kalydeco

Hey guys! I know…this might seem like a random picture but this is actually one of my awesome friends, Spencer. He has a condition called Cystic Fibrosis which is a recessive genetic disorder that affects the lungs, liver, pancreas and intestines. He’s one of the less than 100 people in Canada that can benefit from a drug called Kalydeco which can treat the genetic mutation itself as opposed to just the symptoms.

Unfortunately, this drug has a $300 000 price tag attached to it. That works out to about $400 a pill. His lung function has been dropping dramatically over the last year and he desperately needs this drug if he is going to make it. He was recently hospitalized for a ruptured blood vessel in his lunch which ended up being a 20 day stint in 2 different hospitals, one locally in our smaller one and then he had to fly out to a bigger city for my intensive care. He’s even had to withdraw from university to focus on his health.

His amazing boyfriend Chris has started a campaign to try and get as much funding as possible for Spencer. Even if you are unable to donate, he has also started a petition to try and get the government to assist in funding this. I’m asking from the bottom of my heart that even if it’s a couple dollars, it is appreciated so much. We all love Spencer and he’s such an amazing person.

DONATE HERE

SIGN PETITION HERE

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!!  

youtube

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 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)

WELL IN SON, I’M ECSTATIC.

youtube

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

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.

http://www.birminghammail.net/news/local-news/birmingham-selfridges-worker-denied-cystic-271045

Me

I’m Paul. I’ve had CF since I was 3months old and i’m now 20. It’s been a pretty rough journey and I feel like its getting rougher. The good news is im coughing the mucus out in big chunks but i’m always depressed. I can’t find anyone who understands me, I cant talk to my family about my depression only my psychologist. Not a lot of people know what CF is and they think im contagious when I cough. It sucks to think that everyone has to stay away from you or you got to stay away from them to not get sick. I cant go over 150lbs without feeling sick, I got diabetes from CF in 2006, but I try my best to stay positive and enjoy the things I love. I am always exercising in some way, I just can’t wait to try Kalydeco.

(For the record, Paul. You’ve had CF since birth, that’s how genetic conditions like Cystic Fibrosis work. You may have been diagnosed then, but it was still there)

FDA Approves First Ever Pill Targeting Cystic Fibrosis

image

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.


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!

Tootle-oo!

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.

cure timeline

I don’t believe there will be a cure within the next few GENERATIONS, but I do think more effective drugs such as Kalydeco will be available within the next 30-40 years. It doesn’t upset me either that I’ll likely never have access to these medications. These things take a LOT of time/money.

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.

I'm bigger than you, higher up the food chain, get in my belly!

OK, I’m back!

I’ll apologise again for my lack of blogging over the last month, it’s been hard to find the time to sit down and get everything that’s going on down on paper or on the screen, as the case may be.

It’s hard to write about the attention and questions asked about the blog without pumping up your own tyres too much, but that’s been the case more and more as the time has passed since my last entry. So, thanks to everyone for boosting my motivation and getting my a*** in to gear. And what better timing to sit inside and type one out than a beautiful, sunny, 26 degree Melbourne day! I’d hash-tag sarcasm, but they tell me hash-tags don’t work outside of Twitter or Instagram.

So my last blog was basically a recap of the first month of being on Kalydeco and a trip to Bali that was way too short and way too long ago.

Since then, everything has sort of been cruising along nicely. I got over the trip and the flights there and back with no problems, I haven’t really been sick or well, but I’ve been at a happy medium where things are good. I feel great, but my numbers on paper, where my lungs are concerned, have been either stable or have dropped a touch due to a slight cold/flu about 3 weeks.

I had out patient’s clinic last week where my lung function fell below 30%, which was very disappointing and added to my lack of inspiration to write another update, and was strange given how well everything else has been tracking.

My exercise tolerance is through the roof compared to BK (before Kalydeco), I can run around and kick a footy, I’m getting through cricket training and games with no real issues and being able to day to day stuff is as close to normal as I can be. Like I said, I feel great.

Where the exercise is concerned, it’s almost like my body isn’t creating as much lactic acid – My legs don’t get sore or heavy as much as they used, it’s like I have more oxygen getting around my body, helping everything work as it should. An example of how far I’ve come was at training last week – We set out penalties for bowling poorly, 5 or 10 push-ups per bad ball. True to form, I bowled poorly and went home feeling fresh after doing somewhere between 50-70 push-ups over the course of the night. It’s not a huge number, but when you’ve got spaghetti arms like mine, and could barely blow a candle out after walking a lap of an oval 12 months ago, it’s a big deal.

I’ve put weight on too, I’m now a hefty 61kgs, the heaviest I have ever been and people are commenting on how well I look. That’s something that hasn’t happened for quite a few years, even when I was well previously, I was still under weight and have looked crook in one way or another for years. It’s great to get that sort of feedback from people. It’s refreshing to have shit hung on you because your chest and arms are growing, it’s just never happened before.

I get a real idea of how far I’ve come when I think back to last Christmas – I was 51kgs in January. That’s a BMI of 17.2 – The lowest of the normal range is 18. I was able to put on half the weight by myself, but I was stretching it every day. I had to take a weight gain supplement with every meal and just hated the thought of eating. It’s completely different now, no supplements, minimal junk, I won’t go as far as saying I’m on a lean and mean diet, but it’s nothing like it used to be.

My weight etc. is good on paper too – My BMI is up to 21, which is smack-bang in the middle of that normal range for my age and height. The last time I was anywhere north of the lower side of normal was back in the late 90’s as a kid. That was when my lung function was hovering around 70-80%. 

I feel like my body is stronger now too – It got to a point BK where I felt really weak all the time, my muscles would get fatigued seriously quickly and I’d start to shake like I’d done 100 push ups, just from bringing the shopping in from the car. That’s stopped. I feel stronger, like everything has been rebuilding its self, back to what it was before I got really sick.

All of that has come on the back of very little junk food too – I won’t lie, when I was sick with the flu, my cravings went up/motivation for cooking food went down and I was eating McDonald’s for breakfast every morning, and would push through a Popcorn Chicken here and there if I was desperate for some food. But I didn’t enjoy it; it actually made me feel sick. The only times I’ve really enjoyed take away like I used to was last weekend and the weekend before on the back of two big Saturday nights. It’s strange; I used to live on the stuff!

In the last month I’ve been trying a few different things, tinkering with the way I take Kalydeco, the foods I take it with, etc. all due to the fact I haven’t had the big improvement in lung function. Given that everything else was going OK, I thought it was time to try a few things.

I’ve worked out that I need a serious intake of fat more so than a high calorie meal, and as a result, need to take more Creon to get the full effect. Four Creon before I start, then eating half and taking another Creon with Kalydeco, then finishing off is the routine that seems to work best. Toasted ham and cheese sandwiches, hot dogs and banana flavoured Big M’s have been on high rotation.

Since I started doing this, there’s been a steady improvement, I’ve noticed my cough is more productive, but what’s coming up is lighter and thinner than anything I’ve coughed up in living memory. I have a before and after photo of some sputum, but I’ll save your eyes and stomach the trouble, you can just take my word for it.

My FEV1 is the highest it’s been a long time too – Before I started Kalydeco I posted it was 21%. Two days ago it had cracked 35% and I can still feel improvement coming.

I can’t tell you how exciting that is. It’s still low, but when you’re dealing with sub 20 numbers (the worst one I had done earlier this year was 18%), anything above 30 is amazing.

I’ve got a follow up sweat test booked in for next month sometime which is exciting, it’ll let me know exactly how well Kalydeco is working, looking forward to that. I’ll also be taking part in a 1-hour trial for a new inhaled drug that could help even further, but I’ll save that one for next time. Hopefully there’s something promising and/or entertaining to talk about there!

I think the biggest and most important thing to come out of all of this so far is the confidence I have back that going to hospital and being treated for chest infections isn’t what will keep me alive and, potentially, will let me keep my own lungs for who knows how long.

Hospital has been such a big part of my life forever and in particular over the last 12 months, but I’ve been out now for 18 weeks and don’t even look like going back in. “I ain’t goin’ back, I ain’t goin’ back!” for anyone that has seen Bad Boys 2!

Anyway, that’s about it for now – It feels good to write all this down, so thanks again to everyone that’s been on my back about it.

Give me a week or so and you can feel free to start again!

Good luck to anyone having a punt this weekend too, hope you can back a winner in the Cup.

Peace!