April 6, 2010

reaction to a Lancet Neurology paper on trial of lithium for MND

The paper reports on a trial in the US which has failed to replicate the initial success of lithium as a treatment for motor neurone disease.

Dr Brian Dickie, Director of Research Development at the Motor Neurone Disease Association, said:

“The US trial had a very different design to the UK lithium trial as it set out to look for a very big effect, a 40% improvement in the functional capability of patients, equivalent to that seen in the 2008 Italian study. The US trial was stopped because interim results indicated that the drug was not producing this significant level of improvement.

“The UK lithium trial has been designed differently as we have not set out to meet any predetermined level of improvement and will observe the effects of the drug over the course of the 18 month trial, therefore detecting any smaller but nonetheless important effects.

“We are prepared for the possibility that in a large, rigorous study the effect of lithium might prove to be less dramatic than that seen in the small Italian trial. We believe that to stop our UK trial at this stage would throw away the real possibility that lithium might still have a significant benefit in motor neurone disease, and that detecting that benefit will take longer than the few months exposure experienced by the American participants.”

“Although the UK trial may appear more conservative in its approach, both types of trial design are valuable. For example, a trial of the US size and design may not have picked up the subtle beneficial effects seen with riluzole, a drug which has been shown in more extensive trials to alter disease progression and is currently the only licensed treatment for motor neurone disease.”