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Monday, October 18, 2010

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.

3 comments:

  1. I am right there with you!! Love reading about these drugs. So excited about what they can do for our kiddos. Bryce has the G551D and DF508. Thanks for sharing...I'm going to do the same. :)

    ReplyDelete
  2. Thank you so much for sharing this!! As we are in the UK we don't hear about the vertex trials as soon as you guys. Sophie has one DF508 mutation so I am really excited about how the two drugs will work together.

    Great!

    If you hear anymore down the line would you mind posting or letting me know. Would be very grateful.

    x

    ReplyDelete

I love hearing from you, thank you!

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