Groundbreaking new cystic fibrosis drug offers Montreal teacher a new lease on life

Chelsea Gagnon can finally get back to work and walk her dog. She attributes the shift to a game-changing drug for those living with cystic fibrosis.

Trikafta was approved for use in Canada last June. In late September, four provincial governments — Quebec, Ontario, Saskatchewan and Alberta — announced they would cover the treatment's hefty price tag of around $300,000 for a year's worth of treatment.

For people like Gagnon, it's welcome news. 

"It's a total 180. It's completely different…. I have energy that I didn't know could exist inside a single person," said Gagnon, 32, a teacher who lives in Montreal.

"If healthy people have always had this amount of energy, like, why aren't they climbing Mount Everest every day or going to space?" she joked with Day 6 guest host Rachel Giese.

Gagnon spent hours each day treating the symptoms that come with cystic fibrosis — a progressive, genetic disease that primarily targets the lungs. Often it would leave her physically exhausted, unable to work or visit friends.

Every other month, she says her symptoms became severe enough to warrant significant medical intervention, including hospitalization.

But since starting Trikafta in July, her coughing has subsided and she's been able to put on weight as her body no longer needs to fight regular infections.

"It feels like ... they took my head and put it on a new body," she said.

Drug fixes defective proteins causing disease

John Wallenburg, chief science officer for Cystic Fibrosis Canada, attributes the drug's benefits to its ability to target specific proteins.

For decades medications have only addressed cystic fibrosis symptoms, like persistent coughing and frequent lung infections. Trikafta instead "fixes" the defective proteins that cause the underlying conditions.

"Instead of doing one symptom at a time, you're actually preventing — or maybe reducing — the impact of the symptoms all over all at the same time," said Wallenburg.

A Phase 3 clinical trial suggests patients taking Trikafta have, on average, a 14-per-cent increase in lung function. Some patients, however, have seen improvements more than triple that, Wallenburg notes.

Trikafta specifically targets a gene mutation known as f508del — the mutation responsible for about 90 per cent of cystic fibrosis cases. 

According to Cystic Fibrosis Canada, one in every 3,600 children are born with the disease, and over 4,370 Canadians attend specialized clinics.

With funding for the drug in four provinces so far, Wallenburg says four-fifths of those living with that mutation could access the treatment.

But Trikafta is not a cure, and Wallenburg says that patients should continue standard therapy practices, including physiotherapy.

'I have to start figuring out adulting,' says Emma Chenier

3 years ago

Duration 0:40

Emma Chenier is thrilled to know Ontario will cover the cost of Trikafta, a cystic fibrosis medication. With access to the revolutionary drug, life expectancy is extended, therefore she can start planning further into the future.

Planning for the future

In the 1960s, the average life expectancy for people after a cystic fibrosis diagnosis was less than 10 years, Wallenburg says.

But the development of drugs that treat the disease's symptoms have improved that significantly. It's now expected that half of people with cystic fibrosis will now live to about 54.

Wallenburg believes that Trikafta could boost that number even further.

"You would expect to see an increase of nine years in the median age of survival," he said, pointing to a Dalhousie University study. That study also found that Trikafta could reduce deaths by 15 per cent.

For the first time, Gagnon says that she feels like she can plan for her future.

Growing up and, and in recent years as her condition deteriorated, she says she couldn't imagine living long enough to get married or have kids.

"I get asked all the time now if I'm going to get married with my long-term boyfriend," she said.

"So for me, it's a very happy feeling. A lot of joy, but also unknown territory, because I haven't ever thought about this."

Written by Jason Vermes with files from CBC News. Interview with Chelsea Gagnon produced by Laurie Allan.

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