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Content archived on 2024-05-23

Integrated in vitro and in vivo testing of drugs in prion diseases: screening, development and mechanisms of novel therapeutics

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Drugs that cut cholesterol also halt prion diseases

Scientists have researched the possibility of a treatment for prion diseases based on anti-cholesterol drugs.

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Intense research into prion diseases has led the development of possible drug therapies down various pathways related to the pathogenesis involved. Based on this, the EU funded project STOPTSES formulated its objectives as the prevention of the development of the condition at key stages. Target events included the reduction of infectivity, inhibition of its replication in lymphoid tissue and prevention of the establishment of infection in the central nervous system. The infectious agent behind prion diseases is a protein with a fibrillar structure. Consequently, most research to date had concentrated directly on the disruption of the structure of the protein. Project partners based at the University of Glasgow however, investigated the potential of anti-cholesterol drugs as a means of treatment. Their aim was to determine the action of anti-cholesterol drugs in controlling or preventing the mechanisms by which the prion acts as an infective agent. Disruption of cellular trafficking was one possible mode of action. Also, the processing and formation of prions may be affected by anti-cholesterol drugs. Positive control of these functions was demonstrated in tissue culture models and infected cell lines. The team planned to extend their trials to in vivo models. An alternative therapeutic strategy has been explored that may have potential as a block to the development of prion diseases within the host.

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