Vertex has done it!

It’s official! Vertex has finally found a way to treat the underlying problem with CF, instead of just treating side effects of this disease.  With the combination of two pills, Kalydeco and VX-809, will now help repair the faulty CFTR protein in CF patients who have two copies of the deltaF508 gene! Basically, this will reverse lung damage. We will now be able to have increased lung function, increased weight gain, and decreased hospital admissions and pulmonary exacerbations.

They’re sending it to the FDA to be reviewed and given the thumbs up by the end of this year and hopefully be available for patients by 2015!

I have the the double deltaF508 gene, so I am beyond excited for this new medical advancement and can not wait to be able to try it for myself hopefully next year!!  

FDA Approves First Ever Pill Targeting Cystic Fibrosis

Photo via Eye Dr. DeLengockey

Yesterday the FDA approved a new drug called Kalydeco, which is the fist ever pill that targets causes of cystic fibrosis, a major leap in finding the potential cure for the condition. Cystic fibrosis is a potentially life-threatening genetic disease that causes unusual mucus build-up throughout the body. The development of Kalydeco, which was mostly funded by the Cystic Fibrosis Foundation, is a major breakthrough in the persistent search for new medication and therapies to help people with the condition.

“Kalydeco addresses the underlying cause of CF, and the science behind the drug has opened exciting new doors to research and development that may eventually lead to additional therapies that will benefit more people living with CF.” - Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. 

Read the full story here.


oh lord I’m crying in my office! Congratulations to the Cystic Fibrosis Foundation for creating this wonderful video and winning the doGooder Nonprofit Video Awards. if you watch this video titled “Adding Tomorrows” you’ll see how much they deserved it!

Awesome doctor update for the day:

Today is the first day in ever in my entire 24 years of living that my pft numbers have been at/above 100%.

Technically, the study is blind and I’m not really supposed to know whether I’m on Placebo or actual Kalydeco/Vertex, but I’m fairly positive your baseline doesn’t skyrocket from 80% on a really, really good day, to 101% with absolutely no other medical changes in a matter of months. Also, that whole utter cancellation of birth control kind of clued me in early. 

This drug is freaking amazing and I hope it works for everyone and isn’t a 7 million dollar copay when the FDA finally approves it for DDF508’s (and everyone else!). 

Vertex Pharma Gets Complete Response Letter From FDA For Kalydeco

Vertex Pharma Gets Complete Response Letter From FDA For Kalydeco

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Vertex Pharma Gets Complete Response Letter From FDA For Kalydeco Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that it received a Complete Response Letter from the U.S. Food and Drug Administration (FDA) for its supplemental New Drug Application (sNDA) for the use of KALYDECO®(ivacaftor) in people with cystic fibrosis (CF) ages 2 and older who have one of 23 residual…

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Vertex Pharmaceuticals

This company, the producers of Kalydeco (the latest drug for CFs with the G155D mutation) are currently doing trials on people with one of my mutations, with the hope that with a combination of two drugs, they can control the symptoms of CF and keep all us DF508’s healthier for longer. People that are on Kalydeco sound like they’re experiencing an amazing change in their quality of life - it isn’t a cure but a successful suppressant. They may only be on phase 2 of the new trials but that’s a whole 2 phases closer than they ever were before. (DF508 is one of my genetic CF mutations by the way, the TYPE of CF I have, if you like - this is promising stuff)


Finally, A Breakthrough for Cystic Fibrosis Patients

Cystic Fibrosis is a terrible disease that effects wonderful, young people. It’s a genetic mutation that causes an over production of salt in one’s body, primarily effecting the lungs and the digestive tract. It doesn’t make life impossible, but it certainly makes it harder. Long hospital visits, lots of missed school, daily treatments, a shortened life, and a struggle for every breath. My beautiful friend Alicia has battled CF for all of her 21 years. This summer, we raised over $3,000 for the advancement of CF research. At the final fundraising event, our team joined hands and her father prayed in thanks for all that we had achieved. I’m not one for praying, but when I heard the words “And Lord, as always, we pray for a cure;” I was moved to tears. As I looked around the circle, I realized that I was not the only one. How could this family remain so positive after 21 years of defeat? After 21 years of terrible disease? I will never forget the awe that I felt in that moment; the immense amount of love, faith, and sincerity. 

And I will never forget the look on Alicia’s face today when she told me that Kalydeco had been approved and that she was calling her doctor this afternoon. It was the best damn thing I have ever seen.


Dominic Purcell - Cystic Fibrosis Quest For Kalydeco (by Cystic Fibrosis)

Cystic Fibrosis touches people from all walks of life, even famous actors! Dominic Purcell from the well known show Prison Break to time out of his busy schedule this week to show surport for the Quest For Kalydeco campain by making this personal plea for Kalydeco to become available in the UK and Ireland as soon as possible for all those with the G551D mutation, including his young cousin Charlie who is a member of our Facebook group! Please share this so our voice can be heard!

Thank you

Clinical Trials and Studies are Underrated

Growing up with CF, my family and I have always been approached about me participating in different clinical trials or studies. When I was in pediatrics my parents always said no thank you to the people conducting research studies and I never really asked why. I just assumed that it must be a bad thing if my parents didn’t want me to be apart of them. I’ve now realized that it was a lot of the fact that my parents both worked and weren’t capable of driving me downtown to doctors appointments all the time for the studies or the potential of the any unknown risks of the drug scared them a bit.

Now that I’m an adult and able to make the decisions on my own, I’ve realized how amazing they really are! The first study I ever did was last summer while I was in-patient at my hospital. The study was checking how high doses of Vitamin D interacted with health in people with Cystic Fibrosis. They just gave me extra vitamin D for two days and monitored my blood levels. That easily I made $25.

Monday morning I went in for a study on nutrition in CF patients. They had me come in fasting, took a bunch of blood levels, my vitals, urine and sputum sample, answer a few questionnaires, took a DEXA scan of my body, and did a test to check how many calories I burned while at rest. In roughly two hours, I made $75 and a free lunch.

I was approached today by my CF team and the hospital’s research coordinator about doing a 42-day study for a Phase 2 drug that works basically the same was as Kalydeco and the new Ivacaftor x Lumacaftor combination drugs, just made by a different company. It will be for patients with two copies of the Delta F508 gene (like I do) and work to repair the faulty gene/protein. I will get paid over $600 and have the chance to get this amazing new drug! All I have to do is come in 5 times total (2 visits are 9hrs long and 3 visits are 2-6hrs long), take my meds for two weeks, and basically let them see how my body reacts to either the new medication or placebo. Pretty easy considering it’s $600, plus they pay for my gas, parking, and any food I buy to eat on the visit days. PLUS, Hopefully I can help the CF world become one step closer to find some pretty amazing drugs and eventually a cure! As long as my right arm doesn’t fall off or get killed off by this new medicine, I have no idea why I could ever say no!    

This is one of the most exciting things I’ve heard in a long time. Next year, the hours of treatment, the fighting- it can all be over. I can live like a half normal person.

They’ve been telling me about this for years, and I started to not believe it was ever going to happen. And here it is. The one medication that could completely turn my life around, right on the brink of being given to me.

(I have two copies of the Delta F508 mutation so I’m in that bigger portion that will be effected)

This is me and my cousin Freddie - he has cystic fibrosis. For those of you who don’t have it, you may not think it’s serious and he looks normal, well he isn’t. If you don’t understand cystic fibrosis search it up, it’s the UK’s most deadly genetic disease.

The reason I’m posting this is because I’ve just seen this article (link below) about a 29 year old woman who has cystic fibrosis and is being denied a drug that could save her life as currently she is dying a slow and painful death. This hospital, this story has disgusted me and it made me realise what would I do if this was Freddie, he’s nearly two but this could happen at anytime! Kalydeco needs to be free and available on the nhs and it needs to be now. It’s like watching a person dying, it’s wrong.


Even though I don’t feel any different using it, the effect Kalydeco has on my sister (and everyone else who takes it) is astounding. If it’s hard to tell whether the person even has CF by looking at their various test results, then you have done something extraordinary.

Thanks a thousand times and congratulations to the scientists and researchers. You did good.

It was a very good year .... My name was Brian McGee, I stayed up listening to Queen

So, yesterday marked 12 months since I came home from hospital for the last time.

12 months, it’s incredible.

I could barely last 12 days before noticing the early stages of yet another chest infection, and you could bet your bottom dollar that at some stage around the 12 week mark I’d have either been in hospital already or would be screaming out for an admission, barely able to walk around the house without getting puffed out and/or needing a rest.

I really can’t believe how much one tablet, taken twice a day, has changed my life.

A year ago I needed a lung transplant. No questions asked.

I’ve thought mostly about the operation its self, the risk, then the recovery; the strict regiment of tablets, anti rejection drugs, etc. the rehab, getting your body working again. Then more risk: rejection, infection, etc.

But someone brought it to my attention last week – Before all of that could have happened, there was the waiting.

What if I didn’t get that chance? What if I decided to go down that path and I missed my chance, I’ve seen it happen before, it’s a seriously scary thought.

How lucky have I been?  It’s almost unbelievable.

I’ve said this before, but when you’re that sick, you actually don’t realise how sick you are. Your body learns to cope and function at a certain level that lets everything work. You subconsciously walk slower, avoid lifting heavy things, you sleep more and feel tired all the time no matter how much rest you have, but when things need to get done, you some how find the strength to do it.

It’s only when you’ve recovered a bit that you realise how bad things were. Then once you’ve recovered a bit more, you realise that even that initial recovery didn’t bring you back to an acceptable level.

I’ve had that realisation more and more over the last few months in particular.

How I feel now seems an absolute eternity away from how I felt 12 months ago, yet that’s all it’s been. 12 months. 365 days. And it’s all thanks to one small blue tablet!

Some people get a good 5 minutes of a great time from taking a small blue pill (if you know what I mean…!), I’ve had 12 months, a year and there are no signs of it slowing down.

I still get chest infections, but I think I could count on one hand how many I’ve had in the last year. And they’re no big deal anymore, all it takes is a bit of extra physio and some oral antis and I’m away. A week at the most and I’m back to normal.

No days in bed, no hospital, nothing serious. It’s almost normal!

My weight is now steady at around 10 kilos more than it has ever been steady at. I used to have to work seriously hard at getting to 60kg, using high fat supplements and eating to the point of feeling sick, now I haven’t dropped below 63kg in over 6 months. I’m not doing anything out of the ordinary, I skip meals and don’t eat well enough when I get busy, but the weight stays on. It’s something that wasn’t normal before taking Kalydeco.

I could drop a kilo a day if I wasn’t on top of my eating habits, pill taking, etc. before Kalydeco.

Again, that’s scary! No wonder I got so badly sick, so often.

It’s been a while since I posted here, but I find it hard to write about just being normal. I suppose it’s exciting for me, but for the sake of keeping this interesting (I hope), me writing about how little mucus I’m coughing up or how much weight I’ve put on will be kept to a minimum!

For anyone with any sort of power in the community, doctors, directors, politicians, you know who you all are; this next bit is for you.

This drug is a serious lifesaver. I’m proof of that. There are still people out there who can benefit from Kalydeco and other drugs like it that are being trialled for other CF mutations, you have the power to save people’s lives without the absolute last resort option of transplant. You have the power to prevent CF from ruining people’s lives. Please, use the power you have, use it wisely and use it soon. Like I said, there are people out there that need the second chance that Kalydeco has given me.

I want nothing more than for the people I know, and the people I don’t, that can have the opportunity that I’ve been given, to have it.

For anyone suffering at the moment, this next bit is for you.

Hang in there. I’ve been there. It’s tough, but you’ve got the strength to stick it out. The rewards are coming, new medications are just around the corner, all you need is for the people in power to do the right thing and you’ll have your second chance. Push them, ask the questions you need to ask, believe in them and yourselves and things will work out.

That’s some seriously deep stuff right there, but I thought I’d take the opportunity after reflecting on the last 12 months of my life, a life that if things didn’t fall my way could have been over.

It’s a sobering thought.

Now, wipe your tears and have a drink for Kalydeco this weekend and, more importantly, do me a favour and get behind the Aussies when the Ashes kicks off in just 7 days time! Now that Boof Lehmann is in charge, the sky is the limit!


Sign & Share this link please...

Just a very quick post asking for your help with a petition that is very close to our hearts.

A drug has been developed that will in effect almost ‘switch off’ CF in those with a particular gene in CF. (the G551D gene or 'Celtic’ Gene - due to its prevalence in Scotland). This medicine could prevent further irreversible organ damage being done.

Despite this medicine being available in the USA since January of this year and having been granted a European license in July, it is still not available for those in the UK & Ireland who desperately need it! Due to costs, administrative delays and the system we have here, the prescribing of these tablets may not be for some further time to come.

A Scottish Parliament petition has been created asking for immediate further funding to be put in place to allow the prescribing of this life saving medicine NOW!! Since it is 'immediate’ funding that I have asked for, it means that we only have a matter of weeks to get as many signatures as possible!!
Please share this with as many of your friends, family and work colleagues as you can. It can be shared and signed worldwide.

This is a very cruel situation for the very people that this drug has been developed to help.