Repurposing existing medicines may offer faster route to new EB treatment
UK trial aims to bridge gap between common, rare skin diseases
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Scientists in the U.K. have secured funding for a clinical trial that will test whether multiple drugs used to treat other, more common skin disorders might be repurposed to treat epidermolysis bullosa (EB)
The upcoming study, dubbed the Advancing Repurposed Therapeutics (ART) EB trial, will be led by Su Lwin, PhD, a dermatologist at King’s College London and Guy’s and St Thomas’ National Health Service Foundation Trust.
“My work aims to bridge the gap between rare and common skin diseases,” Lwin said in a news story from the college.
Repurposing existing medicines to treat rare diseases such as EB may have several advantages. Medications that are already approved for other indications have well-established safety profiles, and it’s usually cheaper to test an established drug than to develop a new therapy from scratch.
“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,” Lwin said.
Patients will be matched with a treatment expected to benefit them
EB encompasses several disorders that cause skin to be abnormally fragile and prone to wounds and blisters that don’t heal well. Normally, when a wound occurs in the skin, the area becomes inflamed, allowing immune cells to eliminate any infections. The inflammation then resolves, enabling the wound to heal. However, in EB, inflammation may persist abnormally, contributing to the development of disease symptoms.
Although inflammation is thought to play a role in EB, the biochemical details aren’t well understood. The first part of the ART EB trial aims to measure levels of different signaling molecules that regulate inflammation, known as cytokines, in EB patients. The goal is to divide patients into different groups based on which particular inflammatory molecules are most activated.
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.
Then, in the second part of the study, the researchers will use these inflammatory profiles to match patients with treatments that are expected to benefit them. For example, if some patients have high levels of a particular inflammatory cytokine, they’d be expected to benefit from medicines that can block that particular signaling molecule. The trial will test three different therapies, using an adaptive design where drugs that don’t prove effective will be dropped from the trial and replaced with other medicines.
“This is the first time that a trial for a rare skin disease has utilised this multi-arm, multi-stage trial design,” Lwin said.
The first part of the study that will examine immune profiles is expected to start recruiting in 2026, with the second part testing experimental therapies planned to begin the following year. The trial will be conducted at sites in the U.K., and it is being funded in part by the EB advocacy organization DEBRA UK.
“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,” said Tony Byrne, CEO of DEBRA UK.
Also helping to fund the upcoming trial is Lifearc, a U.K.-based nonprofit that supports medical research related to various diseases.
“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,” said Karen Skinner, Lifearc’s chief operating officer. “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.”
