A new clinical trial at The Children's Hospital in Westmead (CHW) is offering alternative therapies to cystic fibrosis (CF) sufferers.

Already familiar with the long trip from her home in Lightning Ridge to the CHW for CF treatment is 12 year old Abby, who has become the first patient to be enrolled in the trial that aims to beat a potentially devastating infection.

Cystic fibrosis is an inherited disorder that affects the cells that produce mucus, sweat and digestive juices. These secreted fluids are normally thin and slippery. But in people with CF, a defective gene causes the secretions to become sticky and thick. Instead of acting as lubricants, the secretions plug up tubes, ducts and passageways, especially in the lungs and pancreas.

The trial Abby is taking part in uses bacteria-eliminating viral treatments called bacteriophages (phages) to treat Pseudomonas aeruginosa infection. Pseudomonas aeruginosa is a common bacteria found in places such as showers and swimming pools which ordinarily has no lasting impact for healthy individuals, but due to the predisposition of their lungs to infection, those living with CF are at greater risk.

Abby is among the one in four CF patients impacted, after contracting the infection in 2017 and explained the impact of the infection on her condition.

"It feels like you can't breath as much as normal, and your lungs feel really tight," she said.

Phage therapy trial

 Research shows that once established, Pseudomonas aeruginosa develop a unique biofilm, which acts as a barrier against the immune system and antibiotics. Currently, the only available treatment for the infection is prolonged and repeated courses of strong antibiotics. However, this can lead to hearing impairment and kidney disease, and is not always effective in treating the infection.

The new trial will aim to use phage therapy to directly target the infection without these side effects. It is the first major step in demonstrating the suitability of phages as a routine treatment.

“Phages are specific and specialised viruses that can be exactly matched to eliminate bacteria without harming human cells,” Dr Jagdev Singh, trial lead and Paediatric Respiratory and Sleep Consultant at CHW, said.

The phages will be manufactured locally at the Westmead Institute for Medical Research, part of the Westmead Health Precinct.

“If successful, this could open the door for further research and more advanced studies in phase two and three trials and could offer a potentially lifesaving treatment for children with CF, like Abby,” Dr Singh said.

Until now, Abby has travelled 700km from her home to CHW bi-annually for her two-week tune ups, where she receives strong IV antibiotics and intensive physiotherapy to help clear the infection. During her most recent visit though, in addition to the routine antibiotics, Abby received phage therapy, delivered first via bronchoscopy, and then nebulisation, straight to her lungs.

This unique approach to treatment could help alleviate Abby’s dependence on antibiotics, give her the ability to receive treatment at home and reduce the time she needs to spend in hospital due to infections. 

Less time in hospital for CF sufferers is the goal

  Dr Singh said his hope is for the trial to lead to more effective treatment options so patients like Abby can spend less time in hospital, and more time at home, where they belong.

“The 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,” Dr Singh said.

“The earlier we treat these infections, the longer we can keep these children healthy and out of hospital.”

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 difficult to treat lung infections.

“While it was initially a bit nerve-racking to be the first, we are thrilled to have the opportunity to try this new treatment option that may be able to keep Abby healthier for the future,” Abby’s mum, Chloe, said.