June 2025 Research Roundup

30/06/2025

Research Roundup

June 2025

I’ve been volunteering with PSC Support from the moment I understood there was no cure for PSC, 18 years ago when I was diagnosed with the disease. Now, I’m lucky enough to be employed by the charity to focus solely on research. Or more specifically, to help researchers find an effective treatment for everyone with PSC, as soon as possible. This month, I’d like to focus on some of those activities.

Funding Research

First, a quick word about research we're funding. Last year, PSC Support agreed to fund eight research projects that tackle PSC and bile duct cancer associated with PSC. The projects range from understanding the biological processes that lead to cancers, how best to screen for them, understanding fatigue (a symptom that affects most of us) to helping to fund a clinical trial testing a drug to treat PSC.

Funding for the eighth and final project agreed last year was awarded to Christoph Schramm and Jan Philipp Weltzsch from Hamburg, Germany, to assess and compare tests for diagnosing bile duct cancer to determine the best one (or combination) to diagnose this cancer early and without doubt.

With the final 2024 research project announced, we are currently working on assessing brand new research funding applications received this year. We expect to announce which projects we will fund towards the end of the year.

Supporting Research

Your donations allow us to not only fund essential research studies, but also allow us to provide essential research advocacy. We are tireless in this endeavour and are very grateful for your donations and time (be that completing surveys, as Patient Panel members, or volunteering on charity projects and reviewing applications for research funding).

It’s all important for raising the profile of PSC, clearly describing the impact of living with PSC and why developing treatments is so critical and urgent, helping researchers get funds to do more research, and helping patients learn about and take part in clinical trials. And all of this is getting us closer to effective care and treatments, sooner.

The following graphic is a snapshot of what we did last year. Do get in touch with me if you have any questions about these activities.

It is no surprise then to learn that research advocacy is what takes up the majority of my time and this month has been no exception!

It can take 10-15 years to develop new medical tests and drugs. Before they can be available for patients, there must be enough evidence to convince regulatory bodies in charge of approving medicines and payers (e.g. insurance companies or NICE in the UK) that the medicines and tests are safe, effective and offer value for money. This is where strong patient advocacy is vital.

Drug Development Process Drug Discovery - science to understand biological processes involved in PSC. Because we still have much to learn about PSC, much of our research focuses in this area. Understanding how the disease develops may uncover targets for drugs to work on. Drug discovery can involve looking closely at cells and in animals, long before the drug is tested on a human. Clinical Development - this is research on humans - clinical trials. Clinical trials are expensive, and pharmaceutical companies invest millions into developing drugs. There are some studies testing existing drugs that might help PSC. These are often funded by charitable donations (e.g. from PSC Support) and led by academic centres. Market Authorisation - the clinical trial data is reviewed by the regulatory authority. In the UK this is the MHRA. This is extensive and safety is a prime concern. NHS Appraisal - once the drug has been through clinical trials and approved by the regulatory authority, it is reviewed for cost effectiveness before it is made available on the NHS. Safety Monitoring - even once a drug is approved, the drugs continue to be monitored in the real world. Again, safety is critical. In the UK there is a system called the Yellow Card Scheme - you can use it to report side effects of any prescribed medicine. The MHRA constantly monitor this.

Drug Development Overview

Drug Discovery - science to understand biological processes involved in PSC. Because we still have much to learn about PSC, much of our research focuses in this area. Understanding how the disease develops may uncover targets for drugs to work on. Drug discovery can involve looking closely at cells and in animals, long before the drug is tested on a human.
Clinical Development - this is research on humans - clinical trials. Clinical trials are expensive, and pharmaceutical companies invest millions into developing drugs. There are some studies testing existing drugs that might help PSC. These are often funded by charitable donations (e.g. from PSC Support) and led by academic centres.
Market Authorisation - the clinical trial data is reviewed by the regulatory authority. In the UK this is the MHRA. This is extensive and safety is a prime concern.
NHS Appraisal - once the drug has been through clinical trials and approved by the regulatory authority, it is reviewed for cost effectiveness before it is made available on the NHS.
Safety Monitoring - even once a drug is approved, the drugs continue to be monitored in the real world. Again, safety is critical. In the UK there is a system called the Yellow Card Scheme - you can use it to report side effects of any prescribed medicine. The MHRA constantly monitor this.

After the exciting news in May about Phase 3 trial results for NCA (Falk Pharma’s drug) and the new “3-in-1” PSC/ bile duct cancer test (from an international collaboration led by Jesús Bañales and Pedro Rodrigues), we have been busy meeting with the researchers, those with experience of translational research (helping a drug or test developed in the laboratory get to clinics), other PSC patient organisations and regulatory authorities to agree how best to ensure these projects continue to generate solid evidence so that they can be available to patients wherever they live AS SOON AS POSSIBLE.

At the same time, I am not taking my eye off drugs in earlier stages of development. Why? Because there is unlikely to be a single medicine that can either treat everyone with PSC or address all of the biological processes that are involved. We must leave no stone unturned in our quest to develop effective treatments.

If you have any questions about this or any other aspect of our research work, please send me an email.

Martine Walmsley

PSC Support Head of Research Strategy
ERN RARE-LIVER Management Board and PSC Working Group Co-Lead

martine@pscsupport.org.uk