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LITHIUM IN ALS
by Jinsy Andrews, MD
Investigators in Italy at the University of Pisa
(Fornai et al, 2008) proposed that lithium promoted
autophagy which is a mechanism for digestion of cell
contents by enzymes within that cell. They believed this
process may be involved in ALS. Prolonged survival in
mouse models of ALS prompted the scientists to do a small
pilot study in ALS patients. They conducted a small study
with a total of 44 patients with early ALS. Sixteen were
randomized to receive lithium and riluzole and 28
continued to take riluzole only. At the end of 15 months,
none of the patients taking lithium died compared to
almost 30% of the patients taking riluzole alone.
These unprecedented results circulated quickly
among the ALS community and many internet forums such
as "Patients Like Me", "People with ALS (PALS)" and
others. Although these findings are dramatic and exciting,
many researchers are cautious because the number of
patients in the study were small and results can be easily
skewed by many different factors. Additionally, lithium has
significant side effects including seizures, tremors, fatigue,
and conginitive problems among others. The effect of the
medication can also be unpredictable when combined with
other medications, supplements, or other medical
conditions, especially if it is not properly monitored.
This study has prompted ALS researchers to initiate
formal clinical trials quickly. There are multicenter natural
history-controlled and randomized, placebo-controlled
trials are on the way. Our center has begun conducting a
small safety study of lithium and plan to be involved in the
larger trials planned.
Reference:
Fornai F, Longone P, Paparelli A, et al. Lithium delays
progression of amyotrophic lateral sclerosis. Proceedings of
the National Academy of Sciences 2008; 105: 2052-2057
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