Praise Father God from Whom all blessing flow!
Today, July 2, 2015, the FDA approved Orkambi, the second drug developed by Vertex to counteract the underlying genetic defect that causes Cystic Fibrosis, as opposed to treating symptoms.
You can read the press release here: http://www.cff.org/aboutCFFoundation/NewsEvents/7-2-FDA-Approves-Orkambi.cfm
Orkambi has only been approved to treat patients who have two copies of the most common mutation, known as F508del. The initial approval is only for patients 12 and older, but will be available for about 8,500 people.
Can you imagine how those 8,500 people feel today? Can you imagine how their families feel? I am so happy for each and every person who's lives were literally changed today. In my mind I picture a mother and father with a 14 year old boy who has CF. They have watched him struggle for nearly his entire life to grow, thrive, breathe and just be a little boy. They have stayed up countless nights listening to him cough, they have fought hard for weight gain and felt crushed when he required a feeding tube. The mom and dad have shed tears in CF clinic rooms as his lung function numbers declined, and held their breath waiting for cough cultures to come back, only to have to face another new bacteria growing in his lungs requiring IV antibiotics. They have missed work, school, holidays, family gatherings because of hospitalizations. Their 14 year old has always known that he is different and has been left out of sports, sleepovers, birthday parties, school activities, all because of infection risks or because he had to do treatments or because he was just too sick to participate. I can picture all of that, but I cannot imagine how they must have felt yesterday hearing the news that there is now a drug to treat the underlying cause of their son's CF. I imagine that today was probably one of the happiest days for them. What hope for his future was ignited today? What burden was lifted off their hearts? I can only imagine. Praise the Lord!
Orkambi is not considered a cure because it still requires the patient to take the medication daily in order to combat cystic fibrosis, and it is less effective than Kalydeco is for patients with gating mutations. However, it is a significant step forward and it was all made possible because of the investment in research by the Cystic Fibrosis Foundation.
Crosby will not benefit from Orkambi because he does not have the mutations that it affects. However, it is a huge step forward in research alone. Right now the Foundation is investing in more research to pioneer new technologies to repair the defective CF gene. Kalydeco and Orkambi are just the beginning.
I am so thankful for the CF Foundation and for everyone who has donated to help the Foundation realize its mission of controlling and CURING CF. Please believe me when I say that every dollar counts!!! The Foundation has invested hundreds of millions of dollars to help develop CF drugs and therapies.
Thank you for being on this path of hope with us as we await Crosby's cure.
Love,
Carmen
PS: We have several fundraisers coming up in the next couple of months and I will be sharing about them soon.
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