Greetings from the ALS Multidisciplinary Clinic in the Sean M. Healey & AMG Center for ALS at Mass General,
What a year we have had!
- A new FDA-approved drug with another under review;
- Shared first results from our game-changing HEALEY ALS Platform Trial;
- Launched the TechVsALS program to support more centers to provide expanded access programs for their patients,
- Received the first and only grant from the bipartisan ACT for ALS (Accelerating Access to Critical Therapies for ALS) bill to add a compound to the Healey & AMG Center’s expanded access program; and
- Advanced care programs for individuals with ALS and their families.
While these successes were all part of the vision Sean and I had for the Healey & AMG Center for ALS, it is thrilling to see them becoming a reality. All this progress is thanks to your faith in us and generous support for our patient-centered goals of care and cure. This year’s achievements are the culmination of hard work and determination, but we are not at the finish line yet. I am confident the HEALEY ALS platform trial will deliver an effective therapy, so we need to keep testing and adapting. I am overjoyed that the federal government is supporting a new multisite expanded access program, but we need to continue to expand this program so that individuals with ALS have access to the best possible therapies wherever they live. I am proud of the basic science research advances made by our Healey& AMG Center awardees and fellows, and now we need to move these discoveries rapidly from the laboratory to people living with ALS.
Your partnership is essential to our success. We can’t stop, we won’t stop, until we find the cures.
With gratitude,
Merit Cudkowicz, MD, MSc and the ALS Clinic Team
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MERIT CUDKOWICZ CELEBRATES 10 YEARS AS CHIEF OF NEUROLOGY |
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In October, Merit Cudkowicz, MD, MSc, Director of the Sean M. Healey & AMG Center for ALS, and Julieanne Dorn Professor of Neurology, Harvard Medical School celebrated 10 years as the Chair of Neurology at Massachusetts General Hospital. The department is home to over 200 faculty and has raised an incredible $467M in philanthropy from 2012 -2022. With the help of our community, our faculty has led to several discoveries that have contributed not only to innovation in ALS research but also in other fields like Parkinson’s and Alzheimer’s Disease research, as well as in Stroke care and Pediatric Neurology. Under Dr. Cudkowicz’s leadership, the department succeeded in making monumental strides for Neurology care and research and is a leader in the care and therapeutics for people with neurological disorders.
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FDA APPROVES THIRD ALS DRUG: AMX0035 (RELYVRIO) BY AMYLYX |
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In September, the FDA approved RELYVRIO (AMX0035) from Amylyx as a new treatment for people with ALS. This is a monumental step forward for ALS and we are very proud to have been part of this drug development program since the beginning. In partnership with Amylyx Pharmaceuticals, our Healey & AMG Center team designed and led the pivotal clinical trial, whose positive results led to this historic approval. We are grateful to the clinical sites of the Northeast ALS (NEALS) Trial Consortium and to all the trial participants and their families for making this study possible. |
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THE SEAN M. HEALEY & AMG CENTER FOR ALS ANNOUNCED TOPLINE RESULTS IN THE HEALEY ALS PLATFORM TRIAL |
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The results for HEALEY ALS Platform Trial Regimens A, B and C were released in September and October. The HEALEY ALS Platform Trial was designed to accelerate the development of breakthrough treatments for persons with ALS by testing multiple drugs using a shared infrastructure. Zilucoplan developed by UCB (regimen A), Verdiperstat, developed by Biohaven Pharmaceuticals (regimen B) and CMN-Au8, developed by Clene Nanomedicine (regimen C) were the first three investigational treatment regimens tested using this innovative paradigm.
Results from all three regimens provided clear answers and direction for future studies. Regimens A and B were not effective; additional biomarker and subgroup analyses are ongoing. A positive survival signal was found in Regimen C. Therefore, the open-label extension and expanded access programs will continue for Regimen C and plans for a phase 3 trial are underway. The efficient executions of these studies have instilled motivation and promise for future regimens; Regimen D (pridopidine, developed by Prilenia) is ongoing, and Regimen E (trehalose, developed by Seelos) is now enrolling participants. Two more therapies will start in 2023. We extend our sincere thanks to the patient community; because of their efforts, our HEALEY ALS platform trial team will take the data that was gathered and apply it to future studies and continue to make strides in the fight against ALS.
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EXPANDED ACCESS PROTOCOLS: OFFERING OPTIONS TO MORE INDIVIDUALS WITH ALS |
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The Expanded Access Protocols (EAP) Program is an FDA-regulated pathway that allows people with a serious and life-threatening disease to access an investigational drug that is not yet approved by the FDA. It enables individuals, who might not otherwise have the opportunity, to contribute as research participants while receiving access to potential investigational treatments. We are excited to share:
- We continue to grow the EAP program across our nationwide network of clinical researchers to reach more individuals with ALS, identify the best therapies and link these to our HEALEY ALS Platform Trial.
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With philanthropic support from Kulicke & Soffa through the TechVsALS campaign established by Shawn Sarbacker, the Healey & AMG Center and Weinberg ALS Center at Jefferson Hospital announced a partnership to establish and support adding Jefferson Hospital as a participating center to provide patients in the Philadelphia area with the access to ALS investigational treatments. Read the full announcement here.
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Healey & AMG Center investigators Drs. Suma Babu, James Berry, and Sabrina Paganoni were awarded a grant from the National Institute of Neurological Disorders and Stroke (NINDS) to conduct an intermediate-size EAP)in ALS. The grant is supported by the ACT for ALS (Accelerating Access to Critical Therapies for ALS Act). This EAP will evaluate the benefits of the investigational drug trehalose in individuals with ALS. Trehalose (SLS-005, Seelos Therapeutics) is observed to have protective effects in ALS mouse models by activating autophagy, a mechanism that is impaired in ALS. Trehalose is also the fifth regimen in the HEALEY ALS Platform Trial. Read the full announcement here.
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2022 GUPTA FAMILY ENDOWED PRIZE FOR INNOVATION IN ALS CARE |
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The annual 2022 Gupta Family Endowed Prize for Innovation in ALS Care was awarded to the team from ALSUntangled for their work in facilitating more informed decisions on treatments for ALS. The prize was presented to the team during the 21st Annual Northeast ALS Consortium (NEALS) Meeting in Clearwater Beach, FL by Dr. Merit Cudkowicz, Director of the Healey & AMG Center on behalf of the Gupta family.
ALSUntangled was created by Richard Bedlack, M.D, PhD, Professor of Neurology at Duke University and Director of the Duke ALS clinic. The program has three parts: inputs, reviews, and outputs. People with ALS are now able to send their ideas for reviews via email or in-person clinic visit with an ALSUntangled team members. The team prioritizes ideas by a combination of votes from the community and also a multiplier reflecting the amount of useful disclosable information the team can find on a product. ALSUntangled has a team of 130 clinicians and scientists from across 11 countries working on these reviews. Read the full announcement here.
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2022 PRIZES RECOGNIZING INNOVATION IN ALS RESEARCH |
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In addition to the Gupta Family Endowed Prize, The Healey & AMG Center will announce two additional prizes this year for innovation in ALS and transformative scientific discoveries. The 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, and the Drs. Ayeez and Shelena Lalji & Family ALS Endowed Award for Innovative Healing recognizes an individual or team of investigators for excellence in their transformative scientific discoveries focused on the repair of neurological function in ALS. These prizes will be awarded at the 33rd International Symposium on ALS/MND beginning on December 6th.
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Welcome to Dr. Mark Garret, 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 and fellows at Washington University, working in both the Neuromuscular and Physical Medicine and Rehabilitation sections. Dr. Garret has presented academic research on advances in treatments for familial ALS and pre-symptomatic therapies for neurodegenerative diseases. Read his interview with Erika McCarthy here.
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We are thrilled to announce the success of our community events that raised support for ALS care and research!
The Pete Frates 5k on November 6th hosted runners from across New England and raised nearly $70k for ALS care. This year, the money raised will help to fund the addition of a psychologist or psychiatric mental health nurse practitioner to the ALS multidisciplinary care team at the Healey Center.
Additionally, The first annual Captain's Ball in memory of Pete Frates helped to raise a gift of $125,000 for the Healey & AMG Center. Mass General and the Healey & AMG Center are grateful for the Bourque Family Foundation’s support that will help further our progress in pursuit of a cure for ALS.
For more information on upcoming and past events, see our Community Events page here.
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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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