New study brings hope for preventing lung disease in children with cystic fibrosis

Researchers at Perth's Telethon Institute for Child Health Research, the Queensland Children's Medical Research Institute (QCMRI), together with researchers at the Murdoch Childrens Research Institute in Melbourne, are one step closer to preventing serious lung disease which is the main cause of suffering in cystic fibrosis.

Cystic fibrosis is an inherited condition that affects one in 2800 children, with one in 25 people a carrier of the gene that causes the condition. There is no current cure, however, researchers have been working to prevent the onset of lung disease.

The study, which is part of the Australian Respiratory Early Surveillance Team for Cystic Fibrosis (AREST CF) program, sought to determine risk factors for the onset of bronchiectasis. Bronchiectasis is a progressive and persistent form of lung disease.

Professor Peter Sly has published the results of this research in the world's most prestigious clinical medicine journal, the New England Journal of Medicine.

Researchers studied 127 infants diagnosed with cystic fibrosis and reported that they could determine which children were at most risk of developing bronchiectasis by three years of age.

The manuscript, as Professor Sly describes, "shows children with cystic fibrosis who have neutrophil elastase in their lungs at three months are seven times more likely to develop bronchiectasis by 12 months, and four times more likely by three years."

Neutrophil elastase is one of the body's mechanisms for destroying bacteria in the body.

The levels of neutrophil elastase in the body is normally controlled, however in cystic fibrosis excess neutrophil elastase can cause breakdown of the lung structure. This leads to irreversible lung damage like bronchiectasis.

Professor Sly suggests, "If we have a mechanism for mopping up neutrophil elastase then we can potentially stop or delay the start of damage to the lungs. We are currently undertaking a clinical trial using azithromycin which has anti-inflammatory effects to prevent bronchiectasis in infants with cystic fibrosis".

The trial, COMBAT CF, is a clinical trial undertaken in Australia and New Zealand which is the first in the world to try and prevent irreversible lung damage early in life.

This is important Professor Sly states as, "We are seeing lung damage in children with cystic fibrosis as young as three months old. Clearly lung disease starts in the first months of life so early intervention is paramount to improving health and quality of life."

This study was a joint collaboration between the Queensland Children's Medical Research Institute under the direction of Professor Peter Sly, the Telethon Institute for Child Health Research under the direction of Professor Stephen Stick and the Murdoch Childrens Research Institute under the direction of Professor Sarath Ranganathan.

ABOUT THE TELETHON INSTITUTE
The Telethon Institute for Child Health Research is one of the largest medical research institutes in Australia, comprising a dedicated and diverse team of more than 500 staff and students.  Established in 1990, it was among the first to adopt a multidisciplinary approach to major health issues: clinical research, laboratory sciences and epidemiologists all under the one roof, to tackle complex diseases and issues in a number of ways.  Located in Subiaco, Western Australia, the Telethon Institute has strong affiliations with Princess Margaret Hospital for Children and The University of Western Australia.  The Telethon Institute is independent and not-for-profit. The majority of funding comes from success in winning competitive research grants from Federal Government bodies such as the National Health and Medical Research Council and the Australian Research Council. It has also received a number of grants from the US National Institutes of Health. The Western Australian State Government provides valuable support through the Medical and Health Research Infrastructure Fund. www.childhealthresearch.org.au