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:
Improving Care: We attended a major national clinical conference to equip general gastroenterologists and nurses with specialist knowledge, which helps ensure future work is better aligned with what people with PSC need by improving local care and pathways.
Progressing Research: Our 2024 investment into early-stage research to build necessary scientific evidence enabled Dr James Sun to secure major national funding to explore the genetic drivers of the disease. This foundational knowledge is essential for identifying potential new treatments.
Progressing Research: We supported the development of a research manuscript on nutrition in PSC, so that the findings can be effectively shared with the wider medical community. This will help us advocate for the needs of people with PSC to ensure they get the care they need at the right time.
Improving Care: We hosted a clinical consensus meeting to map out an ideal PSC care pathway, which helps ensure future PSC management is better aligned with what people with PSC need. Establishing standard protocols across the NHS is essential for eliminating regional variations in care.
Progressing Research: We contributed to the strategic planning of an international rare liver disease network, which supports progress towards better care for people with PSC. Broadening our influence ensures patient needs are integrated into wider clinical strategies.
Improving Care: We shared firsthand experiences of living with PSC at an international medical school event, which improves clinical understanding of the disease for healthcare professionals. This is important because ensuring future specialists understand the human impact of PSC helps drive patient-centered care and clinical excellence.
Progressing Research: After just over 48 hours on our Big Give Small Charity Week match funding initiative, your donations helped us get to 78% of the way to our £20,000 goal! Thank you! Can we reach 100%?