Cystic Fibrosis - Quest For Kalydeco
Some GREAT inside info from someone who likes our page for those Cystic Fbrosis double F508del people.............."My daughter carries the two copies of delta 508 just before Christmas of 2012 she was really bad she had an infection that was not responding to antibiotics she came home for Christmas and then in January she had a turn for the worst her body couldnt fight the infection she stayed in hospital for 8 weeks she had numerous pneumothoraxs ended up on a ventilator and it was touch and go for a few days but she pulled through it then when she got a bit better came the conversation that we all dread from the consultants its time to start discussing lung transplants my daughter said "im not ready for this" and started to fight again as all cfs do she managed to get het lung function to 38% then a few months after we came of holiday we saw a new trial that was going to be taken place the trial for kalydeco and lumocaftor she had to blow 40% to get on the trial which she did but it was a double blinded study for 6 months. After the 6 months which ended in march she was given the active drug ....so to cut a long story short we went to clinic today sats was 97% weight up to 55 kilos and lung function was a whopping 59% so please whoever decides who gets these drugs please look at the facts of how lives are changing look at the faces of the families the relief it brings to the cfs knowing that they can breathe better and then tell us that its not being funded due to costs. My daughter has not been in hospital for 12 months and has not had any ivs in 12 months she is enjoying life and thats what lifes all about. It really is like looking at a different person!" .... What a fantastic message. Thank you!!)
whoever produced this has been using great strides pages to contact multiple patients, including my family. i am very skeptical that this information is true. the personal details could be easily fabricated by a market mover. we are all on the edge in the CF community.
what a joke!!! i've been following yahoo message for few weeks. I found it's profoudly speculative and all kind of wrong message were sent. for this facebook post, it's so fake that anyone with any scientific background knew this is faked! first of all, it's violating phase 3 clinical trial agreement if this patient is on the TRAFFIC/TRANSPORT trial. any patient is not allowed to talk about their trial results. no one would tell them spirometer results after test. it's against the basic princple of randomized double blinded study!!, common sense. secondly, any patient is not allowed to talk about their study resutls in public domain. otherwise they will be kicked out the study. you know a CF patient won't risk being out of phase 3. third, if FEV1 change is from 40% to 59%, it's 19% absolute change in FEV1. this result is comparable to Kalydeco. if anyone believe Lumo + Kalydeco in F508del homo trial is a miracle drug, he or she is lunative!! if this combo is so much good, there is no need to do press release. you would hear the story all over the internet and within CF community just like Kalydeco 2.5 years ago. fourth, whoever post it is faking the science for the sake of trading. scientifically, in vitro data can't even achieve 19% improvement. CF d508 is most difficult type of CF. is there any data showing combo will produce 19% improvement in any public domain??
I have no idea whether it is true or not, but most investors don't have the inside view of cf living that the post indicates. Re what you wrote...fev is given at clinic to patient so that is normal. Most know not to discuss, but some have on Facebook thinking it is closed group, but that's why I think the analysts take is wrong. Re the 19% improvement, that also is within reason because she was coming off of major problems and some had more than 10%. One thing folks don't recognize is that the drug gives some of the improvement and the improvement gives more improvement because better lungs allow more exercise, which give better lungs. It could be fake...what idiots though to thnk that can really push stock.. But I sure hope it is true.
after rereading your post, I agree. the patients might not talk but those around the patient (parents, relatives, whoever) will see the huge improvement and will talk and buy the stock to 100. So this won't give good data is my guess if you're right.
so you are agreeing with the analyst who talked with the doctors saying the combo would fail??? VRTX up nicely today. data leak??? I agree with your comments though. not sure whether to buy or not. sold some vrtx today and last week. if it goes to 115, I'll be pissed I sold.
Don't get confused about individual's data with group averages. In the 661 Ph 2 trial, one 508 patient who is receiving a placebo had an increase of 26% for FEV1. Does it mean that the patient is faking! All are 508 homozygous patients, but the dispersion is huge.
Getrich, the story quoted by Gladpick from Facebook sounds so real and internally consistent that it is very unlikely a fabrication. The daughter started the trial in September last year and finished the trial in March, then put on the extension study.
Glad, thank you for posting this. The mother's blog is entirely believable. Presumably, the lung function she was referring to was FEV1, and it changed from the prior 38% to 59% at the first several weeks of open-labeled rollover study, whether or not the daughter had received the real drug or not during the double-blinded trial. This is an absolute change of 21% and a relative change of 21/38 = 55%. Astounding. Some placebo effect might be superposed, but it cannot be all placebo effect because it is too large for placebo effect.
Thanks for the post. I am a bit surprised that you copied something so personal into a Yahoo message board, I wonder if you asked the author for permission?
Besides this, obviously the post is intriguing, albeit how could you relate the response of this patient with drug action? This is just anecdotal, and it may or it may not be related to treatment. I hope there were many cases like this in the study arm and not in the placebo groups ... we will learn about the data soon.