Cystic fibrosis: a promising treatment should come

A new treatment against cystic fibrosis holds great promise for patients according to a study published on Wednesday evening.

Cystic Fibrosis, rare and incurable genetic disease that affects 5 to 6000 people in France has found an opponent to its size. A new treatment called Ivacaftor is indeed very promising to fight the disease from the results of a clinical trial conducted by the laboratory Vertex Pharmaceutical. The disease that killed Gregory Lemarchal is characterized according to the Oxford Dictionary of medicine by an "abnormal viscosity of the mucus secreted by the pancreatic and bronchial glands." This results in a particular airway obstruction causing the patient respiratory distress. Whatever the development of the disease, slow or fast, the outcome is almost often fatal.
"A significant step forward"

The results of this treatment is good news for patients because until now they had to settle for a treatment of symptoms and not the mechanism of it. The Ivacaftor allows a major improvement and sustainable development of lung function and acts against the other symptoms. The researchers conducted the clinical trial said Phase 3 - the last step before the green light to put on the market - with 161 participants aged twelve and over, all carriers of a rare genetic mutation known as G551D that affects 4 to 5% of people with cystic fibrosis. The people who took the Ivacaftor twice daily for 48 weeks experienced a significant improvement in several key parameters for assessing the disease, compared with the control group treated with placebo. "The Ivacaftor represents a significant advance in the treatment of cystic fibrosis," said Dr. Michael Lonstan, Chairman of the Department of Pediatrics, Faculty of Medicine, Case Western University in Cleveland, Ohio (north) and one of the authors of the study.