Treatment method for autoimmune diseases such as diabetes, multiple sclerosis, atherosclerosis, and rheumatoid arthritis using skin application of a vitamin D analog where topical treatment leads to systemic immunoregulation.
Immunomodulatory drugs are typically administered systemically and often lead to severe side effects. Moreover, intravenous injections of such drugs are burdensome for patients and drive additional cost and risks. Whenever treatment involves biological compounds it further increases the cost and requires special storage, shipping, and handling.
Many inflammatory diseases are mediated by inappropriately activated T cells. Regulatory T cells (Tregs), on the other hand, limit such inappropriate T cell activation, and prevent inflammatory diseases. Dr. Kambayashi has discovered that topical administration of a vitamin D3 analog MC903 leads to the release of keratinocyte-derived thymic stromal lymphopoietin (TSLP) by skin cells into circulation, which then systemically increases the number of Tregs. He further has shown that this Treg expansion leads to attenuation of inflammatory diseases such as type I and type II diabetes, multiple sclerosis, atherosclerosis, and rheumatoid arthritis in mouse models. Thus use of transdermal patch delivering MC903 provides a promising approach for treatment autoimmune and inflammatory diseases.
Taku Kambayashi, M.D, Ph.D.
This technology was featured as PCI's November 2019 Technology of the Month. Read the blog.
- Topical treatment producing systemic effect
- MC903 has limited systemic absorption and half-life preventing systemic toxicities
- MC903 is used for psoriasis treatment (expired patent, available generics)
Patch or other method of transdermal delivery
Stage of Development:
- Mouse models of type I diabetes (NOD mouse)
- Multiple sclerosis (EAE mouse model)
- Atherosclerosis (ApoE KO mouse model)
- Rheumatoid arthritis (serum-induced arthritis)
Leichner et al. J Autoimmun, 2017 May, 79: 39-52.
Docket # 15-7433