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Sean M. Healey & AMG Center for ALS
Summer 2026 newsletter
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Thanks to your participation and support, this has been an exciting summer at the Sean M. Healey & AMG Center for ALS.
- In the lab, our team identified a shared driver of neuron damage in ALS, frontotemporal dementia, and Alzheimer’s disease — a discovery that could lead to new therapies for multiple neurodegenerative diseases.
- We completed a record enrollment of 250 participants in less than five months for Regimen I (NUZ-001) of the HEALEY ALS Platform Trial — our fastest enrollment period ever, which will help us get trial results more quickly.
- Through a wide range of events, our dedicated ALS community continues to fuel research in the Healey & AMG Center. This month, we spotlight the return of the Ice Bucket Challenge and the Falmouth Road Race.
We could not have come this far without your partnership and support. Thank you for inspiring our efforts as we work together to find a cure.
Warmly,
Merit Cudkowicz, MD, MSc
| | A common underlying cause of neuron damage links three neurodegenerative diseases | | |
Although amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD) and Alzheimer’s disease (AD) all have distinct causes and clinical features, researchers have found that neurons in all three conditions accumulate a distinctive pattern of DNA damage—suggesting they may share a common mechanism of neurodegeneration.
In a recent study led by a team of researchers at Mass General Brigham, Boston Children’s Hospital, Harvard Medical School and other collaborating institutions, scientists compared somatic (non-inherited) mutations in individual neurons in brains affected by ALS, FTD and AD.
| | Strategic research initiatives | | HEALEY ALS Platform Trial | |
Enrollment complete in Regimen I
This was the fastest enrollment period ever for any trial in ALS, and it is because of the HEALEY ALS Platform Trial operational efficiencies and the dedication of the ALS community, which led to shorter startup timelines and fast site activation. Faster enrollment allows us to get answers from the trial faster and speeds up the research process.
This is also the first regimen to enroll under the Next Generation design, a revision of the original Master Protocol that leverages lessons learned from the first seven regimens and the field. Key enhancements include enrollment of participants at earlier stages of the disease (within 24 months since onset), a longer randomized controlled trial (RCT) period (36 weeks), and integration of new informative biomarkers. The revised protocol increases the statistical power of the trial, streamlines operations, and adds more patient-centered features, such as increased opportunities for remote visits in the active treatment extension (ATE).
Thank you to the participants and their families, sites, industry partners, and collaborators on this regimen. This progress would not be possible without your dedication and hard work.
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ANA spotlights ALS research and Healey ALS MyMatch
Dr. Suma Babu recently joined Dr. Ryan Jacobson of Rush University on an ANA Investigates podcast.
They discussed Healey ALS MyMatch, the hope it brings to ALS drug development, and latest updates.
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We’re hosting a webinar in collaboration with NEALS on September 17th at 5:00 pm ET.
Accelerating ALS Drug Development Through Academia–Industry Collaboration: The Next Generation of the HEALEY ALS Platform Trial and Healey ALS MyMatch
Developing effective therapies for ALS requires innovative approaches that connect patients, researchers, clinicians, and industry partners more efficiently than ever before. This webinar will highlight how the Healey ALS MyMatch Program and the HEALEY ALS Platform Trial are working together to create a seamless pathway for patient engagement, trial access, and therapeutic development. Speakers will share insights from these pioneering initiatives, discuss the value of academia–industry collaboration, and explore how integrated research models can accelerate treatment development for ALS while improving the clinical trial experience for people living with ALS.
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Insights from trials and therapy development
Read about how at-home spirometry compares to in-clinic spirometry in the HEALEY ALS Platform Trial, how an innovative funding model could transform ALS therapy development, and how master protocols may decrease cost and improve efficiency in testing potential therapeutics in rare disease.
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Building infrastructure and research tools
Learn about the need for clinical care for people living at genetic risk for ALS and how AI can help identify people living with ALS who might be eligible to participate in clinical trials.
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Researching tofersen
Find updates on the tofersen EAP and the biodistribution of tofersen and its impact on SOD1 levels in human central nervous system tissues.
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Advances in Spinal Muscular Atrophy (SMA)
Learn about Tau as a biomarker in SMA and nusinersen management during pregnancy.
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Research access
Our research programs are dedicated to advancing ALS science, uncovering its underlying causes, and ultimately discovering effective treatments for this disease. If you would like to learn more about how to stay connected to ALS research, please reach out to our research access nurses, Kiersten Pease or Judi Carey at mghalsresearch@mgb.org. You can also visit our clinical trials page here. We are deeply grateful to all past, current, and future participants who are helping to accelerate research and drive the search for life-saving therapies.
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Study Spotlights
Trial of Pridopidine Phase 3
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Purpose of Trial: To evaluate if the investigational study drug (pridopidine) may slow disease progression and improve speech, breathing, and survival.
Trial of AMX0114 Phase 1
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Purpose: To evaluate the safety and tolerability of the study drug in ALS patients.
Clinical research webpage
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Upcoming Healey & AMG Center webinars
Thursday, September 24, 5:00 pm ET: HEALEY ALS Platform Trial Update
| | The Ice Bucket Challenge is back! | | |
We did the Ice Bucket Challenge to advance ALS research! Former NFL player Chris Johnson restarted the challenge to support critical research at the Healey & AMG Center after sharing his ALS diagnosis on Good Morning America.
The NFL commissioner Roger Goodell recently completed the ALS Ice Bucket Challenge in support of Tim Green, Chris Johnson, and people living with ALS. The NFL Foundation has generously pledged $500,000 to the Healey & AMG Center. The funds will support Healey ALS MyMatch, the Center's early phase clinical trial program.
From September 25-26, Olympia High School in Orlando, Florida will honor alumnus Chris Johnson and raise awareness and support for families affected by ALS with CJ STRONG Weekend. The weekend will kick off with an Olympia High School football game on Friday, followed by a community event on Saturday that will include family-friendly activities, alumni participation, food vendors, community organizations, educational opportunities about ALS, fundraising efforts benefiting the Chris Johnson ALS Fund at the Sean M. Healey & AMG Center for ALS, and an alumni/community Ice Bucket Challenge.
| | The Falmouth Road Race - race weekend is upon us! | |
Sunday, August 16th, is the Falmouth Road Race! Our inaugural Healey & AMG Center team will run 7 miles after several months of fundraising for ALS research. The team brings together 9 members of the Healey & AMG Center staff and a remarkable group of community members who have all been touched by the devastation of ALS.
Our 40 team members have now collectively surpassed $110,000 in their fundraising - and donations are still coming in! We hope you'll consider supporting our team with a donation by clicking below. Every contribution helps advance critical ALS research and brings us one step closer to a future without ALS. Thank you for helping us turn every mile on the Falmouth course into hope for people living with ALS and families affected by the devastation of the disease.
Once on the team page, you can support the team as a whole or, if you would like to support a specific runner, simply scroll down to view each of our runners’ individual fundraising pages. We hope you'll consider coming down to Falmouth, MA on Sunday morning to cheer on our team - race start time is 8am and it is one incredible race to run - and watch!
Support the team
| | The Angel Fund for ALS Research | |
Pictured in the photo (from left to right): From The Angel Fund: Ann Hadley, Eugene Nigro, Richard Kennedy; from the Healey & AMG Center: Jen DiMartino, Dr. Jenn Morganroth, Dr. James Berry; and from The Angel Fund: Dr. Neil Gleason
The Angel Fund for ALS Research recently presented a gift to advance the research of Dr. James Berry, Chief of the Division of ALS and Motor Neuron Diseases at Massachusetts General Hospital and Dr. Jenn Morganroth, a principal investigator at the Sean M. Healey & AMG Center for ALS. In-line with their longstanding commitment to accelerating progress in ALS research, The Angel Fund's generosity will enable Drs. Berry and Morganroth and their team to utilize the new Nucleic acid-Linked Immuno-Sandwich Assay (NULISA) platform to analyze over 1,000 existing blood and cerebrospinal fluid (CSF) samples which were collected over the last 8 years in the Dominant Inherited ALS (DIALS) study from people at genetic risk of developing ALS. We are deeply grateful for The Angel Fund's partnership and shared dedication to advancing effective treatments and ultimately a cure for ALS.
| | Richard J. Wolnik Golf Tournament | | The Richard J. Wolnik Memorial Golf Tournament was held in June in memory of Dick Wolnik. Dick was an ALS patient at MGH/Healey & AMG Center and an active participant in their experimental drug trials. Dick was unwavering in his commitment to advocacy—raising awareness and funds to fuel ALS research. | In recognition of the extraordinary work of Dr. Cudkowicz and her team, Dick established The Richard J. Wolnik Foundation for ALS Research, a 501(c)(3) charity, before he passed. His vision was clear: to support the researchers who are driving progress toward a cure. Today, his mission lives on through the foundation, ensuring that funds go directly to MGH and the Healey & AMG Center to advance the most promising clinical drug research. The RJW Memorial Golf Tournament was started in 2025 to honor Dick’s life and commitment to fueling ALS research for others. Over the past two years, the tournament has raised over $50,000 for the Healey & AMG Center. | | Winchester High School & Community Fundraisers | | An enduring legacy! The coaches, staff, and community of Winchester High School continue to make a big impact in the fight against ALS in loving memory of Rick Marks and Billy Fleming, and in honor of so many others who have been or are impacted in their community by ALS. WHS Boys Volleyball Coach John Fleming and his team held their annual Spike Out ALS fundraiser match, 8 members of the WHS coaching staff surpassed their annual 70,000 Step Fundraising Challenge, and a former WHS Volleyball Team player's dad completed his 5-year quest to hike the 48 4000+ foot mountains in New Hampshire - all of these fundraising events were held to support ALS research at the Healey & AMG Center in honor of so many incredible people who meant so much to them and us. | | Joseph Kiley Foundation Walk | The Joseph Kiley Foundation was created to honor the life and legacy of Joe Kiley, who courageously battled Bulbar ALS from 2018 until his passing in 2020. In his memory, we are committed to raising awareness, funding cutting-edge research, and supporting families who face the devastating reality of ALS. What began as a simple community walk has grown into a powerful movement. Each year, hundreds gather in Lincoln, RI to walk side by side, united in the fight against ALS. This year's walk, being held on September 27, marks our 6th annual event, and we’re humbled to have officially raised more than $100,000 for the Healey & AMG Center over the last five years in pursuit of a cure. We hope you'll join us for a morning full of fun, remembrance, and hope in support of those living with ALS. To learn more, please visit our event site. | | Carol and James Herscot Endowed Neuroscience Institute Chair - Executive Director | |
On June 25th, we celebrated Merit Cudkowicz, MD, MSc as the inaugural incumbent of the Carol and James Herscot Endowed Neuroscience Institute Chair - Executive Director.
Dr. Cudkowicz shared, "My hope is that the Neuroscience Institute will always stand for the values that brought us here together, and this chair allows that to continue indefinitely until we have really found those treatments for our patients. Curiosity, urgency, and collaboration - That's what's really important. That's what the Neuroscience Institute is about. And the unwavering commitment to our patients and our families and to each other. I really believe that when we bring people together with a shared purpose, anything is possible. Extraordinary things can be possible."
Congratulations to Dr. Cudkowicz on this well-deserved honor!
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Longitude Prize on ALS Team
In partnership with the Eric and Wendy Schmidt Center at the Broad Institute of MIT and Harvard and colleagues across the Broad Institute, MIT, Harvard, we’re proud to be a part of a team that received the Stage 1 Longitude Prize on ALS! This prize will enable us to use cutting-edge AI to unlock the complexity of ALS. Learn more.
| | Congratulations to Dr. James Berry on his new role as the Director of the Mass General Brigham Neuroscience Institute Clinical Trials Center for Neurotherapeutics! | |  | Congratulations to Dr. Jenn Morganroth for receiving the Golseth Young Investigator Award from the American Association of Neuromuscular & Eletrodiagnostic Medicine! | | | |
Our work at the Sean M. Healey & AMG Center for ALS is accelerating therapeutic breakthroughs and improving the holistic care we deliver to our patients and their families. Your philanthropic support allows our clinicians and researchers to be nimble, pursuing the most promising research and care pathways that make a meaningful difference to individuals with ALS. Together, we will find the cures. Thank you for your consideration.
If you have questions or would like to learn more about how you can support the Sean M. Healey & AMG Center for ALS, please contact Emily Monteiro at emonteiro7@mgh.harvard.edu or visit https://www.massgeneral.org/neurology/als/support/.
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Sean M. Healey & AMG Center for ALS.
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Massachusetts General Hospital
55 Fruit Street
Boston, MA 02114
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