Miller Children’s Hospital chosen for cystic fibrosis clinical trial

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ptc124 clinical research trialsMiller Children’s Hospital Long Beach (MCHLB) is the only center in Southern California and the fourth in the country to be awarded a grant for a clinical research study investigating an oral drug that can potentially improve the status of patients with nonsense-mutation cystic fibrosis (CF). Centers were selected on the basis of their potential to successfully participate in a world-wide study that involves more than 200 patients greater than 6 years of age.

The drug being tested, PTC124, proved promising in early-stage cystic fibrosis trials in Israel and the United States. CF is an inherited disease that causes thick, sticky mucus to build up in the lungs and digestive tract, leading to a shortened life span.

In the current clinical trial, researchers will test PTC124 against a type of cystic fibrosis caused by a mutation in the salt channel gene that blocks critical protein development. The therapy stops the blocking and allows the development of proteins that reverse the effects responsible for nonsense-mutation CF, a genetic form prevalent in approximately 5 percent of cystic fibrosis patients.

“This drug can potentially reverse a life-shortening disease,” says Terry Chin, MD, who is the principal investigator for this study at MCHLB. “After seeing such promising results from earlier trials with PTC124, we are honored to be able to contribute to this cutting-edge study that can potentially represent a major breakthrough for our CF patients.”

The patients selected to participate in the clinical trial will take PTC124 or a placebo pill for 12 months and will be observed for improvements in protein development, lung function and body weight. Changes are expected within 90 days of the first dose.

“By participating in clinical trials such as this one, MCHLB is making leading-edge programs available to our community and contributing to the advancement of medicine,” says Dr. Chin. “Thousands of patients and their families have benefited from our diverse research programs, and we are delighted to do our part in helping find a cure for CF.”

Miller Children’s clinical research programs support clinical trials in many sub-specialties, allowing Miller Children’s physicians—armed with the latest in scientific thinking—to provide state-of-the-art treatment options to children who have illnesses where there is no standard therapy or where conventional therapy is failing.

Also, check out this bbc article about PTC124.

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