New hope in fight against cystic fibrosis

CYSTIC fibrosis sufferers could soon be free of life-threatening lung disease, thanks to pioneering research from the University of Adelaide.

Scientists have shown that CF-affected cells in the airway could be successfully replaced with healthy ones modified through gene therapy.

Dr Nigel Farrow, who gave up a career as a musician and went back to university to become a researcher after his 10-week-old daughter was diagnosed with cystic fibrosis, said the breakthrough could mean an eventual treatment to prevent lung disease in children with CF and an effective treatment for adults with the disease.

CF affects a person's lungs and digestive system causing a build-up of mucus that seriously impairs their breathing and significantly increases the chances of chest infections.

Dr Farrow, a post-doctoral research fellow, and researchers including a team at the Women's and Children's Hospital, applied stem cell transplantation therapy, normally used in bone marrow transplants to treat immunodeficiency disorders, to treat diseased cells in the lungs of patients with CF.

Working in mouse airways, the group successfully tested their new transplantation method, using a marker gene in place of the corrective CF gene in initial studies.

Dr Farrow hopes ta treatment could be available for CF patients within 10 years.

"Our research, which applies stem cell transplantation, involves harvesting adult stem cells from the lungs of CF patients, correcting them with gene therapy, and then reintroducing those cells back into the patient," said Dr Farrow.

"The new transplanted adult stem cells pass on their healthy genes to their 'daughter cells' providing a constant means to replenish the airways with healthy cells, and thereby combating the onset of cystic fibrosis airway disease."

In Australia, one in every 2500 babies has CF, and one in 25 people carry the defective gene. Even though carriers are not affected by the disorder, they may pass the gene to their children.

If both parents are carriers, each of their children has a 1 in 4 chance of being born with the disorder.

"There are 70,000 people worldwide living with CF for which there is currently no cure, and disease in the lungs is the major cause of poor health and a significantly shortened life span," Dr Farrow said.

"The key to these successful transplantations was our innovative method; we first eliminated the existing surface cells, which then created the space required to introduce the new cells."

The research demonstrates that, in principle, human airway stem cells can be transplanted into the lining of the lungs.

"If we can perfect this technique, it will accelerate this exciting research which could significantly improve the lives of those living with cystic fibrosis and potentially combat this chronic life-limiting illness," Dr Farrow said.

The research was published in the journal Stem Cell Research and Therapy.

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