Drugs and Money: Kalydeco

Kalydeco. It's not a cure, but it's a treatment that is a daily cure.

On January 31, 2012, a year and a half before Crosby was born, the FDA approved a drug for CF patients (ages 6 years and older) who have the specific G551D mutation in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene.

About 4 percent of those with CF, or roughly 1,200 people, are believed to have the G551D mutation. Crosby is not in that 4%. {As I have stated on here before, Lance and I have decided not to disclose what Crosby's mutation's are for privacy reasons. We believe that Crosby's genes are personal to him and any information regarding those specific genes needs to be kept private, unless he decides to share later on in life.}

The CF Foundation partnered with a small drug company and contributed more that $100 million for the research and creation of Kalydeco.

For you chemistry nerds out there, here is the chemical structure of Kalydeco (Ivacaftor)

Kalydeco is a pill that is taken two times a day with fat-containing food. It helps the protein made by the CFTR gene function better and as a result, improves lung function and other aspects of CF such as increasing weight gain.

Kalydeco is the first available treatment that targets the defective CFTR protein, which is the underlying cause of cystic fibrosis. Kalydeco was a breakthrough for CF patients because other treatments only combat the symptoms. Kalydeco is so effective, that CF patients on the pill are able to pass sweat tests (test that confirms CF) and their lung function can go up to 100% without any other daily treatments such as the vest or nebulizer being necessary. It's not considered a cure, since the pill must be taken every day, but it is a daily cure.

Taking Kalydeco results in significant and sustained improvement in lung function. I've read other stories and reports where adult CF patients, who have suffered their entire lives, after a week of being on Kalydeco suddenly exclaim, "so this is what it feels like to really breathe!!"

Kalydeco is effective only in patients with CF who have the G551D mutation, so 96% of the CF population is still waiting for their daily cure. And Crosby is still waiting for his.

There was a really interesting article in the New York Times today about the expansion of CF research. I encourage everyone to read it here: http://www.nytimes.com/2014/11/19/business/for-cystic-fibrosis-foundation-venture-yields-windfall-in-hope-and-cash.html

The CF Foundation presser explaining the receipt of $3.3 billion (BILLION!!) can be read here: http://www.cff.org/aboutCFFoundation/NewsEvents/11-19-Expansion-of-Research-and-Programs.cfm

Do you pray? I do and have for as long as I can remember. I'm sure most of you who do pray can remember a time that you prayed to God and said, "If you just give me this one thing, I will never ask for anything ever again.”  Maybe you were 8 and were wanting a new bike. Maybe you were 19 and wanted that boy or girl to ask you out. Maybe you prayed for a job or for a specific outcome on a test, or approval on a home loan.

I've prayed that prayer before for 100 superficial, inconsequential things. But have you ever prayed that prayer for something so important to you that you really would give up everything else?

My prayer is always the same. I would give anything up, never ask for anything again, please just let my baby breathe and live and grow.  Please just ease his suffering and allow him to have the life he deserves. Please don’t make me attend my child’s funeral. Please let there be a cure for cystic fibrosis. 

Thanksgiving is approaching. If I am thankful for one thing this year, it's the Cystic Fibrosis Foundation. Because of the CFF, one day my prayer for Crosby just may be answered and his Kalydeco may be just around the corner!

Momma Bear and her Man Cub.

Love,

Carmen