Sean M. Healey & AMG Center for ALS

September 2026 newsletter

Dear friends,

At the Sean M. Healey & AMG Center for ALS, we are proud to provide research-informed care for people living with ALS and their families. Thank you for your partnership as we accelerate progress. We are also committed to keeping the community informed about programs and research at the Center, and we are moving to a monthly newsletter to provide more regular updates.

This month, we highlight several research programs ongoing at the Healey & AMG Center. As new investigational products are brought into the pipeline, our goal is to find answers as fast as possible and expand access to research for people living with ALS.   

Updates:

• The National Institutes of Health (NIH) awarded the Healey & AMG Center an ACT for ALS grant to fund a new Expanded Access Protocol (EAP) with NUZ-001.
• The first Healey ALS MyMatch trial, the ACACIA Trial, announced top line results. Watch the webinar.
• The second Healey ALS MyMatch trial, the BANYAN trial, enrolled the first participant in the trial. This trial evaluates foralumab, a fully human anti-CD3 monoclonal antibody that has been shown to stimulate T regulatory cells (Tregs) when administered by a nasal spray.
• On September 17, we hosted a NEALS Community Education Webinar​ with the latest about the HEALEY ALS Platform Trial and Healey ALS My Match program.
• Recent community events brought together people whose lives have been touched by ALS - creating connection, honoring loved ones, and building hope – while providing critical support for the research and multidisciplinary care that are at the heart of what we do.
• We are excited to announce the 2026 Young Investigator Awards. Selected early-career researchers receive funding to advance their important work and mentorship to develop into the next leaders of ALS research and care.

Thank you for your continued support of the Healey & AMG Center. This work could not be done without you.  

Warmly,

Merit Cudkowicz, MD, MSc

Expanded Access Spotlight

A team of four Principal Investigators from the Healey & AMG Center (Drs. Sabrina Paganoni, Lori Chibnik, Doreen Ho, and Elisa Giacomelli) recently received an NIH grant to conduct an Expanded Access Program (EAP). The grant program was established under the ACT for ALS and will provide up to 200 people living with ALS with expanded access to NUZ-001. NUZ-001 is an investigational product that is currently being tested as Regimen I of the HEALEY ALS Platform Trial. We look forward to keeping the community updated as the program progresses.

To learn more about this EAP, please join the webinar on October 15^th at 5:00pm ET. Register here.

Strategic research initiatives

HEALEY ALS Platform Trial & Healey ALS MyMatch

HEALEY ALS Platform Trial Regimen I

In July, we completed enrollment in Regimen I. This regimen evaluates the investigational product NUZ-001, an oral drug that targets key pathological mechanisms in ALS, including TDP-43 protein aggregation and impaired autophagy.

Regimen I enrolled 250 participants across 78 participating sites of the NEALS network in less than 5 months. This represents a 50% decrease in enrollment time since the start of the Platform Trial and is faster than any stand alone trial, confirming the time savings and efficiency of bringing a platform trial to ALS. This shorter timeline will provide results and answers on a much faster timeline. Thank you so much to the participants and their families for joining the trial and your dedication to research. 

ACACIA Trial Update

On September 10^th, Suma Babu, MBBS, MPH, Brian Wainger, MD, PhD, and Lori Chibnik, PhD, MPH, along with Eric Foster, PhD of 2b Analytics, presented topline results of the ACACIA Trial.

For more information on the ACACIA results, watch the webinar.

Patient information

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.

Study spotlights

Trial of Pridopidine Phase 3

• Purpose of Trial: To evaluate if the investigational study drug (pridopidine) may slow disease progression and improve speech, breathing, and survival.



Study of Speech, Social Connectedness, and Well-Being Outcomes in ALS

• Purpose of Study: To understand the impact of ALS and associated speech impairments on social connectedness and quality of life.

Clinical trials and studies webpage

Upcoming Healey & AMG Center webinars

Thursday, October 1, 5:00 pm ET: HEALEY ALS MyMatch BANYAN Trial

Thursday, October 15, 5:00 pm ET: EAP Update

Thursday, October 22, 5:00 pm ET: HEALEY ALS Platform Trial Update

In the community

Thank you all for participating in these events, which help raise awareness of ALS, as well as vital funds for research. They are also an invaluable way to bring our community together around a shared purpose. 

Never Surrender's Tomassoni Tour

Never Surrender’s Tomassoni Tour brought together an incredible community for a three-day cycling event spanning 28 communities and more than 165 scenic miles along Minnesota’s Mesabi Trail, September 17–19 in Biwabik, MN. The ride culminated in a special dinner, awards ceremony, and fundraising reception, where Darlene Sawicki, NP, and Jennifer DiMartino of the Healey & AMG Center, spoke about the impact of this extraordinary event - advancing ALS research and care while creating a community where those impacted by ALS can gather, connect, and find purpose.

Never Surrender ALS, the nonprofit behind the Tomassoni Tour, was founded in 1995 by the family and friends of Kevin Kolquist, who was lost to ALS, and has since welcomed multitudes of other families who have experienced the same devastating loss. The Tomassoni Tour was launched in 2023 in memory of Minnesota State Senator David Tomassoni, who was devastatingly lost to ALS in 2022. What began decades ago as a family and friends’ commitment to honoring Kevin has grown into a powerful community dedicated to making a difference for others impacted by ALS.

Never Surrender has supported several initiatives at the Healey & AMG Center, including grants for the HEALEY ALS Platform Trial and the Acceleration Center of Excellence (ACE) site at the University of Minnesota. 

We are truly grateful for the support of this inspiring group of people, whose commitment to the ALS community, to research, and to one another makes such a tremendous difference. It was a room filled with incredibly inspiring people - those who continue to make an impact here on earth, and those whose lives and legacies continue to inspire us from above. Pictured are Darlene and Jen with Terry and Mary Steinbach and David Kolquist of Never Surrender at the event in Biwabik, MN.

Rick Baran's Ice Bucket Challenge

Rick Baran's Ice Bucket Challenge to Help Find a Cure for ALS is bringing family, friends, and supporters together to raise funds for the Sean M. Healey & AMG Center for ALS. Inspired by Rick's determination following his ALS diagnosis, participants are taking on the Ice Bucket Challenge, sharing videos in their favorite team gear, and helping advance innovative ALS research, clinical trials, and hope for families affected by the disease. To join their efforts, visit: Team Rick Baran's fundraising page for Mass General Brigham

Angel Fund Walk for Research

Members of the Healey & AMG Center were proud to participate in The Angel Fund’s 25th Walk for ALS Research on September 12 in Wakefield, MA. This special day began with updates on ALS research from Dr. James Berry, highlighting work underway at the Healey & AMG Center, and Dr. Robert Brown, sharing updates from UMass Chan Medical School, along with inspiring remarks from The Angel Fund’s Chairman of the Board, Gene Nigro, and President, Rich Kennedy. Following the release of doves in honor and remembrance of all those impacted by ALS, we joined more than 500 attendees for an inspiring 3.5-mile walk around Lake Quannapowitt. Pictured L-R are: Dr. James Berry, Jen DiMartino, Rich Kennedy, Dr. Robert Brown, and Judi Carey, RN.

Integrated Pension's 4th Annual ALS Golf Tournament

It couldn’t have been a more beautiful day on the links at Northern Spy Golf Club on September 21st for Integrated Pension Services’ 4th Annual ALS Golf Tournament. IPS’ Will Hackler, along with his family, dear friends, and many colleagues, created a truly meaningful day - from the first practice putt through the final moments of the post-golf reception - all in support of advancing ALS research and care at the Healey & AMG Center and Compassionate Care ALS (CCALS).

This special annual tournament is lovingly held in memory of Robert Hastings, Suzanne Horne, and Stephanie Gonda, three inspiring people who lost their lives to ALS, yet whose lives and legacies continue to help light the path toward a cure. The event brought together an incredible community to honor and remember Rob, Suzanne, and Stephanie, as well as all those lost to ALS and everyone currently living with the disease – including loved ones of many in attendance.

It is through selfless events like this - where people come together in memory, in support, and with a shared commitment to making a difference - that our ALS community continues to move forward. We are deeply grateful to Will, his family, and everyone at IPS for their generosity, compassion, and dedication to helping advance research and care while bringing an incredible community together.

Pictured (L–R): Jen DiMartino, who spoke on behalf of the Healey & AMG Center; Will Hackler, Martha Payne, and Madelyn Hackler of IPS; Mellisa Hastings; and Erin Lajeunesse of CCALS.

Ray Bourque Captain's Ball

Ray Bourque’s annual Captain’s Ball, held in memory of Pete Frates, takes place this Thursday evening at Agganis Arena in Boston, and limited tickets are still available.

We hope you’ll join us for this very special evening - always an incredibly inspiring highlight that kicks off the fall season while helping support ALS research at the Healey & AMG Center. The evening will feature a powerful speaking program, dinner, drinks, dancing, live music, auctions, and more!

It’s sure to be a night to remember, filled with inspiration, celebration, and an incredible community coming together in honor of Pete and all those impacted by ALS. We couldn’t be more grateful for the support of Ray, his family, and The Peter Frates Family Foundation – two incredible organizations that do so much for others and for the ALS community. We are humbled and grateful to stand alongside them on this special night, and every day, as we work together toward the day when ALS is no more. Limited tickets are still available and can be purchased at: 2026- The Captain's Ball in honor of Pete Frates - Campaign.

The Healey & AMG Falmouth Road Race Team

The end of September marks the close of fundraising for our inaugural Falmouth Road Race team, and we couldn’t be more proud or more grateful for the phenomenal $141,000+ raised and the heartwarming experience that brought so many people together around a shared purpose. 

We were incredibly honored to be welcomed as an official charity team for this iconic and very special 7-mile race, fielding a team of 45 passionate and determined individuals—including nine members of our Healey & AMG Center team, led by Dr. Merit Cudkowicz herself, and 36 members of our broader community whose lives have been impacted by ALS. On August 16th, 35 teammates took to the course together in Falmouth, while 10 completed their seven miles from different parts of the country. Though separated by miles, all 45 carried the same purpose in their hearts: to honor and remember those we have lost to ALS; to support those courageously living with the disease today and the families walking alongside them; and to raise critical funds to advance groundbreaking ALS research while helping provide the multidisciplinary care our patients and families rely upon.

We are tremendously grateful to the Falmouth Road Race for giving us this incredible opportunity, to each of our 45 teammates for taking on both the physical and fundraising challenge, and to every person who donated, supported, and cheered us on. We hope you’ll watch our team video to see this remarkable community in action - and the very personal “whys” carried on the backs of everyone throughout those seven miles.

Start your own challenge!

Just like teams drench their winning coaches to celebrate a win, you can host an Ice Bucket Challenge campaign or event all throughout the NFL Season to help tackle ALS!

Start your own campaign here.

Young Investigator Awards

We are thrilled to announce the 2026 Mass General Brigham Healey & AMG Center Young Investigator Awards. Thank you so much to the supporters of these awards. Your generosity helps us to attract and retain the brightest young researchers and innovators in the field of ALS.

Byrne Family and Judith & Pape Adams Fellowship

Paolo Pigini, PhD

Project: Targeting Splicing Dysregulation in ALS Using a Prime-Editing Single-Cell Screening Platform. 

Dr. Pigini’s research focuses on the functional interpretation of ALS-associated genetic variants that are predicted to disrupt RNA splicing, a major but still poorly understood disease mechanism in amyotrophic lateral sclerosis. To address this challenge, he will develop a scalable single-cell platform that combines prime editing, iPSC-derived neuronal models, and multimodal single-cell transcriptomics to directly measure the impact of individual variants in a human neuronal context. This approach will enable systematic mapping of how splicing-altering variants affect RNA processing, gene expression, and neuronal viability at single-cell resolution. In addition, the platform will be used to evaluate brain-penetrant small-molecule splicing modulators for their ability to rescue disease-relevant splicing defects. Together, this work hopes to establish a generalizable framework for linking genetic variation to functional outcome and therapeutic response in ALS.

Corcoran Family Award

Kellen Quigg, MD, MSc

Project: Navigating Uncertainty and Risk in Early Phase ALS Clinical Trials: Development of Community-Driven Consensus Recommendations.

Drug development in ALS is advancing rapidly, with novel approaches to treatment quickly moving into human testing. These advances bring great hope, but may also carry new risks to trial participants. Deciding how to balance the tolerance of risks with the opportunity for benefit is challenging, but important to understand in order to accelerate the advancement of new therapies. This project aims to bring together researchers and patients living with ALS to define how to tolerate risk in early phase trials so that we can balance scientific progress and participant safety and continue moving science forward. The project will use a combination of patient-oriented and researcher-oriented surveys, followed by detailed interviews to characterize the breadth of perspectives throughout the ALS community on how to tolerate risk for new therapeutic development. These community-driven results will inform the development of consensus recommendations to be used for planning and advancement of early phase clinical trials in ALS.

Garrett ALS Clinical Research Award

Mark Levine, MD

Project: Dr. Levine will contribute to several collaborative ALS research initiatives that will enhance his development as a clinical and translational ALS investigator. He will partner with Dr. Mark Albers in the Memory Division to validate TDP-43 mislocalization in cells isolated from nasal brushing biopsies in people living with ALS. He will also serve as a Co-Investigator and Site Principal Investigator for multiple ALS clinical research studies, including trials evaluating a novel therapeutic approach, investigating the biological effects of tofersen in adults without SOD1-associated ALS, and advancing interventions to improve quality of life for patients and caregivers. These experiences will broaden his expertise in biomarker research, clinical trial leadership, and multidisciplinary collaboration.

Garrett Outstanding Achievement Award for Early Career ALS Research

Adam Wahida, MD, PhD

Project: TDP-43, the key pathogenic protein in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).  

TDP-43 mislocalizes into cytoplasmic aggregates that commit motor neurons to death through mechanisms that remain undefined. The goal of Dr. Wahida's project is to apply chemical proteomics to map the structural proteome of disease in ALS, capturing how protein aggregation reshapes protein conformation and interactions in ALS patient tissue and iPSC-derived neurons. In collaboration with the laboratories of Dr. Miguel Rivera and Dr. Clotilde Lagier-Tourenne at MGH, Dr. Wahida's work looks to generate the first ALS Aggregate Atlas and identify new, previously undruggable therapeutic targets for ALS/FTD patients.

Mussallem Transformative Scholar Awards

Matthew Nolan, PhD

Project: ALS and FTD are fatal brain diseases that cause progressive paralysis, changes in behavior, and language difficulties. In most people with these conditions, a crucial protein called TDP-43 malfunctions and leaves the nucleus of the cell. When this happens, neurons lose another vital protein called stathmin-2 (STMN2),which we believe is critical for neuronal health. Without STMN2, neuronal projections breakdown, leading to nerve damage. Our recent work has shown that statins—common cholesterol-lowering drugs—can promote STMN2 levels, suggesting that statins can make brain cells much more resilient. However, scientists do not yet know how statins achieve this effect. Using a cutting-edge functional genomics approach, the goal of this project is to therefore identify exactly which genes and biological pathways allow statins to protect the neurons that are vulnerable in ALS and FTD. 

Su Min Lim, PhD

Project: In ALS and frontotemporal dementia (FTD), the RNA-binding protein FUS mislocalizes from the nucleus to the cytoplasm, where it aggregates and drives motor neuron death. Dr. Lim deployed Optical Pooled CRISPR Screening, a functional genomics platform she adapted to ALS patient-derived fibroblasts and neurons, to identify the genetic regulators of FUS mislocalization at single-cell resolution. Her screen identified the KEAP1-NRF2 axis as a top therapeutic target: silencing KEAP1 activates NRF2, reducing FUS mislocalization across multiple ALS-causing mutations in human iPSC-derived neurons. She will now map the key genetic dependencies of this rescue pathway using targeted CRISPR screening, and testKeap1antisense oligonucleotides in vivo, generating the preclinical evidence needed to advance toward clinical translation. 

Impact of philanthropy

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 our website.

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