The unrelenting crackle of a night-time, torso-flexing cough. The peeling of pounds off her already-tiny 5'3" frame. Frail and fragile, during the day she would regularly stop and hold a wall, or grab a seat in order to catch her breath, which a mere walk to another room or simply opening a bottle from the fridge could shorten.
This all-too-familiar scenario recently landed my wife, Cheryl Corcoran, back in a Halifax hospital for yet another round of weeks-long treatment of powerful antibiotics. The hospitalizations, due to her cystic fibrosis (CF), were piling up. This was the third in a span of six months. The rare, fatal genetic disease was wreaking increasing havoc on the functioning of her 47-year-old lungs.
"I always considered myself one of the lucky ones who managed to be very healthy through childhood, my teenage years and even up until about age 45," Cheryl, of Bridgewater, explained, describing life with the disease she was born with and which also affects 4,300 Canadians, including 200 Nova Scotians.
CF results from a malfunctioning protein, causing thick mucus build-ups in the digestive and respiratory systems and other organs. Sufferers must dedicate themselves to a regimen of physiotherapy, inhaled medication, and the daily consumption of enzymes and vitamins to survive.
"I completed all of my treatments, took all of my medications and never gave another thought to my breathing and how my health would be in the future," said Cheryl.
In Canada, the median age of survival is just over 52 years of age. U.S. information suggests the average age of life expectancy is 44 years.
Thankfully, generations of research and development have improved those figures vastly. For example, in the 1950s CF sufferers were lucky to live beyond Kindergarten. There remains no cure for CF and complications ultimately lead to death for most who have it, while a double-lung transplant is the last resort in improving the length and quality of life.
Last year marked changes in the medical treatment landscape for CF'ers, with the introduction of the combination medication called Trikafta. The breakthrough drug can correct aspects of the defective protein. While there are nearly 2,000 gene mutations that lead to the flaw in the protein, Trikafta is able to target the most common one. Potentially, 90 per cent of CF'ers may benefit. Cheryl found out during her last hospitalization that she's among that unique mutation majority.
Developed by Boston-based Vertex Pharmaceuticals, Trikafta received fast-track approval by the U.S. Food and Drug Administration in 2019, after patient data from drug trials showed dramatic improvements in lung function and a huge reduction in hospital admissions attributable to CF. Medical experts were adamant: Trikafta transforms CF into a manageable chronic condition from a life-shortening disease.
However, Trikafta isn't approved in Canada because Vertex hasn't applied for market authorization. Though there is an exception for some patients, provided they are in rapidly failing health but have bolstered enough energy not to be at death's door.
With her lungs functioning at less than 40 per cent, and after a combined 10 weeks in hospital over a two-year period, and a series of home IV treatments, Cheryl's health care team decided to file a "compassionate grounds" application to Vertex under the special access program (SAP).
Within a month, Vertex granted the request and Health Canada authorized it. Cheryl joined a select number of CF'ers in Canada who can access Trikafta for life and - for now - for free. If she had to pay for it out-of-pocket, it would cost more than $400,000 Canadian dollars a year. The news brought the Corcoran family to tears.
A month into Trikafta treatment of three pills per day, Cheryl's lung function spiked nearly 10 percentage points. The fits of coughing are gone and she's gaining needed weight.
She takes long walks daily and recently returned to work.
"I am not short of breath at all ... I feel like a brand new woman and like I have a new lease on life," she enthused.
Stephanie Stavros, of Pickering, Ontario is the first CF'er in Canada to receive Trikafta on compassionate grounds. She felt the same way when she started it in January.
"Oh, I'm so happy for her," Stavros, 36, commented to LighthouseNOW when told about Cheryl. "Anybody that gets loud about this is going to get us closer to getting [universal access] to this drug."
Stavros had a dismal lung function of 28 per cent, and had ongoing bouts of pneumonia and infections that left her with mobility challenges. Within a week of Trikafta, her lung function went up seven percentage points, and, now, she moves freely and easily. "I won the lottery," she said.
She fought for access to the therapy and now she's among CF'ers across Canada trying to get the attention of the federal government, along with its arms-length board that regulates the prices of patented medicines sold in this country.
Both Vertex and Cystic Fibrosis Canada are concerned about new medicine pricing reforms that were originally due to come into force this summer (now postponed to 2021). The proposed amendments included requiring pharmaceutical companies to steeply drop their prices.
"The current proposal creates significant long-term and far-reaching unintended consequences. We have made every effort to communicate our concerns about these pricing reforms to the Canadian government. We have also communicated several potential alternative solutions," Christina Cunningham, a Vertex spokeswoman based in Ontario, explained to LighthouseNOW.
Cystic Fibrosis Canada suggested the proposed changes will translate into unnecessary delays or denials.
"Phenomenal things are happening, and now we need to make sure people who aren't really sick can get access to this drug so it can help slow the progression [of CF] so people don't have to get that sick and compromise their longevity and lives ahead of them," said Kim Steele, a spokeswoman for Cystic Fibrosis Canada.
As for the SAP option, neither Vertex nor Cystic Fibrosis Canada see that as the overall long-term fix for CF'ers. Steele is surprised SAP appears framed as such in the House of Commons when Health Canada Minister Patty Hajdu was asked by opposition MPs about access.
Hajdu told parliamentarians on May 14 that 95 of 98 patients who applied for special access to Trikafta were approved as of May 6.
"It's a program designed for very, very sick individuals and it's good that it's in place in Canada to help capture some of the very sick, critically ill, but it's not a solution to access by any means," Cunningham said.
Health Canada is saying little beyond that it "recognizes the need for therapies that can help Canadians with serious or life-threatening conditions, and continues to encourage manufacturers to bring their therapies to the Canadian market."
"Health Canada has been in contact with Vertex Pharma with regard to their cystic fibrosis drug, Trikafta. At this time, Vertex Pharma has not provided a submission to market Trikafta in Canada," spokeswoman Maryse Durette said in a statement to LighthouseNOW.
Nova Scotia's Department of Health and Wellness also has its hands tied until Vertex applies and receives approval from the federal government. "This approval is necessary before a drug can be considered for any publicly-funded drug programs," Shannon Kerr, a spokeswoman for the department, told LighthouseNOW.
Those living with CF, and families impacted by the disease, find themselves in the middle of an ongoing debate involving money and politics, when all they want is an unobstructed route to a live-saving therapy.
Nick Evans, 31, a former Bridgewater resident now living in Milton, Ontario, had a double-lung transplant in July 2018 because of the damage CF did to his lungs. He wondered if Trikafta, had it been available at the time, would have negated the need for a high-risk surgery.
"It might have turned a corner for me," he told LighthouseNOW. "But I'm happy some huge progress has been made and the children of today [may] never know how we had to deal with cystic fibrosis."
Kari Trethewey, whose 12-year-old son Ethan has CF, said she learned Trikafta would prove effective for his mutation of the disease.
"Honestly, it was a major relief that there was now a medication that could have the potential to address the issues that will very definitely occur with Ethan's lungs over time," Trethewey, who lives in Bridgewater, explained to LighthouseNOW.
Meanwhile, Cheryl also hopes Trikafta is made available to all CF patients who meet the health criteria necessary to qualify, sooner rather than later.
"It's life-changing," she said.