Insights from therapeutic studies for PrP prion disease

Kenta Teruya, Katsumi Doh-Ura

Research output: Contribution to journalArticlepeer-review

30 Citations (Scopus)


Although an effective therapy for prion disease has not yet been established, many advances have been made toward understanding its pathogenesis, which has facilitated research into therapeutics for the disease. Several compounds, including flupirtine, quinacrine, pentosan polysulfate, and doxycycline, have recently been used on a trial basis for patients with prion disease. Concomitantly, several lead antiprion compounds, including compound B (compB), IND series, and anle138b, have been discovered. However, clinical trials are still far from yielding significantly beneficial results, and the findings of lead compound studies in animals have highlighted new challenges. These efforts have highlighted areas that need improvement or further exploration to achieve more effective therapies. In this work, we review recent advances in prion-related therapeutic research and discuss basic scientific issues to be resolved for meaningful medical intervention of prion disease.

Original languageEnglish
Article numbera024430
JournalCold Spring Harbor Perspectives in Medicine
Issue number3
Publication statusPublished - 2017 Mar


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