An update on novel pharmacological agents for primary sclerosing cholangitis

Camilla Gallo, Bright Oworae Howardson, Laura Cristoferi, Marco Carbone, M. Eric Gershwin, Pietro Invernizzi

Research output: Contribution to journalReview articlepeer-review


Introduction: Primary sclerosing cholangitis (PSC) is a rare chronic cholestatic disease with heterogeneous phenotypes that may lead to liver transplantation and/or end-stage liver disease. Its multifactorial etiopathogenesis remains uncertain, but gut–liver axis and bile composition and excretion are widely demonstrated to influence the immune-mediated fibrogenic reactive cascade. Areas covered: Different experimental therapeutic options are under investigation, mainly aiming at modulating bile acids excretion, limiting inflammatory-cascade reactions, and changing intestinal microbiota composition; none of them yet demonstrated to prolong transplant-free survival. This review provides a comprehensive description of the experimental drugs recently tested and/or currently under investigation. A bibliographical search was performed in PubMed, MEDLINE, EMBASE, OVID, and until July 2021. Expert opinion: The heterogeneity and poor prevalence of PSC, its uncertain pathophysiology, and the lack of surrogate endpoints are the major challenges in drug discovery. Strategies that synergistically target microbiota, bile acids, and liver fibrosis are needed. In parallel, we must enhance biomarkers discovery to develop surrogate endpoints, as biochemical markers’ fluctuations over the time hamper their effectiveness. Magnetic resonance cholangiopancreatography tools that accurately measure bile duct changes represent a potential marker for disease monitoring. A collaboration between academia, research consortia, patient’s associations, and industry is required.

Original languageEnglish (US)
Pages (from-to)69-77
Number of pages9
JournalExpert Opinion on Therapeutic Targets
Issue number1
StatePublished - 2022
Externally publishedYes


  • anti-inflammatory agents
  • antibiotics
  • bile acids
  • fecal microbiota transplantation
  • gut–liver axis
  • Primary sclerosing cholangitis

ASJC Scopus subject areas

  • Molecular Medicine
  • Pharmacology
  • Drug Discovery
  • Clinical Biochemistry


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