Greetings from the ALS Multidisciplinary Clinic in the Sean M. Healey & AMG Center for ALS at Mass General,
Our launch of the HEALEY ALS Platform Trial signaled a new era in the quest to cure ALS. This trial marshals all our collaborators and last month’s [February 2022] publication of an article in Annals of Neurology shares our experience for other clinical researchers in ALS and other neurological fields to follow. We are enormously excited about both the concurrent evaluation of potential therapies, and the trial’s perpetual structure. We will keep testing until we find the cures!
Even as we look forward to results from the first investigational products in the platform trial later this year, we continue to push forward a wide range of ALS research that will bring us the next wave of investigational products. In this newsletter, you will read about our exciting efforts to REPAIR, PREVENT and TREAT ALS.
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Brian Wainger, MD, PhD, inaugural incumbent of the Alexander Healey Endowed Chair in ALS, is accelerating the repair of muscle function. Dr. Wainger and his team are working with neurosurgeons to explore the effectiveness of surgically inserting reprogrammed stem cells to maintain muscle health, especially for breathing strength.
- Our PREVENT ALS initiative 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.
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We are very proud and excited that AMX0035, a treatment whose pivotal clinical trial the Healey & AMG Center investigators designed and led, is undergoing FDA review for full market approval. A decision is expected June 29th, 2022.
- And, we presented awards to two research teams for their efforts to advance predictive analytics in ALS and efforts to repair neurologic function in ALS. We are thrilled at the growing depth and breadth of investigations that are moving us closer to the cures.
Your participation in all these efforts keeps us going. Thank you for your continued faith and support for our work. It is a privilege to care for you and your families.
With gratitude,
Merit Cudkowicz, MD, MSc and the ALS Clinic Team
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HEALEY ALS PLATFORM TRIAL: Accelerating The Path to New ALS Therapies
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The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, in partnership with the Northeast ALS (NEALS) Consortium, designed the first Platform Trial for ALS. The design of the trial leveraged input from multiple stakeholders including ALS scientists and clinicians, the patient community, industry members and regulators. The HEALEY ALS Platform Trial, launched in July 2020, is a multicenter, double blind, placebo controlled, perpetual, adaptive platform trial testing multiple regimens. This approach cuts the time to finding effective treatments in half, decreases costs by one third and increases the likelihood of a participant receiving experimental treatment by two thirds. Every participant who completes the randomized placebo-controlled portion of the study has the option to receive the active study medication in an open label extension (OLE).
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Additionally, you can learn more about the Platform Trial during our weekly webinar. We have connected with ~14,000 patients from roughly 40 countries in an effort to keep the community informed on a weekly basis about all things Platform Trial. |
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EXPANDED ACESS PROTOCOL PROGRAM: Offering Access to Investigational Products For People With ALS Not Eligible For Clinical Trials
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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 three multicenter EAPs –one with Verdiperstat, another with CNM-Au8, and a third with Pridopidine.
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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. Additional information can be found here.
The Healey & AMG Center also offers EAP programs to other investigational therapies to the individuals with ALS cared for at Mass General. 180 individuals have enrolled in one of 16 EAP programs at the center.
The Healey & AMG Center EAP Programs are 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 also 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.
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AN INTRODUCTION TO THE HOUSE CALL PROGRAM |
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Human interaction and personal care are the centerpiece of ALS care. The MGH ALS House Call Program began in November 2017. It is a deeply collaborative effort between the ALS Multidisciplinary Clinic at Massachusetts General Hospital and Compassionate Care ALS (CCALS). We carefully coordinate care and design home visit schedules to integrate seamlessly with the care provided at the ALS Multidisciplinary Clinic at Mass General.
From November 2017 through July 2021, Mass General House Call staff made 1,105 visits partnering with Compassionate Care ALS (CCALS) on 601 (55%). Sixty one percent of patients being followed by the House Call Program are from underserved populations, as indicated medical insurance coverage (e.g., Medicaid).
Through all the challenges of the COVID-19 pandemic, the partnership between our team at Mass General and the Compassionate Care ALS (CCALS) team on the ALS House Call project has been tested and flourished. Using the House Call model, supplemented by video and remote technology, we continued to support our ALS House Call patients, reducing their isolation during the pandemic.
The MGH ALS House call program team includes Nurse Practitioner Debra Skoniecki, Registered Nurse Kristen Kingsley and a Patient Service Coordinator Eunice Terrero.
Our nursing team bring expert care to the home, reduce medical isolation and improve medical connectiveness. Home visits may last 60 minutes or longer. During the visit, our nurses will follow up on the patient’s plan of care, check vital signs and vital capacity and complete an assessment. They will also assess our patient’s homes for safety and equipment needs. We have found the home environment to be an excellent place to continue one on one teaching, provide information on research and discuss patient’s goals of care. Over the last year, we have piloted a home spirometry program and now offer home gastrostomy tube exchanges.
The house call program offers our nursing staff the unique opportunity to meet patients, family, pets and caregivers in their homes; During a home visit, we can see our patient’s life on the walls, learn of their history, legacy, hopes and fears and see how they are living. What an honor!
The program is entirely supported by philanthropy.
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PRIZES FOR INNOVATION IN ALS AWARDED AT VIRTUAL 2021 ALS/MND MEETING |
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During the 32nd International Symposium on ALS/MND in December, the Sean M. Healey & AMG Center awarded: |
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Third annual Healey Center International Prize for Innovation in ALS to the team responsible for the conception, construction, dissemination, and maintenance of the PRO-ACT database that has enabled the new field of ALS predictive analytics. The Pro-ACT team includes Mr. Avichai Kremer of The ALS Association, Dr. Melanie Leitner of Accelerating NeuroVentures, LLC, Dr. Neta Zach of Takeda Pharmaceuticals, and Mr. Alexander Sherman, MS of the Neurological Clinical Research Institute, Department of Neurology, Massachusetts General Hospital. The award was virtually presented to the team during the 32nd International Symposium on ALS/MND by Merit Cudkowicz, MD, MSc, director of the Healey & AMG Center at Mass General and Regan Healey. To read more about this award, click here and to watch the presentation, click here!
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First Drs. Ayeez and Shelena Lalji & Family ALS Endowed Award for Innovative Healing to Professor Michael Coleman, University of Cambridge and Professor Marc Freeman, Oregon Health & Science University for their excellence in transformative scientific discoveries focused on repair of neurological function in ALS. The award was virtually presented to the team by Merit Cudkowicz, MD, MSc Director of the Healey & AMG Center at Mass General and Drs. Ayeez and Shelena Lalji. This award 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. To read more about this award, click here and to watch the presentation, click here!
Special thanks to the members of the Healey & AMG Prize Review Committees who helped select the awardees!
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BRIAN WAINGER, MD, PhD: Using Stem Cells to Restore Motor Function
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Brian Wainger, MD, PhD, inaugural incumbent of the Alexander Healey Endowed Chair in ALS, is developing a novel therapy to help improve neurosurgical outcomes after nerve or spinal cord injury in collaboration with Dr. Justin Brown in the Department of Neurosurgery. Dr. Brown’s team performs nerve transfer surgeries - surgical treatment to replace a damaged nerve with a healthy nerve from a nearby muscle, to restore strength. Unfortunately, in many cases, the muscle has already undergone irreversible damage and nerve transfers do not work. For example, when motor neurons fail, the muscle will no longer flex, and the muscle undergoes unrecoverable wasting from disuse. This degeneration of muscle is the limiting factor to successful restorative nerve surgery.
Dr. Wainger, Dr. Brown and their research teams are pioneering a novel approach to nerve transplants using motor neurons derived in the laboratory from non-embryonic, human stem cells. Such motor neurons have already shown promise in animal experiments where they were injected near the muscle, grew, made connections to the muscle, and maintained muscle health. The muscles did not undergo irreversible damage, setting the stage for a successful nerve transplant.
While initial research focused on nerve injury, a similar strategy can be used to maintain breathing strength in ALS: coupling the injected neurons with a cuff electrode around the nerve that controls breathing has the potential to maintain functional respiration without a ventilator. Because muscle degeneration is implicated in ALS, there may be additional benefits of the motor neurons in ALS.
To confirm these initial findings and provide key safety and efficacy data, rodents and non-human primates will be used to develop this powerful technology. Support for these proof-of-concept experiments will allow Drs. Wainger, Brown and their teams to move more quickly to bringing this technology to humans.
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BYRNE FAMILY AND JUDITH AND PAPE ADAMS FELLOW |
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Byrne Family and Judith & Pape Adams Fellow Yuyu Song, MD, PhD, has spent the last year researching what determines whether a patient will develop Amyotrophic Lateral Sclerosis (ALS), Frontotemporal Dementia (FTD) or ALS/FTD. “Receiving this funding was so important because ALS patients don’t have time to wait. Funding makes it possible to find answers so we can develop new therapies and cures quickly,” Song says. |
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In this video, she discusses what led her to this research topic, the key to this project, what she has discovered and what it may mean for patients.
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At the Sean M. Healey & AMG Center for ALS at Mass General, we are proud to be building the tools that hasten our march toward a cure for ALS. Our determination to uncover the most effective ways to fight ALS underlies our next audacious goal — preventing ALS before it can begin. An initiative we refer to as PREVENT ALS.
The PREVENT ALS initiative builds on the success of the Dominantly Inherited ALS (DIALS) study of people who harbor an ALS-causative gene mutation but do not yet have symptoms to confirm discoveries and foster innovative research. Identifying the earliest symptoms and molecular changes in individuals with ALS will tell us about the cause of ALS, hasten diagnosis and allow us to start treatments before more motor neurons are lost. Participants are evaluated every 6 to 12 months, and provide plasma, serum, DNA, RNA, white blood cells, and in some cases, spinal fluid for analysis. Many DIALS participants also enroll in other biomarker evaluating imaging, cognitive tests, and even the gut microbiome (a study of the bacteria in the GI tract).
The PREVENT ALS initiative engages geneticists and gene counsellors, lab researchers, clinical trialists and families at risk for ALS who work together to identify biological changes preceding ALS and novel therapeutic targets. And our efforts are gaining steam. Currently, we have 169 participants enrolled in the DIALS study, approximately 80 of whom were enrolled in 2021.
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. With additional funding, we will expand, with a goal of enrolling at least 750 participants. Please consider supporting our PREVENT ALS initiative and double your impact through making a gift today.
We envision a world in which ALS is both treatable and preventable. We are confident PREVENT ALS will take us there.
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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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