Overview on Treatment of Prion Diseases and Decontamination of Prions

Abstract

Currently, there are no prophylactic or disease-modifying therapies for prion diseases with proven, significant efficacy. The discovery of treatments by design is hampered by incomplete understanding of prion disease pathogenesis. However, therapeutic considerations have broadly centered on a loss of function of the normal prion protein or possible toxicity of abnormal prion proteins. Potential treatments have been assessed by in vitro cell-free studies, cell-culture studies, in vivo animal experiments, and in human clinical trials. The last of these poses several problems including the rarity of prion diseases, variations in the rates of clinical progression, difficulties in measuring this clinical progress, and in the difficulty of early diagnosis at a time before significant neurological damage has already occurred. Given the transmissibility of prion diseases, one aspect of their prevention involves decontamination of potentially contaminated medical instruments. Unfortunately, prion infectivity is particularly difficult to remove or inactivate, with variations between different prion agent strains and methodological problems in the assessment of the effectiveness of any proposed method. The general principles underpinning prion disease treatment and decontamination are reviewed with reference to past research and current knowledge.