Cystic Fibrosis Letdown Explained Drug Combo: Cell studies suggest way to boost clinical outcomes

Combination therapies are a promising strategy for correcting the most common gene mutation responsible for cystic fibrosis. A “corrector” drug rescues a misfolded ion channel (purple squiggle) and brings it to the cell membrane (yellow). Then, a “potentiator” drug coaxes the channel open, allowing more chloride ions (green) out.

Credit: Sci. Transl. Med.

One of the two drugs in a promising cystic fibrosis therapy might be undoing the beneficial effects of the other, two studies suggest. The work, conducted in cells, may explain the modest results from a major clinical trial and provide an avenue for improved drugs.

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