Stanley Appel, M.D.
Dr. Appel is The Peggy and Gary Edwards Distinguished Endowed Chair for the Treatment and
Research of ALS, Department of Neurology, Neurological Institute, The Methodist Hospital as well
as a Professor of Neurology at Weill Medical College of Cornell University and a Professor of
Biochemistry and Molecular Physiology at Baylor College of Medicine. He was previously Chair of
the Department of Neurology at Baylor College of Medicine as well as Chief of Neurology and the
James B. Duke Professor of Medicine at Duke University Medical Center. Dr. Appel is a native of
Massachusetts and received his Bachelor Degree at Harvard University and his Medical Degree from
Columbia College of Physicians and Surgeons. He is the Director of the MDA/ALS Research and
Clinical Center at the Methodist Neurological Institute, and past Director of the National
Institute of Aging Alzheimer's Disease Research Center. He has received a number of awards for
his accomplishments in Neurology and Biochemistry, including the Gold Medal Award in 1997 from
Columbia College of Physicians and Surgeons; the Sheila Essey Award in 2003 from the American
Academy of Neurology, has been elected a Fellow of the American Association for the Advancement
of Science in 2003, was awarded the Baylor College of Medicine Alumni Association's
"Distinguished Faculty Award"in 2004, the MDA's Wings Over Wall Street Diamond Award in
2004, the Texas Neurological Society Lifetime Achievement Award in 2005 and the Forbes Norris
Award for "compassion and love for humanity in research and treatment in patients with ALS"
from the International Alliance of ALS/MND Associations in 2005.
Merit Cudkowicz, M.D.
Dr. Merit Cudkowicz is an Associate Professor of Neurology at Massachusetts General Hospital, at Harvard Medical School. Dr. Cudkowicz completed medical training at the Health Science and Technology program of Harvard Medical School, and she was a resident in Neurology at Massachusetts General Hospital. She obtained a Master's degree in Clinical Epidemiology from the Harvard School of Public Health in June, 1996. She was also a fellow in the Massachusetts General Hospital (MGH)/ Massachusetts Institute of Technology (MIT) Clinical Investigator Training Program from 1994 to 1996. Dr. Cudkowicz co-directs the MGH Amyotrophic Lateral Sclerosis (ALS) clinic and the Neurology Clinical Trials Unit (NCTU) at MGH and Partners Healthcare System. She co-directs the Northeast ALS Consortium with Dr. Jeremy Shefner. She is on the medical advisory board for the Muscular Dystrophy Association and the Massachusetts Chapter of the Amyotrophic Lateral Sclerosis Association.
Jonathan Katz, M.D.
Now in his fifth year at the Forbes Norris ALS Center in San Francisco, Dr.
Katz continues to gain important exposure to methods of clinical trial design and management at a
busy ALS research center. In this role, he has served as the center PI on numerous clinical trials
and research studies. Dr. Katz serves on the steering committee in the ongoing Lithium in ALS trial,
where Forbes Norris is the lead center of this WALS study. Within this trial, Dr. Katz has been
involved with all aspects of trial management, and has helped design the underlying protocol and
data forms, organize and lead investigator meetings, and interpret data reports. This work,
primarily, has prepared him for the current proposal.
Forbes Norris is also active center in clinical trial participation in ALS, in general. Here, Dr.
Katz has served as the site PI on numerous trials. He has also taken an active roll in trial design
where he has participated locally in early stages of several industry based studies, where he has
worked closely with Dan Moore, PhD. He recently presented his work at the international motor neuron
disease conference on methods for using and interpreting historical control data. The goal of this
work is to improve small trial designs. His work as Co-PI on a proposal for designing small clinical
trials has also lead to Forbes Norris being named one of five national centers within an MDA consortium.
In addition to his clinical trial experience, Dr. Katz is just completing work as PI on a large, ALSA
funded clinical study of advanced MRI in ALS. Working with UCSF researchers, the study involved
longitudinal scanning, data collection, and analysis of ALS patients with advanced MRI techniques.
Dr. Katz joined the Forbes Norris Center after serving as Associate Professor at Stanford University
Medical School. Beyond ALS, Dr. Katz has an extensive clinical background in neuromuscular diseases.
He has led clinics in neuromuscular disease and muscular dystrophy at both centers, and has an extensive
publication background outside of ALS. Much of his academic work has focused on issues regarding
diagnosis, delivery, and decision making in a variety of neuromuscular diseases. He also plays an active
role on national medical advisory boards in myasthenia gravis and CIDP. This background has led to an
interest in understanding different perspectives in how healthcare is delivered and, with respect to ALS,
how decisions underlying clinical trials affect patient outcomes and attitudes towards participation.
Carey Goldberg
Carey Goldberg has been Boston bureau chief of The New York
Times, a longtime Moscow correspondent for The Los Angeles Times, and most recently a science and health
reporter for The Boston Globe, where she primarily covered the brain. She now freelances happily from
home, and is co-author of a book to be published by Little, Brown in April: "Three Wishes: A True
Story of Good Friends, Crushing Heartbreak and Astonishing Luck on the Way to Love and Motherhood."
She graduated summa cum laude from Yale and did graduate work at Harvard and Leningrad State. She also
spent a year as a Knight Science Journalism fellow at MIT. A sampling of her work can be found on her
Website,
bycareygoldberg.com
Steve Perrin, Ph.D.
Dr. Steven Perrin is currently the Chief Executive Officer and Chief Scientific Officer at the
ALS Therapy Development Institute (ALS TDI) in Cambridge, MA. He earned his Ph.D. at Boston
University Medical Center studying the transcriptional regulation of genes during adipocyte and
myocyte differentiation. Steven moved into the pharmaceutical industry in 1997 holding positions
at the Hoechst-Ariad Genomics Center, Aventis Pharmaceuticals and more recently as Director of
Molecular Profiling at Biogen Idec. Steven joined ALS TDI in 2007 as part of historical
collaboration between the Muscular Dystrophy Association, Augie's Quest and ALS TDI to develop
effective therapeutics for ALS patients. Since joining ALS TDI Steven has spearheaded the
development of computational biology capabilities and information management systems to more
clearly understand the molecular mechanisms associated with disease onset and progression in
neurodegenerative diseases. He has expanded ALS TDI's drug screening program to include expertise
in the generation and assessment of gene therapy vectors and protein biologics in preclinical
models of neurodegeneration. He has developed a business plan to facilitate pharmaceutical
partnerships for rapid clinical development and commercialization of promising targets for ALS
patients. Steven is a frequent participant in international conferences in computational biology,
genomics, drug development, and neurodegeneration.
Fernando Vieira, M.D.
Dr. Vieira has worked at ALS TDI since 2001 and has deep ties to the ALS/MND community. With
nearly a decade in ALS research, he has served in a many capacities advancing scientific efforts
at the Institute. He has been integral to the design, execution, and oversight of over 1,000
preclinical studies, the majority of which directly relate to amyotrophic lateral sclerosis. He
worked closely with former ALSTDI President Sean F. Scott to help develop the gold standard for
preclinical survival testing using the G93A-SOD1 mouse model. Since presenting the study design
guidelines to the international ALS research community at meetings and symposiums, Fernando has
helped much of the ALS research community has adopted them. Additionally, he has designed and
validated a novel surgical method for constant infusion of therapeutics to the intrathecal space
of mice. Currently, he manages the scientific project portfolio at ALS TDI, including both
therapeutic and technology development projects.
His previous research experiences include work in the University of Florida Condensed Matter
Physics Lab and the University of Florida Vascular Surgery Lab. A native of Massachusetts,
Vieira received a Bachelors Degree in biological engineering at the University of Florida and
his Medical Degree from Harvard Medical School.
John M. Lincecum, Ph.D.
For four years John co-lead Biogen Idec's therapeutic discovery program in ALS and motor
neuron disease. An expert in gene therapy for motor neuron disease, John has developed several
innovative, patented techniques that have led to gene expression levels far beyond current
published studies. At ALS TDI, John is expanding his work in retrograde transport of gene
therapies injected into muscle, as well as direct intrathecal delivery of promising vectors —
crucial in the validation of pivotal pathways. John received his doctorate in biochemistry
from Boston University and pursued post-doctoral study at Harvard Medical School.