A new cystic fibrosis (CF) drug is being hailed as a medical breakthrough, which may provide hope for many cystic fibrosis sufferers in Macarthur and beyond.
The prescription medicine called Kalydeco, which was recently placed on the Pharmaceutical Benefits Scheme, targets a specific gene mutation that causes cystic fibrosis, and effectively restores normal function to the respiratory and digestive systems.
Sydney Children’s Hospital respiratory department head Adam Jaffe said the drug was a ‘‘major breakthrough’’ in the treatment of cystic fibrosis.
He said the drug had been able to return ‘‘normal lung function’’ to cystic fibrosis sufferers.
‘‘It is the most remarkable thing, one of the biggest things I’ve seen in CF as far as therapy goes,’’ Professor Jaffe said.
‘‘A lot of other small therapies we’ve worked on so far haven’t been as effective as Kalydeco.’’
Professor Jaffe said Kalydeco targeted the G551D gene mutation which affected about 5 per cent of cystic fibrosis sufferers.
He said there were almost 2000 different gene mutations which could cause cystic fibrosis, which affected the respiratory and digestive systems and required extensive daily treatment, but as yet had no cure.
He said work was already under way to see how the gene-specific research that produced Kalydeco could work for other gene mutations.
While Kalydeco has not been found to be as effective on other gene mutations, it is hoped other gene-specific drugs would have a similar result.
‘‘We know trials are under way all the time,’’ he said.
‘‘We’re hoping in the near future there will be another breakthrough.’’
What is Kalydeco?
■ About 200 Australians will be eligible for treatment with Kalydeco
■ Kalydeco is hailed as the most important development in the treatment of the disease since the discovery of the cystic fibrosis gene in 1989
■ Kalydeco restores the function of the defective CFTR protein allowing a normal flow of salt and fluids to the cells
■ Patients over the age of six can take Kalydeco