Parents Seeing Impressive Results With Breakthrough Cystic Fibrosis Drug. But What Are The Unknowns?

Jan 24, 2020, 8:54 PM

SALT LAKE CITY, Utah – Imagine if you learned that your child’s life-threatening disease could now be treated.

That’s the news thousands of parents received about a breakthrough therapy for cystic fibrosis. But as the drug hits the market, what are the unknowns?

Elliot Hales has cystic fibrosis, a life-threatening disease that damages the lungs and digestive system.

“When it’s hard to breathe, it’s kind of like you’re choking on a chip or something,” 12-year-old Elliot said.

He’s used to grueling treatments and hospitalizations, but he may not have to endure them anymore, thanks to a breakthrough therapy.

Elliot’s mother Alta Hales said it was the news she’d been waiting for and working toward for years.

“Tears. It was actually a shout for joy,” she said.

The therapy is called Trikafta: three medications that work together, targeting the underlying cause of cystic fibrosis — defects in a specific protein.

Trikafta is a combination of three drugs that work together, fighting the root cause of cystic fibrosis.

“It’s really the closest thing to a miracle that we have in the cystic fibrosis world, and this is what we’ve been waiting for decades,” said University of Utah Health and Primary Children’s Hospital Dr. Fadi Asfour. “Children are reporting being able to breathe easier, some of them don’t have a cough anymore. We’re talking about children that coughed most of their life.”

Dr. Thomas Ferkol, who is the Alexis Hartman Professor of Pediatrics at Washington University in St. Louis, said it could extend the lives of patients with cystic fibrosis by decades. But it’s not a cure.

“It doesn’t mean that the damage that has occurred in the past will suddenly repair itself. We have no evidence that that will occur,” Ferkol said.

And the treatment is pricey.

“It’s very expensive,” Ferkol said. “It’s $311,000 a year for this drug and that’s a problem. That’s a real problem.”

Most major insurance companies have been covering it so far, but it’s only approved for kids 12 and older, and there are things we don’t know yet.

“This combination therapy appears to be safe, based on the clinical trials, but that’s with a relatively modest number of patients,” Ferkol said.

As more patients take it, more side effects may become apparent.

After just one week of taking Trikafta, Elliot was seeing improvements.

“What’s the difference in your mucus?” Alta asked her son.

“It’s really, really wet and easy to get up,” Elliot said.

Elliot’s life may soon become easier but it’s bitter-sweet for his family. Trikafta is only effective for CF patients with a certain gene mutation, which represents about 90% of them.

“To know that there’s still 10% of them out there not getting this, it breaks my heart. But at the same time, I’m so thrilled that we have it, that it makes me want to fight even harder for everyone to get something like this,” Alta said.

It’s a long-awaited breakthrough for Elliot and many others.

The hope is that Trikafta will soon but approved for kids 12 and under as well. Doctors said the earlier a patient can take it, the more beneficial it will be by preventing lung damage.

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Parents Seeing Impressive Results With Breakthrough Cystic Fibrosis Drug. But What Are The Unknowns?