Outside their home in Tottenham, Ont., brothers Andre and Joshua Larocque take turns shooting on net.
Like countless other Canadian kids, the boys love hockey and spend a lot of time playing their favourite sport outdoors.
“Joshua likes a lot of things that Andre likes because he’s the little brother and he tags along,” said mom Sasha Haughian, adding “they are best friends.”
The brothers, aged nine and seven, have a lot in common besides hockey.
They read the same books, play the same online video game, and they are fighting the same fatal genetic disease — Cystic Fibrosis (CF).
CF Canada’s website notes, the disease ’causes various effects on the body, but mainly affects the digestive system and lungs. The degree of CF severity differs from person to person, however, the persistence and ongoing infection in the lungs, with destruction of lungs and loss of lung function, will eventually lead to death in the majority of people with CF.’
“I just want the boys to be healthy, the healthiest that they can be,” said Haughian.
At present, only one of her boys has access to a medication that is helping.
“Andre has always suffered a little bit more than his brother until he started on a drug trial down at Sick Kids hospital,” explained their mother. “Within a week of taking this miracle drug he immediately stopped coughing, had so much more energy, started gaining weight, just becoming a normal little boy.”
The trial group for the drug, Symdeko, is children aged six to 11. Andre fit the criteria and was chosen at random to take part for free.
Younger brother Joshua was under age six at the time.
With Andre now thriving on the trial, Joshua’s health has started to decline.
“As of today, the future looks good for Andre and questionable for Joshua,” said dad Jamie Larocque. “We don’t want to see Andre lose his brother when he’s a teenager.”
But there is a similar version of the drug, Orkambi, that the family said could help the younger brother too.
“We are pretty much the only developed country that doesn’t have public access to the second generation of these drugs, so Orkambi and Symdeco,” explained Dr. John Wallenburg, the chief scientific officer at Cystic Fibrosis Canada.
“Whereas the traditional drugs treat individual symptoms, these drugs go in and they actually start fixing the protein that’s the cause of the problem, so they’re addressing the problem at the heart of it and so their potential is significantly better,” added Wallenburg, who lost his own daughter to CF.
Orkambi was approved for use in Canada in January 2016 but it costs approximately $250,000 per patient, per year.
“It’s not really realistic that an individual would be able to afford these drugs out of pocket,” said Wallenburg.
And there is more.
The governments of Ontario, Saskatchewan and Alberta have placed prescribing criteria for pediatric patients to qualify for public coverage of Orkambi.
Only patients between six-17 years old who have experienced at least a 20 per cent decrease in lung function over six months and sustained for at least six weeks despite appropriate treatment may be eligible.
“The conditions don’t fit what any clinician would say is a reasonable set of conditions … You’re looking at someone who is declining at such a rapid rate that they’re pretty much in desperate straits by that time,” explained Wallenburg.
Yet half of the CF population in Canada could potentially benefit from Orkambi, including Joshua, Wallenburg pointed out.
CF Canada is calling on the provinces to relax the prescribing conditions significantly “to something much more reasonable.”
“You really don’t want to wait until somebody has that much damage accumulated to their lungs before you get them on the drugs to stop that decline,” said Wallenburg.
Especially because a third generation of drugs to treat CF has just been approved by the U.S. Food and Drug Administration.
“Patients are dying … We need to keep them alive so they can access what’s down the pipe,” Wallenburg said.
In a statement to Global News, Ministry of Health and Long-Term Care spokesperson David Jenson said, the “Canadian Agency for Drugs and Technologies in Health (CADTH) reviews and makes recommendations for cystic fibrosis drugs, including Orkambi, following a thorough, evidence-informed review process.
“CADTH concluded the evidence suggests there might be a small improvement in lung function for some cystic fibrosis patients, but this improvement is unlikely to have a meaningful impact on the lives of cystic fibrosis patients,” the statement continued.
“There was no evidence to suggest Orkambi improves a patient’s growth, cystic fibrosis symptoms or quality of life.”
Jenson added, “Therefore, in Ontario, Orkambi is considered for funding on an exceptional case-by-case basis for pediatric cystic fibrosis patients who are rapidly declining despite appropriate treatment.. Ontario is open to continued dialogue with drug manufacturers regarding forthcoming cystic fibrosis products.”
Haughian said she finds contradiction in that statement.
“If Orkambi is worth funding for someone who has become so sick, why would it not be worth funding to prevent symptoms in a patient from worsening?” she asked.
She joined MPP for Simcoe-Grey Jim Wilson at Queen’s Park on Nov. 21 when he stood up in the Legislature to urge the government to approve medications for patients with CF.
Wilson reminded the government that five years ago he raised the same issue on behalf of another constituent, Madi Vanstone.
Vanstone, now 17, and her mother also attended Queen’s Park to show their support for the fight.
“They just need to prioritize lives … I don’t think it should be a process every time life-saving medications come into the picture. I think it should just be automatic, get it to the people that need them as fast as possible,” said Vanstone.
Wilson said that in the long run the government needs to develop a ‘rare drug strategy’ to fix the problem.
“In an era of trying to end hallway medicine, these children, these young adults, these young parents, they end up in hospital beds, they end up very sick and they end up dying. If they can get these life-saving drugs early on, all of those costs are avoided,” Wilson told Global News.
For the Larocque family, time is of the essence.
“It makes me more and more nervous that Andre is going to grow up healthy and be able to go to school and get a job and contribute to society and Joshua is going to spend his years declining,” said Haughian.
“It’s really hard to see him struggle when we know he could have such a bright future.”
Back from a game of hockey outside, the family is now huddled in the kitchen, snacking on cookies Haughian baked for the boys and their little sister Lucy, just up from an afternoon nap.
They’re talking about Christmas, as lights twinkle around them.
The mood is festive and every room in the house is decked out.
The boys said they would like hockey jerseys for Christmas.
They’re both fans of the Montreal Canadiens, but they would love a signed jersey by Maple Leafs superstar Auston Matthews.
The children are aware Matthews has his own personal connection with CF. His uncle, who took him to see his very first professional hockey game, lived with CF until his death in his early 30s.
What the boys do not know right now is that the disease they are battling is fatal.
“They just know that Andre is on a drug that makes him feel good … and they understand that we are trying to get these types of drugs available to Josh and every CF patient,” said Haughian.
Her wish for her sons, for Christmas and all-year round is “for the boys to just live the healthiest lives that they possibly can.”