Parkinson's Disease Therapeutics Conference
Wednesday, October 26, 2011
Presented by the Michael J. Fox Foundation for Parkinson's Research and the New York Academy of Sciences
The fifth annual Parkinson's Disease Therapeutics conference, chaired by David Weiner, MD Scientific Advisory Board member of the Michael J. Fox Foundation for Parkinson's Research, will highlight novel advances in basic and translational research that impact our understanding of Parkinson's Disease and its treatment. Select Michael J. Fox Foundation-funded investigators will present research on a variety of topics, including novel therapeutic targets, biomarkers for early detection and assessment of disease progression, and strategies to alleviate symptoms and/or to slow disease progression. In addition, a small poster session on innovative Parkinson's Disease therapeutic targets and critical research tools will take place.
October 26, 2011
Welcome by Todd Sherer, PhD and David Weiner, MD
Novel Therapeutic Targets
Discovery of mGluR4 Potentiators for Symptomatic and Disease-Modifying Treatment of PD
The Role of Striatal Serotonergic Terminals in L-dopa Induced-Dyskinesia in PD Patients: An in vivo PET Study
NURR1 as a Target for Parkinson's Disease
Small Molecule Inhibitors of CAV.123 Calcium Channels as Neuroprotective PD Therapies
Networking Break / Poster Viewing
Novel Therapeutic Targets (continued)
Exploiting Ser910/935 Phosphorylation and 14-3-3 Binding to Develop Biomarkers for LRRK2 Activity
AFFITOPE-Based Immunotherapeutic Strategies for Parkinson's Disease
Targeting Glia in PD with Novel Anti-inflammatory Therapeutics
Lunch / Poster Viewing
Research Tools and Resources
Cerebrospinal Fluid Biomarkers of Microtubule-mediated Axonal Transport for Monitoring of Disease Progression and Therapeutic Interventions in Parkinson's Disease
Patient-specific iPS Cell-derived Neurons and Disease-associated LRRK2 Mutations for Parkinson's Disease Research
Cross-comparison of Alpha-Synuclein Assays and Update on the Parkinson's Progression Markers Initiative
Networking Break / Poster Viewing
Hot Topics in Parkinson's Disease Research
Toxic Forms of Alpha-Synuclein
Thoughts on PD Drug Development: Challenges and Opportunities
Cocktail Reception / Poster Viewing
Brian J. Bacskai, PhD, Massachussetts General Hospital
Development and Screening of Contrast Agents for In Vivo Imaging of Parkinson's Disease
Ethan S. Burstein, PhD, Acadia Pharmaceuticals
Development of Nurr1-RXR Heterodimer Selective Agonists for Parkinson's Disease
Sean Clark, PhD, Amicus Therapeutics
Use of the Orally Administered Pharmacological Chaperone AT3375 to Reduce Alpha-Synuclein Levels in the Brain of Thy-1 Alpha-Synuclein Mice
Verne E. Cowles, PhD, Depomed, Inc.
Study to Compare Levodopa/Carbidopa Safety, Tolerability, Pharmacokinetics and Pharmacodynamics following Administration of Gastric Retentive Extended-release Tablets versus a Reference Extended-Release Tablet in Parkinson's Disease Patients
Sonal Das, PhD, The Michael J. Fox Foundation for Parkinson's Research
MJFF-Funded Research Tools and Data Results
Claudio H. Hetz, PhD, University of Chile
Targeting the Unfolded Protein Response (UPR) Transcription Factor XBP-1 to Treat Parkinson's Disease
Sok Kean Khoo, PhD, Van Andel Research Institute
Circulating microRNAs as Biomarkers for Early Detection of Parkinson's Disease
Wassilios Meissner, MD, PhD, Université Victor Segalen Bordeaux 2
Multisite Coordinated Reset Stimulation of the Subthalamic Nucleus in the MPTP Non-Human Primate Model for Treating Parkinsonism
Claire Meunier, The Michael J. Fox Foundation for Parkinson's Research
Fox Trial Finder: An Online Tool to Connect PD Volunteers with Recruiting Clinical Studies
Rachel Saunders-Pullman, MD, MPH, Albert Einstein School of Medicine
Nigral Ultrasound and Olfaction in the Ashkenazi Jewish LRRK2 Cohort
David Weiner, MD
Michael J. Fox Foundation for Parkinson's Research
David Weiner received his medical degree from the State University of New York at Buffalo. He was a National Institutes of Health/Howard Hughes Medical Institute Research Scholar in the Laboratory of Molecular Biology at the National Institutes of Neurological Disorders and Stroke (NINDS) in Bethesda. He trained in neurology at Cornell New York Hospital/Memorial Sloan Kettering Cancer Center, and was a post-doctoral fellow in the Molecular Neuropharmacology Laboratories at the University of Vermont. Dr. Weiner held an active clinical and teaching position in the University of California San Diego Department of Neurosciences, where he was most recently an Adjunct Associate Clinical Professor in Neurology.
Dr. Weiner has worked, both in the pre-clinical and clinical arena, on the discovery and development of novel small molecule therapeutics for human neuropsychiatric disease. He worked at ACADIA Pharmaceuticals Inc. from 1997–2006, where he held various positions including Head of Target Validation and Medical Director-CNS. His research group was involved in the characterization of g-protein coupled receptor targets, as well as studies defining the mechanisms of action of known and experimental drugs. As Medical Director, he fostered the early development (pre-clinical thru Phase 2) of Pimavanserin (ACP-103) for psychosis in Parkinson's Disease, as well as Pimavanserin and n-desmethylclozapine (ACP-104) for the treatment of Schizophrenia. Most recently, Dr. Weiner had global responsibilites for early clinical development in neurology at EMD/Merck Serono.
Anders Björklund, MD, PhD
Anders Bjorklund made his first important contributions, as a young postdoc in the early 1970s, in anatomical studies of the dopaminergic and noradrenergic systems in the brain. These pioneering studies led to the discovery of previously unrecognized projections of the mesencephalic and diencephalic dopaminergic systems to the neocortex and areas of the limbic system, and the spinal cord. In the mid-1970s, Bjorklund's research interests shifted to central nervous system (CNS) regeneration and repair. In 1973, he started to experiment with transplantation of cells and tissue fragments to the intact and damaged rat brain. This led to a series of seminal papers, beginning in 1976, in which he and his collaborators for the first time were able to define the conditions for successful transplantation of developing neurons and neuroblasts to the adult CNS. Strengthened by the arrival of two gifted postdoctoral researchers, Steve Dunnett, PhD, and Fred H Gage, PhD, Bjorklund went on to explore this new cell repair approach in a range of rodent models of neurodegenerative diseases, trauma and aging. In 1987, Bjorklund teamed up with his former student, Olle Lindvall, MD, PhD, to initiate the first clinical trials of fetal neural cell grafting in patients with Parkinson's disease. This pioneering clinical program has been important in that it has given proof of principle for the feasibility of the cell replacement approach in the brain, and paved the way for recent developments in the application of the novel stem cell technology for brain repair. Bjorklund’s current research is focused on the development of stem cells for transplantation in patients with Parkinson´s disease; on studies on the role of the serotonin system in the induction of dyskinesia; and on the use of viral vector gene transfer techniques to model Parkinson-like neurodegeneration in rodents.
P. Jeffrey Conn, PhD
Vanderbilt University Medical Center
Jeff Conn received the PhD degree in Pharmacology from Vanderbilt University in 1986 and pursued postdoctoral studies in the Department of Pharmacology at Yale University. Dr. Conn joined the faculty of the Department of Pharmacology at Emory University in 1988 where he where he rose to the rank of full professor and established himself as a leader in studies of neurotransmitter receptors and their roles in regulating brain function in circuits involved in psychiatric and neurological disorders. In 2000, Dr. Conn moved to Merck and Company to assume the position of Senior Director and Head of the Department of Neuroscience at Merck’s site in West Point, PA. Dr. Conn moved to Vanderbilt University in 2003 to start a new Program in Drug Discovery, with a primary mission of facilitating translation of recent advances in basic science to novel therapeutics.
Dr. Conn is Editor in Chief of Molecular Pharmacology, Regional Editor (North America) of Current Neuropharmacology and serves on the editorial boards of six other international journals. He has served on the Scientific Advisory Boards of multiple pharmaceutical and biotech companies. as well as several foundations, including The Michael J. Fox Foundation.. He also served as Chairman of the Neuropharmacology Division of the American Society for Pharmacology and Experimental Therapeutics (ASPET).
Dr. Conn has received numerous awards and honors, including the NARSAD Essel Investigator Distinguished Investigator Award, the ASPET-Astellas Award in Translational Pharmacology, the Pharmacia - ASPET Award for Experimental Therapeutics, the Charles R. Park Award For Basic Research Revealing Insights into Physiology and Pathophysiology, and was named as an ISI Most-Cited Scientists in Pharmacology & Toxicology. He serves on several national and international committees, including International Union of Pharmacology (IUPHAR) subcommittee on receptor nomenclature, the American Society for Pharmacology and Therepeutics (ASPET) Publications Board of Trust, ASPET Awards Committee, and is an Expert Consultant, Compound Selection Committee, Treatment Units for Research on Neurocognition and Schizophrenia (TURNS). Dr. Conn’s current research is focused on development of novel treatment strategies for schizophrenia, Parkinson’s disease, and other brain disorders.
Nic Dzamko, PhD
University of Dundee
Dr. Dzamko obtained a BSc from Flinders University in South Australia and then worked as a research assistant for two years at the Garvan Institute of Medical Research in Sydney, Australia. Dr. Dzamko conducted his PhD studies on the physiological function of AMPK subunits at the University of Melbourne under the supervision of Professor Bruce Kemp. Dr. Dzamko joined the University of Dundee lab of Dr. Dario Alessi as a postdoctoral researcher in 2008, investigating the biochemical and physiological function of LRRK2, a protein kinase associated with autosomal dominant Parkinson's disease.
Patrizia Fanara, PhD
Patrizia Fanara received her PhD in molecular and cellular neuroscience from the University of Heidelberg, Germany. Her PhD thesis work, under the mentorship of Dr. Roland Brandt, was directed toward the understanding of brain microtubule proteins and their modifications in neurological conditions.
After completing her PhD in 1998, she moved to the Department of Biochemistry at Emory University for postdoctoral work in Dr. Anita Corbett's laboratory, where she developed fluorescence anisotropy assays to measure the interplay of energy and motion in the interactions between components of the nuclear transport machinery and correlate this to transport functions in vivo.
In 2003 Dr. Fanara joined the research team at KineMed, Inc., to apply in vivo stable isotope labeling technology to studying in vivo neuronal cytoskeleton modifications, discovering or evaluating novel therapeutics in ALS, Parkinson's and Alzheimer's diseases. Dr. Fanara was one of the recipients of the 2007 Prize4life award for best ideas in ALS biomarker discovery.
Ole Isacson, MD (Dr. Med. Sci.)
McLean Hospital / Harvard Medical School
Dr. Ole Isacson is professor of Neurology (Neuroscience) at Harvard Medical School. He is the director of the Center for Neuroregeneration Research/Neuroregeneration Laboratories at McLean Hospital. He is principal faculty of the Harvard Stem Cell Institute and a member of the Advisory Board for the Harvard NeuroDiscovery Center.
Dr. Isacson received his Medical Bachelor (1984) and Doctor of Medicine (as a full PhD doctoral degree and training in Medical Neurobiology, 1987) from the University of Lund in Sweden. His postdoctoral fellowship and training were at Cambridge University, England; from there he was recruited to start and direct his own laboratory and program in Boston in 1989, when he also joined the faculty of Harvard Medical School. Dr. Isacson is the past receiving editor of the European Journal of Neuroscience and current editor in chief of Molecular and Cellular Neuroscience. He sits on several editorial boards and has written over 280 original peer-reviewed scientific articles and reviews in his field. He has received several awards for his work.
Markus Mandler, PhD
Dr. Mandler did his PhD–Thesis at Boehringer Ingelheim's Institute of Molecular Pathology in Vienna and received his PhD from the University of Vienna in 2003. As a molecular biologist by training he has extensive experience in academic research and biotechnology industry and his work has lead to numerous publications in high impact journals as well as to the authorship of several patents and patent applications.
Dr. Mandler joined AFFiRiS in March 2004 and currently heads the neurodegeneration department. He is responsible for the pre-clinical development and evaluation of immunotherapies for the treatment of neurogenerative diseases. His main focus is currently set on the development of new vaccination strategies to treat Alzheimer's disease and Parkinson's disease, the two most common forms of neurodegenerative disorders in humans.
Kenneth Marek, MD
The Institute for Neurodegenerative Disorders
Kenneth Marek is President and Senior Scientist at the Institute for Neurodegenerative Disorders and Clinical Professor of Neurology at Yale University. He was graduated from Princeton University (AB, biochemistry) and received his medical degree from Yale University. Dr. Marek was trained in internal medicine and neurology at the Johns Hopkins School of Medicine and is board certified in both of these specialties. He received further training as a post-doctoral fellow in neurochemistry at the Institute of Neurology, Queens Square, London. He has been a faculty member in the departments of neurology at Johns Hopkins University and Yale University. Dr. Marek has been the recipient of numerous grants and awards including those from the National Institutes of Health, Department of Defense, Disease Foundation, and National Parkinson's Foundation. He serves on the scientific advisory board of the Michael J. Fox Foundation. He was a co-founder and continues to lead the AMADEUS consortium, an international SPECT imaging consortium for multi-center neuroimaging in clinical studies. He has served on the executive committee of the Parkinson's Study Group and in leadership roles in the Huntington Study group. He also was a co-founder of Molecular NeuroImaging, LLC, a company providing clinical neuroimaging research services.
Dr. Marek's major research interests include identification of biomarkers for early detection, assessment of disease progression and development of new treatments for Parkinson's disease and Alzheimer's disease and related neurodegenerative disorders. His specific interest has been in in vivo neuroreceptor imaging. He has authored numerous neurology and neuroscience publications on these topics. Dr. Marek has and continues to be the principal investigator of several ongoing multi-center international studies investigating the use of imaging to assess the onset, progression, and effect of treatment in Parkinson's disease, Alzheimer's disease and other neurodegenerative diseases.
Paola P. Piccini MD, PhD, FRCP
Imperial College London
Paola Piccini is a Reader in Neurology at Imperial College London (Division of Neuroscience and Mental Health) and Consultant in Neurology with the Hammersmith Hospitals Trusts. She was awarded her MD from the University of Pisa and her PhD from the University of London. She has worked extensively in the field of Movement Disorders for over 10 years and has published over 120 research articles in peer reviewed journals with high impact factors including Nature and book chapters. Dr. Piccini has been invited to numerous national and international meetings and has lectured in Europe, United States, Australia, India and Korea. She has contributed extensively to the teaching and training of undergraduates and postgraduates within Imperial College and she is the Neuroscience Module Leader for the Imperial College Medical School Graduate Entry MBBS course. Dr. Piccini is a Fellow of the Royal College of Physicians, fellow of Royal Society of Medicine and an active member of several societies including the British Neuroscience Association, the Association of British Neurologists, and the Movement Disorder Society. Currently, she serves as external scientific advisor for the UK PD Society, scientific advisor for the Swedish Research Council, ad hoc member of the MRC board subgroups and external advisor for the Princess Beatrix Foundations Neuroscience Panel. Her primary interest is on functional imaging in movement disorders and predominantly on the use of Positron emission tomography (PET) as a method of investigating aetiology, effects and complications of therapies, particularly new neuroprotective and neurorestorative therapies, in Parkinson's disease.
Darryle D. Schoepp, PhD
Merck and Company, Inc.
Darryle D. Schoepp serves as Senior Vice President and Franchise Head of Merck Research Laboratories. Dr. Schoepp has been Senior Vice President and Director of Neuroscience Drugs of Merck & Co. Inc. since March 2007. Dr. Schoepp has 20 years in neuroscience discovery research at Eli Lilly and Company, where for the past three years he has served as Vice President and overall global Head of neuroscience research and early clinical investigation. Dr. Schoepp serves on ... the Board of Publication Trustees for ASPET and as an Executive Editor for the journal Neuropharmacology. In 2002, Dr. Schoepp was honored with the Pharmacia / American Society for Experimental Therapeutics (ASPET) Award for Experimental Therapeutics for his research on the experimental therapeutics of metabotropic glutamate receptors. Dr. Schoepp received his Bachelor's degree in Pharmacy from North Dakota State University and his doctoral degree in Pharmacology and Toxicology from West Virginia University. He conducted postdoctoral research in Pharmacology and Toxicology at the University of Kansas. He is a member of the American College of Neuropsychopharmacology (ACNP).
Leslie M. Shaw, PhD
University of Pennsylvania School of Medicine
Dr. Shaw is the Professor of Pathology and Laboratory Medicine at the University of Pennsylvania School of Medicine, where he serves at the Director of the Clinical Toxicology Laboratory and Interim Director of the Clinical Chemistry Section. In 2006 Dr. Shaw received the Penn Medicine Award of Excellence. Dr. Shaw is involved in the National Institute of Aging-supported Alzheimer’s Disease Neuroimaging Initiative (ADNI), which is yielding promising results about the structural and biochemical changes in the brain and the genetic variations that mark the onset and progression of Alzheimer’s Disease. His group is interested in the use of biomarkers for grouping study participants into those at highest risk of developing Alzheimer’s disease and those at least risk. A similar effort to identify biomarkers for Parkinson disease progression - the Parkinson’s Progression Markers Initiative (PPMI) – is spearheaded and sponsored by the Michael J. Fox Foundation for Parkinson’s Research.
D. James Surmeier, PhD
Dr. James Surmeier is the Nathan Smith Davis Professor and Chair of the Department of Physiology at the Feinberg School of Medicine at Northwestern University and Director of the Morris K. Udall Research Center of Research Excellence for Parkinson's Disease at Northwestern University. Dr. Surmeier received his PhD in Physiology and Biophysics from the University of Washington in 1983. He trained with leaders in the field of neurophysiology, including Dr. Arnold Towe, Dr. William Willis and Dr. Stephen Kitai. In 1998, he moved to the Department of Physiology at Northwestern University and assumed his current position in 2001.
Dr. Surmeier's research program focuses on the ionic mechanisms underlying neural activity in the basal ganglia and their modulation by activation of G-protein coupled receptors, particularly those for dopamine. He has pioneered the application of modern patch clamp and single cell gene profiling approaches to understanding basal ganglia physiology, authoring over 100 peer-reviewed publications in journals such as Science, Nature, Neuron, Nature Neuroscience and the Journal of Neuroscience.
Dr. Surmeier has served in several advisory capacities lo the National Institutes of Health, including chairing study sections for NINDS and acting as a Councilor for NIAAA. He also serves on the scientific advisory boards of the Hereditary Disease Foundation, Dystonia Foundation and the Tourette Syndrome Association and a number of editorial boards. He has received many scientific awards including the NARSAD Established Investigator award, the Riker Award, the Picower Foundation Award and the Jacob Javits Neurosciencc Investigator Award.
Ronald Tjalkens, PhD
Colorado State University
Dr. Tjalkens is associate professor of Neurotoxicology and Neuroscience in the department of Environmental and Radiological Health Sciences at Colorado State University, in the College of Veterinary Medicine and Biomedical Sciences. He earned his PhD in toxicology from the University of Colorado Health Sciences Center in 1998 and completed a postdoctoral fellowship in Neurotoxicology and Experimental Neuropathology at the University of Michigan in 2001. Dr. Tjalkens' laboratory focuses on gene-environment interactions in neurodegenerative diseases, with particular emphasis on Parkinson's disease. Research interests include neuroinflammation and pathophysiologic signaling responses of astrocytes and microglia, as well as the effects of heavy metals and drugs on neuroinflammatory reponses of glia. Active projects explore both the regulation of inflammatory signaling in glial cells and molecular targets for new therapeutic approaches designed to mitigate inflammatory injury in neurodegenerative diseases.
John Q. Trojanowski MD, PhD
University of Pennsylvania School of Medicine
Dr. Trojanowski obtained his MD/PhD in 1976 from Tufts University in Boston. After a medicine internship at Mt. Auburn Hospital and Harvard Medical School, he began pathology/neuropathology training at Massachusetts General Hospital and Harvard Medical School (1977–1979), and completed training at the University of Pennsylvania School of Medicine in 1980 where he was appointed assistant professor of Pathology and Laboratory Medicine (1/1/1981) and rose to tenured full professor in 1990. Dr. Trojanowski's major leadership positions include: Director of a National Institute of Aging (NIA) Alzheimer's Disease Center (1991–present), Principal Investigator of a NIA Program Project Grant on Alzheimer's (AD) and Parkinson's (PD) disease (1990–present), Director of Medical Pathology (1988–2002), Interim Director (2001–2002) and Director (2002–present) of the Institute on Aging and Co-Director (1992–present) of the Center for Neurodegenerative Disease Research (CNDR) at the University of Pennsylvania School of Medicine. For over 15 years, Dr. Trojanowski has conducted research on AD, PD, motor neuron disease, dementia with Lewy bodies (DLB), frontotemporal dementias and related disorders. Most of his 450 papers, reviews and monographs focus on the pathobiology of neurodegenerative disorders, especially on the role of abnormal filamentous protein aggregates in the onset/progression of these disorders.
Dr. Trojanowski has received several awards for his research including a MERIT Award (1986–1994) from the National Institutes of Health (NIH), the Metropolitan Life Foundation Promising Investigator Award For Alzheimer's Disease Research (1991), membership in the American Society of Clinical Investigation (1991), an Established Investigator Award from the National Alliance for Research on Schizophrenia and Depression (1994), the Metropolitan Life Foundation Award For Alzheimer's Disease Research (1996), the Potamkin Prize For Research In Pick's, Alzheimer's And Related Diseases (1998), the first Pioneer Award from the Alzheimer's Association (1998), ISI Highly Cited Researcher 2000 (most highly cited neuroscientists for 1981–1999), the Stanley Cohen Biomedical Research Award of the University of Pennsylvania (2000), and membership in the Association of American Physicians (2000) as well as the Institute of Medicine (2002). He was elected President of the American Association of Neuropathologists (1997–1998), and is on the editorial board of several neuroscience and pathology journals.
Dr. Trojanowski has served and continues to serve on local and national aging research committees including the NIA Neuroscience, Behavior and Sociology of Aging Study Section (1987–1991), the National Advisory Council on Aging (NACA) of the NIA (1994–1998), the NACA Working Group Chair (1996–1998), the Medical and Scientific Advisory Board of the National Alzheimer's Association (1994–1997) as well as of the Southeastern Pennsylvania Chapter of the Alzheimer's Association (1992–present), the NIA Board of Scientific Counselors (1998–present), the Advisory Board of the Paul Beeson Physician Faculty Scholars In Aging Award (1998–present), the Program Committee of the World Alzheimer Congress 2000 (1998–2000), the Organizing Committee of the 6th International Conference On Progress In Alzheimer's And Parkinson's Disease, in 2003 in Seville, Spain (2001–2003), the Scientific Advisory Board of the Alliance For Aging Research (2002–present) and Chair of the "Biology of Synuclein and Cortical Lewy Bodies Associated with Dementia in AD, LBD, and PD" (July, 2001) and "Genetics of Alzheimer's Disease" (March, 2002) workshops organized by NIA and the National Institute on Neurological Diseases and Stroke in Bethesda, Maryland.
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