SUNY Upstate Medical University is participating in a national clinical trial to determine if lithium, a mood stabilizer, can slow the disease progression of early stage Amyotrophic Lateral Sclerosis (ALS) in humans.
According to Jeremy Shefner, M.D., Ph.D., findings from a study conducted in Italy demonstrated that lithium showed neuroprotective properties in experimental animals and promising results in a small trial of ALS patients. Shefner is SUNY Upstate's lead investigator of the study and professor and chair of the university's Department of Neurology.
"To more fully study the effects of lithium in ALS, the National Institutes of Health /National Institute of Neurologic Diseases and Stroke, the ALS Association and the Canadian ALS Association has funded this important trial," Shefner said. "We will recruit volunteers to participate in the trial. Patient volunteers may be included if they are within three years of their diagnosis and are not already taking lithium. Treatment will be for up to one year."
Lithium is a simple chemical substance approved for use in humans and prescribed as a mood stabilizer. It protects neurons in the brain in animal models of neurodegenerative diseases, including Alzheimer's disease and Parkinson's disease, and has been recently shown to do the same in a mouse model of ALS. In that study, lithium prolonged survival and protected cells in both the brain and spinal cord. While the exact mechanism of lithium's effect is unknown, researchers have proposed it promotes clearance of toxic protein accumulation.
The multi-site investigation will be a double-blind, placebo-controlled trial with 84 patients who will be randomized to either lithium or placebo. A review of data will occur after the 84th person is enrolled, and then a decision will be made on whether to expand the study to 250 patients. The disease course and safety assessments will be measured at regular intervals over that time.