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Lumacaftor and Bennett's Game Pieces

Wednesday, June 25, 2014

Yesterday was a big day in the CF Community.  A medication named Lumacaftor (also known as VX-809) was moved out of a Phase III clinical trial and is now headed for FDA evaluation.  If approved by the FDA, it will hit the market and hopefully be available  in 2015.

Bennett's two CF mutations are F508del are 621+1G>T.  Since he does not have two copies of the F508del mutation, he is not expected to benefit from Lumacaftor.  Nonetheless, we, along with the entire CF community rejoices when any progress is made in the world of Cystic Fibrosis.

The success of Lumacaftor is one in which we expected but were disappointed to learn did not perform nearly as well as we all had hoped.  So, I feel a little bittersweet about it.  It is showed some effectiveness for those it's supposed to benefit but it's no miracle drug.

Thankfully, the CF Foundation (CFF) does not put all of it's eggs in one basket.  The CF Foundation is very busy supporting and following many other medications in the pipeline such as Ataluren, VX-661 and N6022.  (Apparently, there are others but the CFF has not shared specific information about these yet.)

VX-661 as it is thought to be a stronger drug than Lumacaftor and is expected to be more helpful for those carrying one F508del mutation (which Bennett does).  Testing may begin later this year and, if all goes as expected, we may see FDA evaluation for this drug in 2016.

A CFTR Modulation Expansion timeline from the Cystic Fibrosis Foundation

Since Bennett needs only one mutation fixed to be "cured," we are also paying attention to medications that might benefit Bennett's 621+1G>T mutation:  Gene therapy may work for his 621+1G>T mutation but we're not necessarily hopeful this will benefit him any time soon.  There is some indication the 621+1G>T benefits from Kayledeco in the lab.  So, we are also following this study: http://www.clinicaltrials.gov/ct2/show/nct01685801.

Watching these medications move from one clinical trial to another, in hopes of FDA approval, feels a bit like playing the game "Chutes and Ladders."  My child's gene mutations feel like game pieces that I'm watching be moved across a game board.  Maybe this game piece will work with this strategy, if not, maybe this other game piece will work with this other strategy.

Since promising clinical trials can be unsuccessful and successful clinical trials can be refused FDA approval, it can feel like I never really know where we are in the game.  Am I climbing a ladder?  Or are we slipping down a chute?  Are we at the beginning of the game or nearing the end?  If I think I'm near the end of the game, is there a chance I could be thrown back to the beginning?

I am grateful that we have game pieces to play with.  And I am grateful that we have many different clinical trials and medications to follow.  We are much farther in "winning this game" than we were even 20 years ago.

But I have to remember that even if the medicine I'm following is not successful and the clinical trial I'm watching does not result in the kind of promise I was hoping for, Bennett's breath is not ultimately dependent on a clinical result or an FDA approval.  

Bennett's life has and always will exist in God's hands.  Matthew 4:4 

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