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Dundee Scientists on road to sure for "Butterfly Children" condition

13.05.2005


Scientists in Dundee have embarked on a major research programme funded by the charity DebRA which it is hoped will ultimately lead to a successful treatment for a previously incurable genetic skin condition.



DebRA has awarded a grant of £1.6 million to Professor Irwin McLean and Professor Birgitte Lane for a five-year research project into the condition, Epidermolysis Bullosa.

Epidermolysis Bullosa (EB) is a rare genetic condition in which the skin and body linings blister at the slightest knock or rub, causing painful, open wounds. At its mildest, the condition is confined to the hands and feet making holding things and walking extremely painful. In more severe forms all the body is affected and the wounds heal very slowly, giving rise to scarring, physical deformity and significant disability. People with the more severe types of EB also have an exceptionally high risk of developing skin cancers, shortening their lives by approximately 30-40 years. In its most severe form, the condition is fatal in infancy.


Both Prof McLean and Prof Lane have been involved in groundbreaking research into EB at Dundee’s Medical School and School of Life Sciences for several years, particularly into EB Simplex, the most common form of EB. This new injection of grant funding from DebRA will allow them to build on the large body of work they have already completed.

"The genes which cause EB Simplex and other forms of EB were discovered here in Dundee," said Prof McLean. "We now have data which will help us move towards a treatment for EB Simplex. A gene therapy treatment has been developed which involves the use of ’RNA interference’, a new method which can effectively switch off faulty genes. This is a very promising way of bringing about a treatment. The project will hopefully bring a treatment from the lab, through safety tests, and then to clinical trials by the time the five-year programme is drawing to a conclusion."

It is envisaged that clinical trials will be carried out at the new Clinical Research Centre at Ninewells Hospital in Dundee, the roof of which is a design inspired by butterfly wings in recognition of the work carried out by the Dundee scientists. The sufferers of EB are often known as "Butterfly Children" because their skin can be as fragile as a butterfly’s wings.

Prof McLean hailed the development of the Clinical Research Centre as a vital boon to the research programme. "There is space within the new building for us to do gene therapy trials, where we will be able to have small numbers of patients come in and get new, very specialised treatments," said Prof McLean. "To have a facility like that upon our doorstep is a huge boost for us; it formed an important part of this grant application, because we were able to show the various aspects of research and trials that we can carry forward here in Dundee. By the time the Clinical Research Centre is complete, we would hope to be moving our first patients on to clinical testing, so the timing could not be better."

Any major breakthrough on a treatment for EB Simplex could have wider implications, as there are dozens of other conditions caused by keratin genes similar to the ones responsible for EB.

DebRA’s Director for Scotland, Robin Hood, said: "Our charity funds research into an effective cure for EB and also boasts the best specialist Health Care Team in the world, providing support to 400 affected people in Scotland. Our publicity was boosted last year by the award winning Channel 4 documentary, "The Boy Whose Skin Fell Off", featuring Jonny Kennedy.

"Although we are a little-known charity, we are currently third in the league table of those seeking a cure for genetic conditions, lying behind Muscular Dystrophy and Cystic Fibrosis. Our success is due to Prof McLean and Prof Lane’s work into EB and DebRA is delighted to fund the next five-year research programme through the £1.6 million grant. With little state funding available, the majority of our funding is raised by the general public. Without continued financial support from the people of Scotland an effective treatment for this horrific condition will take so much longer to find."

Roddy Isles | alfa
Further information:
http://www.dundee.ac.uk/pressreleases/prmay05/butterfly.html

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