November 16, 17 & 20th, 2023
Together from afar!
6TH ANNUAL ALS RESEARCH SYMPOSIUM
Uniting researchers, clinicians and individuals living with ALS from around the world for three days of sharing important ALS research and hope.
RECORDINGS: Links to view the recorded presentations for all talks (for which we were approved to record) are located beneath each talk title.
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 was an enormous success with over 980 registered attendees from 36 countries! Links to ALL RECORDINGS OF ALL SEGMENTS AND PRESENTATIONS from our 2022 Symposium are located under each talk title/speaker name on the 2022AGENDA tab of this website.
Hosted along with ALS ONE’s Research Team:
DAY 1 (November 16, 2023)
Keynote Speakers
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 an overview of the tremendous speaker lineup and topics which will be updated with confirmed talk titles and timing.
Keynote speakers and talk titles, along with moderators for Day 1 are below.
AGENDA OVERVIEW: talk titles are confirmed themes and will be updated soon.
DAY 1: THURSDAY, NOVEMBER 16, 2023 (all times EST).
DAY 1 (Thursday, 11/16): Keynote Speaker Day
10:30-10:45am: Welcome (Watch recording HERE)
SPEAKERS:
Merit Cudkowicz, MD, MSc.
Jennifer DiMartino, Executive Director, ALS ONE
Brooke Eby
Morning Session: CURRENT STATE OF ALS TRIALS
Moderator: James Berry, MD, MPH
10:45-11:00am: Clinical Trials landscape overview.
Speaker: Merit Cudkowicz, MD, MSc. Chief of Neurology and Director, Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and the Julieanne Dorn Professor of Neurology at Harvard Medical School.
(Watch recording HERE)
11:05-11:20am: Strategies and considerations for trial design in the new era of ALS research.
Speaker: Suma Babu, MBBS, MPH, Assistant Professor of Neurology, Harvard Medical School, ALS clinician at The Healey and AMG Center for ALS at Massachusetts General Hospital.
(Watch recording HERE)
11:20-11:30: Drs. Ayeez and Shelena Lalji & Family Student Scholar Award in Repair Mechanisms in ALS.
Speakers: Shelena Lalji, MD & award recipient & Deland Liu, award recipient.
(Watch recording HERE)
11:30-12:00pm: Asymptomatic gene carriers: Early diagnosis & treatment
Speaker: Tim Miller, MD, PhD, Vice Chair of Research for the department of Neurology and David Clayson Professor of Neurology at Washington University School of Medicine.
(Watch recording HERE)
12:00 – 12:30: Break/Lunch
Afternoon Session: NEXT GENERATION MOLECULAR THERAPIES IN ALS
Moderator: Sabrina Paganoni, MD, PhD
12:30 – 1:10pm: Broad overview of new gene modulation therapies.
TALK TITLE: A comparison of genetic therapies and their relevance to ALS.
Speaker: Jonathan Watts, PhD, Director of The Watts Group at UMass Chan Medical School.
(Watch recording HERE)
1:00 – 1:30pm: Divalent siRNA for SOD-1 suppression.
Speaker: Robert H. Brown, MD, DPhil, Director of the Day Neuromuscular Research Laboratory at the University of Mass. Medical School.
(Watch recording HERE)
1:30-2:00: Silencing FUS and other rare genetic forms of ALS.
Speaker: Neil Shneider, MD, PhD, Director, Eleanor and Lou Gehrig ALS Center at Columbia University Department of Neurology.
(Recording not available)
2:00 – 2:15: Break
2:15 – 2:45: Gene replacement therapy/viruses.
Speaker: Jerry Mendell, MD, Nationwide Children’s Hospital’s Center for Gene Therapy.
(Watch recording HERE)
2:45 – 3:15: “Individualized” investigational therapeutics for neurogenetic disease.
Speaker: Timothy Yu, MD, PhD, Attending Physician, Division of Genetics and Genomics at Boston Children’s Hospital; Associate Professor of Pediatrics at Harvard Medical School.
(Watch recording HERE)
3:15 – 3:30: Break
3:30 – 4:15: Panel discussion with the keynote speakers on topics of the day.
Moderated by Clotilde Lagier-Tourenne, MD, PhD
Keynote Speakers Panelists: Merit Cudkowicz, MD, MSc, Suma Babu, MBBS, MPH, Tim Miller, MD, PhD, Jonathan Watts, PhD, Robert Brown, MD, DPhil, Neil Shneider, MD, PhD, Jerry Mendell, MD, Tim Yu, MD, PhD.
ALS Community Panelist: Blaine Dangel
(Watch recording HERE)
DAY 2 (November 17, 2023)
Industry Presentations
A CALL FOR ABSTRACTS went out to the public from June 2023 until August 30, 2023 for researchers who were interested in speaking on Industry Day (November 17, 2023) during our 6th Annual ALS Research Symposium about an ALS treatment that they or their company/institute/pharma has in development. Anyone interested was asked to submit their abstract title and description as well as the speaker’s name and title by September 1, 2023 for consideration. We then selected 12 presentations from over 20 submissions..
Selections had no bearing on event sponsorships and vice-versa and no speakers for the entire conference are being compensated in any way.
DAY 2 (Friday, 11/17): Industry Day
9:15-9:45am Welcome
SPEAKERS:
Merit Cudkowicz, MD, MSc.
Jennifer DiMartino, Executive Director, ALS ONE
Della Larsen
9am - 12:00pm: INDUSTRY PRESENTATIONS:
Moderator: Fernando Vieira, MD, Chief Science Officer and CEO, ALS Therapy Development Institute.
9:45-10:05am: ACELOT
Speaker: Katie Planey, PhD, MBA
Title: Novel small molecule therapeutics for ALS reduce TDP43 and other amyloid related pathologies in vitro and in vivo by reducing cytoplasmic aggregates and increasing functional nuclear TDP43.
(Watch recording HERE)
10:05-10:25am: AC IMMUNE
Speaker: Tamara Seredenina, PhD
Title: Development of fluid and PET imaging biomarkers for detection of TDP-43 pathology. AND, Discovery and optimization of the first-in-class TDP-43 PET tracer.
(Watch recording HERE)
10:25-10:45am: AMYLYX PHARMACEUTICALS
Speaker: Jamie Timmons, MD
Title: Amylyx Update: CENTAUR and US EAP Data Analyses, Planning for PHOENIX, and New Research.
(Watch recording HERE)
10:45-11:00am: BREAK
11:00-11:20am: BRAINSTORM CELL THERAPEUTICS
Speaker: Bob Dagher, MD
Title: Biomarker exploration of NurOwn’s multimodal mechanism of action on neuroinflammation, neuroprotection and neurodegeneration.
(Watch recording HERE)
11:20-11:40am: CLENE NANOMEDICINE
Speaker: Rob Etherington, President and CEO
Title: CNM-Au8: Path Forward. Clene Nanomedicine clinical development review, advancing with Phase 3 Outcomes Trial.
(Watch recording HERE)
11:40-12:00pm: NEUROSENSE
Speaker: Shiran Zimri, PhD
Title: Shifting the Paradigm - A Biomarker Driven Approach for Studying Amyotrophic Lateral Sclerosis (ALS) Therapy Activity.
(Watch recording HERE)
12:00-12:30pm: LUNCH/BREAK
12:30pm-4:00pm: INDUSTRY PRESENTATIONS CONTINUED
Moderator: Robert H. Brown, MD, DPhil, Director of the Day Neuromuscular Research Laboratory at the University of Mass. Medical School.
12:30-12:50: PRILENIA
Speaker: Michal Geva, PhD
Title: Pridopidine For the Treatment of ALS – Top Line Results From the Phase 2 Healey ALS Platform Trial.
(Watch recording HERE)
12:50-1:10: QURALIS
Speaker: Angela Genge, MD, FRCP(C), eMBA
Title: QRL-201-01 - A multi-center, randomized, double-blind, placebo-controlled multipleascending dose study to evaluate the safety and tolerability of QRL-201 in Amyotrophic Lateral Sclerosis.
(Watch recording HERE)
1:10-1:30: SANOFI
Speaker: Nazem Atassi, MD, PhD
Title: A Phase 2 Trial of RIPK1 Inhibitor SAR443820 in Adult Participants with ALS (Himalaya Study): Study Design and Baseline Characteristics.
(Watch recording HERE)
1:30-1:45: BREAK
1:45-2:05: SOLA Biosciences
Speaker: Akinori Hishiya, PhD
Title: Preclinical proof of concept of SOL-257, a gene therapy targeting misfolded TDP-43 in ALS.
(Watch recording HERE)
2:05-2:25: SPINOGENIX
Speaker: Peter Vanderklish, PhD, CSO
Title: SPG302: a first-in-class synaptic regenerative therapy for ALS.
(Watch recording HERE)
2:25-2:45: TIKOMED
Speaker: Claus Sundgreen, MD
Title: ILB®: A Novel Therapeutic Agent for ALS Targeting Excitotoxicity, Mitochondrial Dysfunction, and Inflammation.
(Watch recording HERE)
2:45-3:00pm: BREAK
3:00-3:45: Roundtable Discussion about abstracts presented.
Moderator: Dr. Robert Brown, MD, DPhil.
Panelists from Acelot, AC Immune, Amylyx, Brainstorm, Clene, NeuroSense, Prilenia, QurAlis, Sanofi, Sola, Spinogenix, and Tikomed.
(Watch recording HERE)
3:45-4:00: Wrap up/closing remarks
The ALS Community: You are welcome!
We welcome all individuals impacted by ALS to attend our symposium to hear about important research and engage with those who are working so diligently to make a difference. We are honored to have seven powerful and inspiring individuals who are currently living with ALS join us to give remarks and join our panel discussions during the entire Symposium.
NEUROTECH DAY: MORNING SESSION
Watch Neurotech Morning Session recording HERE.
9:00 AM - 9:30 AM:
9:00am - 9:30am: Opening Remarks by EverythingALS and Team Gleason, with Keynote by Bill Nuti.
9:30 AM - 10:30 AM: Citizen-Driven Research: Unlocking the power of Agility and Timely Outcomes
Exploring the concept of citizen-driven research and its pivotal role in research, with an emphasis on fostering ethnic diversity and inclusivity.
An overview of EverythingALS' innovative approach to open collaboration and agile methodologies, spanning from care to cure.
The importance of co-creating with individuals impacted by ALS, creating a mutually beneficial feedback loop.
Investigating the transformative potential of multi-modal data and AI in the realm of clinical research.
Unveiling EverythingALS' stakeholder value maps, which encompass their platform, app, and motor examination tools.
Gaining valuable insights from the Radcliff Study Design and the utilization of passive data for digital biomarkers
10:30 AM - 11:30 AM: Future Opportunities of AI for Good in Healthcare: Insights from Global Industry Leaders
Identifying current unmet needs and the role of generative AI and tech innovations in enhancing care and finding a cure.
Gaining valuable perspectives through presentations by distinguished Fortune 500 corporations
Google
Microsoft
11:30 AM - 12:00 PM: Unique Approaches to Navigating Challenges and Opportunities
Engaging in an interactive Q&A and a panel discussion that brings together representatives from key players, including EverythingALS, Google, and Microsoft
BREAK
AFTERNOON SESSION: Implanted Brain-Computer Interfaces
Watch Neurotech Afternoon Session recording HERE.
1:00pm: Welcome/Intro
Speaker: Shelena Lalji, MD, F.A.C.O.G., Founder of ALS Heroes and The Dr. Shel Wellness & Aesthetic Center
1:10pm: BrainGate & Welcome. Speaker: Leigh Hochberg, MD, PhD, Director, Center for Neurotechnology and Neurorecovery, Neurocritical Care and Stroke Services, Department of Neurology, Massachusetts.
1:13pm: The Needs & Perspectives of pALS. Speaker: Zoe Lalji, Neuroscience and Entrepreneurship Major, UT Austin.
Academic Presentations:
Bringing Brain-Computer Interfaces to People’s Homes. Speaker: Jonas Zimmermann, PhD, Senior Scientist, Wyss Center.
BrainGate: Intracortical BCIs for the at-home restoration of speech, communication, and mobility. Speaker: Daniel Rubin, M.D., Ph.D. Physician Investigator (Cl) Neurology, Mass General Research Institute.
Creating BCIs for communication and digital embodiment. Speaker: David Moses, PhD, Adjunct Professor in the Edward Chang Lab at UCSF.
CortiCom: Clinical Trial of ECoG BCI. Speaker: Nathan Crone, MD, Professor of Neurology at The Johns Hopkins University School of Medicine.
Real-world application of implanted BCIs for people with ALS. Speaker: Mariska Vansteensel, PhD, Assistant Professor at the UMC Utrecht Brain Center in Utrecht.
Speaker: Brian Dekleva, PhD, Research Scientist, Rehab Neural Engineering Labs, University of Pittsburgh.
2:30pm: pALS Personal Experience with BCI: Pat Bennett
2:45pm: Panel & Roundtable Discussions:
Florian Solzbacher, PhD, Co-Founder, Executive Chairman & President, Blackrock Neurotech.
Kurt Haggstrom, Chief Commercial Officer (CCO) at Synchron.
Matt Angle, PhD, Founder & CEO at Paradromics.
Michael Mager, Co-Founder & CEO at Precision Neuroscience.
4:00pm Wrap-up
End of 2023 Symposium.
DAY 3 (November 20, 2023)
NEUROTECH DAY
This year, we are excited to once again collaborate Indu Navar, MSCS (EverythingALS), Shel Lalji, MD (ALS Heroes), and Leigh Hochberg, MD, PhD (Center for Neuro-technology and Neurorecovery at MGH), along with Team Gleason, to bring you “NEUROTECH NOW”, a segment dedicated to neurotechnology as it relates to ALS.
Join us for an illuminating conversation with industry leaders, including representatives from renowned companies such as EverythingALS, Microsoft, Apple, Google, Amazon, Service Now, Snowflake, Barracuda and more. The focus of this discussion will revolve around catalyzing new models, increasing efficiencies, and boosting productivity through generative AI automation of workflows. Furthermore, we will explore the role of emerging technologies in developing intelligent decision support systems and enhancing data analytics and reporting for applications in healthcare, clinical research, and care delivery for ALS patients. In the afternoon, we’ll hear the latest advances from academic and industry leaders in implanted Brain-Computer Interfaces, technologies being developed to restore communication and mobility for people with ALS and other forms of paralysis.
To become a sponsor of this year’s Symposium, please email us HERE.
Current list of generous 2023 Symposium sponsors:
EMERALD SPONSORS
SAPPHIRE SPONSORS
GOLD SPONSORS
SILVER SPONSORS
BRONZE SPONSORS
COPPER SPONSORS
