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Parkinson's Disease Therapeutics Conference

Parkinson's Disease Therapeutics Conference

Wednesday, September 30, 2009

The New York Academy of Sciences

Presented By

Presented by the Michael J. Fox Foundation for Parkinson's Research and the New York Academy of Sciences


This conference, chaired by J. William Langston, MD—founder, CEO, and Scientific Director of the Parkinson's Institute in Sunnyvale, California—will highlight novel advances in basic and translational research that impact understanding of PD and its treatment. Select Michael J. Fox Foundation funded investigators will present research on a variety of topics, including novel therapeutic targets, biomarker development, and animal models.

    Presented by

  • Michael J. Fox Foundation


8:00 AM


8:30 AM

Welcome by J. William Langston, MD

8:45 AM

Session I: Novel Therapeutic Targets

Mathias Baehr, MD, University Hospital Göttingen
Granulocyte-colony Stimulating Factor (G-CSF) as a Neuroprotective Therapy for Parkinson's Disease

P. Jeffrey Conn, PhD, Vanderbilt University
Discovery of mGluR4 Potentiators for Symptomatic and Disease-Modifying Treatment of PD

David Bumcrot, PhD, Alnylam Pharmaceuticals, Inc.
Therapeutic Development of siRNA Targeting Alpha-Synuclein

10:30 AM

Networking Break/Poster Viewing

11:00 AM

Session I: Novel Therapeutic Targets (cont'd)

David Standaert, MD, PhD, University of Alabama at Birmingham
Validation of VPS41, A Protein Involved in Lysosomal Trafficking, as a Target for Parkinson Disease Therapy

Gretchen L. Snyder, PhD, Intra-Cellular Therapies, Inc.
Novel Phosphodiesterase Inhibitors for Parkinson's Disease

12:00 PM

Lunch/Poster Viewing

1:15 PM

Session II: Research Tools and Resources

Krystof Bankiewicz, MD, PhD, University of California, San Francisco
Image-Guided Convective Delivery of AAV Vectors

Susan Bressman, MD, Beth Israel Medical Center
Parkinson's Disease, Ashkenazi Jews and LRRK2

Robert L. Nussbaum, MD, University of California, San Francisco
P1 Artificial Chromosome Transgenic Mice as Models for Progressive Parkinson Disease

Michael G. Schlossmacher, MD, University of Ottawa
Characterization of Alpha Synuclein in Human Fluids & Brain as a Biomarker for PD

3:15 PM

Networking Break/Poster Viewing

3:45 PM

Session III: Hot Topics in PD

Franz Hefti, PhD, Avid Radiopharmaceuticals, Inc.
How Should One Design the First Alpha-Synuclein Efficacy Trial?

Andrew Singleton, PhD, National Institute on Aging/NIH
(How) Can GWAS Help Drug Developers?

4:45 PM

Closing Remarks

5:00 PM

Cocktail Reception/Poster Viewing

Poster Presenters

Thomas G. Beach, MD, PhD, Sun Health Research Institute
Brain & Body Donation Program of the Arizona Parkinson’s Disease Consortium

Steven P. Braithwaite, PhD, Mike Monaghan, and Bob Martone, Wyeth Pharmaceuticals
Targeting LRRK2 for Drug Discovery

Luke Esposito, MD, ProteoTech Inc.
New Small Molecule Inhibiting Agents of Alpha-Synuclein & Lewy Body Formation as Disease-Modifying Treatments for Parkinson's Disease

Mark Frasier, PhD, The Michael J. Fox Foundation
The Parkinson’s Progression Markers Initiative: A Prospective Biomarkers Study

David Grabli, MD, Hôpital de la Salpêtrière
Development of a New Primate Model of Postural Disturbance by Lesioning Non Cholinergic Neurons of the Reticular Formation

Ann M. Graybiel and Jill Crittenden, MIT
Evaluation of the Striatum-Enriched Genes, CalDAG-GEF1 and CalDAG-GEF2, as Targets for the Treatment and Prevention of L-DOPA Induced Dyskinesias

Chenjian Li, PhD, Weill Medical College of Cornell University
BAC-Engineered Alpha-Synuclein Transgenic Rats

Pamela J. McLean, PhD, Harvard University
Hsp90 as a Target for Neuroprotective Agents in Parkinson's Disease

Michael T. Rogan, PhD, The Michael J. Fox Foundation
PD Online Research: A Professional Collaborative Community for Rapid-Fire Problem Solving in Parkinson’s Disease Science


J. William Langston, MD

CEO and Founder, The Parkinson's Institute

Dr. J. William Langston is the Scientific Director,CEO Chief Operating Officer, and Founder of the Parkinson's Institute in Sunnyvale, California. A graduate of the University of Missouri School of Medicine, he served as a faculty member at Stanford University and Chairman of Neurology at Santa Clara Valley Medical Center in San Jose, California before founding the Parkinson's Institute. He is internationally recognized for the discovery of the link between a chemical known as MPTP and parkinsonism, which has provided an new tool to study Parkinson's disease and which also stimulated great interest in the possibility that environmental factors may play a role in causing the disease. His current research interests include the study of mechanisms of neuronal degeneration, the etiology of Parkinson's disease, and the development of new strategies to slow or halt disease progression. To date, Dr. Langston has published 300 papers on Parkinson's disease and related disorders and has received many awards for his work, the most recent of which was the Movement Disorders Research Award from the American Academy of Neurology. Dr. Langston serves on numerous editorial boards and advisory committees, and is also Chief Scientific Advisor for The Michael J. Fox Foundation for Parkinson's Disease Research. He is also a devoted father, soccer coach, and skier.


Mathias Baehr, MD

University Hospital Gottingen and University of Gottingen

Prof. Dr. M. Baehr is Director of the Department of Neurology at the University of Gottingen . He graduated from Tuebingen Medical School in 1985. 1987-1989 he was a postdoc in at the Washington-University St. Louis, Department of Anatomy and Neurobiology (Prof. R.P. Bunge). In recent years, he established neurobiological research groups at the Max-Planck-Institute and the Department of Neurology at the University of Tuebingen and Goettingen. Since 1998, Mathias Baehr was Vice-chairman of the Department of Neurology, University of Tuebingen. He was attending the Neurological University Hospital in Tuebingen until March 2001, when he became Director of the Department of Neurology at the University of Gottingen . In 2002 he founded the Research Institute for Multiple Sclerosis Research in Goettingen and was elected Head of this institute. In 2002, he also became member of the Board of the Center for Molcular Physiology of the Brain, Gottingen . Among other awards, he received the Attempto Award of the University of Tuebingen, the Award of the Kuratorium CNS und the Hannelore-Kohl-Foundation, the Research group award of the Ministeriums fuer Wissenschaft, Forschung und Kunst Baden-Wuerttemberg and the Heinrich-Pette-Award, German Society for Neurology. His Research interests focus on the field of neurodegeneration and regeneration.

Krystof Bankiewicz MD, PhD

Brain Tumor Research Center, University of California, San Francisco

Krystof Bankiewicz’s research interests are focused on solving the problems of delivery of advanced therapeutics to the brain. He is widely considered to be a leader in gene therapy, and has pioneered the use of advanced delivery devices, techniques and imaging tools in making it a safer and more effective technique. His therapeutic efforts are directed at gene therapy for Parkinson’s disease, but much of the technology that he has developed is also being applied by him and his collaborators to other serious neurological diseases. Dr. Bankiewicz earned his MD in 1983 in Cracow, Poland, at the Jagielonian University, and his PhD from the Institute of Neurology and Psychiatry in Warsaw in 1986. After a postdoctoral fellowship at the National Institute of Neurodegenerative Disease and Stroke (NINDS) at the National Institutes of Health from 1985 to 1990, he was appointed NINDS’s Chief, CNS Implantation Unit. From 1992 to 1997, he headed preclinical studies for Somatix Corporation in Alameda, California, before returning to NINDS as Acting Chief of the Molecular Therapeutics Section from 1997 until 2001. He then took up an appointment at the University of California, San Francisco (UCSF), as Associate Professor in the Department of Neurological Surgery, and was promoted to Professor in 2003. He also has held the post of Visiting Scientist at the Lawrence Berkeley National Laboratory’s Center for Functional Imaging since 2001. He is listed as a Special Expert in the NIH Grant Review Registry. From 1997 to 1999, Dr. Bankiewicz served on the Editorial Board of the Journal of Experimental Neurology.

Susan Bressman, MD

Beth Israel Medical Center and Albert Einstein College of Medicine

Susan Bressman is Chair of the Department of Neurology at Beth Israel Medical Center in New York City and Professor of Neurology at Albert Einstein College of Medicine. She attended Columbia University's College of Physicians and Surgeons and received her postgraduate training in neurology at the Columbia * Presbyterian Medical Center. After residency she was a Movement Disorders Fellow under Dr. Stanley Fahn and then remained at Columbia where she developed a genetics program in movement disorders, and also saw patients with Parkinson's disease and other movement disorders. While at Columbia, and since her move to Beth Israel in 1997, Dr. Bressman's research has involved many collaborations including investigators at Albert Einstein, Columbia, Harvard, Stanford, and North Shore. The focus has been to identify genes for dystonia and parkinsonism, to characterize genotype- phenotype relations, and to identify imaging and other endophenotypes. Susan B. Bressman, MD Chair, Dept of Neurology Beth Israel Medical Center Professor, Dept of Neurology Albert Einstein College of Medicine.

David Bumcrot, PhD

Alnylam Pharmaceuticals, Inc.

David Bumcrot, PhD, received his degree from the University of Pennsylvania and continued his training in the laboratory of Professor Andrew P. McMahon at Harvard University. With support from the Muscular Dystrophy Association and National Institutes of Health, Dr. Bumcrot investigated the activity of factors controlling the development of the central nervous system. Following his postdoctoral training, Dr. Bumcrot started his career in drug discovery and development by joining the staff of the biotechnology company, Curis (formerly Ontogeny). At Curis, Dr. Bumcrot led pre-clinical studies investigating the therapeutic potential of small molecules and antibodies that inhibit specific cell signaling pathways. This work led to the discovery of novel therapeutics currently being evaluated for cancer and neurological diseases. In 2002, Dr. Bumcrot joined Alnylam Pharmaceuticals as the fifth employee. Serving as Director of Molecular & Cell Biology, he is heading up several efforts to develop drugs based on recent breakthrough discoveries in RNA interference technology to treat cancer and neurodegenerative diseases, including Parkinson's disease.

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 serves on the Scientific Advisory Boards of Addex Pharmaceuticals, Invitrogen Life Technologies, Seaside Therapeutics, Cephalon Inc., AstraZeneca US , Hoffman La Roche, The Michael J. Fox Foundation, the Dystonia Medical Research Foundation and Eyeforpharma Advisory Board on CNS Drugs. He is 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.

Franz Hefti, PhD

Avid Radiopharmaceuticals, Inc.

Dr. Hefti has nearly 20 years experience in the pharmaceutical industry and over a decade experience in academia. Before joining Avid Radiopharmaceuticals, he led the drug development efforts at Rinat Neuroscience Corporation as Executive Vice President of Drug Development for three years. During his tenure, three antibody drug candidates advanced from the discovery stage to preclinical and clinical development and the success of these programs led to the acquisition of Rinat Neuroscience by Pfizer in 2006. Previously, Dr. Hefti was Senior Vice President of Neuroscience Research at Merck & Co., where he coordinated the company's neuroscience research worldwide, serving as site head for the neuroscience research centers in the U.K. and U.S. While working with Merck, small molecule drug candidates covering six drug targets in the neuroscience area were taken into development. Dr. Hefti was also Director of the Neuroscience Research Department at Genentech and previously spent more than a decade in academia as a Professor at the University of Southern California and Associate Professor at the University of Miami, where he carried out seminal research on therapeutic applications of neurotrophic factors. He has published over 250 papers on neurotrophic factors and topics in neuropharmacology, and a recent textbook "Drug Discovery for Nervous System Diseases" (John Wiley & Sons). Dr. Hefti received a Ph.D. from the University of Zurich and did his postdoctoral research at the Massachusetts Institute of Technology and the Max Planck Institute in Munich.

Robert Nussbaum, MD

University of California, San Francisco School of Medicine

Robert Nussbaum is the Lloyd (Holly) Smith Distinguished Professor of Medicine, Chief of the Division of Medical Genetics in the Department of Medicine at the University of California, San Francisco, and a member of the UCSF Institute of Human Genetics. His primary research focus is on the pathogenesis of Parkinson disease. His approach is to find the genes responsible for relatively rarer familial forms of the disease as a way of gaining insight into fundamental pathways leading to Parkinson's disease. These pathways then become targets of early diagnostic and therapeutic interventions. Dr. Nussbaum received his undergraduate degree in Applied Mathematics from Harvard College and his MD from Harvard Medical School in the Harvard-MIT Joint Program in Health Sciences and Technology. After residency training in Internal Medicine at Washington University/Barnes Hospital and Medical Genetics training at Baylor College of Medicine in Houston, he joined the Howard Hughes Medical Institute and the faculty of Human Genetics at the University of Pennsylvania where he was promoted to Associate and then Full Professor of Genetics, Pediatrics, and Medicine. He left the University of Pennsylvania in 1993 to help launch the new intramural research program of what is now the National Human Genome Research Institute. It is there that in 1996, he and his colleague Dr. Mihales Polymeropoulos mapped and identified the first hereditary form of Parkinson's disease, that caused by mutations in the gene encoding alpha-synuclein. He left the NHGRI in 2006 to move to UCSF. Dr. Nussbaum is a member of the Institute of Medicine and served as President of the American Society of Human Genetics in 2004.

Michael Schlossmacher, MD

Ottawa University

The goal of Dr. Schlossmacher's work as a clinician-scientist is to help improve the care of patients with Parkinson disease. Following residency training in general medicine from 1992 to 1995 in Vienna, Austria, he completed adult neurology training in the Harvard Longwood Neurology Program (1995-1999) and a clinical fellowship in the subspecialty of movement disorders at Massachusetts General Hospital and Brigham & Women's Hospital (BWH; 1999-2001). Until 2006, Dr. Schlossmacher was an independent investigator at the Center for Neurologic Diseases in the Department of Neurology at Brigham & Women's Hospital, and an Assistant Professor of Neurology at Harvard Medical School. Currently, he serves as a Scientist in the Ottawa Health Research Institute, a Neurologist at The Ottawa Hospital, an Associate Professor at the University of Ottawa, and Canada Research Chair in Parkinson Disease.

Andrew Singleton, PhD

National Institute on Aging/NIH

Andrew Singleton received his BSc from the University of Sunderland, UK, and his PhD from the University of Newcastle upon Tyne, UK. Dr. Singleton's research initially focused on genetic determinants of dementia, in particular Alzheimer's disease and dementia with Lewy bodies. His postdoctoral studies were spent at the Mayo Clinic in Jacksonville Florida. In 2001 he moved to the National Institute on Aging at NIH, Bethesda, Maryland, and became principal investigator leading the Molecular Genetics Unit in 2002. Dr. Singleton's laboratory studies the genetic basis of neurological disorders including Parkinson's disease. This research is performed using a variety of methodologies, including family based linkage and positional cloning in addition to genome-wide association. The goal of Dr. Singleton's research is to identify genetic variability that causes or contributes to Parkinson's disease in order to facilitate understanding of the molecular processes underlying disease.

Gretchen Snyder, PhD

Intra-Cellular Therapies, Inc.

Gretchen Snyder received her undergraduate degree magna cum laude in psychobiology from Mount Holyoke College. She earned a PhD at The University of Pittsburgh in Behavioral Neuroscience. Her doctoral work with Dr. Michael Zigmond helped to identify the neurochemical compensations that support behavioral function in animals sustaining sub-total dopamine loss after 6-OHDA lesions, a model of sub-clinical parkinsonism. In 1987 Dr. Snyder joined the Biogenic Amines Research Group at Abbott Laboratories, headed by Dr. John Kebabian, as a postdoctoral fellow. Her work at Abbott, and later as a research associate and assistant professor at The Rockefeller University with Dr. Paul Greengard, focused on the identification of biochemical pathways regulating dopamine neurotransmission mediated by the dopamine and cAMP-regulated phosphoprotein, called DARPP-32. Using DARPP-32 knockout mice she identified several neuronal proteins, including glutamate and GABA receptors, as targets for regulation by dopamine, through the regulation of DARPP-32. Dr. Snyder became associate professor in the Department of Neurobiology and Anatomy at Drexel University College of Medicine in 2001 and, in 2003, senior director of Neuropharmacology at Intra-Cellular Therapies, Inc., in New York City, a pharmaceutical company developing therapies for psychiatric diseases such as psychosis and neurodegenerative disorders including Parkinson’s disease and Alzheimer’s disease.

David G. Standaert MD, PhD

University of Alabama at Birmingham

David Standaert graduated from Harvard College in 1982. He received his MD and PhD degrees from Washington University in St. Louis and completed a one-year internship in Medicine at Jewish Hospital of St. Louis followed by a three-year Neurology residency at the University of Pennsylvania. He was appointed a Howard Hughes Medical Institute Physician Research Fellow, and completed a three-year research and clinical fellowship in Neurology (Movement Disorders) at Harvard Medical School and Massachusetts General Hospital in 1995. He subsequently joined the faculty at MGH where he served as Director of the MGH/MIT Udall Center of Excellence in PD Research as well as a Chair of the MGH Institutional Review Board (IRB). Dr. Standaert joined the University of Alabama at Birmingham faculty in July 2006. He serves as Director of the Division of Movement Disorders, Director of the APDA Advanced Center for Parkinson Research at UAB, and Director of the Center for Neurodegeneration and Experimental Therapeutics. He sees patients in a weekly clinic and oversees many clinical trials for new treatments of Parkinson's disease. Dr. Standaert’s laboratory works on understanding both the root causes of Parkinson’s disease as well as the origin of the disabling symptoms that appear after long term treatment of the disease. Recently, his group has focused on approaches to reducing the toxicity of synuclein in animal models of Parkinson disease. Dr. Standaert’s laboratory is also interested in the cause of “wearing off” and dyskinesias in patients treated with levodopa, and has uncovered evidence that may lead to new treatments for these disabling problems.

    Presented by

  • Michael J. Fox Foundation

Travel & Lodging

Our Location

The New York Academy of Sciences

7 World Trade Center
250 Greenwich Street, 40th floor
New York, NY 10007-2157

Click here for directions.

Hotels Near 7 World Trade Center

Recommended partner hotel:

A small number of rooms have been blocked for Conference participants at the New York Marriott Downtown (85 West Street at Albany Street, NYC). To secure a room at the reduced group rate of $229 + tax, please click here, and enter the Group Code pdtpdta when making your booking. Alternatively, you can call 1.800.242.8685 and request a room for the PD Therapeutics Conference.

Other hotels located near 7 WTC:

Embassy Suites Hotel


Millenium Hilton


Marriott Financial Center


Best Western Seaport Inn


Eurostars Wall Street Hotel


Wall Street District Hotel


Wall Street Inn


Ritz-Carlton New York, Battery Park