Merit Cudkowicz, MD, MSc
The addition of the fifth regimen continues this new approach of more rapidly testing new therapies for people with ALS.
Director, Sean M. Healey & AMG Center for ALS
– The Sean M. Healey & AMG Center for ALS
at Mass General received approval both from the U.S. Food and Drug Administration
(FDA) and the Mass General Brigham Institutional Review Board to proceed with adding SLS-005 (trehalose injection, 90.5 mg/ML for intravenous infusion) as an additional regimen in the HEALEY ALS Platform Trial.
Investigational treatment candidates that enter the platform trial are chosen by a group of expert ALS scientists and members of the Healey Center Science Advisory Committee. Investigators from the HEALEY ALS Trial Design Committee worked with Seelos Therapeutics, Inc. to include SLS-005’s
pivotal Phase IIb/III trial in ALS on the platform.Trehalose
is a low molecular weight disaccharide (0.342 kDa) that crosses the blood-brain barrier, stabilizes proteins and, activates autophagy, which is the process that clears material from cells. In animal models of several diseases associated with abnormal cellular protein aggregation or storage of pathologic material, Trehelose has been shown to reduce aggregation of misfolded proteins and reduce accumulation of pathologic material. Trehalose activates autophagy through the activation of Transcription Factor EB (TFEB), a key factor in lysosomal and autophagy gene expression. Activation of TFEB is an emerging therapeutic target for a number of diseases with pathologic accumulation of storage material.
The HEALEY ALS Platform Trial is designed to evaluate multiple investigational treatments simultaneously, thus accelerating the development of effective and breakthrough treatments for people living with ALS. Trehalose would be the fifth regimen in the Platform Trial. The Sean M. Healey & AMG Center for ALS works closely with the Northeast ALS Consortium
(NEALS) and industry partners to tailor the Platform Trial arm to their experimental study drugs.
Several sites from NEALS are currently participating in the HEALEY ALS Platform Trial. The trial is designed to be perpetual and will add clinical sites, participants and experimental treatments until a cure for ALS is discovered.
“The addition of the fifth regimen continues this new approach of more rapidly testing new therapies for people with ALS. We are grateful to all the foundations and individuals who support the HEALEY ALS Platform Trial, including but not limited to The AMG Charitable Foundation, Tackle ALS, the ALS Association, ALS Finding A Cure, ALS One and The Muscular Dystrophy Association
,” says Merit Cudkowicz
, MD, MSc, principal investigator and sponsor of the HEALEY ALS Platform Trial, director of the Sean M. Healey & AMG Center for ALS and chief of the Department of Neurology at MGH, and the Julieanne Dorn Professor of Neurology at Harvard Medical School.
The first four investigational treatments
include zilucoplan, a small macrocyclic peptide inhibitor of complement component 5 [C5], verdiperstat, an oral myeloperoxidase inhibitor, CNM-Au8, catalytically-active gold nanocrystals to support increased cellular energy production and pridopidine, a highly selective Sigma1 receptor (S1R) agonist. Future investigational treatments are in development and will be added to the HEALEY ALS Platform Trial.“We are immensely grateful to all the people living with ALS who have trusted our group and allowed us to continue our long but rewarding path towards finding a treatment for ALS,” says Shafeeq Ladha, MD, neurologist at the Gregory W. Fulton ALS and Neuromuscular Disease Center at Barrow Neurological Institute, in Phoenix, Arizona, and co-lead investigator for the Trehelose regimen in the Healey ALS Platform Trial.
For more information, please visit our website
and view a list of HEALEY ALS 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.