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Kalydeco and Class IV Mutations
- Thread starter pjammer65
- Start date
GenH- Thank you for the post, that was really helpful!
DrRoe - I saw that article also, I when I asked my CF clinic if this was the article they were referring to she said that it was not. She said that it will be an article specifically dealing with other classes. Who knows...
DrRoe - I saw that article also, I when I asked my CF clinic if this was the article they were referring to she said that it was not. She said that it will be an article specifically dealing with other classes. Who knows...
They are pancreatic sufficient and the doc told me 4 or 5 for the class. Yes it's extremely rare, my wife is Thai with chinese origins. They took many months to identify it. I would be more than happy if kalydeco could help.
I assume you are on it based on your mutation, is it?
I assume you are on it based on your mutation, is it?
A
Allansarmy
Guest
I found myself nodding with every sentence. If my son were put in this situation your damn right I would do everything in my power to get my son this medication. If you are being evaluated for actual transplant, then they need to be taking a more drastic approach. You don't have time to sit back and let the politics play out here. There has got to be one doctor out there that would write it for you. Heck if I were a doctor I know I would private message you and write it for you myself <img src="i/expressions/face-icon-small-wink.gif" border="0">
I agree with your comments Allansarmy and saveferris2009, that the doctors never fully understand what its like with CF and the urgency we have for medications like Kalydeco. Marcob- Kalydeco is not available in Australia so I am not taking it. Hopefully vertex will apply to the TGA later this year (like the FDA) which takes 9 months, and then it has to be listed on our health system (so we dont have to pay $294,000!). Hopefully we will have it in the second half of next year. People in Aus below 40% can access it before TGA approval now, but I'm higher than 40%.
GenH,
Now it's a matter of months for you, this is really encouraging. Wish you all the best. I think all the time about this treatment and hope it can also work for us...
There are so many mutations that I hope they will choose a different approach soon, I mean, if the drug is safe why don't we let people try it and see the results, then decide if it works for them or not. If we have to wait for a validation for each mutation it will take a while to determinate the right scope.
Cheers
Now it's a matter of months for you, this is really encouraging. Wish you all the best. I think all the time about this treatment and hope it can also work for us...
There are so many mutations that I hope they will choose a different approach soon, I mean, if the drug is safe why don't we let people try it and see the results, then decide if it works for them or not. If we have to wait for a validation for each mutation it will take a while to determinate the right scope.
Cheers
CyrilCrodius
New member
According to THIS http://saltyspark.blogspot.ca/2012/05/where-ive-been.html
It might work on 621+1G->T which is a splice site mutation!
I have that mutation, I have to build a case.
It might work on 621+1G->T which is a splice site mutation!
I have that mutation, I have to build a case.
Dear all,
during an important meeting in Florence, last week, I spoke with Van Goor: he told me that they hope that kalydeco could work for all mild mutation (class IV and V) and that mutations contained in his paper are the only mutations thay have already tested: Now they want to test as more mutations as possible and, on this purpouse, they are also thinkink on "one to one" trial.
So, let's pray!!!!
during an important meeting in Florence, last week, I spoke with Van Goor: he told me that they hope that kalydeco could work for all mild mutation (class IV and V) and that mutations contained in his paper are the only mutations thay have already tested: Now they want to test as more mutations as possible and, on this purpouse, they are also thinkink on "one to one" trial.
So, let's pray!!!!
J
Jenica
Guest
Such encouraging news! My daughter is also R347P, and I've been very curious about the reach of Kalydeco to the other Class IV mutations. Thank you all for your sharing and input!
Very interesting thread. Thanks to all who have contributed. My daughter is the person referred to above that increased 11% on Kalydeco as monotherapy off label.
@pjammer65-Keep asking for the prescription and change doctors if you don't get it!
My daughter is 14 yrs old with F508del and the Class I splice site mutation 621 + 1G > T . According to researchers and doctors, Kalydeco as monotherapy, should not work with these two mutations. However, you may have seen posts here by dramamama (and she has the blog referred to above "saltyspark.") She has the same mutations as my daughter and had great results with Kalydeco off label. After reading her blog, I asked my doctor about Kalydeco, got the prescription because like me, he said "What can it hurt?" I sent it off to the mail order pharmacy and it showed up on my doorstep in two days! My daughter's lung function had hovered in the mid to high 40's for the last 2-3 years, even after IV antibiotics. After just 28 days on Kalydeco, her sweat chloride dropped from 107 to 79 and her FEV1 increased 11% to 57%--the highest FEV1 she has seen in 3 years--with NO antibiotics!
Now the bad news, both my daughter and dramamama have been denied further coverage by our insurance companies. I got it for one month with a $40 copay. I believe it was so new that the ins company had no policies in place yet for off label use. I am so angry and frustrated that I can no longer get coverage, but above all else, I can tell you beyond a shadow of a doubt that it worked for her! She had a repeat sweat test last week and she is back up to 99 and down to 48% FEV1. Breaks my heart terribly. But again, this is real proof that Kalydeco was working for her. We plan to re-appeal with her new data and may be turned down again. I will not stop until we get this medication back into my daughter's life. I am even starting a medical trust account at the bank for her now to begin a fundraising effort. We may never get enough money for even one month, but I am going to try.
@pjammer65, with your mutation I personally feel like there is a good chance that Kalydeco would work for you. You should do whatever it takes to try it at least one month. My only advice would be to get a sweat test before you start so you will have data other than just FEV1.
Saveferris, you are right on target. I feel absolutely no sense of urgency from anyone, including the CF Foundation, to help us gain more access to Kalydeco. It's safe. What will it hurt to try it with everyone? It will hurt their bank accounts. I apologize if this next statement offends anyone, but don't forget that the CF Foundation is receiving handsome royalties from Kalydeco. Great for future research, but being partners with Vertex also means that they cannot help me fight for off label use. How ironic! Something that ACTUALLY WORKS for CF and they can't help me fight for it! Wow! I thought my daughter's case would be welcomed with excitement and awe. Instead, I feel as though they are saying "There, there, dear. Calm down. Don't tell anyone else and get them all excited too. We will get there eventually." Well eventually is not good enough for me. We are there now and my daughter saw results equal to or better than those with G551D. So I will not "calm down" and I will shout my experience from every rooftop I can find.
There are little blue pills out there that may possibly save my daughter's life. And the reason I can't have them? No, not because they are unsafe. No, not because they are illegal. And no, not because they don't work. It's because I am not rich! If I had $300,000, they would be happy to sell them to me. My daughter doesn't have several years to wait for more trials and testing. She needs help NOW! I will be forever grateful to the CF Foundation, Vertex and specifically Dr. Van Goor for discovering this miracle and getting it to market. However, it blows my mind that CF patients are being denied access to this drug because of money. It is ALL about the money, trust me!
@pjammer65-Keep asking for the prescription and change doctors if you don't get it!
My daughter is 14 yrs old with F508del and the Class I splice site mutation 621 + 1G > T . According to researchers and doctors, Kalydeco as monotherapy, should not work with these two mutations. However, you may have seen posts here by dramamama (and she has the blog referred to above "saltyspark.") She has the same mutations as my daughter and had great results with Kalydeco off label. After reading her blog, I asked my doctor about Kalydeco, got the prescription because like me, he said "What can it hurt?" I sent it off to the mail order pharmacy and it showed up on my doorstep in two days! My daughter's lung function had hovered in the mid to high 40's for the last 2-3 years, even after IV antibiotics. After just 28 days on Kalydeco, her sweat chloride dropped from 107 to 79 and her FEV1 increased 11% to 57%--the highest FEV1 she has seen in 3 years--with NO antibiotics!
Now the bad news, both my daughter and dramamama have been denied further coverage by our insurance companies. I got it for one month with a $40 copay. I believe it was so new that the ins company had no policies in place yet for off label use. I am so angry and frustrated that I can no longer get coverage, but above all else, I can tell you beyond a shadow of a doubt that it worked for her! She had a repeat sweat test last week and she is back up to 99 and down to 48% FEV1. Breaks my heart terribly. But again, this is real proof that Kalydeco was working for her. We plan to re-appeal with her new data and may be turned down again. I will not stop until we get this medication back into my daughter's life. I am even starting a medical trust account at the bank for her now to begin a fundraising effort. We may never get enough money for even one month, but I am going to try.
@pjammer65, with your mutation I personally feel like there is a good chance that Kalydeco would work for you. You should do whatever it takes to try it at least one month. My only advice would be to get a sweat test before you start so you will have data other than just FEV1.
Saveferris, you are right on target. I feel absolutely no sense of urgency from anyone, including the CF Foundation, to help us gain more access to Kalydeco. It's safe. What will it hurt to try it with everyone? It will hurt their bank accounts. I apologize if this next statement offends anyone, but don't forget that the CF Foundation is receiving handsome royalties from Kalydeco. Great for future research, but being partners with Vertex also means that they cannot help me fight for off label use. How ironic! Something that ACTUALLY WORKS for CF and they can't help me fight for it! Wow! I thought my daughter's case would be welcomed with excitement and awe. Instead, I feel as though they are saying "There, there, dear. Calm down. Don't tell anyone else and get them all excited too. We will get there eventually." Well eventually is not good enough for me. We are there now and my daughter saw results equal to or better than those with G551D. So I will not "calm down" and I will shout my experience from every rooftop I can find.
There are little blue pills out there that may possibly save my daughter's life. And the reason I can't have them? No, not because they are unsafe. No, not because they are illegal. And no, not because they don't work. It's because I am not rich! If I had $300,000, they would be happy to sell them to me. My daughter doesn't have several years to wait for more trials and testing. She needs help NOW! I will be forever grateful to the CF Foundation, Vertex and specifically Dr. Van Goor for discovering this miracle and getting it to market. However, it blows my mind that CF patients are being denied access to this drug because of money. It is ALL about the money, trust me!
Your post absolutely broke my heart! It is unbelievable that the CF Foundation wouldn't help you. I thought they were on our side. I am so sorry for you and your daughter! That is one of the cruelest things I have ever heard. Your daughter clearly needs the help NOW! And I agree you should shout all of this from the rooftop to try to get some attention to this horrible situation. Someone told me that I should talk to my congressman. If I were you and had the compelling, and simply outstanding results that your daughter did, I would try speaking to your congressman. I don't know it may not help but then again it might. Good luck and God Bless you!!! Keep us posted.
Thanks, JennyC, your post truly touched my heart and it really helps to know others care. I did contact our congressmen and got a nice personal phone call from one assistant. She basically said without FDA approval, there was not much they could do to force the issue for coverage. I am holding on to hope that Vertex may move forward with the "trials of one" that have been mentioned in some presentations, but I haven't heard anything about them recently.
saveferris2009
New member
Pretty sure that the CF Foundation could get in a bunch of trouble from the FDA for promoting off label use of a drug. Although they are not a drug manufacturer, they are funding a great deal of research for these manufacturers.
So similar to a drug company getting fined for promoting a drug off label, I have a feeling the CFF could be under similar limitations. So I'm not surprised at all that the CFF is staying very clear of any sort of off label use/assistance.
So similar to a drug company getting fined for promoting a drug off label, I have a feeling the CFF could be under similar limitations. So I'm not surprised at all that the CFF is staying very clear of any sort of off label use/assistance.
K
kots66
Guest
@tcfuller: I really can't imagine how difficult it must be for you. I hope you get kalydeco approved by your ins as quickly as possible. Hang in there, I'm sure things will start looking up soon.
LittleLab4CF
Super Moderator
The only possible help I may be adding (sorry, I usually read all posts) has to do with insurance. We periodically have bumped heads with our health insurance. My wife's first call to the insurance company solidfied her next action. We hired a laywer. (Granted, we are expensive, but nearly 20 years of my wife and I as employers, we got employee health insurance when we hired our first employee. We have balanced in the coffers of the insurance industries' pile of money.) Regardless, a compouned prescription costing about $5,000 a month was declined. After my doctor exhausted his influence, the laywer armed with policy, insurance law, and this is the fear of the insurance lawyers. Case precedent in health insurance almost always have cases sympathetic to the patient. Honestly it hasn't been expensive. The lawyer seems to know just what to say ; and open sesame. We haven't nor have we needed to do this often. My wife's employer just changes health insurance often.