<
https://www.theatlantic.com/magazine/archive/2024/04/cystic-fibrosis-trikafta-breakthrough-treatment/677471/>
"They call it the Purge.
You have experienced, in a modest way, something like it in the waning days of
a bad cold, when your lungs finally expel their accumulated gunk. The rattle in
your chest quiets. Your sinuses clear. You smell again: the animal sweetness of
your children’s hair, the metallic breeze stirring a late-summer night. Your
body, which oozed and groaned under the yoke of illness, is now a perfectly
humming machine. Living is easy—everything is easy. How wonderful it is to
breathe, simply breathe.
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.
Cystic fibrosis once all but guaranteed an early death. When the disease was
first identified, in the 1930s, most babies born with CF died in infancy. The
next decades were a grind of incremental medical progress: A child born with CF
in the ’50s could expect to live until age 5. In the ’70s, age 10. In the early
2000s, age 35. With Trikafta came a quantum leap. Today, those who begin taking
the drug in early adolescence, a recent study projected, can expect to survive
to age 82.5—an essentially normal life span.
CF was one of the first diseases to be traced to a specific gene, and Trikafta
is one of the first drugs designed for a specific, inherited mutation. It is
not a cure, and it doesn’t work for all patients. But a substantial majority of
the 40,000 Americans with CF have now lived through a miracle—a thrilling but
disorienting miracle. Where they once prepared for death, they now have to
prepare for life. “It’s like the opposite of a terminal diagnosis,” Jenny
Livingston told me."
Cheers,
*** Xanni ***
--
mailto:xanni@xanadu.net Andrew Pam
http://xanadu.com.au/ Chief Scientist, Xanadu
https://glasswings.com.au/ Partner, Glass Wings
https://sericyb.com.au/ Manager, Serious Cybernetics
