Under The Microscope - Research Update

Promising clinical trial findings provide hope for Adelaide research program

Adelaide researchers are one step closer to finding a cure and treatment for the lung disease in cystic fibrosis, thanks to a significant breakthrough in the UK. This development also validates the need for an increase in funding to fast track the highly promising local research program that Cure4CF Foundation supports in Adelaide.

The findings published in July by the UK Cystic Fibrosis Gene Therapy Consortium revealed that when a healthy copy of the defective CF gene is transferred into a patient’s airways, lung function can improve by an average of 3.7 per cent. This was enough to stabilise CF lung disease and may prevent the otherwise steadily decline of lung function over a lifetime.

This is the first time that a gene therapy had been shown to correct CF lung disease in human clinical trials. And, unlike the latest medications used to treat CF, which are dependent on the particular CF mutation a person has, this type of treatment would benefit anyone with CF.

The UK study focused on a liposome-based gene vector. In this vector, the healthy CF gene is contained in tiny fat globules called liposomes, which were administered monthly over a year.

In contrast, the Adelaide research team has developed an alternate and more potent approach, using their locally developed and highly effective lentivirus-based gene vector. The most exciting aspect of the Adelaide research is that, although a lentivirus method should produce gene therapy treatments that last far longer than those from a liposome, it can also be a potential cure for the airway disease in CF because it is specially designed to also correct the airway stem cells.

This means the new airway cells that continually rejuvenate the airway tissue will already and always have the corrected gene operating within them.

The UK findings will accelerate the development and testing of much more effective gene vectors, and the lentivirus-based gene vector developed by the Adelaide research group is currently leading the field in the ability to alter CF airway health in animals.

Cure4CF Foundation primarily funds the Adelaide CF Airway Gene Therapy Research Group in their pursuit for the cure. We are now calling on the community to increase their support of our fundraising so that the research teams can further develop their world-leading gene therapy approach to treat and cure cystic fibrosis airway disease.

To show your support and make a donation click here.

For more information on the UK research results click here.

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