What about drug repurposing for EB?
Ongoing projects
Drug repurposing involves taking an existing drug and using it to treat a condition for which it was not originally designed. This approach can lead to new treatment options more quickly and at a lower cost.
Drugs already used for other conditions, such as eczema or psoriasis, may have the potential to greatly improve symptoms and quality of life for individuals with EB.
However, before these drugs can be approved for EB, it is necessary to perform clinical trials to confirm their effectiveness for this specific condition.
Drug repurposing presents an exciting opportunity for people with EB and other rare conditions. It has many benefits. It is faster, safer, and more affordable than developing an entirely new drug.
Creating a new drug from scratch can take years and is expensive. In contrast, repurposing an existing drug already on the market is generally less costly and only takes 2-3 years. The known side effects of the drug are already documented, and there are established guidelines for its use.
Drug repurposing is more attractive to pharmaceutical companies since it eliminates the high cost of developing a new drug and the lengthy timelines associated with it to go to market. However, drug repurposing isn’t without risks. The repurposed drug might not work for the new condition, leading to potential setbacks and wasted resources.
Researchers can identify potential drugs for repurposing by understanding the specific function of a medication and how it could address the symptoms of EB. By studying how the drug works and how it may interact with the underlying causes of EB, researchers can pinpoint which drugs are most likely to be effective. These are sometimes assessed on one or two patients to assess their efficacy. These are often published as case studies.
A clinical trial is still necessary to repurpose a treatment to ensure that the positive results observed in the original case study are accurate and applicable to a larger cohort with the condition. Researchers will analyse the data from the drug repurposing study once enough participants have participated, determining whether the treatment is beneficial for the new condition. Many drugs exhibit additional effects beyond their original licensed purpose, and through these trials, researchers can identify how these additional benefits can be harnessed to treat symptoms in new conditions.
Ongoing projects
Debra Ireland and DEBRA UK are funding research on drug repurposing as a treatment option for people with recessive dystrophic epidermolysis bullosa (RDEB). Professor Gareth Inman is leading the study to understand whether repurposing a drug will work in treating squamous cell carcinoma in RDEB.
In doing so, the research team will screen over 3,000 existing drugs already approved for other conditions.
They will test these drugs in a lab setting to see if any can effectively stop the cancer cells from surviving. By doing this, they hope to identify two promising drugs that can quickly and safely move into clinical trials, bypassing the lengthy and expensive process of developing a new drug from scratch.
Debra Ireland and DEBRA UK are also supporting a groundbreaking clinical trial led by Professor Amy Paller in Chicago. This study is exploring the potential of Dupilumab, a drug already approved for eczema and psoriasis, to treat EB-related itch.
Severe itching is a major issue for many with EB, especially in forms such as EB pruriginosa. Preliminary findings suggest Dupilumab could significantly reduce itching and even blistering. Early data shows that individuals with high IgE levels experienced a 70% reduction in pain within just eight weeks. To ensure as many people as possible can participate, Debra Ireland and DEBRA UK are co-funding travel expenses for trial participants based in the US. This initiative aims to break down financial barriers, allowing more individuals to access potentially life-changing treatments.
In collaboration with DEBRA UK and led by Prof. Liam Grover, we are co-funding a project to develop a targeted eye drop solution for all types of EB.
The project will repurpose an existing eye drop technology, which has shown promise in other eye conditions, to address the needs of individuals impacted by EB.
The gellan gum-based eye drops will offer longer-lasting lubrication, reducing the frequency of applications and providing much-needed relief from the pain and risk of blindness caused by eye surface damage. This innovative approach can potentially improve the quality of life for individuals with EB by preventing scarring and protecting the delicate eye tissues from further trauma.
Dr Sinéad Hickey, Head of Research
