Cystic Fibrosis patient able to access breakthrough drug treatment
The international Cystic Fibrosis community has been excited by the positive results of a new drug that promises for the first time to reverse the genetic abnormality seen in a small sub-set of Cystic Fibrosis patients who have the genetic mutation called G551D.
Ernie Bindel has Cystic Fibrosis as a result of the G551D mutation and was enthusiastic about the positive impact the medication has had on his condition. He said,
“I felt better once on the trial and the LIWA clinical trials staff took regular standard tests that showed I wasn’t imagining an improvement.
“I have been on the trial for over a year and this is the longest period I have remained out of hospital in my life. Over all I feel better, cough less and can take part in more physical activities,” he adds.
“Another positive was that the drug was administered orally rather than intravenously, so it was quick and easy to take with no needles involved.”
Director of the Lung Institute of WA, Prof Philip Thompson, said that it was very exciting for the Institute to be involved in studies that trialed medications developed to target the underlying cellular defects.
“LIWA has a highly regarded Clinical Trials Unit evaluating new therapies and internationally recognized Genetics Unit that works to identify the genetic basis of respiratory diseases and identify new molecular targets. Medications such as VX-770 and VX809 demonstrate the end points that such research can generate. It is very encouraging for everyone at LIWA to see how their work at a laboratory level can translate into clinical practice and help individuals such as Ernie enjoy better health.