Source: Forbes | Showing an excellent result, the three-drug regimen of a pharmaceutical company can now soothe the symptoms of cystic fibrosis, a disease considered to be almost impossible to treat. “This is just what we dreamed would someday happen,” says Francis Collins, the director of the National Institutes of Health, who led the discovery of the cystic fibrosis gene two decades ago. “This is a remarkably happy moment to see how far this has come and how incredibly encouraging this news is.”
Known to be a fatal disease, cystic fibrosis is a condition that affects the lungs and the digestive system. Affecting over 75000 people worldwide every year, the disease is characterized by the buildup of thick, sticky mucus that can damage the functioning of the organs.
The drug created by this pharmaceuticals is a combination of three drugs that improved lung function in patients with cystic fibrosis by 9.6 percentage.
Three different studies were carried out and it was said the regimens were generally well-tolerated across all three studies. But the decision of which drug to be used for larger studies are yet to be taken. The efficacy of cystic fibrosis drugs is measured by something called percent predicted forced expiratory volume in one second (FEV1). This is literally the amount of air the patient can blow out in a second, expressed as a percent of what the amount of air a person their age could normally exhale.
This is the turning point in the treatment of cystic fibrosis and can turn out be one of the most important inventions in the history of medical science. But people are still confused about the valuation and affordability of this treatment.