Excellent choice of promising research avenues. A general rule in fast tracking drug development is to improve on an existing product. Orkambi is just such a drug, Kalydeco+a CFTR moderator. In the article, new work with inhaled gene therapy and a better animal model build on a logical approach that has been extensively researched.
Inhaling a CFTR gene impregnated virus was trialed around 1998 with less than impressive results. The animal model was a mouse. Mouse CF was a long way from human CF. What mice responded to was harmful to human tissue. Porcine lungs resemble humans in many ways. An ideal virus can be isolated with reasonable assurance it will work for people. The advancement of genetic technology has improved to the point where healthy CFTR genes are optimized for activation once delivered.
Inhaled viruses loaded with bioavailable CFTR genes enter the cells in the lungs. It has been established that the environment in the lungs change to the extent where the ability to fight infection normalizes according to the article. I find it encouraging to see a return of a promising treatment, albeit not ready for prime time. I hope this comes soon.
LL
