Sean M. Healey & AMG Center for ALS

Fall 2025 Newsletter

Dear Friends,

As we reflect on this season of gratitude, we are filled with appreciation for the incredible community that makes our work at the Healey & AMG Center possible. Your generosity, partnership, and unwavering support fuel our mission every single day—to accelerate progress and bring hope to every person and family affected by ALS.



We are thrilled to share several milestones that you helped make possible:

• Our first Healey ALS MyMatch Trial just completed enrollment. This trial allows for more personalized therapies for individuals with ALS.
• This year marks the 30th anniversary of the Network of Excellence for ALS (NEALS), which brings together families, researchers, industry and others in the ALS community to accelerate progress. We were so pleased to celebrate NEALS’ legacy of impact at the annual meeting in Florida and share new ideas for the next few years.
• Sabrina Paganoni, MD, PhD, has been elected to the National Academy of Medicine, one of the highest honors in health and medicine. Dr. Paganoni has been the driving force behind the HEALEY ALS Platform Trial, which is adding additional regimens to the trial.
• We welcome Jennifer Morganroth, MD, MBA, to the Healey & AMG Center team. Dr. Morganroth cares for individuals living with ALS and is at the forefront of leveraging AI and implementation science to make clinical trials more efficient.

At the Healey & AMG Center, we are grateful every day for all of you who make our ALS progress possible. Together, we will find the cures.

Warmly,

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

30 Years of NEALS

2025 marks the 30^th Anniversary of the Northeast ALS (NEALS) Consortium, recently renamed to the Network of Excellence for ALS (NEALS).

Founded in 1995 by Merit Cudkowicz, MD, MSc and Jeremy Shefner, MD, Phd, NEALS has advanced ALS research through collaboration with individuals, families, researchers, clinicians, industry partners, and ALS organizations. NEALS brought the community together to work in sync and speed discovery. 

Accomplishments in the past 30 years include:

• Sharing data with the scientific community. NEALS worked to launch the PROACT database to make clinical data from trials open. They also established the NEALS biorepository that currently houses over 100,000 cryovials, sharing biofluid data that will drive new discoveries for biomarkers, including neurofilament light chain (NfL).
• Developing and implementing trial outcome measures and trial innovations, including the HEALEY ALS Platform Trial.
• Expanding from 9 academic clinical centers in the New England area to 165 sites, including 12 international sites.
• Providing trainings and webinars for 3,000 members, including people and families living with ALS, health care providers, scientists, and physicians in ALS therapy development.
• Working on the development of therapies for ALS, including QALSODY (tofersen).



This milestone demonstrates the remarkable dedication of the ALS community to accelerating progress, but the work is not yet done. NEALS will continue to build on these achievements in the coming years, devoting time to researching more personalized treatment approaches and accelerating biomarker and gene therapy approaches.

Clinical Spotlights

PACT Program Offers Parenting Support for Families Facing ALS

Learning to navigate life with ALS can be overwhelming for a person with the diagnosis, even more so if that person is also the parent of young children. That’s where the Daniella Lipper ALS Parenting At a Challenging Time (PACT) Program provides support.

The ALS PACT Program offers guidance to parents and grandparents with ALS on how to talk to children about the illness and support their healthy coping with it. PACT Program director, Dr. Cindy Moore, along with Dr. Christine Wu and Dr. Allison White, work with families to help children and young adults, up to age 25, cope with an ALS diagnosis in a parent or grandparent. 

Welcoming Dr. Jennifer Morganroth

Welcome Dr. Jennifer Morganroth to the Division of Motor Neuron Disorders and the Sean M. Healey & AMG Center for ALS. She earned a dual M.D./M.B.A. with a concentration in healthcare management from the Perelman School of Medicine and The Wharton School at the University of Pennsylvania, where she also completed her neurology residency. She then completed a fellowship in EMG and Neuromuscular Diseases at Columbia University. Her research focuses on the application of artificial intelligence and implementation science to improve clinical trial efficiency and expand access to gene-targeted therapies. She is also the recipient of the 2025 Mussallem Transformative Scholars Award. 

Strategic Research Initiatives

HEALEY ALS Platform Trial

Upcoming Regimen

We are pleased to announce that operational start-up for a new regimen, NUZ-001 from Neurizon Therapeutics, in the HEALEY ALS Platform Trial is underway. 



Read the Trial Design Announcement

Healey ALS MyMatch

Completion of Enrollment for First Trial

We are excited to announce that enrollment is complete for our first ALS MyMatch trial, the ACACIA Trial. Over a four-month enrollment period, 41 participants started study medication at three clinical trial sites. We have several other investigational products that will enter Healey ALS MyMatch trial shortly!

Read the Announcement

Watch a Webinar on the ACACIA Trial

From the Bench

Dr. Christine Marques' Work on Digoxin in the Wainger Lab

ALS is a complex disease, with distinct injuries to the command center of a motor neuron, called its cell body, and to its main branch, called an axon, which connects the neuron to muscle. Effective treatments may be needed to prevent both injuries at once. Dr. Christine Marques, an Instructor at the Wainger Laboratory in the Healey & AMG Center, designed drug screens with a goal of identifying drugs that could protect both a neuron’s cell body and axon from ALS. Using libraries of chemicals, including FDA-approved drugs, she performed independent screens to identify drugs that could protect human neurons, made from induced pluripotent stem cell technology, from ALS-relevant disease stressors. Surprisingly, she obtained a class of drugs called cardiac glycosides, including the FDA-approved heart medication digoxin, that protected both neuronal cell bodies and axons in the two independent screens. Dr. Marques and her team have validated the benefit of digoxin in different cellular and mouse models, including showing benefit on motor function in ALS model mice. These results, along with other existing data supporting digoxin, were sufficient to warrant a clinical study. The trial is led by Dr. Suma Babu (Primary Investigator) and Dr. Brian Wainger (Co-Primary Investigator) and is the first study within the Healey ALS MyMatch Program of early-phase clinical trials.

Latest Publications

Autoimmune response to C9orf72 protein in amyotrophic lateral sclerosis

Results from a recent study reinforce the hypothesis that neuroinflammation plays an important role in ALS disease progression.  Healey & AMG Center faculty collaborated with the lead authors to provide important samples and clinical data. The findings identify an antigenic target that could enable more targeted, antigen-specific therapeutics in ALS.

Research access barriers in amyotrophic lateral sclerosis



Some key barriers that limit participation in ALS clinical research studies, and outlined strategies to overcome them are described. These include increasing education and engagement, expanding outreach, and strengthening partnerships. The authors also provide available resources for accessing clinical research.

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, Michelle Redenz 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

Study of ASSESS ALS ALS

• Purpose of Study: To study people diagnosed with ALS and healthy participants to further our understanding of the disease and potential biomarkers of disease progression. The information collected in this study may contribute to future research and development of new treatments for ALS and similar neurological diseases.

Respiratory Comorbidity Detection Using Digital Devices (Empatica)

• Purpose of Study: We aim to improve monitoring of respiratory complications like pneumonia, pulmonary embolisms or deep vein thromboses in people living with ALS using smartwatch sensors (Empatica device). The study could help develop better detection and treatment methods for these respiratory complications related to ALS.

Clinical Research Webpage

Upcoming Healey & AMG Center Webinars

Thursday, November 13th, 5:00 pm EDT: Expanded Access Discussion

Thursday, November 20th, 5:00 pm EDT: Platform Trial Update

Thursday, December 11th, 5:00 pm EDT: Expanded Access Discussion

In the Community

Cans for a Cure

As his Bar Mitzvah project, Elad led a Cans for a Cure challenge, collecting and recycling cans to raise nearly $2k for the Healey & AMG Center, in honor of his uncle who passed away from ALS. We were glad to welcome Elad for a visit to the Center and are proud of his successful fundraising!

5th Annual Joseph Kiley Walk to End ALS

The Joseph Kiley Foundation hosted their fifth annual Walk to End ALS on October 5 in honor of Joe Kiley, a former Healey & AMG Center patient. Doreen Ho, MD and Dario Gelevski attended the beautiful event and spoke on behalf of the Center, sharing progress that's been possible in part to the generosity of the foundation and their support over the past 5 years. This year's walk raised over $25,000, bringing their total fundraising for ALS research here at the Center to over $90,000, with a goal of surpassing $100,000 next year! The Foundation has also recently started the Joseph Kiley Memorial Grant program to support individuals and their families as they participate in clinical trials for ALS research. Click here to learn more.

A Visit with the Will Hardy Foundation

Representatives from the Will Hardy Foundation visited to learn more about the impactful care, research and education taking place across the Mass General Brigham Neuroscience Institute. A huge thank you to Drs. Mike Talkowski, Florian Eichler, Alice Stanton, Suma Babu, Sabrina Paganoni and James Berry for providing tours and presentations. At the end of their visit, Will Hardy, head coach of the Utah Jazz, and Rob Long, executive director of the Will Hardy Foundation, presented their pledge of $100,000 in support of the Sean M. Healey & AMG Center for ALS. These initial funds will support the Healey ALS MyMatch Trial. We are excited about this new partnership. A few of us were able to attend the Celtics/Jazz game and have a little time courtside. 

Announcements and Awards

PRO-ACT Receives Generous Support from Kathy Garrett Research Center for ALS to Advance ALS Research

We are thrilled to announce that the PRO-ACT (Pooled Resource Open-Access ALS Clinical Trials) platform has been awarded a one-year grant from Kathy Garrett Research Center for ALS, marking a pivotal moment in our mission to accelerate ALS research.

This generous support will empower us to expand and enhance PRO-ACT’s capabilities, including new data visualization tools, advanced query features, and improved accessibility for researchers worldwide. As the largest publicly available ALS clinical trials dataset, PRO-ACT has already transformed how scientists and clinicians approach ALS research—enabling data-driven insights, fostering collaboration, and informing trial design.

“This grant from the Kathy Garrett Research Center for ALS is more than just funding—it’s a powerful endorsement of open science and collaborative innovation. With their support, PRO-ACT will continue to empower researchers worldwide in the fight against ALS.”

— Alexander Sherman, Principal Investigator, PRO-ACT

The Kathy Garrett Research Center for ALS’s commitment to innovation and open science reflects a shared vision: that by breaking down barriers to data access, we can drive meaningful progress toward understanding and ultimately curing ALS.

Continued support is essential to build on this momentum – if you are interested in learning more, please contact Emily Monteiro at emonteiro7@mgb.org to explore how you can get involved and help bring new tools and resources to the ALS research community.

Dr. Sabrina Paganoni Elected to National Academy of Medicine

Sabrina Paganoni, MD, PhD, was elected for her leadership in paradigm-shifting ALS research and care, development in innovative national platform trial infrastructures to accelerate testing of novel agents, and advancement of molecular and functional biomarker identification to facilitate precision medicine approaches. Her expanded access programs and collaborative integrative model have propelled the generalizability and impact of these results.

Congratulations Dr. Paganoni!

Dr. Ghazaleh Sadri-Vakili Named Kathy Garrett Endowed Chair in Neurology

On September 29th, we celebrated Ghazaleh Sadri-Vakili, MS, PhD as the inaugural incumbent of the Kathy Garrett Endowed Chair in Neurology at Mass General Brigham.

Dr. Sadri-Vakili is the Director of the NeuroEpigenetics Laboratory at Mass General​, a Scientific Advisor at the Healey & AMG Center for ALS, and an Associate Professor of Neurology at Harvard Medical School. Her work focuses on studying common mechanisms that cause neurodegenerative diseases and identifying novel therapeutic targets.

Dr. Sadri-Vakili shared, "This honor would not be possible without Kathy Garrett. I want to express my sincere gratitude to Kathy for her generosity and her vision. By investing in academic scholarship and innovation, she has ensured that future generations of researchers will continue to benefit from opportunities to pursue knowledge and discovery in the hopes of finding a cure for ALS."

Congratulations to Dr. Sadri-Vakili on this accomplishment!

2025 Innovation Prizes

 A tailor-made drug slowed a man's ALS. Is this the future of treatment?

Dr. Suma Babu, Co-Director of the Neurological Clinical Research Institute at Massachusetts General Hospital and PI of Healey ALS MyMatch, is mentioned in this article about the hope that precision medicine brings for ultra rare genetic forms of ALS.

 Congratulations, Matthew Nolan!

Matthew Nolan, PhD, was awarded a Muscular Dystrophy Association and ALS Network Development Grant for his project, “Developing novel chemical and genetic regulators of Stathmin-2 in ALS.”

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 https://www.massgeneral.org/neurology/als/support/.

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