An Experimental Cystic Fibrosis Treatment Could Make This Patient’s Greatest Wish Come True

According to a story from abc.net.au, Darren Bullock is getting pretty long in the tooth—at least for a cystic fibrosis patient. He is 44 years old, and his lungs currently function at about 40 percent of normal capacity. Even with the most state-of-the-art care, Darren can’t realistically expect to survive for that much longer. However, an all-new experimental drug could change everything.

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.

Changing Patients’ Lives

Darren is happily married and has two eleven year old twins named Alex and Abigail. He has always hoped more than anything that he will be able to see his kids grow up, but it has never felt like a real possibility until recently. The experimental drug, developed in the US, acts on the critical CFTR gene that is mutated in patients. While it does not repair the mutation, it allows the gene to function more normally. The drug acts quickly as well and many patients that have tried it report stunning improvements in breathing ability and reduced infections. It could be real breakthrough.

If approved, the drug is expected to cost a staggering $250,000 annually, and patients would need to take it indefinitely to see the greatest benefit. It will likely still be a few years before the treatment would become available, but if cystic fibrosis patients can survive for that long, this new treatment definitely holds a ton a promise.

 


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