You can read more about my journey to get Spinraza— the first treatment for Spinal Muscular Atrophy— by reading these blog posts: Elizabette & Spinraza
You can read more about my journey to get Spinraza— the first treatment for Spinal Muscular Atrophy— by reading these blog posts: Elizabette & Spinraza
Everyone has that one cool older cousin— the one that just makes you think, “Wow, she’s so rad! I want to grow to be just as cool as she is!” The kind of girl that listens to alternative rock or smooth jazz and wears giant clunky Doc Marten boots heavier than a WWII German Panzer tank.
But, this chick wasn’t so cool that she scoffed at the letters her little cousin wrote to her on bright pink Hello Kitty stationary. No, she wrote back diligently, even though, at the time, the little 12-year-old me was about as cultured and interesting as a really bad episode of Full House (The original one… with the damn Olsen Twins!)
But, it didn’t seem to matter to her. She loved me just the same.
That was my Christine.
Given the age difference between us (8 years), as I grew, so did our relationship. I stopped being the annoying little cousin that talked about Legos and glitter pens. I became a grown-up person that talked about grown-up things. Like why the hell did she think that a kalimotxo was preferable to a 7/7? And even before the ATV wreck that messed up her hand, why on Earth did her handwriting still have to be so awful?
We’d talk current events, books, and how to roast the perfect leg of lamb. We’d strategize ways to castrate our least-favorite politicians while gleefully eating our Aitas’ homemade tripota.
When my mom was diagnosed with the brain cancer, glioblastoma multiforme, she was there each step of the way until the end— hugging, and squeezing me, with a remarkable amount of restraint so that she wouldn’t crush my cripply ribs. If you knew Christine, you knew it was nearly impossible for her to hug with anything less than Superhuman strength. But, for me, she managed.
That was my Christine.
As we were both only children, we shared an extra-special bond. We understood the joys, and sometimes burdens, of such a thing, while relishing in the stories of our Aitas— which we told with great vigor and our very best Basque accents. We commiserated together when our parents wouldn’t let us dye our hair purple, but secretly rejoiced when Christine and her badass-self went out and got her lauburu tattoo.
On that September afternoon, 12-years-ago, when she called me to tell me that the pathology report had come back from her surgery, her voice was strong and steady, like a soldier ready for battle. “It’s malignant. Stage 4.” My heart stopped and then began to pound in a beat that nearly drowned out the sound of her next words… “It’s glioblastoma multiforme.“
I knew in that moment what her eventual fate would be. But, I waited to cry until we got off the phone. And I cried. And I cried. And I said a prayer, “Please let her live long enough to see her little boy become a young man. Let her fight it off until then.” And, the fucking lioness that she was, so she did.
Her loss is great, but the life she lived was even greater. She loved fully and deeply— and that gift lives on in all of us. I know there will come a day, very soon, when I’ll reach for my phone to tell her something, but she won’t be there. And my heart will break just a little, once more. But, I’ll hear her voice in my head, “Don’t be sad, caca. It’s going to be okay.“
That was my Christine.
I’m sure some of you may be wondering how my quest for Spinraza— the first treatment for Spinal Muscular Atrophy (SMA)— is faring. Obtaining this treatment isn’t for the faint of heart, it takes will, perseverance and just a little desperation— like climbing Mt. Everest, running a marathon, or taking a job in the Trump Administration.
But, I’m still waiting to receive the treatment. If I was a female chipmunk, I could have already given birth to nearly three litters of babies in the amount of time I’ve already been waiting. That’s a lot of chipmunks.
Insurance companies around the country have a wide range of policies on Spinraza. While the FDA approved the medication for all ages and types of SMA, at $750,000 for the first year of injections, it is in the insurance companies’ financial interest to limit access to the treatment. So, they have come up with a wide variety of parameters that they are using to approve/deny payment for the drug. Some insurances say you have to be over 15 years old, other insurances say you have to have less than 3 copies of the SMN2 gene, while some say you have to have over two copies of SMN2. Odder still, some are saying that that they won’t pay for those who already use a wheelchair… or a ventilator… or have a last name that ends with “r.”
I have Medicare, so my insurance situation is even stranger. Medicare is requiring something called “Buy & Bill.” Administering hospitals must purchase Spinraza themselves and then Medicare, in theory, will reimburse it. However, under this current set-up, the financial responsibility/burden is on the hospital. And most hospitals are understandably hesitant to assume that risk because they know there is a chance that Medicare will not reimburse 100% of Spinraza’s cost.
Would you fork out nearly a million dollars for a medication if you weren’t sure you’d get paid back for it? Uhm, HELL no. And no one wants to be the bad guy that sends on that kind of bill to the poor cripples in the wheelchair. I may not be in marketing, but even I know that’s not good PR. So, because of this policy, those of us with Medicare are in limbo. We’ve got prescriptions, but no way to get the drug— it’s like having a ticket to the moon, only NASA is broke and all the space shuttles are in museums.
I’ve been told by folks at Biogen (the distributor of Spinraza), and at Stanford, that talks are happening behind the scenes to figure this out. But, as time goes by, all of us with SMA get weaker.
Being patient is difficult, especially for someone like me that doesn’t have a lot of patience from the start. After all, I can barely wait for a grilled cheese to come out of the pan before I take a bite. I hover over the stove like a drone— and I burn the roof of my mouth EVERY. DAMN. TIME. Who can wait for that melty cheesiness? I know I can’t. You’d think that the resulting soreness in my mouth would deter me from doing it again the next time, but I’m just not that smart.
I’m sure some of you reading this are aghast at the price tag associated with Spinraza— and are thinking that there is no need for the treatment to be that expensive. But, please don’t descend upon the makers of this drug with torches and pitchforks on my behalf.
A specialized medication of this caliber is years in the making. There are many costs that go into creating something like this. So, they need to recuperate what they have invested in it. Further, this isn’t a medication for diabetes or high cholesterol— only a very limited number of people can use Spinraza. So, each dose has to be expensive to make it worth their time. If there is no financial incentive to research rare conditions, like Spinal Muscular Atrophy, then no one would bother doing it. That’s a fact. Kindness and altruism doesn’t make the world go around— money does.
In the meantime, today I received this Spinraza welcome packet from Biogen in the mail. It’s a treatment organizer— like the planner I had in high school that I rarely used. This organizer also came with a decidedly cheap pen— emblazoned the Spinraza logo.
No offense, Biogen, but it’s 2017. Who writes with an actual pen, anymore? And who carries a planner? No one.
And I’m going to be frank… Don’t be like Clark Griswold’s cheap boss that bought him that crappy gift in National Lampoon’s Christmas Vacation. At $125,000 an injection, you could buy me an iPad, instead.
I’ll be waiting. You know where to find me.
Ugh. As I sit here typing this, the thermometer is registering 95°F. It’s barely May and only 4 days ago I still had flannel sheets on my bed. And now I’m sweating and my jeans are sticking to my ass. Why couldn’t the summer heat arrive gently? Like the slow bloom of a flower… or water dribbling down a clogged drain. Why, instead, does it have to be so blunt and nasty? Like a bull, or Steve Bannon, in a china shop?
I HATE SUMMER! There, I said it. I don’t like the heat, the sun, or even the clothes. I much prefer the styles of the cooler months— my cabinet full of scarves is a testament to that. Plus, I can’t wear most summer clothes, like shorts, because I sunburn in mere minutes. This isn’t dramatic hyperbole. One time I got a sunburned in the time it took to go from the front doors of the shopping mall to my handicapped parking space— which was IN THE FRONT. Yes, I really am that white.
Now that we’re on the subject of summer attire, I also don’t understand flip-flops. They seem unnatural and highly dangerous. Why would you want to walk around on something so unstable? It’s a shoe that’s barely attached to your body. You are one thin plastic strap away from disaster. It’s a good thing that I can’t walk, because if I had to wear flip-flops in the summer, it wouldn’t end well for me. First I’d get a bad sunburn on the top of my foot, then I’d fall and break my nose on the nearest object, like a ficus plant. It would be like an episode of The Three Stooges, only less funny and less Jewish.
If all that weren’t enough, the slapping sound flip-flops make is also decidedly unappealing. Do you intentionally want to sound like a walrus flapping their hands together? Because, I hate to be the one to say it, but you really do sound like that.
Anyway, thankfully I have air conditioning to help me during these trying times. It’s currently humming in a soothing way that reminds me of those noise-canceling machines that they used to sell at Bed, Bath & Beyond next to the display of soda-making kits that NO one ever buys. The same ones that have been on sale since 2005. If you are thinking of buying one, you might want to reconsider; I’m sure those flavored syrup pods expired back during the days when Bush Jr. lived in the White House.
Those were simpler times, though, weren’t they? We didn’t have a president with orange skin and hair… and Michael Jackson and Whitney Houston were still alive— so we could listen to their music without the sad pang of nostalgia. Those also were the days before the word taxi had been replaced by Uber. Before long the nerdy folks at Oxford will just drop that word from their dictionary entirely… and a hundred years from now little children won’t even know what a taxi is, let alone that it was yellow and usually driven by strange foreign men with accents— or Tony Danza.
It’s only May, though, so I better get used to this heat. If you need me, you can find me sitting in front of the fan, grumbling… and not wearing flip-flops.
The last few days I’ve been battling an infection of some kind— with a sore throat, cough and fatigue. Rest assured, I’m definitely on the mend now, though, thanks to a round of antibiotics, and my nebulizer and Cough Assist machines. Three cheers for respiratory gadgets!
Even though I’m an avid reader (I’ve been known to read 50 to 75 books a year), when I’m under the weather I actually don’t do much reading at all. Rather, I like to sit like a hacking, sniffling lump while I watch long movies. And when I say long, I mean really long movies. The kind of movies involving far away wars, people who sing, Nazis, nuns and, of course, Julie Andrews. If you haven’t gotten the hint that I’m talking about The Sound of Music, I’m not sure if we could ever be friends. I’m serious. That movie is the best movie of all time… I say that with utter certainty. If you dare to disagree me with me, I will run you over. This is not an empty threat.
Anyway, I love to watch this film when I’m sick. It’s long, too, which makes it an especially good choice for long afternoons of lazy, snuffly sickiness. Plus, if all that weren’t enough, Captain VonTrapp is super dreamy. When I was young and home sick from school, my mom used to pop the movie in the VCR. It would keep me occupied, and out of her hair, for at least 174 minutes. Any mother would tell you that this fact alone would make that VCR cassette tape worth its weight in gold. And, as soon as I was old enough to operate the VCR remote control, I would repeatedly rewind the tape to watch the marionette puppet scene over and over again. If you don’t know the scene I’m talking about, you haven’t seen this movie. And, if you haven’t seen this movie, well… you know what will happen to you next.
I think it’s safe to say that I have seen this movie over 50 times. This is not an exaggeration. It’s at the top of my list of Movies to Watch When I’m Sick. Other flicks on the list include: Mary Poppins, the Harry Potter movies, musicals written by Rodgers and Hammerstein, and any film starring a Hepburn (either Audrey or Katharine).
This week, I needed to find a long movie to make this sickness pass faster. There was a free preview of HBO, so I checked out the guide on my television. I found Batman v Superman: Dawn of Justice… which was an impressive 183 minutes long. This was doable. I’m not generally a fan of superhero movies, but I have a decided weakness for Henry Cavill— the hunk that plays Superman. So, it seemed like a good choice.
But, it wasn’t too far into the movie that I began to doubt the merit of my selection. Through the fog of antibiotics and decongestant, I watched car chases, tall buildings being felled, and two men wearing capes fighting each other. This last one had me nearly turning off the television. Batman and Superman are both good guys. Under no reasonable storyline would they be at odds. End of story. With a groggy voice, and no one around to hear me, I actually yelled at the TV (okay, maybe I squeaked), “WHO WROTE THIS MOVIE, ANYWAY? WHAT IS EVEN GOING ON? WHY AM I WATCHING THIS? HOLY SHIT, HENRY CAVILL IS HOT.”
Next time I’m sick, I’ll just stick to Julie Andrews.
While it may not seem like it from the outside, there are definite benefits to being me. There are the obvious ones— preferred parking spaces, event seating in the front (should the venue be equipped for it), and being able to get away with first-degree murder because no one will believe that the perky girl in the wheelchair could possibly poison anyone with arsenic.
Aside from these obvious benefits, there are the not-so-obvious ones, too. Like my shoe budget. I rarely have to buy new shoes because each pair I buy will last a ridiculously long time. This is what happens when you don’t go walking around in your shoes. While Nancy Sinatra may have sung “These Boots Are Made For Walkin’,” she clearly wasn’t talking about me. My boots are made for sitting in my wheelchair while I do things like try not to stain them with dribbled spaghetti sauce. (This is a serious concern. My wardrobe is more susceptible to food-spillage than actual wear-and-tear.)
I’m not ashamed to admit that I still have two pairs of shoes from the 8th grade. I am not kidding. As I know that fashion is cyclical, I’m just biding my time until 90’s chunky heels are all the rage again. If acid-washed jeans can make a comeback, so will my Payless black leather loafers.
If you don’t believe me, I’ve taken this picture of my shoe closet. The average age of a pair of shoes in this closet is 9.7 years.
Anyway, being me is not all sunshine and rainbows. It can be challenging when you get an itch you can’t reach or drop something on the floor and you have to wait for someone to pick it up. Patience becomes a daily practice, an endeavor. It’s not an obscure or unknowable concept — like what the hell is really going on with Trump’s hair.
Yesterday, I had such a challenge. While I was on the phone with my aunt, I felt something weird on my neck. Thinking it was just my hair tickling me, I ignored it. A few minutes later, I felt it again. I looked in the mirror and I saw a spider crawling around on my clavicle… like it owned the place.
To say I freaked out would be an understatement. I hate bugs with a deeper passion than I feel for anything else on this Earth. And that is saying a lot because I really hate manbuns.
Nearly dropping the phone, I went to my dad and shrieked, “THEREISASPIDERONMYNECK! GETITOFFNOW!” With exasperated amusement, he flicked off the spider and it smashed on the knee of my pant leg— leaving a stain on the jeans that would be a reminder for the rest of the day of my traumatizing experience.
In his accented voice, my dad then scoffed, “Bah, eet was juust a leetle bug.“
This was a total lie to try to make me feel better. But, I was the one that had it crawling around on my neck like a fucking Land Rover on safari. In my mind’s eye, heavily colored by my assorted anxieties, the spider was NOT little. And it never, ever will be.
I guess being me isn’t all fun and games…
For a person with Spinal Muscular Atrophy (SMA), like me, respiratory care is a key component to our well-being. The muscles closest to our spines are most effected— while the severity of muscle weakness lessens the further you move out to the tips of the extremities. Therefore, to say that the muscles that control my respiratory system are not great would be a vast understatement. It would be like saying that King Henry VIII only had a slight problem not killing his wives.
You know the satisfaction you get from hocking a really big loogie? Yeah, that’s not so easy for someone with SMA. I would love to easily hock a loogie when the occasion warrants. It’s definitely on my all-time wish list of things to do— which includes marrying George Clooney and traveling back in time to buy stock in Apple when they were still making computers in Steve Jobs’ crappy garage.
As a result of my muscle weakness, I also have scoliosis— which further impairs my respiratory function. In fact, my right lung is so squished that it really doesn’t do much. Despite that, I’m quite surprisingly fond of it anyway. It’s decorative and ornamental— like Melania Trump.
Therefore, keeping colds, viruses and flus at bay are essential. A minor cold that would just make you snuffly for a few days can knock me out for a week or two. And, the specter of pneumonia is always hovering over my shoulder— waiting, watching and listening. Just like Vladimir Putin.
Like many with SMA, I use a BiPAP respirator machine at night while I sleep. While I wear a mask similar to those with sleep apnea, this machine instead ebbs and flows with the rhythm of my breathing— like the tides of the ocean or a politician’s approval rating.
However, this machine is not as soothing as it sounds. It bears no resemblance to the nature-sound CDs that they used to sell at Bed, Bath & Beyond for $9.99. It took me over a year to get used to this damn thing. You know a hurricane? Imagine that in your face while you are trying to sleep. For nearly a year, I vacillated between wanting to throw in the towel and return the BiPAP to the doctor… to getting my dad to run over the f$&@ing machine with a John Deere tractor.
Along with my respiratory gadgets, like my BiPAP, I have an assortment of techniques that I’ve developed over the years to prevent infections:
A few days ago, I headed back to Stanford to see the pulmonary specialist and to finish up the loose ends of my Spinraza evaluation. While there, I saw a kiosk at the entrance of the neuroscience building. It had an automatic hand sanitizer dispenser, tissues, and masks— all in one display case. It was amazing. I felt like a kid on Christmas morning— if that kid was a raging hypochondriac. Since I couldn’t feasibly steal the whole display, like I wanted to do, I took a photo by it instead…
Here’s to a cootie-free Spring!