Following Amy's request for this link (<a href="http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2773991/">http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2773991/</a>) and subsequently reviewing it, I am wondering if any CFers with DF508 saw improvement while participating in the VX-770 trials. One friend of mine participated and saw improvement in PFTs. I wonder if there is still CFTR hanging out at the surface that can be helped? I have DF508 + one random mutation. I realize that theorhetically it will take a "corrector" to get the protein to the surface - but we don't know for certain that there isn't ANY cftr already there, correct?
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DF508ers who participated in VX-770 trials
- Thread starter jmiller
- Start date
Following Amy's request for this link (<a href="http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2773991/">http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2773991/</a>) and subsequently reviewing it, I am wondering if any CFers with DF508 saw improvement while participating in the VX-770 trials. One friend of mine participated and saw improvement in PFTs. I wonder if there is still CFTR hanging out at the surface that can be helped? I have DF508 + one random mutation. I realize that theorhetically it will take a "corrector" to get the protein to the surface - but we don't know for certain that there isn't ANY cftr already there, correct?
Incomudrox
New member
Even if there was cftr at the surface which there is not because f508 is not a gating mutation it would not matter because the defect in f508 is the the proteins do not fold correctly and are discarded by the cells before they have a chance to get to the proverbial surface.
Incomudrox
New member
Even if there was cftr at the surface which there is not because f508 is not a gating mutation it would not matter because the defect in f508 is the the proteins do not fold correctly and are discarded by the cells before they have a chance to get to the proverbial surface.
C
cfsucks
Guest
i too wonder if this new drug will work with other mutations that weren't tested and aren't gating mutations.
C
cfsucks
Guest
i too wonder if this new drug will work with other mutations that weren't tested and aren't gating mutations.
Incomudrox
New member
Yes but DF508 is a trafficking/folding defect so if the protein is not folding properly it's useless this overrides the "reduced CFTR at the surface" theory you have.
Incomudrox
New member
Yes but DF508 is a trafficking/folding defect so if the protein is not folding properly it's useless this overrides the "reduced CFTR at the surface" theory you have.
And scientists are "re-writing" what they thought of our mutations (cftr2 project) given what they are finding as a result of studies with the Vertex drugs and the like. Perhaps I'm an eternal optimist (false hope is still hope right), but I think we have much to learn about what each mutation does and how it's function (or lack thereof) might be affected by these drugs.
And scientists are "re-writing" what they thought of our mutations (cftr2 project) given what they are finding as a result of studies with the Vertex drugs and the like. Perhaps I'm an eternal optimist (false hope is still hope right), but I think we have much to learn about what each mutation does and how it's function (or lack thereof) might be affected by these drugs.
Incomudrox
New member
Well since protein synthesis and protein folding are the same thing, VX770 would not help it at all. There is nothing wrong with your cell "gate" unless there is more to your other mutation perhaps it had a bad cell "gate" maybe.
You need a drug like VX809 or VX661 that fixes the protein folding transcription process. The link you posted mentions nothing about F508 on its own seeing any benefit from VX770 only when in combination with G551D. If you have an iPad download the app CFgeneE it may help you.
You need a drug like VX809 or VX661 that fixes the protein folding transcription process. The link you posted mentions nothing about F508 on its own seeing any benefit from VX770 only when in combination with G551D. If you have an iPad download the app CFgeneE it may help you.
Incomudrox
New member
Well since protein synthesis and protein folding are the same thing, VX770 would not help it at all. There is nothing wrong with your cell "gate" unless there is more to your other mutation perhaps it had a bad cell "gate" maybe.
You need a drug like VX809 or VX661 that fixes the protein folding transcription process. The link you posted mentions nothing about F508 on its own seeing any benefit from VX770 only when in combination with G551D. If you have an iPad download the app CFgeneE it may help you.
You need a drug like VX809 or VX661 that fixes the protein folding transcription process. The link you posted mentions nothing about F508 on its own seeing any benefit from VX770 only when in combination with G551D. If you have an iPad download the app CFgeneE it may help you.
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>jmiller</b></i> And scientists are "re-writing" what they thought of our mutations (cftr2 project) given what they are finding as a result of studies with the Vertex drugs and the like. Perhaps I'm an eternal optimist (false hope is still hope right), but I think we have much to learn about what each mutation does and how it's function (or lack thereof) might be affected by these drugs.</end quote>
You are right to have hope. The mutations are getting "reclassed."
You are right to have hope. The mutations are getting "reclassed."
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>jmiller</b></i> And scientists are "re-writing" what they thought of our mutations (cftr2 project) given what they are finding as a result of studies with the Vertex drugs and the like. Perhaps I'm an eternal optimist (false hope is still hope right), but I think we have much to learn about what each mutation does and how it's function (or lack thereof) might be affected by these drugs.</end quote>
You are right to have hope. The mutations are getting "reclassed."
You are right to have hope. The mutations are getting "reclassed."
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>jmiller</b></i> One friend of mine participated and saw improvement in PFTs. </end quote>
I don't think that double delta patients were included in the study. Any improvement was likely from the correction of his gating mutation, or simply placebo effect. In any case Vertex is pursuing 809 and 661 specifically because 770 does not help those of us with trafficking or folding mutations.
Sure with f508del, you might have a very scant few proteins, but they still don't work properly. Even if 770 did help those mutations it would be analogous to trying to improve the taste of your coffee by adding one grain of sugar.
We will just have to wait and see what 809 and 661 bring.
I don't think that double delta patients were included in the study. Any improvement was likely from the correction of his gating mutation, or simply placebo effect. In any case Vertex is pursuing 809 and 661 specifically because 770 does not help those of us with trafficking or folding mutations.
Sure with f508del, you might have a very scant few proteins, but they still don't work properly. Even if 770 did help those mutations it would be analogous to trying to improve the taste of your coffee by adding one grain of sugar.
We will just have to wait and see what 809 and 661 bring.
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>jmiller</b></i> One friend of mine participated and saw improvement in PFTs. </end quote>
I don't think that double delta patients were included in the study. Any improvement was likely from the correction of his gating mutation, or simply placebo effect. In any case Vertex is pursuing 809 and 661 specifically because 770 does not help those of us with trafficking or folding mutations.
Sure with f508del, you might have a very scant few proteins, but they still don't work properly. Even if 770 did help those mutations it would be analogous to trying to improve the taste of your coffee by adding one grain of sugar.
We will just have to wait and see what 809 and 661 bring.
I don't think that double delta patients were included in the study. Any improvement was likely from the correction of his gating mutation, or simply placebo effect. In any case Vertex is pursuing 809 and 661 specifically because 770 does not help those of us with trafficking or folding mutations.
Sure with f508del, you might have a very scant few proteins, but they still don't work properly. Even if 770 did help those mutations it would be analogous to trying to improve the taste of your coffee by adding one grain of sugar.
We will just have to wait and see what 809 and 661 bring.