Scleroderma – rapid skin improvement seen after treatment with fresolimumab

A treatment breakthrough for total body scarring of the skin that can occur in people with systemic sclerosis (scleroderma) could be on the cards thanks to the findings of new research published in the Journal of Clinical Investigation.
Experts worked with 15 people with scleroderma who were treated with either one or two doses of fresolimumab, a new, unapproved drug that targets a chemical mediator in the body known as TGF-beta.
After seven weeks’ of treatment, the researchers examined the effect on skin scarring and on the expression of molecular markers in the skin. In both clinical and molecular evaluations these patients showed profound decreases in skin scarring.
TGF-beta has long been implicated in causing scarring but this is the first clinical study to clearly show its impact on humans.
In the current study the drug was used for seven weeks, but 24 weeks later the improvements were lost – so fresolimumab may need to be used longer term for its positive effects to be enjoyed.
“Fresolimumab has been well tolerated in several trials at higher doses than those used in this study and multiple doses,” say the authors, but it can have side effects, which would need to be carefully monitored, particularly if the drug were used for longer periods of time.
“Our study shows that TGF-beta plays a critical role in skin scarring in patients with systemic sclerosis,” explains Prof Robert Lafyatis, who is involved in the work. “Our results strongly indicate that targeting the TGF-beta in these patients will block skin scarring.”
The next step is a longer study involving more people and comparison with a placebo (pretend drug).

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Click here to read the original research.