Sean M. Healey & AMG Center for ALS

Winter 2025 Newsletter

Greetings from the Sean M. Healey & AMG Center for ALS,

Here at the Sean M. Healey & AMG Center for ALS, we are proud of our ALS community, a diverse group of people committed to finding the cures for ALS. Together, we are making a difference, with progress on the HEALEY ALS Platform Trial, more sites joining our Expanded Access Protocol program, and encouraging progress in our basic science laboratories. 

Our newest initiative, ALS MyMatch, is an early-phase, biomarker-driven trial program that will identify subgroups of ALS and efficiently pinpoint potential therapies, accelerating the development of effective interventions. A generous partner has offered a challenge grant that we must match to launch the first two, ALS MyMatch early-phase trials next year. We are excited by the promise of ALS MyMatch and are eager to meet the match and move forward. 

 More information about all our initiatives is included in this newsletter. 

 So much of our progress is made possible by your partnership and participation. I know that together, we will find the cures. 

 With deepest gratitude, 

Merit Cudkowicz, M.D., MS.c.

New & Noteworthy

At the Healey & AMG Center for ALS, our patients are our priority. Our work is shaping the landscape of ALS treatment and understanding, and we are excited to share the latest news from our center with you. 

Healey & AMG Center for ALS Launches ALS MyMatch: an Early Phase Biomarker Driven Trial Program 

We are proud to announce the launch of ALS MyMatch, an exciting new initiative for performing early-phase ALS trials. ALS MyMatch is an innovative, multi-center, biomarker-driven trial initiative aimed at bringing new efficiencies to early-phase ALS trials.  

The importance of early-phase trials cannot be understated. Well-designed early-phase trials set the foundation for all of drug development – identifying the optimal dosages for future testing, solidifying biomarker responses, and honing the appropriate trial population. Yet early-phase ALS trials can be expensive and may be prolonged by inefficient trial designs.  

An anonymous Foundation has provided generous support for this initiative and challenged us to match their $2 million philanthropic grant in the next 12 months. With these funds, we will establish a multi-site trial-ready network and launch the first two ALS MyMatch Trials.  

Please consider a gift to help us towards our $2 million fundraising goal. Please contact Emily Monteiro at emonteiro7@mgh.harvard.edu or visit https://www.massgeneral.org/neurology/als/support/.

 Read More 

James Berry, MD, MPH Appointed Chief of MGB Division of ALS and Motor Neuron Diseases 

Mass General Brigham (MGB) has announced that James D. Berry, M.D., MPH, has been named the inaugural Chief of the Division of ALS and Motor Neuron Diseases in the Department of Neurology. A dedicated clinician and researcher, Dr. Berry has played a key role in expanding ALS care, advancing innovative treatment models, and leading major clinical research initiatives. 

As an Associate Professor of Neurology at Harvard Medical School and Co-Chair of NEALS, Dr. Berry has been at the forefront of ALS research, biorepositories, and decentralized clinical trial methodologies. Dr. Berry has been with MGB since his Neurology Residency and has served as Chief of the Division of ALS and Motor Neuron Diseases since 2019. We congratulate him on this well-earned appointment and look forward to the future of ALS and neurodegenerative research under his leadership. His new role begins on March 1, 2025. 

Latest Advances from the HEALEY ALS Platform Trial

The HEALEY ALS Platform trial is an adaptive, perpetual trial allowing multiple treatments to be tested simultaneously, which speeds up the process of finding effective therapies for ALS. Most recently, we reported updates to the Master protocol design, topline results from our 6th and 7th Treatment regimens, and primary publications for the first four regimens tested in this groundbreaking trial. We are accepting applications for new partners, and are currently in discussions with several pharma and bio tech companies about new regimens for the HEALEY ALS Platform Trial.  

First Four Studies of HEALEY ALS Platform Trial Published in JAMA Network: Accelerated Drug-Testing Platform for ALS Paves Way for Therapeutic Innovation 

The ongoing HEALEY ALS Platform Trial completed evaluations of its first four treatment regimens, using a common research setup that makes trials more efficient and cost-effective to bring treatments to patients faster.  

Each of the first four trials included approximately 160 patients, with roughly 120 receiving an investigational drug and 40 receiving a placebo. As phase 2 trials, the goal of these studies was to rapidly assess drug candidates and determine whether to proceed with larger trials. Of the four regimens, two drugs (CNM-Au8 and pridopidine) are moving to phase 3 testing. Though neither met criteria demonstrating statistical significant benefit over 24 weeks, the phase 2 trials showed promising trends in other outcome measures and biomarkers and helped clarify dosage information and target populations, informing the phase 3 design. 

Read the full announcement

Learn more about how to join the trial here

In addition to the publication of the first four regimens of the HEALEY ALS Platform Trial, a recent paper published in Nature Aging highlights the groundbreaking operational and scientific efficiencies of the trial. The study explores how this innovative trial design can accelerate discoveries in ALS and potentially be applied to other diseases.  

This work was made possible through the generous support of the AMG Charitable Foundation, Tackle ALS, The ALS Association, ALS Finding a Cure, the Muscular Dystrophy Association, ALS ONE, the Arthur M. Blank Family Foundation, I AM ALS, the Tambourine ALS Collaborative, and numerous community fundraising initiatives and donors. 

Expanding Access to Experimental Therapies

We are committed to ensuring individuals living with ALS across the U.S., who may not be eligible for clinical trials can access investigational products. Visit our EAP webpages to learn more about Expanded Access. 

UMN, Essentia Health Dose First Participants in Healey & AMG Center, Never Surrender Inc. Expanded Access Protocols 

The Healey & AMG Center and Never Surrender, Inc. announced the successful first dosing of participants in the Expanded Access Protocol (EAP) programs at the University of Minnesota (UMN) and Essentia Health.   

Read More 

HEALEY ALS Platform Trial & EAP Webinar Series

Join us on Thursday, March 13th at 5 pm EST to hear the latest expanded access protocol updates.



Register

From the Bench

We are excited to be expanding our network of ALS researchers and the impactful work being done across our labs at Mass General Brigham. This month, we highlight the contributions of Mass General Brigham neuroscientists Clare Baecher-Allen, PhD, and Andrew Stern, MD, PhD, whose dedication and innovative research are advancing the field of ALS.

Read More

Clinical Spotlight: Healey & AMG Center Biorepository

Pictured left to right: Dr. James Berry, Dario Gelevski, Becky Fillingham, and Ryan Pogemiller. 

The Healey & AMG Center Biorepository, a branch of the larger NEALS Biorepository, houses biofluid samples collected from people living with ALS and healthy volunteers. These samples, including plasma, serum, cerebrospinal fluid, and whole blood, are stored and shared with the ALS research community worldwide. Led by Manager Becky Fillingham, the NEALS Biorepository/ALS Living Library at Mass General has stored and shared over 100,000 such samples, playing a vital role in contributing to the ALS therapeutic pipeline and the discovery of ALS biomarkers for over twenty years. 

Visit the webpage

In The Community

The success of the Healey & AMG Center for ALS would not be possible without the valuable contributions from the ALS community. The year so far has brought together extreme athletes, support from the Major League Baseball & National Football League Associations, and reinvigorated the spirit of the Ice Bucket Challenge, ten years later.

Together, we're making waves of change and moving closer to a future without ALS.

Visit our Community Support page for information about upcoming and past campaigns.

A Day to Remember, A Day to Forget

Three Healey & AMG Center families were invited to the New York Rangers vs. Vegas Golden Knights game as part of Team Goose Foundation's "A Day to Remember, A Day to Forget". The experience included a meet and greet with NY Rangers defenseman, Adam Fox.

Let’s Put ALS on Ice  

ICE ALS is hosting "Let’s Put ALS on Ice", a special hockey game at the Warrior Ice Arena on Saturday, March 29 from 11am-1pm. Inspired by the life of Jon C. Shepard, a dedicated law enforcement officer and hockey enthusiast, a hockey game will be played between The Blue Line Blues and the Boston Bruins Alumni with Ray Bourque as Captain. The feeling of lacing up and having the ability to play hockey where ALS Patients who are athletes can no longer participate takes our passion for hockey to new levels. We hope that you will join us as a spectator, sponsor or volunteer as The Blue Line Blues face-off against Hall of Famer Ray Bourque and the Boston Bruins Alumni. Partial proceeds will benefit the Sean M. Healey & AMG Center for ALS. All are welcomed and the venue is accessible.

You can learn more here. For questions or to sign up, please contact Jamie Shepard at jshepard@iceals.org. 

Celebrating a Milestone



Founded in 2018, the Tackle ALS campaign which supports the HEALEY ALS Platform Trial (Season 1) and ALS repair and regeneration research (Season 2) has surpassed $10M in funds raised! We are so grateful to the Tackle ALS community of over 5,000 donors who have helped us achieve this milestone. To learn more about Tackle ALS or form a team, visit TackleALS.com

Impact of Philanthropy

Our work at the Sean M. Healey & AMG Center for ALS combines outstanding, multidisciplinary care with clinical and basic research informed by the needs of our patients. Philanthropy allows us to accelerate the pace of discovery and make a meaningful difference to our patients and their families. Your support helps to fuel breakthroughs and discoveries in the field of ALS and ensure everyone with ALS can access effective treatments and therapies We are determined to find the cures and we are confident your partnership will get us there. 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/.