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FDA Approves First Targeted Drug against Cystic Fibrosis

The views expressed are those of the author and are not necessarily those of Scientific American.


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conceptual drawing of lungs

Image: Illustration by John Hendrix

The U.S. Food and Drug Administration today approved a new drug that tackles the underlying cause of  cystic fibrosis (CF) in 4 percent of patients. The drug, called ivacaftor (brand name Kalydeco), acts by helping the body make better use of a protein that works incorrectly in cystic fibrosis patients.

The underlying research behind this and other drugs that may help even more individuals with cystic fibrosis was the subject of a feature length article, which I edited, called “A Breath of Fresh Air” [preview], and which appeared in the August 2011 issue of Scientific American.

Two things really intrigued me about the work:

1) the incredible way that the CF community has pulled together to prolong lives and improve quality of life for folks with the condition based on solid science, preventive care and smart nursing (by professionals and others)

2) although CF is a genetic condition, the research does not depend on gene therapy–which has had its ups and downs over the past couple of decades. Rather, researchers are trying to come up with drugs that help make the defective protein responsible for cystic fibrosis more effective.

With any luck, ivacaftor will be only the first of several new CF drugs–each designed to counter a different fundamental cause of the condition. Welcome to the era of personalized medicine.

About the Author: Christine Gorman is the editor in charge of health and medicine features for SCIENTIFIC AMERICAN. Follow on Twitter @cgorman.

The views expressed are those of the author and are not necessarily those of Scientific American.





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