Children with the rare genetic disease Epidermolysis Bullosa face a lifetime of pain due to constant blistering of their skin and other body surfaces. But a new therapy calms inflammation in ‘butterfly’ skin.
The University of South Australia’s Dr Zlatko Kopecki has developed a product to help these kids. The product could potentially treat all people with inflammatory skin conditions.
“We have identified a harmful protein that impairs skin healing in these so-called ‘butterfly children’, and created a new product to address this,” explains Kopecki.
“More broadly, the new therapy we have developed may improve recovery from all kinds of wounds.”
Epidermolysis Bullosa occurs due to failure in scaffold-like structures that link skin cells to each other. With the normal protective barrier to the outside world now leaky, the child’s immune system is forced onto on a never-ending circuit of high alert and repair.
“If the children manage to survive the numerous infections they endure in early childhood, they die from skin cancers induced by this constant cycle,” says Kopecki.
The new therapy for Epidermolysis Bullosa dampens harmful inflammation in the skin by blocking the activity of a protein known as Flightless.
“When extracellular Flightless protein is mopped up by specific neutralising antibodies we have developed, it results in improved healing of blistered skin and improved cellar migration,” says Kopecki.
The effectiveness of the antibody in reducing skin inflammation in mice is described in a recent paper published by Kopecki with colleagues at the Women’s and Childrens’ Health Research Institute, University of South Australia and University of Adelaide.
The researchers are now focused on transitioning this finding to create a new therapy that works in humans.
“We hope to run our first clinical trials in 2016 and aim to develop a marketable product within five years,” says Kopecki.
The new therapy would be a welcome relief for the 500,000 people worldwide who suffer from Epidermolysis Bullosa.
It may also open up new opportunities to treat impaired skin healing due to diabetes, aging, burns and skin blistering, which together cost the Australian federal government in excess of AU$2.6 billion per year.
Kopecki presented his research at Fresh Science South Australia 2015. Fresh Science is a national program that helps early-career researchers find and share their stories of discovery.
– Sarah Keenihan
This article was first published on 5 November 2015 on The Lead and was also shared by Science in Public.