Currently there are no known treatments for Prion diseases, and usually prion diseases are fatal. This paper written by researchers at UCSF (including Stanley Prusiner) titled “2-Aminothiazoles as Therapeutic Leads for Prion Diseases” demonstrates that a small molecule drug class called 2-aminothiazoles might have potential antiprion activity.
Prion diseases happen when a protein is misprocessed, folds into a non-native form, and becomes toxic to the body or is deposited in fatal places. The mechanism of the transformation of the protein into a beta-sheet rich form is still unknown. This paper highlights some of the problems researchers have had in the past in developing therapeutics for prion disease. One example is that most compounds cannot cross the blood-brain barrier and so they do not have activity against brain disease.
From experiments, the researchers suggest that aminothiazoles most likely do not diminish expression of the normal form of the prior protein or denature a pathogenic prion that has been formed. Based on this result, they conclude that the most probabilistic mechanism of inhibition is by inhibiting prion formation to begin with. Additionally, high concentrations of improved analogs were able to reach the brain when they tested the aminothiazole in mice. Lots of future work is needed to determine the exact pathway of action of these drugs and determine how effectively they can reduce symptoms.