vx770 trial results

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LouLou

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Mommafirst</b></i>
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<br />Lauren,
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<br />Is there any indication that VX770 will impact pancreatic sufficiency in addition to lung function?
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<br />I definitely think this sounds promising and exciting -- I hope that the knowledge from this drug will project towards new drugs that adress all the known disease causing mutations.</end quote>
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L

LouLou

New member
I can not speculate how Vertex's drugs are going to effect the future. In fact I can not discuss the trial at all. I was merely discussing the NEJM article. Anyone else read it and want to comment or discuss?
 
L

LouLou

New member
I can not speculate how Vertex's drugs are going to effect the future. In fact I can not discuss the trial at all. I was merely discussing the NEJM article. Anyone else read it and want to comment or discuss?
 
L

LouLou

New member
I can not speculate how Vertex's drugs are going to effect the future. In fact I can not discuss the trial at all. I was merely discussing the NEJM article. Anyone else read it and want to comment or discuss?
 
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mneville

Guest
Our center has patients on the open label now. Their lung function did not go down at all, has stayed steady but they still do the other treatments as well.
 
M

mneville

Guest
Our center has patients on the open label now. Their lung function did not go down at all, has stayed steady but they still do the other treatments as well.
 
M

mneville

Guest
Our center has patients on the open label now. Their lung function did not go down at all, has stayed steady but they still do the other treatments as well.
 
L

LisaGreene

New member
"Is there any indication that VX770 will impact pancreatic sufficiency in addition to lung function?"

I presented a talk at Dr. Orenstein's clinic a couple of weeks ago (he is the one who wrote THE BOOK on CF) and I asked this very question of him. Here's what he said:

We don't really know, however they THINK that it works "system wide." BUT, because the pancrease damage occurs very early on (some research even shows in utero), it will not fix damage already done.

So, speculation again here, these drugs won't correct the pancrease if there is already scarring.

But if all's we have to "worry" about is that- I'll take it! :)

This is SO EXCITING!!!!!
 
L

LisaGreene

New member
"Is there any indication that VX770 will impact pancreatic sufficiency in addition to lung function?"

I presented a talk at Dr. Orenstein's clinic a couple of weeks ago (he is the one who wrote THE BOOK on CF) and I asked this very question of him. Here's what he said:

We don't really know, however they THINK that it works "system wide." BUT, because the pancrease damage occurs very early on (some research even shows in utero), it will not fix damage already done.

So, speculation again here, these drugs won't correct the pancrease if there is already scarring.

But if all's we have to "worry" about is that- I'll take it! :)

This is SO EXCITING!!!!!
 
L

LisaGreene

New member
"Is there any indication that VX770 will impact pancreatic sufficiency in addition to lung function?"
<br />
<br />I presented a talk at Dr. Orenstein's clinic a couple of weeks ago (he is the one who wrote THE BOOK on CF) and I asked this very question of him. Here's what he said:
<br />
<br />We don't really know, however they THINK that it works "system wide." BUT, because the pancrease damage occurs very early on (some research even shows in utero), it will not fix damage already done.
<br />
<br />So, speculation again here, these drugs won't correct the pancrease if there is already scarring.
<br />
<br />But if all's we have to "worry" about is that- I'll take it! :)
<br />
<br />This is SO EXCITING!!!!!
 
S

SarahProcter

Guest
It would be very nice if it could help people who are currently pancreatic sufficient avoid the risk of pancreatitis and/or becoming PI over time...
 
S

SarahProcter

Guest
It would be very nice if it could help people who are currently pancreatic sufficient avoid the risk of pancreatitis and/or becoming PI over time...
 
S

SarahProcter

Guest
It would be very nice if it could help people who are currently pancreatic sufficient avoid the risk of pancreatitis and/or becoming PI over time...
 
M

Mommafirst

Guest
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>SarahProcter</b></i>

It would be very nice if it could help people who are currently pancreatic sufficient avoid the risk of pancreatitis and/or becoming PI over time...</end quote></div>


Agreed. I'm just trying to wrap my brain around how these meds work and change the CF you have. If they are supposed to fix the fundamental protein error from CFTR, then fixing it *should* re-establish functioning. However, I understand that it can mean that any damage already done -- lung scarring or pancreatic scarring, will not be able to heal.
 
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Mommafirst

Guest
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>SarahProcter</b></i>

It would be very nice if it could help people who are currently pancreatic sufficient avoid the risk of pancreatitis and/or becoming PI over time...</end quote>


Agreed. I'm just trying to wrap my brain around how these meds work and change the CF you have. If they are supposed to fix the fundamental protein error from CFTR, then fixing it *should* re-establish functioning. However, I understand that it can mean that any damage already done -- lung scarring or pancreatic scarring, will not be able to heal.
 
M

Mommafirst

Guest
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>SarahProcter</b></i>
<br />
<br />It would be very nice if it could help people who are currently pancreatic sufficient avoid the risk of pancreatitis and/or becoming PI over time...</end quote>
<br />
<br />
<br />Agreed. I'm just trying to wrap my brain around how these meds work and change the CF you have. If they are supposed to fix the fundamental protein error from CFTR, then fixing it *should* re-establish functioning. However, I understand that it can mean that any damage already done -- lung scarring or pancreatic scarring, will not be able to heal.
 
D

dasjsmum

New member
ummm...I would LOVE to comment, but am unable to as my son is in the ph3....send me a p message if you want info....
 
D

dasjsmum

New member
ummm...I would LOVE to comment, but am unable to as my son is in the ph3....send me a p message if you want info....
 
D

dasjsmum

New member
ummm...I would LOVE to comment, but am unable to as my son is in the ph3....send me a p message if you want info....
 
J

john81john

New member
Hi there, I have read through the NEJM G551D article figures... and I found several data very interesting...

1) there were very big improvements in sweat chloride concentrations from baseline to day 28 (people with 150mg or 250 mg drug): 9x very good improvement (40-65 points down), 2x good improvement(35 points down), 4x lower improvement (10-25 points down)

2) there were very different changes in FEV1 %: 3x very good improvement (more than +15--+30%!), 4x good improvement (+10--+15%), 4 x small improvement (+4%--+7%), 4x very small improvement (0--+3%)

3)there is no direct connection between sweat chloride concentrations improvement and FEV1% improvement... 9 people had very great sweat chloride impr., but only 3 people had great FEV1% impr. (of course its different if someone has baseline 85% and he can change it to 100% or someone who has 45% and can improve to 60%)

My conclusion /and only my personal - with maybe some mistakes and tolerance in data showed/ is (have to say that more important for me was FEV1% change)

: 25% people have very good reaction on VX770,
: 35% has good reaction on VX770,
: 40% has small reaction on VX770 (but there is still few % improvement too )

what do you think?
 
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