Scientific Advisory Board

The HDSA Scientific Advisory Board (SAB) is comprised of leading experts in their fields. The Scientific Advisory Board's role is to advise the Board of Trustees and HDSA Management on a range of issues. In general, the SAB provides scientific review of research proposals to ensure that the research programs at HDSA are scientifi­cally sound, pertinent and provide a high impact to the HD research community.

The SAB can be called upon to advise HDSA on any scientific issues that may arise (e.g., stem cell policy, use of animals in research).

The Committee’s specific responsibilities include:

  • Periodically reviewing HDSA's medical and scientific affairs strategy and recommending funding for research grant awards.
  • Significantly expanding HDSA’s research commitments.
  • Define and administer HDSA’s research program, including RFP development, proposal review and grant oversight.
  • Providing speakers and research programs for major HDSA events i.e., the annual HDSA Convention.


Louise Vetter, HDSA’s Chief Executive Officer (left) and Dr. George Yohrling, Director of Medical and Scientific Affairs (sixth from left) welcome members of the Scientific Advisory Board to HDSA’s national headquarters.

Michelle Gray , Ph.D.

Scientific Advisory Board Chair

Michelle Gray , Ph.D.

Dr. Michelle Gray is currently an Assistant Professor in the Department of Neurology at the University of Alabama at Birmingham. She is the Dixon Scholar in Neuroscience, focused on the study of Huntington’s Disease. Dr. Gray earned her Ph.D. from the Ohio State University and performed post-doctoral training at the University of California, Los Angeles.

 

Dr. Gray is a laboratory scientist who has worked on Huntington’s disease (HD) for the last 10 years. She has been an invited speaker and presenter of her work at multiple Huntington’s Disease focused meetings including those sponsored by the Hereditary Disease Foundation and CHDI,Inc. In addition, she presents updates on Huntington’s disease to the Alabama Academy of Neurology and participates in Huntington’s Disease Education Day sponsored by the HDSA Center for Excellence at the University of Alabama at Birmingham.

 

Currently her work focuses on understanding the overall contribution of glial cells, which constitute about 50% of the cells in the brain, to Huntington’s disease. This includes understanding the normal function of the huntingtin protein in glial cells and determining how the mutant protein in glial cells contributes to neuronal dysfunction in HD.

Neil Aronin, M.D.


Professor of Medicine and Cell Biology
 Chief, Endocrinology and Metabolism
 Co-Director, Neuro-Therapeutics Institute
 University of Massachusetts Medical School

Neil Aronin, M.D.


Dr. Aronin’s research focuses on developing gene silencing for therapy in Huntington’s disease, an autosomal dominant neurodegenerative disease. The laboratory uses allele selective RNAi to silence the mutant huntingtin and preserve wild type huntingtin.  The research team explores potential therapies and neuronal pathophysiology attributable to the mutant huntingtin. The team also studies viral delivery of allele selective shRNAmir, with single stranded and self-complementary adeno-associated virus. Dr. Aronin’s team studies next-generation therapeutics that will be applicable to a spectrum of autosomal dominant diseases, as well as studying mechanisms of pathogenesis in Huntington’s disease: mutant huntingtin mRNA kinetics, vesicle recycling and roles of proteins that interact with mutant huntingtin.

Beth Borowsky, Ph.D
.

Director of Translational Medicine
 Clinical Research
 CHDI Management/CHDI Foundation

Beth Borowsky, Ph.D
.

Beth Borowsky is the Director of Translational Medicine at CHDI Foundation.  She manages a portfolio of projects aimed at identifying biomarkers and endpoints for use in clinical trials in Huntington’s disease. Projects she has led include TRACK-HD, TRACK-On, the CHDI Cognitive Assessment Battery Beta and numerous imaging and wet biomarker studies.  Dr. Borowsky is a member of the FNIH Biomarker Consortium, and the Common Data Elements biomarker working group for Huntington’s disease. Prior to joining CHDI in 2007, Beth spent 13 years in CNS drug discovery and development with sanofi-aventis and Synaptic Pharmaceuticals (now Lundbeck), where she held positions of increasing responsibility. As the Schizophrenia Program Leader in the CNS Disease Group at Aventis, she developed a new strategy for developing therapeutics to treat the cognitive deficits associated with schizophrenia. Following the merger with Sanofi, she became an Associate Director, leading the US Psychopharmacology Group within the CNS Therapeutic Department. Beth began her career at Synaptic Pharmaceuticals where she identified, cloned and patented numerous novel G protein-coupled receptors (GPCR). By the time she left Synaptic, she had established the company’s first in vivo behavioral laboratories, was responsible for assessing novel GPCR targets for potential therapeutic indications as well as designing and implementing in vivo and in vitro screening strategies to promote lead compounds to Early Development Candidates. Beth received a BS in Psychology and Neurobiology from Rutgers University, and a PhD in Pharmacology from Duke University Medical Center. She completed her post-doctoral training at the National Institute of Mental Health.

Lucie Bruijn Ph.D.


Chief Scientist
 The ALS Association
Washington, DC

Lucie Bruijn Ph.D.


Lucie Bruijn, Ph.D. joined The ALS Association (ALSA) in January 2001 and is currently the Chief Scientist.  Prior to that Dr. Bruijn led a team at Bristol Myers Squibb developing in vitro and in vivo model systems for neurodegenerative disease. Realizing the potential of stem cell therapy for neurodegenerative diseases, her team worked with experts in academia to establish stem cell studies at Bristol Myers Squibb.

 

Dr. Bruijn received her Bachelor’s degree in Pharmacy at Rhodes University, South Africa.  She received a Master’s degree in Neuroscience and a Ph.D. in Biochemistry, specializing in disease mechanisms of Alzheimer’s disease, at the University of London, United Kingdom.  She joined Dr. Don Cleveland’s laboratory in 1994 where she developed and characterized a mouse model of ALS (mice expressing the familial-linked SOD1 mutation).  Using this model her studies focused on disease mechanisms.  In addition, in collaboration with Dr. Robert Brown she looked for neurofilament mutations in familial and sporadic ALS patients.

 

At The ALS Association, Dr. Bruijn leads a global ALS research effort, Translational Research to Advance Therapies for ALS (TREAT ALS™) with the goal to move treatment options from “bench to beside.”  She has made it a priority to collaborate with other funding agencies, in particular The National Institute of Health, The Department of Defense and many other not-for-profit ALS organizations, as well as other foundations focusing on neurodegenerative research. These collaborations ensure that increased dollars are spent on ALS research. She is involved in project development, encouraging partnerships with academia and biotech, and has played a key role in forging collaborations amongst investigators.  It is her strong belief that only through collaboration among a wide range of disciplines will we be successful in changing the course of ALS and finding a cure.

 

Through participation at scientific meetings both nationally and internationally ALSA receives wide-spread recognition amongst the scientific community.  Dr. Bruijn represents The ALS Association on several scientific and research committees world-wide and acts as advisor to scientists, government officials and industry leaders seeking council in the field of ALS research.  She continues to publish in the field in peer-reviewed journals and remains actively engaged in understanding the most recent research developments.

Ray Dorsey, M.D., M.B.A
.

Associate Professor of Neurology
Director of Movement Disorders Division and Neurology Telemedicine
 Johns Hopkins Medicine 
Baltimore, MD

Ray Dorsey, M.D., M.B.A
.

Ray Dorsey, MD, MBA is an Associate Professor of Neurology at Johns Hopkins where he directs the movement disorders division and neurology telemedicine.  He is active clinical researcher and trialist and serves on the executive committee of the Huntington Study Group.  Together with Dr. Kevin Biglan at the University of Rochester, he is developing models that use technology (web-based video conferencing) to care for patients with movement disorders and enable their broader participation in research studies.

Kenneth H. Fischbeck, M.D.


NIH Distinguished Investigator
 Chief, Neurogenetics Branch

Kenneth H. Fischbeck, M.D.


Dr. Fischbeck received A.B. and A.M. degrees from Harvard University and an M.D. degree from Johns Hopkins. After a medical internship at Case Western Reserve University and a neurology residency at the University of California in San Francisco, he did postdoctoral research on muscular dystrophy at the University of Pennsylvania. In 1982 he joined the faculty in the Neurology Department at the University of Pennsylvania Medical School. In 1998 he came to the NINDS as Chief of the Neurogenetics Branch. He received the Cotzias Award from the American Academy of Neurology and the Jacoby Award from the American Neurological Association, and he was elected to the Institute of Medicine. His research group is identifying the causes and studying the mechanisms of hereditary neurological and neuromuscular diseases with the goal of developing effective treatment for these disorders.

Samuel Frank, M.D.


Associate Professor of Neurology
 Co-Director of Neurology Resident Education at Boston University
 Boston, MA

Samuel Frank, M.D.


Dr. Samuel Frank is Associate Professor of Neurology and co-Director of Neurology Resident Education at Boston University.  Since completing his fellowship in Experimental Therapeutics (Movement Disorders) at the University of Rochester, he has established active general movement disorders, Huntington’s disease, and dystonia clinics at Boston University and the New England regional VA hospital.  He also serves as the inpatient neurology consultant for the Huntington’s disease service at Tewksbury State Hospital, a state run chronic care hospital.

 

Regarding research, Dr. Frank has served as the site investigator for multiple clinical trials including those coordinated through the Huntington Study Group, Parkinson Study Group, Michael J. Fox Foundation, National Science Foundation, NIH and other independent studies.  He played a key role as medical monitor for the clinical trials in the US of tetrabenazine for chorea associated with Huntington’s disease.  He has published in the areas of Huntington’s disease, education and research ethics, determining why patients chose (or refuse to) participate in invasive research studies for Parkinson’s disease. He has also served on data safety monitoring committees.

 

Dr. Frank has played a strong role in advocacy for patients, serving as the consumer representative for the Peripheral and Central Nervous System Advisory Committee for the FDA. He is an active member as a member of the board of trustees for the Huntington Disease Society of America (HDSA) and has chaired the National HDSA Education Subcommittee.  Dr. Frank is also currently serving as a member of the Patient Safety Subcommittee at the American Academy of Neurology.

Marcy MacDonald Ph.D.


Professor of Neurology
 Harvard Medical School and the Massachusetts General Hospital
 Boston, MA

Marcy MacDonald Ph.D.

Dr. Marcy MacDonald obtained her Ph.D. in Medical Biophysics from the University of Toronto in 1980 and is currently a Professor of Neurology (Genetics) at Harvard Medical School and the Massachusetts General Hospital, where she is a founding member of the Center for Human Genetic Research.

 

Dr. MacDonald became involved in Huntington’s disease research in 1985, joining Dr. James Gusella in an international collaboration that identified the Huntington’s disease gene in 1993, improving the diagnosis and management of the disorder. Her research pioneers the use of genetic mouse models and genetic studies in human samples to contribute knowledge essential to the development of effective therapies for Huntington’s disease.

 

Dr. MacDonald has served the HDSA as co-chair of the Grants and Fellowship Committee (1998-2003). She is the Chair of the Medical and Scientific Advisory Committee (2008-2011) and for the more than two decades has spoken about Huntington’s disease research at HDSA events for local chapters and at the annual convention.

Harry Orr, Ph.D
.

Tulloch Professor of Genetics, Department of Laboratory Medicine and Pathology
Director, Institute of Translational Neuroscience
 University of Minnesota

Harry Orr, Ph.D
.

Harry Orr received his Ph.D. from Washington University and completed his postdoctoral fellowship at Harvard. Dr. Orr’s research explores genes that play a role in neuron deterioration. This offers important implications for developing gene therapy and other types of treatments for patients with neurodegenerative diseases. Orr discovered the genetic basis for spinocerebellar ataxia type 1, an inherited and ultimately fatal movement disorder, like HD SCA1 is due to a polyglutamine expansion. Orr directs the Institute of Translational Neuroscience, created to translate laboratory research discoveries into clinical trials of new therapies.

Eric E. Schadt Ph.D.

Jean C. and James W. Crystal Professor of Genomics
 Chairman and Professor, Department of Genetics and Genomic Sciences
 Director, Institute for Genomics and Multiscale Biology
 Mount Sinai School of Medicine

Eric E. Schadt Ph.D.

Dr. Eric Schadt recently joined Mount Sinai Medical School as Chairman and Professor, Department of Genetics and Genomic Sciences and as Director, Institute of Genomics and Multiscale Biology in 2011.  Previously, Dr. Schadt had been the Chief Scientific Officer at Pacific Biosciences, overseeing the scientific strategy for the company, including creating the vision for next-generation sequencing applications of the company’s technology.  Dr. Schadt is also a founding member of Sage Bionetworks, an open access genomics initiative designed to build and support databases and an accessible platform for creating innovative, dynamic models of disease.  Dr. Schadt’s current efforts at Mount Sinai to generate and integrate large-scale, high-dimension molecular, cellular, and clinical data to build more predictive models of disease so that we may better diagnose and treat disease, were motivated by the genomics and systems biology research he led at Merck to elucidate common human diseases and drug response using novel computational approaches applied to genetic and molecular profiling data.  His research helped revolutionize a field in statistical genetics (the genetics of gene expression), has energized the systems biology field, and has led to a number of discoveries relating to the causes of common human diseases.  At the time Dr. Schadt left Merck in 2009, greater than 50% of all new drug discovery programs at Merck in the metabolic space were derived from Dr. Schadt’s work.  Dr. Schadt was also recently appointed as Fellow to the Institute of Systems and Synthetic Biology, Imperial College London.  Dr. Schadt received his B.S. in applied mathematics/computer science from California Polytechnic State University, his M.A. in pure mathematics from UCD, and his Ph.D. in bio-mathematics from UCLA (requiring Ph.D. candidacy in molecular biology and mathematics).

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