Emerging therapeutic targets for primary sclerosing cholangitis

Amir Kalani, James H. Tabibian, Keith Lindor

Research output: Contribution to journalReview articlepeer-review


Introduction: Primary sclerosing cholangitis (PSC) is a chronic, cholestatic liver disease that can progress to end-stage cirrhotic liver disease and/or hepatobiliary malignancy. The etiopathogenesis of PSC remains unknown, and considerable heterogeneity is seen both phenotypically as well as genetically; this, together with its relative rarity and the lack of consensus regarding appropriate (surrogate) clinical endpoints, has hampered the development and implementation of effective pharmacologic therapies. Several different disease mechanisms have been proposed which have led to various potential therapeutic approaches. However, to date, no medical therapies short of liver transplantation have been proven to be effective for PSC, and as such, liver transplantation–an option for only a fraction of patients–remains the only potentially curative therapy. Areas covered: This review will highlight the various different proposed mechanisms of the etiopathogenesis of PSC and explore the therapeutic targets corresponding to them which are anticipated to be or are already under clinical investigation. Expert commentary: The development of new therapies for PSC remains challenging due to numerous factors, not the least of which are the rare and pathobiologically enigmatic nature of the disease. However, with recent advances in the understanding of potential mechanistic underpinnings and lateral insights from other liver diseases, new potential targets and novel therapeutic agents are being evaluated; we believe these have the potential to lead to the establishment of safe and effective medical therapies and thus better outcomes in PSC.

Original languageEnglish (US)
Pages (from-to)393-401
Number of pages9
JournalExpert Opinion on Orphan Drugs
Issue number7
StatePublished - Jul 3 2018


  • bile acids
  • Biliary tract disease
  • drug therapy
  • endoscopy therapy
  • survival

ASJC Scopus subject areas

  • Pharmacology, Toxicology and Pharmaceutics (miscellaneous)
  • Health Policy
  • Pharmacology (medical)


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