Orkambi update - How I'm handling the side effects

I began taking Orkambi a week ago today. I made the decision to take the medication knowing that there was a chance that I could experience severe side effects that could leave my health in a worse condition. Throughout the past year my health has dropped severely and if it continues to deteriorate I won't have long left to live.

Now I read all the paper work on the medication that I could get my hands on, but seeing as it is a new drug the information is very limited. When I began taking Orkambi I really didn't know what to expect, and boy has it been a long week!
Let me premise this by saying before I started the meds I had been feeling a bit congested, and I was dealing with some sinus problems spurred on by the air conditioning.
The first day I took the medication I was so wound up and anxious about looking for the side effects and making sure I was breathing right that I actually ended up having an all day panic/anxiety attack. It wasn't very fun and I exhausted myself. I have been looking forward to this med since they announced it's pre-approval, I was terrified that the side effects would take it away from me. That being said my exhausting day of anxiety probably made me sick.
The second day of the medication I had a lot of shortness of breath and coughing. Now I don't know how much this was spurred by the medication, or if I was experiencing a normal exacerbation. I had the normal symptoms of a regular CF exacerbation so that I how I treated it. I waited another day and I e-mailed my doctor and he sent me some oral antibiotics. The first time I've been on Oral antibiotics this year, the doctor previously would just directly admit me.
It's been a few days and with the antibiotics and I have already noticed a lot of improves, though I am not back to my baseline yet I hope to be soon!
Some other side effects I've noticed are that I sweat a lot. I am usually an oddly cold person, I wear sweaters all year and keep a blanket with me because I am always cold. Since i've started this medication all I do is sweat! All day and all night I feel like I am dripping sweat and it's so gross and I don't really know how to handle it because I've just never been a sweater. It's made sleeping almost impossible because I sweat all night, which leads me to my next side effect.
I am tired all the time! I've had almost non-stop fatigue since i've started the drug. I am not sure how much of this is a direct side effect of the med or if it's a side effect of the other side effects. I haven't been sleeping well since I've had an increase cough, over heating and the sweating. I don't want to put the fatigue all on the med, but I do think it is contributing to my muscle exhaustion. I haven't been quite up to do the running I was before even with the exacerbation, but this is getting better everyday!

Most of the side effects I've noticed are slowly improving and getting more manageable (or i'm just learning how to handle them). I am hoping that it continues to improve and that I can stay out of the hospital for another month at least! Optimistically I would make it to my next scheduled appointment of October 1st! I'll update my journey with Orkambi as it progresses, but for now I am going to go back to updating about my regular life again!
I really hope this medication makes a long term impact on my health so I can make real plans for the future without having to worry about being around to see them through. I am still focusing on my short term dream of designing some cute Disney themed clothes! It's not as easy as I hoped but it's going to be worth it. I hope people will love my designs so I can see them worn across the Disney parks while I'm on my trips!

Wish me luck on my journey!

What am I doing with my life?

2015 has been a really harsh year for me. I've have been grateful to accomplish so much while dealing with being so sick, but for the first time this year I my health has stopped me from accomplishing one of my dreams. But with every bit of bad news comes something good! I have finally received and been taking the new medication Orkambi! I explain with a bit more in this video:

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!  

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!