The Compelling Demand for an Effective PrPC-Directed Therapy against Prion Diseases

Andrea Astolfi; Giovanni Spagnolli; Emiliano Biasini; Maria Letizia Barreca

doi:10.1021/acsmedchemlett.0c00528

The Compelling Demand for an Effective PrP^C-Directed Therapy against Prion Diseases

ACS Med Chem Lett. 2020 Nov 2;11(11):2063-2067. doi: 10.1021/acsmedchemlett.0c00528. eCollection 2020 Nov 12.

Authors

Andrea Astolfi¹, Giovanni Spagnolli², Emiliano Biasini², Maria Letizia Barreca¹

Affiliations

¹ Department of Pharmaceutical Sciences, "Department of Excellence 2018-2022", University of Perugia, 06123 Perugia PG, Italy.
² Department of Cellular, Computational and Integrative Biology (CIBIO) and Dulbecco Telethon Institute, University of Trento, 38122 Trento TN, Italy.

Abstract

Decades of research efforts have conclusively provided overwhelming evidence that the cellular prion protein (PrP^C) plays a central role in prion diseases, a set of fatal and incurable neurodegenerative disorders for which no therapy is yet available. In this Viewpoint, we provide an overview of the drug discovery strategies in the field, highlighting the current therapeutic hypotheses targeting, whether directly or indirectly, PrP^C as well as the antiprion agents closest to clinical application.