I ran across this article today and thought it really showed how far CF treatment has come in recent years. It’s a lengthy interview with longtime CF researcher, Bonnie Ramsey. It’s an amazing article about how drugs to treat the underlying cause of CF are in phase II and III clinical trials and what that means for people with CF. If you know anyone with CF, this is as exciting as it gets. Dr. Black at the CF clinic in Kansas City is very excited about this and also feels this is a watershed moment for CF treatment.
When you read the article, keep in mind that Grace has two different CF genes. She has the most common gene, Delta F508. But, more importantly, she also has the very rare gene G551D, only found in 4% of patients. The article explains it better than I could:
“Then there’s the type 3, the G551D, which is what the Vertex product [VX-770] is working on. That’s where the protein gets to the surface, but it’s not properly activated. So it gets up there to the surface of the cell, but it can’t properly transport the salt.”
“But this [Vertex's VX-770] is actually trying to correct the protein. So if it works, we have shown this concept works. You actually can change a dysfunctional protein into a functional channel. Whether it turns out that Vertex is 100 percent successful or not, this is such a giant step forward, it’s like a man walking on the moon.”
Very powerful stuff. Since VX-770 is in Phase III and the trials have progressed well, this opens up the possibility that Grace will be able to take a pill that could essentially change the dysfunctional protein into a functional one. Simply put, if that works, she could take a pill and not have any CF symptoms.