Dear Friend of the Sean M. Healey & AMG Center for ALS,
Thank you for your kind words and support during this challenging time. Our efforts are focused on continuing to provide the best possible care and access to research for our patients and their families, while keeping everyone safe from the COVID-19 virus.
We want to take a moment to share an update on our progress across the Healey Center. While we contribute to the hospital’s efforts to meet the needs of the many individuals affected by this pandemic, our determination to find the cures for ALS has not wavered. Our ALS research efforts continue both in the laboratory and in providing access to clinical trials for our patients. We quickly flexed our approach to both care and research through virtual platforms. Several of our trials are now completely virtual. For the first time, we are enrolling our patients in trials of new experimental treatments entirely over zoom and other virtual platforms. This also includes the ability to ship medicine directly to our patients and administer intravenous new therapies in the home.
We are very proud to share the announcement of a positive study of AMX0035 in ALS, a study led by Healey Center Investigator Dr. Sabrina Paganoni and the Healey Center Clinical Coordination Center.
We are truly at a turning point in our progress toward the cures. I hope the information in this newsletter encourages and inspires you. The science is guiding us; the commitment of all of you will bring us to the finish line.
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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POSITIVE CLINICAL TRIAL RESULT
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Peer-reviewed results of a trial led by the Healey Center’s Dr. Sabrina Paganoni, will be published soon. The CENTAUR trial, highlighted in a
Wall Street Journal
article, tested a drug that showed promise in slowing disease progression in individuals with ALS.
The CENTAUR trial, led by Amylyx Pharmaceuticals began as an undergraduate project in 2013 by two students from Brown University: Josh Cohen, who studied biomedical engineering, and Justin Klee, who studied neuroscience. After college and post-graduate research, the pair launched Amylyx Pharmaceuticals to study novel approaches to neurodegenerative diseases and turned to Healey Center Director Dr. Cudkowicz and Mass General’s expertise in ALS research and patient care for advice.
“These two scientists took a novel approach to the problem of nerve cell death,” Dr. Cudkowicz said, “and with guidance from our team, and under the direction of Principal Investigator Dr. Sabrina Paganoni, moved the clinical trial forward quickly and carefully.”
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HEALEY ALS PLATFORM TRIAL
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Enrollment in our pivotal HEALEY ALS Platform Trial will begin soon! As a result of the COVID-19 pandemic, we made important changes to strengthen the trial long-term and ensure people with ALS can safely be part of this initiative during this challenging time. These include virtual visits and home safety and outcome measures.
- On January 16, 2020, we received “May Proceed” notice from the FDA notice for first three drugs
- On March 11, 2020 we received central IRB approval for the Platform Trial and the first three drugs
- On March 12th-13th we hosted a virtual Investigator Kick-off Meeting with approximately 250 attendees, including trial organizers, staff from the 54 participating sites, representatives from the HEALEY ALS Platform Trial patient advisory committee, our three industry partners, and from TackleALS, The ALS Association, the Muscular Dystrophy Association and ALS One
- On April 8th, 2020, we hosted a patient webinar to discuss the impact of COVID-19 on the HEALEY ALS Platform trial. Three hundred and forty-four people joined the webinar to talk about impact of COVID-19 on ALS research. Healey Center investigators shared emphasis on patient centered care and research and how the team quickly adapted to increase dramatically our existing TELE ALS program to see all our patients virtually and to continue trials and offer therapeutic options. We answered questions about special care considerations for people with ALS and heard how critical it is for people and families with ALS that research and therapy development continue. Knowing that the team heard peoples' concerns and were working together to provide continued care and access was well received. The Healey Center ALS team at Mass General called all our patients to provide care, support and access to research. We will continue our webinar series for our patients and for people with ALS no matter where they live or receive care.
- The fourth drug for the HEALEY ALS Platform Trial will be submitted to the FDA shortly. Several additional companies have requested participation. Two of these have been selected by the Healey Center Therapy Evaluation Committee to be added as the 5th and 6th therapies.
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2ND ANNUAL INTERNATIONAL PRIZE FOR INNOVATION IN ALS
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The Healey Center International Prize for Innovation in ALS is an annual global prize celebrating excellence in research for a team of investigators who catalyze exceptional discoveries, leading to transformative advancement in therapy development in ALS.
The inaugural International Prize for Innovation in ALS was awarded during the 30th International Symposium on ALS/MND in Perth, Australia on December 6, 2019 to the team that brought to trial the first antisense oligonucleotide (ASO) gene therapy for ALS. Team members include researchers from academia and industry including 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, Toby Ferguson, MD, PhD, for Biogen, and Frank Bennett, PhD, for Ionis Pharmaceuticals.
The call for nominations for the 2
nd
annual International Prize for Innovation in ALS was
announced on May 1
st
and will be awarded at the Virtual International Symposium on ALS/MND in December 2020.
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ATTRACTING AND SUPPORTING YOUNG ALS INVESTIGATORS
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We are excited to announce that Drs. Aaron Held and Matthew Nolan were awarded the first
Cullen Education and Research Foundation Young Investigator
awards at the Healey Center starting July 2020.
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Dr. Aaron Held
completed his PhD at Brown University in 2018 under the mentorship of Dr. Kristi Wharton and Dr. Diane Lipscombe. His graduate work characterized knock-in Drosophila models of ALS and identified BMP signaling as a modifier of motor neuron degeneration. Aaron joined the laboratory of Dr. Brian Wainger at the Healey Center in December 2018, and his current projects focus on the relationship between hyperexcitability and neurodegeneration in ALS.
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Dr. Matthew Nolan
completed a PhD in Clinical Neuroscience at the University of Oxford, supervised by Dr. Olaf Ansorge. During his doctoral work he developed new ways of quantifying ALS pathology in the human brain to understand why some cells appear to be more susceptible to the protein aggregation associated with the disease than others. Dr. Nolan joined the laboratory of Dr. Clotilde Lagier-Tourenne as a postdoctoral fellow at the Healey Center in October, 2019. His interest is in understanding the molecular mechanisms underpinning ALS, with a focus on the role of RNA binding proteins in disease pathogenesis. His group is particularly interested in how a reduction in one RNA-binding protein at the nucleus, TDP-43, can lead to the reduction of another protein called Stathmin-2 elsewhere in the cell. This project will be based around understanding how they can manipulate Stathmin-2 expression, with a view to targeting it therapeutically.
The
Healey Scholars program
launched in 2019 and was first awarded to two young ALS post-doctoral fellows,
Nibha Mishra, PhD and Leslie Nash, PhD
in the laboratories of
Clotilde Lagier-Tourene, MD, PhD,
inaugural incumbent of the Araminta Broch-Healey Endowed Chair in ALS
at Mass General and
Kevin Eggan, PhD
, at Harvard StemCell Institute, for their project
“
TDP-43-dependent loss of stathmin-2 as a target in sporadic and familial ALS
.”
We were very excited to learn that this work led directly to additional funding of Healey Center affiliated researchers and members of our Scientific Advisory Council, Kevin Eggan, PhD, of Harvard University, Robert Brown, Jr., MD, DPhil, Professor and Chairman of the Department of Neurology at UMass Medical Center, and QurAlis, who are among six recipients of a new
AFTD and Target ALS grant program
.
The award allows the researchers to advance their study of the impact of restoring Stathmin 2 in individuals with sporadic ALS.
Identifying and supporting the most promising research at the earliest stages is at the heart of the Healey Center’s mission and allows researchers to leverage that support for significant downstream impact.
In 2020, the Healey Center will launch a global call for nominations to support additional Healey Scholars with the unique focus to train future leaders in ALS therapy development. Awardees will be offered an industry internship in the 2nd year with one of 6 collaborating pharma companies. There is no other training program to develop leaders in ALS therapy development like this in the world, and already there is huge interest from candidates and Industry partners.
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The Sean M. Healey & AMG Center for ALS at Mass General is the only clinical program to offer an Expanded Access Protocol (EAP) for our patients. The EAP provides access to experimental treatments for patients with ALS who might for a variety of reasons be ineligible for clinical trials.
The current EAP program offers active protocols of 6 investigational products with 65 patients enrolled. These experimental therapies have improved patients’ quality of life. Our patients have shared that they gain a sense of purpose and meaning from the opportunity to contribute as research participants. We partner with pharmaceutical companies to provide the drugs, while philanthropy covers the costs of patient qualification, enrollment and coordination, along with the analysis of study results.
In the midst of the COVID-19 crisis, we are continuing this vital program without interruption. We received permission to obtain consent from people with ALS and deliver our EAP entirely through virtual visits.
Our goal is to launch a nationwide EAP program to help people with ALS throughout the United States.
Our patient advisory group for the HEALEY ALS Platform Trial has specifically stated that one of their top priorities is our building an EAP program for people not eligible for the Platform Trial. We are excited to do this and strongly feel that these two synergistic initiatives will transform care and therapy development.
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If you have any questions or would like to learn more, please contact
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