BOSTON – The HEALEY ALS Platform trial led by the Healey & AMG Center for ALS at Massachusetts General Hospital (MGH) and the Northeast ALS Consortium (NEALS) is paving the way for rapid testing of potential treatments for people with amyotrophic lateral sclerosis (ALS), a neurodegenerative disease affecting the brain and spinal cord.

Enrollment is now complete for Regimen E, evaluating Trehalose from Seelos Therapeutics. Seelos is a clinical-stage biopharmaceutical company focused on developing therapies for central nervous system disorders and rare diseases.

Launched in 2020, the HEALEY ALS Platform Trial, the first platform trial for this disease, was designed to evaluate multiple investigational treatments simultaneously, thus accelerating the development of effective and breakthrough treatments for people living with ALS Trehalose marks the fifth regimen to complete trial enrollment in this innovative design paradigm.

“This milestone is a pivotal step forward in the HEALEY ALS Platform Trial and the fight against ALS,” says Merit Cudkowicz, MD, MSc, principal investigator and sponsor of the HEALEY ALS Platform Trial, director of the Healey & AMG Center for ALS and chair of the Department of Neurology at MGH, and the Julieanne Dorn Professor of Neurology at Harvard Medical School. “This achievement would not be possible without our partnerships with people living with ALS; the advice and feedback they provide allow us to develop effective ALS treatments and from the staff and clinicians at the NEALS clinical trial sites.”

SLS-005 (Trehalose) is a low molecular weight disaccharide that crosses the blood-brain barrier and is thought to stabilize proteins and activate autophagy through the activation of Transcription Factor EB (TFEB), a key factor in lysosomal and autophagy gene expression. Participants in Regimen E will be randomized 3:1 (drug: placebo) and studied with a primary endpoint measuring the change from baseline on the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score at 24 weeks. Secondary endpoints are also measured at 24 weeks, including change from baseline in slow vital capacity, muscle strength, quality of life measurements as well as additional signs of disease progression.

“We are so excited to move forward with this regimen now that enrollment is complete” says Shafeeq Ladha, MD Co-Lead of the SLS-005 arm of the Healey Platform trial and director of the Gregory W. Fulton ALS and Neuromuscular Disorders Center at Barrows Neurological Institute in Phoenix, Arizona. “The participation we are seeing is a testament to the power of the platform approach. We are grateful for the support from Tackle ALS, The ALS Association, ALS Finding A Cure®, ALS One, The Muscular Dystrophy Association, I AM ALS, and Tambourine ALS Collaborative. We will continue to add investigational treatments until we find cures for all people living with ALS”

For updates on the trial, please join our weekly Healey ALS Platform webinars.

Background on ALS
Amyotrophic lateral sclerosis, ALS, is the most prevalent adult-onset progressive motor neuron disease, affecting approximately 30,000 people in the U.S. and an estimated 500,000 people worldwide. ALS causes the progressive degeneration of motor neurons, resulting in progressive muscle weakness and atrophy. There are currently four FDA therapies approved specifically for treating ALS symptoms—Riluzole, Nuedexta, Edaravone and Relyvrio. But there is no cure.

About the Sean M. Healey & AMG Center for ALS at Mass General
At the Sean M. Healey & AMG Center for ALS at Mass General, we are on a quest to discover life-saving therapies for all individuals affected by ALS. Launched in November 2018, the Healey & AMG Center leverages a global network of scientists, physicians, nurses, caregivers, patients and families working together to accelerate the pace of ALS therapy discovery and development.
Under the leadership of Merit Cudkowicz, MD, chair of the MGH Department of Neurology, and a Science Advisory Council of international experts, we are reimagining how to develop and test the most effective therapies to treat the disease, identify cures and, ultimately, prevent it.

The key to our success is our tightly integrated research and clinical efforts, encouraging opportunities to bring the challenges our patients face every day into our laboratories, focusing investigations on finding solutions that will make a meaningful difference to our patients without delay. Our collaborative efforts are designing more efficient and effective clinical trials while broadening access to these trials for people with ALS.

About the Northeast ALS Consortium
The Northeast ALS Consortium (NEALS) is an international, independent, non-profit group of 134 research sites around the world that collaboratively conduct clinical research in Amyotrophic Lateral Sclerosis (ALS) and other motor neuron diseases. The mission of NEALS is to translate scientific advances into new treatments for people with ALS and motor neuron disease as rapidly as possible. The NEALS member sites are committed to the principles of open scientific communication, peer review and democratic governance of the Consortium’s organization and activities. Governed by an Executive Committee, the Consortium’s research activities are advised by an experienced Scientific Advisory Board. The NEALS Science Advisory Board also regularly guides academic and industry partners on ALS targets, trial design and conduct.

NEALS is dedicated to educating people with ALS about clinical research and therapy development to invite their participation, empowering them to be advocates for ALS clinical research, along with encouraging them to influence and improve the ALS research process.