Life Science Researchers claim major breakthrough in the fight to cure cystic fibrosis

Researchers claim major breakthrough in the fight to cure cystic fibrosis

Nathan Rae has to take up to 60 tablets and undergo extensive physiotherapy every day. Photo: ABC News
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Adelaide researchers believe a major breakthrough in stem cell transplantation therapy could eventually provide a cure for cystic fibrosis.

It could be another 10 years before the research is trialled on people living with cystic fibrosis but if successful, researchers hope it could remove up to 90 per cent of the disease’s complications.

The University of Adelaide’s lead researcher, Dr Nigel Farrow, said it was a world-first method which could “potentially one day cure cystic fibrosis in the airways”.

Working with mice, researchers managed to remove cystic fibrosis stem cells from the lungs, correct them with gene therapy and then successfully reintroduce the now-healthy cells back into the patient.

The healthy stem cells then pass on their genes and replenish the airways with healthy cells, combatting the onset of cystic fibrosis airway disease.

Dr Nigel Farrow believes they have discovered a world-first breakthrough in the fight against cystic fibrosis. Photo: ABC News

“As a scientist, 95 per cent of our work goes in the bin, so any time we get those small breakthroughs it creates a lot of excitement in the lab,” Dr Farrow said.

“[This breakthrough] has not been seen anywhere else in the world.”

So far, researchers have successfully transplanted stem cells for a three-week period but have more work to do before the cells can be transplanted back into the airway for life.

Dr Farrow stressed that it was early days and there was still a lot of additional experiments to be undertaken.

Defying the odds

Nathan Rae, 30, battles every day with cystic fibrosis.

As a baby, his parents were told he wouldn’t survive to adulthood but he has defied all odds to live a relatively healthy life for someone with the disease.

But staying healthy is no easy feat and takes its toll financially too.

Nathan Rae’s parents were told he wouldn’t survive to adulthood. Photo: ABC News

“Every day we take anywhere from 30 to 60 tablets, nebulisers, extensive physio, all sorts of things just to keep us going,” he said.

“We lose a lot of lung function, so just from walking up stairs or tying a shoelace we get pretty knackered pretty quick so it can be tough.

“I’ve had two admissions [to hospital] this year from three weeks to four-week admissions but some people with CF are a lot different. You could have weekly admissions.”

The self-employed construction worker often has to take large amounts of time off work due to cystic fibrosis, stop playing football and stay inside during cold weather.

For three years, a dozen researchers in Adelaide have worked on this new method of stem cell transplantation to fight the debilitating genetic disorder — something that has stumped some international scientists for up to 30 years.

The breakthrough has Mr Rae and others in the cystic fibrosis community cautiously optimistic about a future free from cystic fibrosis.

“I’d give my body up to anything to try and find a cure,” Mr Rae said.

“There’s always hope but there’s always a long way to go to find that cure, hopefully in the next five to 10 years.”

About 3500 people have cystic fibrosis in Australia with a life expectancy of about 37 years.