Key Takeaways

  • Investigators are testing the potential of pridopidine for the treatment of amyotrophic lateral sclerosis (ALS) in the HEALEY ALS Platform Trial
  • This first ever platform trial for amyotrophic lateral sclerosis speeds up scientific discovery by testing and evaluating multiple treatments simultaneously

The trial infrastructure is perpetual and meant to test more drugs until cures are found for all people with ALS.

Sabrina Paganoni, MD, PhD
Sean M. Healey & AMG Center for ALS, Massachusetts General Hospital

BOSTON – In 2020, the Sean M. Healey & AMG Center at Massachusetts General Hospital launched the first platform trial for amyotrophic lateral sclerosis (ALS), in which multiple treatments are tested and evaluated simultaneously to accelerate the development of therapies for people with ALS. Drug candidates that enter the platform trial are chosen by a group of expert ALS scientists and members of the Healey Center Science Advisory Committee. Investigators have now added a fourth promising drug— pridopidine—to the trial and the first participant received study drug at the MGH site this week.

Pridopidine stimulates the Sigma-1 receptor (S1R), a protein that regulates various cellular processes relevant to neurodegenerative diseases. The drug is developed by Prilenia, a clinical stage pharmaceutical company that is developing treatments for neurodegenerative and neurodevelopmental disorders. Preclinical studies have demonstrated pridopidine’s promise as a treatment for patients with ALS, a degenerative condition without a cure that attacks brain and spinal cord nerve cells.

“S1R is a genetically validated target for ALS, and lab experiments have shown great potential for pridopidine to correct neurodegenerative processes. Further, the drug has shown an excellent safety record in previous clinical trials,” says Merit Cudkowicz, MD, director of the Sean M. Healey & AMG Center for ALS at Mass General, Chief of the Department of Neurology, and Principal Investigator of the HEALEY ALS Platform Trial.

“We are thrilled to add pridopidine to the HEALEY ALS Platform Trial. The platform trial was designed to disrupt the pace of drug development for ALS. The trial infrastructure is perpetual and meant to test more drugs until cures are found for all people with ALS,” says Sabrina Paganoni, MD, PhD, a physician scientist and faculty member at the Healey Center. “The innovative and patient-centered design allowed us to experience record levels of enrollment since launch in the summer despite several challenges posed by the COVID-19 pandemic.”

The platform trial approach speeds up scientific discovery, decreases costs, and allows data from participants in placebo groups to be shared. This results in high statistical power and allows more participants to receive active (rather than placebo) treatments.

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 August 2020, Mass General was named #6 in the U.S. News & World Report list of "America’s Best Hospitals."

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 people who are affected by ALS. Launched in November 2018, we are a diverse group of researchers and clinicians determined to accelerate the pace of ALS therapy development.

Under the leadership of Merit Cudkowicz, MD, chief of Neurology, and a Science Advisory Council of international experts, we partner with other experts around the globe to reimagine how to develop the right novel therapies for the right people at the right time. We are grounded in our strong belief in the partnership between people with ALS, caregivers and our team. Together we can design more efficient and more effective clinical trials and broaden access for people with ALS.

For more information, please email and visit the center’s website

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.