EB Research News Summary: July – October 2025
Read on for an update on the research projects DEBRA is funding and a summary of EB research that has been published over the last four months.
Clinical trials
In this period a new multi-million pound, UK-based clinical trial has been awarded, co-funded by DEBRA UK and LifeArc. Dr Su Lwin will lead this project at Guy’s and St Thomas’ NHS Foundation Trust (GSTT) and King’s College London (KCL). Patient recruitment will start in 2026 with the drug treatments anticipated to begin in 2027.
Our researchers carrying out clinical trials on dupilumab (Prof Paller, USA) and apremilast (Dr Chiaverini, France) have submitted progress reports in this period:
40 people have been recruited to Prof Paller’s dupilumab trial. 23 of them have already completed 8 weeks without dupilumab to set a baseline for symptoms, and 16 weeks of dupilumab injections. At night, participants wear a specially developed itch sensor to detect scratching that they may not be aware of, and they report their experiences of itching and pain. The preliminary results suggest a significant reduction in itching and pain due to the dupilumab injections. DEBRA UK funding towards travel costs for additional participants means the trial is on track to provide significant evidence for repurposing dupilumab to treat EB.
Dr Chiaverini’s trial on how well apremilast works for adults and children with severe EBS began at the end of 2024. Three of the four centres involved are open and ready to welcome participants.
Pre-clinical drug repurposing
Pre-clinical studies are carried out in cells before a drug is tested in people.
A project at the Cancer Research UK Scotland Institute, co-funded by DEBRA UK and DEBRA Ireland, has ended after Prof Gareth Inman successfully screened over 3,000 approved drugs to determine their effectiveness against RDEB cancer cells. 165 of the 3,135 tested were found to slow cancer cell growth or kill cancer cells in the laboratory. The top 50 were tested further at different doses and two will be progressed into pre-clinical testing in a new PhD project in Prof Inman’s lab.
Research to provide evidence for repurposing statins for RDEB cancer is at the pre-clinical stage, with work being carried out in cells rather than people. Dr Zauner’s researchers have confirmed that lovastatin effectively slows the growth of RDEB tumour cells in the laboratory, even showing signs of making them less likely to spread. They’ve found that not all tumour cells respond the same way and have identified a possible way to predict how well a tumour might respond to this statin treatment.
The Oliver Thomas EB Fellowship PhD student, Cameron Ferguson has begun working in Dr Chambers’ lab in London. In the first year of this project, researchers have found that specific types of immune cells are significantly increased in JEB skin, especially where the skin is affected by EB symptoms. Anti-inflammatory drugs planned for repurposing have been assessed and found to be safe. A larger study to understand how they will affect symptoms will be carried out over the next year of the project.
Dr Chambers presented information about her project at Members’ Weekend in May 2025.
Dr Zauner presented in our Research and Health webinar series in February 2025
Outside DEBRA UK, researchers continue to publish their advances on EB:
- Zevaskyn gene therapy treatment for RDEB involves collecting a patient’s skin cells and modifying them in a lab to add a healthy version of the COL7A1 gene. The modified cells are then grown into a thin sheet that is grafted onto a patient’s wounds. There are now three centres in the US where this treatment can be offered including the Children’s Hospital Colorado, and the Lucile Packard Children’s Hospital Stanford in San Francisco. The company that produces Zevaskyn, Abeona, plans to be able to treat up to ten people per month by the middle of 2026.
- Vyjuvek gene therapy is a topical gel treatment for DEB, made by Krystal Biotech, that can now be used from birth in the US and applied in a person’s own home. A case study reports how four weeks of Vyjuvek treatment helped an infant with DEB who was not previously meeting developmental milestones to stand and walk. The benefits of Vyjuvek have been confirmed in a trial in Japan where it may soon also be approved for use.
- Filsuvez is not a genetic therapy but helps wounds to heal. It is made by the company, Chiesi, with extracts from birch trees and approved for use in the UK, as well as EU and US, to treat wounds in DEB and JEB. It has recently been shown to reduce the time and money spent on dressing changes, with people in a German study reporting reduced pain and itching.
- Laboratory studies have highlighted different types of RDEB wound that heal more slowly than others and that specific proteins used by the immune system process, inflammation, may contribute to this.
- Laboratory evidence (pre-clinical) has been gathered to support the repurposing of midostaurin, a blood cancer treatment, to help reduce EBS symptoms.
- Researchers are investigating how EB affects the mouth and teeth and whether laser-based treatments can help with pain.
DEBRA UK is proud to support pioneering research that brings hope to the EB community. We receive no government funding and rely on donations to continue funding vital research and support services.
Check back for the next update in March 2026.