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Repurposing drugs for EB
Drug repurposing is the technique of using an existing drug for a new treatment or medical condition for which it was not indicated before.
This creates an exciting opportunity for people with EB, and other rare conditions too, where the high cost of developing a brand-new drug (up to £1b per drug) and the time to market (10-20 years) often makes it commercially unattractive for pharmaceutical companies.
Drug repurposing in comparison typically costs up to £500k per drug and can take as little as 2 years.
See chart below which compares the timeline for developing a brand-new drug treatment with a drug repurposing timeline.
Initially clinical trials are only carried out on a small number of people in case there are any unforeseen side effects, this is known as phase 1. If this phase shows that a new treatment is safe, it can then be trialled on more people with symptoms in a phase 2 trial. However, if the treatment that is being tested is already in use and successfully treating another condition, phase 1 can be skipped because the treatment has already been shown to be safe. This is called drug repurposing, and is a key part of our EB research strategy.
Drug repurposing is potentially a quicker route to securing effective drug treatments for people with all types of EB, because it doesn’t involve stage 1. It is also cheaper because it is focused on clinically testing existing drugs used to successfully treat related conditions
For EB there are drugs already available within the NHS that successfully treat other inflammatory skin conditions, including psoriasis and atopic dermatitis (severe eczema), which could significantly improve EB symptoms such as blistering and overall quality of life. To prove the effectiveness of these drugs for the treatment of EB though requires testing through clinical trials.
Our EB research strategy prioritises investment in drug repurposing to secure life-changing treatments for every type of EB.
By understanding how a treatment works and how a symptom of EB is caused, EB researchers can identify treatments with the potential to be repurposed.
Specialist doctors may offer a few EB patients the opportunity to try a treatment ‘off-label’. This means it is licensed to treat a condition other than EB. They study the outcomes carefully and publish their results as a case study. However, to repurpose a treatment, a clinical trial involving more patients will need to conducted to be sure that the positive outcomes seen in an initial case study were not just due to chance.
The process of drug repurposing has been proven to saves lives.
You may well have used repurposed drugs yourself. When the COVID-19 pandemic began, there was a rush to repurpose existing drugs that might help. Doctors used their knowledge of the virus to select medications, and clinical trials were initiated to see if their educated guesses were correct.
Aspirin is an example of a familiar drug that has been successfully repurposed. From its initial use against pain, fever, and inflammation, it is now used in lower doses to reduce the chance of heart attacks and strokes.
In some cases, the side effects of a medication allow for repurposing, for example, Viagra was initially developed to treat angina, but a commonly noted side effect led to it being repurposed to treat erectile dysfunction. Also, many different treatments have been successfully repurposed to treat breast cancers including antibiotics, anti-virals, treatments for autoimmune diseases, medications for other cancers and drugs originally used to help with infertility.
Other people living with rare conditions including tuberous sclerosis, alkaptonuria, and autoimmune lymphoproliferative syndrome have also benefited from drug repurposing research and clinical trials that led to the approval of existing drugs to treat these conditions.
Many drugs have additional effects that mean they can be used to treat symptoms other than those they were originally licensed for. Where one effect is to reduce skin blistering, inflammation, itching, or scarring, these drugs may be particularly relevant for people with EB.