Exciting CF drug news!!

Today, Vertex Pharmaceuticals announced that they are starting the clinical trial in the combination of their two drugs, VX-809 and VX-770. These drugs are targeting the underlying cause of CF- defective or missing CFTR proteins.

VX-770 is targeted at the mutation G551D. Gavin has a copy of this mutation (along with DeltaF508, Ben’s two mutations) and should benefit greatly from these two drugs. From what I’ve heard, there is extremely promising results.

The VX-809 drug targets the DeltaF508 mutations which is the most common. Ryan and I are both carries of the DeltaF508 mutation which is why Ben is considered homozygous for DeltaF508 or a “double delta”.

The trial will evaluate the results of the VX-809 drug alone and in combination with the VX-770. The patients sweat chloride will be tested to see if there are any changes. VX-770 is currently in phase 3 and anticipates results in early 2011. Even though Ben doesn’t have the G551D mutation, it is thought that the VX-770 drug could help the VX-809 drug work better. He could end up taking both or just the VX-809. However, these drugs probably won’t be available to children under 5. I’m okay with that because that gives them another 3 1/2 years to get everything right, just in time for Ben.

There are no words to describe the emotions I get from reading news like this. Joy, promise, hope are just some of the emotions I feel. I would give anything and everything I have so Ben could live CF-free. Exciting news like this just gives me the hope I need.

If you’d like to read more, click HERE.