Therapeutic approaches to prion diseases

The prion diseases are neurodegenerative disorders of humans and animals that are sporadic or inherited in origin and can be transmitted. A number of drugs have been tested in an attempt to find an effective treatment for prion diseases, but only a few classes of compounds have had any effect. The need for therapeutics is becoming increasingly urgent because of the emergence of a new variant of Creutzfeldt-Jakob disease (vCJD), which is probably the result of human exposure to bovine spongiform encephalopathy-infected material. Since 1995, more than 130 vCJD patients have been identified in Europe, and there is concern about a future outbreak of this condition. This article presents data on the older and more recent drugs that have shown some effectiveness in vivo or in vitro, with particular focus on their putative molecular mechanism of action.