Optimizing Collaboration
In the past, ALS research has been fractured and duplicative with researchers working in silos—that environment creates a breeding ground for redundancy and inefficiency
The ALS ONE partnership unites leading ALS experts from a wide array of leading institutions as one team working toward a common goal. These stakeholders regularly communicate, share data, discuss findings, and establish best practices. This partnership improves coordination among research, care, and treatment teams, ultimately reducing the duplication of efforts.
The unprecedented linking of minds and resources from Massachusetts General Hospital (MGH), University of Massachusetts Medical Center, ALS Therapy Development Institute (ALS TDI), and Compassionate Care ALS (CCALS) is unique for its leadership in efficiency, dedication to innovative research, and commitment to increasing access to care and treatment.
From The Healey ALS Platform Trial at MGH, the very first ever of it’s kind, ALS TDI’s AT-1501 (Anti-CD40-Ligand Antibody) trial, to the anti-sense oligonucleotide (ASO) trial to target the C9orf72 gene under the direction of Dr. Brown at UMass Memorial; among many others, you can feel confident that your donations are impacting cutting edge and promising collaborative research. Meet our team
Bridging the gap between the ALS community and the brilliant minds behind pivotal ALS research publications.
We’re excited to announce a new resource initiative for the ALS community! Introducing our ALS Research Publication Review series, designed to connect the ALS community with the brilliant minds behind important ALS research publications. Each session will delve into a different publication, offering insights to foster a broader and clearer understanding of the content. We’re proud to collaborate with NEALS on this dynamic new series, and we invite you to watch the inaugural review, where we delve into the publication entitled, which was aired on May 14th at 4 pm EST via Zoom.
REVIEW 1: “Incorporating Genetic Testing into the Care of Patients with Amyotrophic Lateral Sclerosis/Frontotemporal Degeneration Spectrum Disorders”.
Our first review, aired on May 14, 2024, spotlights the publication entitled, “Incorporating Genetic Testing into the Care of Patients with Amyotrophic Lateral Sclerosis/Frontotemporal Degeneration Spectrum Disorders”, and we’re honored to delve into it with two of its authors, Laynie Dratch, ScM, CGC, from the UPenn Neurogenetics Therapy Center, and Lauren Lichten, MS, CGC, from the Emory ALS Clinic with us, along with our panel for this review, which includes Mandi Bailey from the NEALS Patient Advisory Council, Jennifer DiMartino, ALS ONE’s Executive Director, Justin Kwan, MD, Director of the Neurodegenerative Disorders Clinic at the National Institute of Neurological Disorders and Stroke (NINDS), and NEALS member, as well as David Shulman, who is a NEALS Research Ambassador and International Patient Fellow.
We also invite you to read the publication, “Incorporating Genetic Testing into the Care of Patients with Amyotrophic Lateral Sclerosis/Frontotemporal Degeneration Spectrum Disorders,” in its entirety, by going to: https://bit.ly/IGTICOPALS.
REVIEW 2: “Evidence-based consensus guidelines for ALS genetic testing and counseling”.
Our 2nd review of our first series under ALS Genetic Testing and Counseling, was aired on June 11, 2024, spotlighting the publication entitled, “Evidence-based consensus guidelines for ALS genetic testing and counseling”, with two of its authors, Dr. Matthew Harms, MD, Associate Professor of Neurology at Columbia, and Jennifer Roggenbuck, MS, CGC, Genetic Counselor and Assoc Prof of Neurology at Ohio State Wexner Med Ctr. Panelists for this webinar included Dr. Christina Fournier, MD, MSc, Associate Professor of Neurology at Emory Univ. & NEALS NICE Committee member; Lori Andre, who is a NEALS Research Ambassador & Patient Advisory Council Member; Paula Trefiak, a NEALS Research Ambassador and International Patient Fellow, along with our Executive Director, Jennifer DiMartino.
We also invite you to read the publication in its entirety, by clicking HERE.
REVIEW 4: “Identification of Therapeutic Targets for ALS Using PandaOmics – An AI-Enabled Biological Target Discovery Platform.”
Our 1st review of two under the umbrella of Artificial Intelligence (AI) in ALS, aired on November 26, 2024, spotlighting the publication entitled, “Identification of Therapeutic Targets for ALS Using PandaOmics – An AI-Enabled Biological Target Discovery Platform.” We were honored to be joined by one of the publication’s esteemed authors, Dr. Jeffrey Rothstein, MD, PhD, The John W. Griffin Director, Brain Science Institute Professor of Neurology and Neuroscience, Director and Founder, Robert Packard Center for ALS Research. Joining our Executive Director, Jennifer DiMartino on the panel for this review, is Brian Andre, NEALS Research Ambassador; Dr. Christina Fournier, MD, MSc, Associate Professor, Neurology, Emory University, and NEALS NICE Committee Co-Chair; and Kevin Robinson, NEALS Research Ambassador. We hope you enjoy the webinar and we thank all who so generously participated to help the ALS community.
We also invite you to read the publication in its entirety, by clicking HERE.
REVIEW 5: “What do you think caused your ALS? An analysis of the CDC national amyotrophic lateral sclerosis patient registry qualitative risk factor data using artificial intelligence and qualitative methodology”
Our 2nd review of two under the umbrella of AI in ALS, aired on December 17, 2024, spotlighting the publication entitled, “What do you think caused your ALS? An analysis of the CDC national amyotrophic lateral sclerosis patient registry qualitative risk factor data using artificial intelligence and qualitative methodology.” We were honored to be joined by two of the publication’s highly regarded authors, Dr. Danielle Boyce, DPA, MPH, Principal Investigator, Real World Experience at ALS TDI and Jamie Raymond, MPH, Epidemiologist at the CDC, along with panelists: Dr. John Novak, MD, Neurologist at OhioHealth; Dr. Nadia Sethi, NEALS PEACe Committee Co-Chair; Brian Andre, NEALS Research Ambassador; and Jen DiMartino, Executive Director of ALS ONE. This webinar features a poignant conversation about the analysis of an important survey that was completed by over 3,000 participants in the Centers for Disease Control (CDC) and Prevention’s National ALS Registry who answered the question, “What do you think caused your ALS?” We hope you enjoy the webinar and we thank all who so generously participated to help the ALS community.
We also invite you to read the publication in its entirety, by clicking HERE.
Our 6th Annual ALS ONE Research Symposium was held on November 16, 17 and 20, 2023. Hosted along with our research team: Dr’s Merit Cudkowicz, MD, MSc of MGH, Robert Brown, MD, DPhil of UMass Medical School, James Berry, MD, MPH of MGH, and Fernando Vieira, MD of ALS TDI, and held virtually – enabling attendees from 36 countries to attend with ease, our Symposium was an overwhelming success. The conference had over 890 registered guests, including researchers, scientists, and members of the ALS community from around the world, convening virtually for three days to share outcomes and discoveries of critical research that is underway across the globe to help uncover therapeutic options leading to an ultimate cure for Amyotrophic Lateral Sclerosis (ALS). Please click HERE to view the official digital Symposium Speaker and Sponsor Guide. Recordings were made of the presentations which we were permitted to share and they are listed and linked below.
DAY 1: November 16, 2023 – Keynote Speakers
Symposium Segment: Day 1, 11/16/23: Keynote Speakers
Presentation Title: Opening Remarks by ALS ONE Executive Director Jennifer DiMartino, with special guest, Brooke Eby on her inspiring journey with ALS since being diagnosed at age 33.
Moderated by Dr. James Berry, MD, MPH, of The Healey and AMG Center for ALS at MGH.
Click HERE to view this video in its entirety.
Symposium Segment: Day 1, 11/16/23: Keynote Speakers
Segment Title: CURRENT STATE OF ALS TRIALS
Presentation Title: Clinical Trials Landscape Review.
Speaker: Dr. 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.
Moderator: Dr. James Berry, MD, MPH
Click HERE to view this video in its entirety.
Symposium Segment: Day 1, 11/16/23: Keynote Speakers
Segment Title: CURRENT STATE OF ALS TRIALS
Presentation Title: Strategies and considerations for trial design in the new era of ALS research.
Speaker: Dr. Suma Babu, MBBS, MPH, Assistant Professor of Neurology, Harvard Medical School and ALS clinician at The Healey and AMG Center for ALS at Massachusetts General Hospital. Moderator: Dr. James Berry, MD, MPH
Click HERE to view this video in its entirety.
Symposium Segment: Day 1, 11/16/23: Keynote Speakers
Segment Title: CURRENT STATE OF ALS TRIALS Presentation Title: ALS gene-targeted therapies and consideration of early diagnosis/treatment.
Speaker: Tim Miller, MD, PhD, ALS Center Co-Director, Miller Lab Director, David Clayson Professor of Neurology at Washington University School of Medicine.
Moderator: Dr. James Berry, MD, MPH
Click HERE to view this video in its entirety.
Symposium Segment: Day 1, 11/16/23: Keynote Speakers
Segment Title: NEXT GENERATION MOLECULAR THERAPIES IN ALS
Presentation 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.
Moderator: Dr. Sabrina Paganoni, MD, PhD.
Click HERE to view this video in its entirety.
Symposium Segment: Day 1, 11/16/23: Keynote Speakers
Segment Title: NEXT GENERATION MOLECULAR THERAPIES IN ALS
Presentation Title: 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.
Moderator: Dr. Sabrina Paganoni, MD, PhD.
Click HERE to view this video in its entirety.
Symposium Segment: Day 1, 11/16/23: Keynote Speakers
Segment Title: NEXT GENERATION MOLECULAR THERAPIES IN ALS
Presentation Title: Gene replacement therapy/viruses.
Speaker: Jerry Mendell, MD, Nationwide Children’s Hospital’s Center for Gene Therapy.
Moderator: Dr. Sabrina Paganoni, MD, PhD.
Click HERE to view this video in its entirety.
Symposium Segment: Day 1, 11/16/23: Keynote Speakers
Segment Title: NEXT GENERATION MOLECULAR THERAPIES IN ALS
Presentation Title: “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.
Moderator: Dr. Sabrina Paganoni, MD, PhD.
Click HERE to view this video in its entirety.
Symposium Segment: Day 1, 11/16/23: Keynote Speakers
Segment Title: Roundtable Discussion.
Topic: Symposium discussion on presented materials and looking ahead at the future of ALS.
Panelists: Blaine Dangel, Merit Cudcowicz, MD, MSc, Suma Babu, MBBS, MPH, Tim Miller, MD,PhD, Jonathan Watts, Ph, Robert Brown, MD, DPhil, Neil Shneider, MD, PhD, Jerry Mendell, MD, Tim Yu, MD, PhD
Moderator: Clotilde Lagier-Tourenne, MD, PhD
Click HERE to view this video in its entirety.
Symposium Segment: Day 1, 11/16/23
Symposium Segment: Drs. Ayeez and Shelena Lalji & Family Student Scholar Award in Repair Mechanisms in ALS.
Presenter: Dr. Shelena Lalji, MD, FACOG, Founder of ALS Heroes and Dr. Shel Wellness & Aesthetic Center.
Award Recipient: Deland Liu, University of Texas at Austin Medical School Moderator: Dr. James Berry, MD, MPH
Click HERE to view this video in its entirety.
DAY 2: November 17, 2023 – Industry Speakers
Presentation Title: A Phase 2 Trial of RIPK1 Inhibitor SAR443820 in Adult Participants with ALS (Himalaya Study): Study Design and Baseline Characteristics.
Speaker: Nazem Atassi, MD, PhD, Head of Early Neuro Development at Sanofi.
Moderator: Dr. Robert H. Brown, MD, DPhil.
Click HERE to view this video in its entirety.
Symposium Segment: Day 2, 11/17/23: Industry Speakers
Presentation 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.
Speaker: Katie Planey, PhD, MBA, CEO of Acelot.
Moderator: Dr. Fernando Vieira, MD
Click HERE to view this video in its entirety.
Symposium Segment: Day 2, 11/17/23: Industry Speakers
Presentation 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.
Speaker: Tamara Seredenina, PhD, Group Leader of TDP-43/ Neuroinflammation at AC IMMUNE SA
Moderator: Dr. Fernando Vieira, MD
Click HERE to view this video in its entirety.
Symposium Segment: Day 2, 11/17/23: Industry Speakers
Presentation Title: Amylyx Update: CENTAUR and US EAP Data Analyses, Planning for PHOENIX, and New Research.
Speaker: Jamie Timmons, MD, Head of Scientific Communications at Amylyx Pharamaceuticals
Moderator: Dr. Fernando Vieira, MD
Click HERE to view this video in its entirety.
Symposium Segment: Day 2, 11/17/23: Industry Speakers
Presentation Title: Biomarker exploration of NurOwn’s multimodal mechanism of action on neuroinflammation, neuroprotection and neurodegeneration.
Speaker: Bob Dagher, MD, President and Chief Development Officer at Brainstorm Cell Therapeutics.
Moderator: Dr. Fernando Vieira, MD
Click HERE to view this video in its entirety.
Symposium Segment: Day 2, 11/17/23: Industry Speakers
Presentation Title: CNM-Au8: Path Forward. Clene Nanomedicine clinical development review, advancing with Phase 3 Outcomes Trial.
Speaker: Rob Etherington, President and CEO at Clene Nanomedicine.
Moderator: Dr. Fernando Vieira, MD
Click HERE to view this video in its entirety.
Symposium Segment: Day 2, 11/17/23: Industry Speakers
Presentation Title: Shifting the Paradigm – A Biomarker Driven Approach for Studying Amyotrophic Lateral Sclerosis (ALS) Therapy Activity.
Speaker: Shiran Zimri, PhD, VP of Research & Development at NeuroSense Therapeutics.
Moderator: Dr. Fernando Vieira, MD
Click HERE to view this video in its entirety.
Symposium Segment: Day 2, 11/17/23: Industry Speakers
Presentation Title: Pridopidine For the Treatment of ALS – Top Line Results From the Phase 2 Healey ALS Platform Trial.
Speaker: Michal Geva, PhD, SVP, Head of Research at Prilenia
Moderator: Dr. Robert H. Brown, MD, DPhil.
Click HERE to view this video in its entirety.
Symposium Segment: Day 2, 11/17/23: Industry Speakers
Presentation 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.
Speaker: Angela Genge, MD, FRCP(C), eMBA, Chief, Head of Medical Affairs at QurAlis.
Moderator: Dr. Robert H. Brown, MD, DPhil.
Click HERE to view this video in its entirety.
Symposium Segment: Day 2, 11/17/23: Industry Speakers
Presentation Title SPG302: a first-in-class synaptic regenerative therapy for ALS.
Speaker: Peter Vanderklish, PhD, Chief Scientific Officer at Spinogenix.
Moderator: Dr. Robert H. Brown, MD, DPhil.
Click HERE to view this video in its entirety.
Symposium Segment: Day 2, 11/17/23: Industry Speakers
Presentation Title: Preclinical proof of concept of SOL-257, a gene therapy targeting misfolded TDP-43 in ALS.
Speaker: Akinori Hishiya, PhD, Chief Scientific Officer at Sola Biosciences.
Moderator: Dr. Robert H. Brown, MD, DPhil.
Click HERE to view this video in its entirety.
Symposium Segment: Day 2, 11/17/23: Industry Speakers
Presentation Title: ILB®: A Novel Therapeutic Agent for ALS Targeting Excitotoxicity, Mitochondrial Dysfunction, and Inflammation.
Speaker: Claus Sundgreen, MD, Chief Medical Officer at Tikomed.
Moderator: Dr. Robert H. Brown, MD, DPhil.
Click HERE to view this video in its entirety.
Symposium Segment: Day 2, 11/17/23: Industry Speaker Roundtable
Industry Roundtable Topic: Symposium discussion on presented Industry presentations.
Panelists: Katie Planey, PhD (Acelot), Tamara Seredenina, PhD (AC Immune), Jamie Timmons, MD, (Amylyx), Bob Dagher, MD (Brainstorm-Cell) Rob Etherington (Clene Nanomedicine), Shiran Zimri, PhD (NeuroSense), Michal Geva, PhD (Prilenia), Angela Genge, MD, FRCP(C) (QurAlis), Nazem Atassi, MD, PhD (Sanofi), Akinori Hishiya, PhD (Sola Biosciences), Peter Vanderklish, PhD (Spinogenix), Claus Sundgreen, MD (Tikomed).
Moderator: Robert H. Brown, MD, DPhil (UMass Medical School).Click HERE to view this video in its entirety.
Click HERE to view this video in its entirety.
DAY 3: November 20, 2023 – Neurotechnology Day
Symposium Segment: Neurotechnology Day Morning Session (9am – 12pm EST).
Segment Topics: Citizen-Driven Research: Unlocking the power of Agility and Timely Outcomes • Future Opportunities of AI for Good in Healthcare: Insights from Global Industry Leaders • Unique Approaches to Navigating Challenges & Opportunities.
Speakers: Indu Navar, MCSC, Blair Casey, Steve Kowalski, Roseann Hickman, Dr Lyle Ostrow, Dr. Elad Yom-Tov, Dr. Bob MacDonald, Garfield Ha, Fleming Shi, Erin Chu, Christian Rubio, Steffi Henze, and Kristin Hatcher.
Co-Hosted & Moderated by: Indu Navar, MSCS, Founder and CEO of Everything ALS and Blair Casey, Executive Director of Team Gleason.
Click HERE to view this video in its entirety.
DAY 3: November 20, 2023 – Neurotechnology Day
Symposium Segment: Neurotechnology Day Afternoon Session (1pm – 5pm EST).
Segment Topic: Implanted Brain Computer Interfaces
Speakers: Shelena Lalji, MD, FACOG, Leigh Hochberg, MD, PhD, Zoe Lalji, Patricia Bennett, Dr. Nathan Crone, Dr. Brian Dekleva, Vikash Gilja, Kurt Haggstrom, Michael Mager, Dr. David Moses, Dr. Daniel Rubin, Dr. Florian Solzbacher, Dr. Mariska Vansteensel, and Dr. Jonas Zimmermann. Co-
Hosted & Moderated by: Shelena Lalji, MD, FACOG, founder of ALS Heroes and the Dr. Shel Wellness and Aesthetic Center; and Leigh Hochberg, MD, PhD, Director, Center for Neurotechnology and Neurorecovery, Neurocritical Care and Stroke Services, Department of Neurology, MGH.
Click HERE to view this video in its entirety.
Our 5th Annual ALS ONE Research Symposium was held on October 6, 7, and 11th, 2022. Hosted along with our research team: Dr’s Merit Cudkowicz, MD, MSc of MGH, Robert Brown, MD, DPhil of UMass Medical School, James Berry, MD, MPH of MGH, and Fernando Vieira, MD of ALS TDI, and held virtually, our Symposium was a tremendous success. The conference had over 630 registered guests, including researchers, scientists, and members of the ALS community from around the world, convening virtually for two days to share outcomes and discoveries of critical research that is underway across the globe to help uncover therapeutic options leading to an ultimate cure for Amyotrophic Lateral Sclerosis (ALS). Please click HERE to view the official digital Symposium Speaker and Sponsor Guide. Recordings were made of the presentations which we were permitted to share and they are listed and linked below.
DAY 1: October 6, 2022 – Keynote Speakers
Symposium Segment: CURRENT STATE OF ALS TRIALS
Presentation Title: General overview of ALS Trials: New, opportunities, and challenges.
Speaker: Merit Cudkowicz, MD, MSc, Chief, Neurology Dept, Director, Sean M. Healey & AMG Center for ALS at MGH & Julianne Dorn Professor of Neurology at Harvard Medical School.
Moderator: Fernando Vieira, MD, CEO & Chief Science Officer at ALS TDI.
Click HERE to view this presentation.
Symposium Segment: CURRENT STATE OF ALS TRIALS
Presentation Title: Functional Outcomes & Biomarkers: Regulatory Considerations.
Speaker: James Berry, MD, MPH, Assoc. Chief of Neuro-therapeutics and Chief of the Division of ALS and Motor Neuron Diseases at MGH and Director of the MGH NCRI.
Moderator: Fernando Vieira, MD, CEO & Chief Science Officer at ALS TDI.
Click HERE to view this presentation.
Symposium Segment: CURRENT STATE OF ALS TRIALS
Presentation Title: Update on EAP & ACT 4 ALS
Speaker: Sabrina Paganoni, MD, PhD, Co-Director of the Neurological Research Institute (NCRI) at the Massachusetts General Hospital, physician scientist at the Healey & AMG Center for ALS, and Associate Professor at Harvard Medical School.
Moderator: James Berry, MD, MPH, Associate Chief of Neuro-therapeutics and Chief of the Division of ALS and Motor Neuron Diseases at Massachusetts General Hospital (MGH) and Director of the MGH Neurological Clinical Research Institute (NCRI).
Click HERE to view this presentation.
Symposium Segment: CURRENT STATE OF ALS TRIALS
Presentation Title: Update on The Healey ALS Platform Trial
Speaker: Sabrina Paganoni, MD, PhD, Co-Director of the Neurological Research Insitute (NCRI) at MGH & physician scientists at the Healey & AMG Center for ALS & Associate Professor at Harvard Medical School.
Moderator: Fernando Vieira, MD, CEO & Chief Science Officer at ALS TDI.
Click HERE to view this presentation.
Symposium Segment: CURRENT STATE OF ALS TRIALS
Presentation Title: Preclinical Modeling of ALS: Relevance and Applications.
Speaker: Fernando Vieira, MD, Chief Executive Officer and Chief Science Officer at ALS Therapy Development Institute (ALS TDI).
Moderator: James Berry, MD, MPH, Associate Chief of Neuro-therapeutics and Chief of the Division of ALS and Motor Neuron Diseases at Massachusetts General Hospital (MGH) and Director of the MGH Neurological Clinical Research Institute (NCRI).
Click HERE to view this presentation.
Symposium Segment: CURRENT STATE OF ALS TRIALS
Presentation Title: Gene therapies: Biogen’s update on Tofersen.
Speaker: Toby Ferguson, MD, PhD, Vice President, Head Neuromuscular Clinical Development at Biogen.
Moderator: James Berry, MD, MPH, Associate Chief of Neuro-therapeutics and Chief of the Division of ALS and Motor Neuron Diseases at Massachusetts General Hospital (MGH) and Director of the MGH Neurological Clinical Research Institute (NCRI).
Click HERE to view the presentation.
Symposium Segment: CURRENT STATE OF ALS TRIALS
Presentation Title: Update on AMX0035 (Relyvrio)
Speaker: Jamie Timmons, MD, Head of Scientific Communications at Amylyx Pharmaceuticals. Additonal comments by: Justin Klee and Josh Cohen, Co-CEO’s and Co-Founders of Amylyx Pharamaceuticals.
Moderator: James Berry, MD, MPH, Associate Chief of Neuro-therapeutics and Chief of the Division of ALS and Motor Neuron Diseases at Massachusetts General Hospital (MGH) and Director of the MGH Neurological Clinical Research Institute (NCRI).
Click HERE to view the presentation.
Symposium Segment: Continuing lessons from TDP43 and related ALS genes
Presentation Title: Stathmin2: Update on biology and approaches to therapy.
Speaker: Kevin Eggan, PhD, Group Vice President, Head of Research and Early Development at BioMarin.
Moderator: Robert H. Brown, Jr., MD, DPhil, Professor and Chair of Neurology at the University of Massachusetts Medical School. Director of the Day Neuromuscular Research Laboratory at the University of Mass. Medical School.
Click HERE to view the presentation.
Symposium Segment: Continuing lessons from TDP43 and related ALS genes
Presentation Title: Mitochondria, Inflammation and ALS.
Speaker: Erika L.F. Holzbaur, Ph.D., William Maul Measey Professor of Physiology at the University of Pennsylvania Perelman School of Medicine.
Moderator: Robert H. Brown, Jr., MD, DPhil, Professor and Chair of Neurology at the University of Massachusetts Medical School. Director of the Day Neuromuscular Research Laboratory at the University of Mass. Medical School.
Click HERE to view the presentation.
Symposium Segment: Continuing lessons from TDP43 and related ALS genes
Presentation Title: New ALS splicing targets.
Speaker: Aaron Gitler, PhD, Stanford Medicine Basic Science Professor in the Department of Genetics at Stanford University.
Moderated by: Dr. Robert H. Brown, Jr., MD, DPhil, Professor and Chair of Neurology at the University of Massachusetts Medical School. Director of the Day Neuromuscular Research Laboratory at the University of Mass. Medical School.
Click HERE to view the presentation.
DAY 2: November 30, 2021 – Industry Speakers
Symposium Segment: Industry Presentations
Presentation Title: The Relationship Between CSFBiomarkers and Efficacy of Treatment with NurOwn (MSC-NTF).
Speaker: Stacy Lindborg, PhD, Executive Vice President and Chief Development Officer at Brainstorm-Cell Therapeutics.
Moderated by: Robert H. Brown, Jr. MD, DPhil, Chief of Neurology at UMass Chan Memorial Medical School.
Click HERE to view the presentation.
Presentation Title: ENGRAILED-1 homeoprotein transcription factor as a novel therapeutic strategy for ALS.
Speaker: Julien Spatazza, PhD., Head of Pharmacology at BrainEver.
Moderator: Robert H. Brown, Jr., MD, DPhil, Professor and Chair of Neurology at the University of Massachusetts Medical School. Director of the Day Neuromuscular Research Laboratory at the University of Mass. Medical School.
Click HERE to view the presentation.
Symposium Segment: Industry Presentations
Presentation Title: Reduction of oxidized phospholipids or misfolded protein aggregates by AAV-VecTabs in an Amyotrophic Lateral Sclerosis pre-clinical models.
Speaker: Pavlina Konstantinova, MSc, PhD, MBA, Co-Founder and Chief Scientific Officer of VectorY.
Moderator: Fernando Vieira, MD, CEO & Chief Science Officer at ALS TDI.
Click HERE to view the presentation.
Symposium Segment: Industry Presentations
Presentation Title: Accelerating discovery and development of ALS therapeutics with an AI-powered All-in-Human Platform.
Speaker: Irene Y. Choi, PhD, Senior Director/ Head of Drug Discovery at Verge Genomics.
Moderator: Fernando Vieira, MD, CEO & Chief Science Officer at ALS TDI.
Click HERE to view the presentation.
Symposium Segment: Industry Presentations
Presentation Title: Preclinical proof of concept of SOL-257, a gene therapy targeting misfolded TDP-43 in ALS.
Speaker: Akinori Hishiy, PhD, Chief Science Officer at SOLA.
Moderator: Fernando Vieira, MD, CEO & Chief Science Officer at ALS TDI.
Click HERE to view the presentation.
Symposium Segment: Industry Presentations
Presentation Title: QRL-201-01 – A multi-center, randomized, double-blind, placebo-controlled multiple‑ascending dose study to evaluate the safety and tolerability of QRL‑201 in amyotrophic lateral sclerosis.
Speaker: Angela Genge, MD, Chief Medical Officer at QurAlis.
Moderator: Fernando Vieira, MD, CEO & Chief Science Officer at ALS TDI.
Click HERE to view the presentation.
Presentation Title: Sodium Phenylbutyrate/Taurursodiol (AMX0035) ALS Updates
Symposium Segment: Industry Presentations
Presentation Title: Shifting the Paradigm: PrimeC as a novel therapeutic strategy for ALS.
Speaker: Ferenc Tracik, MD, Chief Medical Officer at NeuroSense.
Moderator: Fernando Vieira, MD, CEO & Chief Science Officer at ALS TDI.
Click HERE to view the presentation.
Symposium Segment: Industry Presentations
Presentation Title: Ionis’ ALS Genetic Testing Program.
Speaker: Amy Shea, Director, Diagnostic & Strategic Initiatives at Ionis.
Moderator: Fernando Vieira, MD, CEO & Chief Science Officer at ALS TDI.
Click HERE to view the presentation.
Symposium Segment: Industry Presentations
Presentation Title: Tegoprubart (AT-1501) is safe and well tolerated and reduces inflammation in patients with ALS.
Speaker: Jeffrey Bornstein, MD, Chief Medical Officer, Eledon Pharmaceuticals.
Moderator: Robert H. Brown, Jr., MD, DPhil, Professor and Chair of Neurology at the University of Massachusetts Medical School. Director of the Day Neuromuscular Research Laboratory at the University of Mass. Medical School.
Click HERE to view the presentation.
Symposium Segment: Industry Presentations
Presentation Title: A multi-properties peptide (NX210c) for the treatment of ALS
Speaker: Sighild Lemarchant, PhD, Head of Preclinical R&D, Axoltis Pharma.
Moderator: Robert H. Brown, Jr., MD, DPhil, Professor and Chair of Neurology at the University of Massachusetts Medical School. Director of the Day Neuromuscular Research Laboratory at the University of Mass. Medical School.
Click HERE to view the presentation.
Symposium Segment: Industry Presentations
Presentation Title: Rationale and design of a phase 3, randomized, placebo-controlled trial of tofersen initiated in clinically presymptomatic SOD1 mutation carriers: The ATLAS Study.
Speaker: Michael Benatar, MBChB, MS, DPhil, Walter Bradley Chair in ALS Research; Professor of Neurology; Chief, Neuromuscular Division; Executive Director of The ALS Center; Vice Chair, Clinical & Translational Research Department of Neurology, Miller School of Medicine at the University of Miami.
Moderator: Robert H. Brown, Jr., MD, DPhil, Professor and Chair of Neurology at the University of Massachusetts Medical School. Director of the Day Neuromuscular Research Laboratory at the University of Mass. Medical School.
Click HERE to view the presentation.
Symposium Segment: Industry Presentations
Presentation Title: Immunotherapy targeting the C-terminal domain of TDP-43 decreases neuropathology and confers neuroprotection through microglial engagement in mouse models of ALS/FTD.
Speaker: Tariq Afroz, PhD, Team and project leader for TDP-43 therapeutic antibody program at AC Immune.
Moderator: Robert H. Brown, Jr., MD, DPhil, Professor and Chair of Neurology at the University of Massachusetts Medical School. Director of the Day Neuromuscular Research Laboratory at the University of Mass. Medical School.
Click HERE to view the presentation.
Symposium Segment: Neurotechnology Day Preview.
Presentation Title: NEW BIOMARKERS, BCI, NEUROTECH & BEYOND: A prelude to Neurotechnology Day.
Speakers: James Berry, MD, MPH, Associate Chief of Neuro-therapeutics and Chief of the Division of ALS and Motor Neuron Diseases at Massachusetts General Hospital (MGH) and Director of the MGH Neurological Clinical Research Institute (NCRI). Leigh Hochberg, MD, PhD, Director, Center for Neurotechnology and Neurorecovery, Neurocritical Care and Stroke Services, Department of Neurology, Massachusetts General Hospital; Senior Lecturer on Neurology, Harvard Medical School; L. Herbert Ballou University Professor of Engineering and Brain Science, Brown University; Director, VA RRR&D Center for Neurorestoration and Neurotechnology, Providence VA Medical Center. Indu Navar, MSCS, CEO and Founder of EverythingALS. Shelena C. Lalji, MD, F.A.C.O.G., CEO and Founder of ALS Heroes and The Dr. Shel Wellness & Aesthetic Center.
Click HERE to view the presentation.
DAY 2: October 7, 2022 – Industry Roundtable
A review of the exciting new developments reported during the first two days of the Symposium, and promising future directions in ALS investigations and trials.
Speakers: Merit Cudkowicz, MD, MSc, Chief, Neurology Department – Director, Sean M. Healey & AMG Center for ALS Mass General Hospital and Julianne Dorn Professor of Neurology Harvard Medical School. James Berry, MD, MPH, Associate Chief of Neuro-therapeutics and Chief of the Division of ALS and Motor Neuron Diseases at Massachusetts General Hospital (MGH) and Director of the MGH Neurological Clinical Research Institute (NCRI). Angela Genge, MD, Chief Medical Officer at QurAlis. Bruce Rosenblum, person living inspiringly with ALS.
Moderator: Robert H. Brown, Jr., MD, DPhil, Professor and Chair of Neurology at the University of Massachusetts Medical School. Director of the Day Neuromuscular Research Laboratory at the University of Mass. Medical School.
Click HERE to view the presentation.
DAY 2: October 11, 2022 – Neurotechnology Day
We were excited to collaborate with Everything ALS and ALS Heroes for “NEUROTECH NOW”, an entire day dedicated to neurotechnology as it relates to ALS. During Neurotechnology Day, experts from industry and academia provided exciting updates on BCI technologies and clinical trials, particularly toward maintaining communication for people with ALS. Exciting round tables discussed common challenges and solutions to translating these technologies to clinical use.
Moderators: Leigh Hochberg, MD, PhD, Director, Center for Neurotechnology and Neurorecovery, Neurocritical Care and Stroke Services, Department of Neurology, Massachusetts General Hospital; Senior Lecturer on Neurology, Harvard Medical School; L. Herbert Ballou University Professor of Engineering and Brain Science, Brown University; Director, VA RRR&D Center for Neurorestoration and Neurotechnology, Providence VA Medical Center. James Berry, MD, MPH, Associate Chief of Neuro-therapeutics and Chief of the Division of ALS and Motor Neuron Diseases at Massachusetts General Hospital (MGH) and Director of the MGH Neurological Clinical Research Institute (NCRI). Indu Navar, MSCS, CEO and Founder of EverythingALS. Shelena C. Lalji, MD, F.A.C.O.G., Founder of ALS Heroes and The Dr.Shel Wellness & Aesthetic Center.
Special guest speakers from industry and academics included Biogen Digital Health, MT-Pharma, Novartis, NeuroSense, NeuraLight, Modality.AI, Amazon Web Services, MGH, JHU, UPitt, Wyss Ctr, UCSF, UMC Utrecht, Blackrock, Synchron, and Paradromics.
Click HERE to view Industry Day’s presentation.
Our 4th Annual ALS ONE Research Symposium was held on November 29th & 30th 2021, hosted by our research team: Dr’s Merit Cudkowicz, MD, MSc of MGH, Robert Brown, MD, DPhil of UMass Medical School, James Berry, MD, MPH of MGH, and Fernando Vieira, MD of ALS TDI. The conference had over 630 registered guests, including researchers, scientists, and members of the ALS community from around the world, convening virtually for two days to share outcomes and discoveries of critical research that is underway across the globe to help uncover therapeutic options leading to an ultimate cure for Amyotrophic Lateral Sclerosis (ALS). We are extremely grateful to our 2021 Symposium sponsors: at the Emerald Level: Amylyx, Biogen, and Seelos Therapeutics, at the Gold Level: Cytokinetics, and at the Bronze Level: Brainstorm Cell Therapeutics, Clene Nanomedicine, and Prilenia. Please click HERE to view the official digital Symposium Speaker and Sponsor Guide. Recordings were made of the presentations which we were permitted to share and they are listed and linked below. If you do not see the one(s) in which you are interested, it may be released at a future date so do continue to check back here.
DAY 1: November 29, 2021 – Keynote Speakers
Segment Title: ALS: Biomarkers and Genes in ALS: New Frontiers
Presentation Title: New Disease Progression Biomarkers: Promises and Pitfalls
Presented by: Jeremy Shefner, MD, PhD, of Barrow Neurological Institute and James Berry, MD, MPH, of The Healey Center for ALS at MGH, respectively.
Moderated by: Fernando Vieira, MD, of ALS Therapy Development Institute.
Click HERE to view Dr. Shefner and Dr. Berry’s presentations.
Segment Title: Oligogenetics and Epigenetics
Presentation Title: Epigenetics of Neurodegenerative Disease Genetics – what do we know so far?
Presented by: Ammar Al-Chalabi, MB, ChB, PhD, King’s College London.
Moderated by: Robert H. Brown, Jr., D.Phil., M.D. of UMass Medical School
Click HERE to view Dr. Al-Chalabi’s presentation.
Presentation Title: Epigenetics of Neurodegenerative Disease
Presented by: Nancy Bonini, PhD, of The University of Pennsylvania in Philadelphia
Moderated by: Robert H. Brown, Jr., D.Phil., M.D. of UMass Medical School
Click HERE to view Dr. Bonini’s presentation.
Presentation Title: Retrotransposons and endogenous retroviruses as causal factors in ALS and related disorders
Presented by: Josh Dubnau, PhD, of Stony Brook Renaissance School of Medicine
Moderated by: Robert H. Brown, Jr., D.Phil., M.D. of UMass Medical School
Click HERE to view Dr. Dubnau’s presentation.
Presentation Title: Retroviral elements in ALS
Presented by: Avindra Nath, MD, of The National Institutes of Health
Moderated by: Robert H. Brown, Jr., D.Phil., M.D. of UMass Medical School
Click HERE to view Dr. Nath’s presentation.
Segment Title: Current state of ALS trials
Presentation Titles: Innovative ALS Trial Designs Impact Therapy Development & The ALS Trial Pipeline
Presented by: Jeremy Shefner, MD, PhD of Barrow Neurological Inst and Merit Cudkowicz, MD, MSc of The Healey Ctr for ALS at MGH, respectively.
Moderated by: James Berry, MD, MPH of The Healey Ctr for ALS at MGH
Click HERE to view Dr. Shefner and Dr. Cudkowicz’s presentations.
DAY 2: November 30, 2021 – Industry Speakers
Presentation Title: ALS drug development: designing small molecules to correct dysfunction in metabolic regulation
Presented by: John Thomson, PhD, of 1Base Pharma
Moderated by: Fernando Vieira, MD, of ALS Therapy Development Institute
Click HERE to view Dr. Thomson’s presentation.
Presentation Title: Exploring SIGMAR1 Modulators for the Treatment of ALS
Presented by: Nell Rebowe, BS
Moderated by: Fernando Vieira, MD, of ALS Therapy Development Institute
Click HERE to view Nell Rebowe’s presentation.
Presentation Title: Preventing axonal degeneration in ALS by inhibiting SARM1
Presented by: Thomas M. Engber, Ph.D. of Disarm Therapeutics
Moderated by: Fernando Vieira, MD, of ALS Therapy Development Institute
Click HERE to view Dr. Engber’s presentation.
Presentation Title: Combination of ciprofloxacin/celecoxib as a novel therapeutic strategy for ALS.
Presented by: Shiran Zimri, PhD. of NeuroSense Therapeutics
Moderated by: Robert H. Brown, Jr., D.Phil., M.D.
Click HERE to view Dr. Zimri’s presentation.
Presentation Title: Rationale and preliminary data supporting a synaptic regenerative therapy in ALS using a novel spinogenic small molecule
Presented by: Peter Vanderklish PhD, of Spinogenix
Moderated by: Robert H. Brown, Jr., D.Phil., M.D.
Click HERE to view Dr. Vanderklish’s presentation.
Presentation Title: Revolutionizing Drug Development for Neurodegenerative Diseases
Presented by: Robert Glanzman, MD of Clene Nanomedicine
Moderated by: Robert H. Brown, Jr., D.Phil., M.D.
Click HERE to view Dr. Glanzman’s presentation.
Presentation Title: Sodium Phenylbutyrate/Taurursodiol (AMX0035) ALS Updates
Presented by: S. Machelle Manuel, PhD of Amylyx Pharmaceuticals
Moderated by: Robert H. Brown, Jr., D.Phil., M.D.
Click HERE to view Dr. Manuel’s presentation.
Presentation Title: Combination of ciprofloxacin/celecoxib as a novel therapeutic strategy for ALS.
Presented by: Scott Baver of Apellis Pharmaceuticals
Moderated by: Robert H. Brown, Jr., D.Phil., M.D.
Click HERE to view Scott Baver’s presentation.
Presentation Title: The Journey From FORTITUDE-ALS to COURAGE-ALS
Presented by: Stacy Rudnicki, MD of Cytokinetics
Moderated by: Robert H. Brown, Jr., D.Phil., M.D.
Click HERE to view Dr. Rudnicki’s presentation.
Presentation Title: Pridopidine for the treatment of ALS.
Presented by: Michal Geva, PhD of Prilenia
Moderated by: Robert H. Brown, Jr., D.Phil., M.D.
Click HERE to view Dr. Geva’s presentation.
Presentation Title: Autophagy as a Common Pathway in ALS: SLS-005 (trehalose intravenous infusion) for the Treatment of Familial and Sporadic ALS in the Prominent Mutation Types.
Presented by: Raj Mehra, PhD, of Seelos Therapeutics.
Moderated by: Robert H. Brown, Jr., D.Phil., M.D.
Click HERE to view Dr. Mehra’s presentation.
Presentation Title: Treating TDP-43 proteinopathy by RACK1 Knockdown.
Presented by: Neil Cashman, MD of ProMis Neurosciences.
Moderated by: Robert H. Brown, Jr., D.Phil., M.D.
Click HERE to view Dr. Cashman’s presentation.
DAY 2: November 30, 2021 – ALS Community Updates
Presentation Title: ALS Himalaya Trial- A Phase 2, Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of SAR443820 in Adults with ALS
Presented by: Nazem Atassi, MD at Sanofi
Moderated by: Robert H. Brown, Jr., D.Phil., M.D.
Click HERE to view Dr. Atassi’s presentation.
Presentation Title: Frontiers in ALS Therapy Development
Presented by: Fernando Vieira, MD, CEO & CSO at ALS TDI
Moderated by: Robert H. Brown, Jr., D.Phil., M.D.
Click HERE to view Dr. Vieira’s presentation.
Presentation Title: The HEALEY ALS Platform Trial
Presented by: Sabrina Paganoni, MD, PhD, of The Healey Center for ALS at MGH.
Moderated by: Robert H. Brown, Jr., D.Phil., M.D.
Click HERE to view Dr. Paganoni’s presentation.
Platform Trial Updates & Webinars
We are proud to have been and still be a founding and ongoing benefactor of the Healey and AMG Platform Trial at MGH. This trial, the world’s first platform trial in the field of ALS, accelerates the path to new ALS therapies by testing multiple treatments at once, reducing the cost of research by 30%, decreasing the trial time by 50%, and increasing patient participation by 67%. Funding by ALS ONE has aided significantly in the launch and advancement of this unprecedented trial which began enrolling patients in 2020 and has now tested 7 drugs – a monumental accomplishment.
Weekly Webinars! Join our partners at The Healey Center for ALS at MGH weekly to continue the great communication between the patient and science community for this groundbreaking trial.
Click HERE to register for upcoming weekly webinars and HERE to watch past updates.
To read more about the start of this highly anticipated trial, please click HERE.
To watch the launch video, please click HERE.
Individuals living with ALS who are interested in participating in this trial are encouraged to complete the Patient Interest Form, which can be downloaded HERE.
Since discovering the first gene that causes ALS over 20 years ago, Dr. Robert Brown of the University of Massachusetts Memorial Medical School, has worked to develop gene therapies for people with ALS. Through an ALS ONE partnership with MGH, the team is now altering genes in patients using a harmless virus delivered in a single dose, potentially revolutionizing care and treatment for people with genetic forms of ALS.
Researchers at ALS TDI are developing a drug that restores immune system balance and reduces inflammation. Meanwhile, a team at MGH has developed new imaging tools that measure inflammation in patients with ALS. The ALS ONE partnership has hastened the development of this promising anti-inflammatory therapy to bring it from the lab to patients—and preclinical work is under way.
PMEASURING FATIGUE IN ALS: A study by lead investigator, James Berry, MD, at the Sean M. Healey and AMG Center for ALS at MGH. OVERVIEW: Looking for healthy volunteers and individuals with motor neuron disease (ALS, PLS, HSP, SMA) to complete a survey study to help us learn more about fatigue in individuals with motor neuron disease.
ELIGIBILITY CRITERIA: People with MND and Healthy Participants who are at least 18 years of age and able to complete a series of questionnaires online.
WHAT HAPPENS IF I SIGN UP?
- This is a questionnaire only study.
- All visits will be conducted remotely and online (There will not be any in-person or telephone visits).
- There will be 5 visits over 12 months (baseline, at 4 weeks, 3 months, 6 months and 12 months).
- For each visit, you will be provided with a link to access a series of questionnaires aimed at helping us better understand fatigue.
- It will take you approximately 30-45minutes to complete all questionnaires.
COMPENSATION: There will be no compensation for this study. However, your participation will help develop and implement future studies to quantify the measurement of fatigue, and develop treatments for fatigue in people with motor neuron disease.
IF YOU ARE INTERESTED in participating or learning more, please click this link: https://redcap.partners.org/redcap/surveys/?s=94JPCNPJJF
Principal Investigator: James Berry, MD.
Please contact Zoe Scheier at 617-724-4663 or zscheier@mgh.harvard.edu if you are interested in hearing more details and/or if you would like to know if you are eligible to participate in this study.
The launch of ALS ONE created a unique pathway to unite clinicians with care teams to help individuals living with ALS have access much quicker to resources and equipment needed. Our partners at CCALS and the ALS clinics at MGH and UMass Memorial Medical Center, with our union, have enhanced communication between between care teams in the clinics, at CCALS, and in the homes. As such, increased referrals result in higher-quality care support for those living with ALS and their families.
ALS ONE’s dedicated ALS Research Access Nurse, Judith Carey, R.N., located at the MGH Healey Center for ALS, fields over 700 calls annually from patients worldwide to assist them in navigating clinical trials for which they may be eligible. Judy is a tremendously compassionate and knowledgeable resource who connects patients with clinical trial opportunities, either at MGH or elsewhere, which helps the patients, and helps the trials run faster. Patients and families can contact Judy to learn more about clinical trial opportunities by emailing her at: jcarey8@partners.org.
Patient-Focused Solutions
Patients with ALS can’t afford to wait—the typical life expectancy upon diagnosis is 2-5 years
The partnerships forged by ALS ONE bring efficiency and improved coordination to the fields of ALS research and care. The ALS ONE patient network creates a direct pipeline from therapy discovery, to immediate implementation, and to patient services.
Supporting Clinical Trials
Researchers are faced with myriad challenges that make it difficult to secure necessary data and benchmarks while they work to move novel therapies forward
ALS ONE is investing in technologies that help ease the research process and better enable patients to meet with clinicians and attend clinical trials in a safe environment.