Dear Friend of the Sean M. Healey & AMG Center for ALS,
I am writing today with a renewed sense of optimism and enthusiasm. Despite the challenges of the pandemic, the progress we are making in ALS research is unprecedented. Thanks to your continued support, the initiatives you made possible are now bearing fruit. Our partnership with the young company Amylyx Pharmaceuticals has led to positive results for their CENTAUR trial, which showed improvements in functional outcomes and survival for individuals with ALS; our game-changing HEALEY Platform trial has had the fastest enrollment of any ALS clinical trial and is on track to complete enrollment of four promising therapies this summer; our investigators have won several awards for the design of these new trials, garnering the interest and attention of researchers in other disciplines who want to contribute to the science of ALS; our Expanded Access Protocol program has been embraced by individuals with ALS and their families and we are working hard to fold this research into the larger platform trial and ensure these therapies are available nationwide.
We are enormously proud of Darlene Sawicki, NP, who was recognized for her nursing leadership and patient care with the first endowed nursing chair in ALS, and grateful for the commitment of our donor partners, who have made opportunities for Darlene, and several early-career investigators possible.
Your faith inspires all of us at the Healey Center to redouble our commitment to you, and to our goal of finding the cures for ALS for all.
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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AMX035 POSITIVE ALS RESULTS RECEIVES THREE AWARDS |
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The CENTAUR trial is the first trial to demonstrate positive effects of an investigational product (AMX0035) on both functional outcomes and survival in people with ALS. The study led by Healey & AMG Center investigators, Drs. Merit Cudkowicz and Sabrina Paganoni, in collaboration with Amylyx and the Northeast ALS Consortium, was published in the New England Journal of Medicine. Active regulatory discussions are underway in several territories including the United States, Canada and Europe. As such, this trial has the potential to change clinical practice and improve the natural history of ALS worldwide. Excitingly, Health Canada recently announced that Amylyx can apply for approval based on the CENTAUR single trial!
In addition, these trial results have generated considerable media attention and the three prestigious scientific recognition awards:
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HEALEY ALS PLATFORM TRIAL ENROLLS FASTER THAN ANY PREVIOUS OR CURRENT TRIAL |
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Our pivotal HEALEY ALS Platform Trial, the signature Healey & AMG Center initiative launched in July 2020 to accelerate therapy development has exceeded all hopes! Despite the challenges of the pandemic we are enrolling faster than any other ALS trial and attracting interest from people with ALS, both large and small pharmaceutical companies with promising neurotherapeutic drugs, and other disease groups who want to do the same for the disorders they support.
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Over 50% participants enrolled for first three regimens (treatments)
- 48 sites activated to enroll
- 32 more sites interested to join
- 4th regimen added in record time (Prilenia)
- Participants already enrolling in open label extension
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2 additional Pharma Companies joined (Seelos & Revalesio)
- 20+ companies interested
- Sharing knowledge and experience with investigators and foundations interested in replicating this model for pain, Alzheimer’s disease, Parkinson’s disease, frontal temporal dementia and more.
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EXPANDING ACCESS TO EXPERIMENTAL TREATMENTS FOR PEOPLE WITH ALS |
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The MGH Expanded Access Protocol (EAP) Program continues to grow. To date we delivered 10 investigational treatments and enrolled a total of 116 patients at Mass General who typically could not be part of trials. Learn more here.
We are building on our successful EAP and expanding it across our nationwide network to reach more people with ALS linked to our HEALEY ALS Platform Trial. We propose that all therapies in the HEALEY ALS Platform Trial also provide an EAP arm and all active companies have agreed!
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NEW AWARDS FROM THE HEALEY CENTER FOR ALS |
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Scholars Program
Our first global call for the Healey Scholars program was issued and received 21 applications from 6 countries including Australia, Canada, China, England, Switzerland, and the United States. The review committee convened on March 4, 2021 and selected the first four recipients from an exceptional group of young investigators. The $300,000 two-year awards will be sponsored in partnership with ALS Finding a Cure. These awards encourage more early-career scientists to pursue research in ALS and are only made possible through the support of our generous partners and donors. We know that the more brilliant minds we have focused on ALS, the sooner we will find the answers we need. Your support ensures we can renew this program annually.
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David Richards Endowed Nursing Chair in ALS
Darlene Sawicki, MSN, NP-BC, Director of Advance Practice Providers at Mass General, and a leading clinician at the Healey Center for ALS was named the first incumbent of the MGH Endowed Nursing Chair in ALS. This Endowed Chair will provide permanent resources to support the ALS nurses who play central, multifaceted roles in caring for our patients. A celebration and tribute to Ms. Sawicki’s leadership in ALS nursing, dedication to mentoring and training the next generation of nurses, and commitment to excellence in patient care was held virtually on March 3, 2021. Read more.
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International Prize For Innovation In ALS
Our second annual Healey Center International Prize for Innovation in ALS was awarded to Project MinE, the largest single disease whole genome sequencing project in the world and presented at the Virtual Symposium on ALS/MND December 11, 2020. The winning team was selected from 10 outstanding global team nominations with representation from 8 countries (Belgium, Canada, Germany, Ireland, Netherlands, Sweden, UK, USA).
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Gupta Family Endowed Prize for Innovation in ALS Care
Modeled after the Healey Center International Prize for Innovation in ALS, this new prize will provide an award to the top research team with promising new approaches to improving care for people living with ALS. The award will be given to the research team that best solves the annual key clinical care question and will be based on patients’ priorities and the project’s scientific rigor. We hope to award the first prize in late 2021 or early 2022.
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NEW SCIENTIFIC INITIATIVES |
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Through the advice of our expert Science advisory Council we are thinking big and developing bold new initiatives to REPAIR motor neuron function and PREVENT ALS all together.
These goals are ambitious and require the partnership of a broad community of scientists, clinicians, patients, foundations, industry, and most importantly, all of you.
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OUR CONTINUED SUCCESS DEPENDS ON YOUR PARTNERSHIP |
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We are determined to continue testing the most promising therapies in the HEALEY ALS platform, and need your help to ensure we don’t slow down for a moment. The positive impact of the EAP program on individuals with ALS, and on our strategies to disrupt or reverse disease progression, require we seek out and evaluate more therapies with the potential to make a difference. We are also eager to invite more early-career neurologists and scientists to come learn from our team and pursue the out-of-the-box, cross-disciplinary investigations we know will provide the answers we need.
Thank you for staying with us on this journey. Together we will reach the cures.
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