Emerging pharmacotherapies for Creutzfeldt-Jakob disease.

Only a few years ago, pharmacotherapy of Creutzfeldt-Jakob disease was inconceivable. The enigmatic prion agent causing Creutzfeldt-Jakob disease, consisting solely of a misfolded conformational isoform, the scrapie prion protein, of the normal cellular prion protein was considered hard to treat by routine drug development. However, huge progress has been achieved in recent years, demonstrating principal reversibility of the neuropathological features and protection from clinical symptoms in animal models and introducing potential pharmaceutical agents. Among the most promising ones, antibodies have been shown to be protective against prion disease and heterocyclic small-molecule compounds have been proposed as antiprion lead compounds, initiating clinical trials.