First Participants Enrolled in the Groundbreaking Trial Led by the Sean M. Healey & AMG Center for ALS at Mass General

Though the nation and the world continue to grapple with the COVID-19 pandemic, several sites from the Northeast ALS Consortium (NEALS) are participating in the HEALEY ALS Platform Trial, an initiative led by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, and have enrolled their first patients in the groundbreaking study.

Though COVID-19 has presented unique challenges for clinical research, the Healey Center team and their NEALS colleagues worked tirelessly to adapt the trial over the last few months to ensure patients could safely participate.

As the first ever platform trial for amyotrophic lateral sclerosis (ALS), the HEALEY ALS Platform Trial is testing three proposed drug regimens and will add two more over the next several months. The three experimental treatments include, in no particular order: Zilucoplan, a small macrocyclic peptide inhibitor of complement component 5 [C5], developed by UCB Ra Pharmaceuticals, Inc.; Verdiperstat, an oral myeloperoxidase inhibitor, developed by Biohaven Pharmaceutical Holding Company Ltd., and CNM-Au8 nanocrystalline gold, an intracellular nanocatalyst to support cellular bioenergetics developed by Clene Nanomedicine, Inc.

“We are very thankful to the many patients, colleagues at Mass General, Barrow Neurological Institute and our partner institutions including NEALS, University of Rochester and Berry Consultants who worked closely with us to design the HEALEY Platform Trial,” says Merit Cudkowicz, MD, director of the Sean M. Healey & AMG Center for ALS at Mass General and chief of the Department of Neurology. “These groups worked to develop strategies to add home monitoring technologies, to adapt the trial during the COVID-19 pandemic.”

Including Mass General, there are five sites up and running in Massachusetts, Texas, Connecticut and Florida. The goal is to open all 54 HEALEY ALS Platform trial sites across the U.S. by early fall, on a rolling basis. Teams will recruit up to 480 participants for the first three treatments.

“I am very excited to be participating in the HEALEY ALS Platform Trial, a very innovative trial design that should significantly accelerate drug development for ALS patients and bring more hope to the ALS community,” says Daragh Heitzman, MD, director of the ALS Center at Texas Neurology and site investigator.

“ALS clinicians and investigators are enthusiastic about this approach that brings together the entire ALS community”, says Sabrina Paganoni, MD, PhD, a physician scientist at the Healey Center and co-lead investigator of the trial. “We are grateful to Tackle ALS, the ALS Association, ALS Finding a Cure, sALSa for a cure, ALS One and the Ice Bucket Challenge 2 for their support and we thank the several people with ALS and their families who are contributing with their advocacy and participation.”

Calaneet Balas, President and CEO of The ALS Association, comments, “By testing multiple drugs at the same time, this platform trial will dramatically speed our search for effective ALS treatments for everyone living with ALS. The ALS Association is proud to be supporting this effort, and we are grateful to the Healey Center for their leadership.”

A "platform trial" is a clinical trial in which multiple treatments are evaluated simultaneously. New treatments are added to the platform as they become available, thereby decreasing the gap in time from identification of an exciting therapy to testing. This model, already proven successful in the cancer field, will greatly accelerate therapy development of effective and breakthrough treatments for people with ALS by allowing investigators to test more drugs, increase patient access to trials and reduce the cost by quickly and efficiently evaluating the effectiveness of multiple therapies.

“This trial fulfills our promise to Sean Healey, a visionary leader whose entrepreneurial skills and determination pushed us forward,” says Cudkowicz. “I am forever grateful to Sean, his family and friends for making this possible.”

The HEALEY ALS Platform Trial is perpetual and will add clinical sites, participants and experimental treatments until a cure for ALS is discovered.

For more information, view a list of 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.