UAB says new drug could treat Cystic Fibrosis and other diseases

BIRMINGHAM, AL (WBRC) – A researcher at UAB says that science is closer to creating new drugs that will be able to treat genetic "nonsense mutations" that cause diseases like Cystic Fibrosis.

Dr. David Bedwell with the UAB Department of Microbiology presented his findings on an experimental drug called Ataluren on April 26th at the Experimental Biology 2010 conference in Anaheim. Bedwell claims the drug could be effective in treating over 2,400 genetic disorders.

"When you treat a genetic disease, the bottom line is how much of the missing protein do you need to restore to have a therapeutic benefit," Bedwell says. "It comes down to the threshold of protein rescue. For some diseases, it might be 1 percent of protein you need restored, and for other diseases you may need 50 percent of protein restored."

In the study, Bedwell found that Ataluren restored up to 29% of normal protein function in mice with Cystic Fibrosis. A researcher unaffiliated with UAB has also found the drug to be effective in mice with Duchenne muscular dystrophy.

Ataluren is now being tested in humans.

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