Aboveallislove
Super Moderator
hee hee..but have you ever read all the inserts for the drugs they're on now? The have to list everything that could happen and it is llikely the cf and not the dog that causes the supposed reaction!
Orkambi is approved!
- Thread starter Aboveallislove
- Start date
Lance2020x
New member
Question here from a ΔΔF508....
I've read a ton of articles on Orkambi and how excited everybody is about it, how it "Helps correct the gene" etc. but I still can't figure out exactly what it will DO for my body. Not the technical answer, but what it will do for my body as a result of using the drug, how will my treatments and disease management be different?
The closest I've come to a quantifiable explanation hidden deep in the loads of technical, medical and insurance language is "Showed improved lung functions" and "Seemed to help correct the base cause of the disease" but.... that seems incredibly vague.
As I'm sure everyone here can understand, I'm finding it very difficult to get excited about this development because I don't want to get my hopes up and be let down, but especially because I don't want to get excited about it doing something that it actually doesn't do.... but I'm finding it really hard to figure out what it DOES do.
I want to be able to tell my friends so that they can be excited with me and celebrate but I honestly don't know what to tell them....
"It's a drug that HELPS to correct the underlying issue"
"What does that mean for you?!? Is this a cure?! Are you going to be able to stop treatments???"
"I....... honestly have no idea."
I had thought that the drug Vertex was working on was basically a CURE (stopped all negative side effects from the defective gene) but from reading a bunch of articles I have still not seen any specific explanation. Is this a 'cure'? Would this stop me having to do treatments? Or is it more like the excitement over new antibiotics that 'help us manage the disease more easily'?
I've read a ton of articles on Orkambi and how excited everybody is about it, how it "Helps correct the gene" etc. but I still can't figure out exactly what it will DO for my body. Not the technical answer, but what it will do for my body as a result of using the drug, how will my treatments and disease management be different?
The closest I've come to a quantifiable explanation hidden deep in the loads of technical, medical and insurance language is "Showed improved lung functions" and "Seemed to help correct the base cause of the disease" but.... that seems incredibly vague.
As I'm sure everyone here can understand, I'm finding it very difficult to get excited about this development because I don't want to get my hopes up and be let down, but especially because I don't want to get excited about it doing something that it actually doesn't do.... but I'm finding it really hard to figure out what it DOES do.
I want to be able to tell my friends so that they can be excited with me and celebrate but I honestly don't know what to tell them....
"It's a drug that HELPS to correct the underlying issue"
"What does that mean for you?!? Is this a cure?! Are you going to be able to stop treatments???"
"I....... honestly have no idea."
I had thought that the drug Vertex was working on was basically a CURE (stopped all negative side effects from the defective gene) but from reading a bunch of articles I have still not seen any specific explanation. Is this a 'cure'? Would this stop me having to do treatments? Or is it more like the excitement over new antibiotics that 'help us manage the disease more easily'?
Aboveallislove
Super Moderator
Hey Lance,
Let me share what I sent to my friends and then add a few comments, fwtw:
Wanted to share the good news and thank you all for your prayers over the last 6 years: On Thursday, the FDA approved Orkambi for those 12 and older. This drug corrects the underlying defect for those that have [son's] mutation--two copies of ddf508. They are finishing the trial for those 6-11 with results in October and hopefully approval early next year and then [son] can start taking (if I can get him to swallow a pill!). While not a cure...it will be a help. Currently, [son's] CFTR gene functions about 1%...yours 100%, mine 80/90%. This med gets it to 25% function. Those who have less severe CF mutations and have CFTR function of about 30% have "mild" cases, so we are hopeful this will help him stay healthy longer while they continue. The next generation of drugs will be in clinic by end of year for trials and then next year to combine with this drug and in vitro shows it gets the function to around 50%-60%. That will likely be another 5 years out, but this first step was 5 years away when [son] was diagnosis...and it goes fast! And they are also working on gene modifications...hopefully by the time he's ready for college they'll have his bad gene sliced out and a good one inserted and with the other meds in the meantime, hopefully his lungs won't be permenantly damaged.
Thank you all for your support and friendship.
so no, it's not a cure. no, it won't mean you don't have to stop your other medicines, but it slows the progression of the disease and improves lung function and for those with weight troubles, it helps them gain weight and probably most significantly, it reduces excerbations by something like 40% (don't have the info handy). This is just the first step and gives more time for folks to stay healthier while we wait for "the cure." Those who benefit from Kalydeco have seen in some cases supposed permenant damage reverse (they're learning it wasn't permenant!), and some have had normal sweat tests and able to stop many treatments. Without lung damage, some with ddf508 will be there too when another corrector is added. Hope this helps and that it works well for you.
Let me share what I sent to my friends and then add a few comments, fwtw:
Wanted to share the good news and thank you all for your prayers over the last 6 years: On Thursday, the FDA approved Orkambi for those 12 and older. This drug corrects the underlying defect for those that have [son's] mutation--two copies of ddf508. They are finishing the trial for those 6-11 with results in October and hopefully approval early next year and then [son] can start taking (if I can get him to swallow a pill!). While not a cure...it will be a help. Currently, [son's] CFTR gene functions about 1%...yours 100%, mine 80/90%. This med gets it to 25% function. Those who have less severe CF mutations and have CFTR function of about 30% have "mild" cases, so we are hopeful this will help him stay healthy longer while they continue. The next generation of drugs will be in clinic by end of year for trials and then next year to combine with this drug and in vitro shows it gets the function to around 50%-60%. That will likely be another 5 years out, but this first step was 5 years away when [son] was diagnosis...and it goes fast! And they are also working on gene modifications...hopefully by the time he's ready for college they'll have his bad gene sliced out and a good one inserted and with the other meds in the meantime, hopefully his lungs won't be permenantly damaged.
Thank you all for your support and friendship.
so no, it's not a cure. no, it won't mean you don't have to stop your other medicines, but it slows the progression of the disease and improves lung function and for those with weight troubles, it helps them gain weight and probably most significantly, it reduces excerbations by something like 40% (don't have the info handy). This is just the first step and gives more time for folks to stay healthier while we wait for "the cure." Those who benefit from Kalydeco have seen in some cases supposed permenant damage reverse (they're learning it wasn't permenant!), and some have had normal sweat tests and able to stop many treatments. Without lung damage, some with ddf508 will be there too when another corrector is added. Hope this helps and that it works well for you.
beautifulsoul
Super Moderator
I'm curious, will this help patients that are DDF508 and post transplant? If it helps with overall defects such as stomach issues and weight gain could we still benefit from it???
Aboveallislove
Super Moderator
Theoretically, it would seem so. I don't know if there are concerns with interaction with post-transplant drugs or not. I know I've seen several threads re Kalydeco and post-transplant. If you search those you might be able to see if anyone uses...
A
Allansarmy
Guest
Hey Lance, I am a father of a 19 year old young man diagnosed at 4 mths ddF508. Of course he wasn't gene tested until later mutations were not important back in 1996. Sorry I digress. Not sure if this helps or not but I will share a story.
My son was doing PFTs at the time about a year ago the Respiratory therapist we had known for over 10 years was getting the device ready with clean mouth piece. I decided to ask him an off hand question about the new drugs coming out soon and I chuckled "are they as good as they say". True story here... He stopped dead in his tracts and stared down at the floor for a minute. At first I thought I said something wrong. He looked me right in the eye with tears in his eyes. "I have seen 5 young people with CF on the trials go from being on the lung transplant list to now living a normal life". They have gained weight too. Their lookout on life is not 10 years from now but 30 or 40. It gave me chills. I will never forget that conversation.
Seen some on Kalydeco already trying to sell their VEST and some trying to sell their Kangaroo feeding pump as they say the do not need it. They need money for college. I and many others erge them to keep it around just encase.
Hope this gives you some hope as I wait for the drug for my son. I know Orkambi is different than Kalydeco but there is hope.
My son was doing PFTs at the time about a year ago the Respiratory therapist we had known for over 10 years was getting the device ready with clean mouth piece. I decided to ask him an off hand question about the new drugs coming out soon and I chuckled "are they as good as they say". True story here... He stopped dead in his tracts and stared down at the floor for a minute. At first I thought I said something wrong. He looked me right in the eye with tears in his eyes. "I have seen 5 young people with CF on the trials go from being on the lung transplant list to now living a normal life". They have gained weight too. Their lookout on life is not 10 years from now but 30 or 40. It gave me chills. I will never forget that conversation.
Seen some on Kalydeco already trying to sell their VEST and some trying to sell their Kangaroo feeding pump as they say the do not need it. They need money for college. I and many others erge them to keep it around just encase.
Hope this gives you some hope as I wait for the drug for my son. I know Orkambi is different than Kalydeco but there is hope.
W
windex125
Guest
I am so happy for those with that double D508 mutation that this medication will help. What a wonderful celebration... I am sure they will continue to work on others as well, were making progress. That great news. Pat/60
T
ToriMom
Guest
Thanks to everyone who is sharing their personal stories and honest questions. I have had the same questions too. It helps so much to have people send inspiring messages. It has been hard for me to be hopeful, but I appreciate these posts so I can wait for the drugs in the future.