https://www.gosh.nhs.uk/our-research/our-research-infrastructure/nihr-great-ormond-street-hospital-brc/brc-news/clinical-trial-new-stem-cell-based-therapy-rare-skin-condition-proves-promising/
Clinical trial for a new stem-cell based therapy for a rare skin condition proves promising
9 Sep 2015, 4:52 p.m.
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.
New study finds that nasal cells protect against Covid-19 in children
New research shows that children are less likely than adults to develop severe COVID because cells in their nose are better at fighting off the virus.
New plan announced to get more children access to gene therapy treatments
Great Ormond Street Hospital (GOSH) has announced plans to revolutionise how children living with a rare disease can gain access to life-changing treatments.
First-of-its-kind study into the Ketogenic diet
A ketogenic diet, which is low in carbohydrates and high in fat, shows promise as an alternative treatment option for infants with drug-resistant epilepsy.
Raising awareness on Rare Disease Day
To celebrate Rare Disease Day we invited patients and their families to try out 13 different hands-on and interactive fun and educational science and research activities for children and adults.