The first participants have enrolled in the multisite clinical trial for a first-in-kind therapeutic nanocatalyst, nanocrystalline gold (CNM-Au8; Clene Nanomedicine, Salt Lake City, UT) for the treatment of amyotrophic lateral sclerosis (ALS).
The therapy is a concentrated, aqueous suspension of clean surfaced, noncrystalline gold that acts catalytically to support important intracellular biological reactions. It consists solely of gold atoms organized into faceted, geometrical crystals held in suspension in sodium bicarbonate buffered, pharmaceutical-grade water.
The therapy has demonstrated safety in phase 1 studies in healthy volunteers and showed remyelination and neuroprotection effects in multiple preclinical models.
The goals of the Healy ALS Platform Trial are to reduce trial time and costs and increase individual participation in studies to develop novel therapies for ALS.
“This is a huge milestone for the ALS community and beyond,” said Merit Cudkowicz, MD, director of the Sean M. Healey & AMG Center for ALS and chief of neurology at Massachusetts General Hospital. “We’re grateful to our supporters and collaborators who have worked tirelessly throughout this pandemic to ensure that the trial can begin safely and efficiently.”
Danielle S. Shpiner, MD; Crystal Dixon, MD; Melissa R. Ortega, MD; and Henry Moore, MD
Jason A. Ellis, MD; Benjamin W. Y. Lo, MD; Chirag G. Bhatia, BS; Yona Feit; Steven Mandel, MD; and Dana Shani, MD
Jill M. Giordano Farmer, DO