Featured Plenary Speakers
Frank Bennett, PhD
Ionis Pharmaceuticals, Inc.
Arthur K. Asbury Lecture: Antisense Therapies for Neurological Diseases
I am currently the head of research and executive officer at Ionis Pharmaceuticals (formerly Isis Pharmaceuticals), a biotechnology company focused on RNA targeting therapeutics. For the past 30 years I have served in various research and development positions at Ionis. I have responsibility for our drug discovery research program, core technology projects, medicinal chemistry, research oligonucleotide synthesis and have in the past managed our GMP manufacturing facility. I have help lead the identification of >40 drugs that have entered clinical trials, including five marketed products. Thus, I have broad experience in all aspects of antisense technology, oligonucleotide medicinal chemistry and drug development. In addition to leading the research efforts in the company I also am responsible for leading both the research and development activities for our Neurological Disease Franchise. I have led the discovery and development of our antisense drug for spinal muscular atrophy (Spinraza, nusinersen) which was recently approved by the FDA. Other drugs that I have led the discovery and the development of include antisense drugs for familial and sporadic ALS, Huntington’s disease, Alzheimer’s disease, Parkinson’s disease and myotonic dystrophy. We have over 25 active drug discovery programs to identify drugs for a variety of neurological diseases. I obtained my undergraduate degree in Pharmacy from the University of New Mexico and my doctorate degree in Pharmacology from Baylor College of Medicine. As part of my Ph.D., I studied protein and RNA components in nuclear substructures such as the nucleolus and nuclear speckles. I am the recipient of the 2019 Breakthrough Prize in Life Sciences for the discovery and development of nusinersen and the 2018 Leslie Gehry Brenner Prize awarded by the Hereditary Disease Foundation for the discovery of the first antisense drug to enter development for Huntington’s Disease.
Michael Caterina, MD, PhD
Johns Hopkins School of Medicine
Peter J. Dyck Lecture: Mechanisms of Cutaneous Pain Sensation
Dr. Michael J. Caterina is a professor of neurosurgery, biological chemistry and neuroscience at the Johns Hopkins University School of Medicine and Director of the Neurosurgery Pain Research Institute at Johns Hopkins. Dr. Caterina earned his bachelor’s degree from Pennsylvania State University and subsequently the M.D. and Ph.D. degrees from the Johns Hopkins School of Medicine. He completed a fellowship in cellular and molecular pharmacology at the University of California, San Francisco. Dr. Caterina joined the Johns Hopkins faculty in 1999.Current topics of interest in his lab at Johns Hopkins include: mechanisms underlying pain in hereditary skin diseases and cellular and molecular mechanisms of neuropathic pain, and contributions of transient receptor potential channels to pain.
Aaron DiAntonio, MD, PhD
Washington University School of Medicine
Jack Griffin Lecture: The Molecular Mechanism of Axon Degeneration: SARM1 as a Druggable Target for the Prevention of Peripheral Neuropathy
Dr. Aaron DiAntonio is the Alan A. and Edith L. Wolff Professor of Developmental Biology and the Co-Director of the Needleman Center for Neurometabolism and Axonal Therapeutics at the Washington University School of Medicine in St. Louis. His laboratory combines genetics, cell biology, biochemistry, and physiology in both Drosophila and mouse to investigate molecular mechanisms that control the formation, maintenance, and repair of neural circuits in development and disease. His laboratory is now translating these fundamental mechanistic insights into novel therapies for the treatment of neurological disease.
Laura Feltri, MD
SUNY at Buffalo
Richard Bunge Lecture: Signals to Schwann Cells During Myelination and Peripheral Neuropathies
Dr. Feltri is Acting Director of the Hunter James Kelly Research Institute and Professor of Biochemistry and Neurology at the Jacobs School of Medicine and Biomedical Sciences at the State University of New York at Buffalo. Dr. Feltri received her medical degree from the University of Milano, Italy. She completed her internship in Medicine and Residency in Neurology in the San Raffaele Hospital, University of Milano, Italy. She was a Neuroscience post-doctoral fellow at Thomas Jefferson University and at the University of Pennsylvania. Dr. Feltri worked from 1993 to 2011 at the San Raffaele Scientific Institute of Milano, Italy, where she was the Head of the Unit of Neuro-Glia. Dr. Feltri’s scientific interest is on myelin and myelin diseases. Together, with the laboratory of Lawrence Wrabetz, she developed the first Cre transgene that targets specifically Schwann cells, and pioneered conditional transgenesis to understand the role of extracellular matrix components and their receptors in developing and pathological peripheral nerves. Her laboratory has identified the diverse roles and many of the downstream signals for laminin receptors in nerve development. The NIH, National Multiple Sclerosis Society, Hunter Hope Foundation, Charcot-Marie-tooth Association, Telethon Italy and the European Community have funded her research and she has authored more than 100 peer-reviewed publications. Dr. Feltri has mentored many graduate and MD-PhD students with several of them achieving independent faculty and other leadership positions, and has served the clinical and scientific community on the Board of the Peripheral Nerve Society, the medical board of the Charcot-Marie-Tooth Association and as member of the National Multiple Sclerosis Study Sessions. Dr. Feltri is the current Chair of the Cellular and Molecular Biology of Glia at the NIH and she served as Editorial member of the Journal of Neuroscience, Glia and Experimental Neurology. At SUNY Buffalo, Dr. Feltri was awarded an Exceptional Scholar Award for Sustained Achievements, the SUNY Chancellor’s Award for Excellence in Scholarship and Creative Activities and the Distinguished Post-doc Mentor Award.
Michio Hirano, MD
Pembroke Lecture: Primary Mitochondrial Neuropathies
Dr. Michio Hirano received his undergraduate degree from Harvard College and his Medical Degree from the Albert Einstein College of Medicine. After completing neurology residency at the Neurological Institute of New York, he did a post-doctoral fellowship in neuromuscular genetics under Drs. Salvatore (Billi) DiMauro and Eric Schon at the H. Houston Merritt Neuromuscular Disease Research Center. He joined the faculty of Neurology at Columbia University Vagelos College of Physicians and Surgeons, where he is currently Professor of Neurology, Chief of the Division of Neuromuscular Medicine, and Director of the Merritt Center. For more than 25 years, Dr. Hirano’s translational research focused on mitochondrial disease and inherited myopathies. His laboratory has identified the causative genes for more than a dozen inherited diseases including: mitochondrial neurogastrointestinal encephalomyopathy (MNGIE), LAMP-2 deficiency (Danon disease), and X-linked scapuloperoneal myopathy. To understand how some of these mutant genes cause diseases, the laboratory has been investigating in vitro and mouse models. He has been developing therapies for mitochondrial diseases including deoxynucleoside treatment for thymidine kinase 2 deficiency, which is currently being assessed in clinical trials. Since 2009, Dr. Hirano has been the principal investigator of the NIH-funded North American Mitochondrial Disease Consortium (NAMDC).
Rebecca Schule, MD
University of Tübingen, Hertie Institute for Clinical Brain Research & German Center for Neurodegenerative Diseases (DZNE)
PK Thomas Lecture: Genetics of Hereditary Spastic Paraplegia – Overlap Between Central and Peripheral Axonopathies
Rebecca Schüle is a neurologist and neuroscientist at the University of Tübingen, Germany. She is the coordinator of the TreatHSP.net consortium, a translational network aiming to accelerate development and implementation of novel therapeutic options for HSPs. She is engaged in national and international initiatives for a joint, sustainable, harmonized and standardized capture of longitudinal clinical data for HSPs (HSP Registry) and leads and participates in multiple efforts to develop and validate clinical, molecular and digital biomarkers as outcome parameters for clinical trials. Rebecca Schüle is one of the leading HSP geneticists, initiator of the international HSP OMICS repository and HSP Biobank and models HSP pathophysiology in human stem cell derived model systems.