Evaluation of: Miller RG, Forshew D, Katz J et al. Phase II controlled trial of lithium carbonate in patients with amyotrophic lateral sclerosis. Neurology 77(10), 973–979 (2011).
Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder; riluzole is marginally effective, and as a consequence a large number of trials are regularly reported. Lithium raised marked enthusiasm based on the report of a pilot study that suggested very positive results. Two previous trials were negative, applying a different methodology. The reviewed article reports the results of a third trial using a historical control group. In this trial, lithium was detrimental to ALS progression. Two more trials testing lithium in ALS are in progress. This study is discussed in the context of the great competitive effort that was derived from the unsupported hope created by a false-positive preliminary study.