We have encouraging news to share with you. This morning, Vertex Pharmaceuticals announced promising interim results from a Phase 2 combination study of Kalydeco™ and a potential CF drug called VX-809.
Both therapies are designed to treat the underlying cause of CF and were tested in people who have the most common CF mutation, Delta F508. People with two copies of the Delta F508 mutation who took both drugs in the Phase 2 study showed significant improvements in lung function.
The trial is ongoing and complete results, including data from patients with one copy of the Delta F508 mutation (heterozygous Delta F508), are expected this summer.
These interim findings are important because they show that our approach to target the underlying cause of CF is on the right track. Vertex plans to begin a pivotal trial of Kalydeco and VX-809 in people with two copies of the Delta F508 mutation, pending final study results.
We are pleased that Vertex is accelerating its plans for a pivotal study of the combination treatment in those with two copies of Delta F508. The CF Foundation played a key role in the development of Kalydeco and VX-809, providing significant scientific, clinical and financial support.
We still have much to do, but today’s news is a significant step in our work to defeat cystic fibrosis.
We will not rest until we find a cure.
Thank you for all you do.
Sincerely,
Robert J. Beall, Ph.D."
Sincerely,
Robert J. Beall, Ph.D."
The part in red APPLIES TO ME.
The rest of it applies to over 90% of CF patients.
The CF world holds its breath... I know I am.
This could be it.
The "cure" of my lifetime.
I'm nauseated.
I'm excited.
I'm terrified.
I'm speechless.
If results are very positive, I will cry. If results are not as good as they had hoped, then so be it. Life goes on as expected. But if this thing is for real and I get to be put on it, I will cry. Probably every day. For a long time. Oh look, I'm crying now. Most of you will never go through this experience. Where the thing you've wanted more than anything else in the entire world was never even in sight and suddenly it may not be far away at all... and then may be soon in my hand for me to take in pill form.... I don't know what I'll do if/when the results are good and I have my prescription in hand. I will fall on the floor crying, praising the Lord for this miracle.
This might actually happen.
Soon.
Hold your breath...
-Andrea
