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

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
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.
AUGUST 3, 2020 – THE LAUNCH! We are excited to announce that the first ever ALS Platform Trial has officially begun! Five centers are now open for enrollment in The Healey Center for ALS at MGH’s groundbreaking platform trial, led by Dr. Merit Cudkowicz and team, and it is anticipated that additional sites will be opening over the next days and weeks. As the first ever platform trial for ALS, the HEALEY ALS Platform Trial is testing three proposed drug regimens and will add two more over the next several months. The three experimental treatments include, (in no particular order): Zilucoplan, a small macrocyclic peptide inhibitor of complement component 5 [C5], developed by UCB Ra Pharmaceuticals, Inc.; Verdiperstat, an oral myeloperoxidase inhibitor, developed by Biohaven Pharmaceutical Holding Company Ltd., and CNM-Au8 nanocrystalline gold, an intracellular nanocatalyst to support cellular bioenergetics developed by Clene Nanomedicine, Inc.
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.
JULY 8, 2020: The latest updates on The Healey ALS Platform Trial were provided via zoom webinar on July 7, 2020. This hour-long informative session by Dr. Merit Cudkowicz and team includes many answers to frequently asked questions by pALS and families. The webinar, in it’s entirety, may be viewed by clicking 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.
Posted Date: October 23, 2020
MEASURING 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.
Our partners at CCALS and the ALS clinics at MGH and UMass Memorial Medical Center have streamlined a common release form, allowing enhanced communication between between care teams and treatment teams, and increased referrals for high-quality care.
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.
ALS ONE funds a team at MGH that is solely focused on designing new clinical trials to maximize efficiency. Through this dedicated team, ALS ONE partners are able to be proactive in designing effective trials and models that lead to greater success when applying for grants and greater efficiency in moving from concept to trial.
By centralizing the Institutional Review Board (an entity that protects the rights of people in clinical trials), ALS ONE has accelerated the clinical trial process by an average of 7-12 months.
The MGH ALS imaging group is developing novel tools to measure the biological effect of experimental treatments in a way that can reduce the trial population from 400 to 30 patients, and the trial duration from 12 to 3 months. This added efficiency allows ALS ONE to test many more therapies quickly.
pALS and Families: Have you always wanted to learn more about ALS research and influence others to help improve the research process? If so, we encourage you to nominate yourself or a family member to participate in this year’s Virtual ALS Clinical Research Learning Institute (CRLI), which is being held October 2nd & 3rd. The deadline to apply is September 1st so don’t delay. The ALS CRLI is an annual two-day program dedicated to educating attendees on clinical research and therapy development and empowering this group to be advocates for ALS clinical research. To learn more about the ALS Clinical Research Learning Institute and what it means to become an ALS Research Ambassador, please view this WEBINAR and, if interested, download the application HERE.
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.
The NCRI at MGH serves as the coordination center for ALS ONE clinical trials, as well as the coordination and data management center for the Northeast ALS Consortium, a group of more than 100 academic centers worldwide dedicated to finding new ALS therapies.
The NCRI at MGH serves as the coordination center for ALS ONE clinical trials, as well as the coordination and data management center for the Northeast ALS Consortium, a group of more than 100 academic centers worldwide dedicated to finding new ALS therapies.
ALS ONE funds a transportation program through CCALS to provide rides for patients so they can attend clinical appointments and trials at affiliated institutions.