Topical and Systemic MSC-based Therapies for Wound Healing in Epidermolysis bullosa (Fuentes 1)
Completed| Project lead | Ignacia Fuentes, PhD |
| Organisation | DEBRA Chile, Ñuñoa, CHILE |
| Partner organizations & collaborators | Clínica Alemana-Universidad del Desarrollo, Chile |
| Project budget | EUR 134,000.00 (EUR 109,000.00 DEBRA France/EUR 25,000.00 DEBRA Austria) |
| Start date / Duration | 01. Sep 2021 / 18 months |
| Funder(s) / Co-Funder(s) | DEBRA Austria, EB MSAP/EBEP Recommended, DEBRA France |
| Research area | Symptom prevention & relief |
Project details
Short lay summary
Wounds are a major problem for all EB patients. They can become chronic, infected and can even develop into skin cancer. These last years a special type of stem cells, called Mesenchymal Stem Cells or MSC, has been used in EB trials because of their characteristics for healing. Although these trials showed they are safe, the administration route failed to show a clear benefit for wound healing. In this project we will test two novel strategies, both utilizing MSC and animal models. One strategy consists of using MSC covered in a polymer to be applied directly into wounds, which will result in a localized but also painless treatment. The second strategy is to apply these cells into an artery blood vessel, which will reach large areas of body. Results from this project will pave the way to design better and painless trials to effectively treat wounds in EB patients.
Scientific summary
Impaired wound healing is a major problem in EB patients. If not treated well or on time, non-healing wounds can lead to infections, scarring, pain and even become an aggressive type of skin cancer. On the other hand, Mesenchymal stem cells (MSC) are a special kind of stem cell that has shown a huge potential in the field of regenerative medicine. These cells have previously been shown to be safe for their use in EB patients, and very effective for wound healing when injected at the wound border, allowing even an increase in type VII collagen in healed RDEB skin. However, intradermal injections are also too painful to be used in routine clinical practice for EB patients. In this study we propose to use MSC to improve wound healing, either locally in a biodegradable scaffold applied directly into wounds or systemically through an intra-arterial injection. Preliminary results show a great potential of these two strategies and more importantly, them to be safe. To go further with the characterization and functionality of these strategies for EB wounds, we decided to test them in vivo, utilizing EB mouse models and wild type rats. Results from this project will pave the way to design better and painless trials to effectively treat wounds in EB patients.
Strategic relevance
If one or both of our strategies continue showing great potential as EB wound healing therapies after completing this project, we will proceed firstly with the protection of the invention and secondly a pilot or proof of concept study in EB patients.
What did this project achieve?
Despite not finishing all experiments within the proposed timeline, the group is enthusiastic about the findings and eager to continue investigating this promising topic towards finding a cure for EB wound healing. The research regarding the topical formulation demonstrated that MSC can survive for several days while retaining their beneficial properties within the scaffold. Furthermore, the results indicate that MSC can migrate out of the scaffold and penetrate into the dermis when applied to wounds. The research group is currently in the process of further characterizing the benefits of this formulation using animal models of EB. On the other hand, the systemic approach involving arterial MSC injection in a rat model proved to be safe and effective, with MSC successfully reaching the wound site. This confirms the potential of intra-arterial injection as a systemic delivery method for MSCs. Overall, the findings strongly support the beneficial role of MSC in EB wound healing.