An academic clinical trial for patients with the rare congenital disease epidermolysis bullosa (EB), also known as “butterfly children” disease, has been initiated in collaboration between the CZECRIN infrastructure (MU Faculty of Medicine) and the University Hospital Brno. The aim of the clinical trial is to test the safety of the drug and to obtain the first information on the extent to which the cell therapy administered promotes healing of chronic wounds.
The clinical trial is being conducted by a team of doctors and nurses from the Burns and Plastic Surgery Clinic of the Brno University Hospital and the EB Centre at the Children’s Dermatology Department of the Paediatric Clinic of the Brno University Hospital. In mid-October 2022, the drug was given to the first patient. After the necessary 24-hour observation, the man was discharged home from the hospital in good condition and will have regular follow-ups and repeat applications. He will be monitored in the study for one year. By the end of 2022 and early 2023, three more patients begin with the treatment. An initial safety analysis of the treatment will then take place and, if the results are favourable, additional patients will be included in the study.
The cell therapy drug consists of mesenchymal stromal cells (MSCs). These cells in the human body help repair damaged structures and regulate the exaggerated functions of the immune system. According to existing research, MSCs have healing potential in acute and chronic wounds of various origins. Experts from the Advanced Cell Immunotherapy Unit (ACIU) at the Institute of Pharmacology of Faculty of Medicine (MUNI) have been working on the development of the product for several years, preparing the MSC medicine from fat tissue of healthy volunteers in a “clean room” (GMP) environment. The above-mentioned clinical study is supported by the patient organization DEBRA CZ, z.ú.
Epidermolysis bullosa is a genetic disease caused by changes (mutations) in genes for specific connective tissue proteins. As a result of the mutations, the cohesion of the different layers of the skin, mucous membranes and internal organs is disturbed. Causal treatment is not available; patient care is mainly focused on caring for very fragile skin, keeping wounds clean, promoting healing and preventing infection.
Source – press release CZECRIN