PROSPECTS FOR THE PHARMACOLOGICAL TREATMENT OF HUMAN PRION DISEASES

There is currently no effective therapy available for Creutzfeldt-Jako b disease and related prion disorders. However, a limited number of dr ugs have been found to affect the course of experimental prion disease s and to modify the kinetics of abnormal prion protein accumulation in the CNS. These include polyanions, the amyloid-binding dye Congo red, amphotericin B and its derivatives and, more recently, anthracyclines . At present, the most promising agent appears to be the amphotericin B derivative MS-8209. As a result of its wide spectrum of anti scrapie activity and efficacy in early and late stages of the incubation peri od of the disease in experimental prion models, MS-8209 could be used as a pharmacological tool to contribute to our understanding of the pa thogenic mechanisms involved in these neurodegenerative disorders and to afford a new and valuable base for future therapeutic strategies.