May 2025 Research Roundup
Research Roundup
May 2025
Learning from PBC
This month, I attended a meeting of PBC experts in Amsterdam who want to understand what evidence is required to show that drugs are effective and get market approval so they can be prescribed to patients. Why PBC? It’s similar to PSC but faces the same challenges, and the PBC community recently saw the approval for one of their treatments withdrawn. My goal was to learn from our colleagues in the PBC community to help ensure this does not happen to a PSC treatment. The meeting confirmed to me that we are on the right track with our strategy to help develop biomarkers for PSC, to support the development of tools to allow patients to report how they feel and function, and to collaborate widely.
Bile Duct Cancer Conference
I also attended the ‘Monothematic Conference Liquid Biopsy in Biliary Tract Cancer: from bench to bedside’. The main aim was to consolidate current knowledge and to draw a roadmap towards the adoption of simple tests as key tools to diagnose these cancers and personalise treatment. Thank you to everyone who completed our joint survey with PSC Partners on this topic. So important were the results that the organisers of the meeting allowed us to present the results at the meeting. Only around half of the 623 respondents in our survey had had a discussion about bile duct cancer with their doctor and only a third felt they had enough information on it. There are good reasons for this but with highly predictive tests for bile duct cancer getting closer to clinics, it’s time to change that.
Research YOU'VE funded
Since my last Research Round up in March, we’ve announced that £324,344 of your donations are funding three new research projects to understand pregnancy in PSC, to understand the symptom that affects most of us, fatigue, and to identify the patterns of changes that could be used as markers for the early diagnosis of bile duct cancer:
Dr Deepak Joshi
2024
Understanding how pregnancy may affect patients with PSC, and lead to better management of symptoms and appropriate monitoring during pregnancy.
Rodrigo Motta
2025
DNA Methylation in PSC and bile duct cancer.
Alice Freer
2025
Understanding fatigue in PSC.
International Liver Conference
As well as funding research, our time is spent supporting researchers and ensuring PSC patient voices are firmly embedded in PSC studies. The results of some of our work were presented at the annual international liver research conference of EASL where over 7,000 liver disease experts from 119 countries attended.
Simple Cholestatic Complaints Score
The first was research led by Dr Hussain (you may have seen him on our recent Ask the Expert webinar talking about itch). He has developed a new tool to quickly help assess the main PSC symptoms. It is called the SCCS (Simple Cholestatic Complaints Score) and can be used in clinical trials:
Social Determinants of Health
The second was research conducted by the ERN RARE-LIVER (the network of European rare liver disease experts), and led by Dr Stallbaum. Overall, the findings showed that various social factors appear to be associated with disease outcomes in people with autoimmune liver disease, emphasising the importance of reducing health inequities. Thank you to everyone who completed this survey:
Three Major PSC Announcements
There were three stand out announcements made during the EASL conference:
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- Research that PSC Support helped to fund has yielded positive results. Researchers in Spain have developed a test to detect early bile duct cancer. Although a small risk, it's a serious one and patients report it as one of the most feared complications of PSC. >>READ MORE
- A drug (called NCA) to treat PSC had very encouraging Phase 3 trial results. While there is still more testing to do to ensure it really is safe and effective, this is the closest a treatment for PSC has got to being approved for use. It has taken over 15 years of research to get to this stage. In 2010, Dr Roger Chapman, talking about NCA, said, and I quote, "It's the best we've got right now". How right he was! >>READ MORE
- A drug in an earlier stage of testing (Phase 2) also had positive results. The two drugs work in different ways, addressing different aspects of PSC, so it is good news. >>READ MORE
To everyone that has been on a clinical trial for PSC, we only get results like this because of you. Thank you.
If you are interested in going in a clinical trial for PSC, or want to see if you are eligible, don’t wait for your doctor to mention it (some never do) - check out our list of studies currently looking for people to take part.
Ask the Expert
If you'd like to know more about the NCA research, Dr Trivedi gave up his time last Saturday afternoon to answer our questions and you can watch the recording here.
Do drop me an email if you have any questions:
Martine Walmsley
PSC Support Head of Research Strategy
ERN RARE-LIVER Management Board and PSC Working Group Co-Lead