New grant could lead to a cure for cystic fibrosis

A new grant will enable medical researchers to undergo development of a gene therapy for Cystic Fibrosis (CF) in the laboratory. 

The $140,000 Barbara Stow-Smith Cure4 Cystic Fibrosis Innovation grant was awarded to a team of medical researchers from the University of Sydney’s Faculty of Medicine and Health who are all based at Westmead.

The project will be led by Dr Leszek Lisowski from Children’s Medical Research Institute (CMRI) who is the Head of the Translational Vectorology Unit and Professor Hiran Selvadurai who is Senior Staff Specialist in Paediatric Respiratory Medicine at The Children’s Hospital at Westmead (CHW). It will also involve Dr Predag Kalajdzic from CMRI’s Vector and Genome Engineering Facility (VGEF), and two PhD students Adrian Westhaus and Kimberley Dilworth from CMRI.

Senior Staff Specialist in Paediatric Respiratory Medicine at CHW, Professor Hiran Selvadurai, says he is excited about the prospect of a gene therapy cure for CF patients.

“This grant gives us an opportunity to make a difference to sick children. We are excited to embark on this journey in order to bring real hope for children with CF,” said Professor Selvadurai. 

The medical research team will work to develop a novel pre-clinical model of human lung that will enable studies not only of potential treatments for CF but also other conditions such as asthma and other lung diseases.

Head of the Translational Vectorology Unit at CMRI, Dr Leszek Lisowki, and his team are working to develop a gene therapy that has the potential to extend the life expectancy and improve quality of life for over 10,000 mostly young patients in Australia living with CF.

“Our approach has the potential to alleviate the lung-associated complications of CF, which have major health implications and are usually the most difficult to treat,’’ said Dr Lisowski.

“We are very excited to start this project in an endeavour to help children suffering with CF, and so grateful to for making this possible.’’ 

Cassie Jones' five-year-old son Charlie, is a CF patient at The Children's Hospital at Westmead, and takes 20 pills a day and needs medicine every time he eats plus physiotherapy and use of a nebuliser everyday. 

“Gene therapy eventually aims to cure, not just treat, cystic fibrosis, and the beauty of this is that it could work for anyone with CF, no matter what their mutations are,’’ said Ms Jones.

“This is so important in CF because there are so many different mutations, and more so for us as Charlie carries a rarer one that makes current break-through medications ineffective. 

“We work hard to keep Charlie as healthy as possible because we’re waiting for the day that treatments like this will prevent further damage to his body and add years to his life. News like this just brings that day closer and within reach.’’

The Cystic Fibrosis gene is carried by 1 in 25 Australians. Around 100 newborns in Australia are diagnosed each year with the disease.

For more information, head to the Children's Medical Research Institute's (CMRI) website