Tackling prion diseases: a review of the patent landscape


Expert Opinion on Therapeutic Patents


17 June, 2021


Zattoni M, Legname G

Introduction: Prion diseases are a class of rare and fatal neurodegenerative diseases for which no cure is currently available. They are characterized by conformational conversion of cellular prion protein (PrPC) into the disease associated "scrapie" isoform (PrPSc). Under an etiological point of view, prion diseases can be divided in acquired, genetic and idiopathic form, the latter of which are the most frequent.

Areas covered: Therapeutic approaches targeting prion diseases are based on the use of chemical and natural-based compounds, targeting either PrPC or PrPSc or other putative player in pathogenic mechanism. Other proposed anti-prion treatments include passive and active immunization strategies, peptides, aptamers and PrPC-directed RNA interference techniques. The treatment efficacy has been mainly assessed in cell lines or animal models of the disease testing their ability to reduce prion accumulation.

Expert opinion: The assessed strategies focus on the identification of an efficient anti-prion therapy faced various issues, which goes from permeation of the blood brain barrier to immunological tolerance of the host. Indeed the use of combinatory approaches, which could boost a synergistic anti-prion effect and lower potential side effects of single treatments may represent an extreme powerful and feasible way to tackle prion disease.