Molecular Signatures Identified to Predict Primary Sclerosing Cholangitis Progression and Cancer Risk
Molecular Signatures Identified to Predict PSC Progression and Cancer Risk
Dr Ghada Nouairia
How can PSC progression be predicted?
Predicting the course of primary sclerosing cholangitis (PSC) is a major clinical challenge because the disease varies significantly between individuals. Some people may develop severe liver damage, while others face an increased risk of bile duct cancer (CCA), and others face barely any disease progression at all. Researchers are urgently seeking new, non-invasive tools, known as biomarkers, that can provide a clearer and more timely prognosis than current methods. Dr Nouairia’s study was designed to use advanced data analysis to uncover new biological clues.
What did the research team analyse?
The study analysed blood samples from 33 people with and without PSC, measuring three specific types of circulating molecules: microRNAs, proteins, and metabolites. By applying advanced data analysis and machine-learning methods, the research team has successfully identified distinct molecular "signatures" linked to different presentations, or phenotypes, of the disease. This approach allows for a deeper understanding of the subtle biological differences in a condition known for its varied progression.
What are the key findings?
The results represent a crucial step towards developing diagnostic tools that could ultimately help predict disease progression and identify individuals at a higher risk of complications.
Key findings include:
- Each PSC phenotype has its own unique set of molecules that may serve as future biomarkers for disease progression and risk prediction of CCA.
- The machine learning approach could pick up subtle molecular differences between PSC with and without inflammatory bowel disease (IBD), even though they appear very similar biologically.
- Severe PSC and PSC that progresses to CCA were found to share few molecular features, suggesting possible distinct early markers for cancer risk.
What is the impact for people with PSC?
These promising findings move us closer to a future of more personalised follow-up for people living with PSC. If blood-based molecules can reliably predict who is at higher risk of cancer or severe progression, healthcare professionals could tailor monitoring and treatment strategies more effectively. PSC Support is proud to have funded this work, which directly aligns with our mission to unlock understanding and accelerate the development of effective treatments for the PSC community.
Turning Ambition into Action in 2026:
Our Fortnight in Focus
At PSC Support, we want a world without PSC. We work tirelessly behind the scenes to drive research and improve lives. Here is a snapshot of what we've been up to:
Organisational excellence: We simplified our personal data consent process and streamlined how we record patient stories to ensure that the lived experiences of our community are captured accurately and securely to inform future work.
Progressing Research: We coordinated a series of upcoming meetings with international researchers and pharmaceutical companies, which supports better informed decisions for people with PSC, research, or care. This groundwork helps keep PSC research focused on the primary needs of the patient community.
Progressing Research: We reviewed and contributed to scientific abstracts and posters for the upcoming EASL international liver congress, which strengthens the quality of future research for people with PSC, research, or care.
Progressing Research: We engaged with the MHRA to prepare for the upcoming national consultation on rare disease therapies, representing PSC patients in the wider community. Maintaining this direct dialogue ensures our organisation is prepared to respond formally and protect the interests of those living with PSC
Improving Care: We presented about what it is really like to live with PSC to gastroenterologists This first-hand insight equips healthcare professionals to better understand the day-to-day challenges of PSC.
Income Generation: We secured a place in the prestigious Big Give Small Charity Week match-funding campaign, so that we can maximise future donations from our supporters as every donation between 22 and 29 June will be doubled!
Progressing Research: We recruiting for a research and advocacy officer to expand our core team. This additional capacity ensures we can better support scientific studies and represent the patient voice at every level.