CFTR potentiators 

CFTR potentiators repair defects in chloride transport, and are used to treat cystic fibrosis patients with a specific gene mutation.

Potentiators are CFTR modulators that hold the gate open so chloride can flow through the cell membrane. The drug Kalydeco^® (ivacaftor) is a potentiator. This drug can help patients with gating and conduction mutations in CFTR.

Kalydeco (ivacaftor), a potentiator, binds to the defective protein at the cell surface and opens the chloride channel (holds the gate open) so that chloride can flow through, regulating the amount of fluids at the surface of the cell.

Overexpression of cystic fibrosis transmembrane conductance regulator (CFTR) is associated with human cervical cancer malignancy, progression and prognosis. Gynecol Oncol.

CFTR is activated by a kinase (PKA) which phosphorylates the channel. Here we show that CFTR activation is not caused by phosphorylation, as believed, but by simple PKA binding which promotes reversible release of CFTR’s regulatory (R) domain from an inhibitory position.

The only U.S. Food and Drug Administration -approved CFTR activator, VX-770, was developed as a potentiator to treat CF by correcting the channel gating of certain CFTR mutations (48).