They used to feel like the children Pharmac forgot. But April 1 was a landmark day for the cystic fibrosis (CF) community, with Pharmac now publicly funding the drug Trikafta for those with CF aged over six. The drug has been widely heralded for turning
CF from a life-threatening condition – predominantly affecting the lungs and digestive system – to a manageable one. The Weekly catches up with three of the families affected.
Growing up, Josh Chase never allowed himself to dream about the future.
Born with cystic fibrosis, the genetic lung disorder had clipped his wings and much of his childhood was spent in hospital.
"There was a time in my teens when I was being admitted into Starship [Children's Hospital] almost once a month," he shares. "I wasn't looking after myself because it felt like too much effort if I was just going to die early. I thought, 'If I'm only going to live till 30, then I'm just going to live my life how I like.'"
Now, having access to "wonder drug" Trikafta has changed all that and allowed the 29-year-old to start a new version of himself, unburdened and unlimited.
For several years, Josh, along with the CF community, had been rallying for it to be subsidised, after facing an annual price tag of $330,000 or the prospect of moving to one of 31 countries where it was publicly available.
When he and his mum Moninne found out that Trikafta was going to be funded here, she screamed. Then the tears flowed.
"I was speechless!" says Josh, a personal trainer from Whangaparāoa, north of Auckland. "There were too many emotions to put into words – including anger that it didn't come through sooner – but overall, relief.
"In less than 12 hours after my first dose of Trikafta, my body was already physically changing within. My lungs are now clear, my energy has increased 10-fold and exercise recovery is so much faster."
It's also the first time in six years that Josh has been able to work full-time due to his health. Last year, he was the recipient of a CF Achiever Award for Education after gaining his qualifications in personal training and a diploma in weight management, while having frequent hospital stays for intravenous antibiotics.
Josh says it was following a major lung surgery at 18 − and coming close to death − that his nonchalant perspective changed and he decided to maintain the health he did have. "I'd gone into hospital for a two-week stint on antibiotics for what we all believed was a typical chest infection," explains Josh.
The illness turned out to be pneumonia and while attempting to drain fluid from his lungs, one collapsed. Within less than 48 hours, Josh was in critical condition.
"Doctors told me it was touch and go during the surgery, where they had to peel away the lining from my lung," tells Josh, who was inspired during recovery to pursue a career in fitness. "No one thought I was going to come through that. Once I heard about Trikafta, it was this whole different ball game of having some hope."
Moninne, 45, says there have been several times in her son's life where she thought, 'This is the end.'
"We nearly lost him to an allergic reaction to a fungus spore called Aspergillus, which most people breathe in every day without consequence," she tells. "The long-term steroids brought on early diabetes. He didn't get the childhood he deserved, or his sister Alicia," she says, with tears welling up.
They describe CF as a lonely journey. When Moninne gave birth to Josh at 16, she'd never heard of the disorder and there were no online support groups. But she'll never forget the day Josh was diagnosed.
"One day a specialist came in and said, 'Your son has cystic fibrosis. Here's a booklet and I'll come back if you have questions.' I asked, 'What does that mean?' And she replied, 'It means your son won't live to see old bones.' And then she left.
"Those words have resounded in my head every day throughout Josh's life. The anxiety and panic I've felt to keep him well – being so pedantic with his physiotherapy and medications – fearing I was going to lose him at a young age."
Finally, the future looks much brighter. While Trikafta isn't a "permanent fix" and Josh concedes he'll probably have to go into hospital at some point, for now he's starting to make plans.
"I want to travel and go snowboarding in Japan. I want to be a positive motivator for people and help them achieve their health and fitness goals," smiles Josh.
Adds Moninne, "Josh is the bravest, most courageous person I've ever met. Although breathing is not what CFers do best, I hope we can all breathe a bit easier now knowing that this battle is over."
The last time 15-year-old CF sufferer Grace Paterson featured in the Weekly, she was in palliative care at home with her family on tenterhooks, unsure about her future.
But last November, the gorgeous Taranaki teen got the lifeline she desperately needed – a liver and pancreas transplant.
Now five months post-transplant – and finally on "miracle drug" Trikafta – Grace has had the energy to finally attend a full week of school, a feat she hadn't managed in years.
"Look at Grace's face... the colour of life suits her," beams mum Jess. "There are no words to describe a 25 percent increase in
lung function and being able to open up your lungs by one litre. All this because of Trikafta… she has virtually been saved. Her new liver is tolerating the Trikafta well.
"I don't think we wanted to accept how sick she really was beforehand. It's been months of hard slog to get to where we are today."
Following a nine-hour operation, Grace has been in and out of hospital to help fight infections, but is getting better every day, shares Jess, who along with husband Sean got tattoos to celebrate their daughter's new chance at life and honour the amazing organ donor, "whoever they may be".
Since being diagnosed with CF as a baby, Grace has been admitted to hospital at least once a year. She developed CF liver disease at seven, followed by CF-related diabetes at 10.
The bubbly Year 11 student became progressively sicker and had to have some honest conversations with her parents about her life expectancy.
"It's so amazing to know Grace can now go on school trips, join the youth group in town, and go to university to become a forensic scientist and get married if she wants to!" the doting mum enthuses. "All these things other teenagers take for granted that she simply didn't have the energy to do.
"We bought her a car the other day to start practising in. It was meant to be a 16th birthday present in July, but we couldn't wait that long! She's raring to go. We just can't wait to see her grow into a beautiful young adult and start enjoying life like she should."
'We uprooted our lives for Kairo'
When Ange and Matt Falepau heard the news that Trikafta would be funded in New Zealand, their feelings were bittersweet.
The couple gave up their home in Manawatū a year ago to temporarily live in a caravan in Brisbane so their eight-year-old son Kairo – who has CF – could get on to life-changing medication funded in Australia.
"While we are so thankful and happy for our Kiwi CF community, we do feel resentment towards the massive delay in funding Trikafta, which forced us to move to Australia," says Ange, 36, who has ruled out moving home again.
"We are angry we were essentially forced out of New Zealand to seek medication for Kairo. We were medical refugees and we still are. We uprooted our whole lives and left everything behind to get what Kairo needed. To uproot the children once again would be unfair to them."
She says the other reason for not returning is that since Kairo started on CF modulator drug Orkambi, his health has been "amazing".
"Why change what's not broken? If we were to come home only to find out Kairo reacts negatively to Trikafta, we are back to square one, as New Zealand does not have the option of Orkambi."
While it was a difficult decision for the Falepaus to say goodbye to their whānau, careers and culture, they believe it's a sacrifice that's been worth it for the health of their little boy.
"Kairo is doing so well!" smiles Ange. "He is excelling at school and since starting Orkambi in July 2022, his lung function has increased from 78 to 94 percent.
"He has gained weight and is no longer having coughing attacks in the mornings, and no longer gets out of breath easily, plus he can run around with his friends for longer periods of time."
While the family all miss home, they have a built a new life in Australia.
"We have purchased our own slice of Aussie and move in this month − almost 12 months to the day that we left New Zealand.
"And Kairo currently wants to be an author when he grows up as he loves writing his own stories. He's writing one about our adventure moving to Australia at the moment − you may read about it one day!"