In 2020, Stephanie Stavros was the first Canadian to have received Trikafta, a life-altering medication for people with cystic fibrosis.Shay Conroy/The Globe and Mail
Stephanie Stavros is not a famous medical researcher. But one evening this fall, she found herself unexpectedly receiving a standing ovation at a prestigious science awards ceremony in Toronto.
The Canada Gairdner Awards Gala, held in October, celebrated achievements in biomedical research, from the discovery of stem cells to the development of mRNA vaccines.
For a few moments that evening, the spotlight focused on Ms. Stavros of Pickering, Ont. She was the first in the country who fought to receive Trikafta, a life-altering medication for people with cystic fibrosis, an inherited genetic disease that afflicts about 4,500 Canadians.
Two U.S.-based scientists - Michael Welsh and Paul Negulescu - received international honours for showcasing how science can spur life-saving interventions through their research on cellular and molecular mechanisms underpinning CF. The Gairdner Foundation said their work helped to transform CF from being a fatal disease to a manageable condition.
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“It makes me tear up,” Ms. Stavros said in an interview. “It’s unfathomable that I’d be there that night. It felt like the definition of serendipitous.”
When Ms. Stavros was born in the 1980s, many children living with CF did not live into adulthood.
CF causes a build-up of mucus that primarily affects the digestive system and lungs. In the lungs, the mucus can lead to significant respiratory issues, such as infections and deterioration.
Ms. Stavros's husband Jim, who is a chiropractor by trade, provides daily physiotherapeutic treatment at their Pickering, Ont., home.Shay Conroy/The Globe and Mail
Research advancements and treatments have been credited for improving chances of survival.
Last year, an annual report from Cystic Fibrosis Canada found, for the first time, the estimated median age of survival is 60 years old.
Ms. Stavros, 42, knows she is proof that science saves lives at a time when researchers say their work is increasingly undermined globally, especially as misinformation spreads online. Patients and advocacy organizations also say medications like Trikafta underscore the need for more research.
Prior to 2020, Ms. Stavros’s quality of life was “minuscule.” Her skin appeared blue. She was on oxygen. She was told she would be a good candidate for a double lung transplant.
She was also preparing for the prospect of saying goodbye to her young son Grey and her husband Jim.
Ms. Stavros believed her only hope to live was to campaign to receive the medication, which the U.S. Food and Drug Administration had approved in October, 2019. It reduces thick mucus in the body and was billed as a revolutionary drug for cystic fibrosis patients.
Her appeal worked. The drug maker, Vertex, provided the medication on compassionate care grounds.
Trikafta was not yet available in Canada. Special access from Health Canada had to be granted to allow Ms. Stavros to take the medication.
On Jan. 20, 2020, Ms. Stavros took her first dose and said the drug kicked in almost immediately. After she took two pills at 9 a.m., she remembers feeling her whole body start to change by noon.
“By 10 at night, I was a new woman,” she said.
In June, 2021, Health Canada approved Trikafta.
The following year, it became the most commonly prescribed cystic fibrosis transmembrane conductance regulator in Canada, a class of medication designed to correct for malfunctioning protein.
In a report last year, the Canadian Institute for Health Information found that in 2022, more than half (56 per cent) of the approximately 4,445 Canadians with CF were taking a CFTR modulator and almost all were on Trikafta. It also said it accounted for 25 per cent of the growth in public drug spending in 2022.
The medication is priced at about $300,000 per person per year.
In 2024, Health Canada approved Trikafta for an additional 152 rare mutations. Cystic Fibrosis Canada says Trikafta for this group is funded in some jurisdictions, but access is not equitable across Canada and significant barriers remain for many patients.
Ms. Stavros knows personally about challenges to accessing life-saving medication.
She said she learned in 2024 Vertex would no longer provide compassionate coverage of the medication.
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Ms. Stavros said her private insurance company would also not pay for it and it was not sustainable long-term to pay an annual deductible (usually about 4 per cent of household income after taxes) to access Ontario’s Trillium Drug Program where patients can seek coverage if their prescription costs are high.
“No one in Ontario, or anywhere in Canada, should ever experience this,” she said, adding that the stress adversely affected her health.
Ms. Stavros, who received the King’s Coronation Medal this year in recognition of her CF advocacy work, now receives full public coverage for her medication because she qualifies for home care.
She also continues to advocate for harmonious coverage across Canada so the cost of life-saving drugs is not determined by postal code.
Care, she said, needs to be equal “no matter where someone calls home.”