DebRA has announced that they are to benefit from the generosity of a gift from Goldman Sachs Gives, a donor-advised fund. The donation, which is expected to total around £500,000, is the charity's single biggest individual donation.
DebRA is the only UK national charity working with families affected by Epidermolysis Bullosa (EB), an agonising skin condition which causes the skin to blister with the slightest injury. In its most severe forms, EB is fatal in infancy and can cause fatal skin cancer in young adults. The Goldman Sachs Gives donation will go specifically to support the Sohana Research Fund (SRF) which was set up to fund research into Recessive Dystrophic Epidermolysis Bullosa (RDEB), one of the most severe types of EB.
This donation, which will increase the total projected DEBRA research spend for this year by 50 %, will enable an important clinical trial into RDEB therapy to take place sooner and more comprehensively than expected.
DebRA's CEO, Ben Merrett, said: 'Without this most generous contribution, the research we have planned would not have been able to take place so soon. The gift from Goldman Sachs Gives will enable us to stride ahead in our goal to search for effective treatments and, ultimately, a cure for this most cruel condition'.
The trial, due to start later this year, will be conducted by Professor John McGrath, Professor of Molecular Dermatology at King's College London and take place at Great Ormond Street, London.
Professor McGrath said, 'Repeated skin damage over years leads to scarring, contractures and severe disability, so an effective treatment needs to be given early in life and in a way that can treat the whole skin. We have learnt that Mesenchymal Stromal Cells within the bone marrow can repair skin and even make the collagen Vll protein that RDEB sufferers lack. The aim of this first clinical trial is to show that these cells can improve the skin of children with RDEB, in a safe and efficacious way. If the results are as we expect, more comprehensive cell therapies could be developed to further improve the lives of those living with this devastating condition'.
It is anticipated that between 10 and 20 children with EB will take part in the trial and will receive injections of MSC cells from either a parent of one of the children included in the trial or an unrelated third party donor. The trial is designed to establish the effectiveness of bloodstream delivery of Mesenchymal Stem Cells (MSC), the type of cell in bone marrow which has been found to help repair damaged EB skin and aims to establish the parameters within which this cell therapy can be used safely and effectively to treat patients.
Michael De Lathauwer, Managing Director at Goldman Sachs said, 'We are very excited to be able to fund a potentially life changing trial at this crucial stage of RDEB research. EB is truly a terrible condition, and we hope this contribution from Goldman Sachs Gives will enable DebRA to continue making a real impact in the field of EB and on those affected by it'.
In 2010 Goldman Sachs Gives donated approximately £8,055,153 ($12.8m) to charitable causes. In 2011, the initiative donated £19,091,297 ($30.4m).