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Prion disease drug “ineffective”


A major study into the efficacy of a new treatment for human prion disease has revealed that it does not increase patients’ survival rates.

Human prion diseases include Creutzfeldt-Jakob disease (CJD) and can arise spontaneously, be inherited through a genetic mutation, or develop through infectious transmission.

The most common form is sporadic CJD, which affects between one and two people per million annually worldwide. All human prion diseases are fatal and there is currently no treatment to prevent or reverse their progression.

The latest study, published in The Lancet Neurology, was carried out after quinacrine (mepacrine) – a drug used to treat malaria and some arthritic illnesses – was shown to be effective in treating prion-infected mouse cells. It works by blocking the conversion of normal prion proteins into the abnormal disease-causing form.

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The PRION-1 trial, carried out by researchers from the MRC Prion Unit in London, found no significant difference in survival rates between patients who were given quinacrine and those who were not.

The authors also noted that quinacrine was associated with increased incidence of adverse events, although most were mild. Only two serious adverse events were considered as being connected with the drug.

Copyright © Press Association 2009

The Lancet Neurology

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