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Clinical study shows potential of specific mesenchymal stem cells for symptom relief in RDEB

New scientific publication with EB House contribution

In recent years, RDEB has been recognized as a disease of systemic inflammation and multi-organ involvement rather than a skin-limited disorder, for which no curative therapy exists. Thus, the focus of intensive research is the development of strategies that alleviate symptoms but also prevent irreversible skin and organ damage. Such disease-modifying therapies include the application of small molecule drugs, or the transplantation of bone marrow-derived stem cells.

A group of international EB researchers recently investigated the potential of a specific type of skin-derived mesenchymal stem cells (MSC) for the treatment of RDEB. These MSC, namely ABCB5+ MSC, have the ability to migrate to injured tissue sites when intravenously injected, and possess capacities to reduce inflammation, promote wound healing, and to a certain extent secrete collagen 7 protein. In order to prove the efficacy and safety of an ABCB5+ MSC therapy, a German company together with EB experts performed an international, multicentric clinical trial. 16 RDEB patients between 4 and 36 years, all showing severely affected skin and multiorgan involvement, were included in the study. Each patient received three infusions of a defined amount of MSCs on days 0, 17 and 35. Patients were followed-up for 12 weeks to evaluate efficacy and 12 months to monitor safety.

Two scoring systems specifically developed for EB (iscorEB and EBDASI) were used to measure treatment outcome. These tools allow to assess disease severity and classifying clinically significant change in disease activity and disease‐induced skin and organ damage.

During the 12 weeks follow-up, all patients showed a clinically meaningful decrease in disease activity, associated with a reduction of itch and pain from day 35 after the first injection. The damage score remained unchanged, indicating that no further skin and organ damage occurred during follow-up. In addition, most patients tolerated the treatment very well. Only few suffered from severe side effects such a hypersensitivity reactions, which, however, could be resolved rapidly and without sequelae.

It must be mentioned however, that the improvement in disease severity could be due to other factors such as the additional care during the trial and natural fluctuations in disease symptoms during the short follow-up period. Therefore, further clinical evaluation with a larger number of patients, repeated infusions over a longer period of time, and an extended follow-up period is needed, to confirm the potential benefit of systemic ABCB5+ MSC treatment. Such a trial is currently planned and will include an updated protocol based on knowledge gained during the above mentioned trial.

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