery/research to clinical development. Research interests: Drug discovery including efficacy model development, structure-activity relationships, in vivo pharmacokinetics, and early non-GLP toxicology. Disease areas: Diverse areas within CNS research including learning and memory, pain, feeding, cognition, anxiety, and schizophrenia.
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