Therapeutic possibilities in CJD: patents 1996 - 1999

Abstract

Currently, there is no effective treatment for Creutzfeldt-Jakob disease (CJD). However, an increasing understanding of the underlying disease mechanisms has suggested a number of therapeutic possibilities, many of which are registered as patents. The fundamental disease mechanisms in CJD are the conversion of PrP^Cto PrP^Sc, and the aggregation and deposition of PrP^Sc-related amyloid. The therapeutic proposals can be grouped together under three main headings: reducing the production of PrP^C;prevention of the post-translational alteration of PrP^Sc; prevention of neurotoxicity of PrP^Sc and amyloid deposits. The scientific background to these proposals is outlined and their claims are critically reviewed. In general, the treatment suggestions are based on theory, in vitro experiment and animal model data, with little, if any, definitive evidence of efficacy or safety in human disease.

• amyloid
• Creutzfeldt-Jakob disease
• prion protein
• prnp gene
• treatment