Beautiful Explosion of Hope

     On July 2nd, the FDA announced that it had approved Orkambi, the Vertex-made drug that offers an unprecedented treatment option for people with the most common CF mutation (which happens to be the one I have).  This article contains all of the relevant details from the press release.  The words in black and white represent a level of hope for almost one third of Cystic Fibrosis patients that surpasses what many ever dreamed possible.

     Innumerable people whose lives have been touched by this disease view Tuesday's announcement as a long awaited trumpet which will sally forth a new era in CF care.  With the medicine being shipped to pharmacies as I type these words, I can't imagine or express how it would feel to be the parent of a child with CF, for whom this announcement must seem tantamount to a redefined future.  But as always, I am mindful of the other perspective:  the parents of the ten year old whose child has the mutation (double delta F508) this medicine treats, but is two years away from being allowed to take Orkambi, since it is currently only approved for those 12 and older.  Or the parents of the pre-teen who doesn't have the CF mutation treated by this new drug or Kalydeco, which works in the same way and was approved in 2012.  The gaps in who these incredible medicines can benefit within the CF population, along with the efficacy of the drug, are preventing everyone from using the word "cure," but a quick glance at recent posts here at the CF Blogroll demonstrate how positively most people are reacting to last week's wonderful news.  This one in particular captures the sentiment of a person with CF very well...

     But what about someone like me, whose lungs do not have and will never have the genetic flaw of Cystic Fibrosis lungs?  I have no clue.  The day the announcement was made, I was seeing my pulmonologist, blowing my highest FEV1 yet (95% Wo0t wOot!) and totally unaware that the approval was happening that day.  When I'd asked about Orkambi a few weeks ago, I was given a relatively canned answer:  that they don't like to theorize on giving people medicines that aren't approved by the FDA yet.  Which I can understand.  I also realize that the odds are very slim that anyone with transplanted lungs took part in any sort of trial leading up to the approval of Orkambi.  I will inquire next week about what the drug might mean for transplanted CF people such as myself, and happily volunteer to be in any studies, which I've done before and always had a positive experience with. 

     For now, though, the weekend's fireworks were, in the lives of a large number of CFers and their families, as much about the prospect of breaking free from the restrictions imposed by Cystic Fibrosis as they were about America's enduring freedom.  We continue to hope that Orkambi is one step on the road toward every single person with CF celebrating his or her independence from pills, puffers, and pulmonary problems.