Kalydeco

[creation301]

I know this has been discussed before, but can't find the link. Are there any DDf508's on Kalydeco? In clinic the other day our Dr. said he thought it was worth a try for every DD due to the small subset that responded well in the study. He even went as far as to ask if we would like to be that experiment at our clinic?
My first resoponse was of course, but then in reading other post and articles, I am not so sure, as it may be a really, really long shot. First, we would have to actually get it approved through insurance. Which would be a HUGE if, I am guessing.
It's not going to harm our daughter, so the worst case is that it would do nothing. Right? My one caution is that she is only 6 (almost 7) and would not be able to articulate the differences if any in her body.
We love research and are very open to her being in trials when it is wise and she is able. The sodium cloride levels would be non-subjective, along with her pft's which is tempting. Just hard to think that no other information would most likely be gleaned.
Anyway, just curious if there are any opinions out there that might be helpful.
Also, if there are any other bits of information to help with insurance, I would love you to PM me.

Thank you in advance for your help.

 

[creation301]

I know this has been discussed before, but can't find the link. Are there any DDf508's on Kalydeco? In clinic the other day our Dr. said he thought it was worth a try for every DD due to the small subset that responded well in the study. He even went as far as to ask if we would like to be that experiment at our clinic?
My first resoponse was of course, but then in reading other post and articles, I am not so sure, as it may be a really, really long shot. First, we would have to actually get it approved through insurance. Which would be a HUGE if, I am guessing.
It's not going to harm our daughter, so the worst case is that it would do nothing. Right? My one caution is that she is only 6 (almost 7) and would not be able to articulate the differences if any in her body.
We love research and are very open to her being in trials when it is wise and she is able. The sodium cloride levels would be non-subjective, along with her pft's which is tempting. Just hard to think that no other information would most likely be gleaned.
Anyway, just curious if there are any opinions out there that might be helpful.
Also, if there are any other bits of information to help with insurance, I would love you to PM me.

Thank you in advance for your help.

 

[musclemania70]

DDF508 did not respond to Kalydeco for the most part. Yes, there were a few individuals who may have had decreased sweat chloride, but the change in sweat chloride was NOT SUSTAINED through week 40 of the trial. Neither were the gains in fev1.

I'm not saying don't try it, but don't be surprised if your insurance says no. Studies showed that DDF508's had no where near the improvements that g551d had and they will use this to deny coverage.

If you have the time, energy, resources and stamina to fight the system, I encourage you to try. Please keep us updated.

I can send you the chest article in PDF that shows the results of the trial specifically for DDF508s if you want to pm me your email address. This may help you to make a better decision. I had a personal meeting with a Dr. on this study and I may be able to help answer your questions if you have any.

 

[musclemania70]

DDF508 did not respond to Kalydeco for the most part. Yes, there were a few individuals who may have had decreased sweat chloride, but the change in sweat chloride was NOT SUSTAINED through week 40 of the trial. Neither were the gains in fev1.

I'm not saying don't try it, but don't be surprised if your insurance says no. Studies showed that DDF508's had no where near the improvements that g551d had and they will use this to deny coverage.

If you have the time, energy, resources and stamina to fight the system, I encourage you to try. Please keep us updated.

I can send you the chest article in PDF that shows the results of the trial specifically for DDF508s if you want to pm me your email address. This may help you to make a better decision. I had a personal meeting with a Dr. on this study and I may be able to help answer your questions if you have any.

 

[moxie1]

I would go for it!
I tried to get my doctor to let me try it, but no go. They said insurance wouldn't cover it even though I told them some others had gotten clearance.
You never know what will happen. Remember, gene modifiers play a big role in things, not just genotype. That's why everyone's course of CF is so different.
I read on another site yesterday that inHolland? (can't remember, so that might not be right) they actually test to see if any CFTR is being potentiated and that with some DDF08 patients it is. Kalydeco would then work for them!
I looked for a link to this info, but I can't find it. I was all over the place yesterday looking up CF/Kalydeco stuff so I have no idea where I saw it. <img src="i/expressions/face-icon-small-sad.gif" border="0">

 

[moxie1]

I would go for it!
I tried to get my doctor to let me try it, but no go. They said insurance wouldn't cover it even though I told them some others had gotten clearance.
You never know what will happen. Remember, gene modifiers play a big role in things, not just genotype. That's why everyone's course of CF is so different.
I read on another site yesterday that inHolland? (can't remember, so that might not be right) they actually test to see if any CFTR is being potentiated and that with some DDF08 patients it is. Kalydeco would then work for them!
I looked for a link to this info, but I can't find it. I was all over the place yesterday looking up CF/Kalydeco stuff so I have no idea where I saw it. <img src="i/expressions/face-icon-small-sad.gif" border="0">

 

[musclemania70]

Emailed the pdf to individuals who requested it. Let me know if it you didn't get it.

Gene modifiers CAN affect the course of CFTR. Its just that there has been no way to get this data broken down and collected to provide a case for DDF508s with these modifiers...

 

[musclemania70]

Emailed the pdf to individuals who requested it. Let me know if it you didn't get it.

Gene modifiers CAN affect the course of CFTR. Its just that there has been no way to get this data broken down and collected to provide a case for DDF508s with these modifiers...

 

[Brad]

Correct double Df508 had little respond to Kalydeco .
But people with a single copy of Df508 are having marked improvement taking Kalydeco ,VX770.

I am working on getting it myself.

 

[Brad]

Correct double Df508 had little respond to Kalydeco .
But people with a single copy of Df508 are having marked improvement taking Kalydeco ,VX770.

I am working on getting it myself.

 

[zaj1139]

My grandson has 3 mutations, but one is Delta F508 the other is R560K and Y569C ? Does that make sense? Ican't figure out how to tell the mutation (c.1679G>A and 1706A>G). Do you think since he has the DF508,that Kalydeco would be worth a try? He is 4 and on Medicaid

 

[zaj1139]

My grandson has 3 mutations, but one is Delta F508 the other is R560K and Y569C ? Does that make sense? Ican't figure out how to tell the mutation (c.1679G>A and 1706A>G). Do you think since he has the DF508,that Kalydeco would be worth a try? He is 4 and on Medicaid

 

[rmotion]

Here is the rub. I was able to get my cf doc to write a script but insurance says no, not G551. But my beef is how clinic and the CFF handles this. I know the research says that vx770 it doesnt do anything for 508's but it is how the attitude is we are not going help you get it to try. Their hands are tied they do not want to rock the CFF boat.
So why if this is the 1st drug ever to treat CF are they not just trying it on everyone - the cost, the backlash of a let down that it is not a homerun on its own. IT is very frustrating - its like asking a drowning man how to swim - we are drowning and have to tread rough waters on our own.
So here is how it will play out. VX770 is the first step at $1000 per day then next step (we leapfrogged over vx809) to VX661 (which is at best 2 years away) and the protocol will be vx770 and vx661 at $3000 per day -is it all about a bigger payout?
Please let me hear your viewpoints. Is it worth trying VX770 on its own, I would think yes. Again I make the baseball analogy - I just need a base hit, hell I just want out of the F'ing dugout!

 

[rmotion]

Here is the rub. I was able to get my cf doc to write a script but insurance says no, not G551. But my beef is how clinic and the CFF handles this. I know the research says that vx770 it doesnt do anything for 508's but it is how the attitude is we are not going help you get it to try. Their hands are tied they do not want to rock the CFF boat.
So why if this is the 1st drug ever to treat CF are they not just trying it on everyone - the cost, the backlash of a let down that it is not a homerun on its own. IT is very frustrating - its like asking a drowning man how to swim - we are drowning and have to tread rough waters on our own.
So here is how it will play out. VX770 is the first step at $1000 per day then next step (we leapfrogged over vx809) to VX661 (which is at best 2 years away) and the protocol will be vx770 and vx661 at $3000 per day -is it all about a bigger payout?
Please let me hear your viewpoints. Is it worth trying VX770 on its own, I would think yes. Again I make the baseball analogy - I just need a base hit, hell I just want out of the F'ing dugout!

 

[rmotion]

<strong>creation301</strong><img src="i/dominant/trans.gif" alt=" " width="1" height="8" border="0" />That makes all the difference at least your doc is pushing for you. I had the opposite experience and felt like I had to bring it up I had to push I had to beg. And then when I needed more help they said they are not going to do anything for me - I am on my own with this. Anyway I can have my doc talk to your doc?

 

[rmotion]

<strong>creation301</strong><img src="i/dominant/trans.gif" alt=" " width="1" height="8" border="0" />That makes all the difference at least your doc is pushing for you. I had the opposite experience and felt like I had to bring it up I had to push I had to beg. And then when I needed more help they said they are not going to do anything for me - I am on my own with this. Anyway I can have my doc talk to your doc?

 

[rmotion]

.....DDF508 did not respond to Kalydeco for the most part. Yes, there were a few individuals who may have had decreased sweat chloride, but the change in sweat chloride was NOT SUSTAINED through week 40 of the trial. Neither were the gains in fev1. I'm not saying don't try it, but don't be surprised if your insurance says no. Studies showed that DDF508's had no where near the improvements that g551d had and they will use this to deny coverage.

Well the devil is in the details. F508's had no where near the improvements, but wait there was some improvement 3-8% - hello that is something to hold onto. NOt enough to get a new drug approved but I'll take a small improvement over what we have now! right?
This is the crux of the argument we need to make. Not a homerun but there could be some help !

 

[rmotion]

.....DDF508 did not respond to Kalydeco for the most part. Yes, there were a few individuals who may have had decreased sweat chloride, but the change in sweat chloride was NOT SUSTAINED through week 40 of the trial. Neither were the gains in fev1. I'm not saying don't try it, but don't be surprised if your insurance says no. Studies showed that DDF508's had no where near the improvements that g551d had and they will use this to deny coverage.

Well the devil is in the details. F508's had no where near the improvements, but wait there was some improvement 3-8% - hello that is something to hold onto. NOt enough to get a new drug approved but I'll take a small improvement over what we have now! right?
This is the crux of the argument we need to make. Not a homerun but there could be some help !

 

[musclemania70]

Doctors should not be writing the script according to the CFF if the patient does not have g551d. Can't get mad at the docs for saying no.....

Heterozygotes are a completely different story---they have and do show benefit from Kalydeco even if they don't have g551d.
The OP was discussing homozygotes only.

 

[musclemania70]

Doctors should not be writing the script according to the CFF if the patient does not have g551d. Can't get mad at the docs for saying no.....

Heterozygotes are a completely different story---they have and do show benefit from Kalydeco even if they don't have g551d.
The OP was discussing homozygotes only.