"While I breathe, I hope".
I feel giddily lightheaded as I type this. An indescribable breakthrough took place in the CF community this morning. The first drug of its kind, Kalydeco VX-770, was approved by the FDA to begin treating patients with the G551D genetic mutation. This pill will be taken by patients twice daily and instead of simply treating the symptoms of CF, such as overproduction of mucus, coughing, malnutrition, etc, will actually target the root cause of the disease and prohibit the symptoms from occurring. Quite possibly the closest to a cure that my generation may ever know.
So now, the most common question I'm getting: Am I cured? And the answer is no. There are over 1,000 mutations of the genetic chromosome that causes Cystic Fibrosis. Mine is Double Delta F508, shared with over 75% of the CF population and this particular drug only affects approximately 4%, those suffering from the G551D mutation. In this mutation, a defective protein acts as a locked gate at the surface of the cells, preventing the proper flow of salt and fluid in and out of the cell. Kalydeco helps unlock that gate and restore function of the defective protein, dramatically improving lung function, lowering sweat chloride levels and helping patients gain weight. HOWEVER, the approval of this drug opens doors to fast-tracking other drugs, such as VX-809 which WOULD be my "cure", through the process. VX-809 is currently in Phase 2 of studies with very positive results...Which means within the next 2-3 years, I could begin to live a "CF-less" life. The idea is incredible, almost unfathomable and slightly overwhelming :)
Thank you, first and foremost, Jesus. All of you haters out there can argue that science and man are the reasons that this is happening, but God gave these scientists the brains and knowledge to create the medications that are going to save my life. Thank you to the donors who have given millions and millions of dollars to the CF Foundation on a Hail Mary attempt to do the unthinkable and cure a disease rooted in my DNA. Thank you to my cysters and fibros who have made living with this shitpile of an illness actually worth it. And thank you to my family and friends who have spent days and nights on their knees praying that I would someday live a normal life.
We're halfway there, y'all :)
I feel giddily lightheaded as I type this. An indescribable breakthrough took place in the CF community this morning. The first drug of its kind, Kalydeco VX-770, was approved by the FDA to begin treating patients with the G551D genetic mutation. This pill will be taken by patients twice daily and instead of simply treating the symptoms of CF, such as overproduction of mucus, coughing, malnutrition, etc, will actually target the root cause of the disease and prohibit the symptoms from occurring. Quite possibly the closest to a cure that my generation may ever know.
So now, the most common question I'm getting: Am I cured? And the answer is no. There are over 1,000 mutations of the genetic chromosome that causes Cystic Fibrosis. Mine is Double Delta F508, shared with over 75% of the CF population and this particular drug only affects approximately 4%, those suffering from the G551D mutation. In this mutation, a defective protein acts as a locked gate at the surface of the cells, preventing the proper flow of salt and fluid in and out of the cell. Kalydeco helps unlock that gate and restore function of the defective protein, dramatically improving lung function, lowering sweat chloride levels and helping patients gain weight. HOWEVER, the approval of this drug opens doors to fast-tracking other drugs, such as VX-809 which WOULD be my "cure", through the process. VX-809 is currently in Phase 2 of studies with very positive results...Which means within the next 2-3 years, I could begin to live a "CF-less" life. The idea is incredible, almost unfathomable and slightly overwhelming :)
Thank you, first and foremost, Jesus. All of you haters out there can argue that science and man are the reasons that this is happening, but God gave these scientists the brains and knowledge to create the medications that are going to save my life. Thank you to the donors who have given millions and millions of dollars to the CF Foundation on a Hail Mary attempt to do the unthinkable and cure a disease rooted in my DNA. Thank you to my cysters and fibros who have made living with this shitpile of an illness actually worth it. And thank you to my family and friends who have spent days and nights on their knees praying that I would someday live a normal life.
We're halfway there, y'all :)
Love this post!!! Great Day for all CFer's and their families!!!
ReplyDeleteAwesome news sweetie. You're such a strong woman and amazing fighter. Love you, Linda (Dr Cox's prior nurse).
ReplyDelete