Bits and Pieces: Tecfidera Poll Results Edition

(Photo credit: Ancratyne)

(For those readers who receive Wheelchair Kamikaze via email, this post contains a video that cannot be viewed in standard email formats. To view the video (click here) to open the Wheelchair Kamikaze website in your browser.)

Before we get going with this edition of Bits and Pieces, I’d like to thank all of the wonderful folks who left get well wishes in the comments section of my last post (click here) and who sent me emails full of warmth and support. Your words were appreciated profusely, and I only wish I’d been feeling well enough to respond to each message individually. I did read each and every one, though, and they were far better medicine than anything the doctors could ever prescribe.

I’m still on the mend, even after six weeks since I first took Acthar Gel, which precipitated my unraveling. My physicians are still rather confounded at my reaction to the stuff, and my blood work shows that my endocrine system remains in a bit of a tizzy. I’m feeling much better than I did about four weeks ago, but not nearly back to where I was before things went kablooey. Bit by bit, I suppose, and I’m sure my old friend creeping paralysis isn’t helping with the restorative process. As Samuel Goldwyn once said, in this life you’ve got to take the bitter with the sour…

Anyway, enough about me, as I’ve spent the last six weeks or so stuck in bed contemplating little else but my predicament. Far too much time spent gazing inward, and poking and prodding all the monsters that lie within. Figured a Bits and Pieces post would offer a potpourri of distractions to me and all the folks who read WK, so off we go with a collection of items of interest that were able to catch my attention these last several weeks. Hope you find them  informative and/or entertaining.

♦ First up, a look at the results of a poll that’s been running on Wheelchair Kamikaze for the last 3 ½ months or so, designed to get a snapshot view of the experiences of patients taking the new oral MS drug Tecfidera. If you are taking Tecfidera and haven’t yet participated in the survey, please feel free to do so now, as the poll is still “live”. The more respondents we get the more accurate the results will be, so please do take the time to answer this brief survey if you are a “Tec-y”:

How would you characterize the side effects you’ve experienced as a result of taking Tecfidera?


Have Tecfidera’s side effects forced you to stop taking the drug?


(This question should only be answered by those who answered "no" to the previous question) How would you characterize any benefits you’ve felt since starting Tecfidera therapy?


In order to view the results of the three poll questions, just click the “show results” link beneath each query.

Before I attempt to analyze the above numbers, please allow me a few words about polling bias and how it might affect these results. I know, you’re all probably thinking, “What a blowhard, what can the Wheelchair Kamikaze possibly know about the intricacies of polling?”. Believe it or not way back in the 1980s I worked for a political research firm that conducted well-publicized polls on a national level. In fact, we were responsible for the numbers reported on the NBC nightly news every evening during the 1988 presidential campaign, which pitted Michael Dukakis against George W. Bush.

I was hardly an executive in the firm, but I did supervise the “phone bank”, where 40 or 50 people dialed phones incessantly for five hours every night, asking the people on the other end of the line their preferences in the presidential race and their opinions on the issues of the day. Chief among my responsibilities was making sure that the people responding to our questions were representative of the population at large in respect to sex, age, race, income, and other demographic criteria. Obviously, a national poll with a disproportionate number of Southerners, or too few women, or too many Hispanics, would skew the results and lead to highly inaccurate conclusions. For any poll large or small to have a chance at being accurate the respondents to that poll must be representative of the target population as a whole.

This same element comes into play when looking at the little Internet Tecfidera poll I’ve been posting on this blog. Since there’s no mechanism to screen those responding, there’s no way to control for an over or under representation of the myriad variables among the folks who comprise the Tecfidera taking population. Furthermore, the simple fact that this is an Internet poll automatically skews the results, as it doesn’t capture the opinions and experiences of Tecfidera users who aren’t surfing the Internet. Of those who are, it’s only representative of the relative handful of patients who have stumbled upon the poll here on Wheelchair Kamikaze and have chosen to participate.

My hunch is that the very nature of a wide-open Internet poll such as this probably skews the results a bit towards the negative, since the many patients taking Tecfidera and experiencing few if any problems are very likely to be simply going about their lives and not bothering to search for Tecfidera info on the web or answering a blog questionnaire on the subject. This phenomena can be seen manifesting itself in the comments sections of the Tecfidera informational posts I’ve put up on WK (click here and here), which are primarily comprised of patients recounting their difficulties experiencing the side effects of the drug.

If one took these comments to be representative of the experiences of all Tecfidera patients, it would be easy to surmise that rather severe side effect reactions were the norm rather than the exception, which clearly isn’t the case. This in no way discounts the validity of the accounts of those commenting on my posts, but simply illustrates the skewed nature of much of the info you’ll come across on Internet MS forums, Facebook pages, and other online communities. Such outlets only provide a window on a subsection of the MS population, and all information and opinions gleaned from such sources, both positive and negative, needs to be viewed in that light.

Now, having said all that, I find the results of my little Internet survey to be quite interesting, especially when a bit of negative bias is taken into account. As of this writing, 557 patients had participated in the poll. Let’s have a look at the numbers.

The first question asked is, “How would you characterize the side effects you’ve experienced as a result of taking Tecfidera?” Fully 61% of those responding report experiencing few if any side effects (answering “none” or “mild”), while 23% of respondents report dealing with moderate side effects, and 14% characterize their side effects as severe.

That 14% of folks taking Tecfidera report experiencing severe side effects is concerning (representing approximately 1 in 7 patients), but, as I stated previously, I suspect these results are skewed somewhat towards the negative. Even so, the number does seem high, and is certainly higher than I expected when I first set up the poll. Still, the terminology involved is a bit subjective, in that people have different tolerances for the discomfort caused by side effects, and what one person terms “severe”, another might term “moderate”.

I think the next question demonstrates this subjectivity to an extent. When asked, “Have Tecfidera’s side effects forced you to stop taking the drug?”, 88% of those answering say no, and 11% report that they have indeed stopped taking Tecfidera due to the drug’s side effects. Since 14% reported severe side effects in the first question, it’s apparent that about 3% of those folks didn’t find their side effects to be severe enough to make them stop taking Tecfidera. This question doesn’t touch on a key element in determining whether or not a patient continues on with a drug, namely the length of time that side effects persist. It’s generally been reported that Tecfidera side effects dissipate after 6-8 weeks, so the possibility exists that some folks experiencing severe side effects might be able to stick out their discomfort long enough for those side effects to no longer be a problem.

Interestingly, the two major late stage trials of Tecfidera reported that only 2% of patients taking the drug had to stop due to intolerable side effects, the same number as those who were taking a placebo. Certainly, these trials were far larger and much more controlled than my rinky-dink little Internet survey, but the disparity in results is curious. While much of this difference can probably be attributed to the negative bias inherent in my Internet poll, it’s been reported that drug companies routinely fail to report on the results of people who drop out of their studies, a practice which, if true, is at best abhorrent. It’s been widely demonstrated that drug companies consistently suppress research results that don’t reflect positively on their products, publishing only favorable study results (a phenomenon called “publication bias”), so such charges wouldn’t surprise me. As I’ve stated time and time again, the mechanisms by which medical research is conducted and reported these days are almost completely dysfunctional, much to the detriment of patients and the doctors who treat them.

But I digress, as I can easily work myself up into a frenzy over pharmaceutical company monkeyshines. Before I start frothing at the mouth, let’s get back to looking at the results of my Tecfidera survey. The last question asked is probably the one that patients would ultimately deem to be the most important, “How would you characterize any benefits you’ve felt since starting Tecfidera therapy?”. After all, Tecfidera’s ability to control MS symptoms is the ultimate arbiter of its worth to patients.

Before delving into the numbers, it’s important to remember that I first posted this survey only a few months after Tecfidera was approved by the FDA, and it’s been shown that it takes about three or four months for the drug to fully take effect. Though I’ve no way of knowing this precisely, I’m sure many of those responding to the poll had not been on the drug long enough for it to reach peak effectiveness. Given that fact, I think the results conveyed are fairly impressive. Nearly 50% of respondents (46%, actually) reported experiencing some benefit from the drug, and 21% deemed those benefits to be “moderate” or “great”.

Since many of those responding did so within their first few months of taking Tecfidera, the fact that nearly half had already experienced some benefit is encouraging. Tecfidera affects the body on a number of levels: as an immunosuppressant, an anti-inflammatory agent, and as a potentially powerful antioxidant. Given this variety of mechanisms, one would think that the benefits of Tecfidera might increase with time, and indeed this is what the published research on Tecfidera has shown. It would be very interesting to conduct a poll that queried only folks who had been on Tecfidera for five months or longer, but given the vagaries of the Internet there’d really be no way to accurately capture such a population, at least not with the relatively simple tools currently at my disposal.

So, there you have it, my attempt at playing master researcher, and reliving my youthful sojourn into the world of quantitative research, when Dukakis battled the senior Bush for the Presidency of the United States (it really didn't turn out to be much of a battle, actually). I hope these survey results, and my analysis of them, are helpful to folks currently taking, or who are considering taking, this increasingly popular new drug.

♦ Wow, going over the Tecfidera poll results took a lot longer than I was expecting, so I guess rather than this post being a “Bits and Pieces”, it’s going to be more of “A Bit and A Piece”. I blame the length of the poll results section on the fact that I use voice recognition software to “write” my posts, which makes it far too easy to turn verbal diarrhea into written diarrhea. My apologies.

This last item, though, is quite important. The following video was produced by the Myelin Repair Foundation, one of my favorite MS research nonprofits. It features the family members of my buddy George, known on the Internet as The Greek From Detroit, who passed away last March (click here). The video recounts his struggle with MS, and the impact it had on him and all of those around him, while also telling the story of the MRF and the incredibly important work the organization is doing.

Please watch the video, which is both extraordinarily touching and very informative. The research being done by the Myelin Repair Foundation represents one of our best chances at finding a way to repair the nervous system damage done by multiple sclerosis, and their efforts deserve the support of all MS patients and the people who love them.

And to my friend The Greek, I miss you, pal…



Thanks again for all of the get well wishes, they were appreciated beyond words…

$150,000 in My Fridge, and I Feel like Crap

Okay, to be completely accurate, about four weeks ago I did have $150,000 in my fridge, but now I

only have about $100,000 sitting next to my butter dish. And that small fortune has done nothing but make me feel, at times, worse than I’ve ever felt in my life.

So, how did I acquire such a windfall? Did I find a bag of money in Central Park? No. Did I win the lottery (again)? Nyet. Did I slam my wheelchair into the shins of a Wall Street demonoid (the streets of NYC are lousy with them) and steal his pocket change? Nope. All I did was take delivery of five tiny vials of a medication called Acthar Gel. Each of those vials costs about $30,000, and the stuff in those vials did nothing but make me feel vile. Harrumph. To make the story even more interesting – and disgusting – just about 15 years or so ago those same five vials could be had for about $250. So, this is not only a story of my medical misadventures, but also one of pharmaceutical company shenanigans, which I’ll get into later.

Before I start my tale of woe, let me first state that my reaction to Acthar Gel is entirely atypical, so strange that my physicians are completely confounded as to what went down. The stuff is generally considered quite benign, and the root of my problems with it is my ever baffling, completely fracked up physiology, which the best minds in the business have not been able to figure out. So please don’t use my experience as any kind of example. If you are currently using Acthar Gel, or at some time in the future may be prescribed the stuff, I can just about guarantee that your experience will bear no resemblance whatsoever to mine. The substance is almost universally well-tolerated, and has been used by thousands of patients suffering from a variety of maladies with very few complications. This essay shouldn’t be seen as an indictment of Acthar Gel, but rather a testament to the depth of the medical mystery that is me.

Acthar Gel is simply an injectable, time released form of ACTH, a hormone naturally secreted by the pituitary gland that signals the adrenal glands to produce an increased amount of the body’s natural steroids. When used to treat MS, it’s usually given to RRMS patients who are experiencing an exacerbation, just as intravenous steroids are used to treat MS relapses. In both cases, the steroids – whether generated by the body itself or given intravenously – work in a variety of ways, primarily by suppressing the immune system and reducing inflammation in the central nervous system.

I definitely do not have RRMS, but my illness does look a lot like PPMS (Primary Progressive Multiple Sclerosis), a form of MS that afflicts about 10% of the MS population and is defined by its complete lack of relapses and remissions. Its sufferers instead experience a steady decline in neurologic function over the entire course of their illness. Though my symptoms and disease history do at first appear somewhat typical of PPMS, my diagnostic test results and certain other anomalous features of my disease don’t fit the PPMS mold. In addition to my neurologic problems, I have signs of some kind of systemic autoimmune activity, including widespread endocrine dysfunction. In other words, my hormones are a mess. If you’re interested, you can read more about my medical eccentricities in an older WK essay (click here).

Since people with PPMS generally don’t exhibit much inflammation in their central nervous system, steroids typically have little if any positive effect on them. This is one of the aspects in which my disease seems to differ from PPMS. Back in 2006, about three years after my diagnosis, my disease started to spiral out of control. In an attempt to put the brakes on my deteriorating condition, my neurologist ordered a 10 day course of IV Solu-Medrol, along with a weeklong stay in a highly regarded rehab hospital.

Remarkably, the steroids had an almost miraculous effect on me, restoring physical function that had been lost for months or years. I could suddenly use my right hand again, lift my right arm over my head, and my walking improved dramatically. Unfortunately, these improvements proved to be only temporary, fading within four months of my infusions. Even more unfortunately, I developed a very rare side effect of intravenous steroids called Avascular Necrosis (AVN), a condition which causes the bones in some of the major joints to literally die and then crumble. The AVN attacked both of my shoulders and hips. These days I’m living with the equivalent of two broken hips, and shoulders that are cranky at best. If I were healthier, I’d have had my hips replaced years ago, but in my current state it’s doubtful I’d be able to withstand the surgeries or do the necessary rehab afterwards.

As a result of the AVN disaster, doing intravenous steroids is obviously no longer an option for me, which is unfortunate because steroids are the only treatment I’ve tried which has significantly impacted my disease in any positive manner. That’s where the Acthar Gel comes in. By stimulating my body to produce more of its own natural steroids, it was expected that I would get at least some of the benefits of intravenous steroid treatment without incurring the risk of furthering my Avascular Necrosis. The solution seemed perfect, except for the exorbitant price of the drug, $30,000 for a tiny 5 mL vial, of which I’d need five. Surprisingly, my insurance company didn’t balk at the hefty price tag, and a few weeks after my doctor first mentioned my trying Acthar Gel, I received five little very valuable vials of Acthar Gel via Fed Ex.

Acthar Gel is an injectable drug, and the day after we received the stuff, a nurse came to instruct my wife Karen how to administer the injections. The plan was to do three weeks of injections, with the doses descending each week. The nurse herself gave me the first injection, and things seemed to go smoothly enough. Karen then gave me an injection each evening, and for the first few days I felt similar to the way I had felt on intravenous steroids. Kind of speedy, a little bit agitated, trouble sleeping, but nothing too terrible. I didn’t experience any obvious benefit, but we were just starting on the three-week course prescribed by my doctor.

After the fourth or fifth day, though, I started feeling pretty crummy. I developed a fever (I seem to always run a fever, another strange feature of my disease, but now the fevers were higher), my vision became blurry, my neurologic symptoms worsened, and I generally started feeling like the proverbial wet dishrag. I consulted with my doctors, and the general consensus was that my body was just adjusting to the Acthar Gel, albeit a bit strangely, and there was nothing to worry about. A few days later, much to my chagrin, I found myself feeling just about worse than I’ve ever felt in my life, barely able to get out of bed. I was told to stop taking the Acthar Gel after one more injection at a reduced dose, so as not to shock my body by stopping the stuff completely cold.

I hoped that after coming off the drug, my body would bounce back, and I’d get back to my good old, bad old normal. Instead, a couple of days later I was feeling worse than ever, and began experiencing a most inconvenient symptom. To put it bluntly, I started peeing more than I thought a human being could ever possibly pee. Really, it was incredible. For a few days, I became a living fountain of urine, a perpetual passer of piss, the mysterious source of my own Yellow River. This soggy experience only increased the misery of the situation, and lent an air of the surreal to the whole affair. I managed to stop urinating long enough to make it to my endocrinologist, who did a series of blood tests that came back, surprisingly, fairly normal. Unfortunately, I felt decidedly not normal, but expected that I would soon start feeling better. My expectations, however, were not met.

Now, about four weeks later, I’m still feeling like I kissed the “A” Train between stops (a very New York centric reference, I know. Just use your imagination). As part of my overall endocrine dysfunction, my cortisol levels generally run low, but now they are really low. I’m weak, get dizzy whenever I try to stand up, have absolutely no appetite or energy, and I’m spending the vast majority of my time in bed, and I hate spending non-sleeping time in bed. Suffice it to say, I’ve definitely seen better days.

To think, this dreadful experience only cost my insurance company $150,000, and I have about $100,000 worth of Acthar Gel sitting in my fridge, totally useless to me or anybody else, since it’s illegal to transfer a prescription drug, once delivered, to another patient. I don’t think the stuff has any street value, or I’d send Karen out in a trench coa to t try to sell the stuff to some desperate Acthar addicts. With the profits we could put a 10% down payment on a really small one-bedroom condominium here in NYC.

So, that’s the story behind the lack of new posts on Wheelchair Kamikaze for the last month. I’d like to thank all of my loyal readers for sticking with me. I’m desperately hoping that I’ll be feeling better soon enough, and be able to get back to my fairly loose schedule of regaling the Internet with riveting tales of life with a chronic progressive disabling illness, my take on latest in MS research news, and snippets of the random crap that regularly pops into my noggin.

Before I sign off, a few words about the outrageous price of Acthar Gel. As I mentioned numerous times above, a single small vial of the stuff now sells for about $30,000. One might imagine, then, that it must be some new high-tech compound that cost the drug company that manufactures and sells it, Questcor Pharmaceuticals, millions and millions of dollars to invent, develop, and bring to market. Wrong. The history of Acthar Gel dates back to the 1950s, when the Armour meat packing company was trying to find uses for the animal parts that didn’t make it into the food chain. They discovered that hormones extracted from pig pituitary glands could be used in humans to treat a variety of illnesses. Thus, Acthar Gel was born about 60 years ago, and approved by the FDA in 1952, before the agency required clinical trials to prove a drug’s effectiveness.

For much of its long history, Acthar Gel was used to treat a variety of arthritic, autoimmune, and rheumatologic conditions, and the price of the drug was relatively inexpensive, about $50 for a 5 mL vial by the time the mid-1990s rolled around. By then science had learned to synthesize many of the steroids that Acthar gel stimulates the human body to produce, which severely limited demand for the drug. For the last few decades, Acthar Gel's primary use was to treat a condition known as infantile spasms, a rare epileptic disorder that usually strikes children before the age of one, which can sometimes be fatal. Since infantile spasms afflicts only about 800 patients a year, and Acthar Gel is relatively expensive to produce, making the drug became a losing proposition. Questcor bought the rights to the drug in 2001 for a mere $100,000. Almost immediately, they raised the price of each vial to about $1200, and then in 2007 hiked the price astronomically, to $28,000 for a single 5 mL vial of the stuff. They didn’t change the formula and didn’t refine the compound, but in six years took a cheap, rarely used, 50-year-old drug and turned it into a blockbuster generating hundreds of millions of dollars in profit per year.

How was this financial magic act achieved? All through the graces of a law called the Orphan Drug Act. In the United States, this law grants incentives and privileges to companies that manufacture drugs that treat diseases that affect 200,000 people or less. The intent of the act is to give pharmaceutical companies reason to pursue treatments for diseases so rare that research, manufacture, and marketing of drugs intended to treat them would not otherwise be profitable. Since infantile spasms afflict far less than 200,000 patients, Questcor received orphan drug status for Acthar Gel, and indeed the drug is a godsend for the little victims of that epileptic disorder.

Once a drug receives orphan drug status, though, there’s nothing to prevent the company that sells it from finding other uses for it, and that’s just what Questcor has done with Acthar Gel. Along with raising the price to $28,000 per vial in 2007, Questcor embarked in a massive marketing campaign, in an effort to convince physicians to prescribe Acthar Gel for a wide variety of conditions, including MS, nephrotic syndrome, and rheumatologic conditions. Today, the treatment of infantile spasms only makes up about 10% of the drug’s sales, and Acthar Gel produces hundreds of millions of dollars in sales per year for Questcor pharmaceuticals (in the first nine months of 2012 alone, Acthar Gel sales amounted to about $350 million). Since dramatically increasing the price of the drug in 2007, Questcor’s stock price has skyrocketed, from $.60 to about $58 today. Pretty good investment, right?

For more info on Questcor pharmaceuticals and Acthar Gel, you can read an excellent article in the New York Times (click here), from which much of the above information was derived.

In short, Questcor pharmaceuticals managed to take a rarely used and almost forgotten drug, and in the course of less than a decade turned their initial $100,000 investment into billions of dollars in profit. All this without doing any research and development, attaining incredible returns almost strictly through the magic of marketing, along with a little help from a well-meaning law meant to allow patients suffering from rare diseases at least a glimmer of hope that a treatment for their disease might be developed.

Well, after recounting the above story, I feel even sicker than I did when I started this essay. It’d be bad enough if I was made this ill by the drug at its original price, five vials of which would’ve cost $250. Knowing that the stuff cost my insurance company $150,000, well, all I can say is that I’m going to crawl back into bed as soon as I can muster the strength to leave my computer desk.

PS, all well wishes and healing thoughts will be extremely welcomed…