13th Annual California ALS Research Summit Schedule
All Times Listed Pacific Standard Time
DAY 1: FRIDAY, JANUARY 20, 2023
8:00 AM - 8:10 AM Introduction
Clive Svendsen, PhD, Chair, CA ALS Research Network; Executive Director, Chair, Board of Governors Regenerative Medicine Institute, Cedars-Sinai
Catherine Lomen-Hoerth, MD, PhD, Medical Director, The ALS Treatment & Research Center, UC San Francisco
Sheri Strahl, MPH, MBA, Chief Operating Officer, The ALS Association Golden West Chapter
SESSION I: DEBATE: SHOULD DRUGS SHOWING EFFICACY IN A PHASE II TRIAL FOR ALS BE APPROVED WITHOUT A PHASE III TRIAL?
Chair: Jeremy Shefner, MD, PhD, Barrow Neurological Institute
8:10 AM - 8:40 AM
NO: John Ravits, MD, UC San Diego Health
YES: Jeffrey Rosenfeld, MD, Loma Linda University
8:40 AM - 9:00 AM
Panel Discussion
Lucie Bruijn, PhD, MBA, Therapeutic Area Lead - Novartis
Merit Cudkowicz, MD, MSc - Massachusetts General Hospital
Angela Genge, MD, FRCP(C), eMBA, Chief Medical Officer - QurAlis
SESSION II: HOT TOPICS: TDP - 43, CRYPTIC EXONS AND SYNAPTOPATHY IN ALS
Chair: Justin Ichida, PhD, USC
9:00 AM - 9:20 AM
Don Cleveland, PhD, UC San Diego - Therapeutic Restoration of Stathmin-2 for TDP-43 Proteinopathies
9:20 AM - 9:40 AM
Aaron Gitler, PhD, Stanford - New TDP-43 Targets (And One New Drug)
9:40 AM - 10:00 AM
Rita Sattler, MSc, PhD, Barrow Neurological Institute - Mechanisms of RNA A-I editing Mediated Nuclear TDP-43 Export
10:00 AM - 10:30 AM
Discussion
10:30 AM - 10:45 AM BREAK
SESSION III: INDUSTRY AND CLINICAL TRIAL UPDATES I
Chair: Björn Oskarsson, MD, FAAN, Mayo Clinic
10:45 AM - 11:00 AM
Angela Genge, MD, FRCP(C), eMBA, Chief Medical Officer - QurAlis
11:00 AM - 11:15 AM
Stacy Lindborg, PhD, Co-Chief Executive Officer - Brainstorm Cell Therapeutics
11:15 AM - 11:30 AM
Stacy Rudnicki, MD, Vice President of Clinical Research, Therapeutic Area Lead - Cytokinetics
11:30 AM - 11:45 AM
Linus D. Sun, MD, PhD, Associate Medical Director, Early Clinical Development - Denali Therapeutics
11:45 AM - 12:15 PM LUNCH BREAK
12:15 PM - 1:15 PM
Poster Session
SESSION IV: GENE AND ASO TREATMENTS FOR ALS – WHERE ARE WE NOW?
Chair: Brian K. Kaspar, PhD, CSO, Consultant
1:15 PM - 1:35 PM
Lucie Bruijn, PhD, MBA, Therapeutic Area Lead, Novartis - Advances in Gene Therapies and Challenges for CNS disorders
1:35 PM - 1:55 PM
Martin Marsala, MD, Professor, UC San Diego - Subpial AAV-Based Gene Delivery for Treatment of ALS: Maximizing the Potency While Minimizing Systemic Toxicity
1:55 PM - 2:15 PM
C. Frank Bennett, PhD, Senior Vice President of Research, Ionis Pharmaceuticals - SOD1 Trial for ALSION363 (Jacifusen) An Antisense Drug as A Potential Treatment for ALS Patients with Fus Mutations
2:15 PM - 2:45 PM
Discussion
2:45 PM - 3:00 PM BREAK
SESSION V: STEM CELL CLINICAL TRIALS AND MODELING FOR ALS IN THE STATE OF CALIFORNIA
Chair: Leslie Thompson, PhD, UC Irvine
3:00 PM - 3:15 PM
Lila R. Collins, PhD, Associate Director, Therapeutics Development, CIRM - CIRM Opportunities and Strategic Infrastructure
3:15 PM - 3:35 PM
Richard Lewis, MD, Cedars-Sinai - Cedars-Sinai CIRM Stem Cell Transplant Program for ALS: An Update on Current Clinical Trials
3:35 PM - 3:55 PM
Justin Ichida, PhD, USC - Using A Diverse ALS Stem Cell Panel to Identify Potential Drug Targets with Broad Efficacy
3:55 PM - 4:15 PM
Jimena Andersen, PhD, Emory University - Human 3D Cortico-Motor Assembloids to Model ALS
4:15 PM - 4:35 PM
Clive Svendsen, PhD, Chair, California ALS Research Network, Cedars-Sinai - Large Scale Differentiation of iPSC-Derived Motor Neurons from ALS Patients
4:35 PM - 5:00 PM
Discussion
6:00 PM
Cocktails
7:00 PM
CHAMPIONS FOR CURES AND CARE (Link to Event at http://alschampions.org)
Cocktails, Dinner, Awards, and Entertainment
DAY 2: SATURDAY, JANUARY 21, 2023
8:00 AM - 9:00 AM Breakfast
9:00 AM - 9:10 AM Introduction
Clive Svendsen, PhD, Chair, CA ALS Research Network; Executive Director, Chair, Board of Governors Regenerative Medicine Institute, Cedars-Sinai
Fred Fisher, MSW, LCSW, President & CEO, The ALS Association Golden West Chapter
9:10 AM - 9:30 AM
Presentations from the 2023 Barber ALS Research Award Winners
9:30 AM - 10:00 AM
Plenary I: Merit Cudkowicz, MD, MSc, Massachusetts General Hospital – The Healy Platform Trial, Innovative Trials & Other Approaches
10:00 AM - 10:15 AM
Discussion
SESSION VI: INDUSTRY UPDATES II
Chair: Lucie Bruijn, PhD, MBA, Therapeutic Area Lead, Novartis
10:15 AM - 10:30 AM
Peter Vanderklish, PhD, Head of Biology, Amydis – TDP-43 in the Eye: A Biomarker for ALS?
10:30 AM - 10:45 AM
Stephanie Fradette, Biogen
10:45 AM - 11:00 AM
Robert H. Scannevin, PhD, Chief Scientific Officer, Verge - Discovery and Development of PIKfyve Inhibitors for the Treatment of ALS
11:00 AM - 11:15 AM
Sam Alworth, MS, MBA, CEO, AcuraStem - Discovery and Development of A PIKFYVE Antisense Oligonucleotide Therapeutic for Diverse Forms of ALS and FTD
11:15 AM - 11:30 AM Break
Session VII: AT HOME CARE AND GENETIC TESTING AND ENCOURAGING DEI IN CALIFORNIA
Chair: John Ravits, MD, UC San Diego Health
11:30 AM - 11:50 AM
Björn Oskarsson, MD, FAAN, Mayo Clinic - Standards for Home Care
11:50 AM - 12:10 PM
Matthew Harms, MD, Columbia University – How Has Universal Testing Changed Clinical Care and Clinical Trials/Single Nucleotide Effects on Protein etc.?
12:10 PM - 12:30 PM
Catherine Lomen-Hoerth, MD, PhD, UC San Francisco – How Do We Increase DEI in Both Research and Patient Trial Selection: Challenges and Opportunities
12:30 PM - 1:00 PM
Discussion
1:00 PM - 2:00 PM Lunch Break
SESSION VIII: THE SIGMA RECEPTOR AND ALS
Chair: Richard Smith, MD, Center for Neurological Study
2:00 PM - 2:20 PM
Vladimir Zhemkov, PhD, Cedars-Sinai - The Role of the Sigma-1 Receptor in The Endoplasmic Reticulum: Relevance for ALS
2:20 PM - 2:40 PM
John Ravits, MD, UC San Diego Health - Sigma Receptor Binding in the Human Motor Cortex
2:40 PM - 3:00 PM
Richard Smith, MD, Center for Neurological Study - Novel Sigma 1 Therapeutics
3:00 PM – 3:15 PM Break
SESSION IX: DETECTING ALS EARLY AND FOLLOWING PROGRESSION – NEW TOOLS AND IDEAS
Chair: Catherine Lomen-Hoerth, MD, PhD, UC San Francisco
3:15 PM - 3:35 PM
Timothy Miller, MD, PhD, ALS Center Co-Director, Washington University - Following Pre-Symptomatic ALS Gene Carriers: What Can We Learn About a Prodromal Period in ALS?
3:35 PM - 3:55 PM
Hiroshi Mitsumoto, MD, Columbia University - Hastening the Diagnosis of Amyotrophic Lateral Sclerosis
3:55 PM - 4:25 PM
Robert Bowser, PhD, CSO, Barrow Neurological Center - CSF Biomarkers That Predict Disease Progression
4:25 PM - 4:50 PM
Discussion
4:50 PM - 5:00 PM
Closing Remarks and Thoughts for 14th California ALS Research Summit