Cystic Fibrosis cure getting closer at San Diego lab

No one will call it a cure yet, but a new drug developed in the San Diego lab of Vertex Pharmaceuticals was designed as an actual fix for the gene defect called cystic fibrosis. Ivacaftor, trademarked as Kalydeco,  is a drug that currently is used to treat 4-5% of sufferers of cystic fibrosis, those who have the G551D gene defect. However, if used in combination with another potential CF therapy, VX-661, recent clinical testing results have shown a significant improvement in lung function in people with two copies of the CF mutation Delta F508. The actual government study is available here: Study of VX–661 Alone and in Combination With VX-770 in Subjects …

30,000 people in the United States have cystic fibrosis, most of them will not live to be 40. Cystic fibrosis is caused by any one of several defects in a protein, the cystic fibrosis transmembrane conductance regulator, which regulates fluid flow within cells and affects the components of sweat, digestive fluids, and mucus. The defect is caused by a mutation in the individual’s DNA. The CF gene causes clogging in several vital organs, with the most damage usually occurring in the lungs.

There has been a continual increase in the success of CF treatment in the last 50 years. In 1960, most sufferers of the disease did not live to be 10 years old.

The two treatments are currently going through the last phase of trial on humans. The next step would be to gain FDA approval to use Kalydeco with VX-661.

Part of the cost of developing the new drug was paid for by the Cystic Fibrosis Foundation. The organization has raised funds for a CF cure since 1955. San Diego & Imperial Counties CFF spokesperson Laurent Quenaud said,  “There’s little to no government funding for cystic fibrosis research so we are involved in every single drug that comes out.

(Editor’s Note: This article has been changed to make it more accurate and clear since its initial publication. Thanks goes to Jenn Whinnem for the comments that provided some of the additional information about this topic.)