There has been a ton of hype going around the Cystic Fibrosis circles about a new drug developed by Vertex Pharmaceuticals.  Actually there are two drugs being studied, the names get confusing because the names are numbers (VX-770 and VX-809) to fix genetic mutations which are also numbers (G551D and delF508).  Both drugs are oral pills that are taken twice a day, both drugs work on the actual genetic mutation within the cell that causes all the troubles with CF at the cellular level.

Neither drug is a “cure” for CF.  To me a cure would mean that the CF mutation would be completely fixed with no further treatments needed, nor would the CF mutation be passed on through the future generations.  The Vertex drugs do neither of that.  Regardless, both drugs are hoped to be almost a miracle for us with CF if the drugs actually do what they are predicted to do.

Before I go on about these drugs (in a future post), I want to air a frustration I am having with the CF community specifically regarding the excitement and hope that this drug will be “the one” to fix all our problems.  Not trying to be Debbie Downer, and don’t get me wrong because yes I am excited about these drugs too, but it seems like everyone is getting wrapped up in the hype and is forgetting the reality that we have been here before specifically with the gene therapy studies of both Copernicus and Targeted Genetics.  Both companies developed a gene therapy (like early to mid ’90’s? maybe) that would also “fix” CF at the mutation/cellular level (but just in the lungs where most of the life shortening damage is done), both showed great promise, but both companies struggled to actually make things happen mostly because of funding and then some other issues… I feel right now the same excitement of the Copernicus and Targeted Genetics studies started up many years ago, but this time I have some reservations to throw all my hope into one drug.  To me, the Vertex drug it is not YET considered to be “the biggest breakthrough since finding the gene in 1989.”

The biggest fear I have is that if for any reason these drugs don’t make it to market or don’t work the way they hoped, or have some horrible side affect that does more harm than good, what is going to be the affect on CF research overall?  I fear that with the excitement and energy put into this one drug we are putting all our hope in one basket, all our effort in one place, that if this doesn’t work, we are screwed because we all assumed it would work.  I wonder if there are other drug companies that may have great ideas, who may be putting their ideas on hold waiting to see if this Vertex thing works out.

This really concerns me because I’m one of the adult Cystics who didn’t grow up with some of the major life extending drug therapies that the kids have now, like Pulmozyme and Hyper Tonic Saline.  I have no extra time to spare. I don’t have time to waste putting all hope and energy in one place.  So please don’t blame me for not getting all psyched up about the Vertex studies.  I will get pissed off when the media claims there is a new “cure” for CF, because if I have to take a pill twice a day (along with the others I am already taking due to permanent lung damage and because I have absolutely no pancreas left), yet if I have children of my own, or grandchildren if I am ever so lucky, there is still a chance they may have to do the same. That, to me, is no cure. So the fight for a cure must go on.