European Union funded research Interview with Prof. Meneguzzi

European Union funded research

Interview with Prof. Meneguzzi

What if healthy skin grown in a laboratory could successfully replace the damaged skin of EB patients?
That is the question driving the research team conducting the three-year Skintherapy project in Europe, in which DebRA is a partner.
With an idea borrowed from the treatment of burns patients, when skin is taken from elsewhere on the body and grafted onto the burn site, researchers are hoping to adapt the practice to suit people with Recessive Dystrophic Epidermolysis Bullosa (RDEB).
Instead of skin being taken from one site on the body and grafted over a burn however, the skin cells of the RDEB patient will be genetically modified – the faulty EB gene altered to create normal cells – before being grafted over damaged skin.

If successful, the project will have an enormous impact on EB patients.
It raises the possibility that one day the damaged skin of all RDEB patients might be repaired – taking away many of the most severe symptoms of the devastating condition.

Led by Dr Guerrino Meneguzzi, the team is learning many lessons from a different, more advanced project – involving most of the teams in the Skintherapy consortium – which aims to develop similar gene therapy for Junctional EB patients.
For instance the teams will learn the best grafting procedures, the best care of the grafted wounds and the best follow-up procedures.
And they will also be able to identify the best viral vector, which allows a corrected gene to be introduced to the skin cells to change them from abnormal to normal.
There are differences between developing treatments for RDEB and Junctional EB, particularly with the type of vector used, which is why one project could not cover both forms of the condition.
It is also why the Skintherapy team must adapt the lessons learned from the gene therapy project before applying them to their own study.
Dr Meneguzzi said: “The idea was, that since the technology of growing fresh skin was well known in the treatment of burn victims, why shouldn’t we try to adapt this technology to try to treat EB patients? From a certain point of view they are like burn patients but in this case we must adapt the treatment to the particular condition.”
A major part of the project will be focused on determining the correct vector which will do the required job and will be completely safe for the patient.
Dr Meneguzzi said: “The aim of this project is to find a good vector which is able to carry the curative DNA into the cells, starting from the knowledge that we have already acquired. We can then identify the patients who will be best suited to the trials.”
The clinical trials will be carried out with at least four RDEB patients and will be a continuation of the Skintherapy project. A separate project application is expected to be submitted in 2008.
There are many potential problems with the treatment such as rejection of the skin but the Skintherapy project, and the subsequent trials, aim to resolve any such issues and make way for a potential treatment.
Although any treatment would be available immediately after a successful trial, that could still be a number of years away.