molecules of the month

The Pfizer CFTR potentiator, CP-628006, enhances CFTR function in cystic fibrosis patient-derived airwell cells with a distinct mechanism of action from existing potentiators such as ivacaftor. It has a unique chemical structure, and in contrast to other potentiators does not seem to inhibit the channel at high concentrations of drug and appears to improve the plasma membrane stability of F508del-CFTR rather than destabilize it. Greater clinical benefit in CF might be achieved with combinations of different classes of potentiators such as CP-628006, making it an interesting albeit synthetically challenging starting point for further investigation.