Drug repurposing is an exciting opportunity for EB and is essential for conditions described as 'rare diseases' where high costs can make traditional drug discovery unviable.

There are drugs already available to treat other inflammatory skin conditions, such as psoriasis and atopic dermatitis, which could significantly improve blistering and overall quality of life for people living with EB. To prove the effectiveness of these drugs for the treatment of EB requires clinical trials.

We are prioritising investment in drug repurposing to accelerate research progress and find life-changing treatments for EB.

 

 

Contents:

• What is drug repurposing?
• What are the benefits of drug repurposing?
• How does drug repurposing happen?
• Drug repurposing success stories
• Our drug repurposing projects

Diagram demonstrating drug repurposing.

What is drug repurposing?

Drug repurposing is a way of finding new uses for existing treatments and increasing the treatment options for people with EB more quickly and more cost-effectively than creating brand new treatments.

There are drugs already available within the NHS to treat other conditions which could significantly improve symptoms and overall quality of life for people living with EB. 

Well-designed clinical trials are required to prove the effectiveness of these drugs before they can be licensed for use by people with EB.

 

What are the benefits of drug repurposing?

Drug repurposing is quicker, safer and cheaper than creating a completely new treatment.

Developing a new treatment to the point where it can be prescribed for patients can take decades and cost hundreds of millions of pounds or more. A drug repurposing study can cost less than half a million pounds and be carried out in 2-3 years.

Drug discovery vs drug repurposing diagram.

Drug repurposing means that the treatment has already been tested and shown to be safe for people to use. There will already be suggested doses and timings for taking the medication to maximise its effectiveness and any side effects will have been identified.

 

How does drug repurposing happen?

Understanding how a treatment works and how a symptom of EB is caused can help researchers to choose which treatments might be most successfully repurposed.

Many licensed drugs may be tested in the laboratory before one or two are identified that show most promise.

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’.

To repurpose a treatment, a clinical trial involving more patients needs to be funded to be sure that the positive outcomes seen in an initial case study were not just due to chance.

People with EB will be asked by their care team if they would like to participate in research studies. There is no obligation to do so and anyone can withdraw from a study at any point without giving any reason.

When enough people have participated in a drug repurposing study, the researchers will analyse the results and see if they can prove that the treatment will be helpful.

We use recommendations from experts and our members opinions to help DEBRA fund trials that are most likely to provide strong evidence towards licensing approval.

 

Drug repurposing success stories

The process of drug repurposing saves lives. You may well have used repurposed drugs yourself.

When the COVID-19 pandemic began, there was a rush to repurpose any existing drugs that might help. Doctors used their basic science knowledge of the virus to select medications that might help, 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 repurposing. Viagra was initially developed to treat angina, but a commonly noted side effect led to Viagra being repurposed to treat erectile dysfunction.

Many different treatments have been successfully repurposed to treat breast cancer including antibiotics, anti-virals, treatments for autoimmune diseases, medications for other cancers and drugs originally used to help with infertility.

Patients with rare conditions such as tuberous sclerosis, alkaptonuria and autoimmune lymphoproliferative syndrome have benefited from drug repurposing research studies and clinical trials leading to approval for existing treatments to be used in 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 to EB patients. Drug repurposing is the process of validating new uses for currently existing treatments.

 

Our drug repurposing projects

DEBRA’s first drug repurposing clinical trial for EB will use an existing anti-inflammatory drug (apremilast) which is licensed to treat patients with psoriasis. Encouraging results published after an initial small trial, showed that it appears to reduce blistering in patients who have the severe form of epidermolysis bullosa simplex (EBS). We are now funding a larger trial to measure not only blistering but also whether it could improve quality of life for people with the severe form of EBS by reducing pain and itch.

Using treatments that successfully treat other inflammatory skin conditions such as psoriasis, and are already available within the NHS, means we could find treatments for EB sooner. This is the first of what we hope will be many clinical trials that we will fund which could take us a step closer to having approved drug treatments for EB.