Grand Challenges in Parkinson's Disease

Dilan Athauda is an Honorary Consultant Neurologist at the Department of Clinical and Movement Neurosciences at The National Hospital for Neurology & Neurosurgery, and a Senior Clinical Lecturer at the Institute of Neurology, University College London. His primary interests involve neuroprotection in Parkinson’s disease, with an emphasis on how Type 2 diabetes and metabolic dysfunction influence neurodegeneration. His work involves utilising human-induced pluripotent stem cells to explore cellular pathways underlying neurodegeneration, and as a tool for drug discovery and biomarker evaluation. He is a co-investigator on the Exenatide-PD3 and Exenatide-MSA clinical trials, which are evaluating the use of exenatide, a Glucagon-like peptide-1 (GLP-1) agonist, as potential treatment for Parkinson’s disease and Multiple System Atrophy; and is a member of the Edmond J. Saffa ACT-PD working group – a project bringing together a range of experts to successfully produce a multi-arm, multi-stage clinical trial which would greatly advance current challenges in Parkinson’s research and deliver much-needed results to those in need.

Prof. Bandmann went to Medical School in Munich, Germany and did his PhD at the Institute of Neurology, Queen Square, London. He is now based at the Sheffield Institute for Translational Neuroscience (SITraN). His group undertook the first ever drug screen in genetically stratified Parkinson’s disease patient tissue. 2000 compounds were assessed for their rescue effect on mitochondrial function. Ursodeoxycholic acid (UDCA) was identified as a particularly promising compound. UDCA has already been in clinical use for > 30 yr and is therefore an ideal candidate for the drug-repositioning strategy. He subsequently took UDCA into a phase II, proof of concept clinical trial, the “UP Study” (UDCA in Parkinson’s disease, NCT03840005). The novel trial design included 31PMR-spectroscopy to confirm target engagement and sensor-based assessment of motor impairment to objectively quantify disease progression. 

Dr. Denise Barbut is a Professor of Neurology & Former Chief of the Neurovascular Division and of the Stroke research Program at Weill-Cornell Medical College, Cornell University. Her scientific interests have centered on neuroprotection and neurodegenerative disorders.  She trained as a neurologist in the UK and the USA, receiving her MD. from University College, London. Her post-graduate training included Imperial College, the Brompton, the National Hospital for Neurology and Neurosurgery and Cornell University. She is a Fellow of the Royal College of Physicians of the UK. Subsequently she became Professor of Neurology at Cornell University. She holds close to 200 patents and has published over 70 peer-reviewed articles. Her current research interests are focused on the role of aminosterols in the prevention and treatment of neurodegenerative diseases. She co-founded Enterin with Michael Zasloff in 2016, and currently serves as President, CMO, and Board Member. 

Lena Burbulla, PhD, serves as Heisenberg Professor for Metabolic Biochemistry of Neurodegenerative Diseases at the Biomedical Center, LMU Munich, since July 2021. She spent the past 9 years at MGH, Harvard Medical School, Boston, and Northwestern University, Chicago, where she conducted compelling research on pathologic mechanisms in Parkinson’s disease with a focus on dysfunctional dopamine metabolism and its contribution to diminished glucocerebrosidase function during her time as postdoctoral research fellow and Research Assistant Professor in the laboratory of Dimitri Krainc.  

The overarching goal of her own laboratory is now to further define the underlying determinants of vulnerability of dopaminergic neurons in the substantia nigra leading to midbrain neuron loss in PD and related disorders. Therefore, iPSC cultures are differentiated into midbrain dopaminergic neurons, but also astrocytes and microglia, to examine cell autonomous as well as the contribution of non-cell autonomous factors to neuron degeneration. Specifically, her lab is interested in the interactions between dopamine, iron, and oxidant stress with an emphasis on deciphering species-specific differences that may explain the incomplete recapitulation of human pathology in currently available animal models.  

Philippe Damier attended the University of Grenoble in France and, after completing his undergraduate studies in Biological Sciences, graduated from the Medical School in 1989. He was resident in Neurology at the University of Paris VI Pitié-Salpêtrière. He obtained a thesis in Neurosciences (PhD) in 1994 and spent a year as a postdoctoral fellow in Ann Graybiel’s lab (Boston, MA). Philippe became an assistant professor in Pitié-Salpêtrière hospital in 1995, before heading the Clinical Research Center in 1997 (under Yves Agid’s chair).

In 1999, he was appointed as a full professor of Neurology at the University of Nantes. Amongst Professor Damier’s responsibilities at the Medical School, University Hospital of Nantes, he chaired both the Department of Neurology and the Clinical Research Center. He is currently in charge of the expert center in Parkinson’s disease for the West part of France and president of scientific committee of the French Parkinson patient association.

He has published more than 250 scientific articles in international journals, most of them in the field of Parkinson’s disease and movement disorders.

In 2011, he got a Master in Business Administration in Melbourne (Australia). He published three books about managerial decision (2014, 2017, 2022).

Dr. Tim Greenamyre is the Love Family Professor and vice-chair of neurology and director of the Pittsburgh Institute for Neurodegenerative Diseases (PIND) and the American Parkinson Disease Association Advanced Center for Parkinson’s Disease Research at the University of Pittsburgh. He completed his MD, PhD and residency training in Neurology at the University of Michigan. He has been listed as one of the ‘Best Doctors in America’ since the mid-1990s. His laboratory studies mechanisms of neurodegeneration in Parkinson’s disease, with a focus on gene-environment interactions. Translational studies use pharmacological and ‘gene therapy’ approaches. He was Chair of the 2019 Parkinson’s Disease Gordon Research Conference. For 17 years he was editor-in-chief of the scientific journal Neurobiology of Disease, and he is a current member of the SAB of Science Translational Medicine.

Ashley S. Harms is an assistant professor at the University of Alabama at Birmingham (UAB) in the Department of Neurology and the Center for Neurodegeneration and Experimental Therapeutics. As a dedicated neuroimmunologist, Dr. Harms studies the cellular and immunological mechanisms underlying the initiation and progression of alpha-synucleinopathy disorders, with emphasis on antigen presentation and adaptive immunity. Utilizing novel genetic and viral model approaches to study the role of immune cell subsets, her lab focuses on how the protein alpha-synuclein contributes to microglial activation, peripheral immune cell infiltration, and subsequent activation of the immune response in Parkinson’s disease and multiple system atrophy. 

Ravi Jagasia, Ph.D., is a Neuroscientist, lab head and CNS drug discovery project leader in Neuroscience and Rare Disease Discovery in Roche Pharma and Early Development. Dr. Jagasia holds a Ph.D. from the Max-Planck Institute für Neurobiologie and LMU München, and Master ’s and Bachelor’s of Science from the University of Toronto Department of Physiology. His lab focuses on modelling neuronal development disorders, lysosomal storage disorders, Alzheimer’s and Parkinson’s using pluripotent stem cell derived neurons. Projects started in his lab in both rare diseases such as Angelman’s Syndrome or in Alzheimer are now in clinical trials.

Dr. Lang is Professor and previous Director of the Division of Neurology at the University of Toronto where he holds the Jack Clark Chair for Parkinson’s Disease Research. He is the Director of the Edmond J. Safra Program in Parkinson’s Disease, the Rossy Progressive Supranuclear Palsy Program and the Morton and Gloria Shulman Movement Disorders Clinic and holds the Lily Safra Chair in Movement Disorders at the Toronto Western Hospital, University Health Network. He has published over 900 peer-reviewed papers and is one of the most highly cited investigators in the field of Movement Disorders. Among his awards and distinctions he was appointed as an Officer of the Order of Canada in 2010; in 2011 he was elected a Fellow of both the Canadian Academy of Health Sciences and the Royal Society of Canada; in 2014 he was elected by the International Parkinson and Movement Disorder Society (MDS) as an Honorary Member “in recognition of his extraordinary contribution to the field of Movement Disorders”; and in 2017 he was the recipient of the first MDS Pan-American Section Leadership Award. In 2018 he received the Weston Brain Institute International Outstanding Achievement Award for work in accelerating the development of therapeutics for neurodegenerative diseases of aging and in 2020 he received the Dean’s Lifetime Achievement Award for global impact from the University of Toronto.

Prof. Meissner is Head of the Department of Neurology for Neurodegenerative Diseases at the University Hospital Bordeaux, Co-chair of the French Reference Center for MSA, and Deputy Director of the Institute for Neurodegenerative Diseases (CNRS UMR 5293) at the University Bordeaux. He received his medical degree in 1997 from Humboldt University Berlin in Germany and his Board Certification in Neurology in 2005 after completing his residency at the Charité University Hospital in Berlin and the University Hospital Bordeaux. In 2005, he was awarded a PhD in Neuroscience at the University Bordeaux. He was appointed Professor of Neurology at the University Bordeaux in 2012.

Prof. Meissner has served on several MDS Committees since 2013, including a leadership role as Chair of the International Education Committee between 2017-2019, and is the current MDS Treasurer-Elect. He has authored more than 190 peer-reviewed publications in the field of movement disorders, mostly dealing with PD and MSA, and has given over 125 invited lectures in more than 25 countries. His current research focuses on biological and clinical markers of disease progression in PD and MSA, and the development of new preclinical models and treatments for these disorders in a translational approach.

In his current research activities, Prof. Meissner chairs the MDS UMSARS Revision Task Force. He is co-coordinator of the LIXIPARK trial and has recently led a phase 1 trial with two vaccines directed against a-synuclein in MSA patients as part of the SYMPATH consortium. He has further coordinated the ARTEMIS consortium, which has evaluated the efficacy of distinct strategies targeting a-synuclein in preclinical models of MSA.

Dr. Narayanan is the Juanita J. Bartlett professor of Neurology Research and Vice Chair for Basic and Translational Research in the Department of Neurology at the Carver College of Medicine in the University of Iowa.  He also is an Associate Professor an Associate Director of the Iowa Neuroscience Institute, and Associate Director of the Clinical Neuroscience Training Program.

He is originally from Seattle, Washington.  He received BA from Stanford University and received an MD and PhD from Yale Medical School, where he also completed a residency in neurology. He came to the University of Iowa in 2012 to launch his lab studying the basic mechanisms of prefrontal dopamine. He leads a multidisciplinary clinic focused on Parkinson’s disease.

He received the Donald B. Lindsley Prize for the best dissertation from the Society for Neuroscience, the S. Weir Mitchell Award for best residency research from the American Academy of Neurology, and the Jon Stolk Award for movement disorders research from the American Academy of Neurology.

Prof. Tiago Outeiro graduated in Biochemistry at the University of Porto and was an Erasmus student at the University of Leeds in the UK. Prof. Outeiro did his PhD thesis at the Whitehead Institute for Biomedical research – MIT and worked as a Research Scientist at FoldRx Pharmaceuticals as a Research Scientist and Consultant. Prof. Outeiro was a Postdoctoral Research Fellow in the Department of Neurology of the Massachusetts General Hospital – Harvard Medical School where he focused on the study of Neurodegenerative disorders such as Parkinson’s and Alzheimer’s disease. Prof. Outeiro directed the Cell and Molecular Neuroscience Unit at IMM, Lisbon, from 2007 to 2014, and is currently Full Professor and the Director of the Department of Experimental Neurodegeneration at the University Medical Center Goettingen, in Germany. Prof. Outeiro holds a joint Professor position at Newcastle University in the UK. Prof. Outeiro has authored >250 research articles in international journals and participates in various international boards and in collaborative projects with the aim of identifying the molecular basis of neurodegenerative disorders such as Alzheimer’s and Parkinson’s disease. He has been awarded multiple prizes and grants in Germany, from the European Union, and from other international funding agencies. Currently, Prof. Outeiro is a visiting Professor at UFRJ.

Dr Judith Peterschmitt is the Global Project Head for Parkinson’s disease in Neurology Development at Sanofi; prior to that she served as the Clinical Lead of the Rare Disease Therapeutic Area at Sanofi Genzyme.

Dr. Peterschmitt has over 18 years of experience in clinical development. She has worked in multiple programs, including venglustat for GBA-Parkinson’s disease and Gaucher disease type 3, Cerdelga®, an oral GCS inhibitor for Gaucher disease type 1, and enzyme replacement therapies for lysosomal storage disorders: Olipudase alpha, in development for acid sphingomyelinase deficiency, Myozyme® (alglucosidase alfa) and Aldurazyme® (laronidase) for Pompe disease and mucopolysaccharidosis type I respectively.

Prior to joining Genzyme in 2004, Dr. Peterschmitt was a full-time geneticist and the Medical Director of the Center for Treatment of Lysosomal Storage Diseases, and of the Newborn Screening Program at Hospital Nacional Posadas, in Buenos Aires, Argentina.

Dr. Peterschmitt gained her MD at the National University of Buenos Aires, Argentina and a Masters in Medical Sciences (Clinical Investigation) from Harvard–MIT Division of Health Sciences and Technology, MA, USA. She completed her training in pediatric and child neurology in Buenos Aires, newborn screening in Japan, and clinical and biochemical genetics at Boston Children’s Hospital, MA, USA.

Dr. Simuni joined the faculty of the Northwestern University Feinberg School of Medicine in 2000 to build a multidisciplinary movement disorders center that is recognized by the Parkinson’s Foundation, Huntington Disease Society of America and Wilson’s Foundation as a Center of Excellence and serves as a training model in the region. She is the lead investigator of a number of clinical trials on experimental pharmacology, non-motor manifestations, and pharmacological management of PD. She serves on a number of Steering Committees for the PD national clinical trials, several committees for PSG and the PF. She is the Site PI and Steering Committee member for the largest PD biomarker initiative (PPMI study) and site PI for the Network for Excellence in Neuroscience Clinical Trials (NEXT). Dr. Simuni is an active member of the American Academy of Neurology, American Neurological Association, the Movement Disorders Society as well as the Parkinson’s Study Group

Günther Staffler is a biotech industry executive and multidisciplinary researcher with more than 25 years’ experience in bio-chemical and immunology research and in the establishment and management of drug development programs. He has extensive leadership and strategic oversight experience in early discovery to early clinical phase drug development phases with strong involvement in manufacturing operations. Before joining AC Immune, he was Chief Technology Officer at AFFiRiS AG where he led the development of different active immunotherapies for the treatment of non-communicable chronic diseases, including Parkinson’s disease and Hypercholesterolemia. Günther completed his degree in biochemistry and immunology at the University of Vienna. Apart from his research work at the Institute for Immunology he has been lecturer at the University of Vienna and at the University of Applied Sciences, FH Krems & Vienna.

Dr. Standaert graduated from Harvard College and received M.D. and Ph.D. degrees from Washington University in St. Louis. Following Neurology residency at the University of Pennsylvania, he was appointed a Howard Hughes Fellow and completed a three-year research and clinical fellowship in Movement Disorders at Massachusetts General Hospital. He was a member of the faculty at Harvard Medical School from 1995 to 2006 and then relocated to the University of Alabama at Birmingham (UAB). Currently he is the John N. Whitaker Professor and Chair of the Department of Neurology and a senior member of the faculty of the Division of Movement Disorders. He directs the NIH-funded Alabama Morris K. Udall Center of Excellence in Parkinson’s Disease Research. He is Chairman of the Scientific Advisory Board of the American Parkinson Disease Association, a Deputy Editor of the journal Movement Disorders, a Fellow of both the American Neurological Association and the American Academy of Neurology, a Councilor of the Association of University Professors of Neurology, and a member of the NIH/NINDS Board of Scientific Counselors. His lab has a long-standing interest in the basic mechanisms underlying Parkinson disease as well as the complications of therapy. 

Dr. Michele Tagliati is a clinician and clinical researcher, investigating advanced therapeutics of Parkinson’s disease, dystonia and other movement disorders. He was among the pioneersdeveloping the use ofdeep brain stimulation (DBS) for movement disorders in the United States. Dr. Tagliati attended medical school and neurology residency in Rome, Italybefore moving to New York in 1993. He completed a second residency in neurology at the Mount Sinai School of Medicine, during which time he was Chief resident. Following residency, he completed a fellowship in Movement Disorders at Beth Israel Medical Center and afterward joined the Departmentof Neurologyas an attending and faculty at Albert Einstein College of Medicine. In 2004, Dr. Tagliati moved back to MountSinai, where he was anAssociate Professor of Neurology and Division Chief of Movement Disorders. In 2010, accepted the position of Vice Chairman of Neurology and Director of Movement Disorders at Cedars Sinai Medical Centerin Los Angeles. In2015, he was awarded the Caron and Steven D. Broidy Endowed Chair for Movement Disorders.Dr. Tagliatihas received research grants and support in excess of 4 million dollars fromvarious agencies and foundations and has been a Principal Investigator in over 45 clinical trials exploring new medical and surgical therapies for Parkinson’s disease and dystonia. Currently, his research is directed at the study of non-motor and pre-motor symptoms of Parkinson’s disease, including investigator-initiated trials of liraglutide in Parkinson’s disease and adrenergic blockers in subjects at high risk of developing PD. Dr. Tagliati holds 2 international patents on subjects related to Parkinson prevention and DBS optimization.Dr. Tagliati is a diplomate of the American Board of Psychiatry and Neurology.He has published over 120 peer-reviewed articles and 30 book chapters. In addition, heis co-author of the patient reference book Parkinson’s Disease for Dummies.Dr. Tagliati has lectured at conferences and academic institutions around the world and is one of the premier educators of DBS management, having directed –among others -theDBS programming course at the annual meeting of the American Academy of Neurology. An active member of the American Academy of Neurology and the Movement Disorders Society, Dr. Tagliati also serves in theEditorial Boardof the Journal of Parkinson’s Diseaseand Nature Parkinson’s disease.

Pete Thornton is currently Director of Translational Science at NodThera Inc., a biotech focused on the discovery and development of NLRP3 inhibitors for the treatment of inflammatory and neuroinflammatory disease. Previously, he has worked on multiple anti-inflammatory drug projects at GSK, Biogen-Idec, MedImmune, and AstraZeneca. For the most part, Pete has supported early and late discovery projects in the neurodegeneration and pain therapy areas, gaining a wealth of experience in leading small and large molecule projects from target validation, through lead optimisation to first time in human studies. Pete holds a Batchelor of Science in Pharmacology from the University of Bristol and a Ph.D. in Neuroinflammation from the University of Manchester. He has published widely in the field of neuroinflammation with particular emphasis on glial and cerebrovascular inflammatory mechanisms in disease.

Dr. Milton Werner is the President and Chief Executive Officer of Inhibikase Therapeutics, a company developing novel protein kinase inhibitor therapeutics to treat neurodegenerative disease and viral infection inside and outside of the brain. Previously, Dr. Werner served as Vice President of Research at Celtaxsys, a cell-free immunotherapeutics company. From September 1996 until June 2007, Dr. Werner was a Head of the Laboratory of Molecular Biophysics at The Rockefeller University in New York City. Throughout his scientific career, Dr. Werner has been an innovator integrating chemistry, physics, and biology into a comprehensive approach to solving problems in medicine, including an explanation of the origin of “maleness” in humans, the mechanistic basis of several forms of leukemia and lymphoma and, more recently, the development of therapeutics that can halt and potentially reverse functional loss in neurodegenerative disease. 

Dr. Werner is the author or co-author of more than 70 research articles, reviews, and book chapters and has given lectures on his research work throughout the world. He is the recipient of numerous private and public research grants totaling more than $30 million. He is the recipient of several awards, including the Naito Memorial Foundation Prize, the Young Investigator Award from the Sidney Kimmel Cancer Foundation, the Research Chair from the Brain Tumor Society, and a $1 million Distinguished Young Scholars in Medical Research Award from the W. M. Keck Foundation. Dr. Werner received his Doctor of Philosophy in Chemistry from the University of California, Berkeley, and his Bachelor of Science in Biochemistry from the University of Southern California, and he was an NIH intramural postdoctoral fellow prior to his tenure at the Rockefeller University.