Therapeutic Approaches for Prion Diseases

Although there are no known ways to cure transmissible spongiform encephalopathy (TSE) diseases, scientists around the world are working to develop treatments. Using infected tissue culture cells for fast initial screening, NIAID researchers have tested thousands of compounds and identified hundreds of molecules that inhibit the formation of the abnormal form of prion protein. Further testing of the most potent of these inhibitors has revealed several that can prolong the lives of rodents if treatments begin soon after infection.

In collaboration with NIAID scientists and through a contract with NIAID, researchers at the Institute's Rocky Mountain Laboratories (RML) and at Utah State University in Logan are testing these compounds in animals. Other groups are further testing two of the inhibitors in Creutzfeldt-Jakob disease (CJD) patients. New research at RML has shown increased survival times in mice given combination drug treatment therapy 14 to 28 days after scrapie inoculation. Although this will need further exploration, it is a positive step in treatment after the onset of clinical signs of TSE diseases.

RML researchers also have identified antibodies and short synthetic protein molecules (fragments of prion protein) that can block the conversion of normal prion protein to the abnormal form. If successful, these investigations will lead to safe and effective methods to prevent prion infections as well as therapies that work in either the pre-symptomatic or symptomatic phases of disease.​

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