Healey & AMG Center Announces Update in ALS Platform Trial with Trehalose

BOSTON – The HEALEY ALS Platform Trial led by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH) reports that Regimen E evaluating trehalose, developed by Seelos, versus placebo in adults with amyotrophic lateral sclerosis (ALS) did not meet the primary or key secondary endpoints.

Data from the trial showed there was no statistically significant difference between the trehalose treated group and the placebo group     from baseline through week 24 in disease severity over time as measured by ALS Functional Rating Scale-Revised (ALSFRS-R, a 12-item functional activity scale) and accounting for survival for the trehalose treatment group vs placebo. In addition, the key secondary endpoints including muscle and respiratory function were not met.

The HEALEY ALS Platform Trial was designed to accelerate the development of breakthrough treatments for persons with ALS by testing multiple drugs in parallel. The trial leverages a shared placebo group and central infrastructure to make efficient use of resources. Each drug regimen is designed to provide go / no go decisions for a particular drug. Trehalose was the fifth treatment regimen tested using this innovative paradigm. Two other regimens, testing two other drugs, are currently enrolling participants and more regimens are expected to be added in the future. The central infrastructure allows the study team to learn from each regimen and continue to refine and adapt the trial

“Though we did not achieve success with this trial, we remain committed to using the data to advance ALS science, and our experience with the trial to understand ALS," says Merit Cudkowicz, MD, MSc, principal investigator and sponsor of the HEALEY ALS Platform Trial, director of the Sean M. Healey & AMG Center for ALS, chief of the Department of Neurology at MGH, and the Julieanne Dorn Professor of Neurology at Harvard Medical School. “We are thankful to the many people who participated in this study. We will learn from these results and continue to use this data to inform future advances in ALS trial design.”

“We would like to extend our sincere gratitude to participants in the trehalose regimen who committed their invaluable time and energy in the trial as well as the NEALS investigators and study staff for their dedicated effort and expertise," says Shafeeq Ladha, MD, Barrow Neurological Institute and co-lead investigator of the trehalose regimen. “Even though the trial results were negative, participants' involvement in the trial contributed greatly to ALS research by providing substantial clinical and biomarker data that will help us better understand ALS disease mechanisms and the role of the cellular catalysts in people living with ALS and may help unlock keys to the next ALS treatment.”

The Healey ALS Platform Trial is supported by funding from The AMG Charitable Foundation, Tackle ALS, The ALS Association, ALS Finding A Cure^®, ALS One, The Muscular Dystrophy Association, Arthur M. Blank Family Foundation, Tambourine ALS Collaborative and countless individuals who support the HEALEY ALS Platform Trial.

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 leading to death by respira. There are currently few FDA therapies approved for treating ALS—riluzole, edaravone (IV and oral formulation), Relyvrio, and Nuedexta.

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 Center leverages a global network of scientists, physicians, nurses, caregivers, people with ALS and families working together to accelerate the pace of ALS therapy discovery and development.

Under the leadership of Merit Cudkowicz, MD 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 Massachusetts General Hospital
Massachusetts General Hospital, founded in 1811, is the original and largest teaching hospital of Harvard Medical School. The Mass General Research Institute conducts the largest hospital-based research program in the nation, with annual research operations of more than $1 billion and comprises more than 9,500 researchers working across more than 30 institutes, centers and departments. In July 2022, Mass General was named #8 in the U.S. News & World Report list of "America’s Best Hospitals." MGH is a founding member of the Mass General Brigham healthcare system.