Vertex 770 question

[dasjsmum]

My kids all have DF508/G551D.

The drug only has to work on one mutation....then the cf lungs act like a carrier's lungs ie your parents. The G551D mutation has the channel that transports sodium but not the 'gate' that allows it to transfer across the cell (sorry about my rudimentary knowledge of CFTR, but this is the way it has been explained to me/us).

The DF508 mutation does not have the channel (or has a lessor channel) and does not have the 'gate' either. That's why the 770 works for G551D but not so much for DF508. Vertex hopes that the 809/661 (?) combined with the 770 will enable both functions to work correctly (the channel and the gate), thus allowing sodium to move though the cell, the lack of which is the base cause of cf problems.

Various mutations work in different but similar ways to create the problems pwcf have (which is due to lack of sodium transport). To add to Bill, G551D is about 2% of cf population (I think, could be higher).

 

[dasjsmum]

My kids all have DF508/G551D.

The drug only has to work on one mutation....then the cf lungs act like a carrier's lungs ie your parents. The G551D mutation has the channel that transports sodium but not the 'gate' that allows it to transfer across the cell (sorry about my rudimentary knowledge of CFTR, but this is the way it has been explained to me/us).

The DF508 mutation does not have the channel (or has a lessor channel) and does not have the 'gate' either. That's why the 770 works for G551D but not so much for DF508. Vertex hopes that the 809/661 (?) combined with the 770 will enable both functions to work correctly (the channel and the gate), thus allowing sodium to move though the cell, the lack of which is the base cause of cf problems.

Various mutations work in different but similar ways to create the problems pwcf have (which is due to lack of sodium transport). To add to Bill, G551D is about 2% of cf population (I think, could be higher).

 

[dasjsmum]

My kids all have DF508/G551D.
<br />
<br />The drug only has to work on one mutation....then the cf lungs act like a carrier's lungs ie your parents. The G551D mutation has the channel that transports sodium but not the 'gate' that allows it to transfer across the cell (sorry about my rudimentary knowledge of CFTR, but this is the way it has been explained to me/us).
<br />
<br />The DF508 mutation does not have the channel (or has a lessor channel) and does not have the 'gate' either. That's why the 770 works for G551D but not so much for DF508. Vertex hopes that the 809/661 (?) combined with the 770 will enable both functions to work correctly (the channel and the gate), thus allowing sodium to move though the cell, the lack of which is the base cause of cf problems.
<br />
<br />Various mutations work in different but similar ways to create the problems pwcf have (which is due to lack of sodium transport). To add to Bill, G551D is about 2% of cf population (I think, could be higher).

 

[dasjsmum]

...anyhow, have a look at the FB page above, they have a great diagram that shows how the difference between g551d & df508 (under photos) <img src="i/expressions/face-icon-small-smile.gif" border="0">

 

[dasjsmum]

...anyhow, have a look at the FB page above, they have a great diagram that shows how the difference between g551d & df508 (under photos) <img src="i/expressions/face-icon-small-smile.gif" border="0">

 

[dasjsmum]

...anyhow, have a look at the FB page above, they have a great diagram that shows how the difference between g551d & df508 (under photos) <img src="i/expressions/face-icon-small-smile.gif" border="0">

 

[anien2]

In my opinion if Vertex is waiting for the Anaheim conference to make public the results of phase 2a for the combo it can only mean one thing: good news.

No one would wait for an annual meeting just to say "sorry guys,it is not working", the waiting only makes sense if yo have something big to tell.

 

[anien2]

In my opinion if Vertex is waiting for the Anaheim conference to make public the results of phase 2a for the combo it can only mean one thing: good news.

No one would wait for an annual meeting just to say "sorry guys,it is not working", the waiting only makes sense if yo have something big to tell.

 

[anien2]

<br />In my opinion if Vertex is waiting for the Anaheim conference to make public the results of phase 2a for the combo it can only mean one thing: good news.
<br />
<br />No one would wait for an annual meeting just to say "sorry guys,it is not working", the waiting only makes sense if yo have something big to tell.

 

[Kaethe108]

Well, you can already read the abstracts on the conference website...
About the combo study it says:
"VX-809 coadministered with the 250 mg dose of VX-770 led to >2-fold reduction in sweat chloride compared to VX-809 alone. A slight increase in lung function was evident in the VX-809 plus 150 mg VX-770 group, but not for 250 mg VX-770, though the small sample size challenges interpretation. The safety profile was comparable between groups. These early data support the hypothesis that the coadministration of VX-770 plus VX-809 can significantly improve CFTR function compared to VX-809 alone in patients homozygous for F508del-CFTR and supports further clinical investigation."

 

[Kaethe108]

Well, you can already read the abstracts on the conference website...
About the combo study it says:
"VX-809 coadministered with the 250 mg dose of VX-770 led to >2-fold reduction in sweat chloride compared to VX-809 alone. A slight increase in lung function was evident in the VX-809 plus 150 mg VX-770 group, but not for 250 mg VX-770, though the small sample size challenges interpretation. The safety profile was comparable between groups. These early data support the hypothesis that the coadministration of VX-770 plus VX-809 can significantly improve CFTR function compared to VX-809 alone in patients homozygous for F508del-CFTR and supports further clinical investigation."

 

[Kaethe108]

Well, you can already read the abstracts on the conference website...
<br />About the combo study it says:
<br />"VX-809 coadministered with the 250 mg dose of VX-770 led to >2-fold reduction in sweat chloride compared to VX-809 alone. A slight increase in lung function was evident in the VX-809 plus 150 mg VX-770 group, but not for 250 mg VX-770, though the small sample size challenges interpretation. The safety profile was comparable between groups. These early data support the hypothesis that the coadministration of VX-770 plus VX-809 can significantly improve CFTR function compared to VX-809 alone in patients homozygous for F508del-CFTR and supports further clinical investigation."

 

[anien2]

can´t find the abstracts about the combo. Can you please copy-paste the address for that??

Thank you!!

 

[anien2]

can´t find the abstracts about the combo. Can you please copy-paste the address for that??

Thank you!!

 

[anien2]

can´t find the abstracts about the combo. Can you please copy-paste the address for that??
<br />
<br />Thank you!!

 

[bigstar]

Im so looking forward for the conference!

 

[bigstar]

Im so looking forward for the conference!

 

[bigstar]

Im so looking forward for the conference!

 

[LouLou]

Just wanted everyone to know that my son and I are going to be THE video at the NACFC. I have seen the video and I like it very much. I will share it with you after it shows at NACFC. I have been participating in the clinical trial for Vertex 770 since Ph. 2B in 2008. I don't write this often because I no longer share details on the forum due to Vertex's request. I did however share graphic details of my experience in Ph. 2B that may or may not exist on this site anymore. At the time it was against the code of conduct of the site for multiple reasons but since then they allow people to share at their own discretion.

Through sharing my story and though support by another member; Joanna embarked on getting her children involved with Vertex Ph. 3 in Australia. This is just one example of many on this site of a cross-continental friendship that bloomed into something so much bigger.

My family and I feel very blessed that I have been able to participate in the trial. I travel 8 hours every 8-12 weeks, leaving my son and husband home in order to participate. I am now on open label. It has been a very rewarding experience & I wholeheartedly encourage others to enroll in clinical trials.

 

[LouLou]

Just wanted everyone to know that my son and I are going to be THE video at the NACFC. I have seen the video and I like it very much. I will share it with you after it shows at NACFC. I have been participating in the clinical trial for Vertex 770 since Ph. 2B in 2008. I don't write this often because I no longer share details on the forum due to Vertex's request. I did however share graphic details of my experience in Ph. 2B that may or may not exist on this site anymore. At the time it was against the code of conduct of the site for multiple reasons but since then they allow people to share at their own discretion.

Through sharing my story and though support by another member; Joanna embarked on getting her children involved with Vertex Ph. 3 in Australia. This is just one example of many on this site of a cross-continental friendship that bloomed into something so much bigger.

My family and I feel very blessed that I have been able to participate in the trial. I travel 8 hours every 8-12 weeks, leaving my son and husband home in order to participate. I am now on open label. It has been a very rewarding experience & I wholeheartedly encourage others to enroll in clinical trials.