Cystic fibrosis is a terrible disease, which often results in fatal lung failure by the age of 30. It is the most common genetic life-shortening disease in the Caucasian population, and it is caused by mutations in one single gene. The first gene therapies were tested in clinical trials in the early nineties. It felt like the cure was round the corner. However, more than 20 years later, we are still trying to find the best way of restoring the function of the damaged gene. Last month, the scientific journal Lancet respiratory Medicine published a study where a new gene therapy was tested on cystic fibrosis patients. This therapy was reported by several news outlets as a potential cure for cystic fibrosis by 2020. However, the truth is that the therapy failed to detect a relevant clinical benefit for the patients.
Why the lack of progress?