Trikafta for cystic fibrosis

"... word began spreading of a forthcoming three-drug combination from Vertex. In clinical trials, neither patients nor doctors are told who is on the placebo and who is on the experimental drug. But in this trial, everyone could tell. The triple combo made patients’ lung function jump by a shocking 10 percentage points. Overnight, they woke up smelling for the first time the distinctive scent of their home. They could even taste their sweat becoming less salty. This was Trikafta.

In the fall of 2019, Trikafta was approved by the FDA... Jenny received her first box of Trikafta on November 17, 2019, at the end of yet another two-week hospital stay. She had gotten sick again in Nashville. Actually, she had been fighting off a cold before she left, and despite assiduously staying in her hotel room to keep up her treatment routine, she felt an infection settling into her lungs. At the conference, she heard a lot about Trikafta, but she didn’t expect to get it so quickly. CF centers were being inundated with calls from patients asking for the new drug.

In the hospital in Utah, she recorded a video that she sent to her sister with CF, Teresa, who now lived in Ohio. She is sitting on her hospital bed. “My Trikafta is here,” she says, her voice shaking and her eyes tearing up. The miracle drug she had been promised her whole life was now in her hands.

Teresa was also able to start the drug not long after. For her, Trikafta’s impact was immediate and unmistakable. The Purge [expectoration of voluminous amounts of sputum] started on the drive back from the doctor’s visit where she took the first dose. The mucus coming up was so thin that she was confused; it was nothing like the sticky gunk she’d had to work so hard to cough up. A month later, she went back for a sweat test, and her salt level was normal. Based on the results, you would not know she had cystic fibrosis.

“I think of it like, ‘Oh, back when I used to have CF,’ ” Teresa said on a recent call with Jenny and me. “I don’t feel like I have CF. I feel completely normal.” She has been able to stop using her vest and inhaled medications, freeing up that time for her adopted children and the farm where she lives with her family. Before Trikafta, every small exertion was a negotiation with her lungs. Should she go upstairs? How many breaths would that take? Now she’s running around milking the goats, trimming their hooves, throwing 30 bales of hay into the barn..."

If you have a family member with CF or a friend with such, or a friend who has a family member etc., I urge you to read The Cystic-Fibrosis Breakthrough That Changed Everything.  It's quite a remarkable (and happy) story.  

Image cropped for size from the original at the link.  Credit Fumi Nagasaka for The Atlantic,