Dear Friend of the Sean M. Healey & AMG Center for ALS,
I am so proud to provide you with an update on what has been an extraordinary year here at the Sean M. Healey & AMG Center for ALS at Mass General. Every member of our staff has been profoundly touched by the loss of Sean this past spring, but we also know our accomplishments are the result of his spirit and determination to build a team that can help others.
We accomplished our first big goal – to dramatically change how therapies are developed to speed up the process, ensure the best drugs move forward both in clinical trial and in compassionate use programs.
We also increased access so that more individuals with ALS can receive the benefits of these therapies, either through our House Call program or our Early Access Protocol (EAP) program, always working to make sure we meet patients where they are.
Despite the challenges of COVID-19, enrollment in our exciting new HEALEY ALS Platform trial has been four times faster than any previous ALS trial. Three therapies are currently in testing, a fourth will be added shortly and we hope to add two more in the coming months. Incredibly, we will have our first results next fall – not from one, but from all of the first four drugs! Interest in this innovative approach extends to industry as well as other neurological disease groups, including Alzheimer’s and Parkinson’s.
Fifty scientific articles have been published just this past year from Healey Center investigators, including three in the premier New England Journal of Medicine describing positive clinical trial results for the sporadic and familial forms of ALS. At the upcoming December meeting of the International Motor Neuron Disease Symposium, Healey Center-affiliated scientists, including Drs. Sabrina Paganoni, Jeremy Schmahmann and me, are featured in six presentations, with Dr. Rudolph Tanzi, an expert in Alzheimer’s disease, presenting the inaugural Stephen Hawking Memorial Lecture on “Targeting neuroinflammation across neurodegenerative diseases.” We are thrilled to have the opportunity to share our discoveries with the world so others can join the research effort and contribute to the development of effective therapies for patients everywhere.
We have made thrilling progress this year, and so much of it is made possible because of the generosity and commitment of friends like you. We are determined – and inspired by Sean M. Healey and each member of our community – to reach the finish line that we know is getting closer every day. In the year ahead, we seek to support research to “Arrest; Promote and Prevent” – arrest disease progression, promote regeneration of function, and prevent ALS altogether! We hope you will continue to be our partners in reaching these goals. If you are interested in learning more about how you can help to make an impact, please contact Emily Monteiro at 617-643-7376 or emonteiro7@mgh.harvard.edu.
With all best wishes,
Merit Cudkowicz
Merit Cudkowicz, MD, MSc
Director, Sean M. Healey & AMG Center for ALS at Mass General
Chief, Neurology Department & the Julieanne Dorn Professor of Neurology, HMS
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The CENTAUR trial, led by investigators at the Sean M. Healey & AMG Center for ALS at Mass General in collaboration with Amylyx Pharmaceuticals, Inc., tested the effect of a novel oral drug called AMX0035, which is a combination of sodium phenylbutyrate and taurursodiol. We recently reported in the New England Journal of Medicine that administration of AMX0035 resulted in significant slowing of ALS disease progression, offering hope to individuals with ALS and their families. The same drug combination also demonstrated the potential to prolong patient survival as reported in the journal Muscle and Nerve. Read the New York Times article about the CENTAUR Trial.
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Top-line results of the NurOwn ALS Phase 3 clinical trial, sponsored by Brainstorm Cell Therapeutics and supported by so many in our community, were released this week. Read more here. While the trial did not meet its primary or secondary clinical endpoints, the results were promising in a pre-specified subgroup analysis. Robust biomarker data did indicate a biological effect in fighting this disease. Dr. Cudkowicz will present these results at the upcoming virtual 31st International Symposium on ALS/MND.
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HEALEY ALS PLATFORM TRIAL |
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The HEALEY ALS Platform Trial is the first of its kind in ALS. It is a new way of conducting trials that is allowing us to perform trials more rapidly, more efficiently, and with fewer participants receiving placebo. The first participant in our revolutionary new approach to therapy evaluation enrolled on July 14, 2020 at Texas Neurology and the next here at the Healey Center for ALS at Mass General. We are confident this approach marks the beginning of a new era of progress toward the cures as we quickly and efficiently evaluate promising therapies and move them forward to reduce the impact of ALS. We are so grateful for the enthusiasm of individuals with ALS, who have contributed to record-breaking enrollment numbers. As of November 23, 2020, 30 centers across the United States are enrolling participants and more being activated in the coming weeks.
We have 207 participants enrolled to date. ALS trials usually enroll at a rate of approximately 1 participant for every 2 activated sites per month. The HEALEY ALS Platform trial is enrolling up to 8 times as fast! We are enrolling 3-4 participants per site per month! In the words of one of our site investigators, Dr. Daragh Heitzman of Texas Neurology, “I have not seen this level of patient interest since the ‘90s.”
⦁ A fourth investigational treatment regimen from Prilenia Therapeutics will be added soon, and we are negotiating with three additional companies to join the Platform trial in 2021. The speed at which we can add promising therapies is unprecedented, and, combined with the number of companies eager to join us, and the knowledge that it will take months, not years to see results, make us enormously optimistic about what we will accomplish for individuals with ALS.
⦁ Interest in the HEALEY ALS Platform Trial extends far beyond our patients and clinical research teams. More than 360 individuals – scientists from a wide range of fields, patients, advocates and industry representatives from around the world – participated in a HEALEY ALS Platform Trial Roundtable. Hosted in collaboration with ALS ONE as part of the 2020 Virtual ALS ONE Research Symposium, roundtable panelists discussed the progress, statistics, outcomes and regulatory process of the HEALEY Platform Trial. During the roundtable panel, Biohaven Pharmaceuticals provided positive feedback on their experience integrating with the Platform Trial, a welcome endorsement of our careful planning and thoughtful engagement with our partners.
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FIRST ALS EXPANDED ACCESS PROTOCOL (Compassionate Use) PROGRAM |
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At the Healey Center, we are determined to provide more patients with access to more promising therapies while ensuring the best treatments move forward quickly to FDA approval. We are now ready to roll out our successful Expanded Access Protocol (EAP) program across our nationwide network of clinical researchers to reach more patients, identify the best therapies and link these to our HEALEY ALS Platform. Through recent philanthropic gifts, we will launch our pilot HEALEY EAP at three participating centers in early 2021. Our goal is to increase to 10 sites throughout the United States in 2021.
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ATTRACTING TALENTED RESEARCHERS TO ALS |
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Healey Scholars Program: We believe strongly in identifying, supporting and mentoring the next generation of researchers with a passion for bringing treatments to people living with ALS. To accelerate discovery and encourage great ideas, the Healey & AMG Scientific Advisory Council developed a one-of-a-kind program designed to engage clinician-scientists and post-doctoral fellows in therapy development for ALS at their home institution with the option of industry experience in year two. Healey & AMG Center for ALS issued a global call for applications for the 2020 Healey Scholars Program and selected Ana Rita Quadros, PhD, as the first Healey Scholar.
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“As a Portuguese neuroscience enthusiast, the opportunity to be here in Boston to study the mechanisms of ALS is truly a dream. Exciting studies published in 2019 by our lab and others, have shown, for the first time, that production of the protein Stathmin-2 is impaired in patients with ALS. The Healey Scholar grant has given me and my lab the opportunity to test drugs and genetic tools that can restore the levels of this protein in cellular models with known ALS mutations. We are working now on expanding and validating these tools with the aim of finding some that can later be used in humans to stop ALS progression. In addition, we are working on developing strategies to detect this protein with high specify and affinity, so it can be used as a biomarker for ALS. Perhaps even more important, being part of the Healey Center allowed me to connect to patients, clinicians and other scientists dedicated to find ways to ameliorate ALS disease. Being part of such an enthusiastic team has inspired me and given me both tools and novel perspectives that helped push our studies forward. Finding a cure to ALS is a challenging, but fundamental goal, and I am very happy to be part of Healey Center and contribute to such an outstanding effort,” Healey Scholar Ana Rita Quadros, PhD.
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CARE IN THE TIME OF COVID |
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Nowhere has the impact of the COVID-19 pandemic struck more at the heart of what we do than in the MGH ALS Multidisciplinary Clinic at the Sean M. Healey & AMG Center. The clinic is a Center of care, where people with ALS and providers meet to find comfort and to create hope in the face of ALS. Fortunately, we were ready. Having built a telemedicine program for people with ALS over the course of a decade, the clinic transitioned smoothly to virtual care. Building on years of experience, we maintained clinical care through video calls successfully, and we were seen as leaders in the hospital in transitioning to virtual care.
At the height of the COVID-19 pandemic, we maintained contact with patients in a variety of new ways:
- We employed phone calls, virtual visits, and urgent visits in-person, as needed.
- Our nurses reached out to all patients to address concerns.
- We held a multidisciplinary care webinar to provide information to patients about ALS and COVID-19.
- We updated the clinic website with FAQs about COVID-19 and ALS care.
We adjusted to the “new normal” as quickly as possible:
- We collaborated with other departments across Mass General to transform our process for placing feeding tubes to reduce hospitalizations when needed.
- We worked with vendors to create new processes to obtain non-invasive ventilators and cough assist machines, without the need to see patients in person.
- We have created the means to see people with ALS safely in person with visitors whenever needed for communication or care, accommodating the use of respiratory equipment.
Today, we are seeing more and more people in person:
- Our multidisciplinary clinic is open, and we are still allowing patients to choose virtual care, if it works better for them and their families.
- Our ALS House Call program, briefly halted to retool for COVID precautions, is again seeing people with ALS and their families in their homes to extend our care beyond the walls of our clinic.
An article from Business Insider highlighted the ways the team at the Healey & AMG Center for ALS adjusted to COVID restrictions and transitioned elements of research to patients homes.
On Saturday, November 7th 2020, Dr. Merit Cudkowicz, Dr. James Berry, and Jennifer Scalia, NP virtually hosted the 6th Annual Your Day: ALS Care and Research Symposium. The latest updates in ALS research and care were presented by Drs. Merit Cudkowicz, James Berry, Sabrina Paganoni, Suma Babu, Sarah Luppino, NP and Jen Scalia, NP.
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New Impact Award Program: We are launching a new impact award program to inspire solutions to key unsolved ALS problems in 2021 focused on key scientific areas that will have the greatest impact on ALS therapeutic development. The progress we have made in the first two years of the Healey Center has made us bold about what we can accomplish in 2021. Our Science Advisory Council has outlined three priorities:
⦁ ARREST: Stop the progression of disease
⦁ PROMOTE: Promote regeneration and repair
⦁ PREVENT: Prevent ALS altogether
These goals are ambitious and require the partnership of a broad community of scientists, clinicians, patients, foundations, industry, and most importantly, all of you. Thank you for your continued commitment and support.
Together, we know we can solve ALS.
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https://www.massgeneral.org/neurology/als/support/
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