2 ms·
Your cynicism might evaporate if you read the article carefully without jumping to conclusions and make an effort to understand some background of CF: >“There
by InSteady 3y ago
Your cynicism might evaporate if you read the article carefully without jumping to conclusions and make an effort to understand some background of CF:
>“There was a lot of scepticism that this could be done,” Hadida recalls. ... The drug-discovery process required a marathon effort, testing the effects of more than one million compounds on isolated human lung cells to identify candidate drugs for clinical trials.
All of this research into these drugs was for the purpose of treating CF. They aren't developing drugs that target a CF-related protein in lung tissue to treat any other disorder. These drugs do not work on their own to any meaningful degree because protein function in the immune system is freaking complex.
Retraining proteins that are dysfunctional due to a genetic defect is not trivial stuff. They found a combo of drugs that allow a misfolding protein to function properly; two that when combined deliver the protein to the cell surface and then a third to get it to work properly once it is manipulated into the correct place.
CF also only effects a tiny percentage of the population. The only way the economics of this research makes sense is if they charge insurers a high price for it. If we don't reward this behavior, 70,000 people worldwide die at age 35 after having a medically miserable life. Instead they now have stunningly high chance of living until age 80 with significant quality of life improvement.