Stem cell therapy boost for cystic fibrosis treatment
Health & Medical
Researchers in South Australia have shown that cells causing cystic fibrosis airway disease can be successfully replaced with healthy ones using cell transplantation therapy.
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The University of Adelaide research published in the journal Stem Cell Research and Therapy applies cell transplantation therapy, normally used in bone marrow transplants to treat immunodeficiency disorders.
“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 Nigel Farrow, a Post-Doctoral Research Fellow from the University of Adelaide’s Robison Research Institute.
“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 combatting the onset of cystic fibrosis airway disease.”
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.
“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,” said Dr Farrow, who conducted the study alongside Associate Professor David Parsons from the Women’s and Children’s Hospital in Adelaide.
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.
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.
The pioneering 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,” said Dr Farrow.
“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.”
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