Promising results for stem cell treatment of RDEB

Promising results noted from the latest clinical trial into the use of stem cells to treat recessive dystrophic epidermolysis bullosa.

We take some time to talk to our in-house expert; Osama Shaikh , to learn more about this progressive new trial.

We have seen some very promising results from the latest clinical trial for a new stem-cell based therapy for a rare and debilitating skin condition; recessive dystrophic epidermolysis bullosa. The treatment which involved the infusion of stem cells was found to provide pain relief whilst also reducing the severity of the skin condition.

Recessive dystrophic epidermolysis bullosa (RDEB) is an extremely painful skin disease; even the smallest skin injury will lead to blisters and wounds which never fully heal. There are around 1,000 people in the UK currently living with this atrocious disease. The fragile skin caused by RDEB scars, develops contractures and is prone to life-shortening skin cancers. Unfortunately, at the present time there is no cure for RDEB.

This latest clinical trial, led by King’s College London in collaboration with Great Ormond Street Hospital, recruited 10 children with RDEB. Each participant received 3 infusions of mesenchymal stem cells (MSCs) from unrelated donors. They were then closely monitored for a year after the stem cell infusions.

Dr Gabriela Petrof, of King’s College London said; We found that wound healing improved in all 10 children and the skin was less inflamed. Many of the kids also mentioned their skin was less painful and itchy, and their parents noted how much more energetic they were.

Professor John McGrath, also of King’s College London said, In 25 years of my research on RDEB, this is the only time I have seen any treatment change the nature of the condition. The outcomes are truly impressive and injecting the MSCs raised no safety concerns. At the same time, it’s important to note that this is not a cure and that the benefits wear off after about six months

Overall, outcomes of the trial are promising. However, further work is needed to better understand the mechanisms involved in stimulating wound healing and reducing inflammation in the skin. Additional studies will also be required to confirm the effectiveness of this treatment and to establish the optimal dose of cells to use for RDEB patients.

For more information on this new trial visit: Stem cell therapy inherited skin

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