Therapeutic strategies for prion disease: a practical perspective

Human prion diseases are usually rapid neurodegenerative illnesses that are invariably fatal. Despite several clinical trials, no effective treatment has been discovered in humans. Although prior clinical trials have not been successful, they provided information that is vital for the formation of future clinical trials. Among these findings is the realization that there are several prion disease-specific aspects that must be considered when conducting clinical trials. The rarity, rapidity, and clinical heterogeneity of prion disease affect study enrollment and the ability to measure treatment effects. In addition to affecting results, study methodology may also influence study enrollment. In this review, we explore several challenges to conducting clinical trials in prion disease and suggest some practical considerations.