From the national cystic fibrosis conference going on in Florida this weekend.  Frank Accurso is one of the ringleading researchers of Cystic Fibrosis (also my former childhood doctor and current boss).  In the video below, as principle investigator, he speaks of an amazing new study drug, Vertex VX-770. This is not a cure, but it does treat the basic defect, not just the symptoms.  Probably the closest thing I will see to a cure in my lifetime.

Quick and dirty run down of what this treatment means:

CF affects how water and chloride moves in and out of the cells.  In a non-cf body, when working properly the cells keep everything (mostly mucus, digestive juices and such) lubricated and flowing.  With CF, chloride and water doesn’t flow as it should and causes mucus to become very thick and sticky which builds up in the organs, causes infections and destruction.  This drug will treat CF at a level that forces the water and chloride to move in a more “normal” way.  Which is a step ahead of the thick sticky mucus, stopping the domino effect of destruction from happening.

What that means to a Cystic?   If this drug works as planned, a person with CF will take a pill (once a day? twice a day? not sure), this pill will force their cells to work properly, to what degree is still being studied. But even the slightest improvement resulting in thinner mucus would be a savior to a cystic.  Their bodies would work in a more normal fashion. Unfortunatly any distruction already done to the lungs, pancreas, liver, …etc,  would not be reversed. But frankly, I would take that compromise.

CF is caused by a genetic mutation.  There are several mutations that cause CF. VX-770 only targets one specific mutation, which few people with CF have. (Personally, I do not have that mutation) But this is such exciting news. The preliminary data says it is successful. Which means a few more years of studying the drug before it can go to market.  In the mean time maybe they will find a way to treat other mutations using the same technology.