Completing enrollment for the fourth regimen in the Platform Trial provides further hope towards discovering treatments for people living with ALS using this novel approach to therapy development in the disease.

Merit Cudkowicz, MD, MSc
Director, Sean M. Healey & AMG Center for ALS, Massachusetts General Hospital


BOSTONThe HEALEY ALS Platform trial led by the Northeast ALS Consortium (NEALS) and the Healey & AMG Center for ALS at Massachusetts General Hospital 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.

Drug candidates that enter the platform trial are selected by a group of expert ALS scientists and members of the Healey & AMG Center Therapy Evaluation Committee. Enrollment is now complete for the fourth investigational treatment (pridopidine) 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 uses the Mass General Brigham central IRB for additional efficiencies and includes 52 sites from NEALS and will continue to add geographically clinical sites, participants and investigational treatments until effective approaches for ALS are discovered.

Pridopidine, the fourth treatment regimen, is an oral drug candidate, acting as a highly selective Sigma-1 receptor (S1R) agonist. The S1R is a protein that regulates various cellular processes relevant to neurodegenerative diseases. Preclinical studies have demonstrated pridopidine’s promise as a treatment for patients with ALS.

“Completing enrollment  for the fourth regimen in the Platform Trial provides further hope towards discovering treatments for people living with ALS using this novel approach to therapy development in the disease,” 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 and chief of the Department of Neurology at MGH, and the Julieanne Dorn Professor of Neurology at Harvard Medical School. We are grateful for our partnerships with people with ALS who provide advice and input and without whose participation we could not develop effective ALS treatments.” 

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. The first three investigational treatments have already completed enrollment.

“The HEALEY ALS Platform Trial is disrupting the pace of drug development by testing several investigational products in close collaboration with the patient community and with several clinical trial sites, industry and academic partners. The exceptional enrollment rates we are seeing are 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,” says Sabrina Paganoni, MD, PhD, a physician-scientist at the Healey & AMG Center and co-lead investigator of the 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. 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.