New Cystic Fibrosis Therapy Brings Doctors and Patients to Tears

After three decades of hope, a long-awaited treatment for cystic fibrosis is finally within reach: a new therapy has proven to target the genetic root of the disease, significantly improving patients’ lung function.

Due to the immensely persuasive data, the Food and Drug Administration in the United States was quick to approve Trikafta – a three-drug combination used in the therapy that could benefit 90% of patients with the disease, making it a huge upgrade over the significantly less effective predecessor drugs and having doctors and patients in tears.

“I’m overjoyed,” says Francis Collins, director of the National Institutes of Health in the U.S. state of Maryland, who participated in the 1989 discovery of the gene defect that causes cystic fibrosis. “Thirty years along, with many bumps along the road and so many people waiting and hoping that something like this would happen — and here we are.”

“With treatments like this, we can actually anticipate that if a young child were started on this therapy, they could actually expect to have a normal life expectancy,” says Deepika Polineni, a pulmonologist at the University of Kansas Medical Center, who was involved in the largest Trifakta therapy trial consisting of 403 cystic fibrosis patients – with results showing decreases in lung problems and an increase in quality of life. “This is a breakthrough therapy for people with cystic fibrosis.”