Greetings from the ALS Multidisciplinary Clinic in the Sean M. Healey & AMG Center for ALS at Mass General,
Progress in our quest for ALS cures continues to accelerate.
- New discoveries on ALS biology by Dr. Clotilde Lagier-Tourenne, Araminta Broch-Healey Endowed Chair in ALS;
- Three additional investigational therapies were added to the HEALEY ALS Platform Trial;
- Results from the first four investigational therapies in the HEALEY ALS Platform Trial are helping refine our therapeutic targets with two therapies proceeding to phase 3 testing in ALS;
- Growth of our expanded access protocol (EAP) program, making participation more available across the US;
- Our clinicians are finding creative ways to improve the care we deliver – including testing a robotic wearable that supports arm function.
At the Healey & AMG Center for ALS at Mass General we are committed to every aspect of ALS – from basic science investigations of the molecular basis of the disease through programs that improve the quality of life for individuals living with the disease.
We have reached what I believe is a critical turning point in this work thanks to your support – as a participant, as an advocate, as a caregiver, and as a donor. I know I speak for every member of the Healey Center network, when I express my heartfelt gratitude for all you do.
Thank you for being part of our team. We cannot do it without you. Together, we will find the cures.
Warmly,
Merit
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FROM THE LAB OF DR. CLOTILDE LAGIER-TOURENNE |
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The lab of Clotilde Lagier-Tourenne MD, PhD, Araminta Broch-Healey Endowed Chair in ALS, investigates the molecular mechanisms driving neurodegeneration in amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and Huntington’s disease.
Dr. Lagier-Tourenne's innovative, biomarker-driven work has greatly impacted the work being done in the HEALEY ALS Platform Trial. Recently, Dr. Lagier-Tourenne co-led a study regarding the mechanism of STMN2. The study was published in Science on March 17th, and findings include that TDP-43 binding in the first intron of the STMN2 pre-mRNA sterically blocked access of RNA processing factors that would otherwise recognize and use a cryptic 3′ splice site.
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Updates from the Pivotal HEALEY ALS Platform Trial |
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The HEALEY ALS Platform Trial continues to make tremendous progress- testing multiple investigational drugs. Each drug is tested in a dedicated “regimen” and the trial continues to reach milestones and add new regimens to accelerate the development of effective treatments for ALS. Two of the first four treatments are proceeding to phase 3 testing, based on results from the HEALEY ALS Platform Trial. |
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Testing pridopidine, announces study results
Healey & AMG Center & the Northeast ALS Consortium announce results in platform trial with pridopidine.
On February 23rd, the topline results for regimen D, evaluating investigational product pridopidine (Prilenia), were announced. Some[CMEM1] highlights include substantial improvements in sustained phonation, speaking rate, articulation rate, and articulatory precision in participants taking pridopidine compared to those taking placebo, and slower progression among participants within 18 months of onset who also met El Escorial criteria for definite ALS. Important biomarker changes in neurofilament were also found.
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Regimen E,
Testing trehalose, announces enrollment completion
The HEALEY ALS Platform Trial completes enrollment for Regimen E evaluating SLS-005 (Trehalose) by Seelos Therapeutics
In February 2023, enrollment was completed for Regimen E, evaluating SLS-005(Trehalose_ from Seelos Therapeutics. SLS-005 is a low molecular weight disaccharide that crosses the blood-brain barrier and is thought to stabilize proteins and activate autophagy through the activation of Transcription Factor EB (TFEB), a key factor in lysosomal and autophagy gene expression. Results expected in fall 2023.
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Regimen F,
Testing ABBV-CLS-7262
ABBV-CLS-7262 developed by Calico Life Sciences LLC in collaboration with AbbVie is the sixth regimen for the HEALEY ALS Platform Trial. Enrollment for Regimen F is underway!
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Regimen G,
In start-up
Healey & AMG Center for ALS announces new drug regimen for testing DNL343 in HEALEY ALS Platform Trial
In December of 2022, the Healey & AMG Center for ALS signed an agreement to work with Denali Therapeutics to test an investigational drug in the HEALEY ALS Platform Trial.
DNL343 is an investigational drug and is being developed by Denali Therapeutics, a publicly owned biotechnology company based in South San Francisco, CA, and is not yet approved for use in any country. Drug candidates that enter the platform trial are chosen by a group of expert ALS scientists and members of the Healey & AMG Center Science Advisory Committee. Enrollment is planned to start in Spring 2023.
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Expanding Access to Experimental Therapies |
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At the Healey Center, we are dedicated to developing effective treatments for ALS and to providing people with ALS access to experimental therapies through our Expanded Access Protocol (EAP) programs. We continue to partner with several stakeholders to create new EAP models for ALS. Here are the most recent updates:
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TechVS ALS
To support EAP opportunities at more centers, we are thrilled to collaborate with Shawn Sarbacker, founder of the TechVsALS campaign. Shawn spoke with us about his experience with EAPs and how to support this program.
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The axeALS Foundation in partnership with the Sean M. Healey & AMG Center for ALS establishes Expanded Access Protocol (EAP) Program at the University of California, Irvine
The axeALS Foundation is partnering with the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital to establish a new EAP program at the University of California, Irvine. Under the direction of, Namita Goyal, MD, professor of neurology, UCI School of Medicine, and director of UCI Health ALS Services, thanks to multiyear support from the axeALS Foundation, UCI will add expanded access as an option for people living with ALS who do not qualify for trials.
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Impact on Scientific Knowledge
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Soft robotic wearable restores arm function for people with ALS
Researchers from Massachusetts General Hospital and the Harvard John. A Paulson School of Engineering and Applied Sciences have created a soft robotic wearable to support arm function in people with ALS. While the device is still in development, initial tests have been promising showing that this wearable can improve functional activity and improve performance of basic activities of daily living. Sabrina Paganoni, physician-scientist at the Healey & AMG Center for ALS, and David Lin, MD, Director of the MGH Neurorecovery Clinic, serve as co-authors.
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From the Lab of Mark W. Albers, MD, PhD
Clinical Trials: NADALS Basket Trial screened first ALS patient
Funding: New Agreement between the Healey & AMG Center and Holy Cross Hospital to support the NADALS Basket Trial over the next two years.
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PRIZES, AWARDS, AND RECOGNITION |
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Merit Cudkowicz, MD, MSC announced as recipient of the 2023 MDA Legacy Award for Achievement in Clinical Research
Congratulations to Chief of Neurology and Director of the Sean M. Healey & AMG Center for ALS Dr. Merit Cudkowicz for being awarded named the recipient of the Muscular Dystrophy Association’s 2023 Legacy Award! The award was presented at the 2023 Muscular Dystrophy Association Clinical & Scientific Conference on Monday, March 20.
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Annual Healey Center International Prize for Innovation in ALS to the CENTAUR Trial Team
In December, Healey & AMG Center awarded the fourth annual Healey Center International Prize for Innovation in ALS to the team that brought innovation, rapid development, and efficacy in Phase 2 clinical trials that led to the FDA approval of Relyvrio as a new treatment for ALS. The award was presented to the team during the virtual 33rd International Symposium on ALS/MND by Merit Cudkowicz, MD, MSc, and Regan Healey. The annual Healey Center International Prize for Innovation in ALS is a global prize celebrating excellence in research for a team of investigators who catalyze exceptional discoveries leading to a transformative advance in therapy development in ALS.
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Annual Drs. Ayeez and Shelena Lalji & Family ALS Endowed Award for Innovative Healing
The Sean M. Healey & AMG Center for ALS was proud to award the second annual Drs. Ayeez and Shelena Lalji & Family ALS Endowed Award for Innovative Healing to two outstanding teams at the 34th International MNDA Symposium in December. The award was given to two outstanding teams; The Answer ALS team led by Dr. Clive N Svendsen of Cedars-Sinai Medical Center of Los Angeles and Dr. Jeffrey Rothstein of Johns Hopkins University, and the team responsible for identifying Stathmin-2 as a therapeutic target for ALS.
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Hop on a Cure Awards first grant to Healey & AMG Center for ALS
Hop on a Cure was founded by musician John Driskell Hopkins from the Grammy-award-winning Zac Brown Band following his diagnosis of ALS in 2021 at the Healey & AMG Center. He launched Hop On A Cure to increase awareness of ALS and raise funds for new treatment research. In January, Hop On A Cure announced the awarding of its first grant — $100,000 to the Healey & AMG Center to fund the development of effective therapies, identify cures, and, ultimately,
prevent the disease.
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Krivickas Memorial Neurology Grand Rounds
On Thursday, January 19th, James Berry MD, MPH presented his research on digitizing ALS using digital data to quantify behavior and function in ALS and hasten drug development at the Lisa S. Krivickas, MD Memorial Lecture. Dr. Berry is working to develop tools to leverage digital data to more accurately quantify behavior and function in ALS, reduce clinical trial burden for people with ALS and hasten drug development. Read the full article here
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CLINICAL CARE AND COMMUNITY EVENTS |
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Ride to Endure
On Wednesday, March 15 Miron Golfman completed the Iditarod Trail Invitational raising awareness and funds in support of the Expanded Access Protocol Program (EAP) at the Healey & AMG Center in honor of his uncle, Bruce Rosenblum. Miron set out from Anchorage, AK on February 26 traveling 1,000 miles to Noam, AK. An incredible accomplishment! To learn more, or support Miron’s fundraising efforts, visit ridetoendure.com
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BID Group Annual Golf Tournament
On Friday, March 3, BID Group hosted their second annual golf tournament to benefit the Healey & AMG Center. Their former employee, Rick Wilson was honored at this year’s event, held at the Santee Cooper Country Club in Santee, SC. Erin Clampffer, RN attended and accepted a $25,000 check on behalf of the Center.
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The Gregg Bonheur ALS Fundraiser
Join friends and family of Gregg Bonheur on April 28th at the Four Oaks Country Club in Dracut for a night in support of ALS. There will be live music by Jamie Cain, a stand-up comedy routine by Justin Hoff, and music by DJ Soul. Net proceeds from ticket sales and the silent and live auctions will benefit the Healey Center for ALS. Purchase tickets to the event here
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ALS Clinic Community Research Day: Saturday, May 6th, 1:00 PM - 4:00 PM ET: This event will be held both in-person and virtually. Sign up here.
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Gelevski D, Addy G, Rohrer M, Cohen C, Roderick A, Winter A, Carey J, Scalia J, Yerton M, Weber H, Doyle M, Parikh N, Kane G, Ellrodt A, Burke K, D'Agostino D, Sinani E, Yu H, Sherman A, Agosti J, Redlich G, Charmley P, Crowe D, Appleby M, Ziegelaar B, Hanus K, Li Z, Babu S, Nicholson K, Luppino S, Berry J, Baecher-Allan C, Paganoni S, Cudkowicz M.
Muscle Nerve. 2022 Dec 19. doi: 10.1002/mus.27775.
Proietti T, O'Neill C, Gerez L, Cole T, Mendelowitz S, Nuckols K, Hohimer C, Lin D, Paganoni S, Walsh C.
Sci Transl Med. 2023 Feb;15(681):eadd1504. doi: 10.1126/scitranslmed.add1504. Epub 2023 Feb 1.
PMID: 36724237
Causal inference in medical records and complementary systems pharmacology for metformin drug repurposing towards dementia. Charpignon, M.-L., B. Vakulenko-Lagun, B. Zheng, C. Magdamo, B. Su, K. Evans, S. Rodriguez, A. Sokolov, S. Boswell, Y.-H. Sheu, M. Somai, L. Middleton, B. T. Hyman, R. A. Betensky, S. N. Finkelstein, R. E. Welsch, I. Tzoulaki, D. Blacker, S. Das and M. W. Albers (2022). Nature Communications 13(1).
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All 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. We are determined to find the cures. Your partnership will make this possible.
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