2018 Conference

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James Goldman, MD, PhD
Columbia University

James Goldman, MD, PhDJames Goldman, MD, PhD, has been on the neuropathology staff at Columbia University for more than 30 years where he is a professor of pathology and cell biology. He received an MD and PhD from the New York University School of Medicine and then did his residency training in neuropathology at the Albert Einstein College of Medicine. Goldman participates in clinical neuropathology and also runs a research laboratory, in which he has pursued an interest in astrocyte biology and myelin disorders for many years. He has investigated the neuropathology of Alexander disease as well as the mechanisms by which the accumulation of mutant GFAP in Alexander’s alters the forms and functions of astrocytes. Goldman is currently interested in how Alexander astrocytes promote an inflammatory environment in the central nervous system.

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Fly Models of Disease

Mel Feany, MD, PhD
Harvard Medical School

Mel Feany, MD, PhDMel Feany, MD, PhD, is a professor of pathology at Harvard Medical School and Brigham and Women’s Hospital. She pioneered modeling of human neurological diseases in the powerful genetic model organism Drosophila. Her laboratory currently has a major focus on understanding how glial cells control neuronal function and viability in Alexander disease and related neurological disorders. In addition to uncovering new molecules and mechanisms using unbiased approaches in Drosophila, work in the Feany laboratory is highly collaborative and integrates data from human cells and tissue, and rat and mouse models to identify core pathways that are dysregulated in Alexander disease. These pathways represent attractive therapeutic targets in patients with Alexander disease, and possibly also other neurological disorders in which glial cells are abnormal.

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Rat Model of Disease

Tracy L. Hagemann, PhDTracy Hagemann, PhD
University of Wisconsin-Madison

Tracy Hagemann, PhD, is a senior scientist at the Waisman Center, University of Wisconsin-Madison. She received her PhD from Rush University in Chicago. Hagemann works to better understand astrocyte function in neurodegenerative disease. Her research focuses on Alexander disease as one of the few examples of a specific disorder of astrocytes. Hagemann has generated several rodent models with GFAP mutations to mimic those found in patients with the disease, allowing her to study the effects of astrocyte dysfunction on other cell types in the central nervous system. She is currently using these same models to test suppression of GFAP as a strategy to alleviate disease symptoms in mice and rats and eventually for treating patients with the disorder.

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Protein Clearance from the Brain

Maiken Nedergaard, M.D., D.M.Sc.Maiken Nedergaard, MD, PhD
University of Rochester

Maiken Nedergaard, MD, DMSc, is a professor of neurosurgery and the co-director of the Center for Translational Neuromedicine at the University of Rochester Medical Center (URMC) in Rochester, NY. She is also a professor of glial cell biology and co-director of the Center for Translational Neuroscience at the University of Copenhagen, Denmark. Her multiple interests range from basic research on neuron-glia interactions to their role in aging, small vessel disease, seizure disorders and cerebral blood flow. Foremost amongst her discoveries is the identification of the glymphatic system, a brain equivalent of the lymphatic system within which cerebrospinal fluid diffuses rapidly and mixes with interstitial fluids, thereby filtering metabolic byproducts that accumulate due to neuronal activity. She published a landmark study in Science showing that the glymphatic system dramatically expands during sleep compared to waking – brain cleaning and detoxification is thus greatly facilitated during sleep, providing a novel and direct explanation for what we all generally consider sleep’s restorative effect.

Chemical Modifications of GFAP during AxD

Natasha Snider, PhDNatasha Snider, PhD
University of North Carolina Chapel Hill

Natasha Snider, PhD, is an assistant professor of cell biology and physiology at the University of North Carolina-Chapel Hill. As a graduate student with Paul Hollenberg in the Department of Pharmacology at the University of Michigan (2004-2009), she studied cytochrome P450-mediated pathways of endocannabinoid metabolism. Snider was an NIDDK-funded post-doctoral fellow (2009-2011) and research investigator (2011-2014) with Bishr Omary in the Department of Molecular & Integrative Physiology at the University of Michigan, studying keratin intermediate filaments and mechanisms of liver injury. Snider started her independent research program at the University of North Carolina-Chapel Hill in January 2015. She is also a full member of the Center for Gastrointestinal Biology and Disease (CGIBD).

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Induced Pluripotent Stem Cells

Su-Chun Zhang, MD, PhD
Waisman Center 5/30/14
(Photo @ Andy Manis)

Su-Chun Zhang, MD, PhD
University of Wisconsin-Madison

Su-Chun Zhang, MD, PhD, is a professor of neuroscience and neurology at the University of Wisconsin-Madison and a Waisman Center investigator. Zhang received his MD from Wenzhou Medical College in China and his PhD from the University of Saskatchewan. In 2005, Zhang demonstrated that stem cells can be coaxed into becoming spinal motor neurons that relay messages from the brain to the body, and midbrain dopamine neurons that coordinate movement. Zhang continues to develop stem cell-based therapies to protect and repair diseased and damaged neurons, including transforming adult stem cells into early-stage nerve cells bypassing the pluripotent stem cell stage. Eventually this process could produce cells used to treat spinal cord injury and to screen drugs to treat neurodegenerative diseases.

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Antisense Oligonucleotide Therapy

Anne Smith, PhDAnne Smith, PhD
Ionis Pharmaceuticals

Anne Smith, PhD, is an executive director of clinical development at Ionis Pharmaceuticals, a California-based biotechnology company and world leader in RNA-targeted drug discovery and development. She obtained her PhD in chemical engineering from North Carolina State University where she developed computational techniques for simulations of protein folding and aggregation. Smith has worked in the biotechnology industry for more than 15 years and joined Ionis in 2014 to focus on developing treatments for neurological conditions. While at Ionis, she led development of an investigational drug targeting the Huntingtin protein as a possible disease-modifying treatment for Huntington’s disease, including successful demonstration of proof-of-concept in a clinical trial last year. Smith recently joined Ionis’ Alexander disease research and development team and is looking forward to working with the community to develop potential treatments for Alexander disease.

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Planning for Clinical Trials

Amy WaldenAmy Waldman, MD
Children’s Hospital of Philadelphia

Amy T. Waldman, MD, MSCE, is an assistant professor of neurology at the Perelman School of Medicine at the University of Pennsylvania (Penn). She received her medical doctorate from Jefferson Medical College, Thomas Jefferson University, and completed her pediatrics residency at the Children’s Hospital of Philadelphia (CHOP) and child neurology residency at both CHOP and the Hospital of the University of Pennsylvania. Waldman completed a fellowship in pediatric and adult MS at CHOP and the Hospital of the University of Pennsylvania. During her fellowship, she obtained a Master of Science in clinical epidemiology degree at Penn. In 2005, she co-founded the Pediatric MS Center, and in 2014, the Leukodystrophy Center of Excellence, both at CHOP where she is the medical director. Waldman is currently extending her expertise in clinical outcome measures to leukodystrophies, with the goal of assisting in clinical trial design and interpretation for these disorders. She is leading a natural history study of Alexander disease, including collecting data on performance-based outcome measures in affected individuals.

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