Abstract

Every year, Alzheimer’s, Parkinson’s and Creutzfeldt-Jakob disease kill or cripple millions of people worldwide. We adopted a very different approach to developing treatments for these diseases. Our goal is to use drugs approved by the FDA for treating other diseases alone or in combination for the treatment of prion diseases whenever possible. This must occur in concert with creating tests for early diagnosis. We hope to be able to detect the disease process long before the brain is severely damaged. We plan to perform studies in humans and genetically engineered mice that faithfully recapitulate prion disease in humans. Optimizing drugs and other compounds that inhibit prion formation or enhance prion clearance in cultured cells followed by studies in mice should lead to effective therapies in the near future. Because these are uniformly fatal diseases, any therapeutic breakthrough in laboratory animals will find rapid application in people.