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Novel therapeutic targets in primary biliary cirrhosis

Lookup NU author(s): Dr Jess Dyson, Professor Derek Mann, Professor David Jones


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Primary biliary cirrhosis (PBC) is a chronic immune-mediated liver disease characterized by progressive cholestasis, biliary fibrosis and eventually cirrhosis. It results in characteristic symptoms with marked effects on life quality. The advent of large patient cohorts has challenged the view of PBC as a benign condition treated effectively by the single licensed therapy-ursodeoxycholic acid ( UDCA). UDCA nonresponse or under-response has a major bearing on outcome, substantially increasing the likelihood that liver transplantation will be required or that patients will die of the disease. In patients with high-risk, treatment-unresponsive or highly symptomatic disease the need for new treatment approaches is clear. Evolution in our understanding of disease mechanisms is rapidly leading to the advent of new and re-purposed therapeutic agents targeting key processes. Notable opportunities are offered by targeting what could be considered as the 'upstream' immune response, 'midstream' biliary injury and 'downstream' fibrotic processes. Combination therapy targeting several pathways or the development of novel agents addressing multiple components of the disease pathway might be required. Ultimately, PBC therapeutics will require a stratified approach to be adopted in practice. This Review provides a current perspective on potential approaches to PBC treatment, and highlights the challenges faced in evaluating and implementing those treatments.

Publication metadata

Author(s): Dyson JK, Hirschfield GM, Adams DH, Beuers U, Mann DA, Lindor KD, Jones DEJ

Publication type: Review

Publication status: Published

Journal: Nature Reviews Gastroenterology & Hepatology

Year: 2015

Volume: 12

Issue: 3

Pages: 147-158

Print publication date: 01/03/2015

Online publication date: 03/02/2015

Acceptance date: 01/01/1900

ISSN (print): 1759-5045

ISSN (electronic): 1759-5053



DOI: 10.1038/nrgastro.2015.12