November 13, 14 & 15th, 2024
Together from afar! 100% via Zoom to allow anyone from anywhere in the world to attend at no cost and without the impact of travel.
7TH ANNUAL ALS RESEARCH SYMPOSIUM
A NOTE ABOUT RECORDINGS: LINKS TO THE RECORDINGS OF ALL PRESENTATIONS AND SPEAKERS ARE LOCATED UNDER EACH TALK TITLE IN THE AGENDA’S BELOW.
Our annual Symposium unites researchers, clinicians and individuals living with ALS from around the world for three days of sharing important ALS research and hope.
“This Symposium is one of the best that I’ve ever attended in all the ten years that I’ve been working in ALS. Thank you for all your hard work on such an outstanding and important meeting”
Our annual symposium is hosted by our ALS ONE research team: Dr. Robert Brown, MD, DPhil, of UMass Memorial Medical School; Dr’s Merit Cudkowicz, MD, MSc, James Berry, MD, MPH, and Sabrina Paganoni, MD, PhD, of the Sean M Healey & AMG Center for ALS at Massachusetts General Hospital; and Dr. Fernando Vieira, MD, of The ALS Therapy Development Institute. This year’s symposium promises to be a robust and informative forum to discuss and review current research that is underway across the globe to help uncover therapeutic options leading to an ultimate cure for Amyotrophic Lateral Sclerosis (ALS). We believe that we are stronger when we are sharing together and our annual conference has proven to be a tremendous forum to do just that.
Last year’s Symposium, held November 16-18th 2023, was an enormous success with over 900 registered attendees from 35 countries! Links to ALL RECORDINGS OF ALL SEGMENTS AND PRESENTATIONS from our 2023 Symposium and our 2022 Symposium, are located under each talk title/speaker name on the tabs above on this website.
UNITY BRINGS US CLOSER TO A WORLD WITHOUT ALS
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UNITY BRINGS US CLOSER TO A WORLD WITHOUT ALS ⬆️
Hosted along with ALS ONE’s Research Team:
DAY 1: November 13, 2024
Keynote Speakers
Renowned Keynote speakers will give presentations and share insight on many of today’s most relevant and important topics in the world of ALS research.
Below is our full lineup of Day 1’s Speakers and Moderators.
DAY 1: NOV. 13 (Keynote Speaker Day) all times EST
10:00am-10:15am: Opening Remarks:
Dr. Merit Cudkowicz, MD, MSc
Jennifer DiMartino, Executive Director of ALS ONE
Chuck and Nicole Johnson, family living with ALS.
Watch the recording of Chuck & Nicole’s inspiring talk HERE.
Morning Session
10:15 – 12:00: CURRENT STATE OF ALS TRIALS.
Moderator: Dr. Sabrina Paganoni, MD, PhD
10:15am – 10:30am (15 min)
Speaker: Dr. Sabrina Paganoni, MD, PhD
Talk Title: Clinical Trials landscape overview.
Watch the Recording HERE.
10:30am-10:45am (15 min)
Speaker: Dr. Jeremy Shefner, MD, PhD, FAAN
Talk Title: Outcome Measures in ALS: Newer Possibilities and Old Ones Reconsidered
Watch the Recording HERE.
10:45am – 11:00am (15 min)
Speaker: Dr. Melanie Quintana, PhD
Talk Title: Evidence-Based Clinical Trial Design in ALS
Watch the Recording HERE.
11:00am – 11:15am (15 min)
Speaker: Dr. James Berry, MD, MPH
Talk Title: An overview of the ALS clinical research consortium called Access for All in ALS (ALL ALS)
Watch the Recording HERE.
11:15am – 11:30am (15 min)
Speaker: Dr. Jaime Raymond, MPH
Talk Title: National Amyotrophic Lateral Sclerosis (ALS) Registry: Identification of ALS Risk Factors.
Watch the Recording HERE.
11:30am-11:45am (15 min)
Speaker: Dr. Sheena Chew, MD
Talk Title: How to use Neurofilament as a tool in ALS clinical trials.
Watch the Recording HERE.
11:45am-12:10pm (25 Min): PANEL DISCUSSION OF MORNING SPEAKERS
Moderator: Dr. Sabrina Paganoni, MD, PhD
Watch the Recording HERE.
12:15pm – 12:45pm: 30 MIN LUNCH/BREAK
Afternoon Session: 12:45 – 4:30pm NEW APPROACHES TO UNDERSTANDING SPORADIC ALS.
Moderator: Dr. Robert Brown, MD, DPhil
12:45pm – 1:10pm (20 min talk + 5 min Q&A)
Speaker: Dr. Ammar Al-Chalabi, PhD
Talk Title: The multistep hypothesis of ALS.
Watch the Recording HERE.
1:10pm – 1:35p (20 min talk + 5 min Q&A)
Speaker: Dr. Eran Hornstein, PhD
Talk Title: miRNAs in ALS: mechanism, diagnosis and therapy development.
Watch the Recording HERE.
1:35pm – 2:00p (20 min talk + 5 min Q&A)
Speaker: Dr. Justin Ichida, PhD
Talk Title: Using iPSCs to model sporadic ALS and evaluate therapeutic strategies.
Watch the Recording HERE.
2:00pm – 2:25p (20 min talk + 5 min Q&A)
Speaker: Dr. Eva Feldman, MD, PhD
Talk Title: The ALS Exposome: Where Science Meets Patient Care
Watch the Recording HERE.
2:25pm-2:40pm: 15 MIN BREAK
2:40pm – 3:05pm (20 min talk + 5 min Q&A)
Speaker: Dr. Zinan Zhou, PhD
Talk Title: Somatic mosaicism in amyotrophic lateral sclerosis and frontotemporal dementia.
Recording unavailable for sharing.
3:05pm – 3:30pm (20 min talk + 5 min Q&A)
Speaker: Dr. Dame Pamela Shaw, DBE, MBBS, MD, FRCP, FMedSci, FAAN, FANA, FAAAS
Talk Title: Subclassification and therapeutic approaches for sporadic ALS.
Watch the Recording HERE.
3:30p-3:45p: 15 MIN BREAK
3:45p – 4:30p (45 min panel discussion)
Panel Discussion with afternoon speakers.
Moderator: Dr. Robert Brown, MD, DPhil
Watch the Recording HERE. (This video also contains Dr. Pamela Shaw’s talk just prior to the panel).
DAY 2: November 14, 2024
Industry Presentations
A CALL FOR ABSTRACTS went out to the public from June 2024 until August 31, 2024 for researchers who were interested in speaking on Industry Day (November 14, 2024) during our 7th Annual ALS Research Symposium about an ALS treatment that they or their company/ institute/pharma has in development.
Industry Day Agenda (all times are EST):
9:00am – 9:10am: Dr. James Berry, MD, MPH, Jennifer DiMartino
9:10 - 9:30am: The Lalji Student Scholar Award presentation: Anushka Sanyal, undergrad student studying Human Biology at Stanford University. Presented by Dr. Ghazaleh Sadri-Vakili, MS, PhD , Assistant Professor of Neurology at the Harvard Medical School and Assistant in Neuroscience at the Massachusetts General Hospital.
AWARDEE: Anushka Sanyal, Undergrad Student studying Human Biology at Stanford University.
Watch the Recording HERE.
Session 1: 1 Hour & 20 min. Each presentation: 20 minutes in total w/Q&A.
Moderator: Dr. James Berry, MD, MPH
9:30 – 9:50: Neurizon Therapeutics (formerly PharmAust)
Title: Exploring the Benefits of mTOR Inhibition in Patients with Amyotrophic Lateral Sclerosis.
o Speaker: Dr Michael Thurn, Managing Director
Watch the Recording HERE.
9:50 – 10:10: Zydus Lifesciences
Title: A phase 2, proof-of-concept, placebo controlled, randomized, multicenter, doubleblind study of Usnoflast (ZYIL1) to evaluate the efficacy, safety, tolerability, pharmacokinetics, and pharmacodynamics in patients with amyotrophic lateral sclerosis.
o Speaker: Dr. Maulik S. Doshi, MD, DM, General Manager Clinical Research and Development
Recording not available for viewing.
10:10 – 10:30: Neurosense
Title: Shifting the PARADIGM: PrimeC, an Oral Candidate for ALS, Demonstrates Safety, Efficacy, and Target Engagement Biomarkers in a 12-Month, Phase 2b Trial.
o Speaker: Dr. Shiran Zimri, PhD, VP of R&D and Dr. Merit Cudkowicz, MD, MSc.
Watch the Recording HERE.
10:30 – 10:50: Kadimastem
Title: Safety and efficacy of first-in-man intrathecal injection of human astrocytes (AstroRx®) in ALS patients: Preclinical and phase I/IIa clinical trial results.
o Speaker: Dr. Kfir Molakandov PhD, VP Research and Development.
Watch the Recording HERE.
BREAK: 10:50-11:00 (10 min)
Session 2: 1 Hour & 20 min. Each presentation: 20 minutes in total w/Q&A.
Moderator: Dr. James Berry, MD, MPH
11:00 – 11:20: PLL Therapeutics
Title: A new diagnostic and therapy paradigm for ALS.
o Speaker: Dr. Jean-Pascal Zambaux, CEO.
Watch the Recording HERE.
11:20 – 11:40: Aclipse Therapeutics
Title: M102, an Nrf2 and HSF1 activator, entering Phase 1 for the treatment of Amyotrophic Lateral Sclerosis.
o Speaker: Dr. Ira Kalfus, MD, Chief Medical Officer.
Watch the Recording HERE.
11:40 – 12:00pm: Mitochon Pharmaceuticals
Title: Micro-doses of Mitochondrial uncoupler MP101 in hSOD1 G93A Provides the First Ever Small Molecule with Functional Recovery from Paralysis and Hints that ALS is a Solvable Problem.
o Speaker: Dr. John G. Geisler, PhD, CSO.
Watch the Recording HERE.
12:00 – 12:20: Spinogenix
Title: Clinical advancement of SPG302 as a first-in-class synaptic regenerative therapy for ALS.
o Speaker: Dr. Peter Vanderklish, PhD, CSO.
Watch the Recording HERE.
BREAK/LUNCH 12:20 – 1pm.
Session 3: 1 Hour & 20 min. Each presentation: 20 minutes in total w/Q&A.
Moderator: Dr. Fernando Vieira, MD
1:00 – 1:20: Acelot
Title: Novel oral small molecule for sporadic ALS treats both gain- and loss-of-function TDP-43 pathology, restores cryptic mis-splicing in vitro and extends survival and decreases NfL in vivo.
o Speaker: Dr. Katie Planey PhD, MBA.
Watch the Recording HERE.
1:20 – 1:40: Modality.ai
Title: Multimodal Speech Biomarkers for Remote Monitoring of ALS Disease Progression.
o Speaker: Dr. David "DSO" Suendermann-Oeft, PhD, CEO.
Watch the Recording HERE.
1:40 – 2:00: OrphAI Therapeutics
Title: OrphAI Therapeutics, Apilimod dimesylate development in Amyotrophic Lateral Sclerosis.
o Speakers: Suma Babu, MBBS, MPH, Co-director of the Neurological Clinic Research Institute at Massachusetts General Hospital and Principal Investigator of Phase 2a trial of AIT-101 in C9orf72 ALS; Brigette Roberts, MD, CEO OrphAI Therapeutics Inc.
Watch the Recording HERE.
2:00 – 2:20: Transposon Therapeutics
Title: A Phase 2A study of TPN-101, a nucleoside reverse transcriptase inhibitor, in patients with C9ORF72-related ALS/FTD.
o Speaker: Dr. Andrew Satlin, MD, Chief Medical Officer, Transposon.
Watch the Recording HERE.
BREAK: 2:20 – 2:40pm (20 min)
Session 4: 1 Hour & 40 min. Each presentation: 20 minutes in total w/Q&A.
Moderator: Dr. Fernando Vieira, MD
2:40 – 3:00: Eikonizo Therapeutics
Title: Development of a highly selec3ve CNS-penetrant small molecule HDAC6 inhibitor for the treatment of ALS and companion PET imaging technology to measure in vivo target occupancy.
o Speaker: Dr. Tonya Gilbert, PhD. Executive Director of Translational Research.
Watch the Recording HERE.
3:00 – 3:20: Leal Therapeutics
·Title: Development of LTX-002, an ASO for the treatment of ALS.
o Speaker: Dr. Asa Abeliovich, CEO and Founder, Leal Therapeutics.
Recording not available for viewing.
3:20 – 3:40: UniQure
Title: A Phase 1/2, Multicenter Study to Evaluate the Safety, Tolerability, and Exploratory Efficacy of Intrathecally Administered Gene Therapy AMT-162 in Patients with SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS).
o Speaker: Dr. Brenda Vicenzi, Executive Director, Clinical Development.
Watch the Recording HERE.
3:40 – 4:00: Arbor Biotechnologies
Title: Gene Editing with a Type V CRISPR-Cas Enzyme Disrupting the Aberrant Splicing of STMN2 as a Potential Treatment for ALS.
o Speaker: Dr. Priscilla Negraes, PhD, Associate Director CNS Therapies.
Recording not available for viewing.
BREAK: 4:00 – 4:10pm (10 min)
4:10 – 4:30: Amylyx Pharmaceuticals
Title: Amylyx Update on Development of AMX0114, an Antisense Oligonucleotide Targeting Calpain-2.
o Speaker: Dr. Lauren Kett, MD, PhD; Head, Medical Director of Clinical Development.
Watch the Recording HERE.
4:30 – 4:50: Axoltis Pharma
Title: Phase II clinical trial evaluating NX210c drug candidate efficacy in ALS patients (“SEALS”): rational and innovative design.
o Speaker: Dr Annette Janus, MD – Chief Medical Director.
Watch the Recording HERE.
4:50 – 5:10: Prilenia
Title: Pridopidine For the Treatment of ALS – Significant Improvements in Definite, Probable, and Early (<18mo from onset) Subjects in the Phase 2 Healey ALS Platform Trial.
o Speaker: Dr. Michael R Hayden, MBChB, PhD, CEO.
Watch the Recording HERE.
5:30pm EST Closing Remarks/End Day 2
DAY 3: November 15, 2024
NEUROTECH DAY
Watch the full Neurotech Day recording HERE.
10:00am -10:15am - Opening Remarks
James Berry, MGH
Lyle Ostrow, Temple University
10:15am -11:00am - Sensor Labs: Scaling Innovation & Avoiding Status Quo
Moderator: Lyle Ostrow, M.D. Ph.D
Nadine Tatton, CPath
Patrick Mercier, UCSD, Center for Wearable Sensors
Ajay Verma, Neurologist + Venture Capitalist
11:00am -11:45am - From Nice to Have to “Must Have” - What it Takes
Moderator: Karl Sillay, M.D.
Alvaro Pascual-Leone, Linus
David Suendermann-Oeft, Modality
Dmitriy Richard Starson, Passio
Ernest Fraenkel, MIT
11:45am -12:00pm - Demo AI in ALS CEP+ Patient Story
Speaker: Shawn Penno, Person Living with ALS & PathFinder
Natalia Luchkina, EverythingALS
Stephanie Henze, EverythingALS
12:00pm - 12:30pm - Big Tech and ALS: GenAI & AI for Good
Moderator: Dao Jensen, Compass UOL
Kely Norel, IBM
Luca Foschini, Sage Bionetworks
Geoff Green, Salesforce
12:30pm-2:15pm - BCI Discussion and Q&A
Intro and moderator - Leigh Hochberg, MGH
Kurt Haggstrom, Synchron
Romina Nejad, Neuralink
Nick Ramsey, University Medical Center Utrecht, Netherlands
Sergey Stavisky UC Davis
Benjamin Rapoport, Precision Neuroscience
Matt Angle, Paradromics
Rob Franklin, Blackrock
Jessica Kelemen,iBCI-CC
Watch the full Neurotech Day recording HERE.
2:15pm-2:25pm - Closing Remarks
Indu Navar, EverythingALS and Jen DiMartino, ALS ONE
The ALS Community: You are welcome!
We welcome all individuals impacted by ALS to attend our symposium so that they can hear about important research and engage with those who are working so diligently to make a difference. Last year, were were honored to have six powerful and inspiring individuals (at left) who are currently living with ALS join us to give remarks and join our panel discussions. This year, we hope you’ll join us to learn more about Chuck and Nicole Johnson’s inspiring journey living with ALS, which they are courageously maneuvering with the help of their “Chucklings”.
