BOSTON – The HEALEY ALS Platform trial led by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital in collaboration with the Northeast ALS Consortium (NEALS) has successfully enrolled its first participants for the investigational product SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion).
Investigational product candidates that enter the platform trial are selected by a group of expert ALS scientists and members of the Platform Trial Therapy Evaluation Committee.
The HEALEY ALS Platform Trial is designed to evaluate multiple investigational products simultaneously, thus accelerating the development of effective and breakthrough treatments for people living with ALS. The Sean M. Healey & AMG Center for ALS works closely with the Northeast ALS Consortium (NEALS) and industry partners to tailor the platform trial regimen to their investigational product.
“This is an important step forward to learning about the safety and efficacy of SLS-005 for people living 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, chief of the Department of Neurology at MGH, and the Julieanne Dorn Professor of Neurology at Harvard Medical School.
SLS-005 is a low molecular weight disaccharide (0.342 kDa) that crosses the blood brain barrier and is thought to stabilize proteins and activate 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. In animal models of several diseases associated with abnormal cellular protein aggregation or storage of pathologic material, SLS-005 has been shown to reduce aggregation of misfolded proteins and reduce accumulation of pathologic material. SLS-005 has previously received Orphan Drug Designation for the treatment of ALS from the U.S. Food and Drug Administration and from the European Medicines Agency in the EU. SLS-005 is an investigational treatment and is not currently approved by any health authority for medicinal use.
Additional products under investigation in the platform trial include: a) zilucoplan, a small macrocyclic peptide inhibitor of complement component 5 [C5], b) verdiperstat, a brain-penetrant myeloperoxidase enzyme inhibitor, c) CNM-Au8, a cellular energetic nanocatalyst, and d) pridopidine is an oral, highly selective Sigma-1 receptor (S1R) agonist. Enrollment is completed for the first four investigational products.
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.
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, people with ALS and families working together to accelerate the pace of ALS therapy discovery and development.
Under the leadership of Merit Cudkowicz, MD 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.