After months of waiting, Quebecers with cystic fibrosis were able to breathe a little; the Legault government announced on Monday that it would fund Trikafta for patients aged six and over.
Nearly 1,220 people have cystic fibrosis in Quebec. Of this number, just over 130 are in Saguenay–Lac-Saint-Jean. This is particularly the case of little Gabrielle, aged twenty-three months.
“This is news we’ve been waiting for. Gabrielle will be two years old in a month, so she is not yet old enough to have the Trikafta,” said her mother, Stéphanie Dionne.
In four years, Gabrielle will be able to receive the drug for free, which is a major relief for her mother, who no longer has to worry about having to pay to treat her daughter.
For Cindy Lessard, the mother of little Derek, it’s almost unreal.
“I expected the delays to be much longer than that. Looks like I don’t realize it yet,” she confessed.
“We are already seeing so many improvements in the oldest who take these drugs, who already have some damage to the lungs. To know that my boy will be able to start at a young age where his health is still going well, despite his illness, I tell myself that it can just go up even more”, rejoiced Mme Lessard.
Since Derek is already old enough for the medication, he will be eligible for it today.
Much more accessible
The Trikafta has been authorized since April 20 for children aged 6 to 11, and for a little over a year for adults. However, a restrictive criterion prevented its accessibility to several patients. To qualify, the lung quality of an affected person had to be less than 90%. This criterion is now removed.
“Waiting to deteriorate below 90% lung function sometimes means multiple hospitalizations and complications. The fact that the medicine now can be given before that condition deteriorates is another hope. We’ve been waiting for this for a long time,” explained Gabrielle’s mother.
It is also one less financial concern since the Trikafta costs $300,000 annually per person with the disease.
A better quality of life
Even if it is still an incurable disease, the fact of administering the drug Trikafta to people affected allows, among other things, to prolong their life expectancy by ten years in addition to improving the conditions the patient’s lungs, allowing him to have an almost normal life. Without the drug, life expectancy is about fifty years.
“Modulators, what it does is it corrects the protein that is defective in patients. The younger we can start this drug, the less we will have an accumulation of damage,” said Dr. John Wallenburg, chief scientific officer for Cystic Fribrosis Canada.
There are over 2000 mutations in cystic fibrosis. Additionally, 10% of sufferers cannot use Trikafta since they lack the gene called DeltaF508.
“The reason they don’t benefit is because they don’t produce any protein at all, so they have mutations that interrupt the gene,” mentioned the Dr Wallenburg.
“I know at least three people that I know their children don’t have access to Trikafta, so it’s super important to get involved and continue the search,” Ms.me Dione.
Fundraising
The superhero car wash will be back this year. It will be held at the Alma Center on August 28. The fundraiser will collect donations to help research with Cystic Fibrosis Canada.
“We raised more than $12,000 last year with the car wash event, it was beyond our expectations,” exclaimed Ms.me Dionne, who will take part in the event.
For the 10% of sufferers who still cannot benefit from Trikafta, research continues with researchers from all over the world. Cystic Fibrosis Canada assures that it will not let them down.
“We are looking for ways to treat mutations that do not produce proteins. Research will continue, but we still have a long way to go,” admitted John Wallenburg.