In my life, I’ve seen lots of advancements in CF treatment. These have all been medicines/techniques/what-have-yous don’t treat the underlying cause of the disease, but mitigate the symptoms. None of it is easy or fun, but it does provide a concrete answer to the age old question of what I would do for a Klondike bar: I would take 8 pills and 8 units of insulin.
Kalydeco is different. Kalydeco is designed to treat the underlying cause of CF. Unfortunately, while all Cystic Fibrosis is a result of a defective Cystic Fibrosis Transmembrane Regulator Gene, they are many unique ways in which the CFTR gene can tell a person to go fuck themselves. So a treatment that works for one CF mutation may not work for another (for more information on CF mutations, stop by the Cystic Fibrosis Foundation Website http://www.cff.org/AboutCF/Testing/Genetics/CFMutations/).
Kalydeco currently works very well for people with the G155D CF mutation. In this mutation the defective CFTR protein—the product of a mutated Cystic Fibrosis Transmembrane Regulator gene—moves to it’s proper place on the surface of a cell, but instead of letting water and salt through like it should, it gives them the finger and tells them to piss off. Kalydeco unfucks this protein and allows salt and water to pass through the cell normally.
The protein for my mutation, Delta F508 Homozygous, can’t be bothered to come to the surface of my cells. It’s like an internet troll, lobbing insults from afar to make sure it never knows the touch of a woman—or, in this case, salt and water. So the prevailing idea is that another compound could be used to draw the protein to the surface and then Kalydeco could give it a proper talking to. My proteins could use a talking to, so I enrolled in a research study to see how Kalydeco (also known as Ivacaftor or VX-770) works when combined with a compound called Lumacaftor or VX-809.
This was a potentially life changing study, but I tried to keep my expectations in check. Besides, since it was a double blind study with two dosage groups and a control, there was a 1 in 3 chance of getting a placebo. I would have been okay with the placebo though, as I’d still get free meals and money just for showing up. I’ve had CF for over 30 years; I know what having CF is like and I’ve already planned to deal with it until I die. I’d never been paid to go to the hospital. THAT sounded exciting.
But when we got word that I would be in the study, a thought came knocking on my thought-door. At the outset, it seemed harmless enough, but I sensed something insidious about it, so I never invited it in. The thought was this:
“Wow, wouldn’t it be nice if I could finally stop all that other shit?”
I did not want that thought in my thought-house for a multitude of reasons. We’ll get to those.
Personal drama aside, let’s talk about the actual process. I went for 6 “study visits” over a six month period. At these visits, I was issued packets of pills designed to both sort out the pills and to make sure my floor always had at least 5 small cardboard ovals on it. I was to take 5 pills in the morning and 4 at night, always with food.
Due the time of my study visits, it had been established that I would take my morning pills around 10am, which proved difficult. At the time I was working from 6pm-2am, so I had to wake up, get something to eat, take some pills and go back to sleep. More than once, I woke up, ate, then went back to bed, only to wake up an hour or so later to repeat the process, this time with the pills. I’m not proud to admit that I threw the container of pills in anger more than once, as if it were the pills fault that I have trouble waking before noon (in my defense, I also threw the container of pills a couple of times for things that the pills did do, but we’ll get to that too).
So did these pills work for me?
First, a disclaimer: Being that this was a double blind study, I have no idea what I was taking. I very well could have been taking the placebo. However, I’m currently enrolled in a two year continuation study in which I know I’m on the real stuff. I’ve been in the continuation for about 12 weeks and not much has changed, so I’m pretty confident that I was on some dosage of the drug. But correlation not being causation, maybe my digestive system decided to start being terrible at the exact moment I enrolled in the study.
I thought this study might be a big turning point for me (and I sensed an opportunity for a sequel to my book), so I kept a journal for the first month of the study. I present excerpts from that journal here:
I sent Bekka a picture of my face after I got out of work last night and she said she thought it looked larger, then we Facetimed so she could confirm her diagnosis. I thought it was because my beard trimmer broke so my face hairs are all big and bushy right now, but she swears that my cheeks are finally retaining water. She also says that my voice is clearer. I will have to take her word on these things. Hopefully, mucus is not the key ingredient to my golden pipes.
As I write this, it’s midnight on the 3rd and my chest actually feels a little tight. Whether that means it’s getting ready to do something or it’s just the standard bullshit it always does, I have no idea. The suspense is killing me!
My sugar was running a little high (195mg/dl) when I ate dinner, so that may have something to do with it. It’s still a little high (185mg/dl) two hours after, so maybe I should have had another Prandin for dinner.
I’ve been pretty tired all day, which is either a function of the fact that I have to get up at 10am to take my pills or that I got the placebo. No chest pain though, so that’s good. Last night, my chest felt like a very tiny balloon that had been over-inflated to lung size.
Bekka says I’m tired because my body is sending all my white blood cells to fight the crap in my lungs. I certainly hope so. This is the first day since I started the pills that I feel under the weather. I wanted to write “like shit” there, but that’s hyperbolic. I’m just a little run down today, which is usually how I feel once I get three days deep into the workweek. I was hoping the pills would be kicking in by now and I’d be fighting camels in underground arenas, but alas, it’s just a regular ol’ workday for me.
My mucus today is thinner than it was yesterday, but still thicker than it was earlier in the week. I seem a little less productive as well, but I still got a decent amount moving. Around 9pm, I started to feel the chest tightness creeping up and after I ate lunch it wasn’t getting any better. I tried drinking a shitload of water and that seemed to help, but I slowed down once I started pissing so hard I thought I was going to damage my blowhole. Still, I don’t feel the same fatigue I had yesterday, so that’s a good thing.
I called Bekka around 9:15. She said I sounded much more phlegmy than I have in the past few days, but she seems to think that it’s just me working through some of my older mucus. It also could have been excitement in my voice because she secured herself a fancy salon stylist job today and I am very proud of her.
I don’t seem quite as angry today as I have lately. I thought maybe the study drug was affecting my mood slightly, but I think I was just mad at it for not working. It’s not like a tremendous mood swing or anything, as I usually end each day thinking everything is bullshit, I just feel like I’ve been getting there much faster over the past few days. The feeling reminded me of taking Bactrim, which I usually take to clear up infections. It seems like Bactrim makes me mad as a goat at a dog show, but it’s tough to tell because I only take it when I’m already sick and angry. I’m going to give the study drug the benefit of the doubt and say that I’m just naturally angry. For now.
I’ve officially wiped my ass raw. The poops look well formed, but they’re coming out soft serve and making a damn mess. That’s certainly happened before, but never for a sustained period. This, combined with the fact that my chest seems to get tight after I eat leads me to think that maybe I should try dropping my enzyme dosage and seeing what happens*. Maybe my body is rejecting them.
I’ve also been getting a little more in the way of acid reflux, which is interesting, because I’ve been hitting the omeprazole just like I always have. It’s not painful, but it’s noticeable. This could also be a function of the fact that I tend to eat a lot and my food always seems to be percolating at the back of my throat. I’ve felt this feeling before I started the study drug, but it seems more pronounced now. Maybe I’m just paying more attention.
*I don’t know where that “drop my enzyme dosage” nonsense came from, but it’s one of the stupidest fucking things I’ve ever written. That’s as close to optimistic as I get: I thought that my body was working so good that shitting five times a day was its way of saying it’d like to digest its own food.
I woke up again at 2:45pm, thought I felt like I could’ve slept until 2:45am. However, it was fairly easy to get up because my stomach was very persistent in letting me know it had to make a delivery. It was quite a delivery–bountiful and solid, but orange and hot. It was through this experience that I decided to keep taking 8-9 enzymes when I eat. No more of this 6-7 bullshit.
I meant to get an intense workout in today to see if I could move up some crazy mucus, but my body felt like it was moving in slow motion, so I just laid in bed reading until it was time for work. Even when I finally did force myself to get up, I felt like I was underwater. It was odd, because I didn’t feel like I was sick or even that I wanted to sleep; I just wanted to lay down for a while. Duty called though and I got my ass up and went to work.
Bekka might be right about my face getting fatter too, because I’ve bitten my cheek twice today. Maybe I have to start doing some sort of mouth workout.
Why does that sound dirty?
I went to Wal Mart last night after work to see if I could hunt down something that was easily microwavable and over 20 grams of fat. I settled on some generic sausage, egg and cheese biscuits.
They did not, however, settle in my stomach. The one I ate this morning went down smooth enough, but I awoke this afternoon at 3pm with a powerful rumble in my stomach.
Turns out that rumble was a pile of turds. A healthy pile too. It was apparently prefaced by a number of sleep farts, because my room had quite a note of bunghole to it when I woke up. I did not feel like a champion today.
This was probably the first day that these pills have felt like more trouble than they are worth. I haven’t been coughing as much, so I guess that’s good, but I’m pretty sure they are interfering with my acid reducer and that means my enzymes are getting torn up before they get a chance to do their thing.
Today went fairly smooth, health wise. My morning sausage biscuit did not beat my stomach up today and I was able to get a workout in. I took one shit that smelled like Beggin’ Strips but it was solid, so that’s an improvement.
I had a decently productive cough on the ride into work, but that seemed to dam up once I got in. I was hoping we were about to see a deluge of old mucus liberated from my lungs. Instead, I got some false hope and swampy feeling lungs. It was nice while it lasted.
My current theory is that the pills might move the upper and lower limits of how I feel, but not affect the median much. So even if I get sick when I’m on them, I won’t get as sick as I would have if I didn’t have the pills and when I feel super great, it will be even better. But I spend most of my time driving in the center lane and there’s no change there. Hopefully I’m wrong.
On the bright side, I did make a poop that smelled exactly like an old basement. That has to count for something.
Today was the first day that I felt like I can’t wait for this study to be over. Waking up at 10am is a pain in the ass and I feel like I’m not seeing enough return on this investment. I feel like my tune will change once the checks start rolling in though. I’d really like those checks to show up.
Today brought one interesting development in that when I woke up at 10am, the first thing I did was cough up some mucus in which one tiny sliver had the rough consistency of a dried booger. Am I finally moving mucus plugs? We can only hope.
I keep waiting for a study check to show up and I keep getting disappointed when I get nothing. A little cash would really make this feel worthwhile, because though I’ve evened out pretty well, I’m not sure that I feel these pills are worth the hassle. Bekka says I look better though, so I guess there’s that. At least these pills are doing something for one of us!
It was another one of those days where you could have easily slipped these pills into my oatmeal and I wouldn’t have noticed a difference either way. I don’t feel terrible, I just don’t feel any better. Bekka has been comparing old pictures of me and making a lot of Facebook posts, because I think she really wants to believe that these things will work, but I just don’t feel it. Maybe tomorrow I will.
The only real item of note is that a massive globule of snots flew out of my nose while I was exercising today. I was outside, so that was good, but it splattered everywhere, dropping shrapnel on my shoe and making a terrible mess. It had roughly the same consistency of loose lung mucus. Perhaps it’s old stuff moving out?
End Journal Entries
You’ll notice that as early as Day 20, I start wondering when I’m going to get paid, which isn’t a good sign. Day 30 is the point I stopped keeping the journal, because I got sick of writing “Nothing has changed” again and again. Then when things finally did start changing, I was out of practice. But here’s what the journal missed:
I had been told before starting the study that it may interfere with the way my Prandin—a small pill I took to control my blood sugar—worked and that I might end up on insulin. So the Wednesday before Thanksgiving, I started taking insulin. You can read more about that here.
I also dealt with some pretty impressive acid reflux, which resulted in a doubling of my omeprazole dose and the acquisition of a moderate Tums habit. However, none of this improved the efficacy of my enzymes—the pills I use to digest my food—so I eventually upped my dose from 8 per meal to 10. When that didn’t help, I went to 12. Twelve enzymes finally brought my shits down to a more manageable 2-3 a day, rather than the 5-6 I’d been spraying over the toilet previously.
Some patients have seen an increase in liver enzymes in this study, so monthly liver enzyme tests are now a thing. My liver enzymes didn’t spike, but my testosterone almost doubled. Maybe that’s why I threw the pill packet a couple of times and recently pounded my first on the armrest of my car when Bekka asked why I didn’t run a yellow light (I usually do).
It hasn’t all been bad though, as my mucus does seem to be a little thinner than it used to be, though no less sticky. I can now manage a mild laugh without choking, which is an improvement, but that Batman boner comic still brings me right to the edge of hospitalization.
Pushing aside the anecdotal stuff, I was not allowed to see my lung function in my study visits. And though I would have been allowed to see any lung function tests performed during my regular clinic visit, I declined. Partly because I wanted to stay as blind as possible and partly because I showed up late and missed the test anyway.
Given the way my appointments fell, I saw my pulmonary function test from one week before the study and then one week before the end of the study. It was a grand total of no improvement. But there was no decline either, so I guess that counts for something. (I don’t remember the actual numbers or I would print them here).
However, for the first time in my life, these pills made me not want to eat.
One time I went to an all you could eat seafood and steak buffet where I ate 12 lobsters and 5 steaks. Later that night, I went to Denny’s with some friends and when one of them couldn’t step up to the challenge of the Philly Cheese Melt, I ate that too. I threw up 14 times that night and was pretty sure I was going to die in my sleep. I didn’t. But you know what I wanted when I woke up? A goddamn lobster.
My appetite could not be defeated by the 2.5 hour time limit imposed by that buffet, but these pills brought it to the mat. And it wasn’t that I wasn’t hungry or didn’t feel like making food: I just didn’t want to eat. Granted, it wasn’t every day that I didn’t feel like eating, but it happened. And it chilled me to my bones.
Once we figured out that I had to double my omeprazole, add 50% more enzymes to my meals and switch to insulin, this happened less often, but I sometimes get a little nauseous right after a meal. Now, these pills never stopped me from eating—I’ll be goddamned if I let some pill boss me around—but they took some of the fun out of it.
So why am I still on them? Well, the main reason is that I really want a laptop and I’m only in it for the money. But part of me is scared to stop taking them. I haven’t had a pleasant time on them, but what if it gets worse if I stop? It’s a decision I’m scared to make myself, but if it was made for me—say by elevated liver enzymes or something—I’d probably do a little dance. Or I’d spend the rest of my life thinking about the pills that got away. Also, being in a research study makes me feel like I’m doing something to help, even if that something is shitting 5 times a day.
I have no doubt that someone somewhere will eventually cure CF, though the chances of me stumbling across the answer via the chemistry kit I bought from Fingerhut are getting less and less likely. I don’t know if I’ll be around when the cure is found or if I will have done enough damage that it will be too late for me even if I’m still alive. I believe there is another study being done to gauge the efficacy of Kalydeco and another compound, so maybe that will work out better. Hell, just the fact that these pills have done something—even if that something wasn’t all beneficial—is a good sign.
But seriously, how many years is it going to be until I can 3D print myself some lungs?