Clinical trial for a new stem-cell based therapy for a rare skin condition proves promising

Promising findings from a trial for a new stem-cell based therapy for a rare skin condition have been published in the Journal of Investigative Dermatology. The study, which involved intravenous injections of stem-cells, has led to an improvement in the quality of life for the subjects and their carers.

Recessive dystrophic epidermolysis bullosa (RDEB) is an inherited skin disease which results in the severe blistering of the skin. It is caused by a mutation in the protein, COL7A1, which leads to reduced anchoring of the upper skin layers to deeper tissue. Currently there is no effective cure for this condition.

The clinical trial, which recruited 10 patients with RDEB, was led by Professor John McGrath at Kings College London and BRC-supported Principal Investigator Dr Anna Martinez at GOSH. The patients were followed up for one year following the stem cell infusions, which took place three times over a six month period. Questionnaires revealed an improvement in skin healing following blistering and a reduced pain score after therapy. The severity of the condition has also reported to be lessened.

Overall, the results from this study appear promising. While the patients in this study were not blinded, which the researchers’ state could lead to a positive bias towards the new treatment, patient benefits such as better sleep and reduced caring needs remains a positive from this study. 

Read the full press release here.