Abstract
Converging experimental data suggested attempts of using in human beings modulator of ion channels to counteract the neurodegenerative process in progressive cerebellar ataxia (CA). The availability of riluzole, which is already approved for use in clinical practice, with a good safety profile, prompted a repurposing approach.
Two trials supported the attempt to repurpose riluzole in CA. Its symptomatic action (and possibly its slowing effect on disease progression) suggested to consider riluzole in clinical practice as a general front-line therapy, while the diagnostic process is ongoing, as well as an add-on therapy in forms with etiologic treatment.
The research on the possible use of riluzole in CA is currently a hot topic: a precursor of riluzole, troriluzole, is now under scrutiny in two phase 3 trials; in patients with spinocerebellar ataxia type 2 another phase 3 trial is ongoing; a pilot trial is currently in progress in patients with spinocerebellar ataxia type 7.
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Romano, S. et al. (2023). Riluzole in Progressive Cerebellar Ataxias. In: Soong, Bw., Manto, M., Brice, A., Pulst, S.M. (eds) Trials for Cerebellar Ataxias. Contemporary Clinical Neuroscience. Springer, Cham. https://doi.org/10.1007/978-3-031-24345-5_14
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DOI: https://doi.org/10.1007/978-3-031-24345-5_14
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