Epidermolysis Bullosa (EB)
Clinical Trial Phase I/IIa
Epidermolysis bullosa (EB) is an inherited, clinically and genetically heterogenous severe skin disorder. The disease affects about 500,000 individuals worldwide (Rashidghamat and McGrath, 2017). EB patients suffer from extremely fragile skin and mucosa, caused by cytoskeletal disorganization followed by the loss of anchorage of the epidermis to the underlying dermis. Thus, even a minor mechanical trauma or friction can lead to blisters, erosions and open, slowly healing wounds (Boeira et al., 2013). The life expectancy of EB patients is highly dependent on the subtype, inheritance pattern and severity of the disease. In its severest forms, EB can lead to death already in early childhood or increase the risk of dying from metastatic skin carcinoma in early adulthood (Rashidghamat and McGrath, 2017).
At present there is no effective therapy or cure for EB, so current treatment options are primarily preventive and supportive (Watkins, 2016). Since EB is characterized by devastating tissue damage accompanied by severe inflammation (Cianfarani et al., 2017), mesenchymal stem cells (MSCs) could offer a new chance for these patients, due to their immunomodulatory and anti-inflammatory properties. Current literature and clinical trials (Conget et al., 2010, El-Darouti et al., 2015, Petrof et al., 2015) support the clinical benefits and safety of MSCs in EB patients.
First evidence for efficacy of ABCB5-positive (ABCB5+) MSCs in recessive dystrophic EB (RDEB), a particularly severe EB subtype, has been provided by the research group around Jakob Tolar (University of Minnesota, Minneapolis, USA) (Webber et al., 2017). In various preclinical models the safety of ABCB5+ MSCs could be shown.
The clinical trial investigates the efficacy and safety of allogeneic ABCB5+ MSCs in Patients suffering from RDEB. HERE you can find more information about this clinical trial.