Emerging pharmacologic therapies for primary sclerosing cholangitis

Curr Opin Gastroenterol. 2017 May;33(3):149-157. doi: 10.1097/MOG.0000000000000352.

Abstract

Purpose of review: The only currently approved treatment for primary sclerosing cholangitis (PSC) is liver transplantation, with a median time to transplant of 12-18 years after diagnosis. There are a number of emerging drugs that have the potential to meet this critically unmet need that will be summarized and discussed herein.

Recent findings: Although the cause of PSC is unknown, there are a number of novel therapeutics under development. These drugs target presumed pathogenic mechanisms largely extrapolated from ex-vivo and in-vivo preclinical models, as well as translational observations.

Summary: Future therapeutic strategies for PSC may include a multitude of complex pathogenic mechanisms encompassing pathways of immunomodulation, the microbiome and inflammation-related fibrosis.

Publication types

  • Review

MeSH terms

  • Anti-Bacterial Agents / therapeutic use
  • Chenodeoxycholic Acid / analogs & derivatives
  • Chenodeoxycholic Acid / therapeutic use
  • Cholangitis, Sclerosing / drug therapy*
  • Cholangitis, Sclerosing / microbiology
  • Clinical Trials as Topic / methods
  • Fecal Microbiota Transplantation
  • Fibric Acids / therapeutic use
  • Gastrointestinal Microbiome / drug effects
  • Humans
  • Immunologic Factors / therapeutic use
  • Molecular Targeted Therapy / methods*
  • Molecular Targeted Therapy / trends
  • Organic Anion Transporters, Sodium-Dependent / antagonists & inhibitors
  • Probiotics / therapeutic use
  • Symporters / antagonists & inhibitors
  • Ursodeoxycholic Acid / therapeutic use

Substances

  • Anti-Bacterial Agents
  • Fibric Acids
  • Immunologic Factors
  • Organic Anion Transporters, Sodium-Dependent
  • Symporters
  • obeticholic acid
  • Chenodeoxycholic Acid
  • sodium-bile acid cotransporter
  • Ursodeoxycholic Acid