Riluzole ‘a candidate treatment’ for cerebellar ataxia

Sep 9 2015

By Lucy Piper, Senior medwireNews Reporter

Riluzole may be an effective treatment for cerebellar ataxia, say researchers who call for further studies to confirm their findings.

The 1-year trial showed that patients with hereditary cerebellar ataxia were nearly 10 times more likely to have a treatment response with riluzole than with placebo, with an absolute risk difference of 39% and a number needed to treat of 2.6.

“Given the well known safety profile of riluzole and the need for new treatments for hereditary cerebellar ataxias, this trial might have potential implications for clinical practice, if further studies in larger and disease-specific populations support our findings”, say researcher Giovanni Ristori (Sapienza University of Rome, Italy) and co-workers.

The team randomly assigned 60 patients to receive riluzole – a small-conductance potassium channel opener – or placebo. Among the 55 who completed the study, 28 were assigned to riluzole and of these patients, 19 had spinocerebellar ataxia and nine had Friedreich’s ataxia. This compared with a respective 19 and eight patients among those assigned to placebo.

Patients in the riluzole group were a significant eight times more likely to have a decreased Scale for the Assessment and Rating of Ataxia (SARA) score at the end of the 12 months than those in the placebo group, at 50% versus 11%. A significant difference between the two groups was evident at 3 and 12 months and the proportion of patients taking riluzole with reduced SARA scores at 3 months remained stable at 12 months, whereas it declined among those taking placebo.

The clinical effects of riluzole were unaffected by adjustment for different forms of ataxia, age and gender, after which the odds ratio for treatment response was 9.76.

Post-hoc analyses also indicated significant benefits of riluzole over placebo on activities of daily living and quality of life at 12 months, with 32.1% of patients achieving a SARA score of 5.5 or lower (mild dependency in the performance of daily living) versus 7.4% and 60.0% versus 6.7% showing an improvement in at least four Short Form-36 dimensions.

There were no serious adverse events with the drug. Two patients had an increase in liver enzymes, which has been shown before with riluzole, but to no higher than two times above normal, while two patients from each group had sporadic mild adverse events.

“Overall, this trial lends support to the idea that riluzole might be efficacious in the treatment of patients with cerebellar ataxia, in addition to its present indication for amyotrophic lateral sclerosis”, says the team.

“The virtual absence of effective therapies for the inherited forms of cerebellar ataxia, which affect a large number of patients and at-risk individuals worldwide, warrants further investigation of riluzole as a candidate treatment”, they conclude in The Lancet Neurology.

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