News for gating, residual function and conductance mutations!
Hello all, With this post I'm hoping to reach patients with gating, residual function and conductance mutations. We have found a combination of two food supplements that have a synergistic effect on CFTR and can potentiate CFTR up to therapeutic levels (around 50%). This effect is backed up by 'in vitro' and 'ex vivo' (human intestinal CF cells) research. I have posted the news on several Facebook groups last week. Some people have started trying the combination and are already noticing effectsÂ…Â… (including some people that are already on K) I know some of you (like ourselves) are waiting for Kalydeco to be approved soon...
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - October 18, 2013 Category: Respiratory Medicine Authors: samaruba Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Information about how VX809 helps F508del and other mutations helped by VX809
I summarised a scientific article released in August that discusses how VX809 helps F508del, how the folding can be improved further and other mutations that were helped by VX809 in this study: http://sixtyfiverosesblog.wordpress....508del-part-2/ (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - October 18, 2013 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Nitric oxide pilot trial demonstrates an 8.8% FEV1 increase after 2 weeks
Novoteris recently announced that the FDA has granted orphan drug designation for inhaled nitric oxide as a treatment for Cystic Fibrosis. The pilot trial demonstrated an 8.8% absolute improvement from baseline after 2 weeks. Phase 2 trials are planned for 2014. More info here: http://sixtyfiverosesblog.wordpress....oxide-therapy/ (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - October 11, 2013 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

ProQR Receives Orphan Drug Status for F508del Therapy
ProQR Therapeutics recently received orphan drug designation for their Cystic Fibrosis therapy that targets the F508del mutation. This approach is different to the CFTR modulation approach (potentiators and correctors), which targets the defective protein. ProQR are targeting the Cystic Fibrosis defect at the RNA level. I have explained this further here: http://sixtyfiverosesblog.wordpress....08del-therapy/ (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - October 11, 2013 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

First Time Post
Hi! I have a 14 year old daughter who just was recently diagnosed with CF...well CRMS a few months ago and now after a hospitalization 3 weeks ago with a mucus plug and partially collapsed lung they have changed the diagnosis to CF. Her genome is M470V and r75q with a 7t/5t variance. She has a borderline sweat test. We are told this is a mild CF. Her lung functions is 75% and she is pancreatic insufficient. This is the first time I have done a forum. I woke up this morning angry, scared, and feeling very alone. My mom suggested I should join a group, so here I am. I am confused by all this genome stuff and just need some s...
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - October 7, 2013 Category: Respiratory Medicine Authors: pootersmom14 Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Just lost my phase 3 place. :( DF508 /i507del
DF508/i507del (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - September 27, 2013 Category: Respiratory Medicine Authors: Rachie Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Back from Denver National Jewish.....(Kalydeco trial)
Hey Everyone! I know there were a few people interested, so I decided to start my own thread about my Denver experience. :) Sooo.... I made it to Denver on Tuesday to be screened for the Kalydeco trial. For those who don't know, it is the trial for residual function mutations. In order to get in, one must meet at least one of 3 criteria: A sweat test under 80, pancreatic sufficient, or diagnosed at or after age 12. We know that I don't meet the second two, so I can only get in if my sweat test comes in at or under 80. It was 83 when I was diagnosed in 1981, so it's really questionable. However, they seemed optimisti...
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - September 6, 2013 Category: Respiratory Medicine Authors: triples15 Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Ataluren Update- Phase 3 trial planned for 2014
PTC had their first investor conference yesterday, here is a brief summary: - Second phase 3 Ataluren study planned to start in the first half of 2014 with CF nonsense mutations - Plan to apply to the EMA (Europe) this year - Potential for combination with Vertex's Kalydeco discussed during investor conference, PTC looking at ways to evaluate this concept More info here: http://sixtyfiverosesblog.wordpress....aluren-update/ (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - August 13, 2013 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Calista Therapeutics: F508del Research
I was just looking through my blog and realised I have not posted about Calista here. Calista Therapeutics are developing medications that target F508del. Pre clinical studies are underway and they are looking for funding for a phase 2a trial. The lead candidate is Procaftor, which appears to work to about 116% of VX809 (see the graph). I've added more info and the graph here: http://sixtyfiverosesblog.wordpress....8del-research/ (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - August 9, 2013 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Proteostasis: F508del Research
Proteostasis have identified multiple compounds that improve the folding and trafficking of F508del. These compounds have a similar level of activity to VX809, and when added to VX809, about 40% of normal function is reached. Trials are planned to start in 2015. I've added more info and some images here: http://sixtyfiverosesblog.wordpress....8del-research/ (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - August 7, 2013 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

809 vs 661
My husband has been thinking of getting on the phase three trial of lumacaftor plus ivacaftor. However we both have some reservations. His mutations are both delta F508. He is 28 years old. He has an FEV1 of 75%. This has been stable for the last decade. And his enzyme levels were in the normal range last time they were checked (the center still suggested he take one enzyme pill with each meal). When I read the phase 2 results for this combo, the results are dismal. There was no FEV benefit. So now for the phase III, they triple the 809 dose. This seems to be somewhat grasping at straws. Like maybe his FEV wi...
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - August 5, 2013 Category: Respiratory Medicine Authors: Pebbles8 Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Vertex Update: New data for other gating mutations and new trial for G551D
I've summarised the latest Vertex investor conference and added some images from the conference here: http://sixtyfiverosesblog.wordpress....vertex-update/ Main points: G551D A new study with VX661 & Kalydeco is planned for the second half of 2013 with patients who have G551D & F508del. Data is expected in late 2013 or early 2014. 2-5 year old Gating Mutations Part 2 is enrolling, results expected in the middle of 2014 Other Gating Mutations Phase 3 data: Mean absolute FEV1 improvement of 7.5% from baseline & 10.7% compared to placebo at 8 weeks (both p<0.0001). Vertex plan to apply to the FDA and...
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - August 1, 2013 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Elvated ALT levels
Hi all I have been taking Kalydeco for almost 5 months now. My liver test have been going downhill since. From normal range to an ALT level of over 250. Any words of wisdom? I have not spoken to the doc yet, but I am soooo worried. I don't want to stop Kalydeco - it is such a life changer. (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - July 31, 2013 Category: Respiratory Medicine Authors: sdeuber Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Australian wanting to get Kalydeco from USA or UK (or anywhere!)
Hi all. Unfortunately Kalydeco (Ivacaftor) is not yet available in Australia. Well it is, but it is $300,000 per year (well outside the price range we can afford unfortunately). We are desperately seeking a way of getting the drug from USA or UK. Does anyone know if this is possible? Has anyone done it? Can anyone work out a way it can be done? It is unbearable to know that Kalydeco is out there and can help my daughter lead a normal life, but we can't get it. Any offers of information would be most appreciated. We are prepared to do what needs to be done to get access to this life saving medication. Thank you (Sou...
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - July 27, 2013 Category: Respiratory Medicine Authors: Shani Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Looking for anyone who has tried to get Kalydeco off label
***KALYDECO OFF LABEL*** We are trying to get together some information to help CFer's in the future get the help they need. If you feel comfortable sharing your information for the benefit of our wonderful CF community please email me at jcurrie@1starbank.com. We are looking for anyone who has tried to get on Kalydeco off label whether successful or not. We are needing your mutations and whether you were approved or denied and any information that could be helpful. Thank you in advance for any information. (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - July 19, 2013 Category: Respiratory Medicine Authors: JENNYC Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums