6 October 2021
October 2021, Brno – An academic clinical trial phase I/II for patients with rare congenital epidermolysis bullosa (also known as butterfly wing disease) is about to start in the Brno University Hospital. Thanks to the cooperation with Masaryk University and the patient organisation DEBRA ČR, patients will receive cell therapy developed over several years by experts from the Advanced Cell Immunotherapy Unit (ACIU) at the Department of Pharmacology, Faculty of Medicine. The aim of the trial is primarily to verify the safety of cell therapy injected into chronic wounds. Scientists and physicians are also interested in the first information about the extent of wound healing promotion. During five years, 15 patients will participate in the study. Team of doctors and nurses led by MUDr. Jitka Vokurková, PhD from the Department of Burns and Plastic Surgery and prim. MUDr. Hana Bučková, PhD from the EB Centre at the Children’s Dermatology Department of the Paediatric Clinic will care for the participants. The study was organised with the support of research infrastructure CZECRIN and the project CZECRIN_PRO PACIENTY funded by the European Regional Development Fund.
In the first part of the study, cell therapy will be given in two doses to adult patients enrolled at the turn of 2021 and 2022. After safety assessment in the next year, the recruitment can be opened to paediatric patients. Children will successively participate in defined age categories as required by regulatory authorities in these early stages of clinical research. State Institute for Drug Control has authorised the clinical trial in May 2021.
According to the previous preclinical and clinical research, mesenchymal stromal/stem cells (MSCs) that will be administered to trial participants have healing potential for skin lesions of various origins. MSCs are physiologically present in the human body and repair damaged tissues. They also affect the immune system’s functions by suppressing exaggerated inflammatory response occurring in chronic wounds. MSCs are found abundantly in the bone marrow. Recently, adipose tissue has been shown to be a rich source of MSCs, as well. This tissue is available as a waste product after liposuction, and it is usually disposed of without further use.
The experts from ACIU adapted a method for obtaining MSCs from adipose tissue collected by liposuction and developed a set of laboratory tests to verify the microbiological and immunological quality of the manufactured medicinal product. Adipose tissue for the development and production of cell therapy was donated by volunteers and collected at the Department of Burns and Plastic Surgery. If the safety and effect of injectable cell therapy prove successful, researchers will continue with a less invasive method. In cooperation with the Technical University of Liberec (TUL), they are already developing wound dressings that combine the benefits of advanced nanomaterials developed by TUL and cell therapy produced at ACIU.
About Epidermolysis bullosa
Epidermolysis bullosa is a genetic disease caused by mutations in genes for specific connective tissue proteins. Because of mutations, the cohesion of individual layers of the skin, mucous membranes and internal organs is disturbed. Causal treatment is not available. Therapy is mainly focused on caring for very fragile skin, wound cleansing, healing support and prevention of infection.
ACIU is a part of the Department of Pharmacology, Faculty of Medicine at Masaryk University. The Unit operates in the good manufacturing practice (GMP) regimen. Clean rooms for the manufacture of cell therapy medicinal products are available and regularly inspected by the State Institute for Drug Control. ACIU cooperates intensively with the Transfusion and Histic Treatment Department and the Department of Clinical Microbiology and Immunology of the Institute of Laboratory Medicine, University Hospital Brno, in cell therapy research and development projects.