Not My Miracle

“A vacation!” I squealed.

“To where?” my husband asked.

“Anywhere! After Orkambi, I’ll probably be able to go anywhere. I read that someone traveled to Europe after years of being too sick to do much of anything…

…Ohh!! We can also go see Billy Joel at Fenway next summer! Remember how I was in the hospital when we had 7th row tickets 2 years ago and we had to sell them?

Oooohh…We can also visit your parents next Christmas!”

On a random day this past November, we were hanging out in the living room, watching a movie. My husband was sitting on the love seat, and I was laying on the couch, clutching the area of my chest where my lungs felt so tight.

I was taking a new medication, developed specifically for people with my gene mutation of Cystic Fibrosis. The medication with the funny name – Orkambi – held so much promise, but was known to cause chest tightness and shortness of breath in the initial stages of treatment.

My husband and I were okay with that – what’s a week or two, or maybe a couple of months, of discomfort, if it meant it would change my – our – entire lives?

At 44 years old, I’ve had many ups and downs due to CF. As I’ve aged, I’ve had more downs than ups, and lately, it’s felt like I was just traveling downward into a mineshaft.

The promise of a second chance was almost too much to handle. We tried to temper our enthusiasm, but it seemed like everyone was excited.

My entire medical team was excited.

Patients all over the world were excited. Some of them, I know well and consider friends. I knew of more than a few who had participated in the clinical trials. Hope was contagious.

On the backs of these ground-breaking tests, the FDA fast tracked this novel medication to market because it had so much medical potential for patients with Cystic Fibrosis.

Orkambi’s retail price tag of $259,000 a year was a shock, but we reasoned, what do 2-3 hospitalizations cost? What do my everyday medications cost each year? The answer is- a LOT more than a quarter of a million dollars. We’d be saving our insurance company money!

The First Few Weeks

I finally had a lull between infections when my doctors felt it was safe to start taking Orkambi.

My doctors warned me not to freak out over possible side effects that might come up. Online, where I communicate with hundreds (and sometimes thousands) of other patients, I read stories of rough beginnings as well.

Many of them experienced chest tightness, shortness of breath, fatigue, cough, and a “purge” of sorts, whereas the patients coughs out lots and lots of mucus. The medication thins the mucus that so typically thick and sticky in the organs of CF patients, most dangerously accumulating in the lungs and digestive system.

The now-thinner mucus would be easier to clear, but as it came out, it could cause symptoms that mimicked a lung infection.

So the first week, when I was immediately sick, we thought, “this is what it will be for a while…we can handle this!”

Indeed, my mucus was getting thinner. It was easier to cough out. SUCCESS! The basic component of Orkambi was working.

But I kept getting sicker over the next week. I was exhausted – more beat than I had ever felt before. I almost passed out in the shower because I ran out of breath. When my fever hit 103.5, I called my doctors. It turned out that I WAS sick, and that these symptoms were not necessarily all due to the new medication. I had waited because I didn’t want to rush to conclusions.

Long story short, I was sick, and it probably started just before/as I had started this medication. It likely had nothing to do with the new medication.

It was rough. My lung function had plummeted over 30 percentage points, more than 50%, and because I had just been in the hospital only six weeks earlier, when it was nearly impossible to find a place to put IVs in me, my doctors and I decided to try to manage some of my bugs at home. Some of the bugs required IV antibiotics, but they weren’t as active as the ones we could treat at home. We decided to save the IV medications for when those bugs were really making a racket.

Why IV?

Basically, people with CF culture bugs all the time. The thick mucus makes a nice warm environment for bacteria to grow in and set up shop. Lately, 4-5 different bugs have been acting up and show up on sputum tests

Remember petrie dishes from Biology class? They take some of my lung junk and put it on a petrie dish, and see what grows. Then they take antibiotics and see which ones work on those bugs. Several of my bugs are resistant to just about everything, minus a couple of antibiotics only available in IV form. So if I have 4 bugs growing in me, 2 of them can only possibly be managed via IV.

Due to a history of blood clots and scarred veins, placing IVs of any sort – even the longer-term PICC lines (so that I can do 2-3 week courses of strong IV antibiotics), was getting nearly impossible. The last PICC line took 3 attempts and 4 hours, only to end up buried under the muscles in my chest, instead of thread through the veins in my arms.. The one prior to that required a double angioplasty to drill through scarred and clotted veins.

At this point, we had spoken to my doctors getting a PORT – basically a permanent, surgically implanted IV. But my doctors wanted me to be in the best health possible – and not actively sick from my bugs – to do the procedure. We didn’t want those bugs to end up in my blood!

The Next 2 Months

I got a better from my infections for a while – long enough to see if Orkambi was doing anything. I was finally able to see the changes. Unfortunately, for me, most of them were negative.

I had one really emotional doctor visit where I just crumpled into tears, nearly hyperventilating as I told my doctor about the amount of pressure I felt to do whatever I could to keep taking this medication, and deal with the side effects and medication interactions that forced me off several of my maintenance drugs.

I was taking this medication with hopes to help me in the future, but all the work required, and the existing and new issues I was dealing with, presently made me feel absolutely awful.

  • I still had chest tightness and shortness of breath. I was still exhausted – the fatigue wasn’t about needing to take a nap, or the kind of tired that happens from exercising. It was an encompassing, overwhelming feeling that gravity was pulling me down. Every attempt to do the most basic thing left me feeling like I would collapse.
  • Taking Orkambi required me to eat about 20g of fat twice a day with each dose – which had to be taken 12 hours apart. So if I ate breakfast at 8am – which was tough, considering my many digestive issues, the number of medications i have to take before eating anything, and the other things i had to do in the morning for my diabetes and other issues – I couldn’t take my second dose until 8pm. If I couldn’t manage to choke that food down until 10 or 11am, I’d be forced to wait until 10 or 11pm to do it all over again.Given my existing issues eating – nausea and pain from pancreatitis (fat makes it worse), issues relating to GERD, my lack of appetite in general due to regular life or the antibiotics I was often taking – this was a tough feat.Finding something that didn’t make me sick to my stomach, that wouldn’t aggravate my other issues, or upset my easily upsettable stomach was difficult. I had a lot of anxiety over food, because put simply, eating fat – especially so much of it, and twice a day – made me sick.
  • I had uncontrollable Acid Reflux, because the Orkambi basically blocked my Reflux medication from working. Over time, the acid got so bad that it was backing up into my lungs and sinuses. It wasn’t just heartburn – the acid can literally eat through esophageal tissue, and cause pneumonia when present in the lungs. I could no longer treat something that I had managed for years.
  • It also blocked my antidepressant, which had successfully managed my chronic depression and generalized anxiety. They either didn’t do much to help me, or they caused other side effects.
  • I had insomnia. At first, I thought I was just sleeping better, because for the first time in over 10 years, I was able to wake up in the morning and pop out of bed. What propelled me out of bed was not necessarily new-found energy, but a physical sensation that I can only describe as adrenaline or anxiety, that prevented me from falling back to sleep. I HAD to get up, sometimes as early as 5am, whether or not I slept enough the night before.
  • I had a much harder time sensing drops in my blood sugar, to the point where I was almost unconscious before I became aware of it and could safely correct it on my own. Normally, when my blood sugar drops (I have CF related Diabetes), I feel it immediately. If “normal” for me is 90-120, I normally felt my blood sugar drop when it was in the 80s. Now, I was hitting the 50s, 40s, even 30s before I sensed it, and it was very difficult to be lucid enough at that point to drink or eat carbohydrates.

And then, I realized that everything that I had hoped would happen, just was not happening.

I wasn’t getting any better in the lung department. I got sick again just 2 weeks later, and started another round of oral antibiotics. And again. And once again more. That’s 4 new rounds of antibiotics in 3 months, treating just 2 of the 4 bugs active in my lungs right now.

Living for Now, or the Future?

At the 3-month mark, I stopped and asked myself – was I feeling any better? Was it worth a dozen negative effects now, and for who knows how long, for the hope that maybe a year or two down the line, I might collectively have one or two fewer exacerbations?

Over the previous months, each time I talked to my doctors and the manufacturer, they told me to stick with it…that even though I wasn’t feeling any real benefits, the hope what that eventually, I would. Eventually, it should hopefully decrease the number of infections I had, the number of hospitalizations I had to endure, and better my overall functioning.

The theory was that if my body wasn’t producing the crazy thick mucus, that the bugs wouldn’t stick around and multiply like they have been, that perhaps I’d have a lot more energy, that perhaps my Diabetes would be more manageable, that maybe my chronic Pancreatitis would ease up.

So I made a list.

One or two symptoms seemed a little better, but i had a laundry list of negatives.

I had side effects that made me feel miserable. In addition, Orkambi interfered with 3 of my most important daily medications, setting off as domino effect of worsening symptoms. Not having them meant that I was experiencing unpleasant/painful issues that had been controlled for the years I have been taking them.

So now I was at a point where I had 2 issues that seemed better, but now a dozen or so issues that were new, worse, or had returned.

What do I do?

I had read stories about patients for whom this medication gave them back “life”. But I also read about patients who had to stop taking it, whether due to side effects, lack of improvement, or interactions with their maintenance medications.

I wasn’t alone. Other patients experienced similar problems, and had to stop taking it.

No medication works for everyone – just like how some people swear by Tylenol for a fever, others only take Motrin.

The problem is, there was so much hope surrounding this new drug. People even used the word “Miracle”. I had tried not to get too hopeful, but it was the very first time the CF community has had any hope since scientists first discovered the gene responsible for the disease, back in 1989. Back then, everyone said a cure was “around the corner”. But my husband, my family, my friends – we were all excited about the possibilities. Maybe, just maybe, we thought…

It’s done.

We did it. I took my last dose Tuesday night.

My doctors are working with us to make sure that they’ve covered all bases while coming off the medication. Some patients have reported complications, so my husband and I, and my medical team, are ready in case I need any medical care while my body adjusts. Ideally, because I didn’t feel a lot of benefits, I won’t feel too much discomfort as the drug leaves my system.

I am just starting to feel the emotional effects of stopping what I once thought could be my miracle. I am slowly understanding that this was not my second chance…

…that there’s no extra hope that maybe this year, we can take our first real vacation in 9 years.

…that we can make plans, plunk money down on plane or concert tickets, and have any certainty that I’ll be able to go.

…because we’ve tried that so many times, and so many times have had to reschedule and cancel.

…because I’m tired of losing money to airlines and missing events or special gatherings that I so very much wanted to attend.

…because I’m tired of disappointing people I care about, when we have to say “no, we can’t go” or “sorry, I’m going into the hospital again.”

So we move forward, one moment at a time.

I don’t know what the future holds. Medically, I know that sooner or later, I will end up needing IV antibiotics, and based on what the past several years have brought me, it will likely be within the 4-6 month timespan since my last hospitalization.

As for getting to see Billy Joel at Fenway Park – we didn’t buy tickets this year. And the plan to go on vacation, perhaps returning to sunny Puerto Rico, where we had hoped to see again – probably won’t happen. I just haven’t been well enough that I can choose any random date or week at some point in the future, and expect that I will be well enough to attend or travel.

I don’t want to discourage anyone from trying this new medication, or any new medication. I also don’t want to detract from the stories of patients who’ve found new life with the first CF therapy of its kind.

It just was not right for me. I can’t say that I’m not disappointed. It’s just now slowly sinking in, and I’ll have to process it somehow.

I am thankful to have had at least the chance at a “second chance”. Not everyone gets to experience that kind of hope.





One thought on “Not My Miracle

  1. Andrea says:

    We will continue to fundraise and cross fingers for you and everyone with CF. You are very brave, a quality you share with many people with CF. Thank you for you candor and sharing your Orkambi experience, as heartbreaking as it is. I hope your positive attitude and inner strength allows you to take a vacation one of these days.

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