My work aims to bridge the gap between rare and common skin diseases. By repurposing existing drugs that are already licensed for use for other conditions, we can accelerate therapeutic innovation and hopefully bring much-needed treatments to the people who need them sooner.
Dr Su Lwin, Senior Research Fellow at King’s and Consultant Dermatologist at Guy’s and St Thomas’
09 December 2025
World's largest drug repurposing trial to fast-track treatments for rare 'butterfly skin' disease
Dr Su Lwin from King’s College London and Guy’s and St Thomas’ NHS Foundation Trust has been awarded funding from LifeArc and DEBRA UK to lead a multi-million-pound clinical trial to repurpose existing drugs to help treat epidermolysis bullosa (EB) – a rare, painful genetic skin condition that causes fragile, blistering skin, often called ‘butterfly skin’.
The planned five-year Advancing Repurposed Therapeutics (ART) EB trial will investigate whether drugs already used to treat more common skin conditions, such as psoriasis and eczema, can be repurposed to treat EB.
Understanding the role of inflammation in EB
EB describes a group of rare and painful genetic skin disorders that cause the skin to blister and tear. As the skin becomes so fragile, like a butterfly’s wings, it is often called ‘butterfly skin’. EB is estimated to affect at least 5,000 people in the UK, and currently has no cure.1
Emerging evidence suggests that inflammation in the skin – caused by the body’s immune response to the wounds and blisters – worsens EB, increasing pain and the chances of scarring and infection. But little is known about which key immune signals, called cytokines, cause the inflammation in EB.
The first stage of the ART EB trial will be to better understand the inflammation in EB through a process called immune profiling. Dr Lwin will aim to group people in the trial based on the ‘immune profiles’ (patterns of cytokines) driving their inflammation. Depending on these profiles, people will then be matched with the available treatment most likely to be effective for them.
Bridging the gap between rare and common skin conditions
The trial will use an innovative approach to test multiple drugs at the same time across multiple clinical trial stages, with patients being matched to one of up to three drugs. Drugs that aren’t effective will be dropped from the trial and replaced with a different drug.
“This is the first time that a trial for a rare skin disease has utilised this multi-arm, multi-stage trial design,” Dr Lwin adds.
The team is aiming to recruit adult patients from EB centres across the UK, including the Rare Disease Centre at St Thomas’ Hospital. Drugs which show promise will move into the next phase of the trial, to be tested in larger numbers of people.
Recruitment to the immune profiling arm of the study is expected to begin in 2026 and the drug trial in 2027.
DEBRA UK CEO, Tony Byrne said: “We are delighted to have agreed this groundbreaking new research partnership with Dr Su Lwin and LifeArc. Effective treatments for all forms of EB are the no.1 priority for the EB community, and this new project could develop a clinical trial platform that can test three different drugs at the same time whilst also supporting future EB clinical trials. On behalf of DEBRA UK and the EB community, I would like to thank LifeArc and Dr Lwin for their commitment to this project and to improving quality of life for people living with all forms of EB. I would also like to thank our many supporters who have helped contribute vital funding”.
Karen Skinner, Chief Operating Officer at LifeArc, said: “We are delighted to support the ART-EB trial, a project which exemplifies LifeArc’s commitment to accelerating scientific innovation that delivers real impact for patients. By repurposing existing drugs, ART-EB offers a faster, more cost-effective route to treatments that could significantly improve wound healing and quality of life for people living with EB. Collaboration is at the heart of this effort – by working together with DEBRA UK, Dr Su Lwin, and the wider EB community, we can move faster and bring more than hope to those living with EB.”
References
- DEBRA. Epidermolysis bullosa (EB): symptoms, treatment, and care. Available at: https://www.debra.org.uk/get-support/eb-support-and-resources/epidermolysis-bullosa-eb/ (Accessed on 6/11/2025)
In this story
Dermatology Registrar, Honorary Clinical Research Fellow, Genetic Skin Disease Group