Cure4CF Update
Phage therapy offers new hope for children with cystic fibrosis
In a major advancement for personalised medicine, The Children’s Hospital at Westmead has launched a world-first clinical trial investigating the use of phage therapy to treat a chronic bacterial infection in children with cystic fibrosis (CF).
Funded by the Cure4 Cystic Fibrosis Foundation (Cure4CF) in collaboration with Sydney Children’s Hospitals Foundation, the trial will use bacteria-eliminating viral treatments called bacteriophages (phages) to treat children with CF who have chronic Pseudomonas aeruginosa infection.
The phages will be manufactured locally at the Westmead Institute for Medical Research, part of the Westmead Health Precinct.
Minister for Medical Research David Harris has welcomed the trial which is being supported by a $3.5 million NSW Government funding boost to manufacturing capacity. “It’s pleasing to see the recent investment making a meaningful difference by supporting this very important clinical work,” Mr Harris said.
Pseudomonas aeruginosa is a common bacteria found in places such as showers and swimming pools. For healthy individuals, the bacteria ordinarily have no lasting impact; but those living with CF are at greater risk due to impaired mucus clearance in their lungs, with almost one in four children living with CF infected by this bacteria.
Currently, the only available treatment is prolonged and repeated courses of strong antibiotics. However, the bacteria often develops a protective film that enables it to evade antibiotics and the body’s natural defences, making it extremely difficult to treat.
The ongoing use of such antibiotics can also lead to hearing impairment and kidney disease and is not always effective in treating the infection. Dr Jagdev Singh, Paediatric Respiratory and Sleep Consultant at The Children’s Hospital at Westmead, is leading the trial, with his PhD supported by Team Simon and said phage therapy offers a new way to directly target the infection.
“Phages are specific and specialised viruses that can be exactly matched to eliminate bacteria without harming human cells. If successful, this could offer a potentially lifesaving treatment for children with CF who have a Pseudomonas aeruginosa infection, helping to alleviate dependence on antibiotic use and as a result, reduce hospital visits due to infection,” Dr Singh said.
The clinical trial is the first major step, both locally and internationally, in demonstrating the suitability of phages as a routine treatment for chronic Pseudomonas aeruginosa bacterial infection in children with CF.
“Pseudomonas aeruginosa infections increase the risk of death for children with CF by almost nine times, with the infection often meaning these children aren’t eligible for lifesaving lung transplants due to the risk associated with post transplant infection.” “Current treatment involves strong antibiotics but the bacteria can become resistant to medication over time. The unique advantage of phages is that they can adapt as the bacteria evolves, meaning we have more chance of effectively treating and stopping the infection altogether.”
Unique to this trial, phage therapy will be delivered through the lungs via bronchoscopy and nebulisation, giving children the ability to receive future treatment at home and reduce time spent in the hospital. Suzy Dimaline, Cure4CF CEO, said it is fantastic to be contributing to potentially ground-breaking research.
“This clinical trial could offer a potentially lifesaving treatment for young people with cystic fibrosis and we are so proud to be working alongside Dr Singh and his team at The Children’s Hospital at Westmead to support this vital research,” Mrs Dimaline said. “Through this trial, we are truly seeing the impact fundraising has not only in driving transformational research but also in changing the care that can be provided to young people living with CF for the better.”
Dr Singh added, “We couldn’t do what we do without the support of communities and organisations like Cure4CF and Team Simon. Their funding has been integral to this trial and we are incredibly grateful for their continued and generous support.”
Phase one of the Cure4CF personalised phage treatment of Pseudomonas aeruginosa for children with cystic fibrosis clinical trial will involve approximately ten children. If successful, the trial will then expand nationally and internationally, before furthering the treatment to target other bacteria that causes lung damage in patients with CF.
Watch Jagdev Singh’s exciting work featured on The Project below.