So many amazing news headlines and stories have been popping up this month in the CF community - and amazing might just be an understatement... more along the lines of life-changing for a small percentage of those with cystic fibrosis! This is the news that anyone who battles this disease on a daily basis has been waiting to hear. I decided to break down all the stories I've read over the past week to highlight some of the key information as drug makers get closer and closer to new treatments for CF that could stop the horrible impacts of the disease.
So many of you who know Kaelyn and our family have reached out earlier this year to help us raise funds for cystic fibrosis, and while you know in your mind that you did a wonderful thing, you might not know just how big of an impact your contribution really is. Sure you could do what I did - read all the news/information about what's happening on your own. However, it might not all make sense to you - so here I go..... I'm by no means a scientist, but let me try my best to break down what's happening so you can see what a difference your help has made.
First off, no 2 cases of CF are exactly the same. Cystic fibrosis is progressive and the progression of this disease along with the severity of it's symptoms vary from person to person. While it's true the average life expectancy is mid-30's and some live well beyond those years, we unfortunately are still losing lives entirely too young. I'm talking about children who still aren't making it to elementary school, teenagers who don't get the chance to graduate high school, and the list goes on. How is that fair? Is living a life to the age of 35 a long enough life for you? I'm going to safely assume probably not, so why should we settle for it be a long enough life for someone with CF?
I've seen the destruction CF can bring to families, through the eyes of the others in the CF community. It's devastating what this disease has done to some of these families and to the young lives that fought the fight and unfortunately lost their battle. These children so weak, so sick but at the same time so very strong & brave and they are now in the hands of God, as an angel watching over the rest of us like Kaelyn. I know every time I blow out the candles on my birthday cake, I'm wishing for a cure for our little lovebug and I'm certain Chad wishes for the same thing every year too. We've said prayers over and over again to watch over Kaelyn and keep her healthy through the years. Well it looks like some of the wishing and prayers are being heard...
Just last week on October 20th an announcement was made by Vertex Pharmaceuticals that the drug Kalydeco (vx-770) is finally under review for approval by the FDA. This will be the very first drug on the market that will target the underlying cause of cystic fibrosis. Currently any treatments CF'ers receive to date only help to relieve symptoms. While these treatments are effective and prolong life, the new drug should be able to add decades of life over a short period of time! How amazing, right?!? This phase of the drug however will only benefit those who are 6 years or older and who have at least one copy of the rare G551D mutation. Only 4% of the CF population have this copy, but that's approximately 1,200 lives that could potentially be changed in a dramatic way!
HUH?!?? I'm sure some of you are saying that to yourself right now. As some of you might remember reading or hearing me say before, cystic fibrosis is a genetic disease which has thousands of known CF mutations - G551D is just one of them. Kaelyn unfortunately doesn't fall into either of the 2 criteria for the drug - she's only 3 and does not have a copy of the G551D mutation. Her genetic mutations are DeltaF508 and P5L. DeltaF508 is the most common gene mutation of CF. The good news is, right now clinical trials are taking place to evaluate Kalydeco in combination with another drug (vx-809) that would benefit those with a copy of DeltaF508 - And Kaelyn has a copy! It is their hope that with time they will be able to benefit ALL people with CF and this is a major step in that direction!
It's sometimes hard for friends and family to grasp the enormity of cystic fibrosis. Most of the time with Kaelyn, no one would even know she was affected. Right now, we couldn't be more thankful for her health and her pretty much symptom-free 3 years of life she's lived so far. We know in the back of our minds though that cystic fibrosis IS progressive. What's unknown is the path and rate of that progression. We need to stop that progression so kids like Kaelyn can live long, healthy lives!
If you want to be a part of curing a disease that will give futures to many little kids, please continue with us in our fight against CF. As I said in the beginning, life-changing things are happening right now! Keep your prayers coming, we're getting closer!!
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