Investigational drug SLS-005 (Trehalose) receives “May Proceed” notice and Central IRB approval for the HEALEY ALS Platform Trial – Massachusetts General Hospital

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Press Release5 Minute ReadSep | 21 | 2021
The addition of the fifth regimen continues this new approach of more rapidly testing new therapies for 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 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.
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.
Merit Cudkowicz, MD
The Healey Center at 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.
Drug represents the fourth medication tested in this accelerated trial format.
Drug will be tested simultaneously with multiple treatments in novel, accelerated trial format
The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH) awarded the 2nd annual Healey Center International Prize for Innovation in amyotrophic lateral sclerosis (ALS) to Project MinE for developing the largest single disease whole genome sequencing project in the world.
The antiepileptic drug ezogabine reduced pathologic excitability of cortical and spinal motor neuron cells that are early signs of clinical dysfunction in people with amyotrophic lateral sclerosis.
An experimental medication that was recently shown to slow the progression of the neurodegenerative disease amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, has now demonstrated the potential to also prolong patient survival.
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