Experimental Drug Shows Early Promise Against Cystic Fibrosis

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A longer trial probably will be started "early next year," Kerem said.

"We are planning a larger long-term study that will be international, in the United States and Canada and also in Europe," said Dr. Langdon Miller, chief medical officer of PTC Therapeutics, which sponsored the study.

PTC124 was developed through a screening program in which hundreds of thousands of compounds, with a light-emitting molecule called luciferase, were aimed at the genetically altered "stop" signal, Miller said. Several compounds hit the target and lit up.

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"We selected the best ones, did chemistry to modify them and came up with PTC124," he said.

Several initial studies, including the one in Israel, have shown that PCT124 can help produce salt-conducting channels that are "full length and fully functioning," Miller said.

The drug may be useful for a number of other genetic disorders caused by the same sort of mutation, he added. It already is being tried for another genetic condition, Duchenne muscular dystrophy.

More information

You can learn more about cystic fibrosis and its treatment from the Cystic Fibrosis Foundation.

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