Specialty: Neurology and Complex Disease Genetics
Ammar Al-Chalabi is a Clinician Scientist at King’s College London. His research focuses on causes, modifiers and potential treatments for ALS. He co-leads the UK National MND Research Institute, chairs the International Symposium on ALS/MND, and is a National Institute for Health Research Senior Investigator. His work has been recognized by multiple prizes, including the Forbes Norris Award from the International Alliance of ALS/MND Associations, the Healey Center International Prize for Innovation in ALS, the Sheila Essey Award from the American Academy of Neurology, a Gold National Clinical Excellence Award, and the Charcot Young Investigator Award from the MND Association.
Multistep Process in ALS
Understanding the Genetic Architecture of ALS
Specialty: Neurology and Complex Disease Genetics
Ammar Al-Chalabi is a Clinician Scientist at King’s College London. His research focuses on causes, modifiers and potential treatments for ALS. He co-leads the UK National MND Research Institute, chairs the International Symposium on ALS/MND, and is a National Institute for Health Research Senior Investigator. His work has been recognized by multiple prizes, including the Forbes Norris Award from the International Alliance of ALS/MND Associations, the Healey Center International Prize for Innovation in ALS, the Sheila Essey Award from the American Academy of Neurology, a Gold National Clinical Excellence Award, and the Charcot Young Investigator Award from the MND Association.
Innovations in Trial Design – From Phase 1 to Registration
Angela GENGE Canada
Dr Angela Genge is the Executive Director of the Clinical Research Unit (CRU) and Director of the ALS Clinical at the Montreal Neurological Institute and Hospital. Under her direction, the CRU has grown from a small centre with a handful of staff and trials to encompass over 60+ staff members and over 100 industry and investigator-sponsored Phase I-IV clinical trials spanning the spectrum of neurological diseases. It is now a globally renowned Phase 1 Unit and ALS Global Centre of Excellence. Dr Genge herself has been principal investigator on well over 150 sponsored trials since 1994, and she has mentored a dozen fellow physicians to become clinical trialists in their own right.
Over the course of the past 30 years, Dr Genge has come to be a respected key opinion leader in the space of trial design and development. She has served as Global PI for both ALS-Pharma and Alexion; on steering committees for MT-Pharma, Cytokinetics, Alexion, Biogen, among others; and consulted on trial design for dozens of trials. Her expertise has also led her to be a sought-after chair for advisory boards, where she brings together the voices of all stakeholders and successfully managing the dynamics between sponsors and key opinion leaders.
Though now double board certified in Neurology and Internal Medicine, Dr Genge was originally trained as a physio. It should come as no surprise therefore that Dr Genge is deeply committed to the development of better outcomes measures in Rare Diseases in Neurology, for which she is currently leading four global initiatives.
Innovations in Trial Design – From Phase 1 to Registration
Dr Angela Genge is the Executive Director of the Clinical Research Unit (CRU) and Director of the ALS Clinical at the Montreal Neurological Institute and Hospital. Under her direction, the CRU has grown from a small centre with a handful of staff and trials to encompass over 60+ staff members and over 100 industry and investigator-sponsored Phase I-IV clinical trials spanning the spectrum of neurological diseases. It is now a globally renowned Phase 1 Unit and ALS Global Centre of Excellence. Dr Genge herself has been principal investigator on well over 150 sponsored trials since 1994, and she has mentored a dozen fellow physicians to become clinical trialists in their own right.
Over the course of the past 30 years, Dr Genge has come to be a respected key opinion leader in the space of trial design and development. She has served as Global PI for both ALS-Pharma and Alexion; on steering committees for MT-Pharma, Cytokinetics, Alexion, Biogen, among others; and consulted on trial design for dozens of trials. Her expertise has also led her to be a sought-after chair for advisory boards, where she brings together the voices of all stakeholders and successfully managing the dynamics between sponsors and key opinion leaders.
Though now double board certified in Neurology and Internal Medicine, Dr Genge was originally trained as a physio. It should come as no surprise therefore that Dr Genge is deeply committed to the development of better outcomes measures in Rare Diseases in Neurology, for which she is currently leading four global initiatives.
Large Patient Registry -based Drug Development for ALS
Gen SOBUE Japan
Professor Gen Sobue currently worked as a director of Brain and Mind Research Center at Nagoya University since 2015 and is a president at Aichi Medical University since 2019. He graduated from Nagoya University, School of Medicine in 1975. From 1981, he worked as a lecturer and an associate professor at Aichi Medical University. Professor Sobue worked as an assistant professor at the University of Pennsylvania, USA from1982 to 1985. He was appointed as a professor of neurology at Nagoya University Graduate School of Medicine in 1995. From 2009 to 2012, Professor Sobue served as the dean of Nagoya University Graduate School of Medicine.
Large Patient Registry -based Drug Development for ALS
Professor Gen Sobue currently worked as a director of Brain and Mind Research Center at Nagoya University since 2015 and is a president at Aichi Medical University since 2019. He graduated from Nagoya University, School of Medicine in 1975. From 1981, he worked as a lecturer and an associate professor at Aichi Medical University. Professor Sobue worked as an assistant professor at the University of Pennsylvania, USA from1982 to 1985. He was appointed as a professor of neurology at Nagoya University Graduate School of Medicine in 1995. From 2009 to 2012, Professor Sobue served as the dean of Nagoya University Graduate School of Medicine.
iPSC-based Drug Discovery and Development for ALS Medicine
Haruhisa INOUE Japan
Specialty: Department of Cell Growth And Differentiation
Professor Inoue is currently a Professor at CiRA, Kyoto University, Kyoto, Japan. He is also a Senior Visiting Scientist at RIKEN Center for Advanced Intelligence Project (AIP), Medical-risk Avoidance based on iPS Cells Team. Since 2017, Professor Inoue is the Team Leader at the RIKEN BioResource Research Center (BRC), iPSC-based Drug Discovery and Development Team. He was a postdoctoral fellow for a year at the Neuroregeneration Laboratory, McLean Hospital/Harvard Medical School, Boston, MA, USA in 2004. Aside from being a Research Resident in Kyoto University and National Institute of Neuroscience, Tokyo, he was also a Research Fellow at the Laboratory for Neuropathology, University Medical School of Pecs in Hungary.
iPSC-based Drug Discovery and Development for ALS Medicine
Specialty: Department of Cell Growth And Differentiation
Professor Inoue is currently a Professor at CiRA, Kyoto University, Kyoto, Japan. He is also a Senior Visiting Scientist at RIKEN Center for Advanced Intelligence Project (AIP), Medical-risk Avoidance based on iPS Cells Team. Since 2017, Professor Inoue is the Team Leader at the RIKEN BioResource Research Center (BRC), iPSC-based Drug Discovery and Development Team. He was a postdoctoral fellow for a year at the Neuroregeneration Laboratory, McLean Hospital/Harvard Medical School, Boston, MA, USA in 2004. Aside from being a Research Resident in Kyoto University and National Institute of Neuroscience, Tokyo, he was also a Research Fellow at the Laboratory for Neuropathology, University Medical School of Pecs in Hungary.
The ALSFRS-R: Continued Utility and Global Harmonization
Jeremy SHEFNER United States
Specialty: Neurology
Jeremy Shefner, MD, PhD, is Professor of Neurology at the Barrow Neurological Institute. Dr. Shefner is co-founder of the Northeast ALS (NEALS) Clinical Trials Consortium. He continues to direct NEALS outcomes and clinical monitoring cores. His research focuses on development of novel outcome measures for ALS trials.
Dr. Shefner has published more than 240 chapters and papers and has served on multiple grant review panels. In 2014, Dr. Shefner received the Sheila Essey Award for ALS Research, and in 2022 was an awardee of the Healey Center International Innovation Prize in ALS. He on the editorial boards of the ALS Journal and Neurotherapeutics. He is neuromuscular section editor of UpToDate.
The ALSFRS-R: Continued Utility and Global Harmonization
Specialty: Neurology
Jeremy Shefner, MD, PhD, is Professor of Neurology at the Barrow Neurological Institute. Dr. Shefner is co-founder of the Northeast ALS (NEALS) Clinical Trials Consortium. He continues to direct NEALS outcomes and clinical monitoring cores. His research focuses on development of novel outcome measures for ALS trials.
Dr. Shefner has published more than 240 chapters and papers and has served on multiple grant review panels. In 2014, Dr. Shefner received the Sheila Essey Award for ALS Research, and in 2022 was an awardee of the Healey Center International Innovation Prize in ALS. He on the editorial boards of the ALS Journal and Neurotherapeutics. He is neuromuscular section editor of UpToDate.
ALS – Roadmap to Therapies
Matthew KIERNAN Australia
Professor Matthew Kiernan is the Bushell Chair of Neurology at the University of Sydney and Co-Director of the Brain and Mind Centre. He is Chair of the World Federation of Neurology ALS/MND Specialty Group and established the Pan-Asian Consortium for Treatment and Research in ALS (PACTALS). He is President of the Brain Foundation and Editor Emeritus of the Journal of Neurology, Neurosurgery & Psychiatry.
ALS – Roadmap to Therapies
Professor Matthew Kiernan is the Bushell Chair of Neurology at the University of Sydney and Co-Director of the Brain and Mind Centre. He is Chair of the World Federation of Neurology ALS/MND Specialty Group and established the Pan-Asian Consortium for Treatment and Research in ALS (PACTALS). He is President of the Brain Foundation and Editor Emeritus of the Journal of Neurology, Neurosurgery & Psychiatry.
New Therapies for ALS
Merit CUDKOWICZ United States
Dr. Merit Cudkowicz is the Chief of the Massachusetts General Hospital Neurology Service, Director, Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and the Julieanne Dorn Professor of Neurology at Harvard Medical School in Boston. Dr. Cudkowicz is one of the founders and former co-directors of the Northeast ALS Consortium (NEALS), a group of over 140 clinical sites in the United States, Canada, Europe and the Middle East dedicated to performing collaborative academic-led clinical trials and research studies in ALS.
She is leading the first Platform Trial initiative in ALS and is also the Principal Investigator of the Clinical Coordination Center for the National Institute of Neurological Disorders and Stroke’s Neurology Network of Excellence in Clinical Trials (NeuroNEXT). Dr. Cudkowicz mentors neurologist in careers in experimental therapeutics.
New Therapies for ALS
Dr. Merit Cudkowicz is the Chief of the Massachusetts General Hospital Neurology Service, Director, Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and the Julieanne Dorn Professor of Neurology at Harvard Medical School in Boston. Dr. Cudkowicz is one of the founders and former co-directors of the Northeast ALS Consortium (NEALS), a group of over 140 clinical sites in the United States, Canada, Europe and the Middle East dedicated to performing collaborative academic-led clinical trials and research studies in ALS.
She is leading the first Platform Trial initiative in ALS and is also the Principal Investigator of the Clinical Coordination Center for the National Institute of Neurological Disorders and Stroke’s Neurology Network of Excellence in Clinical Trials (NeuroNEXT). Dr. Cudkowicz mentors neurologist in careers in experimental therapeutics.
Impairment of Proteostasis and Ribonucleostasis as Common Cell Death Mechanisms in ALS and Neurodegenerative Disorders
Seung-Hyun KIM South Korea
Dr. Seung H Kim is currently a neurology professor, director of the ALS clinic, and Korean NIH-sponsored Stem Cell Therapy Center at Hanyang Univ. Hospital, Seoul. And, He serves as President of the Korean Society of Neuromuscular Disorder and the Korean Society of ALS.
A therapeutic strategy using autologous Bone Marrow that originated Mesenchymal Stem Cell Therapy for ALS was approved as an orphan drug in the Korean NIH research project. He put autologous stem cell therapy into clinical practice to treat ALS and is now conducting translational research in the field of neurodegenerative diseases for the development of stratified and precision medicine based on the unique genetic background of the Korean and Asian populations.
Impairment of Proteostasis and Ribonucleostasis as Common Cell Death Mechanisms in ALS and Neurodegenerative Disorders
Dr. Seung H Kim is currently a neurology professor, director of the ALS clinic, and Korean NIH-sponsored Stem Cell Therapy Center at Hanyang Univ. Hospital, Seoul. And, He serves as President of the Korean Society of Neuromuscular Disorder and the Korean Society of ALS.
A therapeutic strategy using autologous Bone Marrow that originated Mesenchymal Stem Cell Therapy for ALS was approved as an orphan drug in the Korean NIH research project. He put autologous stem cell therapy into clinical practice to treat ALS and is now conducting translational research in the field of neurodegenerative diseases for the development of stratified and precision medicine based on the unique genetic background of the Korean and Asian populations.
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