Every newborn in Canada is tested most commonly by a sweat test.
Cystic Fibrosis Canada chief scientific officer Dr. John Wallenburg says CF affects mainly the respiratory system, particularly their lungs. As a way to counteract the inflammation that occurs, patients usually have a daily routine that involves methods such as devices that help get rid of mucus build-up.
“Patients will spend between two and four hours every day, no vacations, no weekends,” said Wallenburg. “Just to maintain their health. This combination of intense therapy and a high burden of the disease and a lot of drugs has enabled patients to extend their lives.”
Wallenburg says the progressive disease over time leads to respiratory system failure.
He says all three generations of drugs work in various stages of repairing proteins broken by CF. However, it’s the third-generation drug Trikafta, currently moving through the drug approval process in Canada, that covers the largest range of those with the disease.
“It allows to treat up to 90 per cent of Canadians living with cystic fibrosis,” Wallenburg said. “What’s really important is you are not treating the symptoms that are the downstream result. Now you are going in and you are fixing the product.”
CF patient Christ MacLeod was granted exceptional drug status in 2012 after his lung function dropped to 30 per cent. He says if it wasn’t for the first-generation gene modulator Kalydeco, he more than likely would have died eight years ago. Instead, the drug doubled his lung function.
“Incredible,” said MacLeod. “I went from four litres of oxygen to walking quickly up hills and back to work for 80 plus hours a week.”
MacLeod says these drugs don’t just move the dial on a small scale, they are a game-changer.
“The fact that I am sitting here in 2020, no oxygen, not in a hospital says everything you need to know about the drug(s).”
Saskatoon resident Emily Brennan says she has had to do her CF medical treatments and therapies for up to eight hours per day while dealing with a recent lung infection. Last year, she spent a lot of time in the hospital or on home IV antibiotic therapies or related measures for roughly half the year.
“It’s definitely a strain trying to do all those things that really just keep you alive.”
Brennan says earlier this year she had to step away from her job to maintain a certain level of health.
Brennan says she pays attention to the progress of CF gene modulators such as Trikafta. She says their accessibility in the not distant future provides hope and courage to keep on fighting.
“I thought I just need to keep going. Hopefully, I’ll be able to see my son (Declan) graduate high school,” said Brennan. “I could plan for life well after my 40s.”
The Nechvatal family is well known in Saskatoon with their yearly Hoops 4 Hope basketball tournament that raises funds for cystic fibrosis. Their two boys Dominic, 12, and Benjamin, 4, both have CF.
They put in a handful of hours each day to clear their lungs of mucus with devices and dozens of pills and vitamins. Their father Shaun remains cautiously optimistic a deal for access to Trikafta comes to fruition. He says the drug corrects the protein to the point where a person with CF that’s a given a baseline test would register negative for the disease.
“(At) the cellular level it’s addressing the most root cause of cystic fibrosis,” said Shaun. “That blows my mind.”
Nechvatal says full-scale access for anyone with CF would allow for people to live fuller lives.
According to the Cystic Fibrosis Canada website, Trikafta is being reviewed by the Canadian Agency for Drugs and Technologies in Health (CADTH) for use for those ages 12 and up. Studies for ages under 12 have not yet been completed and therefore the drug can’t be given the green light for that age range.
This process typically takes up to two years but could be fast-tracked to under a year.
The release says that if the drug is approved sooner than later, it would result in 15 per cent fewer deaths and decrease those with severe lung complications by up to 60 per cent by the year 2030.
Wallenburg says he expects Trikafta to be available to some in 2021 across the country, and for it to be in the hands of those who are eligible for it by 2022.
Countries such as the United States, the United Kingdom, and many throughout Europe have granted access to Trikafta to CF patients as of roughly a year ago.
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