North Knoxville, TENN (WVLT) -- 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.
Robert J. Beall, Ph.D.
President and CEO
Cystic Fibrosis Foundation
Your Volunteer Spirit is in needed to help kids fighting CF like Tucker Jack.
You wouldn't know that the 3- year old has Cystic Fibrosis.
Only at medicine time, or when it's time to eat.
He takes 12 medications daily.
Tucker also is hooked up for 30 minute breathing treatments with a "shake" vest-machine twice a day.
This allows the mucus in his body to break up and not clog up.
Father, Rod Jack says "In CF patients, it makes mucus in your body sticky, and nothing can flow properly properly through lungs, intestines, to clean them out."
Jennifer adds, "When certain germs get in our lungs, we can get rid of them, a CF child germs go into their lungs and just stick there, and they can't get rid of it because of the thick mucus."
CF patients have a life expectancy of 37 years.
That's why they need to find a cure now, and they need need funding for the research/medicine.
Rod says, "We need money to buy science, we are fighting the clock, so the fundraising is a big part of our life."
"We're just trying to keep him (Tucker) as healthy as possible until a cure is found so his lungs won't be too far gone," Says Jennifer.
"We pray everyday."
The community can show Volunteer Spirit and help add years to the lives of CF patients on Saturday, May 12 during the "Great Strides" walk and Pajama Run at Victor Ashe Park.
The fun starts at 9am!
**Also- for the month of May - Papa Murphy's take and bake pizza is giving $1 to CF from every Mini Murph Pizza Kit sold! The kits are only $3!