For people living with Epidermolysis bullosa, any potential form of relief is welcomed and the fact that there may now be a new treatment option available is great news.
Epidermolysis bullosa is a rare genetic skin disease that is often referred to as ‘butterfly skin’, as a sufferer’s skin is so sensitive that it is equivalent to a butterfly’s wings. Children who suffer from this condition have been given the moniker ‘butterfly children’ and suffer greatly, as the disease is very painful.
A possible new treatment is currently being tested in Spain. Should this prove successful, those with Epidermolysis bullosa could see their lives greatly changed.
The aim of the new trial is to evaluate how mesenchymal cells derived from bone marrow can be used to improve any wounds caused by the condition. The trial has been promoted by the Foundation for Biomedical Research of La Paz University Hospital and clinical trial assistants with special skills, perhaps recruited from providers such as http://www.gandlscientific.com/clinical-trial-assistants/, will oversee the process. The trials will be carried out by the Madrid Hospital’s Department of Haematology and Dermatology, with all parties involved hoping for a good result.
A chance for improvement
Not only is Epidermolysis bullosa incredibly painful and debilitating but also it can be fatal, with some patients given a life expectancy of only 30 years. The trial has been approved by the Spanish Agency of Medicines and Sanitary Products and 75 per cent of the cost has been financed by the Carlos III Institute of Health; meanwhile, a patients association by the name of DEBRA The Butterfly Children Charity will carry the other 25 per cent of the costs.
DEBRA will also be covering the costs of a molecular geneticist for the period of one year, which will give butterfly children better access to a generic diagnosis and provide a greater insight into the root causes and lineage of their condition. This insight is incredibly valuable to both the patient and their families, as it defines the strain of Epidermolysis bullosa from which they suffer and provide incredible prognostic value. The affected can also use the results of their genetic testing for future family planning and to map their future in the hope that the condition will not be passed down to another generation.