Look who's getting Orkambi!

This is it, it’s finally happening. The doctor sent my prescription off for Orkambi yesterday afternoon! I don’t think I’ve ever been so excited for a medication or so anxious. I have been e-mailing my doctor non-stop since the medication was approved by the FDA on July 3, now a few weeks later I’m waiting for it to really arrive. I still have to wait for confirmation from the pharmacy, and there is no word on whether or not insurance will cover it yet, I am really hoping they will because filling out the assistance program paperwork will just delay the meds arrival. This medication will cost a whopping $259,000 a year, but for some people with Cystic Fibrosis it could mean life or death.

I fall into a danger zone for the medication. People with PFT’s below 40 have reported good results but also some danger side effects including shortness of breath and lowered lung function. If I get lucky and have no side effects in the first two weeks hopefully I’ll be good to go!  The medication is still brand new so it requires blood level checks for liver function every three months, which is a small price to pay compared to the reward.

So what does this medication do? Well to explain that I have to explain CF a little better. Cystic Fibrosis is often called a lung disease, but really it’s a genetic condition. This medication is really neat because it actually targets the broken genes and helps to correct them and function better. Orkambi consists of two separate medications that have their own function, without getting to specific one medication helps the gene’s work better and the other helps the body receive the fixed genes better. This may not be the perfect explanation but this is how I understand it. This medication isn’t going to fix the damage that’s already been done to my body over the years, meaning the scar tissue in my lungs and my lung function itself won’t improve as a direct result from the medication BUT with my genetics doing what they’re supposed to do it will greatly slow down the progress of the disease. It’s kind of like taking the disease progression and pushing the slow motion button. That will give me the opportunity to focus on healing the damage I have now which over time will hopefully increase my lung function and also extend my life.

Back in June my doctor was extremely concerned about my condition because it was deteriorating at such a rapid rate, but now with the prospects of this new medication I may have a shot of getting better for the first time in a long time.

That being said this medication only works on certain people with CF and it does not help everyone. I am lucky that I have the gene’s that this medication is effective in. Even with this medication it’s still really important that we keep fighting for a cure, we’re so close I really believe we can do it!  

musings before bed

There are a lot of times that I find myself questioning my decisions in life. I often wonder how the decisions i've made have effected people, but I don't mean the people around me. I try to consider the people who I had the chance to meet but never did, how many people I will still meet down the line anyway. Then I can't help but wonder how many people I run into that i've left an impression on that never say a word, have I ever?
Sometimes I spend to much time wondering who I would be if I didn't have Cystic Fibrosis. I only assume these kinds of thoughts are normal to have occasionally. Part of me is so grateful to CF, I see the world in such a beautiful way and I know it's only s a result of my CF. I just can't help but wonder what it's like to be healthy, to be kind of normal?  I've never really been a person to hold pent-up aggression about being sick or having this problem, I see my questions as being curious more than anger induced. I've always had way to strong of an imagination and far to often I let my dreams loose and they run wild crashing into reality.
Sometimes I think I dream to big, to big for anyone person. My dreams stem from this distorted idea that anything is possible if you work hard enough, and it's become apparent that some dreams aren't part of my lifes' bigger picture. Some dreams are meant to fill just a small time in our lives, and the pain that comes from their fulfillment can be just as bad as losing them all together.

A selfish wish that I could hold onto every dream forever, hold onto all those people I could meet forever. I just want to keep the dreams in my line of site ad never even blink because they could disappear. How is it possible that the correct decisions cause so much pain and anxiety? Nothing worth it is ever easy right? It wouldn't be right if I didn't consider every little variable.

And to all those people i'll never meet: I hope you have the best time.

The future is mine!

There has been an amazing development this month for the Cystic Fibrosis community. The FDA has approved a medication called Orkambi that will treat the underlying cause of Cystic Fibrosis in CFers with the double delta gene type (my gene type). This medication will act as a pause button of sorts helping to stop the progression of CF from getting worse!
I really can't express what a big deal this is for the community and for myself personally. In the past I'd never really considered planning my life more then 1 year our at a time, and with this new drug I could have a whole future out there!
I don't think people on average realize what a privilege it is to have a future. I've spent so much time from my life preparing for it to end and now I feel like this weight is being lifted and I can focus on a tomorrow that is less foggy and unsure. There is no day guaranteed for any person, but when you live with this cloud over you that any wrong move could put your tomorrow in jeopardy you really start to notice more when the sun comes out.

I was admitted into the hospital around the middle of May for the third time this year, the doctors were really concerned that my health had deteriorated so much in the 5 month span that 2015 had started they were looking into lung transplant evaluations and giving me around 2 years left to live if things continued progressing as they were. The hard part is hearing that and knowing that you are doing everything you can already to get healthy. I was doing my best to improve my health and the outcome was just the opposite, it was getting worse.  Finding the motivation to keep moving forward and to keep doing your best isn't easy and there are days where you refuse to get out of bed because it just feels useless but the important thing is pushing past all those negative thoughts and just doing it all anyway.
I worked really hard through the month of June and for the first time in two years I saw a positive increase on my health! My lung function improved 6% to 36%. My numbers have been that high since November of 2013! The number sounds so small, but it feels so great! I was really nervous when I went in to do the breathing test, but so excited when the technician told me the numbers that I insisted on doing more to aim for a high percentage and in the extra 3 tests I did I improved my percentage by 3!  That tiny bit of optimism I had made a huge difference in my tests so I can only imagine what the optimism I have for live can do with my time.

I was also incredibly received to a point of tears that I was showing signs of improvement in another area, my weight. I have been struggling with my weight since December after having influenza. I dropped over 40 pounds in 3 months which is incredible considering i was hardly at the proper weight to begin with. Talking about my struggle with weight gain is difficult because so many people ignore the fact that for me it is a challenge. I have a very strict diet that is difficult to complete, even though it may seem like the dream diet to some people it isn't as easy as it looks. Anyhow, I managed to gain 8 lbs in the few weeks I was out of the hospital finally pulling me back over the 100b mark. I would have been grateful for 5 lbs but a whopping 8! Again it sounds like such a small number, but so much work and effort went into the little 8 that is feels like a monumental victory!

I don't have another appointment until October and I am really scared that the start of Autumn and winter will throw all the progress I've made away. I plan on working hard and improving all of my numbers even more! I hope to get my lung function back into the 40% range and my weight at least over 120 lbs by my October appointment. I also plan on continuing or restarting my training for a half marathon! I completed my first half marathon November of 2014 and failed at the one I had registered for this past May but I am confident with these improvements I'll be able to finish another one in no time!

I am so excited for the future! After all we have to keep moving forward!