Trial of Valproic Acid in Patients With Progressive Supranuclear Palsy (Depakine)
Status:
Completed
Trial end date:
2010-07-01
Target enrollment:
Participant gender:
Summary
Progressive Supranuclear Palsy (PSP) is a relentlessly progressive neurodegenerative
disorder, clinically characterized by parkinsonism with prominent axial involvement and
postural instability, bulbar symptoms, supranuclear ophthalmoplegia, and executive
dysfunction. Abnormal neuronal and glial tau aggregations affecting the basal ganglia and
selective brainstem structures result in dysfunction of the five frontosubcortical circuits
and brainstem functions. There is no effective treatment for PSP. One of the key feature in
the aggregation of tau is its phosphorylation by kinases such as glycogen synthase kinase 3b
(GSK3b). Recent reports have shown that valproic acid was able to inhibit the activity of
GSK3b and could exert a neuroprotective effect through this inhibition. The investigators
thus decided to conduct this controlled study to assess the putative neuroprotective effects
in patients with PSP.