New data on experimental drug combination being developed by Vertex Pharmaceuticals of Cambridge, Mass., should give hope to tens of thousands of people suffering from cystic fibrosis, the deadly lung disease that afflicts 70,000 people worldwide and kills most of them in their 20s. If the drug combo proves successful, it could also open up new avenues for genetic research to lead to treatments for terrible diseases, raising the possibility that pairs of drugs developed in tandem could succeed where single pills have failed.
But big questions remain because the data are still early and the results are not quite what researchers expected.
The new combination pairs Vertex’s Kalydeco, a newly approved treatment for CF patients whose disease is caused by a rare mutation called G551d, with another pill called VX-809. Together, the pair of drugs are designed to help patients whose cystic fibrosis is caused by a second mutation, called F508del. Kalydeco, which appears to be incredibly potent in patients with the G551d mutation, doesn’t help the eight in ten CF patients with F508del mutations at all.
It appears that may change when the Kalydeco pill and VX-809 are given at the same time, according to results revealed by Vertex via a press release this morning and shared with Forbes under an agreement they would not be publicized until now.