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15th ALS Network Research Summit Agenda
What Can ALS Learn From Other Neurological Diseases?
DAY 1: THURSDAY, JANUARY 16, 2025
12:00 PM – 1:00 PM WELCOME LUNCH
1:00 PM – 1:10 PM INTRODUCTION
Clive Svendsen, PhD, Professor and Executive Director, BOG Regenerative Medicine Institute, Kerry and Simone Vickar Family Foundation Distinguished Chair in Regenerative Medicine, Cedars-Sinai;
Chair, ALS Network Scientific Advisory Committee
Aaron Gilter, PhD, Director, Taube/Koret Center for Neurodegenerative Disease Research
Senior Investigator and Associate Director, Gladstone Institute of Neurological Disease Professor Department of Neurology, Stanford University: Member, ALS Network Scientific Advisory Committee
Jonathan Day, MD, PhD, Professor of Neurology and Neurological Sciences, Stanford University
Sheri Strahl, MPH, MBA, President & CEO, ALS Network
SESSION I: FTD AND ALS – WHAT CAN ONE LEARN FROM THE OTHER?
Chair: Daniel Mordes, MD, PhD, University of California, San Francisco; Member, ALS Network Scientific Advisory Committee
1:10 PM – 1:40 PM OPENING PLENARY - The Pathology of FTD and How it Relates to ALS
Bill Seeley, MD, Professor of Neurology, Weill Institute for Neurosciences, University of California, San Francisco
1:40 PM – 2:00 PM
Rita Sattler, MSc, PhD, Barrow Neurological Institute; Member of the ALS Network Scientific Advisory Committee
2:00 PM – 2:20 PM
Edward B. Lee, MD, PhD, University of Pennsylvania
2:20 PM – 2:40 PM
Eric Huang, MD, PhD, University California San Francisco
2:40 PM – 3:00 PM DISCUSSION
3:00 PM – 3:15 PM BREAK and POSTERS
SESSION II: INDUSTRY AND CLINICAL TRIAL UPDATES
Chair: Jill Goslinga, MD, MPH, Assistant Professor, Neurology ALS and other neuromuscular patients, University California San Francisco Weill Institute for Neurosciences
Member of the ALS Network Scientific Advisory Committee
3:15 PM – 3:30 PM
QurAlis: Neuro Pioneers on Quest to Cure
ATAXIN Update
3:30 PM – 3:45 PM
Neuvivo Inc. – NP001
3:45 PM – 4:00 PM
Joe Lewcock, PhD, CSO - Denali Therapeutics
4:00 PM – 4:15 PM TBD
4:15 PM – 4:30 PM DISCUSSION
SESSION III: NEW CIRM FUNDED PROJECTS
4:30 PM – 4:50 PM
Correction of C9 using ASO
Claire Clelland, MD, PhD, University California San Francisco
4:50 PM – 5:10 PM
C9orf72 Repeat Expansion-Tuned Allelic Suppression by CRISPRi as an ALS Therapy
Ritchie Ho, PhD, Cedars-Sinai
5:10 PM – 5:30 PM DISCUSSION
5:30 PM – 7:00 PM POSTERS and RESEARCHER COCKTAIL HOUR
DAY 2: FRIDAY, JANUARY 17th, 2025
7:30 AM – 8:30 AM BREAKFAST and POSTERS
SESSION IV: NEUROMUSCULAR DISEASES AND ALS
Chair: Jonathan Day, MD, PhD, Professor of Neurology and Neurological Sciences, Stanford University
8:30 AM – John Day with introduction – Lessons from SMA.
MD, PhD, Professor of Neurology and Neurological Sciences, Stanford University
8:40 AM – 9:00 AM
Treating the Muscles in ALS; What are the Possibilities?
Helen Blau, PhD, Stanford University
9:00 AM – 9:20 AM
Kenneth Fischbeck, MD, National Institutes of Health
9:20 AM – 9:30 AM
Charlotte Sumner, MD, Johns Hopkins University
9:30 AM – 10:00 AM DISCUSSION
10:00 AM – 10:15 AM BREAK and POSTERS
SESSION V: TDP 43 AND ALS – WHAT’S NEW THIS YEAR?
Chair: Aaron Gitler, PhD, Genetics Professor, Stanford University
10:15 AM – 10:35 AM
Alternative Polyadenylation in ALS or Single Cell Transcriptomics of Motor Neuron Degeneration in ALS
Yi Zeng and Olivia Gautier – Gitler Lab
10:35 AM – 10:55 AM
New Mechanisms of TDP-43 Nuclear Export
Sitao Zhang – Cleveland Lab
10:55 AM – 11:15 AM
TDP- 43 Phase Separation
Jim Shorter, PhD, University of Pennsylvania
11:15 AM – 11:45 AM DISCUSSION
11:45 AM – 12:15 PM
Helping ALS Patients Speak Again with AI and Brain Machine Interface Systems
David Brandman, MD, PhD, University California Davis
12:15 PM – 12:30 PM DISCUSSION
AI and Language Processing and ALS
12:30 PM – 1:30 PM LUNCH and POSTERS
SESSION VI: THE WALS CLINICAL UPDATE
CHAIR: Jill Goslinga, MD, MPH, UCSF
1:30PM – 1:45 PM
Why have Phase III Trials Struggled to Replicate Phase II Trials
Jeremy Shefner, MD, PhD, Barrow Neurological Institute
1:45 PM – 2:00 PM
What is Working and What isn’t Working with Current Clinical Trial Outcome Measures
Angela Genge MD, McGill University
2:00 PM – 2:15 PM
Challenges and Opportunities in Clinical Trials
John Ravits, MD, University of California San Diego
2:15 PM – 2:30 PM
The Future of ALS Clinical Trials
Merit Cudkowicz, MD, Massachusetts General Hospital
2:00 PM – 3:00 PM DISCUSSION
Clinical Trials and ALS
3:00 PM – 3:15 PM BREAK
SESSION VII: SPORADIC ALS AND CIRM PROGRAMS
Justin Ichida, PhD, University Southern California
Member of the ALS Network Scientific Advisory Committee
3:15 PM – 3:35 PM
Clustering of ALS Cases Using ML-based Phenotyping of Patient Fibroblasts
Steven Altshuler, PhD and Lani Wu, PhD, University California San Francisco
3:35 PM – 3:55 PM
Tofersen and ALS – Where are We Now?
Stephanie Fradette, Pharm. D, Biogen.
3:55 PM – 4:15 PM
Development of a VAV2 Antisense Oligonucleotide (ASO) Treatment for ALS
Justin Ichida, PhD, University Southern California - CIRM DISC2 Project
4:15 PM – 4:35 PM
Development of an UNC13A ASO for ALS
Wen-Hsuan Chang, PhD AcuraStem – CIRM TRAN1 Project
4:35 PM – 4:45 PM DISCUSSION
4:45 PM – 5:15PM SPECIAL PLENARY LECTURE
Synaptic Plasticity in Alzheimer’s Disease – Lessons for ALS
Thomas Sudhof MD, PhD, Avram Goldstein Professor in the School of Medicine, Professor of Neurosurgery,
Nobel Laureate 2013
5:15 PM – 5:30 PM DISCUSSION
5:30 PM END OF DAILY SESSION
6:00 PM
CHAMPIONS FOR CURES AND CARE
(Link to Event at http://alschampions.org)
Cocktails, Dinner, Awards, and Entertainment
Announcement of the Barber ALS Research Award Winners
DAY 3: SATURDAY, JANUARY 18th, 2025
8:00 AM – 9:00 AM BREAKFAST
9:00 AM – 9:20 AM
Spotlight on the Barber ALS Research Award Winners
SESSION VIII: INDUSTRY AND CLINICAL TRIAL UPDATES II
Chair: Richard Smith, MD, Medical Director of the Center for Neurologic Study
Member of the ALS Network Scientific Advisory Committee
9:20 AM – 10:20 AM TO BE ANNOUNCED
10:20 AM – 10:30 AM DISCUSSION
10:30 AM – 10:45 AM BREAK
Session IX: ASSEMBLOID MODELS OF C9ORF ALS
10:45 AM – 11:00 AM
Longitudinal Characterization of Reproducible ALS Phenotypes in iPSC-derived Sensorimotor Organoids
Joáo Pereira, PhD, University of Alabama, Birmingham
11:00 AM – 11:15 AM
Benchmarking Region-specific Organoids and Cortico-motor Assembloids to Study ALS
Jimena Andersen, PhD, Emory University
11:15 AM – 11:30 AM
Probing Consistent and Reproducible Pathophysiological Phenotypes in the Novel Forebrain-spinal Circuitoid Models Derived from ALS Patients
Liang Oscar Qiang, MD, PhD, Drexel University and Daryl Bosco, PhD University of Massachusetts
11:30 AM – 11:45 AM
A Novel Human Cortico-spinal Assembloid Model of ALS/FTD
Kornelia Szenbenyi, PhD, HUN-REN Research Centre for Natural Sciences, Hungary
11:45 AM – 12:00 PM
Characterization of the Human ALS Motor Neuraxis Organoid System for Disease-Modeling
András Lakatos, PhD, University of Cambridge, UK
12:00 PM – 12:30 PM DISCUSSION
12:30 PM – 12:45 PM Closing Remarks and 2026 Planning
Clive Svendsen, PhD & ALS Network Scientific Advisory Committee Chair
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