Key Takeaways

  • Investigators are planning to test the potential of RNS60 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.

RNS60 was selected by the Healey Therapy Evaluation Committee based on strong science and safety in early phase trials in both ALS and other diseases.

Merit Cudkowicz, MD,
Director, Sean M. Healey & AMG Center for ALS at Mass General

BOSTON – In summer of 2020, despite the challenges posed by the pandemic, the Sean M. Healey & AMG Center at Massachusetts General Hospital (MGH) and the Northeast ALS (NEALS) Consortium 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. The trial opened at several sites of the NEALS Consortium and has been enrolling participants to test four investigational products so far.

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 selected the next— RNS60 —for possible entry into the trial.

RNS60 is a novel neuroprotective and immune-modulatory agent that has shown significant effects in pre-clinical models of ALS and other neurodegenerative diseases. RNS60 activates intracellular signaling pathways that support mitochondria and have anti-inflammatory effects and thereby promote cellular survival and differentiation.  The drug is being developed by Revalesio Corporation, a clinical stage pharmaceutical company dedicated to developing treatments for neurological disorders.

“RNS60 was selected by the Healey Therapy Evaluation Committee based on strong science (including work in cellular and animal models of ALS) and safety in early phase trials in both ALS and other diseases. There is an ongoing clinical trial led by Drs. Ettore Beghi and Letizia Mazzini at 22 centers in Italy” 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 work closely with our colleagues in Italy and the results of their study will help inform the final design of the RNS60 regimen in the HEALEY ALS platform trial.”

“RNS60 was also safe and well tolerated in a previous open label pilot study at MGH,” says Sabrina Paganoni, MD, PhD, a physician scientist and faculty member at the Healey Center.

The Healey Platform Trial Design team will work closely with Revalesio to design this new regimen. As for all new regimens in the HEALEY ALS Platform Trial, FDA and central Investigational Review Board approvals are required prior to enrollment.

The platform trial approach speeds up scientific discovery, reduces trial start-up times, 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.

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