Phage therapy offers new hope for children with cystic fibrosis

Phage therapy offers new hope for children with cystic fibrosis

Photo of young girl sitting on hospital bed ith legs crossed accompanied by female carer sitting beside her on the bed.

In a major advancement for personalised medicine, The Children’s Hospital at Westmead (CHW) 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).

Twelve-year-old Abby, from Lightning Ridge in outback NSW, is the first patient to be enrolled in the trial, which will use 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.

“It feels like you can’t really breathe as much as normal, and your lungs feel really tight,” Abby said.

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, funded by Cure4 Cystic Fibrosis (Cure4CF) in collaboration with Sydney Children’s Hospitals Foundation, 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.

Photo on young girl sitting on hospital bed with two hospital clinicians dressed in PPE gear beside her

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.”

Suzy Dimaline, Cure4CF CEO, said she is proud to be contributing to this potentially groundbreaking research.

“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,” Mrs Dimaline said.

Dr Singh, whose PhD is supported by Team Simon, 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 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.