A Phase III Clinical Trial on Epidermolysis Bullosa Treatment with Regenerative Medical Products Initiated

Dr. Akira Ishiko
Professor and Chairman
Department of Dermatology
Toho University Omori Medical Center

The Department of Dermatology at Toho University Omori Medical Center has started a clinical trial (Phase III: validation clinical trial) using regenerative medical products for patients with epidermolysis bullosa. The department conducted an exploratory clinical trial on patients in 2017. At present, it is a validation study aiming for regulatory approval.

Epidermolysis bullosa is a hereditary disease that causes blister, erosion, and ulcer formation all over the body, especially in areas susceptible to mechanical stimulation; the pathogenesis is linked to a congenital deficiency, or an extreme lack of factors important to the adhesion between the epidermis and dermis, such as type VII collagen. Since it persists throughout life, it has been labelled as an intractable disease by the Ministry of Health, Labor and Welfare.

The treatment under investigation involves the application of a regenerative medical product containing mesenchymal stem cells derived from adipose tissue of a healthy donor to the ulcer site. This is expected to supply the affected areas of skin with various growth factors that promote skin formation from other stem cells, as well as type VII collagen, which is lacking in these patients, to the lesions, thereby promoting ulcer healing and improving skin fragility.

The Department of Dermatology at Toho University Omori Medical Center has started a clinical trial (Phase III: validation clinical trial) using regenerative medical products for patients with epidermolysis bullosa. The department conducted an exploratory clinical trial on patients in 2017. At present, it is a validation study aiming for regulatory approval. Epidermolysis bullosa is a hereditary disease that causes blister, erosion, and ulcer formation all over the body, especially in areas susceptible to mechanical stimulation; the pathogenesis is linked to a congenital deficiency, or an extreme lack of factors important to the adhesion between the epidermis and dermis, such as type VII collagen. Since it persists throughout life, it has been labelled as an intractable disease by the Ministry of Health, Labor and Welfare. The treatment under investigation involves the application of a regenerative medical product containing mesenchymal stem cells derived from adipose tissue of a healthy donor to the ulcer site. This is expected to supply the affected areas of skin with various growth factors that promote skin formation from other stem cells, as well as type VII collagen, which is lacking in these patients, to the lesions, thereby promoting ulcer healing and improving skin fragility.