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Expert Rev Neurother. 2011 Dec;11(12):1693-8. doi: 10.1586/ern.11.164.

Lithium treatment in amyotrophic lateral sclerosis: do we have enough trials?

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  • 1Neuromuscular Unit, Instituto de Medicina Molecular, Faculty of Lisbon, Portugal.


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.


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