vx-661

[dasjsmum]

it didnt sound to me that the 809 isnt working....and the trial 770x809 has only just started, so there arent any results for that yet (770x809)...and we know that the 809 doesnt work by itself. It sounded like they're developing a second drug, 661, which is fantastic for df508ers!!! Your hopes dont just hinge on one drug anymore. And, either or 809 or 661 may be used in conjunction with 770...this is great news.
<br />
<br />Also, to result in normal non cf functioning, you only need one of your mutations to work properly, so there doesnt have to be anything that targets dd508, as long as it works on the df508, it doesnt matter what your other mutation is. My son has G551D and d508. The 770 works on the G551d mutation with results as per the vertex release and so the person then functions the same as a carrier does aka your parents.

 

[knobby]

From what I've always read, Vertex never believed that vx-809 would completely treat CF, even when combined with vx-770 so continued development of more effective compounds was always the goal.
One can speculate as to how effective vx-809 combined with vx-770 can be based on some of the in vitro and in vivo studies and I'm hopeful for those that have the df508 defect(s), it will be clinically beneficial.
Now for some wild speculation...
Looking at the Vertex vx-809 trial for those homozygous for the DF508 defect, the high dose treatment reduced sweat chloride by 8 mmol/L (<a target=_blank class=ftalternatingbarlinklarge href="http://investors.vrtx.com/releasedetail.cfm?releaseid=442429).
">http://investors.vrtx.com/rele...fm?releaseid=442429).
</a>In vitro studies show that vx-770 can increase CFTR activity by ~4+ fold for the DF508 defect (<a target=_blank class=ftalternatingbarlinklarge href="http://cfnews.us/).
">http://cfnews.us/).
</a>Mixing measurements here makes it difficult to see what the combined effectiveness is so here comes the guess work.
Lowering the sweat chloride by 8 points would translate to about ~2% CFTR activity (very speculative here). Multiply that by 4 fold increase for vx-770 and that would take you to about 8+% CFTR production for double DF508 CFers but a total of only 4% if the other mutation is a type 1 for example.
8% would be significant but still probably slightly above the 60 mmol/L threshold.
Based on the in vivo studies, it's hopeful that vx-770 will be effective against any missense mutation. There is no in vivo data on vx-809 on any missense mutations that I've seen but I'm hoping it will show some benefit there too.

 

[knobby]

From what I've always read, Vertex never believed that vx-809 would completely treat CF, even when combined with vx-770 so continued development of more effective compounds was always the goal.
One can speculate as to how effective vx-809 combined with vx-770 can be based on some of the in vitro and in vivo studies and I'm hopeful for those that have the df508 defect(s), it will be clinically beneficial.
Now for some wild speculation...
Looking at the Vertex vx-809 trial for those homozygous for the DF508 defect, the high dose treatment reduced sweat chloride by 8 mmol/L (<a target=_blank class=ftalternatingbarlinklarge href="http://investors.vrtx.com/releasedetail.cfm?releaseid=442429).
">http://investors.vrtx.com/rele...fm?releaseid=442429).
</a>In vitro studies show that vx-770 can increase CFTR activity by ~4+ fold for the DF508 defect (<a target=_blank class=ftalternatingbarlinklarge href="http://cfnews.us/).
">http://cfnews.us/).
</a>Mixing measurements here makes it difficult to see what the combined effectiveness is so here comes the guess work.
Lowering the sweat chloride by 8 points would translate to about ~2% CFTR activity (very speculative here). Multiply that by 4 fold increase for vx-770 and that would take you to about 8+% CFTR production for double DF508 CFers but a total of only 4% if the other mutation is a type 1 for example.
8% would be significant but still probably slightly above the 60 mmol/L threshold.
Based on the in vivo studies, it's hopeful that vx-770 will be effective against any missense mutation. There is no in vivo data on vx-809 on any missense mutations that I've seen but I'm hoping it will show some benefit there too.

 

[knobby]

From what I've always read, Vertex never believed that vx-809 would completely treat CF, even when combined with vx-770 so continued development of more effective compounds was always the goal.
<br />One can speculate as to how effective vx-809 combined with vx-770 can be based on some of the in vitro and in vivo studies and I'm hopeful for those that have the df508 defect(s), it will be clinically beneficial.
<br />Now for some wild speculation...
<br />Looking at the Vertex vx-809 trial for those homozygous for the DF508 defect, the high dose treatment reduced sweat chloride by 8 mmol/L (<a target=_blank class=ftalternatingbarlinklarge href="http://investors.vrtx.com/releasedetail.cfm?releaseid=442429).
">http://investors.vrtx.com/rele...fm?releaseid=442429).
</a><br />In vitro studies show that vx-770 can increase CFTR activity by ~4+ fold for the DF508 defect (<a target=_blank class=ftalternatingbarlinklarge href="http://cfnews.us/).
">http://cfnews.us/).
</a><br />Mixing measurements here makes it difficult to see what the combined effectiveness is so here comes the guess work.
<br />Lowering the sweat chloride by 8 points would translate to about ~2% CFTR activity (very speculative here). Multiply that by 4 fold increase for vx-770 and that would take you to about 8+% CFTR production for double DF508 CFers but a total of only 4% if the other mutation is a type 1 for example.
<br />8% would be significant but still probably slightly above the 60 mmol/L threshold.
<br />Based on the in vivo studies, it's hopeful that vx-770 will be effective against any missense mutation. There is no in vivo data on vx-809 on any missense mutations that I've seen but I'm hoping it will show some benefit there too.

 

[rmotion]

<P>Yeah I called a Research clinic that was doing the vx770/809 trials and that trial got put on hold. Wont know until October when they meet with the company.  She did not know why. Ergh. Maybe the vx-661 is moving up.</P>
<P> </P>
<P> </P>

 

[rmotion]

<P>Yeah I called a Research clinic that was doing the vx770/809 trials and that trialgot put on hold. Wont know until October when they meet with the company.She did not know why. Ergh. Maybe the vx-661 is moving up.</P>
<P></P>
<P></P>

 

[rmotion]

<P><BR>Yeah I called a Research clinic that was doing the vx770/809 trials and that trialgot put on hold. Wont know until October when they meet with the company.She did not know why. Ergh. Maybe the vx-661 is moving up.</P>
<P></P>
<P></P>

 

[bkc3]

I saw this on the fda site relating to 661 http://clinicaltrials.gov/ct2/show/NCT01531673
Hope to hear some details. I hope they participate at our clinic.

 

[bkc3]

I saw this on the fda site relating to 661 http://clinicaltrials.gov/ct2/show/NCT01531673
Hope to hear some details. I hope they participate at our clinic.

 

[Anomie]

Awesome!! Thanks for linking that for us. I've been waiting a long time for them to start trials for the 661. I think we're all going to have alot to look forward to hearing about at the conference next fall! Cheers!!

 

[Anomie]

Awesome!! Thanks for linking that for us. I've been waiting a long time for them to start trials for the 661. I think we're all going to have alot to look forward to hearing about at the conference next fall! Cheers!!

 

[cfsucks]

damn wish it would be used on hetero df508

 

[cfsucks]

damn wish it would be used on hetero df508

 

[Anomie]

I think they'll test it on the heterozygous delta's in the second part of the study just like the 809. If you look the completion date isn't until August 2013 so they have alot of time to play around with things.

http://clinicaltrials.gov/ct2/show/study/NCT01225211

 

[Anomie]

I think they'll test it on the heterozygous delta's in the second part of the study just like the 809. If you look the completion date isn't until August 2013 so they have alot of time to play around with things.

http://clinicaltrials.gov/ct2/show/study/NCT01225211

 

[musclemania70]

THANKS for posting this update!
I always wonder why the CFF does not post this information as quickly as it appears. If they want more participation then why do they not post items as they come available?
I find items of value MUCH FASTER on this site than on the CFF website.

 

[musclemania70]

THANKS for posting this update!
I always wonder why the CFF does not post this information as quickly as it appears. If they want more participation then why do they not post items as they come available?
I find items of value MUCH FASTER on this site than on the CFF website.

 

[Juggler54]

the one posted by anomie is actually scheduled to be completed this summer 2012

 

[Juggler54]

the one posted by anomie is actually scheduled to be completed this summer 2012

 

[MCGrad2006]

THATS the one my doctor mentioned a few weeks ago! If I remember correctly, he said 770 alone did NOTHING for d508ers. And then I think he said the 770/809 was not yielding the results they had hoped. He said 661 is doing very good things in pre-clinical trials.