According to a story from The Washington Post, data regarding the recently approved drug Trikafta, which is designed to treat cystic fibrosis, was so convincing that the US Food and Drug Administration (FDA) actually moved to approve the drug five months before the official deadline. The trial results, which have been released at a national conference and have also been published in two medical journals, appear to describe a treatment that not only can improve lung function, but can also help counter the effects of the underlying genetic mutation that causes cystic fibrosis.
About Cystic Fibrosis
Cystic fibrosis is a type of genetic disorder which can have impacts throughout the body, but it is most characterized by the build up of abnormally thick, sticky mucus in the lungs. This mucus becomes a fertile breeding ground and habitat for potentially infectious bacteria. Many patients must take antibiotics for much of their lives. This disorder is caused by mutations of the CFTR gene. Symptoms of cystic fibrosis include progressive decline in lung function, lung and sinus infections, coughing up mucus, fatty stool, poor growth, infertility in males, clubbed digits, and digestive problems. Treatment includes antibiotics and medications or procedures intended to maintain lung function. Lung transplant is an option when lung function declines severely. Life expectancy ranges into the 40s and 50s with good care. To learn more about cystic fibrosis, click here.
A Revolution in Treatment?
Another exciting characteristic of this new drug is that it is expected to be effective for around 90 percent of the patient population, which is a dramatic improvement over current treatments which are limited in effectiveness and only work for a limited portion of patients.
The director of the National Institutes of Health Francis Collins, who was part of the research team that first uncovered the mutation that caused the illness, says that he is “overjoyed” by the data from the trials. While Trikafta is still far from a cure for cystic fibrosis, it still has the potential to truly revolutionize patient treatment and transform the disorder from an ultimately lethal diagnosis to a chronic condition that can be effectively managed, such as diabetes.
Take this patient story as an example of the potential of this drug. 28 year old Sarah Carollo, a special needs teacher, could barely make it down a hallway without stopping to catch her breath because of cystic fibrosis. After participating in the Trikafta trial, her and another trial participant were able to successfully complete a 5k race earlier this month. That’s a story with results that speak for themselves.
