The ALS Foundation is funding a major clinical trial to see if lithium can slow disease progression in patients with early-stage Amyotrophic Lateral Slerosi, or ALS, commonly called Lou Gehrig’s Disease.

Lithium is a simple chemical substance approved for use in humans and prescribed as a mood stabilizer. Lithium 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.

A study in a small number of ALS patients compared lithium plus riluzole (an approved treatment for ALS) to riluzole alone. After 15 months, no deaths occurred in the 16 participants treated with lithium plus riluzole, while 29% of those taking riluzole alone had died. Patients on lithium also had a markedly lower decline in disease-related disability.

However, the trial was limited by the small numbers of patients and the absence of a placebo treatment ─ both the patient and the doctor knew whether the patient was taking lithium or not, which may have influenced the response to treatment or interpretation of disability.

The new trial 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 to 250 patients.

The trial is being supported through The Association’s TREAT ALS (Translational Research Advancing Therapies for ALS) initiative, and will use the TREAT ALS/NEALS Clinical Trials Network. Lead investigators for the study will be Merit Cudkowicz, M.D., and Swati Aggarwal, M.D., of Massachusetts General Hospital, Lorne Zinman, M.D., from the University of Toronto, Petra Kaufman, M.D., from Columbia University, and Jeremy Shefner, M.D., of SUNY Upstate Medical University.