Will Riluzole really be good for cerebellar ataxia?

This is just a quick post on a recent paper in Lancet Neurology which looked at the potential benefit of Riluzole in the treatment of cerebellar ataxia.

Neurologist know Riluzole very well. It is the only drug that has even the slightest hint of slowing down the dreadful motor neurone disease (MND). It is however fraught with side effects and demands a strict regime of regular blood tests to monitor for liver toxicity; this puts many people off it. Many neurologists are also sceptical of its reported benefits (in whispering tones of course).

It is therefore with some surprise for me to see a trial of Riluzole in another disease at all. Why Riluzole I wondered? The paper is titled Riluzole in patients with hereditary cerebellar ataxia: a randomised, double-blind, placebo-controlled trial. And it answers my question in the introduction- Riluzole just happened to be one of a bunch of drugs the authors had previously tried at random, and somehow the one that showed any hint of a benefit. Sounds strangely familiar, or is it just déjà vu?

 

The authors studied subjects with the genetic conditions spinocerebellar ataxia (SCA) and Friedreich’s ataxia (FA). They used the SARA scoring system to monitor for improvement in the study subjects. And their faith was rewarded; 50% of the subjects on Riluzole improved compared to only 11% of the subjects on placebo. The authors suggest further trials to confirm the benefit. The academically minded will pick apart the methodology and statistical significance; the neurologist on the coal face will hold on to any hope.