Evaluation of cell therapy developed by the CZECRIN infrastructure for patients with butterfly skin disease is underway at the Brno University Hospital
An academic clinical trial for patients with the rare congenital disease epidermolysis bullosa (EB), also known as butterfly skin disease, has been initiated in collaboration between the Faculty of Medicine of Masaryk University in Brno and the University Hospital Brno. The aim of the clinical trial is to test the drug's safety and obtain the first information on the extent to which the administered cell therapy promotes the healing of chronic wounds.
The cell therapy drug consists of so-called mesenchymal stromal cells (MSCs). These cells help repair damaged tissues and direct the exaggerated functions of the immune system of the human body. 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 Department of Pharmacology FM MU, have been working on the development of the product for several years, preparing MSCs from the adipose tissue of healthy volunteers in a "clean room" (GMP) environment.
The clinical study is being carried out by a team of doctors and nurses from the Department of Burns and Plastic Surgery of the Brno University Hospital and the EB Centre at the Children's Dermatology Department of the Department of Paediatrics of the Brno University Hospital. In mid-October 2022, the drug was administered to the first patient. After the necessary 24-hour observation, the man was discharged home from the hospital in good condition and will regularly attend further check-ups and repeat administration of the drug. He will be followed in the study for one year. By the end of 2022 and early 2023, three more patients will be administered the drug, after which an initial safety analysis of the treatment will take place and, if the results are favourable, additional patients will be enrolled in the study.
The above-mentioned clinical study was supported by the patient organisation DEBRA ČR, z.ú.
Epidermolysis bullosa
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, cleaning wounds, promoting healing, and preventing infection.
ACIU
The ACIU of the Department of Pharmacology of the Faculty of Medicine of Masaryk University is operated under the Good Manufacturing Practice (GMP) regime, has clean rooms for the production of cell therapy medicines, and is regularly inspected by the State Institute for Drug Control. In cell therapy research and development projects, the ACIU cooperates intensively with the Transfusion and Tissue Department, the Department of Clinical Microbiology and Immunology of the Department of Laboratory Medicine, and the clinical departments of the University Hospital Brno.
CZECRIN
CZECRIN is a research infrastructure supporting the implementation of academic clinical research in the Czech Republic. As the Czech node of the European Network of Clinical Research Infrastructures ECRIN-ERIC, it contributes substantially to the involvement of academic institutions in international clinical research projects.
