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Sean M. Healey & AMG Center for ALS
Spring 2025 Newsletter
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Greetings from the Sean M. Healey & AMG Center for ALS, | |
Spring is here, with its sense of hope and renewal, sentiments we feel strongly here at the Sean M. Healey & AMG Center for ALS. With this issue, we are celebrating the recognition of several of our investigators for their dedication to solving the mysteries of ALS and research progress in innovative studies, including a smell test to detect ALS earlier, advances in our understanding of the neural dynamics of conversation, and a reinforcement of multidisciplinary approach to ALS care.
We are excited about upcoming initiatives and have recently opened our call for experimental therapies to be considered for the new ALS MyMatch ALS early clinical trial initiative. Our matching gift challenge for this project is currently underway. While we have already surpassed the halfway mark toward our goal of matching the $2 million philanthropic grant, the challenge is still ongoing, and we are eager to reach our target to launch the first two ALS MyMatch Trials.
We also look forward to announcing a new group of promising scientists whose work will be supported by awards generously supported by our donors.
We are inspired, as always, by our growing ALS community and your continued commitment to our work.
Warmly,
Merit Cudkowicz, M.D., MS.c.
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Novel Nasal Brushing Technique
Dr. Mark Albers, MD, PhD, from the Healey & AMG Center in collaboration with Dr. Eric Holbrook of Mass Eye & Ear, are pioneering a novel nasal brushing technique to detect TDP-43 pathology in ALS patients, a protein mislocalized in 97.3% of ALS cases. The technique involves using a camera-aided brush to harvest olfactory neurons, which regenerate, ensuring no loss of smell for participants. This five-minute procedure aims to identify TDP-43 presence in living individuals, potentially indicating ALS risk and aiding in neurodegenerative disease research. Dr. Albers hopes to detect pre-symptomatic changes and evaluate drug efficacy in clinical trials by comparing TDP-43 levels before and after treatment. Learn more here.
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Integrated Target Discovery Program 43 (iTDP43)
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TDP-43 is a protein expressed in most cells that is mislocalized in neurons in 97% cases of ALS, in addition to other neurodegenerative diseases. It is critical to identify and validate novel therapeutic targets that address central mechanisms of TDP-43 pathology.
An important step in drug development is to demonstrate that the potential new therapy corrects pathological changes in different models of the disease. To date, validation studies have been conducted under varying conditions, with distinct outcomes, and different analysis methods. To improve this drug development pipeline, the laboratories of Drs. Clotilde Lagier-Tourenne, Mark Albers and Brian Wainger have come together to launch the integrated Target Discovery Program 43 (iTDP43). In this program, careful curation of multiple ALS disease models with clinically relevant endpoints forms a suite of assessments for new drugs that target TDP-43 mislocalization and pathology. This suite was developed in part by applying learnings from our own clinical trial experience.
iTDP43 aims to transform the translational pipeline for ALS, by assessing efficacy of newly identified drugs across multiple and complementary in vitro (stem cell-derived neurons) and in vivo ALS models that span different aspects of TDP-43 dysfunction (e.g., TDP-43 mislocalization, Stathmin-2 loss, neuroinflammation). We will first test novel drugs identified by the laboratories at the Healey & AMG Center and utilize this platform to prioritize drugs for future translational studies. Once established, this preclinical framework can be utilized to interrogate other newly identified drugs, with the long-term goal of improving clinical trial success rates for ALS and other neurodegenerative diseases with TDP-43 disruption.
For more information, please contact Dr. Alexander Henderson, Program Director, ahenderson9@mgh.harvard.edu.
| | | At the Healey & AMG Center for ALS, our patients are our priority. We are dedicated to advancing ALS treatment and understanding, and our work is making significant strides in care and research. We are thrilled to share the latest news and developments from our center with you. | The Sean M. Healey & AMG Center for ALS is thrilled to announce the 2025 MGH ALS Young Investigator Awards. These awards celebrate the work of researchers and innovators in the field of ALS research. Learn more about the 2025 awardees here. |
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2025-2027 Mussallem Transformative Scholars in ALS
Supported by the Linda and Mike Mussallem Foundation, the Mussallem Transformative Scholars in ALS are brilliant young scientists committed to advancing the most innovative and exciting ALS research.
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YuYu Song, MD, PhD: Selective Vulnerability in ALS and FTD: from cells to molecules
Assistant Professor, Department of Neurology, associated with the MassGeneral Institute for Neurogenerative Diseases
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Seung Hun Park, PhD: Development of Mitochondria-Targeted Probes for Non-Invasive Monitoring and Therapeutic Intervention in ALS
Instructor, Gordon Center for Medical Imaging, Division of Radiological Sciences, Mass General
| | | 2025 Byrne Family and Judith & Jean Pape Adams Endowed Fellowship | | | | |
Adel Boudi, Ph.D.: Investigating gene fusion-driven impairments in Amyotrophic Lateral Sclerosis.
Post-doctoral Research Fellow, Department of Neurology, Massachusetts General Hospital Neuroepigenetics Lab at the MassGeneral Institute for Neurodegenerative Disease.
| | Congratulations to Clotilde Lagier-Tourenne, M.D., Ph.D., Araminta Broch-Healey Endowed Chair in ALS at Massachusetts General Hospital on receiving the prestigious Javits Neuroscience Investigator Award from the National Institute of Neurological Disorders and Stroke (NINDS). This distinguished award, established by the U.S. Congress in honor of the late Senator Jacob K. Javits (R-NY)— a dedicated advocate for research — recognizes investigators with an exceptional record of scientific excellence and productivity in neuroscience. Dr. Lagier-Tourenne’s outstanding work and contributions to the field, particularly in neurodegenerative disease research, have earned this honor. | | | | | |
On Sunday, April 6th, Dr. Merit Cudkowicz was awarded the American Academy of Neurology (AAN) Lifetime Achievement Award for Clinically Relevant Research at the AAN 2025 Annual Meeting in San Diego, recognizing extensive contributions to ALS research.
Watch her acceptance speech.
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The Healey & AMG Center issued a Request for Applications of experimental therapies for inclusion in our newest initiative, Healey ALS MyMatch. This is a patient-centered, efficient approach for screening investigational products in early-phase biomarker driven (Phase 1b/2a) clinical trials in ALS across several experienced trial-ready enrolling centers. Applications are due May 16, 2025 for investigational products from the academic and industry community to be considered for inclusion in Healey ALS MyMatch in 2025. We received an anonymous matching gift from a foundation to launch this new initiative. Learn more here
Watch our Informational webinar
| | Clinical Spotlight - Access for ALL in ALS | | |
The Access for ALL in ALS (ALL ALS) research initiative - a nationwide effort to accelerate discoveries in ALS through broad participation and deep data collection - continues to make exciting progress with over 400 individuals enrolled across 33 sites activated so far. MGH is enrolling particularly well, with strong participation in both ASSESS and PREVENT studies with 52 and 63 participants enrolled, respectively.
Recruitment and retention activities remain very active, and will be propelled with the addition of two new patient engagement team members: Emma Allen and Emma Goldberg. Their roles include building relationships with ALS communities, guiding participants through the study process, and ensuring consistent communication and follow-up.
Every participant moves us closer to understanding more about ALS with the hope of discovering meaningful treatments. We are ALL in - join us in this vital effort!
To learn more about ALL ALS, please visit: ALL ALS Consortium | ALS Research | ALS Disease Progression
| | Latest Academic Publications | | 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 about more Expanded Access. | | |
Call for Nominations Recognizing Innovations in ALS
At the Healey & AMG Center for ALS we are committed to recognizing scientific breakthroughs and clinical transformations that are advancing ALS therapy development and solving clinical care challenges for people living with ALS. We are pleased to announce that nominations are open for our 2025 prizes, awards, and recognitions:
· Gupta Family Endowed Prize for Innovation in ALS Care
· Sean M. Healey International Prize for Innovation in ALS
· Drs. Ayeez and Shelena Lalji & Family ALS Endowed Award for Innovative Healing
· Drs. Ayeez and Shelena Lalji & Family Student Scholar Award
Please submit your nominations through our new application portal here.
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Let’s Put ALS on Ice
On March 29, ICE ALS hosted its inaugural "Let’s Put ALS on Ice", a special hockey game at the Warrior Ice Arena. Inspired by the life of Jon C. Shepard, a dedicated law enforcement officer and hockey enthusiast, a hockey game was played between The Blue Line Blues and the Boston Bruins Alumni with Ray Bourque as Captain. The event raised over $70,000 for ALS research, with partial proceeds benefitting the Sean M. Healey & AMG Center for ALS. You can learn more about the event here.
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Boston Red Sox Vs. Texas Rangers Ceremonial First Pitch
On May 6, Matt Barker, Healey & AMG Center patient will be throwing out the ceremonial first pitch at Fenway, in hopes of raising ALS awareness and funds for the Center. Good luck, Matt!
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Oxford to Boxford ALS Ride for Hope 2.0
On June 2nd to 4th, a group of highly committed cyclists (and their supporters), will cycle approx. 150 miles for Oxford, ME to Boxford, MA to raise awareness and funds for ALS research and patient care. This is the third annual mighty and meaningful ride in honor of Doug McIlvaine. A portion of proceeds will support ALS research at the Healey & AMG Center for ALS. Join or support the ride here.
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Annual Lou Gehrig Day June 1-2
Major League Baseball Commissioner’s Office, in partnership with the Lou Gehrig Day Committee, has selected the Healey & AMG Center for ALS as the beneficiary of their 2025 Lou Gehrig Day fundraising efforts. The MLB will be hosting an online auction of autographed Topps baseball cards and limited-edition Lou Gehrig Day t-shirts. Funds raised through these initiatives will support the Expanded Access Protocol (EAP) Program at the Healey & AMG Center for ALS. A link to the auction site will be shared when available.
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3rd Annual Team Goose Hockey Tournament
On June 21 in Wayne, NJ Team Goose Foundation will be hosting their annual 5v5 hockey tournament. Last year's tournament included 12 teams across two divisions with over 300 spectators. A portion of the proceeds raised through the tournament and it's auction support ALS research and care at the Healey & AMG Center for ALS. Details about the tournament can be found on the Team Goose website.
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Richard J. Wolnik Foundation for ALS Research Inaugural Golf Tournament
Join the Richard J. Wolnik Foundation for ALS Research for a day of golf, laughter, and community as they come together to raise funds and support groundbreaking ALS research and the search for a cure in memory of Dick. The tournament will take place on Monday, June 30 at Indian Ridge Golf Club in Andover, MA. Details and registration can be found on the Ricard J. Wolnik Foundation website.
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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/.
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Massachusetts General Hospital
55 Fruit Street
Boston, MA 02114
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