Dr. Darryl C. De Vivo, Dr. Claudia Chiriboga (PI), and the Spinal Muscular Atrophy (SMA) Clinical Research Center team achieved a major milestone this week, conducting the first-ever clinical trial in a human with SMA. The study involves intrathecally dosing SMA patients with a promising antisense oligonucleotide therapeutic that has been found, preclinically, to modify this devastating pediatric disease by altering the splicing defect in the SMN2 gene. Columbia is the first of four sites to conduct this trial, and is currently the only site that is actively recruiting. Dr. Chiriboga plans to dose five more SMA patients in the coming months.
A landmark natural history study led by Drs. Darryl C. De Vivo, Salvatore DiMauro, Petra Kaufmann, and NI colleagues, shows a measurable decline in clinical and imaging outcomes for patients with MELAS carrying the mitochondrial DNA m.3243A>G mutation. [read more]
Dr. Elan D. Louis
P&S FIVE IN FIVE SERIES
Elan Louis, MD, MS, Professor of Neurology and Epidemiology answers questions on "the essentials of essential tremor" in the most recent P&S Five in Five series. [watch the video]
Dr. Edward HueyJill Goldman, MS, MPhil
CME PROGRAM
Edward Huey, MD, Assistant Professor of Psychiatry and Neurology and Jill Goldman, MS, MPhil, Genetic Counselor, both in the Taub Institute, are co-directing a CME program, "Is it Alzheimer's Disease or Frontotemporal Degeneration? An Update on Diagnosis, Management, and Research," on Monday, December 12, 12:15-4:45 PM, in the NI Auditorium. [register here]
Dr. David Sulzer
JPB FOUNDATION GRANTS
David Sulzer, PhD, Professor of Neurobiology in Neurology, Pharmacology and Psychiatry was awarded two new grants from the JPB Foundation (formerly The Picower Foundation). Both projects, "Mechanisms of Substantia Nigra Neuronal Death in Parkinson's Disease" and "Mechanisms of Parkinson's Disease Pathogenesis" seek an explanation as to why substantia nigra (SN) neurons die in Parkinson's disease and why this process increases with age, to provide insight toward developing clinical approaches.
Dr. Scott A. Small
Scott Small, MD, Professor of Neurology in the Taub Institute and Sergievsky Center and colleagues have reviewed recent neuroimaging findings that indicate common brain disorders, from Alzheimer's disease and cognitive ageing to schizophrenia and depression, differentially target distinct functional and molecular subregions of the hippocampus, suggesting a unified pathophysiological framework of hippocampal dysfunction. [read more]
Karen Marder, MD, MPH, Sally Kerlin Professor of Neurology and Psychiatry was appointed as one of three site investigators to serve on the Executive Committee of the NINDS NeuroNEXT project. The NeuroNEXT network of 25 sites nationwide, including Columbia in partnership with Weill-Cornell, will provide a standardized, accessible infrastructure to facilitate rapid development and implementation of protocols in adult and pediatric neurological disorders.
Dr. Hiroshi Mitsumoto
MDA'S WINGS OVER WALL STREET GALA
The MDA's Wings Over Wall Street gala event raised over $500,000, some of which will be used by the Eleanor and Lou Gehrig MDA/ALS Center for Dr. Hiroshi Mitsumoto's research.
Dr. Hiroshi Mitsumoto
INTERNATIONAL ALS CONFERENCE
Dr. Mitsumoto, Wesley J. Howe Professor of Neurology, led the 2nd International ALS Conference at the Tarrytown HouseEstate & Conference Center in Tarrytown on September 6th – 9th. This unique, discussion–based conference brought together 150 of the world's greatest minds in ALS to promote clinical, patient-oriented research into pathogenesis and causes of ALS. Dr. Mitsumoto plans to follow–up this accomplishment with a supplement in the Annals of Neurology.
Dr. Salvatore DiMauro
UNIVERSITY OF PADOVA (PADUA)
Dr. DiMauro, Lucy G. Moses Professor of Neurology and Director Emeritus of the H. Houston Merritt Clinical Research Center, has been invited by the University of Padova (Padua), one of the oldest and most prestigious medical schools in the world, to present a special Morgagni Lecture on "Mitochondrial Medicine" this Saturday, September 17th. The occasion of this special lecture series is the 300th Anniversary of Giovanni Battista Morgagni's inaugural speech at Padua, and the 250th Anniversary of the publication of his seminal textbook, "De Sedibus," which established Morgagni as the founder of anatomical pathology. Congratulations on this distinguished honor.
Dr. Neil Shneider, Assistant Professor of Neurology, has co-authored a study led by Brian McCabe, PhD, Assistant Professor of Physiology & Cellular Biophysics and Neuroscience, which demonstrates that two genes associated with familial ALS function together to support the longevity of motor neurons. [read more]
U.S. Department of Health and Human Services
Dr. Jennifer Manly, Associate Professor of Neuropsychology in Neurology and the Taub Institute has been invited to serve on the U.S. Department of Health and Human Services non-federal Advisory Council on Alzheimer's Research, Care, and Services, as part of the National Alzheimer's Project Act. [read more]
Dr. Serge Przedborski and Dr. Eric Schon's recent Neuron article entitled, "Mitochondria: The Next (Neurode) Generation," suggests a new explanation for the role of mitochondria in neurodegenerative disease. [read more]
