Luca Rooney uses a nebuliser most days - like the one his teddy is wearing. Photo: Supplied/Liam Rooney
Two-year-old Luca Rooney's parents are living in hope their wee boy will soon have access to a "miracle" life-extending treatment for his cystic fibrosis.
When Luca was diagnosed at two weeks old, his parents Liam and Courtney Rooney had no idea what the condition was.
They quickly learned a lot, including the devastating news that without treatment Luca would probably only live until his thirties, due to organ damage.
Cystic fibrosis causes a sticky mucus to build up in many vital organs including the lungs and pancreas - trapping bacteria and meaning those with the condition easily get very sick.
There is no cure, but there is hope.
"We were sort of told by our doctors that there was a miracle drug that had just been developed called Trikafta, and that would extend the lifespan of kids to... at least double," Liam Rooney said.
When Liam Rooney’s son was diagnosed with cystic fibrosis at two weeks old, he and his wife Courtney had “no idea” what it was. Photo: Supplied/Liam Rooney
Doctors at Starship told the Rooneys that Trikafta was "the most transformative drug they've ever seen outside of any cancer drugs," he said.
In New Zealand, Trikafta has been funded for those six years old and over since 2023 - but it has just been added to Pharmac's Options for Investment (OFI) list for two to five-year-olds.
Being on the OFI list does not guarantee funding, but once a drug is on the list Pharmac aims to fund it when it has available budget, the agency said.
Drugs on the list are ranked, but the rankings are not public.
The Rooneys could not afford to pay the roughly $400,000 a year that Trikafta costs. Instead they were forced to wait until Luca turns six.
"We're sort of constantly on edge that... he would get too sick and if he required a lung transplant or anything before six, it could mean that he could not be eligible to take Trikafta in the future," said Rooney.
That meant drastic preventative measures including keeping Luca out of daycare, twice-daily physio, a nebuliser (a type of inhaler) most days, vitamins three times a week, and keeping him away from any harmful bacteria which is often in dormant water.
"So anytime he's playing anywhere, we're basically standing over him just to make sure that he's not anywhere near any water that could affect him."
Luca Rooney Photo: Supplied/Liam Rooney
But despite their best efforts, Luca had been in and out of hospital in his two years, and has lived half his life on antibiotics.
Rooney said he and his wife lived in hope every day that the drug would be funded for littlies like Luca.
"It would mean everything to us."
Starship respiratory specialist Dr Julian Vyas told RNZ that Trikafta works by stopping the mucus being produced so thick, clearing the chest over a few days.
Making it available for preschoolers would help prevent some lifelong harm, he said.
"We know even by a year or two of age children have evidence of damage and evidence of harm to their lungs on CT scans.
"So the rationale is, if we can intervene sooner, by starting the Trikafta sooner, we can maintain wellbeing for longer, and there's a number of benefits from that. It's the idea of nipping things in the bud."
Luca Rooney has spent his life in and out of hospital. Photo: Supplied/Liam Rooney
Vyas also pointed out the massive burden of care on parents, having to protect their children and administer medication - which can be a battle with a preschooler.
"It's an ongoing thing, it's not you need it for two weeks, job done. This is for the foreseeable future," he said.
Parents do what they need to for their kids, but it was a huge responsibility, said Vyas.
"A number of parents have said to me, it kinda shifts that dynamic from being mum or dad, into being a nurse, doctor, physio all rolled into one."
At two years old, Luca Rooney still has four years to wait until he’s eligible to receive publicly funded Trikafta. Photo: Supplied/Liam Rooney
The head of Cystic Fibrosis NZ (CFNZ) Simone Brown said the inclusion of Trikafta for two to five-year-olds on Pharmac's OFI list was an important milestone.
"Starting this medicine from two years of age is vital to prevent deterioration, preserve lung function, and protect children with cystic fibrosis from significant decline," she said.
"We've already seen how transformative Trikafta has been for hundreds of people living with CF in New Zealand."
CFNZ would continue to push for the access to be expanded, Brown said.
Trikafta has been funded in New Zealand for those aged six and over since April 2023.
CFNZ estimated more than 50 two to five-year-olds would benefit from Trikafta.
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