Wednesday, January 23, 2013
Victory for Kalydeco!
I've been following this drug since 2009 when I first tried to get on the trial; and although it was all very promising (I tried to enrol in the trail but was a few weeks too late due to my genes being lost in my notes, grrr) I think the penny finally dropped when I read this article:
It was this bit which amazed me, the first Eureka moment for CF R&D?
"At first, she thought maybe she was just having a good week, or month. Then, she started to bring up more mucus. “Actually, it felt like a flash flood,” she said. “I would cough and it would just come flying out of me, not to be too gross. It was just everywhere. It was much thicker than it used to be. It was constant, all day long, and I thought, ‘Oh, crap, I’m getting sick,’ but I never actually got sick.”
“And as time went on, my amount of mucus decreased, and my cough decreased. Then I started noticing bigger and bigger changes. I was sleeping. I was able to maintain my blood sugars. I could basically eat and not have to worry. I no longer needed any medication for constipation. And the biggest thing for me was, that year was the first year in my entire life that I never got sick.”
"Roe has a port in her chest for intravenous antibiotics. She hasn’t used it since she started the trial in 2009. And more: before she started on the trial, she had a “huge mucus plug” in her chest so bad that she was considering surgery to remove part of her lung; now it appears to be gone.
As she spoke to the Children’s Hospital cystic fibrosis team, she mentioned that her FEV, a measure of her lung function, had been 62%. Now it’s 81%."
The first months supply for me arrives tomorrow. We shall see...:-)