Modern Medicine

Our friends and family have been incredibly supportive since finding out about Sienna's CF diagnosis.  They have researched Cystic Fibrosis thoroughly to understand what we are dealing with.  They have attended CF fundraising events.  They have called us just to share positive stories about a friend of a friend that's lived a long, happy life despite having CF. We are so thankful and appreciative of this support!

One common topic that comes up often is the advancement made in medicine for treatment against CF.  Therefore, I wanted to post about the newest FDA approved drug on the market for a small percentage of those with Cystic Fibrosis, Kalydeco™.  Kalydeco™ is the first FDA approved drug that actually remedies the way the defective gene works in those with the genetic mutation G551D.  Unfortunately, Sienna does not have this mutation.  The good news for us is that there are plenty of trials in progress testing the effectiveness of Kalydeco™ in conjunction with another drug, VX-809, for those individuals with at least one copy of DF508 (which Sienna does have).  According to The Cystic Fibrosis Foundation website, 50% of the CF population has a double copy of DF508 and about 40% have a single copy.  We are optimistic that a drug combination for Sienna's mutations will become available over the next several years!  

The Today show had a story this week about a 40 year old man with CF battling his insurance company, United Healthcare, for over a year to get Kalydeco™ covered.  The monthly cost for this drug is $25,000.  His health was deteriorating and Kalydeco™ was his only real chance to survive.  He was denied coverage because he does not carry the G551D mutation, which is the only gene mutation the FDA has approved Kalydeco™ for.  The "twist" in the story is that his sister, who has the exact same mutations, doctor, and insurance company, had gotten Kalydeco™ approved for coverage from the start.  If she hadn't been approved, I doubt this would have made National news...but I'm thankful it did as it really brought awareness to the disease!  The price tag on medicine these days is a scary thing.  The one thing that stuck with me after our initial clinic visit is that we were told "Cystic Fibrosis is one of the most expensive diseases one can have." Of course panic set in as no parent wants to feel like they may not be able to afford the absolute best treatment for their sick child.  I hope we never have to battle insurance to cover a life saving medicine! 

Check out the Kalydeco™ and CFF sites (both linked above) to read more about the trials taking place today.  Both Joe and I are open to getting Sienna involved with a drug trial if her CF clinic team thinks it would be of benefit to her.  We will do anything to ensure our little girl lives a happy and healthy life!

Find a cure for Me!