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Sean M. Healey & AMG Center for ALS
Summer 2025 Newsletter
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Thanks to your participation and support, this summer has been an exciting one for all of us at the Sean M. Healey & AMG Center for ALS. We are working across research areas, sharing critical data gathered through the HEALEY ALS Platform Trial to inform other studies, even as we design trials for additional promising compounds; and collaborating with bioengineers (like our newest faculty member, Alice Stanton, PhD, interviewed below) and experts in Artificial Intelligence/Machine Learning in our efforts to get ahead of ALS for earlier diagnosis, effective treatments and ultimately, prevention.
We also took a moment to congratulate clinician-scientist Sabrina Paganoni, MD, PhD, named the inaugural incumbent of the Robert H. Brown, Jr., DPhil, MD Endowed Chair in Neurology, thanks to the generosity of the Estate of Kathy Garrett, and recognized three new 2025 ALS Scholars in Therapeutics Awards, a global program made possible through our partnership with ALS Finding a Cure® and FightMND.
We are deeply grateful to every member of our ALS community. We walk this path together, inspired by the patients we care for and united by a sense of purpose and urgency.
Warmly,
Merit Cudkowicz, M.D., MS.c.
| | Stanton Lab Shares Inspiring Vision for the Future of Neuroscience | |
A new member on the team at Mass General Brigham's Center for Genomic Medicine and Department of Neurology, Alice Stanton, PhD, is ready to hit the ground running.
Dr. Stanton is committed to advancing precision medicine for the brain and is pursuing several lines of research aimed at transforming the pace and scope of therapeutic discovery. This includes developing more advanced brain models and nanotechnology to address disease burden, advancing strategies for drug delivery to the brain, and tailoring therapies to individual patients based on their genetic profiles.
| | Strategic Research Initiatives | | HEALEY ALS Platform Trial | | Data from Regimens A through E successfully integrated into PRO-ACT database | | |
We are pleased to announce the successful integration of data from participants who received placebo during the randomized -controlled period of the first five regimens (A-E). Retired data from the HEALEY ALS Platform Trial is now available in the PRO-ACT database - the world’s largest ALS clinical trials dataset.
By contributing this data to PRO-ACT, scientists around the world will have access to the tools they need to make discoveries faster and design better, more efficient trials. We are committed to data sharing to accelerate ALS research and hope to foster research partnerships by making this information available.
We would like to thank everyone who has taken part in the HEALEY ALS Platform Trial. Your contribution is now helping researchers answer critical questions about how ALS works and how to improve research.
Read the press release here.
We also invite you to attend an upcoming community Q&A webinar on Thursday, September 25th at 5:00 pm, featuring members of the ALS community. A panel of guest speakers will share their perspectives on the integration of placebo data into the PRO-ACT database and why this milestone matters for the ALS community. Register here.
Design of TPN-101 for the Treatment of ALS
In collaboration with Transposon Therapeutics, Inc., we initiated design of a potential new HEALEY ALS Platform Trial regimen for TPN-101. Learn more.
| | Expanded Access Protocols (EAPs) | | 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 and stay connected with the latest developments in Expanded Access. | | We are excited to launch Healey ALS MyMatch, our newest program focused on a series of patient-centric, biomarker-driven Phase 1b/2a clinical trials. On the website, please find detailed information about the program. We also include links to past webinars that provide insights into the science, goals, and collaborative spirit behind ALS MyMatch. | | | |
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 webpage 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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Upcoming Webinars
Thursday, August 28th, 5:00 pm EDT: Biomarker Learnings from the HEALEY ALS Platform Trial
Thursday, September 11th, 5:00 pm EDT: Expanded Access Discussion
Thursday, September 25th, 5:00 pm EDT: HEALEY ALS Platform Trial Panel Discussion
Thursday, October 23rd, 5:00 pm EDT: Decoding HEALEY ALS Platform Trial Publications with Dr. Lori Chibnik
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Fourth Annual Rick Wilson Invitational Golf Tournament
On May 21-22, Comact hosted their 4th annual Rick Wilson Invitational golf tournament, in Marksville, LA, with partial proceeds benefiting the Healey & AMG Center. This two-day tournament is held in memory of former ALS patient and advocate, Rick Wilson. Healey Center nurses, Erin Clampffer, RN and Danica Sanders, RN attended and spoke about the mission of the Center and how grateful we are for Comact’s ongoing partnership. This year, they raised over $30,000 for ALS research, bringing their total raised for the Center to over $100,000!
| | Never Surrender Inc. Kolar Toyota ALS Fishing Tournament | |
On May 30–31, Drs. Merit Cudkowicz and Eric Rosenthal joined Never Surrender Inc. in celebrating their 30th Anniversary Fishing Tournament in Duluth, MN. Held over two days on Island Lake and virtually, the event drew participation from 218 anglers and over 100 volunteers and supporters. Never Surrender Inc. proudly supports the Healey & AMG Center for ALS, expanding access to cutting-edge ALS research for patients across Minnesota and neighboring states through partnerships with Essentia Health and the University of Minnesota.
| | Richard J. Wolnik Foundation for ALS Research Inaugural Golf Tournament | | |
On June 30th friends and family of Dick Wolnik gathered at Indian Ridge Country Club in Andover, MA for the inaugural Richard J. Wolnik Foundation for ALS Research Golf Tournament. An impressive 136 golfers were competitively generous on and off the course, raising $100,000 for Healey ALS MyMatch.
| | The Bourque Family Foundation Captain's Ball | | |
The 4th annual Bourque Family Foundation Captain's Ball in honor of Pete Frates will take place on Thursday, September 25 at Agganis Arena. The evening includes live music, silent and live auction and dancing to raise funds for the Frates Family Foundation, Healey & AMG Center for ALS and the Bourque Family Foundation. Join us to celebrate Pete's legacy and support the fight against ALS. Tickets can be purchased here.
| | Recently, Dr. Merit Cudkowicz sat down for an inspiring conversation with Mass General Brigham Neurology resident Dylan Neel. She discussed how she became interested in neuroscience and her passion for advancing clinical trials in ALS. Read the interview here. | | Dr. Sabrina Paganoni Named Robert H. Brown, Jr., DPhil, MD Endowed Chair in Neurology | |
On July 10th, we celebrated Dr. Sabrina Paganoni as the inaugural incumbent of the Robert H. Brown, Jr., DPhil, MD Endowed Chair in Neurology, thanks to the generosity of the Estate of Kathy Garrett.
Dr. Brown (UMASS Chan Medical School) is a leading scientist in amyotrophic lateral sclerosis (ALS) and has mentored ALS physician scientists and lab based scientists from around the world. He and his team also discovered the first gene, SOD1, that is linked to ALS. Dr. Paganoni (Massachusetts General Hospital) has served as principal investigator (PI) on multiple ALS clinical trials, including her role as co-PI of the HEALEY ALS Platform trial.
Dr. Brown shared, "Through Dr. Paganoni's work, this chair will extend the Mass General Hospital legacy of success in decoding and trying to find treatments for neurodegenerative diseases like ALS, Huntington's disease, and hopefully, many others."
We look forward to all that Dr. Paganoni will accomplish through this chair. Congratulations to Dr. Paganoni and Dr. Brown on this well-deserved honor!
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2025 ALS Scholars in Therapeutics Awards
The Sean M. Healey & AMG Center for ALS in partnership with ALS Finding a Cure® and FightMND is excited to announce the 2025 ALS Scholars in Therapeutics. This program funds young investigators researching new treatments for people living with ALS. Three recipients were selected for this global two-year program.
| American Neurological Association (ANA) Award | | Dr. Merit Cudkowicz received the 2025 ANA Award for Clinical and Scientific Excellence. Congratulations Dr. Cudkowicz! | | 2025 Corcoran Family Award | | |
Eric Rosenthal, MD is the 2025 recipient of the Corcoran Family Award. He is the medical director of the Mass General Neurosciences Intensive Care Unit and program director of the MGB NeuroAI Center. For over a decade, Dr. Rosenthal has been leveraging artificial intelligence and machine learning (AI/ML) to develop tools for predicting patient outcomes, including for brain aneurysms, coma recovery, cardiac arrest, and seizure management. Recently, he has focused on creating large, AI-ready datasets from diverse sources to improve patient care.
With the Corcoran Family ALS Award, Dr. Rosenthal will extend his work to ALS, aiming to develop AI/ML tools for earlier ALS diagnosis. He explains, "The number of treatments for ALS continues to grow. To help patients with ALS receive the greatest benefit from these treatments, we need to diagnose patients at the earliest stage possible. We aim to learn the earliest time at which a pattern or trend in these clinical findings should be referred for definitive diagnosis by an ALS specialist." He and Morteza Zabihi, PhD, will recruit a postdoctoral fellow in AI to analyze existing ALS research data and create new datasets for AI/ML model training.
Dr. Rosenthal noted that, “Large research datasets developed through the participation of patients with ALS are incredibly helpful to train AI/ML models that can then be validated or fine-tuned in other settings. In this way, the broad community of ALS patients can benefit from their power of those who have participated in research.”
Congratulations to Dr. Rosenthal!
Previous Corcoran Award recipients include Doreen Ho, MD (2024) and Mark Garret, MD (2023).
| | 2025-2026 Byrne Family Endowed Fellowship in ALS Research | | |
Adel Boudi, PhD is the recipient of the 2025-2026 Byrne Family Endowed Fellowship. His project is titled “Investigating gene fusion-driven impairments in Amyotrophic Lateral Sclerosis.”
Dr. Boudi is a postdoctoral research fellow working under the mentorship of Dr. Ghazaleh Sadri-Vakili at Massachusetts General Hospital and Harvard Medical School. Dr. Boudi earned his master’s degree in Neuroscience from Sorbonne University in Paris and completed his PhD at King’s College London, where he investigated a novel cell death pathway in a mouse model of Dentatorubral-Pallidoluysian Atrophy, a rare inherited neurodegenerative disorder. In 2019, he joined MGH, contributing to the development of promising therapeutic strategies and biomarkers on Huntington’s disease. He joined Dr. Ghazaleh Sadri-Vakili’s lab in 2024, bringing his expertise to advance research in Amyotrophic Lateral Sclerosis (ALS).
Dr. Boudi is leading a pioneering study on fusion genes, a genetic anomaly well known in cancer, but only recently reported in individuals with ALS by Dr. Sadri-Vakili’s team. His research centers on the FAM69A-EVI5 fusion gene, one of the top candidates linked to ALS. By studying FAM69A-EVI5 effects in neuron-like cells and iPSC-derived motor neurons, this project aims to uncover its role in ALS pathogenesis. These findings have the potential to reveal novel mechanisms driving ALS and pave the way for the development of new therapeutic strategies.
Congratulations to Dr. Boudi!
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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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Massachusetts General Hospital
55 Fruit Street
Boston, MA 02114
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