MEETING PURPOSE
A one-day workshop bringing together invited clinical experts across academia and biopharma to focus on clinical trial design and advancing drug development in ALS
THURSDAY, MARCH 20, 2025
THURSDAY, NOVEMBER 3 2022
8:00 – 8:30a
8:30a Breakfast and Registration
Welcome & Meeting Goals
Jinsy Andrews, MD, MSc (NEALS Co-chair) and James Berry, MD, MPH (NEALS Cochair)
Workshop on Advancing ALS Clinical Trials
NEALS
8:35a
What’s Needed to get into Human Clinical Trials?
Moderator: Nicholas Maragakis, MD (Johns Hopkins University)
Panelists: Lauren Black, PhD (Charles River Laboratories); Erin Flemming, BA (ProJenX); Clotilde Lagier-Tourenne, MD, PhD (Mass General Hospital); Piera Pasinelli, PhD (Thomas Jefferson University); Jeffrey Rothstein, MD, PhD (Johns Hopkins University)
What Biomarkers Would Enhance Your Trial Development?
Moderator: Robert Bowser,PhD (Barrow Neurological Institute)
Panelists: James Berry, MD, MPH (Sean M. Healey & AMG Center for ALS at Mass General); Joshua Cohen, BS (Amylyx Pharmaceuticals); Stephanie Fradette, PharmD (Biogen); Melanie Leitner, PhD (ALS Investment Fund)
10:55a
11:10a 1:20p 2:30p
12:20p 3:40p 9:45a
Break
Beyond the Status Quo: Charting a New Course for Patient-Centered and Precision-Drive ALS Clinical Trials
Moderator: Merit Cudkowicz, MD, MSc (Sean M. Healey & AMG Center for ALS at Mass General)
Networking Lunch
Stats Crash Course in ALS Clinical Trials
Moderators: Lori Chibnik, PhD (MGH Biostatistics); Melanie Quintana, PhD (Berry Consultants)
Panelists: Eric Macklin, PhD (MGH Biostatistics); Peng Sun, PhD (Biogen); Jenny (Jianing) Wang, PhD (MGH Biostatistics)
Outcome Measures: The search for common ground
Moderator: Zachary Simmons, MD (Penn State University)
Panelists: Sheena Chew, MD (Biogen); Ram Miller, MD, MBA, FRCPC (Novartis); Lauren Kett, MD, PhD (Amylyx Pharmaceuticals); Jeremy Shefner, MD, PhD (Barrow Neurological Institute)
Wrap Up
Jinsy Andrews, MD, MSc (NEALS Co-Chair) and James Berry, MD, MPH (NEALS CoChair)
SPEAKER BIOGRAPHIES
(Listed in alphabetical order by last name)
Asa Abeliovich, MD, PhD (Leal Therapeutics)
Dr Asa Abeliovich is CEO and Founder of Leal Therapeutics, which is a near-clinical biotechnology company developing novel therapeutics for major neurodegenerative and neuropsychiatric disorders Asa previously served as CEO and Founder at Prevail Therapeutics until its acquisition by Eli Lilly in 2021, and was Co-Founder at Alector Inc Prior to that, Asa was a tenured Associate Professor of Pathology, Cell Biology, and Neurology at Columbia University, as well as a member of the Taub Institute for Alzheimer’s Disease and the Aging Brain He also served as an Attending Physician in Neurology at the New York-Presbyterian Hospital and the New York Psychiatric Institute
Asa holds M D and Ph D degrees from Harvard Medical School and the Massachusetts Institute of Technology (MIT), respectively, and Bachelor’s degrees in life sciences and humanities from MIT Asa completed his residency training in neurology at the University of California, San Francisco, and conducted Postdoctoral Research at Genentech, Inc Asa is on the Scientific Advisory Board of The Silverstein Foundation for Parkinson’s with GBA
Jinsy Andrews, MD, MSc (NEALS Co-Chair; Columbia University)
Dr Jinsy Andrews is the current Co-Chair of NEALS and the Director of Neuromuscular Clinical Trials at Columbia University. She has been actively involved with the NEALS consortium for the past 10 years and has participated in several NEALS clinical trials She also serves as the co-chair of the Upper Motor Neuron Disease Committee of NEALS and has been doing so for the past seven years
Prior to her current position at Columbia University, Dr Andrews was the Head of Neuromuscular Therapeutics and Senior Director of Clinical Research and Development at Cytokinetics, Inc overseeing international clinical research development in Amyotrophic Lateral Sclerosis and North American clinical research development in Spinal Muscular Atrophy Prior to Cytokinetics, she was the Director of the Clinical Trials Unit and Co-director of the MDA/ALS Clinic at Hospital for Special Care in Connecticut and an Assistant Professor of Neurology at the University of Connecticut She completed her fellowship training in Neuromuscular Disease and Clinical Neurophysiology at Columbia University and subsequently held a faculty position as an Assistant Professor of Neurology while actively participating in clinical trials and caring for patients with ALS and other neuromuscular diseases at the Eleanor and Lou Gehrig MDA/ALS Center at Columbia University She is board certified in Neurology, Neuromuscular Disease and Electrodiagnostic Medicine
Dr Andrews received her B S at Union College, her Master's Degree in Biostatistics (Patient Oriented Research) from Columbia University's Mailman School of Public Health, and her M D from Albany Medical College
SPEAKER BIOGRAPHIES
(Listed in alphabetical order by last name)
James Berry, MD, MPH (NEALS Co-Chair; Sean M. Healey & AMG Center for ALS at Mass General)
Dr. James Berry is the current Co-Chair of NEALS. He is a dedicated ALS clinical researcher at the Sean M. Healey and AMG Center for ALS at Massachusetts General Hospital, where he is the Winthrop Family Scholar in ALS Sciences and the Averill Healey Endowed Chair for ALS. Dr. Berry is the Director of the Neurological Clinical Research Institute and Chief of the Division of ALS and Motor Neuron Diseases at Mass General Hospital. He is committed to developing novel therapeutics by translating discoveries in the lab into clinical trials and leading clinical trials that will transform our approach to care in ALS. He has been a member of NEALS for over a decade and started the Technology Subcommittee in NEALS. He helps to oversee the NEALS Biorepository. His research focus is on digital and biofluid biomarker development and ALS clinical trial design and conduct. He is committed to increasing trial participation opportunities for people with ALS and incorporating research into clinical care for ALS.
Dr. Berry graduated from Tufts University, worked as a middle school teacher with Teach for America in Houston, and then attended medical school at Northwestern University. He completed neurology residency and fellowship training at Mass General Brigham.
Lauren E. Black, PhD, (Charles River Laboratories)
Dr Lauren Black served in FDA/CDER and CBER for 11 years, assessing approximately 1000 transplant drugs, oligos, monoclonals for autoimmune disease, and cell products’ impact in both animals and phase 1 trials in patients She contributed to FDA guidances, including immunotoxicology, oligos, xenotransplantation, and human starting doses Since 2002, she has worked at CRL in the global SAS group, where she advises on trials (including jacifusen for FUS/ALS) and preclinical program support She also supports internal scientists (interpretation of pharm/tox/biomarkers), and consults for drug developers (from large pharma to parents) on their development strategy She was recently promoted to Distinguished Scientist and received the Career Recognition award from the Society of Toxicology for accelerating high risk programs and advancing translational science Lauren trained at Carnegie Mellon University, Virginia Commonwealth School of Medicine, and NIH/NINDS (dopamine receptor dynamics and signal transduction) before working at FDA
SPEAKER BIOGRAPHIES
(Listed in alphabetical order by last name)
Robert Bowser, PhD (Barrow
Neurological
Institute)
Dr. Robert Bowser is an internationally-recognized leader in ALS research. He has contributed to pioneering efforts to discover and validate biomarkers for ALS. These biomarkers can be useful as diagnostic indicators of disease, predictors of disease progression, and also in determining the effectiveness of drugs in clinical trials. Dr. Bowser has extensive experience in the translation of basic science discoveries to the clinic to impact patient care.
As director of the Barrow ALS Research Center, Dr. Bowser directs research to determine the underlying mechanisms of ALS, identify new targets for drug treatment, develop improved therapies for ALS, and lead clinical research studies performed in numerous medical centers throughout North America.
Dr. Bowser is a graduate of Carnegie Mellon University and obtained his PhD from Yale University. He performed fellowship training at the Albert Einstein College of Medicine and was a faculty member at the University of Pittsburgh from 1994 to 2011, rising through the ranks to full professor. He joined the faculty at Barrow in 2011.
Joshua Cohen, BS (Amylyx Pharmaceuticals)
Josh Cohen co-founded Amylyx Pharmaceuticals (NASDAQ: AMLX) in 2013 and continues to serve as its co-CEO Josh, alongside co-CEO Justin Klee, has overseen the growth of Amylyx – from a concept dreamed up in a dorm room through its IPO, FDA and Health Canada approvals of its first product, and product launch – to the company it is today, with a mission to usher in a new era for treating diseases with high unmet needs
Josh co-invented the oral, fixed-dose combination of sodium phenylbutyrate (PB) and taurursodiol (TURSO, also known as ursodoxicoltaurine), which is being explored for the potential treatment of neurodegenerative diseases Amylyx’ clinical trial results in collaboration with leading experts have been published in several peer-reviewed medical journals, including theNew England Journal of Medicine, Muscle & Nerve, and theJournal of Neurology, Neurosurgery and Psychiatry
Josh and Justin have been named to Business Insider’s 30 Under 40 in Healthcare list and PM360’s ELITE Drug Researchers & Developers and Entrepreneurs lists In 2022, they received the Harvey and Bonny Gaffen Advancements in ALS Award by the Les Turner Foundation, the Henri Termeer Transatlantic Connections Award, and the Northeast ALS Consortium (NEALS) Big Data Award The CENTAUR clinical trial team made up of Amylyx, the MGH Neurological Clinical Research Institute, NEALS, and Barrow Neurological Institute was also awarded the 2022 Annual Healey Center International Prize for Innovation in ALS Stemming from his background in biomedical engineering, Josh is passionate about research that improves outcomes where there is a significant unmet need Josh was a National Merit Scholar and earned his Bachelor of Science in biomedical engineering from Brown University While attending Brown, Josh founded the Brown Biotechnology Investment Group and published research at the National Institutes of Standards and Technology (NIST) and in theJournal of Pharmaceutical Sciences
SPEAKER BIOGRAPHIES
(Listed in alphabetical order by last name)
Sheena Chew, MD (Biogen)
Dr. Sheena Chew is a neuromuscular neurologist and a Medical Director in Neuromuscular Clinical Development at Biogen. In her role at Biogen, she supported the development of tofersen for SOD1-ALS, and she led the global clinical development program for BIIB105, an antisense oligonucleotide targeting ataxin-2 protein for ALS. She received her MD from Harvard Medical School, completed her neurology residency and neuromuscular medicine fellowship training at Mass General Brigham, and completed the Anne B. Young Fellowship in Therapeutic Development, focusing on ALS, at Biogen and Mass General Hospital.
Lori Chibnik, PhD (MGH Biostatistics)
Dr Lori Chibnik is a biostatistician and Associate Professor with appointments in the Departments of Neurology at Massachusetts General Hospital and Harvard Medical School the Department of Epidemiology at the Harvard T H Chan School of Public Health and She received her MPH in International Health and her PhD in biostatistics from Boston University where she worked on predictive modeling methods for disease risk Over her career she has developed and assessed predictive models for diseases such as HIV, pre-natal screening and autoimmune diseases and continues to apply her methods to various diseases Dr Chibnik’s current research focus is in two primary areas, genetics and genomics of neurodegenerative diseases with an emphasis on longitudinal cohorts and genetic studies in ethnically diverse populations She serves as one of the primary statisticians for the Healey ALS Platform Clinical Trial and is the co-lead of the Data and Statistics Core of the Massachusetts Alzheimer’s Disease Research Center (MADRC)
In addition to her research, she is internationally renowned for her training programs and innovative teaching techniques, having developed multiple courses in biostatistics for varied audiences While at Boston University she managed the Summer Institute for Training in Biostatistics, an NHLBI funded, 6-week summer program designed to bring undergraduate students into the fields of Biostatistics and Public Health She has developed and implemented multiple courses on research methods, biostatistics and programming specific to the needs of scientists in sub-Saharan Africa and currently she serves as Principal Investigator of the Global Initiative for Neuropsychiatric Genetics Education in Research (GINGER) at the Harvard-Chan School and the Stanley Center for Psychiatric Research at the Broad Institute of Harvard and MIT
SPEAKER BIOGRAPHIES
(Listed in alphabetical order by last name)
Merit Cudkowicz, MD, MSc (Sean M. Healey & AMG Center for ALS at Mass General)
Dr. Merit Cudkowicz is the inaugural Executive Director, Massachusetts General Brigham Neuroscience Institute, the Director of the Sean M. Healey & AMG Center for ALS, and the Julieanne Dorn Professor of Neurology at Harvard Medical School in Boston. Dr. Cudkowicz completed her medical degree in the Health Science and Technology program of Harvard Medical School and a master’s degree in Clinical Epidemiology from the Harvard School of Public Health. She completed her neurology residency and fellowship at Mass General.
Dr. Cudkowicz’s research and clinical activities are dedicated to the study and treatment of individuals with amyotrophic lateral sclerosis (ALS). She is a leader in neurotherapeutics. She co-founded the Neurological Clinical Research Institute at Mass General. She is also one of the co-founders of the Northeast ALS Consortium (NEALS), a group of more than 150 clinical sites in North America dedicated to performing collaborative, clinical studies to develop treatments for people with ALS. She co-directed NEALS for the first 18 years and currently serves on the Executive Committee and Science Advisory Board. Dr. Cudkowicz has led many clinical trials in ALS and leads the first Platform Trial in ALS. She also the Principal Investigator for the Clinical Coordination Center for NeuroNEXT, a National Institute of Health supported early phase network that conducts high-impact, efficient early phase trials for neurological disorders. She is a member of the National Academy of Medicine and a council member of the American Association of Physicians. She is the ecipient of numerous awards including the 2006 Wings over Wall Street MDA award, 2009 Sheila Essey Award from the American Academy of Neurology, 2012 Prize4life Pioneer Award, 2017 Forbes Norris Award from the International MND Society and the 2018 Pinnacle Award from the Boston Chamber of Commerce.
Erin Fleming, BA (ProJenX)
Erin Fleming is a co-founder and Chief Operating Officer at ProJenX Prior to ProJenX launching in November 2021, Ms Fleming was Director of Research Operations at Project ALS, a non-profit 501(c)3 organization that identifies and funds promising scientific research toward the first effective treatments and a cure for ALS
Previously, Erin was associate director at Project ALS, project manager at the biotech Applied Therapeutics, and project director at the boutique life sciences consulting firm Clearpoint Strategy Group She holds a BA in English and Comparative Literature from Columbia University
SPEAKER BIOGRAPHIES
(Listed in alphabetical order by last name)
Stephanie Fradette, PharmD (Biogen)
Dr. Stephanie Fradette is the Head of the Neuromuscular Development Unit at Biogen. In her role, she oversees development of therapies for Amyotrophic Lateral Sclerosis (ALS), Spinal Muscular Atrophy (SMA), Friedreich’s Ataxia (FA), and other neuromuscular diseases. Stephanie has spent the last 15 years in various roles across Research and Development, including Safety, Regulatory, and Clinical Development helping to bring meaningful therapies to patients around the world. Recently, she led the development of the first approved therapy for a genetic form of ALS (SOD1-ALS), which supported significant advancement of neurofilament as a biomarker and optimization of clinical trial design in ALS.
Lauren Kett, MD, PhD (Amylyx Pharmaceuticals)
Dr Lauren Kett is a Medical Director in Clinical Development at Amylyx Pharmaceuticals, where she oversees the design and conduct of clinical trials in ALS and other neurodegenerative diseases Lauren received her MD/PhD at the University of Michigan Medical School She completed neurology residency and neuromuscular fellowship at Massachusetts General Hospital and Brigham and Women's Hospital
SPEAKER BIOGRAPHIES
(Listed in alphabetical order by last name)
Clotilde Lagier-Tourenne, MD, PhD (Mass General Hospital)
Dr. Lagier-Tourenne is Associate Professor of Neurology at the Massachusetts General Hospital and Harvard Medical School. She is the recipient of the Araminta Broch-Healey Endowed Chair in ALS, and associate member at the Broad Institute of MIT and Harvard. She trained as a medical geneticist and neuroscientist in Strasbourg (France), Columbia University and UC San Diego. She was awarded the 6th International Paulo Gontijo Award in Medicine, the 2017 Grass Foundation –American Neurological Association Award in Neuroscience, the 2022 Drs. Lalji & Family ALS Endowed Award for Innovative Healing and the 2023 World Medical Innovation First Look Award. Her team investigates the molecular mechanisms driving neurodegeneration in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). She has established collaborations with academic and pharmaceutical partners to develop novel approaches to therapy, including RNAtargeting antisense oligonucleotides and immunotherapies for ALS and FTD.
Melanie Leitner, PhD (Accelerating NeuroVentures LLC, ALS Investment Fund)
Dr Melanie Leitner currently serves as the Chief Scientific Officer for the ALS Investment Fund (a life sciences VC) and is the founder of Accelerating NeuroVentures, LLC, a solo consulting practice providing strategic, programmatic and business development services to investors, biopharma, and nonprofit clients interested in funding, initiating or advancing programs within the neuroscience translational space Prior to launching Accelerating NeuroVentures, Leitner was Chief Scientific Officer at the ALS nonprofit Prize4Life, and she has also held director positions at Biogen and at the nonprofit organizations FasterCures and the Society for Neuroscience Earlier in her career she was a member of U S Senator Dick Durbin’s Health staff Leitner was awarded a Howard Hughes Predoctoral Fellowship and a Lucille P Markey Fellowship for her Ph D work at Washington University, and graduated Magna Cum Laude with an Sc B from Brown University all in the field of neuroscience
SPEAKER BIOGRAPHIES
(Listed in alphabetical order by last name)
Eric Macklin, PhD (MGH Biostatistics)
Dr. Eric A. Macklin is an Assistant Investigator in Biostatistics at Massachusetts General Hospital and an Assistant Professor in the Dept of Neurology at Harvard Medical School. His research interests focus on neurodegenerative and neurodevelopmental diseases with an emphasis on clinical trial design.
He is currently a lead statistician for the HEALEY ALS Platform Trial, a statistical advisor for the PSP Trial Platform, and study statistician for several other clinical trials studying biologic and pharmacologic interventions for ALS, observational studies in PD, trials of psychosocial interventions for TBI, trials conducted by the Center for Rare Neurological Diseases, and a study of cerebral amyloid angiopathy. He was an organizing member and lead statistician for the Airlie House Clinical Trial Guidelines for conduct of ALS clinical trials, has been the Biostatistics Director for the Executive Committee of the Parkinson Study Group for the past 12 years, and is an advisor to the Critical Path for Parkinson’s and the Critical Path Rare Neurological Diseases programs. His previous work in neurology and neuro-oncology includes many other trials in ALS and PD, design of phase I/II MS trials and AD and PD prevention trials, studies of brain infarction in sickle cell disease, acupuncture for treatment of chronic stroke symptoms, imaging of intracranial aneurysms, and treatment and prognostics of glioblastoma.
Dr. Macklin earned a PhD from Duke University and also holds an MS from Stanford University and an SM from the Harvard T.H. Chan School of Public Health.
Nicholas Maragakis, MD (Johns Hopkins University)
Dr Nicholas Maragakis is Director of the ALS Center for Cell Therapy and Regeneration Research, Co-Director of the Johns Hopkins ALS Clinic and Professor of Neurology at Johns Hopkins University The ALS Clinic at John Hopkins is a world recognized leader in providing medical care and offering the latest in clinical trials and therapies to ALS patients
A common theme to his research has been in the study of the astrocyte biology and its role in disease pathogenesis with a particular emphasis in how astrocytes may contribute to Amyotrophic Lateral Sclerosis (ALS) His laboratory in collaboration with others, has been interested in the development of induced pluripotent stem cells from ALS patients His laboratory has created over 50 iPS cell lines from familial and sporadic ALS patients as well as controls His current efforts have been to characterize iPSC-derived motor neurons and astrocytes both in vitro and in vivo with an effort towards understanding disease mechanisms; in particular modeling ALS disease heterogeneity with regard to disease progression
Dr Maragakis has also been heavily involved in clinical research as the principal investigator, site principal investigator, or co-investigator of numerous clinical trials in ALS, many coordinated by the Northeast ALS Consortium on which he served as an Executive Board member and currently a member of the Scientific Advisory Board
Dr Maragakis has received several awards for his outstanding research efforts and received the Certification of Meaningful Use Stage 1 EHR from the Centers for Medicare& Medicaid Services (CMS) in 2013 He is member of the American Board of Psychiatry and Neurology, the American Academy of Neurology (AAN) and the Northeast ALS Consortium (NEALS)
Dr Maragakis received his bachelors of science degree and medical degree from the University of Utah and he completed his residency and fellowship in neurology at John Hopkins University
SPEAKER BIOGRAPHIES
(Listed in alphabetical order by last name)
Ram Miller, MD, MBA, FRCPC (Novartis)
Dr. Ram Miller is a Senior Clinical Development Medical Director at Novartis Pharmaceuticals. A geriatrician and an epidemiologist by training, Ram has many years of experience conducting clinical research in neuromuscular disease. He has a particular interest in innovative clinical trial designs and the development of novel clinical endpoints for ALS.
Piera Pasinelli, PhD (Thomas Jefferson University)
Dr Piera Pasinelli is the Frances & Joseph Weinberg Professor in Neuroscience at Thomas Jefferson University and the founder and Director of the Jefferson Weinberg ALS Center, a Clinical-and-Research Integrated Program
She graduated with a dual Bachelor and Master in Science degree from the University of Milan, Italy, where she studied Chemistry and Technology in Pharmaceutical Industry and obtained her PhD in Neuro-biochemistry from the University of Utrecht, The Netherlands She performed postdoctoral training at Massachusetts General Hospital-Harvard Medical School and, in 2006, joined Thomas Jefferson University as the co-director of the Frances & Joseph Weinberg Unit for ALS Research In 2016, under her leadership, Jefferson expanded the ALS program adding a multidisciplinary clinic and a clinical-research program to complement the existing research laboratories and launching the Weinberg ALS Center
Her research focuses on the cell death mechanisms that overtake the motor neurons in ALS and the role played by glia cells in disease She also leads clinical research studies aimed at identifying disease biomarkers and new therapeutic targets
Dr Pasinelli has been the Scientific Director of the Robert Packard Center for ALS Research from 2008 to 2020 and now serves on the scientific advisory board of the Center She is also member of the scientific advisory board of AriSLA and of the ALS/MND International Alliance She is passionate about educating the ALS patients about research and over the years has taken an active role in raising awareness of ALS in the community
SPEAKER BIOGRAPHIES
(Listed in alphabetical order by last name)
Melanie Quintana, PhD (Berry Consultants)
Dr. Melanie Quintana is a Director of Consulting and Senior Statistical Scientist at Berry Consultants, LLC. She is an expert in utilizing Bayesian statistics to design innovative clinical trials and answer complex clinical questions across a wide range of therapeutic areas. She earned a PhD in Statistics from Duke University and went on to pursue a Postdoc in Biostatistics at The University of Southern California. During her PhD and Postdoc, she worked closely with collaborators within multiple large studies and consortiums around the nation to develop and implement analytical strategies to assess genetic risk factors for various complex diseases. This work led to a passion for consulting and collaborating with clinical experts to find analytical solutions to hard medical problems, which has only expanded since joining Berry Consultants in 2013. At Berry Consultants, she collaborates with a wide range of clinical experts to find the best solution for answering complex clinical questions and mentors and oversees a group of consultants to do the same. Her work includes the design of over 100 clinical trials in a wide range of therapeutic areas including adaptive designs, platform trials & statistical innovation in rare disease and unmet medical needs. Her work in clinical design also involves support for regulatory interactions and guidance on regulatory considerations, and support for clinical trial implementation as a member of statistical analysis committees and data safety and monitoring boards.
Bernard Ravina, MD (Atlas Venture)
Dr Bernard Ravina is CEO of a Stealth NewCo and has over 20 years of drug development experience in government, academia, and industry Most recently he was CMO of Praxis Precision Medicines Prior to that he served as CMO of Voyager Therapeutics and worked in clinical development at Biogen Dr Ravina holds an M D from the Johns Hopkins University School of Medicine and a Master’s in Clinical Epidemiology and Biostatistics from the University of Pennsylvania, where he completed training in neurology After training, Dr Ravina held positions at NINDS/NIH in neurogenetics and at the University of Rochester School of Medicine where he was Associate Professor of Neurology and Director of the Movement and Inherited Neurological Disorders Unit
SPEAKER BIOGRAPHIES
(Listed in alphabetical order by last name)
Jeffrey Rothstein, MD, PhD (Johns Hopkins University)
Dr. Jeffrey Rothstein is the John Griffin Director of the Brain Science Institute and Professor of Neurology and Neuroscience at Johns Hopkins University, Baltimore, MD, USA. He is the Founder and Director of the Robert Packard Center for ALS Research at Johns Hopkins, the largest collaborative academic organization dedicated towards understanding ALS and developing novel therapeutics and the Director of the Muscular Dystrophy Association ALS Clinic. He received a B.A. in Neuroscience from Colgate University, Johns Hopkins University, Baltimore, Maryland, USA, a Masters at the University of Chicago, Chicago IL, USA and his MD and Ph.D. in Physiology and Biophysics from the University of Illinois, Chicago, IL, USA. He did Neurology residency and neuromuscular fellowship training at Johns Hopkins University, Baltimore, MD USA. He has been the principal and/or local investigator in almost a dozen National or International trials in ALS His laboratory research interests range from basic neurobiology of CNS glia and their role in regulating neurotransmission, to the pathophysiology of neurodegenerative disease, focusing on ALS. Specifically, he uses model systems including rodent and human IPS models and human tissue to dissect the underlying mechanisms of neuronal and glial injury in ALS as a means of illuminating effective therapies. He is the author of over 250 peer-reviewed articles on basic and clinical neuroscience. He holds more than a dozen patents on candidate drugs and biomarkers for ALS and Neurodegenerative disease.
Jeremy Shefner, MD, PhD (Barrow Neurological Institute)
Dr Jeremy Shefner is Professor of Neurology and Chief Medical Officer for Clinical Research at the Barrow Neurological Institute in Phoenix, AZ He is trained in neurology and clinical neurophysiology, and has devoted his clinical and research efforts to experimental therapeutics, clinical trial design, and outcome measure development primarily directed towards ALS He received a PhD in experimental psychology from the University of Illinois in 1976, and an MD from Northwestern University in 1983 After residency and fellowship training at Harvard Medical School and the Brigham and Women's Hospital in Boston, Massachusetts, Dr Shefner started the ALS Clinic at BWH and participated in multiple industry sponsored ALS clinical trials He co-founded the Northeast ALS Clinical Trials Consortium (NEALS) in 1995, and co-chaired NEALS through 2013 He remains on the Executive Committee for NEALS NEALS has grown to include more than 100 sites throughout the US and Canada, and has performed more than 50 clinical studies since 1995 Dr Shefner has held leadership positions in many NIH and industry supported clinical trials He has published more than 230 peer reviewed scientific articles, is neuromuscular section editor of UpToDate, and serves on the editorial board of Neurotherapeutics and the ALS Journal He is also past Chair of the ALS Research Group and the New York Neurological Society In 2014, he was awarded the Sheila Essey Award for ALS Research by the ALS Association and the American Academy of Neurology
SPEAKER BIOGRAPHIES
(Listed in alphabetical order by last name)
Zachary Simmons, MD (Penn State University)
Dr. Zachary Simmons received his medical degree from the University of Florida, and then trained in neurology at the University of Iowa and in neuromuscular diseases and electromyography at the University of Michigan. He now serves as Professor of Neurology and Humanities at Penn State University, where he founded and directs the ALS Clinic and Research Center.
The research center now has a neural engineer, 3 physicians, a research manager, and 3 research coordinators. Dr. Simmons had serves as site principal investigator for more than 20 clinical treatment trials, and a large number of investigatorinitiated studies in ALS. He has published extensively on quality of life in ALS and was the co-developer of the ALS Specific Quality of Life Instrument (ALSSQOL) and the more recently validated short form (ALSSQOL-SF). Related to this, he has published on ethics of care and end-of-life issues. The Penn State ALS Center has pioneered the use of telemedicine in ALS for clinical and research purposes, including home monitoring of respiratory function, of gait and falls, and of speech and swallowing. Other ALS-related research interests include cognitive and behavioral dysfunction, symptom management, optimization of multidisciplinary care, psychological well-being and mindfulness.
Dr. Simmons currently serves as Editor-in-Chief of Muscle & Nerve. He has published more than 150 peer-reviewed articles. At the organizational level, he has served on the NEALS technology, bulbar, and upper motor neuron committees, and on the AANEM Board of Directors and multiple AANEM committees. Institutional responsibilities include Vice-Chair for Research in Neurology and membership in his Department’s Executive Committee. He has been a member of the Ethics, Law, and Humanities Committee of the American Academy of Neurology, and of the Penn State Hershey Ethics Committee.
Peng Sun, PhD (Biogen)
Dr Peng Sun received his Ph D degree in statistics in 2006 from the University of Iowa He worked in the clinical pharmacology statistics group at Merck for four years before joining GSK At GSK, Peng was the lead statistician for the global submission of a combination treatment for metastatic melanoma that received accelerated approval from the FDA Peng joined Biogen in 2015, and he severed as the lead statistician for the global submission of Spinraza, the first approved drug for the treatment of spinal muscular atrophy (SMA) Peng is currently the statistics head for the rare disease franchise at Biogen and he has been closely involved in the successful development of tofersen for the treatment of SOD1 ALS
SPEAKER BIOGRAPHIES
(Listed in alphabetical order by last name)
Jenny (Jianing) Wang, PhD (MGH Biostatistics)
Dr. Jianing Wang is an Assistant Investigator at MGH Biostatistics and a faculty member in the Department of Medicine at Harvard Medical School. She specializes in statistical methodologies for epidemiology, knowledge transfer between largescale health surveillance systems, and ALS clinical trial research. Her current focus at MGH is on Phase 2 ALS clinical trials and biomarker analyses, where she plays a key role in identifying and evaluating meaningful endpoints and assessing outcome measures to advance trial methodologies. She is involved in the HEALEY ALS Platform Trials and other neurological studies. Dr. Wang completed her PhD in Biostatistics at the Boston University School of Public Health and her MS in Statistics at Boston University.
ABOUT THE NORTHEAST ALS CONSORTIUM (NEALS)
In 1995, the Northeast ALS Consortium (NEALS) was founded as an independent, non-profit group of research sites that collaboratively conduct clinical research in Amyotrophic Lateral Sclerosis (ALS) and other motor neuron diseases. While retaining its original acronym and focus on ALS trials, NEALS has since developed into a national, and increasingly an international, consortium of ALS clinical research centers Together, the scientists at these centers work to improve the efficiency and quality of ALS clinical trials and research, expand access to clinical research, support biomarker development with a robust biorepository, innovate ALS clinical care, and educate the community about ALS research Through these efforts, NEALS strives to rapidly translate scientific breakthroughs into new therapies and help people with ALS live longer and maintain a higher quality of life.
Governed by a seasoned Leadership Team, NEALS has developed into an active network of 156 trial-ready sites with extensive training on conducting clinical research and robust experience in designing and managing FDAregulated clinical trials for ALS NEALS has successfully partnered with industry and academic researchers to conduct high-quality ALS studies for over 25 years and has grown to be the largest ALS research consortium in the world.
ABOUT THE SEAN M. HEALEY & AMG CENTER FOR ALS AT MASS GENERAL
At the Sean M Healey & AMG Center for ALS at Mass General, we are committed to bringing together a global network of scientists, physicians, nurses, foundations, federal agencies, and people living with ALS and their loved ones and caregivers to accelerate the pace of ALS therapy discovery and development.
Launched in November 2018, the Healey & AMG Center, under the leadership of Merit Cudkowicz, MD and a Science Advisory Council of international experts, is reimagining how to develop and test the most promising therapies to treat the disease, identify cures and ultimately prevent it
With dozens of active clinical trials and lab-based research studies in progress right now, we are ushering in a new phase of ALS treatment and care Together, we will find the cures
