Kalydeco or VX-770 off lable use?

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rmotion

New member
In order for a drug to be developed and go through FDA approval it has to show some significant benefit and activity. Those with the G551 gene have been taking Kalydeco have shown some real benefit.
In the research there was some benefit with just DF508 in the range of 5-8%, this is not enough to get a drug to pass but it could help.
Question:
Anyone with DF508 in the trials noticed any benefit?
ANyone know of anyone with just DF508 taking Kalydeco off label?
 
R

rmotion

New member
In order for a drug to be developed and go through FDA approval it has to show some significant benefit and activity. Those with the G551 gene have been taking Kalydeco have shown some real benefit.
In the research there was some benefit with just DF508 in the range of 5-8%, this is not enough to get a drug to pass but it could help.
Question:
Anyone with DF508 in the trials noticed any benefit?
ANyone know of anyone with just DF508 taking Kalydeco off label?
 
M

marcob

New member
this can interest you maybe
<a href="http://saltyspark.blogspot.com/2012/03/kalydeco-for-class-i-and-class-ii.html">http://saltyspark.blogspot.com/2012/03/kalydeco-for-class-i-and-class-ii.html</a>
 
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marcob

New member
this can interest you maybe
<a href="http://saltyspark.blogspot.com/2012/03/kalydeco-for-class-i-and-class-ii.html">http://saltyspark.blogspot.com/2012/03/kalydeco-for-class-i-and-class-ii.html</a>
 
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JENNYC

New member
I'm curious too. I've been keeping up with Mandy's blog and soooo happy for her!!! <img src="i/expressions/face-icon-small-smile.gif" border="0"> And I loved Rebecca's latest blog as well!! But so far Mandy is the only one that I've seen that is sounding off about taking it off label. Anyone know of anyone else? We still don't know what our daughters second mutation is, have to be approved by CF Foundation to start the process, but it shouldn't be long!!! I love reading all of the feedback!! It feels so good to know that by 2016 we might possibly have something so great for the DF508's!
 
J

JENNYC

New member
I'm curious too. I've been keeping up with Mandy's blog and soooo happy for her!!! <img src="i/expressions/face-icon-small-smile.gif" border="0"> And I loved Rebecca's latest blog as well!! But so far Mandy is the only one that I've seen that is sounding off about taking it off label. Anyone know of anyone else? We still don't know what our daughters second mutation is, have to be approved by CF Foundation to start the process, but it shouldn't be long!!! I love reading all of the feedback!! It feels so good to know that by 2016 we might possibly have something so great for the DF508's!
 
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musclemania70

New member
Is all that info saying that its basically impossible for HOMO delta 508's to see benefit from the Kalydeco?
No one has piped in from this group to say if they are trying the drug.
 
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musclemania70

New member
Is all that info saying that its basically impossible for HOMO delta 508's to see benefit from the Kalydeco?
No one has piped in from this group to say if they are trying the drug.
 
R

Rummage

New member
No, it is just talking about research that shows one reason why heterozygotes may respond. There are other reasons why homozygotes might respond, like gene modifiers.
 
R

Rummage

New member
No, it is just talking about research that shows one reason why heterozygotes may respond. There are other reasons why homozygotes might respond, like gene modifiers.
 
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saveferris2009

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>musclemania70</b></i> Is all that info saying that its basically impossible for HOMO delta 508's to see benefit from the Kalydeco? .</end quote>

There was a small subset of DDF508's that did very well on the drug
 
S

saveferris2009

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>musclemania70</b></i> Is all that info saying that its basically impossible for HOMO delta 508's to see benefit from the Kalydeco? .</end quote>

There was a small subset of DDF508's that did very well on the drug
 
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Incomudrox

New member
There is also a running theory that because of the auto immune response in CF once the body is making CFTR and doesn't recognize it as it's own it is attacking it which maybe why some cases there is temporary improvement or no improvement off the bat. I just entered a study on this at my hospital.
 
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Incomudrox

New member
There is also a running theory that because of the auto immune response in CF once the body is making CFTR and doesn't recognize it as it's own it is attacking it which maybe why some cases there is temporary improvement or no improvement off the bat. I just entered a study on this at my hospital.
 
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musclemania70

New member
I suggest anyone that has a copy of Delta 508 be granted an RX for this drug. I believe if we all rally together and get this out there, our doctors will submit. Everyone should have the chance to benefit from this drug. Even if its not 'statistically significant' enough to get approved for the deltas, does not mean it won't help.
There is enough evidence to show there MAY be benefit.
WE need to empower ourselves and become advocates for our health. WE DESERVE THE CHANCE TO TRY THIS DRUG.
Who is with me on this?
 
M

musclemania70

New member
I suggest anyone that has a copy of Delta 508 be granted an RX for this drug. I believe if we all rally together and get this out there, our doctors will submit. Everyone should have the chance to benefit from this drug. Even if its not 'statistically significant' enough to get approved for the deltas, does not mean it won't help.
There is enough evidence to show there MAY be benefit.
WE need to empower ourselves and become advocates for our health. WE DESERVE THE CHANCE TO TRY THIS DRUG.
Who is with me on this?
 
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