When an incident on top-rating drama show Married At First Sight goes viral – and picked up in earnest by old-school news media – it usually involves someone acting as a grub or a termagant (harsh-tempered, overbearing).
Good news and gravity doesn’t usually cut through.
That has changed, at least for the moment.
The bride who didn’t think she’d live so long
The biggest story out of MAFS this season is that of prime-time bride Lyndall Grace, a 27-year-old accountant from WA who wasn’t confident she’d live to 30 because of cystic fibrosis. It’s an anxiety shared by about 3500 Australians.
As Ms Grace explained on an Instagram post last year:
“It’s a genetic condition that mainly affects the digestive and respiratory systems.
“My lungs don’t know how to work properly so they create a really sticky lining that makes catching bugs really easy and getting rid of them really hard. My tummy can’t break down food so I take tablets with everything I eat…
“I’ve also had hundreds of litres of antibiotics pumped into my veins, thousands of tablets and inhalers, I’ve had stomach infections and chest infections to the point of being unable to eat, having my lungs collapse, and even going bald once (it was SO bad holy heck haha).”
Some days, she wrote, “I do feel very scared and very alone”.
Then a breakthrough drug was put on the PBS
Soon after that posting, in March last year, then-Health Minister Greg Hunt put out a sober press release announcing the PBS-listing of a new treatment that “would be life changing for many Australians with cystic fibrosis and their families”.
The new treatment was called Trikafta.
Without the PBS subsidy, “around 1900 Australians would pay more than $250,000 a year for access to Trikafta”, Mr Hunt said.
On the PBS, people aged 12 and over, would “only pay a maximum of $42.50 per script, or as little as $6.80 with a concession card”.
The announcement was widely reported, but there wasn’t the visceral grunt the story deserved.
The new treatment actually works
The sticky build-up in the lungs and other organs as described by Lyndall Grace – the cause of her breathing difficulties and hard to treat infections – is a consequence of poor flow of water and chloride through cell membranes.
The new treatment, as described by Mr Hunt, “works by improving the flow of chloride and water in patients who have a certain genetic defect and helps improve lung function and breathing”.
This is a big deal for many, but not all people with cystic fibrosis. For those who can be helped by the drug, their quality of life appears to significantly improve.
It’s a shame Mr Hunt didn’t have a real-life case study – someone photogenic and charismatic like Lyndall Grace – to wheel out in front of the media.
Because in the last week, with the MAFS branding, she has drawn a mountain of attention to the “miracle” treatment that she believes might add 30 to 40 years (depending on which “exclusive” interview you’ve been reading) to her deeply challenged life.
Improvements felt after three days
A baby is born with cystic fibrosis every four days.
While life expectancy for people with CF has improved, the median life expectancy for Australians with CF is 47 years.
Ordinarily, most children survive to the age of 12, and then life expectancy drops away.
Trikafta is expected to significantly extend life expectancy and profoundly improve quality of life.
Ms Grace has said she experienced improvements to her health within just three days of taking the medication.
“I noticed my voice came back fully, everything was coming up and clearing out, and now I wake up in the morning with a clear cough,” she reportedly said.
For other happy changed-my-life stories see here and here.
But Trikafta can have side effects, including depression. See here.
What does the research say?
Canadian research published in the Journal of Cystic Fibrosis examined the outcomes if people were given access to the treatment in 2021.
The researchers found that by 2030, Trikafta could reduce the number of people living with severe lung disease by 60 per cent and reduce the number of deaths by 15 per cent.
The findings also showed:
- A significantly slower disease progression, with an 18 per cent increase in people with only mild lung disease
- 19 per cent fewer hospitalisations or home intravenous antibiotics for pulmonary exacerbations.
- A reduction in the number of transplants required for severe lung disease.
- The estimated median age of survival for a child born with cystic fibrosis would increase by 9.2 years.
As an extensive 2021 review concluded: “The effectiveness of Trikafta, seen in clinical trials, outperforms currently available therapies in terms of lung function, quality of life, sweat chloride reduction, and pulmonary exacerbation reduction.”
The genetic aspect
Trikafta is a combination of three drugs (elexacaftor, tezacaftor, and ivacaftor) that target the F508del mutation of the CFTR gene.
This mutation causes dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) protein.
Trikafta is known as a modulator therapy, where the expression of a gene is altered – in this instance CFTR – with a view to repairing the problem.
Some things to keep in mind
Lyndall Grace may well be blessed with decades of additional life. But the long-term efficacy of the treatment is not yet known.
Jo Armstrong, CEO of Cystic Fibrosis Australia, told The New Daily:
“Trikafta has been reported as a life-changing medication for many people with cystic fibrosis who have reported an increased quality of life and we hope this means an increase in life expectancy too.
“But it is a new medication here, as it has only been available in Australia for people over the age of 12 years with at least one F508del mutation since April 2022.”
Ms Armstrong and her organisation are advocating for PBS subsidy for Trikafta to be extended to children aged 6-11 years old.
She said there are many people with cystic fibrosis who do not respond to modulator therapies, or are not eligible in Australia, and therefore other therapies are being sought.
“While Trikafta may have profound effects for some people with cystic fibrosis, it is not a cure and not available or suitable for everyone living with cystic fibrosis,” she said.
