An overview of current and future therapeutic strategies for the treatment of primary sclerosing cholangitis

Ahmad H. Ali, Elizabeth J. Carey, Keith D. Lindor

Research output: Contribution to journalReview articlepeer-review

2 Scopus citations


Introduction: Primary sclerosing cholangitis (PCS) is a cholestatic liver disease characterized by chronic destruction and stricturing of the biliary tree, leading to fibrosis and liver cirrhosis. The underlying mechanisms of the disease are poorly understood. Areas covered: Multiple agents with various pharmacological activities have been examined in PSC patients with disappointing results. Ursodeoxycholic acid (UDCA) is by far the most extensively studied agent in PSC. Large prospective trials have failed to demonstrate a positive long-term effect of UDCA in patients with PSC. Antimicrobials have been examined in PSC patients but long-term outcome data are lacking. Immunosuppressive agents were tested in patients with PSC but the majority of these agents were associated with adverse side effects. Nor-UDCA is a newly emerging C23 homolog of UDCA that has shown promising results in experimental studies. The farnesoid X receptor agonists are an attractive class of drugs and are currently being studied in patients with primary biliary cirrhosis. Antifibrotic agents have yielded encouraging results in animal models of PSC but are yet to be evaluated in humans. Expert opinion: Currently, there is no effective treatment for PSC. It is our hope that the newly discovered potential therapeutic routes will lead to the discovery of a curative agent for this debilitating disease.

Original languageEnglish (US)
Pages (from-to)545-556
Number of pages12
JournalExpert Opinion on Orphan Drugs
Issue number6
StatePublished - Jun 2014


  • Antimicrobial
  • Farnesoid x receptor
  • Primary sclerosing cholangitis
  • Ursodeoxycholic acid

ASJC Scopus subject areas

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


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