Skin-derived ABCB5+ mesenchymal stem cells for high-medical-need inflammatory diseases: From discovery to entering clinical routine
International Journal of Molecular Sciences
School of Medical and Health Sciences
NIH/National Eye Institute (NEI) grants RO1EY025794, R24EY028767 / National Heart, Lung, and Blood Institute (NHLBI) grant 1R01HL161087
The ATP-binding cassette superfamily member ABCB5 identifies a subset of skin-resident mesenchymal stem cells (MSCs) that exhibit potent immunomodulatory and wound healing-promoting capacities along with superior homing ability. The ABCB5+ MSCs can be easily accessed from discarded skin samples, expanded, and delivered as a highly homogenous medicinal product with standardized potency. A range of preclinical studies has suggested therapeutic efficacy of ABCB5+ MSCs in a variety of currently uncurable skin and non-skin inflammatory diseases, which has been substantiated thus far by distinct clinical trials in chronic skin wounds or recessive dystrophic epidermolysis bullosa. Therefore, skin-derived ABCB5+ MSCs have the potential to provide a breakthrough at the forefront of MSC-based therapies striving to fulfill current unmet medical needs. The most recent milestones in this regard are the approval of a phase III pivotal trial of ABCB5+ MSCs for treatment of recessive dystrophic and junctional epidermolysis bullosa by the US Food and Drug Administration, and national market access of ABCB5+ MSCs (AMESANAR®) for therapy-refractory chronic venous ulcers under the national hospital exemption pathway in Germany.
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Niebergall-Roth, E., Frank, N. Y., Ganss, C., Frank, M. H., & Kluth, M. A. (2023). Skin-derived ABCB5+ mesenchymal stem cells for high-medical-need inflammatory diseases: From discovery to entering clinical routine. International Journal of Molecular Sciences, 24(1), Article 66. https://doi.org/10.3390/ijms24010066