A research team at Dalhousie University has found that a drug for cystic fibrosis (CF) has the potential to treat 90 per cent of people living with the disease, but it’s not available in Canada.
The drug Trikafta was described as “life-changing” for people with cystic fibrosis, a genetic disorder that attacks the respiratory system, by lead researcher Sanja Stanojevic.
“For the first time in the history of the disease we have a treatment that corrects the underlying defect,” Stanojevic said.
A study, released on Monday, analyzed how the health of Canadians living with CF would be impacted by the year 2030 if they started receiving Trikafta in 2021, if it was delayed until 2025 and if it wasn’t made available in the country at all.
Results showed an 18 per cent increase in people living with mild lung disease, but 60 per cent fewer people living with severe lung disease.
It also showed 19 per cent fewer hospitalizations for chest infections and a reduced number of transplants required for severe lung disease.
There are around 5,000 Canadians living with cystic fibrosis, Stanojevic says.
One of those Canadians is the host of Sickboy Podcast Jeremie Saunders, who says some days are really tough.
“When my lungs aren’t doing too well it’s a struggle, even just going on a walk.”
Saunders says he takes about 40 pills a day and spends two hours a day doing other treatments.
The 32-year-old says when he was born his parent were told he wouldn’t live into his twenties.
“I spent the majority of my life with this notion that I wouldn’t make it past 30.”
Now, he says he’s living on “bonus time.”
Stanojevic says one of the key findings of the drug’s impact was that the estimated age of survival for an individual with CF would improve by 9.2 years over the course of 10 years. Death rates would reduce by 15 per cent, she says.
Stanojevic says this therapy could potentially be effective for 90 per cent of people living with cystic fibrosis in Canada.
”It’s thousands of people in Canada where their disease could be completely changed,” she said.
Saunders was given a copy of the study’s findings last week and he says he was in shock.
“Just reading that one-pager brought me to tears. It was probably one of the most mind-bending experiences of my adult life, to see how much of an impact this one little pill can have on so many people,” Saunders says.
“It really made me think about how amazing it would be for a parent who has recently found out their child has been diagnosed with cystic fibrosis, and to know they could get their hands on this drug, how much of an impact that would have on that child’s life.”
In the United States, Trikafta was approved by the FDA in November 2019. Last month, it was lauded in the United Kingdom.
Health Canada does allow Canadian patients to apply for medications like Trikafta via the Special Access Program, and just over 120 Canadians have been given access.
Health Canada told Global News in July there are many steps involved in the regulation and reimbursement of drugs in Canada, and recognizes delays can be frustrating for families.
“Disappointed doesn’t even begin to explain how it feels to know that there’s a drug out there that could fundamentally change my entire life and yet its currently not possible for me to get my hands on it,” Saunders says.
Stanojevic says she hopes the results of this study help advocacy for cystic fibrosis and access to the therapy, so that it may be made available in Canada.
”As someone who lives with cystic fibrosis, I feel like I’m just sitting here crossing my fingers that enough people hear about how effective this drug is, and that enough noise is made,” he said.
–With files from Dan Spector