Somedays I wake up and think Piper's life has been plotted out for her. I get lost in the realization that her disease is fatal and I prepare myself for the road to come, the hardships we will experience and as morbid as it sounds, I prepare myself to one day have to let go of my little girl. It's a tough thing to face on a daily basis, but I make a conscience effort to live in the "real world" so that it won't come crashing down around me when the day arrives.
That being said...today I received the most incredible email from one of the team member's at the Tampa Chapter Office for the Cystic Fibrosis Foundation. It appears that all of our fundraising efforts, prayers and pushes for new legislation are beginning to bear fruit. I had tears in my eyes. Light at the end of the tunnel...a ray of sunshine in a rainstorm...a needle in a haystack...whatever you want to call it, here is the announcement from the Cystic Fibrosis Foundation. They are BUZZING over there about this new development, as are we all!!
Foundation and Vertex Announce Positive Early Results for VX-770
March 27, 2008
The Cystic Fibrosis Foundation and Vertex Pharmaceuticals announced today that VX-770, an oral drug in development that targets a basic defect in CF, showed promising results in an ongoing Phase 2a clinical trial for patients who carry the G551D mutation of CF. The drug is being developed by Vertex Pharmaceuticals Incorporated.
Patients who took the drug for 14 days showed significant improvements in several key indicators of cystic fibrosis, including lung function, nasal potential difference measurements and sweat chloride levels. The findings suggest that VX-770 improves function of what is known as the faulty CFTR protein. This early data is promising and could have important implications for studies of other drugs in development.
This is the first time that any potential therapy has improved the abnormal sweat chloride (salt) levels in a person with CF. Excessive sweat chloride is a key clinical indicator of cystic fibrosis. The “sweat test” is the traditional diagnostic test for CF.
“These early results are an extraordinary endorsement of our hypothesis—that small molecules can correct the basic defect and affect the clinical indicators of cystic fibrosis,” said Robert J. Beall, Ph.D., president and CEO of the Foundation. “The emerging data for VX-770 represents the most exciting results we’ve seen from a Phase 2 trial and increase our confidence that we’re on the right track.”
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This could mean SO much for Piper! All I can visualize is land on the horizon in what has been a sea of worry, wait and concern. I pray that this continues to grow and that we are able to actually knock this horridly wretched disease out. To hear them say this is the "first time" about something is truly remarkable, as CF has been around for so many years and has taken so many precious, young lives.
So, that oughta teach me...never give up hope, keep the faith and pray for the best!
~Courtney :-)
such great news!! so excited to hear it!!!!! we love you all... xoxo
Posted by: Keri | April 01, 2008 at 05:16 PM
Your news brought the happiest tears to my eyes!
~Daphne
"Don't give up. Don't ever give up." -Jimmy Valvano
Posted by: Daphne Duncan | April 03, 2008 at 01:41 PM
Courtney, that is wonderful news. As a mother myself, I cannot imagine what it is like to live with what you know about your beautiful girl & the disease on a daily basis. It really makes me realize what to be thankful for in life and what to 'not sweat' over! Keep up hope and faith!
Posted by: Gina | April 09, 2008 at 04:52 PM