The HEALEY ALS Platform Trial completes enrollment for first three investigational treatments

BOSTON – The HEALEY ALS Platform trial led by the Healey & AMG Center for ALS at Massachusetts General Hospital 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 the first three investigational treatments in the ongoing study. 

The HEALEY ALS Platform Trial, the first platform trial for this disease, is designed to evaluate multiple investigational treatments simultaneously, thus accelerating the development of effective and breakthrough treatments for people living with ALS. It currently includes 52 sites from NEALS and it  will continue to add clinical sites, participants and investigational treatments until effective approaches for ALS are discovered.

“This is a critical milestone, guiding the timeline for trial completion and result reporting, ultimately putting us on the path forward towards finding treatments for people with ALS. We thank all the people with ALS who provided invaluable advice and support and are taking part in this new approach to therapy development in the disease,” says Merit Cudkowicz, MD, principal investigator and sponsor of the HEALEY ALS Platform Trial,  director of the Sean M. Healey & AMG Center for ALS and chief of the Department of Neurology at MGH, and the Julieanne Dorn Professor of Neurology at Harvard Medical School.

The first three investigational treatments include a) zilucoplan, a small macrocyclic peptide inhibitor of complement component 5 [C5], b) verdiperstat, an oral myeloperoxidase inhibitor, and c) CNM-Au8, a cellular energetic nanotherapeutic. Enrollment is expected to be completed very shortly for the fourth investigational treatment, pridopidine, a sigma 1 receptor agonist.

“This important enrollment milestone was made possible by the extraordinary collaboration with so many in the ALS community, including patients and families, clinical trial sites, industry partners and research collaborators and by funding from the Healey & AMG Center, Tackle ALS, The ALS Association, ALS Finding A Cure^®, ALS One, The Muscular Dystrophy Association, I AM ALS, and Tambourine ALS Collaborative,” says Sabrina Paganoni, MD, PhD, a physician scientist at the Healey Center, co-lead investigator of the trial and assistant professor of Physical Medicine and Rehabilitation at Harvard Medical School. “Participation in clinical trials is vital to the development of new treatments for people living with ALS.”

For more information, please see our website and view a list of HEALEY ALS Platform Trial sites.

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 three FDA therapies approved specifically for treating ALS symptoms—riluzole, nuedexta and edaravone. 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 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, chief of the Department 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.