DEVELOPING A SINGLE DOSE TREATMENT FOR CYSTIC FIBROSIS
Researchers from the Women’s and Children’s Hospital are one step closer to finding a treatment for cystic fibrosis that could last for most of a person’s lifetime.
The researchers have developed and tested a special method for preparing airways in mice to accept a gene that could effectively counteract the severe lung disease.
This novel therapy uses a modified virus particle to carry in the ‘wanted’ gene and is designed to reach airway stem cells to produce a very long lasting gene transfer.
Chief Medical Scientist in the Respiratory and Sleep Medicine Department, Dr David Parsons, his colleague Dr Don Anson, and PhD student Alice Stocker have for the first time discovered that a single application of this unique gene treatment can last for most of the mouse’s lifetime, and in a few cases for their entire life.
Dr Parsons says this break-through is a promising step towards finding an effective treatment.
“Imagine if you had a drug that you could take once, say for your severe asthma that would fix your asthma for most of your life – that is what we are aiming for here, we’re using gene therapy as a CF lung disease treatment,” Dr Parsons said.
“There is no cure yet for cystic fibrosis (CF). It's a life-long disease and that means any treatments for this genetic disease will need to work for a lifetime.
“For most diseases like CF, this means treatment has to be given on a regular basis.
“We think we are now getting the ‘wanted’ gene into the stem cells in the lung, which effectively makes the gene therapy last forever because all the ‘daughter’ cells produced from those stem cells also carry the gene. We are excited by these findings because this might mean that only one effective treatment is required.
“This is especially important in cystic fibrosis as the lungs have evolved to reject any repeated attempts to transfer genes, even the beneficial ones needed to treat CF.
“So far, this unique method has only been tested in mice. Although we know the same gene transfers can work immediately in other animal lungs, we now need to see if it also works safely into ‘old age’ in animals with more human-like lungs. This would give us confidence to move towards clinical trials.”
The research findings have been published in the Journal of Gene Medicine this week.
“There is still a large amount of work that needs to be done before we can say our latest findings could form the basis for a cure for cystic fibrosis but we are really encouraged by these results,” Dr Parsons said.
“This work has taken 15 years of research to get to this point, with major funding from the National Health & Medical Research Council, as well as CF Associations in Australia and the USA. Without the generous financial and resource support from corporate and personal sponsors via the Cure4CF Foundation, and the Women’s and Children’s Hospital Foundation, we would never have made it this far.”
Cystic fibrosis is an inherited condition that causes severe, unrelenting lung disease in many children and adults world-wide. Approximately, 1 in 2,500 infants are born with this disease and only half survive past 35 years of age.
For more information on the Cure4CF Foundation go to www.cure4cf.org and for more information on the WCH Foundation visit www.wchfoundation.org.au