UK-PBC began in 2007 as a national effort to establish a large cohort of high-throughput genetic studies. Since 2008 the project has been on the portfolio of the National Institute for Health Research Comprehensive Research Network. UK-PBC Stratified Medicine is an exciting new project aimed at transforming the capacity to treat Primary Biliary Cirrhosis (PBC) and to establish a national trial resource.
The study consists of three workstrands:
Workstrand 1 – Recruitment, Phenotyping and Disease Stratification
Led by Dr Richard Sandford at the University of Cambridge, will work with the PBC Foundation to recruit patients via the UK-PBC consortium, a research network consisting of over 170 NHS Trusts throughout the UK. It will increase the size of and stratify the existing clinical cohort and to create a nested cohort of patients of high risk and non-responsive to UDCA.
Workstrand 2 – Mechanisms of UDCA Non-Response
Led by Professor John Kirby at Newcastle University, will answer key questions relating to the recovery of stressed biliary epithelial cells by therapeutic normalisation of bile acid with UDCA, why this might fail in UDCA non-response, and what implications this holds for how might treat non-responding patients either through informed re-use of existing agents or through new agent development.
Workstrand 3 – The User Interface
Led by Dr Gideon Hirschfield at the University of Birmingham, will address the need for UK-PBC to ensure strong and effective partnerships between patients, clinicians, industry and the NHS. It seeks to future-proof our unique research platform, and ensure long-term value from UK-PBC, by enabling better understanding of new treatments and by laying the foundations for implementing the first ever national standard-of-care for patients with PBC.