New cystic fibrosis drug could bring relief to patients, doctors

Medical Watch
Data pix.

CHICAGO — Patients are breathing a sigh of relief as results begin to flow in for a novel drug to treat cystic fibrosis. It's offering doctors and their patients hope.

Just a few weeks after a four-month hospital stay, Sierra May is breathing easier.

“It was really rough. The coughing was horrible,” May said.

It was the lowest point in the course of her life-long disease – cystic fibrosis. Patients suffer with chronic lung infections due to mucus build up in their airways.

Sierra May

“This is a normal lung right here, it has a nice grey texture. But the rest shows windpipes that are dilated and filled with mucus, and this is classic with CF.,” said Dr. Marc Sala, Northwestern Medicine pulmonologist and critical care physician.

Last fall, Sierra was 70 pounds with just 19% lung function. She required high levels of oxygen. As her doctors tried to knock down the inflammation in her lungs with antibiotics, they also braced the 23-year-old for her likely last chance -- a transplant. But in late October, the FDA approved what Dr. Sala calls a landmark drug.

“The results have been pretty dramatic. Not only have patients been able to regain on average lung function of 10 to 15 percent back by their breathing test, but their symptoms they’re reporting dramatic changes.”

Clinical studies have shown Trikafta is effective in treating 90% of patients.

Called Trikafta, the drug is a combination of three medications that target a faulty protein in those with CF. Clinical studies have shown it’s effective in 90% of patients, but there’s a price to pay -- Trikafta is expensive at $24,000 dollars a month.

“Medicaid actually pays for it. You have to go through a few hoops. We had to do a prior authorization and practically beg them, but we got it. They are paying for it for at least a year,” said May.

It’s not the only obstacle she’s faced.

“I had a really bad rash, which is one of the number one side effects,” May added. “I’ve got pictures of that rash, and I wouldn’t wish that on my worst enemy. There has been vision changes I’ve noticed, which is a known side effect.”

But the burden has not outweighed the benefits. Her most recent breathing test showed she’s climbed to 37% lung function since starting the drug — and she’s no longer tethered to an oxygen tank.

“You still have some crackles, which we’re not expecting to go away even with the drug, but you don’t sound mucusy, no wheezing. You sound wonderful,” said Dr. Sala.

Sierra — who drafted a bucket list last summer is now considered too healthy to be on the transplant waitlist.

“I know a lot of people are calling it a miracle drug,” said May. "To an extent it is, but it’s not a cure, and it won’t reverse the damage already done. But it does give us longer time to find a cure.”

Trikafta is approved for patients 12 years of age and older. The Cystic Fibrosis Foundation is helping those who need the expensive drug get access.

For more information visit https://www.cff.org/

Listen to the Bair Facts on Health

Get the real facts on everything from diet trends to cutting-edge treatments, brought to you by Dina Bair and actual experts, so you can ignore the noise on social media and make informed decisions about your health.

Subscribe to the podcast

Apple Podcasts

Pocket Casts

Spotify

Stitcher

Popular

Latest News

More News