Deep Brain Stimulation for Cerebellar Ataxia: A Systematic Review on Indications, Targets and Outcomes

Background. Cerebellar ataxias are a heterogeneous group of disorders characterized by progressive impairment of coordination, gait and balance, for which effective symptomatic treatments remain limited. Abnormal activity within the deep cerebellar nuclei, secondary to cerebellar cortical degeneration, represents a key pathophysiological mechanism. Deep Brain Stimulation has therefore emerged as a potential therapeutic strategy to modulate cerebellar output pathways. Objective. To systematically review the available clinical and preclinical evidence on the efficacy, safety, and stimulation targets of Deep Brain Stimulation for cerebellar ataxia. Methods. A systematic search of MEDLINE (PubMed) and Scopus was conducted in accordance with PRISMA 2020 guidelines. Studies involving human subjects with cerebellar ataxia treated with Deep Brain Stimulation were included, alongside relevant preclinical animal studies. Data on ataxia aetiology, stimulation targets, clinical outcomes and adverse effects were extracted and qualitatively analyzed. Results. Fifteen clinical studies (27 patients) and seven preclinical studies were included. The Dentate Nucleus was the most frequently targeted structure in human studies and was associated with modest but clinically meaningful improvements in ataxic symptoms, particularly in selected hereditary and post-lesional forms. Tremor-dominant phenotypes benefited consistently from thalamic stimulation. Preclinical studies highlighted the Interposed Nucleus as a promising novel target, demonstrating robust restoration of motor coordination and cerebello-thalamo-cortical signaling in animal models. Conclusions. Deep Brain Stimulation appears to be a feasible and potentially effective therapeutic option for selected patients with cerebellar ataxia. While current clinical evidence remains limited and heterogeneous, converging data suggest that modulation of cerebellar nuclei—particularly the Dentate Nucleus and the Interposed Nucleus—represents a promising avenue for future translational research.