Epidermolysis Bullosa: Drug Repurposing Trials

Jim Shannon Excerpts

Tuesday 9th May 2023 Commons Chamber

[Gareth Bacon (Orpington) (Con)]

Epidermolysis bullosa is dreadful. It is often referred to as butterfly skin. It is a little known and rare genetic skin blistering condition, which causes skin to blister and tear at the slightest touch. BE—I will be kind to myself and refer to it in the rest of my speech as BE—causes excruciating, lifelong pain, and can have a devastating impact on the physical and mental wellbeing of patients. It can also greatly affect patients’ families because of the constant treatment needed. In many cases, this means several hours of bandaging and unbandaging the most affected parts of the body daily. In its most severe form, EB also affects internal organs and eyes, causes severe problems when it comes to eating and drinking, and requires extensive healthcare—and there is no cure. It is believed that EB affects around 5,000 people in the UK, although there is limited information available from the NHS. Although that is a relatively small number, the severity of the condition is such that it has a deep impact.

I was asked to secure this Adjournment debate by some of my constituents, DEBRA, which is a UK-wide charity supporting people with EB and their care givers, healthcare professionals and researchers who work with EB. The charity exists to improve quality of life for people living with EB, and to fund pioneering research to find effective treatments. The charity works in partnership with the NHS to deliver EB healthcare services. There are four centres of excellence delivering specialist care in the UK, along with additional hospital care and clinics at other locations. The charity has a proud record of funding EB research in the UK and internationally, funding the first clinical trials in gene therapy. Its current focus is on fulfilling its mission to help people with EB live a life free of pain, a point I will return to later in my speech.

Many colleagues may be familiar with DEBRA from some of its network of more than 100 charity shops, which are the main source of the charity’s income. I was able to visit one of the DEBRA shops, located in Locksbottom in my Orpington constituency, a few months ago, and I met not only the volunteers there, but a local constituent called Wendy, who suffers with EB. This particular lady has been suffering from EB since birth, and she told me of her experience and the experience of others who have an even more severe type of the condition.

As a child and even as a young lady, Wendy did not know that she had EB because doctors had not diagnosed the condition, which, particularly in the early years, is difficult to spot. She did, however, suffer constantly from blisters on her feet and her legs. Wendy told me about how, while she was growing up in constant pain, she would try to hide her wounds from friends and classmates, and would avoid wearing skirts to avoid causing unpleasant reactions from others. Yet as terrible as this sounds, Wendy told me her case was not uncommon and not as severe as it was for other patients.

[Jim Shannon (Strangford) (DUP)]

I commend the hon. Gentleman for bringing this debate forward. He raises an issue that affects not only his constituents, but mine and those of others across Northern Ireland, so I commend him. Does he not agree that this dreadful disease, which is named the butterfly disease because it is unusual, has the capacity to limit life and the quality of life, and that the funding for the treatments he refers to, which can provide relief to constituents in all of our constituencies—in his, mine and those of others in this House—is vital? Would he urge the Minister, who is a very compassionate man and understands the issue very well, to give even the smallest bit of funding towards research to try to find a cure for the disease and to help the lives of those who have it?

[Gareth Bacon]

I thank the hon. Gentleman for his kind words and the way he expressed them, and I agree entirely with those sentiments. Without giving out too many spoilers at this stage, there will be a request for a small amount of funding towards the end of my speech.

What I learned during the visit I referred to was truly moving, and I am particularly grateful to have met Wendy. I also thank DEBRA’s director of research, Dr Sagair Hussain, and the excellent staff at the charity shop in my constituency, for inviting me to visit them and learn more about how they help individuals who live with this painful condition. In the spirit of thanking people, I also thank the Minister for his interest in this subject and for being here this evening to respond to the debate, and the Minister for Social Care for recently answering a written parliamentary question that I tabled about EB.

I stress that we cannot merely wait for a cure for this condition. We need to make a difference for patients who are suffering today and those who will be living with the condition for the foreseeable future. All EB patients are crying out for better therapeutic treatments, which have the potential vastly to improve their lives. DEBRA has set an objective of securing two to three treatments from drugs that are already licensed for other conditions, to radically improve the quality of life experienced by people with EB. In reply to my recent written question, the Minister for Social Care said that medicines that are potential candidates for repurposing in this way should be put forward for consideration for support from the Medicines Repurposing Programme. I am grateful for her guidance, and officials from the MRP have been in touch with DEBRA since to talk about the programme’s work. That is excellent news.

In addition, I was delighted to hear that some innovative treatments for EB are either in trials or are being considered by the National Institute for Health and Care Excellence. Specifically, NHS England is working with NICE on the evaluation of two products for EB: birch bark extract for skin wounds, and a gene therapy with a name that I find particularly difficult to pronounce, although I will give it a shot—beremagene geperpavec. I have almost certainly mispronounced that, but it is still encouraging news. However, my understanding is that those two treatments will be available only to a fraction of the total number of people suffering from EB. That is why the repurposing process for more mainstream therapeutics is so important.

DEBRA has identified six anti-inflammatory drugs that could help with EB. Several of those are already available for people with more common skin conditions such as eczema and psoriasis, but for people with EB they could be nothing short of life-changing. They have the potential to transform thousands of lives by improving wound healing, reducing pain, and lowering the burden on the family members and carers of those with EB.

--- Later in debate ---

[Will Quince]

If I am available I would be happy to do that. I thank my hon. Friend for his support for that charity, which is based in his constituency but works nationwide. The support that constituency Members of Parliament provide to charities through this place should not be underestimated. If I am able to attend, I will.

[Jim Shannon]

When we spoke earlier, I was quite sure that the Minister is truly a compassionate man. He has given us the answers that we wish to hear, and I thank him for that. He talks about the possible cures for EB. Will that information be shared with all devolved Administrations? I think he said that it would be, but I wanted to check.

[Will Quince]

Absolutely. We work on clinical and medical research across our United Kingdom, and rightly so. We work very closely across all four nations.

I am conscious of time, and I want to pick up on the final question from my hon. Friend the Member for Orpington about generic versus branded medicine and the MRP. Branded medicines are potentially eligible for the medicines repurposing programme. I understand that the eligibility criteria state that the programme can support generic, biosimilar and branded medicine. I would be happy to look at my hon. Friend’s list.

In closing, I give my thanks again to my hon. Friend for securing today’s important debate, and to all Members who have contributed. I also pay tribute to the whole EB community and charities such DEBRA and Cure EB, which I know work tirelessly to improve the lives of people affected by the condition. They are fortunate to have my hon. Friend in this place as their champion.

Question put and agreed to.