Novel therapeutic challenges in cerebellar diseases

Abstract

In the last decade, substantial scientific progress has enabled a better understanding of the pathogenesis of cerebellar diseases and the improvement of their diagnoses. Extensive preclinical work is expanding the possibilities of using experimental models to analyze disease-specific mechanisms and to approach candidate therapeutic strategies to create a rationale for clinical trials that might finally lead to successful treatment. At present, drug treatment of cerebellar disorders has shown limited effectiveness and current treatment is primarily supportive. Until effective and selective pharmacological treatment leading to better quality of life as well as increased survival of patients with cerebellar diseases is found, physical and sensory rehabilitation techniques are revealing effective approaches for improving the patient’s quality of life. The objective of this chapter is to provide an updated summary of the treatments currently available for cerebellar disorders, in particular for spinocerebellar Ataxias, and to discuss the new emerging therapeutic strategies that are resulting from the intensive ongoing basic and translational research devoted to cerebellar diseases.