Low-dose calcipotriol can elicit wound closure, anti-microbial, and anti-neoplastic effects in epidermolysis bullosa keratinocytes

Scientific Reports, 2018 · DOI: 10.1038/s41598-018-31823-6 · Published: September 12, 2018

Simple Explanation

This study investigates the potential of calcipotriol, a vitamin D3 analog, to improve wound healing in patients with recessive dystrophic epidermolysis bullosa (RDEB). RDEB is a genetic skin disorder characterized by fragile skin and chronic wounds. The researchers found that low doses of calcipotriol can promote wound closure, fight microbes, and prevent tumor growth in RDEB skin cells grown in the lab. These findings suggest that calcipotriol could be a helpful treatment for RDEB patients. A single-patient observation showed that a low-dose calcipotriol ointment led to complete wound closure and improved skin bacteria diversity in a patient with a related skin condition, indicating potential benefits and safety.

Study Duration

One month

Participants

Single 75-year-old dominant dystrophic EB (DDEB) patient and in vitro cell lines

Evidence Level

Single-patient observation study and in vitro experiments

Key Findings

1
Calcipotriol treatment enhanced wound closure in RDEB cell monolayers, with up to a 2-fold increase observed in scratch closure rates.
2
The secretome of calcipotriol-treated cells exhibited increased antimicrobial activity, specifically against C. albicans, indicating enhanced innate immune defense.
3
Calcipotriol demonstrated anti-neoplastic effects by suppressing the clonogenicity and proliferation of RDEB tumor cells.

Research Summary

The study demonstrates that low-dose calcipotriol can induce the expression of cathelicidin, enhance wound closure, and increase local antimicrobial defense in RDEB keratinocytes in vitro. A single-patient observation study showed that low-dose calcipotriol ointment improved wound healing and reversed skin microbial dysbiosis in a DDEB patient. Calcipotriol also exhibited anti-neoplastic potential against RDEB tumor cells, suggesting a comprehensive therapeutic benefit for RDEB patients.

Practical Implications

Therapeutic Potential for RDEB

Calcipotriol may represent a vital therapeutic option for RDEB patients due to its wound healing, anti-microbial, and anti-neoplastic effects.

Repurposing Existing Medications

The study supports the re-purposing of calcipotriol, already approved for psoriasis, for off-label use in RDEB wound management.

Enhancing Innate Immune Response

Calcipotriol's ability to enhance the local antimicrobial defense of RDEB keratinocytes could significantly benefit patients susceptible to wound infections.

Study Limitations

1
In vitro systems cannot fully replicate the wound healing process.
2
The effects of calcipotriol were robust in only a subset of RDEB cell lines, indicating potential variability in patient response.
3
Single-patient observation study lacks a placebo control and may not be generalizable.

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