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Greetings from the ALS Multidisciplinary Clinic in the Sean M. Healey & AMG Center for ALS at Mass General,
We are so grateful to you for your continued advocacy and support of research and care for individuals with ALS. We are excited to share that a new therapeutic we helped test in people with ALS received conditional approval in Canada, with a decision by the FDA here in the US, just weeks away.
In addition, results from the first four clinical investigations in our game-changing HEALEY ALS Platform Trial will be ready in the coming months, even as we added a fifth regimen and prepare several more.
We are making significant strides in our basic research labs, moving ever closer to understanding the genetic and molecular changes that occur in ALS. At the same time, we continue to respond to the multifaced needs of our patients and families with new programs to support emotional health.
The launch of the Healey Center allowed us to lay a foundation that embraces a global community, all working together to support the brightest minds and the best ideas that will bring us to the cures.
Thank you for your partnership. Your determination inspires us every day.
Warmly,
Merit Cudkowicz, MD, MSc and the ALS Clinic Team
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AMX0035 APPROVED IN CANADA, FDA DECISION PENDING | |
On June 13 ALBRIOZA (also known as AMX0035) from Amylyx Pharmaceuticals, Inc., received Approval with Conditions from Health Canada. This is a wonderful step forward based on a community effort to test and advocate for this new therapeutic.
We are proud to have been part of this ALS drug development program since the beginning. In partnership with Amylyx Pharmaceuticals, Inc, our MGH team designed and led the CENTAUR trial, whose positive results led to this historic approval. We are grateful to the clinical sites of the NEALS Consortium and to all trial participants and their families for making this study possible.
In the US, a second virtual meeting of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee is scheduled for September 7, 2022, with the FDA decision about Albrioza approval expected by September 29, 2022.
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The Healey & AMG Center, in partnership with the Northeast ALS Consortium (NEALS), designed and launched the first Platform Trial for ALS in 2020, with the goal of accelerating the development of new treatments for ALS.
Since the trial launch:
- Over 800 people with ALS have participated in this study.
- All participants enrolled in the first four clinical investigations (Regimens A-D with investigational products: zilucoplan, verdiperstat, CNM-Au8 pridopidine) completed their last visit for the 24-week randomized controlled portion of the trial.
- Trial results are expected this fall.
- The fifth investigational product (trehalose – Regimen E), began recruiting participants in February 2022.
- Two additional industry regimen partners (Revalesio and Calico Life Sciences) are currently working with us on trial design.
- We continue to review new investigational product applications on a rolling basis for possible inclusion in the platform trial and are proud to report that two additional potential therapies have been recommended by the committed.
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Cullen Education and Research Fund (CERF) Young Investigators
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We are excited to announce that the Cullen Education and Research Fund (CERF) has awarded the third annual young investigator awards at the Healey & AMG Center. These awards are used to catalyze significant progress in basic science investigations, moving us closer to discovering therapies that can be tested in patients. The Fund supports and encourages research into a cure for ALS.
Aaron Held, PhD, joined the laboratory of Dr. Brian Wainger at MGH in December 2018, and his projects focus on the fundamental cellular processes leading to neurodegeneration in ALS. He is developing and leveraging new stem cell technologies and computational approaches to identify molecular changes that are shared between ALS subtypes and contribute to the development and progression of ALS. Dr. Held’s new stem cell and analytical tools allow Dr. Wainger’s team to quickly identify and evaluate interventions that could positively impact motor neurons damaged by ALS.
Sandeep Aryal, PhD, completed his PhD at the University of Delaware in 2021 under the mentorship of Dr. Salil A. Lachke. His graduate work involves the discovery of RNA-binding proteins' role in congenital developmental defects of the lens and the eye. Dr. Aryal joined the laboratory of Dr. Clotilde Lagier-Tourenne at the Healey & AMG Center in May 2021 to expand his expertise on disease mechanisms of ALS. In collaboration with Dr. Nolan, a former CERF Young Investigator, Dr. Aryal uses state-of-art CRISPR-based genetic screens to identify new therapeutic targets for ALS. He focuses on a gene called stathmin-2 that is profoundly altered in both sporadic and familial ALS as well as in other neurodegenerative conditions including frontotemporal dementia and Alzheimer’s disease.
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Byrne Family and Jean & Pape Adams Fellowship
Suma Babu, MD, MPH, MBBS is the recipient of the 2022-2023 Byrne Family Endowed Fellowship and Judith & Jean Pape Adams Charitable Foundation Early Career Research Award.
Dr. Babu is an Assistant Professor of Neurology at Harvard Medical School and provides clinical care for people with ALS, other motor neuron diseases, and neuromuscular disorders at the multidisciplinary ALS clinic in the Healey Center for ALS and in the Neuromuscular division. As the 2022-2023 Byrne Family and Pape Adams Early Career Researcher in ALS she will pursue a single-site, cross-sectional, magnetic resonance imaging (MRI) study of neuroinflammation in people with ALS. This study builds on the premise that in early ALS, neuroinflammation remains focal around motor neurons located in the gray matter and spreads to other brain regions later in the illness. To design informative clinical trials testing neuroinflammation-targeted drugs in ALS, it is important to accurately characterize neuroinflammation in the early and focal stages, when it may be more amenable to intervention.
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ALS SCHOLARS IN THERAPEUTICS | |
The Healey & AMG Center, ALS Finding a Cure (ALSFAC), and FightMND, selected three recipients for the second global, two-year ALS Scholars in Therapeutics program. The program received an outstanding pool of applicants from several countries. This year’s recipients are: Dylan Galloway, PhD, of the Washington University School of Medicine, Jacob Mann, PhD, of Northwestern University Feinberg School of Medicine, and Roberta Piovesana, PhD, of the University of Montreal. | |
Dr. Galloway is recognized for his research in identifying microRNAs as novel regulators of differential motor neuron susceptibility. He earned his PhD in Neuroscience at the Memorial University of Newfoundland where he researched how microRNAs modify neuroinflammation and brain repair in multiple sclerosis. | |
Dr. Mann is recognized for his research in investigating the efficacy of microtubule-targeting agents and uORF ASOs to target NEK1 loss-of-function in ALS. He earned his PhD in Neurobiology at the University of Pittsburgh under the mentorship of Dr. Christopher Donnelly, where he studied the role of RNA in the regulation of aberrant phase transitions/aggregation of ALS-linked proteins like TDP-43 and FUS. | |
Dr. Piovesana is recognized for her work on Neuromuscular Junction and the identification of ALS biomarkers. She earned her Master’s in Medical Biotechnology and later PhD in cellular and developmental biology at the University of Rome where she established adipose stem cell models that led to the production of Schwann cells and their role in axonal support and plasticity. | |
Read the full article here
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2022 PRIZES RECOGNIZING INNOVATION IN ALS | |
In addition to our exciting advances in basic research, philanthropic support is critical to our ability to accelerate our efforts to encourage innovation in research for therapies, promote repair of function, and improve the care of those living with ALS. Thanks to the generosity of our patients, families, and friends, the Healey Center now awards three annual prizes to teams and investigators dedicated to this research, including the Sean M. Healey International Prize for Innovation in ALS Research (announced at the Virtual 33rd International Symposium on ALS/MND in December 2022); the Gupta Family Endowed Prize for Innovation in ALS Care (announced at the 21st Annual NEALS Meeting November 1 - 3, 2022 in Clearwater, Florida); and the Drs. Ayeez and Shelena Lalji & Family ALS Endowed Award for Innovative Healing (announced at the Virtual 33rd International Symposium on ALS/MND in December 2022).
We look forward to sharing the recipients of these awards in our next issue.
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NEW FACULTY SPOTLIGHT- DR. MARK GARRET | |
We are excited to welcome Dr. Mark Garret as our newest faculty at the Healey & AMG Center for ALS. Dr. Garret received his medical degree from the University of Pennsylvania followed by a neurology residency and neuromuscular fellowship at Washington University in St. Louis. During his fellowship, Dr. Garret participated in multiple clinical trials of targeted genetic therapies for inherited forms of ALS. Dr. Garret has also served as a lecturer to residents at Washington University, working in both the Neuromuscular and Physical Medicine and Rehabilitation sections. Dr. Garret has presented research on several impactful studies such as insights into clinical trials for inherited ALS, and acute nutritional axonal neuropathy.
In joining the Healey & AMG Center, he will be working with the Dominantly Inherited ALS Network (DIALS), an ongoing study of asymptomatic carriers of familial ALS genes to improve our understanding of the earliest changes in ALS. Dr.Garret is committed to joining the team of researchers working together to understand the biology of ALS and the early cascade of pathology that leads to the onset of symptoms, first in individuals with the ALS genes, and then use this information to understand sporadic (non-genetic) forms of ALS. Using the biology of genetic forms of ALS as a guide, we hope to unlock the first steps in ALS onset and unravel the heterogeneity of this disease.
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NUTRITION AND MENTAL HEALTH CLINICAL INITIATIVES | |
We are thrilled to announce that registration is now live for the 2022 Pete Frates 5K! Visit the website for more information and to register today.
All proceeds from this year’s race will fund the addition of a psychologist or psychiatric mental health nurse practitioner to the ALS multidisciplinary care team at the Healey Center.
The primary symptom of ALS is the progressive loss of strength, yet the psychological impact of the disease is also tremendous. Access to expert psychological support to navigate these changes and offer coping strategies will provide a positive impact on the quality of life for people with ALS.
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PREVENT ALS Matching Gift Opportunity
PREVENT ALS is gaining momentum as more and more people and families at risk for developing ALS join us to search for the triggers that initiate the disease, so that we can stop it before it starts. We received a generous matching gift opportunity for $1 million from the Eddie and Jo Allison Smith Family Foundation to inspire raising an additional $1 million for the expansion of the PREVENT ALS initiative. Please consider supporting our PREVENT ALS initiative and double your impact through making a gift today.
Tackle ALS
Tim Green - fundraising champion and advocate for ALS research at the Healey Center - was recently featured in an article on the sports news website The Athletic. The interview encompasses his numerous successful careers, the importance of family, and how his ALS diagnosis continues to motivate him. Tim launched the Tackle ALS fundraising campaign in 2018 and has raised over $5.5M in support of the HEALEY ALS Platform Trial.
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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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