Friday, March 8, 2024

Another reminder of the possibilities of working in pharma

Via The Atlantic Monthly, this wonderful article on Vertex's CF drug Trikafta: 

...Imagine, though, that you had never been able to simply breathe. Imagine that mucus—thick, copious, dark—had been accumulating since the moment you were born, thwarting air and trapping microbes to fester inside your lungs. That you spent an hour each day physically pounding the mucus out of your airways, but even then, your lung function would spiral only downward, in what amounted to a long, slow asphyxiation. This was what it once meant to be born with cystic fibrosis.

Then, in the fall of 2019, a new triple combination of drugs began making its way into the hands of people with the genetic disease. Trikafta corrects the misshapen protein that causes cystic fibrosis; this molecular tweak thins mucus in the lungs so it can be coughed up easily. In a matter of hours, patients who took it began to cough—and cough and cough and cough in what they later started calling the Purge. They hacked up at work, at home, in their car, in bed at night. It’s not that they were sick; if anything, it was the opposite: They were becoming well. In the days that followed, their lungs were cleansed of a tarlike mucus, and the small tasks of daily life that had been so difficult became unthinkingly easy. They ran up the stairs. They ran after their kids. They ran 10Ks. They ran marathons.

Read the whole thing. 

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looks like Blogger doesn't work with anonymous comments from Chrome browsers at the moment - works in Microsoft Edge, or from Chrome with a Blogger account - sorry! CJ 3/21/20