Thursday, October 1, 2015

Birthday Wish

In 67 years I have had some fantastic birthday celebrations, including a dinner in Rome on my 60th birthday. But today's celebration is the best so far. Today my son starts taking Orkambi.
       This expensive drug -- which costs a quarter of a million dollars every year -- may not be a cure for Cystic Fibrosis but it is the closest thing so far. It's actually a double drug. A couple of years ago, a Boston company, Vertex,  released Kalydeco (ivacaftor), a drug that helps correct the genetic defect in about 5 percent of CF patients. Orkambi, also released by Vertex,  adds lumacaftor to ivacaftor to expand the effectiveness to the vast majority of CF patients. Clinical trials showed the combination to be somewhat effective, increasing lung function about 4 percent, but not as effective as the 10 percent increase that Kalydeco delivers to patients with the rarer genetic defect.
      Nevertheless it's a huge dose of hope to CF adults, whose days have been filled with medications and therapy treatments since they were infants.
       First of all, I want to thank God for keeping Ryan safe through 38 years, way beyond the predictions, so he could live long enough to see this medical advancement. I want to say thank you to everyone who ever supported me in one of those annual CF walkathons because your donations really did help provide research. And I am grateful to the scientists who have made these discoveries. Of course I appreciate Vertex for producing the drug, but I am concerned that the outrageous price is more about greed than need. I pray that  wisdom will prevail and this life-saving drug will be available to all who need it.
       As I blow out the imaginary candles on my fantasy cake (thanks to Diane Carroll Burdick for the ones in the photo!!)  I wish for this drug to work its magic, to go into every cell and make those ion transfers work so the mucus that lubricates my son's exocrine system will no longer be sticky and trapping infections. And God's masterpiece machinery will be returned good working order.

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