RHEACELL >< EB-Trial

Mode of action of ABCB5+ MSCs in EB

ABCB5+ MSCs are infused into blood streams

ABCB5+ MSCs can migrate to the wounds, where they decrease inflammation and promote wound healing

ABCB5+ MSCs target all wounds – internal and external

ABCB5+ MSCs release and deposit collagen VII

Anti-inflammation as the basis of wound healing

ABCB5+ MSCs have an anti-inflammatory potential by interacting with surrounding immune cells and initiating the reprogramming. The release of interleukin-1 receptor antagonist (IL-1RA) induces a shift from a pro-inflammatory (due to the presence of M1 macrophages) to an anti-inflammatory stage (with pro-healing M2 macrophages). In addition, collagen VII and the structural proteins laminin and keratin 14 are produced, which are also stored in the wound and thus support its healing³.

ABCB5+ MSCs: The therapeutic concept in EB

Reducing the burden of the disease as an important goal

The completed phase 2a trial with ABCB5+ MSCs in 16 RDEB patients proved the general anti-inflammatory effect and showed a significant reduction in number and severity of wounds. In addition to the recorded wound healing potential, a significant reduction in itch and pain did improve the general disease burden in these patients⁴. Larger, pivotal Phase 3 studies are now important to further substantiate the benefit-risk profile in a larger patient population.

Even though the effects seen in the first clinical trial are very promising, due to the genetic nature of this condition, a full cure is unlikely to be seen in the near future. Nevertheless, preclinical studies in mice suggest that ABCB5+ MSCs may have the potential to slow or halt disease progression, according to the published findings of an internationally renowned research group⁵.

Hope for the future of patients with EB starts with research

More research on the efficacy, tolerability, and safety as well as possible interactions of ABCB5+ MSCs is in progress. A pivotal (phase III) trial with 74 patients with severe forms of EB starts in various renowned study centers across Europe (EU and UK), Israel, Chile, Argentina, and the US. The study is recruiting and looking for patients (children and adults) with a confirmed diagnosis of recessive dystrophic EB (RDEB) or junctional EB (JEB).

If you are interested in participating, talk to your doctor about this clinical trial, whether you meet the inclusion criteria, and which study site would be suitable for you.

Find study centers here

1 Kerstan A, et al., (2022). Allogeneic ABCB5+ mesenchymal stem cells for treatment-refractory chronic venous ulcers: a phase I/IIa clinical trial. JID Innov. 2022 Jan;2(1):100067. doi: 10.1016/j.xjidi.2021.100067. Epub 2021 Oct 25. PMID: 34870260; PMCID: PMC8635035.

2 Instructions for use and expert information AMESANAR^®

3 Vander Beken S. et al., (2019). Positive Dermal Mesenchymal Stem Cells Promote Healing of Chronic Iron-Overload Wounds via Secretion of Interleukin-1 Receptor Antagonist. Stem Cells. 2019 Aug;37(8):1057-1074. doi: 10.1002/stem.3022. Epub 2019 May 13. PMID: 31002437; PMCID: PMC6663647.

4 Kiritsi D. et al., (2021). Clinical trial of ABCB5+ mesenchymal stem cells for recessive dystrophic epidermolysis bullosa. JCI Insight, 2021;6(22): 51922. https://doi.org/10.1172/jci.insight.151922.

5 Webber B.R. et al., (2017). Rapid generation of Col7a1-/- mouse model of recessive dystrophic epidermolysis bullosa and partial rescue via immunosuppressive dermal mesenchymal stem cells. Lab Invest. 97(10):1218-1224.