Tuesday, January 12, 2010


Just missed out on the VX-770 trial. I asked my CF Team what my genotype was in September. Unfortunately it was buried in my notes somewhere so the team had to get a third party Gopher to find it from my notes. I got the letter back on Christmas Eve, probably missed out on the trial by a matter of days. C'est la vie...

From: J
Sent: 11 January 2010 15:00
Subject: VX-770 Trial

Hello ,

I am a 38 year old male with cystic fibrosis. I have recently learned that my CF Genotype’s are G551D / N1303K. I have been told that I may be eligible for the VX-770 medical trial. Would it be possible for you to give me any further details?

Kind Regards, J

From: DJC
Sent: 11 January 2010 17:22
To: J
Subject: RE: VX-770 Trial

Dear J

I am really sorry to disappoint you, but recruitment to the trial you mention closed just before Christmas as the drug company had exceded the numbers they needed.

On a brighter note, they are planning an interim analysis after everyone has completed 6 months of treatment, ie. early summer, and if things are looking good, they will be applying for a license to allow your clinical team to prescribe this agent. It's difficult to predict how long this will all take, but they will certainly be trying to get it through as fast as they can.

I'm sorry not to have more positive news for you personally

Best wishes, J

I'm not too disappointed by this really; in the long term this is good for the CF Community because we are actually getting to the stage where we may be able to take a gene-correcting drug that may stop things like pseudomonas getting established in our lungs, it may stop our pancreases becoming progressively more damaged, and it may prevent us developing diabetes. Fingers crossed!

Further reading here on the cystic fibrosis transmembrane conductance regulator (CFTR), and on G551D and N1303K. If anyone can explain to me in more detail how the CFTR works then please let me know ;-)


Gemma said...

ohh you have different genes! I dont get all this stuff but I assume it will only help people with your gene type? It's all good news though xx

Woody said...

Although the drug is targetted at the G551D mutation, I think they are testing the drug on DF508 patients as well. I have no idea how it works!

Anonymous said...


Robbie said...

Sorry you missed it Woddy, Good news about the 6 months time though!