The Sean M. Healey & AMG Center at Massachusetts General Hospital will launch the first platform trial for amyotrophic lateral sclerosis (ALS) to accelerate the development of effective and breakthrough treatments for people with ALS.

A "platform trial" is a clinical trial in which multiple treatments are tested and 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 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.

“The Healey Center brought together top leaders from around the world to boldly re-think how we design ALS trials, to create a groundbreaking platform trial program, and deliver compassionate care,” says Merit Cudkowicz, MD, MSc, director of the Sean M. Healey & AMG Center for ALS at Mass General. “This approach cuts the time to find an effective treatment in half, decreases costs by a third or more, and is supported by our patients, the FDA, ALS clinicians and scientists and our pharma colleagues.”

In addition, because different treatments are tested using a shared infrastructure and a common master protocol, data from participants in all placebo groups can be pooled. This results in high statistical power to see a treatment and increase the participant’s individual chance of being on active treatments much higher than in traditional trials.

"This design optimizes chances for people to receive active treatment, provides answers faster, and ensures we keep learning about the disease," says Cudkowicz.

The HEALEY ALS Platform Trial will include several promising biomarkers and novel outcome measures and will be an engine to develop exciting novel tools to revolutionize how therapies are developed.

“We are thrilled to be in a position to test several treatments for ALS and, at the same time, facilitate the development of breakthrough biomarkers that can more quickly predict therapeutic success,” says Sabrina Paganoni, MD, PhD, a physician scientist and faculty member at the Healey Center. “In this new paradigm, more patients will have access to promising therapies and the trial will provide important contributions to our knowledge about ALS.”

The Healey Center issued a call for the best therapeutic ideas ready to enter the HEALEY ALS Platform Trial and received an outstanding response with almost 30 applicants from 10 countries. A group of expert ALS scientists and members of the Healey Center Science Advisory Committee chose the top five therapeutic ideas ready to enter the platform trial. The Center will launch the platform trial with three therapies first, with plans to add two more shortly afterwards. The 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.; Bioenergetic Nanocatalysis (CNM-Au8, nanocrystalline gold) developed by Clene Nanomedicine, Inc.; Pridopidine, a highly selective S1R agonist, developed by Prilenia Therapeutics; and IC14 immunotherapy, developed by Implicit Bioscience Ltd.

About the first 5 treatments

Industry partners from these first five companies are working closely with the Healey Center Trial Design Team, the Northeast ALS Consortium (NEALS) and Berry Consultants to tailor the arm of the HEALEY ALS Platform Trial. 54 NEALS trial-ready sites across the U.S. were selected for participation in the HEALEY ALS Platform Trial.

“The investigators in NEALS are enthusiastic about this approach," says Timothy Miller, MD, PhD, co-chair of NEALS and David Clayson Professor of Neurology at Washington University School of Medicine. "The ability to test multiple drugs at one time will move us more quickly toward the goal of finding a treatment for ALS.” 

Partial financial support to initiate these first treatments is made possible thanks to the generosity of the Healey family and friends and the AMG Charitable foundation along with partners at TackleALS. The Healey Center at Mass General is working closely with industry partners to finalize contracting in the coming weeks, while continuing to seek additional partnerships with foundations to support, sustain and expand the program over time.  Several other exciting therapies for people with ALS are in the pipeline and the Healey Center’s goal is to add new treatments to the platform until effective treatments are found.

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

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 at the Healey Center at Mass General determined to accelerate the pace of ALS therapy development.

Under the leadership of Merit Cudkowicz, MD, MSc, 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.

Terri Janos, Mass General Public Affairs,, 617-726-0954