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SUNY Upstate Medical University to study if lithium can slow progress of ALS in humans

SYRACUSE, N.Y. — 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 that we project will begin in January 2009," Shefner said. "As soon as we begin the study, 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.

The trial is being supported through the ALS Association's TREAT ALS (Translational Research Advancing Therapies for ALS) initiative, a drug discovery program and clinical trials process created by the association that accelerates discovery and testing of clinical candidates. In addition to SUNY Upstate, investigators from Massachusetts General Hospital, the University of Toronto and Columbia University will participate in the trial.

ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. When the motor neurons die, the ability of the brain to initiate and control muscle movement is lost, leading to progressive paralysis.

For more information about the study or to participate in the study, call 315-464-5004.