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Topical and Systemic MSC-based Therapies for Wound Healing in Epidermolysis bullosa (Fuentes 1)

Ongoing
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, 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.


Keywords

Fuentes
DEBRA Chile
stem cells
mesenchymal stem cells
MSC
wound healing
Woundhealing
RDEB
JEB
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