The Road To Kalydeco The Little Blue Pill
The Road To Kalydeco The Little Blue Pill
If you regularly read my blogs you may know that I suffer from the incurable illness Cystic Fibrosis, if you have just landed on this site, welcome, and where the hell have you been? As you may also notice from our website, my wife and I love to travel and I just happen to have an incurable disease, which to simply sum up is sh*t but I pass it off as an “inconvenience”. Nevertheless I’m not going to moan on about how difficult life is and how I am special. I’m just a regular bloke who hasn’t a clue what he wants to do in life, except for travel and take each day as it comes. As long as my wife and family find me special and I keep breathing, then life’s good. I won’t go into the ins and outs of Cystic Fibrosis, you can check our CF blogs for more, but thankfully I am doing ok and heading towards my mid thirties and a life of grey hair and moaning about how life was better in my day!
Any incurable illness is harrowing and a sad fact of life but I have been one of the lucky ones to have kept reasonably well and to avail of the closest thing to a cure for Cystic Fibrosis so far, the little blue pill as it’s known, Ivacaftor or brand name, Kalydeco. I thought after three and a half years of being on the drug that it was a good time to write a little about it. The pros and cons of the drug and also to show what a sick world we live in, as one hell of a fight was needed to ensure people like me benefited from this minor miracle. So please join me as we follow The Road To Kalydeco The Little Blue Pill.
The Road To Kalydeco The Little Blue Pill – The Journey to A Cure?
I am currently 34 years of age, have just returned from two amazing years travelling the world, living again for one year in Australia and am now back in Ireland and I still don’t have a clue what I am doing with my life! It is certainly not a bad thing however, the conventional life never really appealed to me, mortgage, kids, career. I don’t know if I was born with this attitude or if it is as a result of my late diagnosis at 17 years of age, a difficult year as it is in any boys life! At 17 I was one year away from leaving school, to be honest I wasn’t a fan of school.
Sitting in boring classes that would have no considerable influence on my future, all those hours of algebra, religious studies and science, boy I wish I could take them back! I can safely say that Pythagoras theorem really has got me where I am today! Going into my last year of school is when the bombshell was dropped on me that I had CF. It didn’t really affect me, aside from missing a couple of weeks of term but I didn’t shed any tears over that! To be fair my school was great, well wishes from my classmates, teachers and principal, which was nice, but to be honest many hadn’t a clue what CF was, I think they thought I was dying!
After I left school I still had no idea what I was doing, I never even bothered to fill out a college application form, as I don’t believe a person should be defined by education, and there is no point wasting time doing something you don’t enjoy. Yes I respect doctors, professors, academics etc, but some of the most messed up people I have met in life are these well-respected and revered academics, and lets not mention the many ineffectual politicians who really have much to answer for! After leaving school I had one goal, to travel, but that’s easier said than done. I done the standard two weeks holidays around the beach resorts of Europe seeing such glorious sights as Kelly’s, Murphy’s, Flanagan’s bars and eating the fine local cuisine from Spain to Greece of steak and chips. Not exactly cultured travel!
That’s life however for many young 20 somethings and I enjoyed every second regardless. Work Monday to Friday, party till Sunday with my biggest worry at the time running out of hair gel! Looking back I was extremely lucky. I never discussed my illness with people back then, not that I was ashamed I just didn’t feel the need to. If somebody asked I would tell them all about it but I didn’t want sympathy or special treatment. I continued to take oral medication each day and attend regular check ups in my hospital, that was it. For eleven years I did not have one admission to hospital, which is amazing for somebody with this condition and I can’t say it was from clean living. I ate quite poorly, drank quite frequently but exercised regularly, which looking back was a big help. When I met my now wife at 27 years of age life started to change.
Almost from day one I knew I had met someone special, someone I could spend the rest of my life with. I was up front almost immediately about my condition and she thankfully accepted me. That’s not to say us CF’ers are there to be pitied or that we are not worthy of a loving partner. On the contrary, but there is no denying that many “healthy” people do run at the sign of illness, but that’s human nature I guess. Cystic Fibrosis does come with complications, there is no denying that, so many just can’t or won’t face up to that, which is understandable.
The Road To Kalydeco The Little Blue Pill – Growing Older With Cystic Fibrosis
We move into my thirties, a landmark birthday, a birthday many medical books at the time of my diagnosis said that most Cystic Fibrosis sufferers would be dead by. I am fit, as healthy as I could be and marrying my now wife. I was doing what I usually did to stay well, which wasn’t really a hell of a lot to be honest. I still exercised frequently, started to eat reasonably well and took my medication. It was my wife who was doing all the fussing, looking up cures, recipes, anything to improve my quality of life. From my diagnosis at 17 there were always murmurs of a cure on the grapevine but being realistic I took it with a pinch of salt, pardon the pun.
A cure would be fantastic but I have always been cynical, even if a cure was discovered would the pharmaceutical companies dare give up the cash cow of a pill munching sufferer of a genetic illness? Hey that pharmaceutical CEO needs to drive his Bentley or Jaguar and those shareholders need their holiday homes in Barbados, don’t they? Cystic Fibrosis was really something I only had to face up to when I stayed in hospital or attended my check ups. The two minutes a day popping my pills being the other “inconvenience”. Maybe you mature or maybe you just get angry with the injustices around you but the plight of those with serious illness around the globe really gets to you and becomes more evident as you grow. I started to become more aware of my situation and the fact that many sufferers around me were having a more difficult time with the condition than me.
I really floated through my twenties more concerned about buying the car, travelling and clubbing with hospital visits an inconvenience. Little did I realise that in the next hospital room there were fellow sufferers being told they hadn’t long to live or they were anxiously awaiting new lungs. At this point in my early thirties I was asked to participate in some trials, this would involve a few trips to the hospital for various checks and blood tests etc and some survey filling. Maybe in the earlier days I would have declined, being too busy, but maybe I was put on this earth to help someone else? My few minutes being a guinea pig could make the world of difference to a kid born with the illness in the future.
As it turned out the wonder drug Ivacaftor was being developed and I just happened to possess the gene type that this drug would work with, Phe508del + Gly551Asp. These letters and numbers didn’t really mean much to me previously, all I knew was they caused my lungs to fill up with mucus and gave me the physique of a famine victim during my teenage years! Now I realised that this drug may actually halt many of the hallmark symptoms of CF. After many tests, cat scans, chest x-rays, blood tests and an uncomfortable eye-watering thin tube shoved up my nose, it was
now time for the day to receive my first Ivacaftor tablet. The road to obtaining the tablet was not easy for the 120 Irish people with CF who were deemed suitable for treatment. It is estimated that treatment of a single patient with the new ‘wonder drug’ costs €234,000. Now don’t get me wrong, I am grateful that the “government” covered the costs but frankly that’s what we elect them for, to protect their citizens. I am more grateful for the hard work done by sufferers and the families of the sufferers themselves who tirelessly worked hard for many years to find a cure. I am also thankful for the gifted and talented medical professionals who made this wonder drug possible, maybe that college education isn’t such a bad thing after all! I understand research and development costs a lot of money but I still think it is disgusting to charge nearly a quarter of a million euros per person for a life changing drug. It is because of these high costs that many governments around the globe reject the drug and now campaigners are fighting to bring about another life changing drug, Orkambi for the many other gene types.
It is clear evidence of greed that people have to fight to avail of a drug to keep them alive but there seems to be no problems for governments to give the go ahead to invest millions in military hardware and bank bailouts. All I can say is that the little blue pill has dramatically improved my lung health. I no longer wake in the middle of the night coughing like a lifelong smoker, I don’t wake up first thing in the morning with clogged lungs, my lung function improved immediately by 10%, no more agonising stomach cramps and my weight stabilised and even rose by 13 kilos during my time in Australia, this certainly would not have been possible pre-Ivacaftor.
The National Centre for Pharmacoeconomics in Ireland previously recommended against the drugs sanction for use at a cost of more than €234,000 per patient per year and the performance of the drug will be closely monitored to ensure that the results promised by the company are delivered on. All I can say is the little blue pill is a miracle, maybe it is too late for me to fully be 100% healthy, the damage has been done from birth, but the outlook for many future CF’ers is a bit brighter. If the young sufferers have access to this little blue pill then many complications of the disease can be avoided. For me there has only been two negatives of the treatment, first, the high costs involved has meant my chances of living full-time and continuing to receive the drug in Australia are slim, so for now I must stay in Ireland, and second the colour, being a Manchester United fan I would prefer any colour except light blue, the colours of city!!
Seriously though and to finish, If the National Centre for Pharmacoeconomics wants statistics and a balanced bottom line I cannot oblige, all I can say is my wife is happy I don’t wake her up in the morning with an earth-shaking cough and I’m sure there are many other fortunate users of the drug out there. My hope for the future is that more breakthroughs can be made and that people can be put before profit for a change, can you really put a price on life? Sadly some people can.
The Road To Kalydeco The Little Blue Pill – It’s been a long one.
The Positives of Having Cystic Fibrosis
Hey Bro,
Im a CF’er as well, and like you have found Kalydeco to be a real game (life) changer. Also like you I have a passion for traveling, and at 38 years old, I am working to find a way to move to Mexico, Dominican Republic, or somewhere similar to pursue a simple life without so much of the rat-race I’ve been stuck in thus-far.
But unlike you, as a US Citizen with a bloated, expensive, for-profit healthcare system, for the past 20+ years I chose “security” and health insurance over enjoyment of life. Ugh. Just the thought of sitting behind a desk in a maze of cubicles for another 30 years (and hoping my lungs last that long) just makes me want to puke.
The #1 thing preventing me from doing that now? The outrageous cost of Kalydeco. Here’s hoping something will happen and the cost of this drug will come down and I’ll have a chance to realize my dreams as well.
Cheers!
Dave
Hi Dave,
Thanks for the message, much appreciated. Difficult to know what to say, on one hand this drug is simply amazing but on the other the greed and profit made from illness is disgusting. CF is like a roll of the dice, you are born with it but the differing gene types ensure it affects people in differing ways. The same roll of the dice determines where we come from, Ireland has many problems but I certainly cannot complain about the healthcare system and the fact I receive my meds for free. I certainly sympathise with my American friends and others from around the world who either have to pay or cannot access treatment. The sad part of life is that people will prey on the sick and vulnerable and there is profits to be made.
I understand many cannot just simply quit their jobs and travel like I have done. I am fortunate to have done it but it does come with sacrifice. I, like you, worked in those office cubicles at a desk wondering “what am I doing here” so I changed the direction of my life. It is risky and I am sacrificing a pension but I’ve rolled that dice and decided to cash in my chips today. Even if I do make it to retirement age I’ll survive, I’ll worry about it then!
I hope you do make it to Mexico or the Dominican Republic or wherever you simply feel free. Hopefully the cost of this drug drops significantly, or better still a cure is found, but pardon the pun, I won’t hold my breath! Stay strong and I hope all your dreams come true or at least you wake up feeling well. When you do make it to that beach in the Caribbean send me some pictures, life is for living and having no regrets so if you do get a chance to pursue your dreams do it while you can.
Best of luck with your continued battle with this illness, hopefully we will be all celebrating its demise some day.
Best wishes
Continent Chasers
Greetings to all,
I am also a cystic fibrosis patient. I was searching for reviews on Kalydeco in order to know what to expect from this drug. I was diagnosed at age 63 years with Cystic Fibrosis. My entire life was a continuous fight with pneumonia, bronchitis and sinus infections. I wheezed, coughed and was getting to the point of struggling to breathe. The primary doctor gave me antibiotics every six weeks for over 30 years. He never suggested that I see a pulmonologist, just kept dosing me. As an adult, my daughter was tested for medical problems. She was diagnosed as a carrier when in her 30’s. She called me and said I had to have CF since I had all the symptoms and had passed CF to her. No one in my family had ever talked about having it. In the 50′, 60’s, 70’s and 80’s testing wasn’t done. My father lived to age 93 years and my mother lived to age 100 years, six months. I visited a pulmonologist that tested immediately when I explained why I was there. I was put in treatment immediately to fight the infections in my lungs and organs. I was diagnosed as a Cystic Fibrosis Related Diabetic and am on insulin now. My CF doctor has now prescribed Kalydeco for me. I have struggled with several medical issues and medications that have extreme side effects. I am very leery of what this drug could cause. I have been told the outrageous cost of this drug and being a US citizen, on Medicare with supplements that I must pay for myself, am not sure I can afford it. I’m happy to hear you both have done well using Kalydeco. Hopefully, it will be a benefit to me if I choose to take it.
Best wishes to you,
Judith
Hi Judith
Thank you for the comment. Wow your story is amazing and I cannot believe you were not diagnosed until such a late age! I’m sure you hear that all the time. I was diagnosed at 17 and I thought that was late. I can totally relate to your story about the doctor’s neglecting to investigate further. I was given inhalers, cough medicines and antibiotics until finally I found a wonderful GP. Again there was no history of CF in our family but my parents had siblings who died at young ages back in the 40s and 50s which, as you say, was a time when testing wasn’t done. I have been on Kalydeco for almost 5 years now and it has been very beneficial. I understand your concerns with the drug but personally I have not suffered any side effects, only good ones. My cough isn’t as bad or frequent as it used to be and I find that putting on and keeping weight on is easier than it used to be pre-Kalydeco. It is sad that you have to pay for the drug in the US. If I had to pay for it I just simply could not afford it, luckily the Irish Government funds the costs for patients in Ireland. Overall I hope you receive some help but you are obviously built of strong stuff if you reached your sixties without medication. The fact that your parents lived to such a good age shows you have strong genes I guess despite CF. I really think your story is very interesting and goes to show how many most likely remain undiagnosed around the world. We would love to hear more of your story and perhaps you would like to draft a version for us to share with our readers. A little about your life, how you were pre-diagnosis and how you are now coping? It is a fascinating story and also gives hope that we can live to beyond our live expectancy.
We wish you the best of health and hopefully you can find a solution to the problem with Kalydeco. Personally I would fight for it but if you feel you can cope without it then chat with your medical team about the best future plan.
Thanks again for contacting us and hopefully you enjoy our posts.
Hope to hear from you soon and if you need any further advice or have any queries please do not hesitate to contact us.
Kind Regards
Continent Chasers.