Lithium clinical trial results published

The results of a small Italian clinical trial of the drug lithium carbonate have recently been published in the journal Proceedings of the National Academy of Sciences.
During the 15 month trial, 16 people with MND took lithium carbonate in addition to riluzole. The progress of their disease was compared with that of 28 people taking rilzuole alone. The Italian researchers reported that the lithium carbonate significantly slowed the progression of MND.

Consider with caution

Whilst the results of this trial may be encouraging, they do need to be considered with some caution and should not be seen as conclusive evidence that lithium is an effective treatment for MND.
Sixteen people are an inadequate number in which to establish with confidence that a drug is beneficial. The methods used in the trial also had other significant weaknesses that make the results less reliable:

  • The participants in the trial knew whether they were taking the trial treatment or whether they were in the comparison group. This can produce bias in the way participants report how they feel and also means that the placebo effect can have an influence.
  • A larger proportion of the participants given lithium had disease characteristics (e.g. age and site of disease onset) that tend to be associated with slower disease progression.
  • The participants were not put into the treatment and comparison groups at random - they were chosen for each group by the researchers. Random selection is usually performed by a computer and prevents bias in choosing which patients go into each group.

In addition, adverse events (possible side effects) were not described by the researchers, which is extremely unusual for a clinical trial report. For more information on the procedures used in clinical trials, see Research Info Sheet D - Clinical trials (324 kb) .


More trials needed

Laboratory studies have shown that lithium can have a protective effect on motor neurones, and it has demonstrated positive effects in the mouse model of MND. The data from this research may well warrant further large-scale clinical trials of lithium carbonate in people with MND and the MND Association intends to discuss the issue further with leading researchers in the field. Such trials, if rigorously conducted, could eventually provide the evidence that is needed to prove whether or not the drug is effective.
However, until such trials are completed, lithium should not be regarded as a proven treatment. It is also important to remember that lithium carbonate is known to have side effects. If you have MND and are considering taking lithium now, the MND Association would encourage you to discuss the matter with your neurologist.