Stratified medicine in Primary Biliary Cholangitis

Liver disease research in Newcastle has identified areas of unmet need in PBC; in particular, sub-optimal response to established therapies.

Primary Biliary Cholangitis (PBC) is a chronic liver disease affecting 20,000 patients in the UK. Work performed in the UK, funded in part by the NIHR Newcastle Biomedical Research Centre (to support the liver disease research), has demonstrated important areas of unmet need in this condition; in particular, associated with sub-optimal response to established therapies, particularly in younger patients.

This work has enabled the birth of the unique UK-PBC research platform, which involves collaboration between academic centres across the UK, patient groups (the PBC Foundation and LIVErNORTH) and 11 industrial partners.

The UK-PBC Project is funded by the Medical Research Council, the NIHR Newcastle Biomedical Research Centre and the NIHR Rare Diseases Gastrointestinal TRC. It was established to develop unique patient cohorts with PBC and to allow deep phenotyping of individuals in order to understand the mechanisms underpinning both aggressive disease and symptomatic disease. It also allowed the development of an experimental and trials platform to evaluate and implement stratified medicine in this disease. We have currently phenotyped over 4000 patients.

Research of this kind allows us to understand why some patients with PBC are unresponsive to therapy and develop significant symptoms, and what therapies may be appropriate and effective at treating these individuals.

The project aimed to improve outcomes for PBC patients; both lengthening their life and improving the quality of life. It intended to provide unique opportunities for industry, for which clinical trial activity is already ongoing in the UK.