Single-blind, placebo-controlled pilot study of pregabalin for ataxia in cortical cerebellar atrophy

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Abstract
To preliminarily compare the efficacy of pregabalin with that of placebo on the cerebellar signs caused by cortical cerebellar atrophy (CCA). A deficiency of gamma-aminobutyric acid (GABA) has been described in the cerebellum in CCA, and pregabalin has been shown to enhance GABA release in rat hippocampus. Two consecutive patients with clinical diagnoses of CCA took part in the study. A placebo and pregabalin, 225 mg per day, were administered in a single-blind scheme during 15 day periods to every patient; cerebellar function was evaluated with the Scale for the Assessment and Rating of Ataxia (SARA) at the end of each period. A video recording of the SARA items performed by the first patient accompanies this article. Total SARA scores of 19 and 15 were obtained for the patients after placebo administration. The SARA scores decreased to 11 and 8, respectively, with the administration of pregabalin; an important amelioration of the ataxia was also evident. Both patients preferred continuing treatment with pregabalin when the trial was over. Pregabalin was superior to placebo in the improvement of the cerebellar signs caused by CCA. Further studies are needed to confirm the present results.