Delivering cells for treatment of children with EB
Epidermolysis Bullosa is a very painful genetic skin condition that causes blistering and skin loss at the mildest trauma. A recent clinical trial suggested that mesenchymal stromal cells could bring benefit to children with the condition and we aim to deliver this at Great Ormond Street Hospital
December 2017 - December 2020
Charity information: Cure EB
Epidermolysis Bullosa is currently an incurable condition with no effective treatment. Children with the condition have symptom management in the form of dressings and pain relief. Blisters have to be pricked and wounds dressed on a daily basis which is very painful and arduous time for the children concerned. Every aspect of a sufferers life is affected and every action is painful, even simple everyday tasks such as eating and walking. The condition is life shortening due to skin cancer
The project aims to improve the quality of life for children with EB. Evidence from the trial suggests that the cells reduce inflammation and therefore reduce discomfort and pain. Wound healing times improved and the frequency of trauma and blistering reduced. The time taken to complete the dressings was shortened which had an impact on family life. Children were happier and more able to participate in daily activities. Whilst not a cure this would establish an interim treatment.
Production of stem cells in a cell stack system at Kings College, London
Activities» Funding the cell stack system consumables and production scientist to produce cells dedicated for use in treating children with RDEB at GOSH
What success will look like
A rigorous process of validation needs to be completed prior to being able to deliver cells to patients. Quality control testing shows cell suitability
Technical support for the bioreactor
Activities» Fund the technician who will responsible for generating mesenchymal stromal cells for clinical use
What success will look like
A technician will be recruited to ensure successful validation of the cell stack. The first cells delivered to patients as a treatment will demonstrate success
This project would aim to establish a cell delivery system that is dedicated for epidermolysis bullosa therapy and research. In the recent past, clinical trials cells have been shipped from Utrecht in Holland which has been expensive. A facility in the UK which is dedicated for treatment and further research would be cost efficient and would enable easier access.
The risks are
1) generating cells that are safe and effective for use in people which conform to GMP standards
2) Transporting the cells to the hospital where they will be delivered to children. In the first instance this will be Great Ormond Street Hospital
By using experienced and highly competent clinical teams at Kings and Great Ormond Street we are hopeful that they can 'de-risk' the process
Donors receive updates on all our work on a regular basis through newsletters, email updates, letters and meetings where we have scientists speak. We will also gather video testimonials from patients and use social media to disseminate information
Budget - Project Cost: £143,000Loading graph....
Amount Heading Description £94,000 Cell Stack Consumables A system for generating cells £49,000 Technician A person responsible for running the bioreactor
Current Funding / Pledges
Source Amount Trust £20,000 Guaranteed Corporate donor £30,000 Guaranteed
The cells will be generated at Kings College London and delivered initially to patients at Great Ormond Street Hospital in London. Whilst generation is based in London, these cells could be transported to other hospitals within the UK depending on demand.
Children with epidermolysis bullosa within the Uk who the medical teams have assessed will benefit from infusions of mesenchymal stromal cells given intravenously.
We were instrumental in the delivery and have solely funded both the recent clinical trials testing the use of mesenchymal stromal cells in patients with recessive dystrophic epidermolysis bullosa, which took place at Great Ormond Street Hospital and Guy's Hospital in London. We are solely dedicated to funding research and clinical trials leading to treatments and are collaborating with funding organisations in the the UK and US to make this happen.
Read more about the Charity running this project.
Founder and Trustee of the Sohana Research Fund who works closely with the medical teams
Professor Francesco Dazzi
Professor of Regenerative and Haematological Medicine at Kings College London who will oversee production of the mesenchymal stromal cells
Professor John McGrath
Professor of Molecular Dermatology at Kings College London, who is the leading EB expert and researcher in the UK
SRF 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