March 2025 Research Roundup
Research Roundup
March 2025
March has been a positive month at PSC Support: we've seen exciting clinical trial results, announced another new research project funded by your donations, seen our quality of life research enter its next phase (and is seeking volunteers right now), have new surveys on the go so we can share your views and influence research and are working with the UK medicines regulator and rare disease research experts to pave the way for PSC treatments.
If you are interested in helping with some of our research activities, please consider joining our amazing Patient Panel, a pool of people affected by PSC interested in PSC research.
Aspirin May Stop Some Cancers Spreading
Research published in Nature showed that aspirin may stop some cancers from spreading. This makes our Asp-PSC clinical trial even more important than ever. I don't think this is a good enough reason to rush out to buy some to take, but if it does cross your mind, it is worth looking into the Asp-PSC study, where you would be have close medical supervision.
Our Latest Research
I’m delighted to share that we have awarded £51,060 to a leading expert on PSC in Sweden, Professor Annika Bergquist, to help fund her study on how an existing drug called Simvastatin (a type of drug used to lower cholesterol, known as a “statin”) could be used as a treatment for people with PSC.
2025 Research Grants
Talking of research funding, if you are a PSC researcher, we’ve extended the deadline to submit a Letter of Intent for a 2025 grant from PSC Support.
We’re setting up a database to reduce the administration involved in reviewing and managing grant applications and we’d like up and running before we receive full grant applications.
Rare Disease Research UK Annual Conference
Research is hard. Rare disease research is even harder! Last week I attended the Rare Disease Research UK (RDR UK) Annual Conference in Manchester. I emphasised the value of high quality registry data on a discussion panel and there were many other fruitful discussions throughout the day.
Of note, we considered how to build capacity in specialist rare disease care and pave the way for more specialist doctors and researchers (we need more specialists), and discussed ways RDR UK can make rare disease research easier to do in the UK. RDR UK brings together leaders in rare disease research and I hope this collective expertise can accelerate the development of treatments for PSC and other rare diseases.
Latest PSC Surveys
Surveys are a bit like buses: they all come at once or you see none for months, but they are always important. We use survey results to improve care, influence research topics and help secure research funding (such as FARGO and Asp-PSC). Check out our current live surveys.
We've been working with PSC Partners to develop a quick survey to get your views on bile duct cancer and monitoring. We’re attending a conference of cancer experts in May and will stress the urgent need for early, accurate detection. Your views will help strengthen our case. If you’ve got some time, please take a few moments to tell us. The survey will be open for 2 weeks.
Clinical Trials and Studies
Promising news...
We’ve been working with the body that regulates medicines in the UK for many years as a member of the MHRA Patient and Public Community. I had a 1-1 meeting with the MHRA this month to discuss PSC clinical trials in the UK. Why?
- We're talking to several pharmaceutical companies about ways we can support potential new clinical trials in PSC, and I hope they will be brought to the UK. For example, last week Chemomab reported more encouraging results from the extension phase of their recent SPRING Trial.
- We're currently eagerly awaiting the results of Phase 3 research* into a drug called norUrso. If those results are positive, the medicine must pass further evaluation of its risks, benefits and costs before it can be prescribed. This takes time, and the patient voice is critical here, so we must be as prepared as possible. As I said, this is only IF there are positive results; we simply don’t know what they’ll be yet, but we’re expecting them soon.
*More about the phases of trials here
Quality of Life in PSC Study
Those of you who have had PSC for a while may recall that a research study began in 2016/17, funded by the PSC Support and British Liver Trust. The study aimed to develop a scientific questionnaire to properly assess the quality of life in people with PSC. We’re now recruiting volunteers for the next stage of this important research.
Why did we start this research? Because there was a disconnect between what patients were saying about PSC and how the medical and research community viewed it.
Patients said that the uncertainty and unpredictability of PSC was hard, dealing with symptoms was hard, living with the risk of cancer was hard and living with an incurable disease was hard (spot the theme?) while our doctors were focusing on physical test results such as blood tests and scans.
It was clear that if we were to improve care for PSC, we needed to understand the true burden of living with the disease. And to do that, we needed to ask the right questions - so the idea of a PSC quality of life measure was born. We also carried out research looking at Wellbeing in PSC and since then these results and those from our surveys have been used many times to secure millions of pounds for PSC research towards developing a treatment and improving quality of life.
Special thanks to PSC Support Patient Panel members who have been helping ensure that Ryan’s study documentation is clear and makes sense.
If you’d like to take part in the quality of life study, or find out more, click here.
Do drop me an email if you have any questions:
Martine Walmsley, PSC Support Head of Research Strategy martine@pscsupport.org.uk