Dear Friend of the Sean M. Healey & AMG Center for ALS,
Thank you for another year full of positive momentum as we move ever closer to our goal of effective therapies for individuals with ALS. We are sending this on the third anniversary of the opening of the Healey & AMG Center, a remarkable moment marking the start of our accelerated efforts, made possible by the generosity of Sean Healey and his family and friends, AMG and so many of you.
As we wait for the FDA decision on marketing approval of AMX035, the two-drug combination therapy that we helped guide through clinical trials, we are busy adding therapies to the HEALEY ALS Platform Trial; rolling out our multicenter expanded access program (EAP) companion program; discovering new targets for therapy development in basic science laboratories; welcoming new faculty and award winners and recognizing our emerging leaders in ALS research and clinical care. We work across all these areas – from basic research through clinical trials – thanks to our global network of collaborators, including scientists, clinicians, nurses, industry partners, patient advocates, and most importantly, you.
Our growing community fuels our determination and inspires us every day. Thank you for continuing to support this work, which we are confident will bring us to the finish line.
With gratitude,
Merit Cudkowicz, MD, MSc
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HEALEY ALS PLATFORM TRIAL UPDATE - ACCELERATING THE PATH TO EFFECTIVE TREATMENTS |
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We successfully reached our enrollment milestone of randomizing 160 participants in each of the first 3 investigational therapies in the HEALEY ALS Platform Trial and anticipate that the 4th investigation therapy will reach this milestone very soon! Currently, we have:
- 52 participating centers activated for enrollment
- 859 participants consented to the Master Protocol
- 633 participants assigned to a Treatment Regimen
- 162 to Treatment Regimen A (zilucoplan, UCB)
- 167 to Treatment Regimen B (Verdipistat, Biohaven Pharmaceutical Holding Company Ltd.)
- 161 to Treatment Regimen C (CNM-Au8, Clene Nanomedicine, Inc.)
- 143 to Treatment Regimen D (Pridopidine, Prilenia Therapeutics)
- 281 participants enrolled in Open Label-Extension where all participants receive the active therapies, of Treatment Regimen A-D.
In addition to completing enrollment in the first treatment regimens, we continue to work towards adding more treatment regimens to this perpetual trial. We received FDA approval for our 5th investigational treatment, Trehalose by Seelos, and will begin enrollment in the coming weeks.
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Our Therapy Evaluation Committee, composed of leading scientists and trialists from around the world, reviews new applications from prospective treatment regimen industry partners looking to join the platform trial. We are currently in active negotiations with new treatment regimen industry partners that will enter the platform trial in 2022. |
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THE MULTICENTER EXPANDED ACCESS PROTOCOL (EAP) COMPANION TO THE HEALEY ALS PLATFORM TRIAL |
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The Multicenter EAP Companion to the HEALEY ALS Platform Trial is a powerful approach to provide access to investigational product for people with ALS who are unable to participate in the HEALEY ALS Platform Trial. Expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.” Expanded Access is also sometimes referred to as “Compassionate Use”.
The Multicenter EAP Companion Program recently launched two multicenter EAPs –one with Verdiperstat and another with CNM-Au8. A third EAP –with Pridopidine is ready for launch at three sites in December 2021.
The EAP Companion Program is a unique opportunity to leverage the HEALEY ALS Platform Trial infrastructure to bring more people with ALS access to potentially beneficial therapies. While each EAP may enroll only a limited number of participants, the Healey & AMG Center in partnership with Duke University is developing enduring training, advocacy and best practice documents and processes which will be shared publicly to educate key partners and catalyze more widespread EAP availability and access for patients.
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The Healey & AMG Center also offers EAP programs to other investigational therapies to the individuals with ALS cared for at Mass General. 147 individuals have enrolled in one of 16 EAP programs at the center.
The Healey & AMG Center EAP Program is funded and made possible by the AMG Charitable Foundation, Tackle ALS, Clearing Corporation Charitable Foundation, Eddie and Jo Allison Smith Family Foundation, I AM ALS and many other community fundraising initiatives and donors. We would like to acknowledge the industry partners for their partnership and drug donations in providing access to their investigational therapies and thank the ALS community including people living with ALS, their families, and advocates for their participation in this important program. Learn more here: Expanding Access to Therapies (massgeneral.org). On July 29th, 2021, Dr. Merit Cudkowicz addressed members of the House Energy & Commerce Subcommittee on Health in a hearing entitled, "The Path Forward: Advancing Treatments and Cures for Neurodegenerative Diseases.” Click here to read Dr. Cudkowicz’s full testimony or you can watch here, starting at the 7:41:00 mark.
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NEW INNOVATION AWARDS FROM THE HEALEY & AMG CENTER FOR ALS |
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Announcing the Winner of the Gupta Family Endowed Prize in ALS Care
At the annual meeting of the North East ALS Consortium (NEALS) meeting in October 2021, we awarded the recipients of our first annual Healey & AMG Center Gupta Family Endowed Prize for Innovation in ALS. This prize is awarded to those who have developed promising new approaches to improving care for people living with ALS. The goal of this prize is to encourage idea sharing, innovation, and forward thinking on scalable ongoing and ground-breaking projects that will directly improve ALS patient care.
The award recipients include Amit Chahwala, BS; Andrew Geronimo, PhD; James Grogan, MD, Yojana Kanade, MS, CCR; Mansoureh Mamarabadi, MD; Zachary Simmons, MD; and Susan Walsh, RN, MSN of Penn State Health Milton S. Hershey Medical Center ALS Clinic. The team was awarded the Gupta Family Endowed Prize for Innovation in ALS Care for their pioneering work using digital health technologies to revolutionize ALS care. To read more about the team and this award, click here.
Special thanks to our wonderful committee who helped make this year’s award possible: Merit Cudkowicz, MD, MSc; Suma Babu, MD, MPH, MBBS; Jen Scalia, NP; Allison Bulat; Phil Green; Ghazaleh Sadri-Vakili, MD, PhD; Shelena C. Lalji, MD, FACOG; and Andrea Lytle Peet.
Two Healey & AMG Innovation Awards will be announced at the Virtual 32nd International Symposium on ALS/MND on December 10th
1) The 3rd Healey International Prize for Innovation in ALS will be presented by Regan Healey and Dr. Merit Cudkowicz to a team of investigators who catalyze exceptional discoveries leading to a transformative advance in therapy development in ALS. The Sean M. Healey International Prize for Innovation in ALS Research recognizes an individual or a team of investigators who catalyze exceptional discoveries leading to a transformative advance in therapy development in ALS. Past winners include:
- 2020: Project MinE, for developing the largest single disease whole genome sequencing project in the world. Award recipients include Professor Ammar Al-Chalabi of Kings College London, Professor Jan H. Veldink of University Medical Center Utrecht, Professor Orla Hardiman of Trinity College Dublin, Professor Phillip Van Damme of University of Leuven, Belgium, Professor Leonard H. van den Berg of University Medical Center Utrecht, Professor Jonathan D. Glass of Emory University, Professor John Landers of University of Massachusetts Medical School and Ms. Evy Reviers of EUpALS, Belgium.
- 2019: First clinical trial of antisense oligonucleotide (ASO) therapy for ALS. Award recipients include Timothy Miller, MD, PhD, of Washington University School of Medicine in St. Louis; Don Cleveland, PhD, of the Ludwig Institute at the University of California at San Diego; Richard Smith, MD, of the Center of Neurological Study in La Jolla, California; Toby Ferguson, MD, PhD, for Biogen and Frank Bennett, PhD, for Ionis Pharmaceuticals.
Special thanks to the members of the Healey & AMG Science Advisory Committee who helped make this year’s award possible!
2)In 2021, the Drs. Ayeez and Shelena Lalji & Family ALS Endowed Award for Innovative Healing was established to recognize an individual or team of investigators for excellence in their transformative scientific discoveries focused on repair of neurological function in ALS. The goal of this award is to identify therapies and modalities to regain lost function of ALS.
We will share the announcement of winners in a future communication as the news is currently embargoed until Dec 10th.
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NEWLY NAMED ENDOWED CHAIRS FOR ALS RESEARCH AT THE HEALEY & AMG CENTER FOR ALS
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Congratulations to James Berry, MD, MPH as the inaugural incumbent of the Averill Healey Endowed Chair in ALS and Brian Wainger, MD, PhD as the inaugural incumbent of the Alexander Healey Endowed Chair in ALS.
As part of the creation of the Healey & AMG Center for ALS, four named endowed chairs were established to be held by Mass General faculty who hold academic rank at Harvard Medical School, thus honoring and supporting these individuals as pivotal leaders in Neurology and the field of ALS.
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Dr. James Berry is Associate Professor of Neurology at Harvard Medical School and Associate Neurologist for Massachusetts General Hospital. Dr. Berry is the Winthrop Family Scholar in ALS Sciences, the Director of the Massachusetts General Hospital Multidisciplinary ALS Clinic and Chief of the Division of ALS and Motor Neuron Diseases. His work is focused on the identification of markers of ALS in blood and spinal fluid, with an emphasis on markers of abnormal inflammation. He is also actively working to develop mobile health and digital phenotyping methods to better understand ALS and hasten the development of new therapies, while reducing the burden of trial participation for people with ALS. |
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Dr. Brian Wainger is Assistant Professor in Neurology and Anesthesiology at Harvard Medical School and an attending physician at Massachusetts General Hospital. His clinical expertise is focused on treating pain in patients with neurological diseases. Dr. Wainger’s research using stem cell modeling led to identification of a mechanism of abnormally increased motor neuron electrical activity in ALS and a clinical trial to correct the hyperexcitability. His lab has also developed a stem cell-based model of the neuromuscular junction in ALS.
“I’m honored and grateful to be part of the Healey & AMG Center and especially to be named the Alexander Healey Endowed Chair in ALS. The connection to Alex and the Healey family will be something that guides and inspires me throughout my career and life,” said Dr. Wainger.
We look forward to celebrating our newest Healey Chairs in 2022.
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7th ANNUAL YOUR DAY: CARE AND RESEARCH SYMPOSIUM
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On Saturday, October 30th, we hosted the 7th Annual ALS Your Day: Care and Research Symposium. The symposium was a great success with over 100 registrants and presentations by our faculty. Among the speakers were Dr. Merit Cudkowicz; Dr. James Berry; Dr. Sabrina Paganoni; Dr. Suma Babu; Jennifer Scalia, NP; and Sarah Luppino, NP. This year’s event focused on innovative clinical programs, exciting clinical trial results and the robust ALS trial landscape at the Healey & AMG Center.
The ALS Parenting at a Challenging Time (ALS PACT) program provides education and guidance to parents and grandparents with ALS to support some of the most vulnerable members of our ALS families. Through the ALS House Call program, we support people with ALS in their homes. Through this program, we also began at-home monitoring of respiratory function and home g-tube replacements. And, of course, our telemedicine program continues to help us support people with ALS at a distance, a program that preceded the COVID era by a decade and has helped provide care for people in their homes since its inception.
The Healey & AMG Center has been central to the design and conduct of critical clinical trials that are reshaping the ALS research landscape, from the Platform Trial, to the CENTAUR trial of AMX0035, and to the VALOR trial of tofersen for SOD1 ALS. And with scores of trials beginning enrollment, our chances of finding effective therapies are greater than ever before. #HealeyHope!
The event also included a Q&A session where attendees asked questions about all things ALS. If you missed the event, don’t worry! The full recording of ALS Your Day is available to watch here!
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UPCOMING TRIAL HOLDS PROMISE FOR ALS AND ALZHEIMER'S DISEASE |
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Healey & AMG Center faculty member, Mark Albers, MD, PhD, is leading the launch of a basket trial of baricitinib, an FDA-approved therapy currently used to treat inflammation in rheumatoid arthritis and Emergency Authorization Use for COVID. While the goal of this trial is to determine the impact of this drug on the death of neurons in both ALS and Alzheimer’s disease, the design and development of this new basket approach to testing – the first ever in neurology and certainly among neurodegenerative diseases – is also providing critically important information on how to bring research in these two disease areas together, and about the variability of both Alzheimer’s and ALS, while helping to define the subgroup of patients who are most responsive to this therapy.
“The work we are doing in advance of the trial represents the foundational building blocks that will help us learn not only whether or not this drug will reduce inflammation and neuron death, but also provide us with valuable information about why some people respond to this therapy and others don’t,” Dr. Albers says.
Two recent studies published this summer support the mechanics of this approach:
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Nature Communications: Machine learning identifies candidates for drug repurposing in Alzheimer’s disease
Dr. Albers in collaboration with clinical investigators James Berry, MD, and Katharine Nicholson, MD are working with collaborators at the Alzheimer’s Clinical Trials Research Unit at Mass General. The basket trial, now scheduled to launch in early 2022, will provide 10 participants with Alzheimer’s disease and 10 participants with ALS, with one daily baricitinib pill, for 24 weeks. At the end of the trial, participants will be measured for a reduction in inflammation and neuronal death markers.
Although the study covers a modest number of participants, it lays the groundwork for exciting new approaches to identifying both effective treatments, and more clearly defining subsets of patients who will respond to specific therapies.
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NEXT GENERATION OF CLINICAL AND SCIENCE LEADERS IN ALS |
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Welcome Dr. Doreen Ho
We are excited to welcome Dr. Doreen Ho as our newest faculty to the Sean M. Healey & AMG Center for ALS. Dr. Ho completed her medical school and residency training at Tufts Medical Center and her neuromuscular fellowship at Mass General Brigham, where she was colleagues with Dr. James Berry, now chief of our ALS and Motor Neuron Diseases Division. To learn more about Dr. Ho, her experience and her hope for the future of ALS, click here.
As we continue to grow our team and work collectively towards finding a cure for ALS, we are excited about our next generation of ALS investigators:
- Neurodegenerative Clinical Research Fellow
- Stephen Johnson, MD
- Byrne Family and Judith & Pape Adams Fellow
- Yuyu Song, PhD
- Healey & AMG Center Postdoctoral Fellow
- Dylan Galloway, PhD
- Cullen Young Investigator
- Aaron Held, PhD
- Matthew Nolan, PhD
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2021 ALS Scholars
- Juliet Goldsmith, PhD of University of Pennsylvania
- Matthew Anthony White, PhD of Kings College London
- Brian Jude Joseph, PhD of Harvard University
- Su Min Lim, PhD of Massachusetts General Hospital, Harvard Medical School
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All of 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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