BOSTON – The Sean M. Healey & AMG Center for ALS at Mass General has received approval from the U.S. Food and Drug Administration (FDA) to proceed with administering three proposed drug regimens in the HEALEY ALS Platform Trial – the first trial of its kind for amyotrophic lateral sclerosis (ALS).

The three experimental treatments include, in no particular order: Zilucoplan, a small macrocyclic peptide inhibitor of complement component 5 [C5], developed by 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.

“This is a huge milestone for the entire ALS community,” says Sean Healey, who with AMG and his family and friends, helped launch the Healey Center and the platform trial.

Merit Cudkowicz, MD, MSc, director of the Sean M. Healey & AMG Center for ALS at Mass General adds, “Through dedication, enthusiasm, and collaboration, the Healey Center, and its many collaborators are one step closer to accelerating ALS therapy development. We are very grateful for the support of the Healey Center, Tackle ALS, the ALS Association, sALSa for a cure, ALS One and the recent ice bucket challenge 2 to help launch this initiative. We thank the many individuals with ALS and their family members who provided invaluable input and advocacy from IAMALS and the NEALS Clinical Research Learning Institute. The success of this new initiative depends on the entire ALS community working together to solve ALS.”

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.

Fifty-four Northeast ALS Consortium (NEALS) trial-ready sites across the US were selected for participation in the HEALEY ALS Platform Trial. The next steps in the process before individuals can officially enroll in the study include obtaining approval from the central Institutional Review Board (IRB), training the NEALS sites on the study protocol, contracting with the different vendors key to drug distribution, safety monitoring and biomarker development.

“Obtaining a ‘May Proceed’ notice from the FDA means there are no major issues with the study and the FDA agrees that we can move forward with the first three experimental treatments to be tested,” says Sabrina Paganoni, MD, PhD, a physician scientist at the Healey Center and co-lead investigator for the HEALEY ALS Platform Trial. “This is a crucial step in providing more access and more options for people with ALS.”

The HEALEY ALS Platform trial is a perpetual study that will continue to add interventions until cures are found for all people with ALS.

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—riluzole, nuedexta and edaravone.

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, clinicians, project managers and information technologists 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 are partnering with other experts around the globe to reimagine how to develop the right novel therapies for the right people at the right time.

Key to our success is our spirit and dedication to collaboration. We partner with the best scientists and clinicians from all over the world in a shared mission to develop treatments to cure ALS. We believe strongly 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.

About the Northeast ALS Consortium

The Northeast ALS Consortium (NEALS) is an international, independent, non-profit group of 128 research sites around the world who 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 network is governed by an Executive Committee and the Consortium research activities are advised by an experienced Scientific Advisory Board. Each Leadership team member brings a depth of experience and a unique perspective to the governance of NEALS. Through this leadership, active oversight is provided for ongoing operations along with new approaches to trial design evaluated and implemented.