The Sean M. Healey & AMG Center at Massachusetts General Hospital is taking a bold new approach to therapy development for amyotrophic lateral sclerosis (ALS), launching a “platform trial,” in which several therapies are tested simultaneously.
The Healey Center effort is a first for ALS research, although the model has been successful in cancer research.
The Healey Center effort is a first for ALS research, although the model has been successful in cancer research. The trial 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.
ALS, also known as “Lou Gehrig’s Disease,” is an illness that attacks motor neurons that control muscle movement, causing progressive paralysis. Currently, only limited treatments are available and there is no cure. Recently, however, amazing advances in understanding the biology of ALS create more hope that effective treatments will be discovered soon.
Dozens of Promising Therapies
“Traditional clinical trials study one treatment to answer one question, and often take up to two years to complete,” says Merit Cudkowicz, MD, MSc, director of the Healey Center and chief of Neurology at Mass General. “The master trial allows us to test several therapies and add or drop therapies once their effectiveness is determined without the need to stop and restart the trial. With this tool, testing can continue until cures are found.
“The combination of advances in genetics, along with an infusion of research funding thanks to the Ice Bucket Challenge led to the identification of dozens of promising therapies in the laboratory,” Dr. Cudkowicz adds. “The challenge was getting those therapies into clinical trials where we could evaluate their effectiveness quickly and provide meaningful options for our patients.”
Sean M. Healey, founder and executive chairman of Affiliated Managers Group (AMG), was diagnosed with ALS in 2018. The Healey Center opened a few months later, thanks to a $40 million donation driven by Mr. Healey, along with members of AMG’s board and senior management team, supported by The AMG Charitable Foundation.
Healey Center Collaboration
The Healey Center’s goal is to accelerate the development of new therapies to treat ALS by creating a worldwide collaborative research effort that includes academic and pharmaceutical scientists and patients and their families.
Plans for the platform trial were developed with advice and guidance from the experts from Healey Center’s scientific advisory committee, Berry Consultants and the Northeast ALS Consortium (NEALS), the largest ALS clinical trial network in the world.
The enrollment of patients for the trials will begin in early 2020 …
The enrollment of patients for the trials will begin in early 2020 and 54 treatment centers from across the U.S. are expected to participate, ensuring broad access for people with ALS.
Home of the nation’s largest hospital-based research program, Mass General has been on the forefront of medical innovation for more than 200 years, providing compassionate care while bringing bold breakthroughs from the lab to patients’ bedsides.
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