Skin equivalent grafts for epidermolysis bullosa
This is a clinical trial which aims at using gene corrected grafts to treat chronic wounds of three patients with recessive dystrophic epidermolysis bullosa. As the grafts are gene corrected in all layers they have the potential to permanently heal the areas they are grafted on to.
December 2018 - December 2020
Charity information: Cure EB
Epidermolysis Bullosa is currently an incurable condition with no effective treatment. It often leads to chronic wounds that take time to heal, or do not heal at all, sometimes for years. Management of these wounds includes applying dressings and ointments in an effort to promote closure and avoid infection. These wounds are very painful, like severe burns and they lead to contracting scars and and increased risk of skin cancer.
The project aims to improve the quality of life for people with EB. If the grafts work and wounded areas are healed up permanently this will be a huge step in managing EB on a local level. The grafted area should not re-blister and there will be a significant reduction in pain. Whilst this is not a whole body treatment, this should represent a very effective treatment for painful open wounds that do not heal easily
Manufacturing skin grafts from a skin sample taken from a patient with recessive dystrophic EB
Activities» Funding the production, quality control and shipping of the gene corrected grafts for the patients on the trial.
What success will look like
The skin grafts are produced under strict GMP conditions and undergo rigorous quality control. The shipping of the product for grafting will demonstrate success
These grafts should allow durable if not permanent treatment of grafted areas. This will mean a reduction in blistering and wounds. This is turn will lead to fewer skin infection and much less pain and itching of those areas. If the skin heals and does not re blister there is less need for dressings. there will be a reduced risk of skin cancer with closure of chronic wounds. Quality of life for patients would be substantially improved
1) Production of a GMP graft that is safe and effective in people
2)Transporting the graft
3) Grafting onto a patient wound
The clinical and scientific teams are highly experienced and the necessity of gaining regulatory approval should de risk the procedure substantially
Donors receive updates on all our work on a regular basis through newsletters, email updates, letters and meetings where we have scientists speak. Whilst trial sensitive information cannot be divulged prior to final reports, that in the public domain will also be disseminated via social media
Budget - Project Cost: £328,838Loading graph....
Amount Heading Description £191,977 Skin graft production Production, quality control and shipping of the grafts £44,500 Hospitalisation Hospitalisation during the grafting procedure for patients on the trial £16,279 Clinical research assistant Monitoring the patients for the duration of the trial £40,695 Clinical Study Technician Technical monitoring for the study £35,387 Statistician Statistical analysis of the project
Current Funding / Pledges
Source Amount Trust £100,000 Guaranteed Corporate £50,000 Guaranteed Donor £50,000 Guaranteed
This project has been a multinational collaboration between clinical teams in Europe, including Guy's and St Thomas' Hospital in the UK, Necker Hospital in Paris. Eufets in Germany. The skin grafts will be produced in Madrid, be grafted in Paris and will include a UK patient
People with epidermolysis bullosa will benefit from this project. The ability to use a gene corrected skin graft that will heal a wound and then last for a long time has huge implications for the community and for the quality of life of patients who are in so much pain, most of the time.
We are solely dedicated to funding research and clinical trials aiming to lead to treatments and are collaborating with funding organisations to make this happen. We have funded all of the most recent clinical trials for recessive dystrophic epidermolysis bullosa in this country and were approached to fund this multinational collaboration to trial skin equivalent grafts
Read more about the Charity running this project.
Professor Alain Hovnanian
Head of the research laboratory on genetic skin disease at Imagine Institute, Paris
Professor Fernando Larcher
Universidad Carlos 3 Madrid
Professor John McGrath
Professor of Molecular Dermatology at Guy's and St Thomas' NHS Foundation Trust, Kings College London
Cure EB has given us hope for the future that there could possibly be a cure for this terrible condition. We can only imagine how happy we could be as parents if our one and only dream came true and Poppy could live with a life without pain.