Van Allens thrilled CF drug Trikafta approved, continue fight to get it to their son

·8 min read

The persistence of the Canadian cystic fibrosis (CF) community was rewarded with some exciting news on June 18.

Health Canada has approved the drug Trikafta as a triple-combination therapy for people with CF, ages 12 and up, who have at least one F508del mutation, the most common CF-causing mutation.

This decision is expected to give about 1,100 people access to a therapy that directly addresses the underlying cause of their disease, according to Trikafta’s developer, Vertex Pharmaceuticals. A study drawing on Canadian data estimated that CF-related deaths across that country could drop by 15 per cent over the next 10 years with this medication’s use at the start of this decade.

“The approval of Trikafta marks a significant milestone for Canadians with CF, their families and Vertex,” Reshma Kewalramani, MD, president and CEO of Vertex, said in a press release.

“I’m absolutely thrilled that it’s been approved in Canada,” said Perth-Wellington MP John Nater. “By all accounts this is a game-changing life-saving medication and the fact that it has been approved in Canada for anyone 12 and over, I think that is amazing and I think it’s going to change a lot of lives and bring a lot of hope to a lot of families.”

He has been an outspoken advocate for the CF community but Nater said he believes it was the families and the CF community itself that made this happen.

“They have been campaigning since the fall of 2019 when it was approved in the United States and they haven’t let up,” he said. “They have been working so hard … I’m just thrilled because it gives so much hope to those families.”

Nater said it is his understanding that additional studies are being conducted and once

Trikafta is proven safe it may even be approved for CF patients younger than 12 which will be welcome news for Shane and Sarah Van Allen, whose son Wade is eight.

“One of the things with CF is the earlier many of these treatments start the better for the long-term health of the individual,” said Nater. “So if we get to the point where Trikafta is available to those who are that much younger it really does improve the long-term outlook.”

It was surreal when Shane heard the news that the drug was approved for people 12 and over. He felt that he better read the fine print in the announcement because he couldn’t believe it happened.

“It’s all very exciting and we’re blessed,” he said. “We’re at a time – 20 years ago it was a different lifestyle for an eight-year-old with CF so we’re pretty lucky … in the United States the FDA has already approved it for six and over and that’s normally the trend, that we follow suit so there is a pretty good chance that one month, six months, who knows but that Canada will follow suit and the drug will be available for Wade.”

Sarah said they will continue their fight until the drug in their hands.

“Right now it has been approved federally and because healthcare is regulated provincially, now it trickles down, each province has to create their guidelines and permission to allow the drug,” she said. “You don’t know what is going to be covered by Ontario Hospital Insurance Program (OHIP) and you have to negotiate with the insurance companies to see what they will pay so it does get to be a little bit of a game where there is some red tape in the way until we get that pill in our hands.”

Shane said they have learned that the fight for Trikafta is required in stages.

“We can never presume it is inevitable,” he said. “I can’t believe it has taken the fight at this point to get it to this point. I mean to me it is a bit of a no-brainer when there are life-saving drugs available in other countries of the world, why was Canada dragging their feet.”

Sarah said there has been a precedent set for approving CF drugs developed by Vertex in Canada because Kalydeco, an earlier drug, was approved for public funding by Ontario.

“We’re beyond hopeful,” said Shane. “This is hugely optimistic news. This is great. We’re pretty excited but … there is too much at stake to take this softly and stop the fight. I think people like John Nater, they understand that and he knows the inside and out of politics better than we do so he’ll know what areas still need to have their thumb on it and keep pushing because we are learning you can’t let off. If there is something near and dear to you that you want to see happen politically it takes some constant effort and rightfully so, a lot is going on.”

Shane said they realize there is more in the political sphere than what is happening in the CF community’s little bubble.

“So we’re grateful to see change because knowing how much of an effort it takes to make it happen but yes, you can’t let the foot off the gas,” he said.

Sarah said Trikafta is the fourth CF drug Vertex has created.

“The way it was explained to us when the very first one came out it was like the very first iPhone,” she said. “Then you just keep building on it so after Trikafta there is going to be another one that’s even better.”

Trikafta is not a cure for CF.

“It’s two pills every day,” said Sarah. “It corrects the cystic fibrosis transmembrane conductance regulator (CFTR) protein and that controls the salt and water in your body. So with CF, those proteins get blocked with mucus, so this drug helps move the salt and water and that mucus out so that the body acts like it doesn’t have CF.”

“Wade is excited (for Trikafta) because he’s been well-groomed to be active,” said Shane. “It’s a healthy lifestyle the CFer is required to have and Wade does it very well but for him, the thought of being capable of even more yet is mind-numbing. We’re very excited to see what the future holds now that we know that this is an option. It’s just a sigh of relief to know that Canada has stepped to the plate and we’re seeing progress. We’re pretty optimistic still.”

The Van Allens realize that as important as it is to get Trikafta to Wade there are older people with CF who need it sooner because they have suffered damage to their body, but the younger a CF patient is when they get to take it the better.

“It doesn’t repair the damage that CF has caused to lungs,” said Sarah. “Also, if you have a lung transplant you can’t have it, so the younger they can have it they may not get the damage or they are not going to have the hospital stays.”

Sarah pointed out that if Trikafta keeps CF patients from having frequent expensive hospital stays that will be less of an overall OHIP expense.

“There are so many CFers this drug will allow to get back into the workplace,” said Shane. “It’s just huge. So many opportunities for people to live their lives, to raise their children and be able to laugh without that deep cough or rasp … I know the damage has already been done to a certain extent but this is finally going to allow them to take that deep breath.”

The Van Allens are grateful for the support that has been given to us, by both the community and by Nater, to try to make this extra push at the political level.

“We never signed up for any of this,” said Shane. “We don’t know politics. We didn’t know the proper way to go about or how to even make sure (Trikafta) was going to come to Canada but … we can’t do it without our local community and that’s pretty heartwarming. It makes a huge difference.”

He said it’s nice to hear the good news stories that have come with the approval of the drug. “We don’t always complain about our government,” said Shane. “We’re not always saying everything is so ho-hum but sometimes we need to take a stand on something we believe in or that we think should happen and it’s nice to be able to have a feel-good story where it did happen and it is happening. We don’t want to take away from what has happened just because there is more progress to be made. We’re grateful for what has happened to this point and we’re excited about what the future holds.”

Colin Burrowes, Local Journalism Initiative Reporter, Listowel Banner

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