Dear Friend,
As we approach the one-year anniversary of the Sean M. Healey & AMG Center for ALS at Mass General, we want to share several amazing success stories – successes made possible by Sean, his family and friends, and all of you!
Our goal is to accelerate the pace of ALS therapy development by bringing together a global community of the best minds to conquer ALS. We have done this!
With amazing input from our Scientific Advisory Committee, we:
- Chose the top five most promising therapies from 27 applications submitted from 10 countries. We have resounding support from the FDA, from patients all over the globe, from our 132 investigators from sites all over North America and from biopharma companies.
- For the first time, our Healey Center team brought three companies, and the FDA, together in the same room to confirm final approval of the HEALEY ALS Platform Trial design.
- Galvanized and partnered with the patient community to revolutionize how treatments are developed for ALS.
- Collaborated across disciplines and disease areas to bring more scientists together to focus on finding cures. This led directly to funding the first study of B cell dysfunction in ALS.
- Awarded the first two Healey Scholars to attract the brightest young minds to the ALS field.
- Created the only center to offer five expanded access protocols to people with ALS.
Our Healey investigators spoke at conferences around the world and published more than two dozen scholarly articles. Sharing our work and bringing researchers together accelerates our efforts to find the cures. We are making enormous progress, thanks to your generous support of our efforts. Every day brings us closer to answers.
Warmly,
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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HEALEY ALS PLATFORM TRIAL
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The experimental treatments include: zilucoplan, a small macrocyclic peptide inhibitor of complement component 5 [C5], developed by Ra Pharmaceuticals, Inc.; Verdiperstat, an oral myeloperoxidase inhibitor, developed by Biohaven Pharmaceutical Holding Company Ltd.; Bioenergetic Nanocatalysis (CNM-Au8, nanocrystalline gold) developed by Clene Nanomedicine, Inc.; Pridopidine, a highly selective S1R agonist, developed by Prilenia Therapeutics; and IC14 immunotherapy, developed by Implicit Bioscience Ltd.
We worked closely with Berry Consultants, the leading statistical group for adaptive platform trials, and the Northeast ALS Consortium (NEALS), the largest ALS clinical trial network in the world, to design the HEALEY ALS Platform. The result is a truly revolutionary clinical trial approach that cuts the time to finding an effective treatment by 50 percent and decreases development costs by about 30 percent.
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OUTREACH AT THE HEALEY CENTER FOR ALS
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Work at the Healey Center is gaining attention around the globe. Our outstanding faculty were invited to speak at fourteen conferences across the globe about our research, our organizational approach and on our efforts to bring patients, families, communities and scientists together to focus on solving one problem – ALS.
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HEALEY FAMILY ALS ENDOWED CHAIR FOR RESEARCH
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On May 20, one of the world’s leading ALS researchers, Clotilde Lagier-Tourenne, MD, PhD, was named the inaugural incumbent of the Healey Family ALS Endowed Chair for Research. Dr. Lagier-Tourenne’s creativity and skill have already led to the identification of a disease pathway common to all forms of ALS, and indeed, all neurodegenerative diseases. When Dr. Lagier-Tourenne and Healey Center Advisory council member Dr. Kevin Eggan published back-to-back research on the promise of STATHMIN-2, suggesting that ALS might be treated by restoring this protein, the Healey Center advisory council made pursuing that work a priority.
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This year’s Healey Scholars,
Nibha Mishra, PhD
(photo left)
and
Leslie, Nash, PhD
(photo right), are dedicated to advancing the research around the effect of STATHMIN-2 on ALS.
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THE POWER OF COLLABORATION
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One of the greatest strengths of the Healey Center is the international expertise of the Scientific Advisory Council (SAC). Our meetings are opportunities to brainstorm about ALS science, identify research gaps and pool our contacts to encourage researchers from other scientific specialties to join our cause. We made extraordinary progress this year thanks to the resources the Healey Center and our partners have made available, but also thanks to the dedication of the SAC to direct those resources where they can make the largest impact.
At our May, 2019 meeting of the Healey SAC we invited several new investigators from other fields to learn about therapeutic approaches in other disease areas that might be effective in ALS. At the meeting, we heard from Mark Poznansky, MD, PhD, who is the director of the Vaccine and Immunotherapy Center (VIC) at Mass General and an attending physician in Infectious Diseases medicine. Dr. Poznansky spoke about how the progress in our understanding of immunotherapy has revolutionized treatment of cancers and autoimmune diseases.
This led directly to the launch of an exciting collaboration with Dr. Poznansky’s team at VIC: a research effort to determine if there is a defect in B cells associated with ALS.
If the hypothesis is proven, there is the potential that ALS could be treated using B cell therapy.
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SUPPORT OUR PROGRAMS AT THE HEALEY CENTER
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Please join our quest to discover life-saving therapies for individuals who are affected by ALS. At the
Sean M. Healey and AMG Center for ALS at Mass General
, we partner with the best scientists, our patients and friends like you to turn laboratory successes into promising therapies.
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