Symdeko

The last time I updated I was in the middle of a 2 week (turned into 3 week) course of Zosyn. Without too many boring details I went in after 10 days of meds and my lung function only climbed a bit. I went from 26% in clinic (dipped to 22% in the hospital, BUT I hadn't had my bronchodilator and I have horrible asthma and basically can't breathe without it so I totally don't think that one counts) to 28% in the ten days. A 2% increase is absolutely crushing because it isn't clinical significant. I felt totally defeated and super frustrated that the hospital and IVs were all a waste. We agreed to give it 14 days and I also added Bactrum since some of my bugs were sensitive to it. Around day 13 I actually started to feel better and called to extend my IVs to 21 days.

I just went into clinic today (28 days after starting IVs and a full week off of IVs) and my PFTs were 36% which is basically my baseline. Whew! I am so relieved to have improved so much. It just proves to me that my body really does need longer doses of IVs.

Getting back to baseline came at a perfect time because I am getting Symdeko tomorrow!! I wasn't overly optimistic I would have the opportunity to try Symdeko because it is more expensive that orkambi and I didn't have confidence my insurance would approve it. Somehow my insurance did approve it and I am starting soon! I am so curious to see if I feel any different between Orkambi and Symdeko.

Two Years and Counting

This month officially marks TWO years hospital free!! I can't remember the exact date I was admitted so I am sure I am a week or two early, but I am celebrating now anyway. It is also 1 1/4 years IV free!


It has also been a little over 2 years on Orkambi. Coincidence? I think not! I can't wait to see what new meds are around the corner and how much healthier all of us in the CF community can be!!

Restarting Orkambi

Due to issues with my pharmacy I did not receive my January shipment of Orkambi on time. Of course, I was upset that due to issues with my pharmacy and their lack of organization and communication was causing me to miss medicine that is beneficial to my body. But the reason I was most upset is that when I started Orkambi I had a lot of issues with side effects specifically chest tightness and shortness of breath. I was very concerned that restarting my Orkambi would bring back some or all of these side effects.

When I finally received my Orkambi I was so relieved that I wouldn't have to fight Accredo anymore. I momentarily forgot my concern with side effects. I was abruptly reminded of this concern a few hours after taking my morning dose. Although the side effects weren't nearly as intense as when I first started Orkambi I did notice a huge difference in my breathing pretty immediately. I often felt as if someone was sitting on my chest. I also had random sporadic bouts of extreme shortness of breath that seemed to come out of nowhere and disappear just as mysteriously. The biggest issue I had was I was too short of breath to exercise efficiently so I felt like my lungs weren't getting cleared properly by swimming which seems to be my best defense against mucus build up.

The side effects only lasted about fours days and yet after they subsided my lungs felt congested and heavy. I currently feel like I am on the brink of an infection. I will never know 100% if stopping and restarting Orkambi caused this infection, but I do think adjusting off and back on was really stressful to my body. I also think missing/more lax workouts didn't help the mucus build up nor did the tightness caused by restarting Orkambi. I seriously hope that I am never forced to go off of Orkambi again unless a new and improved drug comes out.

I Hate Accredo

I am not even sure how to explain how impossibly hard it was dealing with Accredo these past few weeks. I think instead of writing a post that could easily go on for hours I will give you the quick and dirty run down. I think it is important to note that a rep from Vertex as well as my clinic coordinator was also helping me deal with Accredo and I have no doubt it would have taken significant longer to get my meds if I was doing it alone. Which is so beyond ridiculous because it took over two weeks for me to get the medicine as it was.

• At one time my clinic nurse called and explained in great detail what she discussed with Accredo. I called Accredo and when I told the rep what my clinic nurse just explained the rep told me there is no record of such conversation in their computer system. Huh? What? How do you even respond to that?
• Every time I called the rep would flip flop what they were saying. Sometimes they would say it wasn't covered by my primary which meant my secondary wouldn't cover it either. Other times my primary would cover it, but my secondary didn't leaving me with a co-pay of $5,300.
• Two times my Orkambi was scheduled to ship out the next day only to be cancelled at the last minute due to "issues."
• Between Thursday and Friday I spent FOUR hours on the phone! About 90% of this time was spent on hold with Accredo.

We eventually found out a lot of these issues were because someone opened two accounts under my name and neither had both my insurance plans. So depending on which account the rep opened there was differing insurance information. Hence the reason some reps said that my nurse or Vertex never called in. They had the wrong account open. After this issue was resolved I assumed we would be good to go. Oh, but no that would be much to easy!

• Accredo kept insisting over and over again that my secondary insurance didn't cover Orkambi although it absolutely DOES cover Orkambi and I was told over and over again that the only way to get my medicine is to fork over $5,300. (The issue was that they were using the wrong authorization number)
• My case had been escalated a few times to people "higher up" in order to try to expedite (HA!) my shipment. However, there was complete lack of communication between the higher up "center of excellence" and the reps. The center of excellence (I use the term excellence very very loosely) made a plan and was told to me by my Vertex Rep. When I called Accrued explaining the plan I was told by both the rep and her supervisor that, "Nope, it doesn't work that way!" I ended up hanging up and calling back until I found a rep that would do what I said.
• I was told by a supervisor and someone in center of excellence that they would call me back within a specific time frame. Did it happen ever? Nope. And when I called them, the rep said the person I needed to speak to was nowhere to be found.
• After about two weeks I talked to a rep that said that maybe I should let me doctors office know I was having trouble getting my medication. Uhhh, if you look at my log they have been calling in for almost two weeks now...I think they know! This seemed to be another common theme... reps that didn't bother to read the notes in my file and wanted to d the bare minimum as far as work goes.

So we finally figured everything out and I was starting to believe I would finally receive my Orkambi. But wait, that would be too easy right?

• I was scheduled to get my Orkambi and although I had already ran out of my supply and it was coming super late I was beyond relieved to know it was on its way. Just when I thought I was safe my phone rang.... it was Accredo... nothing good comes from those calls...
• They were calling to say that their inventory was low and it would take 7 days for me to receive my shipment. SEVEN DAYS!!!
• At this point, I will not mention the things running through my head. However, the rep that delivered the news was one of the few reps that knew how to do her job. She had talked to me several times and I kept telling her if there is ANY inventory ANYWHERE they needed to send it to my pharmacy to get it to me. She told me to hold.... came up with a potential plan and told me she would call back.
• And guess what? Whoever is in charge of updating their inventory electronically didn't do their job (notice a theme??) and they actually did have some to send me. So I was going to miss another entire week of medicine because someone "forgot" to update their inventory list.

And then I got Orkambi and we celebrated. The whole family. Kaylee wouldn't be neglected anymore while I spent all day on hold. My husband didn't have to put up with my mental breakdowns anymore. And best of all I had my medicine. Except, restarting also meant my body had to readjust to Orkambi which my body doesn't do well...

To be continued...

Proof Orkambi Works

Several weeks ago I wrote my one year review on Orkambi. I said that I felt it was helping, but did not have the clinical proof to back up the way I felt. My PFTs are unchanged and even though I had a health streak that was unheard of for me following Orkambi, I fell into a typical health slump around the nine month mark. Although Orkambi didn't feel life altering, I also did not feel any worse so I continued to take Orkambi with hope of slowing the decline of the progressive nature of Cystic Fibrosis. Well, I now officially have proof that Orkambi is working for me!

I want to preface this post by saying CF has taught me to be cynical and I am not a blind believer in anything. So many times I have believed that certain procedures or medicine would benefit me only to be left disappointed. Antibiotics that my cultures are sensitive to (meaning they should knock down an infection) have failed me, I have left hospital stays with numbers just as horrible as I entered with, I have gotten worse with no rhyme or reason and I don't fully trust anything medically based until I try it and see it working for me. The medical field isn't fool proof and CF doesn't seem to follow any rules.

To explain my proof I need to back up a few years (this will be short, I promise). In 2014 I was going through an exhausting and frustrating health slump. My doctors, perplexed by my inability to get better despite everyone's best efforts, ordered a CT scan. The CT scan didn't show anything overly telling despite mucus plugging, scar tissue, bronchiectasis, and other typical CF problems in someone with more advanced lung disease. I continued the roller coaster of a CF slump for a few months and eventually stabilized.

Fast forward to summer 2016 (now post Orkambi) and I was experiencing another CF slump. After IVs and months of oral antibiotics we weren't getting anywhere. My doctors were once again stumped, this seems to be a pattern with me, we decided a CT may be needed to see exactly what was going on. I didn't anticipate it to show much and didn't even bother to call for results knowing if something horrible showed up (say a collapsed lung or pneumonia) my doctors would notify me. Honestly, I didn't want to hear about how much more my lungs progressed because there is nothing more depressing than proof your lungs are dying.

When I did get the results they shocked me! Remember, this is a CT scan taken while doctors couldn't get my lungs to stabilize so clearly, I was not in great health. This CT was a comparison to a 2014 CT scan and CF is progressive in nature meaning things usually just get worse and worse. My thirties have been extraordinarily hard on me and good news in regards to my CF just doesn't happen anymore.

When my results were finally sent to me I skimmed through the write up, holding my breath and feeling that uneasiness that comes with impending disappointment. And yet words like stable, significant improvement, markedly improved jumped off that page at me. Wait, what?! There were improvements in my CT over that past two years? And these improvements are showing up despite how sick I was when the CT was taken? My progression of bronchiectasis has stabilized (it is impossible for bronchiectasis to improve so stability is the best case scenario). Nodules in my lungs were shrinking? There was significant interval improvements in my right lung (which has always been my problem lung)?

I had to read through everything several times before it sunk in. My CT scan did not get worse over the past two years and in fact it actually got better! Although I have not seen the miracle results many people have seen on Orkambi including increased lung function, increased energy, decreased exacerbations, I have seen an improvement in my CT scan. When your disease is progressing at a dizzying rate seeing any improvement can feel nothing short of miraculous.

Yes, I wish I had seen an improvement in my lung function especially since I sit so very close to transplant now. Yes, I wish I had increased energy to make each day count just a little bit more. Absolutely, I wish these infuriating slumps in my health that seem to be nonresponsive to antibiotics would disappear forever. However, at this stage in the game I will take any improvement I can get and even if those improvements are only noticeable through an improved CT scan then I can't complain. Without Orkambi I am positive that CT scan would have continued to look worse. Honestly, I don't even want to think about what my life would look like today without Orkambi.

One Year of Orkambi

This post was incredibly hard for me to write. Like all matters related to CF there was no black and white with Orkambi, for me at least. I wrote extensively on what the first four months were like, which were a rollercoaster to say the least. My purge was intense and utterly miserable if I am perfectly honest. Walking often induced shortness of breath that was so intense I wondered if I would ever walk again at times. In fact, sitting often brought on shortness of breath that left me gasping for air. At the four month mark my negative side effects disappeared and I continued on to have one of the healthiest winters in my recent history which I wrote about at the 24 week mark.

Was orkambi responsible for my winter health streak? I would like to say yes. If you asked me mid winter without a doubt I would have said, "Yes!!" Last winter, my first on Orkambi, was by far my healthiest winter ever. However, somewhere around June sickness struck again and I had three weeks of IVs, followed by weeks and weeks of oral and only now have I seemed to stabilize at a number that is still below my pre-Orkambi baseline. Would this last exacerbation be worse if I wasn't on Orkambi? Possibly. Could Orkambi be causing some inflammation that is keeping me from hitting my pre-Orkambi baseline? Potentially. So many factors come into play with CF that it can be hard to know exactly what is causing problems and what is helping. The progressive nature of the disease can complicate this issue even more. However, outside of several rough months this summer, I do feel that Orkambi has improved the consistency of my mucus and has made my lungs cleaner and clearer. I just can't quantify the way I feel and I have no real clinical data to back this feeling up.

Even though my lung function didn't improve like I had hoped and wished with all my might, my GI system has absolutely improved and that is unwavering to this day. Despite being sick for a couple months without reprieve my weight has remained stable. I no longer have days that I am so bloated my stomach feels stretched and painful. As long as I take my enzymes I really do not notice or think about my GI system at all. Of course, even Orkambi couldn't get rid of my CF belly, but as they say beggars can't be choosers and so I will take what I can get!

24 Weeks of Orkambi

Two years ago I tried to get in the Orkambi trials, but after going through all the pre-trial testing I was denied. When I finally received the medication Sept 2015 I was curious as to how my life may have differed today if I had been the trial two years ago (and had the same dose medication that was approved by FDA which would not have necessarily been the case). Would I have been healthier with better lung function? Would my two years of decline and instability have been less "eventful"? There is no way of knowing how things would have been different had I started the trial 2 years ago, but I was still curious as to what my experience on the trial would have been like. So after starting my Orkambi I marked my calendar for 24 weeks (the duration of the Vertex trials) and waited.

I blogged about the first couple of months here if you want more detail as to the rough start I had with Orkambi. The biggest changes from the last 24 weeks of Orkambi:


1. Plugs!
I coughed out my first plug about 2 months into Orkambi and I had no idea at the time, but they would continue to come out at an alarming rate peaking at 4 months. There were days I would cough out up to four or five plugs in a single day. Around 4 months I started to lose sinus plugs at an equally alarming rate. There were weeks that I got either a lung or sinus plug every.single.day and often multiple times a day! 
**For reference, prior to Orkambi the only time I would cough out a plug was during a round of IVs and these plugs were usually "newer" plugs that were more gelatinous and lighter in color. Orkambi plugs were dark and were so foul I often gagged as they came out sometimes verging on throwing up. They were clearly bacteria ridden and extraordinarily old. Good riddance!**

2. Sickness
I have had a rough couple of years. There was an entire year that every single time I started to feel better I would instantly catch a cold and be incredibly sick again. It seemed that once I felt well for a few days I would wake up the next day feeling a little off only to be sick with high fevers or completely bed ridden by afternoon. Sickness came quickly and often. I hardly left the house and bathed in hand sanitizer and was still constantly sick. I also ended up with reoccurring infections because my lungs couldn't handle the stress and mucus overload.

Since starting Orkambi I have had the first winter in a very long time that I was NOT SICK AT ALL! (Yes, I shouted that). What is even more amazing is that Kaylee and I have not restricted ourselves due to risk of getting sick like we have in the past. We continued storytime at the library, had regular playdates, and generally acted like normal human beings. And somehow I did not get sick! The most insane part is that I never even touched a bottle of hand sanitizer this winter. I ran out and never replaced it. Clearly, my body working more properly has allowed my immune system to function again (years ago I had a pretty amazing immune system that was so taxed the past few years it seemed to completely stop working).

There were 3 times over winter that I thought I was getting sick (the achy tired feeling is always a red flag that sickness is approaching). All three times I mentally prepared for a hospital stay because the last few years sickness always ended with IVs because my lungs just couldn't handle the overload. But unlike pre-Orkambi somehow I got over whatever was making me feel off. As in, I never actually got sick. This was such a sharp contrast from the past few years that the first sign of ache always left me feverishly shaking in bed within 24 hours.

3. FEV
So unfortunately, I have not seen an increase in FEV1. In fact, my last appointment I was down a little. However we are in the throws of allergy season at the moment which is my hardest season. My allergies present themselves as asthma and my lungs often feel like they completely shut down during allergy season. At this point I do not believe I will be one of those people with CF that see a positive change on FEV1. I won't lie I would absolutely love extra lung function and really wish I would have seen some improvement in that regard.

I am a skeptic when it comes to... well most things. I did not assume Orkambi would be a miracle and I did not assume every positive (or negative) change in my body would be from Orkambi. Our bodies are complicated and CF can be so unpredictable that it makes distinguishing correlation vs causation hard to separate. However, the hundreds of plugs I have coughed out in the past 6 months and the fact that my completely unstable health (especially in winter that usually left me a hermit for months on end) seemed to stabilize (for now) is enough to make this skeptical person confident that Orkambi has greatly improved my quality of life and I am so thankful it came into my life when it did.

Four Months Of Orkambi

*This is a long version of my experience on Orkambi*

This is a post I keep starting, but can't seem to get myself to post. To be honest, my Orkambi journey started torturous at best. As much as I wanted to gripe and complain about the sickness that came with this new pill I knew I was unbelievably lucky to have the ability to even try this medicine. I know how many people with CF would love the opportunity to try this medication that was already in my hands and that none of them wanted to hear me complain. At the same time, when I felt as sick as I did, it was hard to feel grateful and lucky for a medicine that I wasn't sure could ever help me. And so I kept quiet and internally kept note of the changes, both good and bad, going on in my body. I am just finishing four months of Orkambi now and feel I have a clearer picture of what Orkambi means for me and my disease.

I started Orkambi feeling cautiously optimistic and well aware that the first month or so would be tough. However, I had no idea what the next several months had in store for me. My symptoms were not that unusual and many people that start the medicine experienced the same side effects. Some of the symptoms that came with this little pink pill were shortness of breath that was so extreme I had trouble walking across the room. In fact, there were times I felt like I was suffocating while sitting still. I had fevers, chills, aches, streaking of blood in my mucus, a cough that never stopped, but didn't feel productive, which meant I hardly slept. I was mentally prepared for these side effects, but I wasn't prepared for how long they would last. I was warned by fellow Orkambi users that the first week is the hardest and the worst of it was usually gone by a month. My symptoms seemed to get worse as time went on and as I passed the first and second month with no relief I started to lose hope that this medicine could work for me.

The physical aspect was tough, but the mentl aspect felt torturous. I dropped from 40% lung function (fev1) to 29% and despite the passing weeks, my numbers wouldn't budge. I worried endlessly as the weeks passed that I was harming myself rather than helping. I was so tight, coughing was not super productive and my exercise consisted of walking in slow motion around the house. I worried that my lack of movement and my tight chest meant that more mucus was pooling in my lungs and that I would end this Orkambi journey worse off than when I began. I felt like I was sicker than I had ever been in my life and I knew I was doing it to myself by taking this medicine. I kept waiting for a sign that things may improve, that my body was still okay, but nothing seemed any better.

And then one evening 2.5 months of worsening symptoms I broke down and cried. I had decided in the morning I would call the doctor and tell him that I just couldn't do it any longer. Physically, I was tired and mentally, I was terrified I was hurting myself and I don't have a lot of lung function to spare (at 29%). Orkambi didn't seem to be the right medication for me.

That night I went to bed and slept through the night for the first time in months. I didn't cough once! The next morning my shortness of breath had miraculously disappeared! It was a complete transformation. It was all I needed to stay on course and continue using Orkambi. My next clinic visit my lung function was 35% and although that was better than the previous 29%, it still wasn't to baseline. Regardless, I felt a huge sense of relief that at least my lungs were moving in the right direction.

During the fourth month I suddenly starting seeing all the changes in my body I had secretly hoped would happen. I started coughing up old putrid plugs from my lungs. Sinus plugs were coming out at an alarming (yet wonderful) rate. I could climb stairs and even run in the yard with my daughter. My mucus lightened in color and was much less copious compared to years past. My mucus came up easily and I started to feel stronger and more sure of my body. my morning cough has completely disappeared. I am starting to see my weight steadily climb despite starting at a healthy weight to begin with.

Clinically, I am not sure my lungs seem much different, but I feel like I just came off a round of IVs. I am in extreme awe of how well I feel without needing a hospital stay and weeks of IVs. I feel amazing and I have done nothing to work for it, no oral meds, no hospital stay, no prednisone, this hardly feels like real life. Orkambi may be considered a marginally beneficial medication and yet I feel stronger, healthier, and my lungs feel clearer. I may not gain lung function or see a huge change clinically, but I am taking something that is making my body work more like it should and that is amazing!

Rain Showers and Orkambi

"Maybe we will see a rainbow" my 3 year old shouted with eyes wide at the thought.
My dad and I exchanged glances. While smirking I replied, "Maybe" not believing it one bit.
It hardly rains in the summer. We haven't seen rain in four months or so, but a recent drizzly day made its way into the end of summer leaving my daughter with the optimistic belief that she would see a rainbow. It was true the rain had stopped, but the sun was still hiding behind dense clouds and evening was quickly approaching. My daughter had a date with my father that evening and I was hoping the excitement from the date would be enough to make her forget the disappointment of a rainbow that would never be.

I started Orkambi today. The day I found out that Orkambi was approved, my application was accepted, and the pharmacy was preparing to ship my pills to me I was filled with a multitude of conflicting emotions. I am an optimist by nature, but a realist by life experience. My heart kept shouting, "This is a dream come true! Your body will be functioning more normally than it ever has before. This is what you have been waiting for" But my brain kept shouting just as loudly, "But the benefits are minimal and your lung function is already so low." My heart would rebuttal, "Improvement is not the point, stability is what matters. " The fight between my jubilant heart and my practical cautious mind roared on throughout the week

This morning I woke excited and anxious for the postal carrier. And once the meds were in my hand around 9am I suddenly felt nervous in a way I haven't felt in so long. This was the moment of truth. All the potential benefits and all the potential let downs were running through my mind. I wanted so badly to let my optimism take over my cautious mind, but I was equally concerned about being disappointed. I sat there for a long time before swallowing my first pill. It is okay to hope for the best, I kept telling myself. It is okay to be optimistic for once and believe that good things, no matter how small, are coming your way.

I could hear the giggles and high pitched voice of my little girl running up the front steps. I greeted both her and my dad at the door with a big smile. Before I could even ask how the date night went my daughter, hardly able to contain her excitement shouted, "We saw a rainbow, mommy! We really did!"

Orkambi Troubles

I am not sure how one quantifies the value of a life. Apparently, some people know the secret equation, but I wasn't lucky enough to be one of them. Although, I do not know exactly what my life's value is I do know according to my insurance company my life is not worth $259,000/year. In other words, my insurance is not covering the new drug Orkambi for any of their Cystic Fibrosis sufferers.

I was actually really surprised, naïve as it was, because my insurance gladly covers the $300,000/year Kalydeco for patients with the correct mutation. I thought that if they would pay a higher dollar amount for one groups of CFers they would pay for a less expensive version for another. We all know life isn't fair and insurance companies rarely make sense so I guess thinking my insurance would cover this medication was faulty logic.

The really horrendous part of it is I am not sure I will be eligible for the Vertex patient assistance program for people who have insurance refusing to cover the cost of the drug. This could mean that unless I magically become extraordinary wealthy, an extra $20,000 a month wealthy, I may never have access to this medication.

The only aspect of this whole situation that is giving me hope is that CF doctors in my state are coming together to fight for their patients so that we can all access this medication. The CF community is full of doctors who genuinely care for their patients and for that we are lucky. The only other fiber of hope I have is that potentially my insurance company is negotiating price and may eventually strike a deal with Vertex. The part that is the most frustrating is that I am being left in the dark. The morning after I found out my insurance is refusing to cover Orkambi through another cyster I called my insurance company, my clinic (the CF nurse is on vacation), my pharmacy and vertex. I got zero response and no answers. Now that it is the weekend I have to wait until Monday to hope that someone will answer their phone to provide me with some answers on where we go from here.

Cystic fibrosis patients wake up every morning from the moment they are born until they die ready to fight. It is the only way to ensure we wake up every single day. I am ready to fight my insurance in any way possible, my life is on the line!

Anticipation

There is a flurry of emotions whirling through my mind. I feel as if I am teeter tottering between every emotion that a human can possible feel throughout each and every day. Why all this emotional instability? The newest Vertex drug, Orkambi, is the culprit of all this emotional confusion.

The two years of utter free falling into a dark abyss from this disease made me wish with every fiber of my being for Orkambi to be approved. Every wish my heart held so dearly revolved around something, anything that could pull me up, even just a little, from this bottomless well. Now that Orakmbi is here I feel such a mix of emotions I feel I may go crazy with uncertainty.

The optimistic side of me is feeling excitement so strongly that I can feel it buzzing in my bones. The optimistic side of me wants to scream and shout and jump up and down that there is something, anything that may help me maintain the last 5 months of stability for the next several years. The optimistic side of me want to believe that this drug will work well for me and I will gain 4% lung function or more! I want to have more energy to chase and swim and bike with my daughter. I want to gain weight easily, and have fewer aches, and have more stability in my day to day life when it comes to health.

And then a voice, just as loud and adamant as my optimistic voice, comes bursting into my mind. The voice is my realistic (pessimistic?) side and it is reminding me that not ALL people experienced increased lung function and there were a few that experienced side effects that required them to stop the medication (mainly elevated liver levels). Some felt nothing and others had symptoms that were troublesome and even a bit scary like extreme shortness of breath.

To add icing to my troublesome cake I feel paralyzed with fear that my insurance, which has been reviewing my case for over a week now, will deny my ability to even try this medication.

So much of life with Cystic Fibrosis is luck. What mutations you have, how the disease presents itself, what bugs you catch and how much havoc they wreak on your body, how well you respond to meds and if you are allergic to them. Here is just another example of luck. I have heard this drug has been a life saving miracle for some people and for others it is a nuisance not worth their time. How I feel about this medication is anyone's guess and sheer luck just as most things with CF. And of course, luck will determine if my insurance will even cover this $259,000 a year medication.

I just hope that this time, lady luck is on my side!

Orkambi

As most of the CF world is probably aware of by now, the vertex drug developed for DDF508 has a good chance of becoming available to patients this July. Everyone in the CF community was hopeful this drug targeting DDF508 would be a life changing miracle drug just like Kalydeco was for people with the G551D mutation. Unfortunately, the results of the study quickly dashed many of our dreams that this drug would be our miracle drug.

As much as I am disappointed that Orkambi, which will work on my mutation, is not the life changing and life extending drug that Kalydeco is for a select few with CF. I can say I am beyond excited, despite having all the facts, that we may have another medication that could potentially help slow the progression of my disease. When you live with a progressive disease new treatments are never developed fast enough. In fact, discoveries to treat CF are far and few between. And even when therapies are developed I have found myself allergic and unable to take advantage of many of these medications and treatments. The progression of the disease is always three steps ahead of the treatment and watching your lungs and body deteriorate is heartbreaking at best. It development of treatments are very much a matter life or death for so many with CF. This is why I have to roll my eyes at the naysayers that complain about the humble 3% increase in lung function that were seen in the Orkambi study.

What the naysayers don't realize is that 3% may not seem clinically significant, the truth is that the drug is so much more than 3% increase (on average) in lung function. It is the fact that is can slow the guaranteed progression of this disease. That is something that can not be taken lightly. To me, it  means I may have a few more years added to the end of my life to watch my daughter turn into a woman. It may mean one or two more anniversaries I spend with my husband. It may mean another birthday and a few more grey hairs. So yes, 3% may not seem like a big deal to someone who hasn't had an expiration date stamped to their lungs. It may not seem like 3% could change a life, but pessimists are stuck on a low percentage and not the potential for what slowing, even minutely, can do to a life. How a few years can mean so much to a mother, a wife, a sister, a friend who would do anything to hold on to their life for just a little longer.

So yes, lets celebrate the accomplishment of the researchers, the foundation, the  CF community that we may (as long as the FDA gives the okay this July) have one more tool in our fight again CF, we may have a little more hope for tomorrow, and we may have a little more time on this Earth.