Thursday, December 19, 2013

'tis Better to Give… (Worthy MS Nonprofits for Holiday Giving)

English: Santa Claus as illustrated in , v. 56...

English: Santa Claus as illustrated in , v. 56, no. 1449 (1904 December 7), cover. (Photo credit: Wikipedia)
(For those who receive these posts via email, this essay contains videos which can only be viewed on the Wheelchair Kamikaze website – click here)

Yes, it’s that time of year again: the holidays are upon us. It’s incredible how they always manage to just kind of sneak up on you even though it’s impossible to miss the signs of the season, most notably the endless stream of holiday themed advertisements and TV commercials that these days start running even before Halloween.

Maybe it’s precisely because of that constant commercial bombardment with manufactured Christmas cheer that the actual day of the holiday inevitably comes as something of an anticlimactic  shock. In an effort to simply maintain our sanity, perhaps we tune out all of the faux happy holiday chatter and go into a survival mode state of denial. After all, there are only so many Mercedes, BMW, Lexus, and Audi holiday advertisements one can watch before the brain simply perceives them all as white Christmas noise. And does anybody ever actually ever get a luxury automobile for Christmas? Anybody normal, I mean, folks in the “1%” not included? Back in 1981, my mother’s boyfriend gave me his recently deceased father’s 1970 Oldsmobile, but it wasn’t for Christmas and the car needed a new transmission and left rear quarter panel. Still, at 17, I was thrilled with my first set of wheels, and in my adolescent exuberance that 11-year-old Cutlass may as well have been Santa’s sleigh complete with flying reindeer.

Whatever the reason, I always seem to find myself scrambling to order last-minute Christmas gifts for the folks on my list, even though these days my schedule isn’t exactly bursting at the seams. Thank heavens for the Internet, where even gimps can spread Christmas cheer without much fuss. With just a few clicks of the mouse, it’s ho ho holy crap, I just maxed out my credit card. But far be it from me to play the part of Scrooge, as I do derive great satisfaction in giving gifts to the ones I love. In fact, I honestly much prefer giving gifts to getting them. These days, given my limited physical capacities, there isn't really much that I need, except maybe a brand-new central nervous system, which I can only imagine would be especially hard to wrap and would make quite a mess under the tree…

In the spirit of giving I thought I’d provide a list some lesser-known MS nonprofits that would greatly benefit from the holiday largess of MS patients and those who care about them. In the world of MS nonprofits, the National Multiple Sclerosis Society is the great big hairy ape in the room. Due to its ubiquitous MS Walks, high public profile, and aggressive fundraising, the NMSS has become the face of MS to the general public. When most people think about making a donation on behalf of MS patients, it’s to the NMSS that the money flows. The National Multiple Sclerosis Society is to MS nonprofits as Kleenex is to tissues, melding in the public’s mind as one and the same.

In reality, though, there’s a plethora of worthy smaller MS nonprofit organizations out there, many of them starving for funds and some of them doing incredibly important work in the nuts and bolts research trenches that will with any luck eventually cure this damned disease. Trust me, when the cure comes it will most likely be from one of these little guys, not from the monoliths most often associated with MS research, who, though well intended, may be just a little too invested in the status quo, even if on an organizationally subconscious level. I by no means want to disparage the work being done by the NMSS, and the folks I know who work for the organization are all extremely caring and dedicated people, but there are lots of other guys in the sandbox who tend to get crowded out by the well hewn NMSS fundraising machine.

Here then are a handful of nonprofit MS research groups whose voices are all too often drowned out by the booming fundraising megaphones of larger organizations. The below groups all get the much coveted Wheelchair Kamikaze stamp of approval, and I’d urge WK readers to request that their family and friends who might be inclined to make MS related donations this holiday season to consider the following groups in lieu of some of the more obvious candidates.

♦ The Myelin Repair Foundation (click here) – As its name implies, the MRF is entirely devoted to researching strategies and methods for repairing the damage done by the MS disease process. Founded by Scott Johnson, himself a PPMS sufferer, the Myelin Repair Foundation aggressively seeks to break down the barriers that slow down medical research by encouraging collaboration rather than competition, and actively partnering with research groups and organizations pursuing the tangible goal of myelin repair and neuroregeneration.

Now maintaining its own research laboratories, the MRF has set its goal to have a therapeutic agent that repairs MS nervous system damage available to patients by 2019. That may seem like a distant date, but it’s only five years away, and for decades patients have been told that a cure for MS will be had within 10 years. So far that promise has been nothing but a lie, but I’m confident that the MRF stands an excellent chance of turning promises into reality. I’ve had the pleasure of having dinner with Scott Johnson, and I can personally attest to the drive and dedication of the man and his organization. The MRF is already making great strides towards reaching their goal, a goal that once achieved will have tremendous positive impact on each and every patient stricken with multiple sclerosis.

I’ve previously posted the below video, but it’s exceptionally well done and conveys the mission and vision of the MRF in an extremely personal and emotional fashion, while memorializing my late friend and comrade in arms, George Bokos, The Greek from Detroit. I’m featuring it once again in the hopes that readers will forward it to friends and loved ones, some of whom will hopefully choose to help the MRF achieve its audacious goal.

♦ The Tisch MS Research Center of New York (click here) – The privately funded Tisch Research Center is an integral part of the MS clinic at which I am treated. Headed by Dr. Saud Sadiq, the Tisch Center is at the cutting edge of MS research, investigating and innovating paradigm shifting methodologies for combating multiple sclerosis and repairing the damage that the disease inflicts on its victims.

In extremely exciting news, the Tisch Center recently received FDA approval to begin only the second US trial using adult stem cells to repair damaged central nervous system tissues in MS patients. Utilizing breakthroughs made through years of intense research at the Tisch Research laboratories, the clinical trial will use stem cells specifically targeted at repairing nervous system damage (neural progenitor cells) injected directly into the spines of trial subjects in an attempt to achieve the Holy Grail of MS treatment, the regeneration of cells damaged or destroyed by the MS disease process. One of the trial subjects will be noted journalist and author Richard Cohen, husband of TV personality Meredith Vieira, who, along with Dr. Sadiq recently appeared on The Dr. Oz Show to talk about this groundbreaking clinical trial. You can view clips from The Dr. Oz Show featuring the trio (click here-part one) and (click here-part two).

Dr. Sadiq is my MS neurologist, and I can testify to the man’s obsessive passion for finding the cure for MS and his deep compassion for the patients he treats. Dr. Sadiq is a bit of a maverick and a very “outside the box” thinker, a fiercely independent physician who refuses to permit pharmaceutical representatives to even enter his clinic so as to keep clear of the influence of Big Pharma. Given the frustrating and unrelenting nature of my illness, I consider myself lucky to have Dr. Sadiq on my side, as I’m confident that if anybody will come up with the answers I seek, it will be The Big Guy (as I affectionately call him). The Tisch MS Research Center of New York is certainly worthy of whatever tax-deductible donations come its way, as in addition to stem cell research the center vigorously conducts a wide range of studies specifically targeted at finding a cure for MS. Hopefully, the upcoming stem cell trial will culminate in results that will forever change the way MS is treated, and for the first time restore function pilfered by the disease.

♦ The Accelerated Cure Project (click here) – The ACP is an organization dedicated to speeding up the pace of multiple sclerosis research and treatment by engendering collaboration among researchers through a variety of mechanisms. Perhaps the most valuable resource provided by The Accelerated Cure Project is the ACP Repository (click here), a storehouse of blood and spinal fluid samples collected from over 3000 subjects, along with voluminous data on the medical and familial history of those subjects. These samples and data sets are available to scientists and organizations conducting research that can positively impact patients with MS.

The ACP Repository contains samples of Wheelchair Kamikaze blood and spinal fluid, which I understand is being kept in lead lined containers under the watchful eyes of Seal Team Six. Heaven forbid any WK derived substances fall into the hands of nefarious evildoers, as pure bedlam would be sure to follow. Labrador Retrievers would take their rightful place as the dominant species on the planet, and hordes of crazed wheelchair drivers would terrorize any who dared stand in the way.

All joking aside, The Accelerated Cure Project is devoted to the all-important goal of eradicating multiple sclerosis. Along with the ACP repository, The Accelerated Cure Project maintains The Multiple Sclerosis Discovery Forum (click here), an online community connecting, educating, and challenging MS researchers worldwide. Although intended for MS researchers, The Multiple Sclerosis Discovery Forum is a valuable resource for anybody interested in the latest MS info and research findings. Additionally, the ACP maintains the OPT-UP program (click here), a wide-ranging clinical study designed to evaluate the effectiveness of MS drugs in real-world settings, identify predictors an early indicators of response to specific drugs, and detect biomarkers specific to progressive MS.

The Accelerated Cure Project is an exceptionally important resource for MS researchers around the globe, and the organization has provided specimens and data for over 70 groundbreaking MS research studies. The biosamples, metadata, and interactive resources provided by the ACP are playing a vital role in research that could very well unlock the answers for which we as MS patients so ardently hope.

Well, there you have it, three smaller MS nonprofits all well deserving of charitable donations this holiday season (or any season, for that matter).

Please allow me to wish Wheelchair Kamikaze readers a very Merry Christmas, a happy belated Hanukkah, a tremendously good Kwanzaa, a happy Festivus (for the rest of us), or just a particularly good couple of weeks for those who don’t celebrate any of the aforementioned holidays. And of course, may all of us have a New Year filled with abundant health, happiness, and laughter.

As Charles Dickens wrote in A Christmas Carol, “It is a fair, even-handed, noble adjustment of things, that while there is infection in disease and sorrow, there is nothing in the world so irresistibly contagious as laughter and good humour.”

Amen to that…

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Saturday, December 7, 2013

How Are You?

English: "Three Gentlemen Greeting Each O...

English: "Three Gentlemen Greeting Each Other" (Photo credit: Wikipedia)
How are you?

I’ve always hated that question. It’s asked countless times a day, most often in passing as part of a quick and breezy greeting. “Hey, how are you”, says one person acknowledging another, typically with all the sincerity of a society lady blaming a loud fart on the dog. For that very reason – that lack of any real authenticity behind it – I’ve long disliked this social grace, even back when I was healthy. The standard response to “how are you” is almost always “good” or “great” regardless of whether or not things are good or great. Sometimes the question is met with a tired attempt at sarcasm, “same shit, different day…”, or some equally worn-out rejoinder.

Now that I’m stuck with a chronic debilitating illness, I find this most frivolous of salutations especially grating. I’m sitting here half paralyzed with my ass firmly planted in a wheelchair, how the hell do you think I am? Footloose and fancy free?

Of course, one’s condition at any particular moment is all relative, and despite the fact that I am indeed sitting here half paralyzed with my ass firmly planted in a wheelchair there are naturally times that I am better off than others, but all in all my situation, even when broken down to its most basic elements, is a complex matter. Coming up with a response that summarizes my current state in just a few words is a virtual impossibility, and even on those occasions when I happen to be feeling pretty good or even great, a quick “good” or “great” just doesn’t seem sufficient to capture the manifold nuances of the situation. My usual response these days is a tepid “oh, I’m hanging in there, I guess”, but more and more, when I’m feeling particularly grumpy I find myself tempted to pin my interrogator against a wall with my 300 pound wheelchair and proceed to let them know precisely how I am.

So how am I?

Physically, I’m a wreck. My right arm and leg are completely useless, most often more hindrance than help, and my left side is gradually (but not gradually enough) spiraling the drain as well. Due to a rare and disastrous reaction to intravenous steroids called avascular necrosis, my hips and shoulders are quite literally broken, keeping me in a nearly constant state of pain. That very same pain keeps me from sleeping for more than an hour or two at a time before whatever position I’ve dozed off in becomes so excruciating it wakes me, so I’m sleep deprived and constantly exhausted. Whatever disease process has hacked away at my spinal cord has also taken a buzz saw to my endocrine system, sending my hormones completely out of whack, the physical impact of which can be debilitating, setting up a medical situation so complicated it’s proving almost impossible to untangle. For reasons unknown I get fevers almost every night, which don't play well with my extreme heat sensitivity. Other than that I’m as fine a specimen of human physiology as can be found on God’s green earth.

How am I?

Emotionally, hard as it may be to believe, I’m in a better place now than I was for most of the time I was healthy. Back then I was often wildly neurotic, angst ridden, and for reasons only my decades long list of therapists may ever know, perpetually intent on finding reasons to be miserable. Not that I was friendless or socially isolated, for despite all of my psychological foibles I managed to maintain a boyishness and wield a sarcastic wit, the combination of which came together to form a certain kind of charm (or was my thriving social life due to the fact that I developed the habit of pinning cash to my garments?). By and large, though, I was almost always in a constant state of discontent, an emotional expanse whose boundaries ranged from mild trepidation to downright anguish. Though I was always quick to laugh, quite often at myself, nasty little gremlins were always flitting around inside my brain, twiddling with knobs, switches, and dials labeled insecurity, anxiety, and self-doubt.

Now, physically saddled with a mysterious and chronic progressively debilitating disease, most of my old psychological kinks have somehow been vanquished. Not that I’ve become the poster boy for robust mental health, but it seems that having a genuinely horrendous problem to deal with has negated any pathological need I had for seeking out reasons to be anxious. Now that my overactive psychology has been given a tangible target on which to focus, albeit a dreadful one, I find my anxieties have quieted down considerably. There’s not much time or energy to be wasted on existential angst when your entire right side is doing its best impersonation of a mannequin. Not saying my situation doesn’t suck, as it sucks huge hairy monkey balls, but it definitely keeps me grounded in reality, and has given me reason to channel my energies towards self-preservation rather than self-doubt.

How am I?

Frightened. Nah, that’s too tame a word. Let’s try absolutely, completely, thoroughly scared shitless. That’s more like it. How does this omnipresent state of fear wash with my aforementioned newfound mental stability? Very easily; anybody who wouldn’t be scared shitless at the future prospects presented by a constantly progressing crippling illness would have to be considered a prime candidate for Basket Weaving 101 at an inpatient mental facility. Try as I might to stay centered and focused on the now, it’s almost impossible to completely shield oneself from glimpses of a potentially horrific future. And we’re not talking about an abstract threat here, as the reality of the situation has been personified by some of the most famous victims of the disease (Annette Funicello, Richard Pryor, etc.). Can there be any prospective future more dreadful than the real possibility of being reduced to a very alert brain miserably serving a tortured life sentence silently trapped inside a prison of completely useless flesh and bone? Enough said. I’d better stop now before I soil myself.

How am I?

Dauntless. Since my diagnosis almost 11 years ago, I’ve endured trials and tribulations that my former hypochondriacally neurotic self would never have imagined I could withstand – much less simply shrug off, as I have most of them. I’ve been poked, prodded, irradiated, pounded and punctured more times than I can count, all with nary a yelp of protest or consternation. I’ve been PET scanned, CT scanned, gallium scanned, and MRI scanned so many times that I now possess far more pictures of the inside of me than the outside of me. I’ve racked up well over a dozen spinal taps, had needles the size of fire hoses simultaneously stuck into the veins in both arms during plasmapheresis, and have orally and intravenously ingested all kinds of bizarre and potentially poisonous concoctions dressed up as medicines, the vast majority of which have been as effective as voodoo powder (maybe less so, as I haven’t yet tried voodoo powder). I’ve watched more and more and more of my body be transformed from vital to useless over the course of a short decade as this creeping paralysis has done its dirty work, enduring ever mounting indignities as the endless losses have piled up, and yet I’ve somehow been successful in fending off the impulse to call it a life and permanently take up the fetal position. Big ups to the powers of mindfulness and the teachings of the ancient Eastern philosophers.

How am I?

Angry. Pissed off at the universe for cursing me with this vexation, derailing my life just when it seemed I’d learned some hard lessons and things were finally going my way. Despite my inner demons I found myself a great girl and forged a successful career in a highly competitive industry, only to watch it all come crashing down around me. Well, all except the great girl, who’s so great that she miraculously hasn’t headed for the hills. I’m furious at modern medicine, which despite all of its whizbang technology and blaring headlines of paradigm shifting medical advances is left dumbfounded and rendered completely impotent by dozens of horrific maladies, the vast majority of which leave those whose job it is to cure them not knowing their asses from their elbows. I’m seething at so-called healers who are content to label diseases “autoimmune”, when it’s clear that an immune system gone haywire is a symptom of some much greater ill. Newsflash: we haven’t evolved over millions of years to simply have our immune systems wake up one day and decide, “fuck it, I think I’ll go rogue and just attack this son of a bitch”. I’m infuriated at a medical research model that has become so corrupted by the corrosive influence of big money that it’s completely lost sight of its primary objective: finding ways to eradicate diseases, not fancy new formulas for turning them into cash cows.

How am I?

Grateful. Despite all the terrors of and the destruction wrought by my disease, I’m cognizant that in its own twisted way getting sick was my ticket to freedom, and maybe even to a dash of wisdom. Yes, I’d found success in a “glamour” industry, but truth be told I always hated working. Though the positions I held required varying degrees of creativity, many of them found me stuck in buttoned-down corporate environments, the kind of places that the younger, more idealistic me proclaimed loudly to all who would listen that I’d never wind up. Some people flourish in such a business environment; I suffocated. I found the regimentation of the 9 to 5 lifestyle absolutely soul sucking, asphyxiating my spirit and smothering my passions. I somehow allowed myself to stray far from the path I had once sworn to follow, and found myself too trapped by the realities of adult responsibilities and my own fears to find my way back. My disease, or more precisely, the early “retirement” it forced upon me, turned out to be my emancipation.

Some find the transition to life on long-term disability nearly impossible. Not me. I took to this relative life of leisure as if I were to the manor born. I suddenly had the time to rekindle interests and appetites that had far too long been neglected; writing, photography, history, zombie movies, ancient aliens. Perhaps more importantly (but really, what could be more important than zombies and ancient aliens) I was afforded the perspective to examine the healthy life I was forced to shed, and thus come to fully understand just how trivial were most of the concerns that had previously tied me up in knots. Worries over relatively minor financial setbacks, social status, broken romances with the wrong people, fixations on material goods – all so pathetically frivolous in the face of the disease that now beset me. The Eastern philosophies I'd  read but had never been quite able to incorporate into my healthy daily life now became vital instruments of survival. That Siddhartha wasn't just whistling Dixie.

Perhaps the most important thing I've learned is that the greatest blessing on earth is a quiet night spent with the people you love who love you back (this includes dogs).

So, how am I?

Oh, I’m hanging in there, I guess…

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Friday, November 22, 2013

Bits and Pieces: Tecfidera Poll Results Edition

Just heard from my dr office.  I will be start...
(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…

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Friday, November 8, 2013

$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…

Thursday, October 24, 2013

At What Price Health? (Repost)

Well, I'm still in recovery mode from my last failed attempt at trying a new MS med. For all of those who have inquired, no, the medication was not Tecfidera, so if you have recently started the drug, no cause for alarm. The med I tried is not used all that often to treat MS, and I had a very atypical reaction to it (naturally). I'll provide full details in my next post, which I will put up as soon as I'm feeling better. Just a teaser to keep you interested: the drug costs $150,000 for a three-week supply, and I was only able to use about one week's worth. So, I have about $100,000 sitting in my refrigerator, just taking up space and doing nothing to help anybody. Way to go, Medical Industrial Complex!

In the meantime, I'm reposting the following piece, which first appeared on Wheelchair Kamikaze way back in 2009. It generated lots of comments back then, and I hope current WK readers find it just as interesting. Please stay tuned for a new post, coming just as soon as I'm feeling up to it…

For the last few days, I've been pondering a thought exercise that recently popped into my mind. Imagine, for a moment, that an almost miraculous cure for MS has been discovered, one that can alleviate all MS symptoms with a single injection. A patient simply has to go to their doctor's office, get the shot, and, voilĂ , 24 hours later they are completely symptom-free, their nervous systems restored to pristine condition and their general state of well-being suddenly better than even before they were diagnosed with Multiple Sclerosis.

Great, right? Sign me up!

Only, there's a catch. This "cure" comes with a terrible cost: after a certain amount of time, every patient treated with this injection dies painlessly in their sleep. In the "X" amount of time before they die, the patient remains in the full bloom of health, right up until the night they go to bed for the final time. The question, then, is what would be the minimum duration of guaranteed health that would entice you to take the shot? In other words, would being restored to perfect health be worth it to you if you knew with utter certainty that you would die in six months? One year? Five years? 10 years?

Would you be willing to trade a full life of chronic illness for a blissful time during which you would be completely unshackled by the chains of Multiple Sclerosis? For a time free of fatigue and cognitive dysfunction, of muscle spasms, spasticity and paralysis , of bladder and bowel issues, of the constant daily struggle of dealing with the rigors of this miserable disease? An interval during which you'd have no reason to even think about braces and canes and walkers and wheelchairs and MRIs and neurologists and lesions and a medicine chest full of pills that hardly even seem to matter? When you could walk and run and dance (dance!), drive and play and travel, and finally, finally, once again be that fully functional man or woman that you used to be, that you've dreamed of being since the day MS started taking its dreadful toll?

How many months or years of restored health would be enough to entice you to undergo a simple but profoundly effective treatment that carried with it the ultimate price? Of course, the answer must differ for each of us, based on our own current state of disability, our rate of progression, the level of our misery, and the amount of hope we have that a cure, or even a truly effective treatment, can be found in time to help us. The flavor of the disease a patient suffers from also enters into the calculus . Folks with relapsing remitting disease that is currently being managed effectively by disease modifying drugs might reject such a proposition out right, while patients with progressive disease, especially advanced progressive disease, would probably be much more open to trading longevity for a period of perfect health.

Certainly, marital status and family situations factor greatly into the equation. Single people, or those without children, might be more willing to sacrifice longevity for a chance, though brief, to be healthy once again. For those who are married, and especially those with children, the calculus gets infinitely more complicated. How much time with a healthy parent would it be worth for a child to then lose that parent? Difficult questions all, and ones I think reach to the very core of our beings.

Personally, after much thought, I think I'd put my "X" at somewhere around a year or a year and a half. If a physician approached me with a syringe, and told me that the injection would guarantee me 12 months of perfect health, but at the end of that 12 months, I would die painlessly in my sleep, I would give the offer serious consideration. Of course, I'd want more time, all the time in the universe, but this thought exercise requires that we consider the absolute minimum amount of time we would settle for.

One year would give me time enough to experience all of those pleasures in life that I now miss so terribly, to travel to the places my wife and I have always wanted to see together, and to spend time with those who I hold closest to my heart. I don't have children, so that's not a consideration. I do have hope that stem cells offer real promise as a treatment, but I'm unsure that this promise will be fulfilled in time to help me. I have my doubts about many of the avenues currently being explored by MS researchers, and though strides are being made, I'm uncertain that the mysteries of MS will be fully unraveled anytime soon, and given my rate of progression, soon is the only timeframe that really matters to me. In addition, there is now question as to whether what I have is even really MS, and what chance is there that some mystery illness will be solved before it puts me into a state I deem to be simply unbearable?

So what about you, dear reader? What's the minimum amount of time for which you'd be willing to trade your life for perfect health? What's your "X"?

Tuesday, October 8, 2013

Remembering Stella (Repost)

(Some good news and some bad news. First, the bad news. After trying a new drug (for me) to calm my mysterious  beast, I am instead caught in the throes of a struggle with it. Weaning off the drug now, so hopefully I'll be back to my "old normal" sooner rather than later. The good news is that all this should make for a pretty interesting blog post, including some outrageous pharmaceutical company shenanigans, the mysteries of my illness, and general adventures in medicine.. In the meantime, for your hopeful enjoyment, I'm reposting the below essay, written in January, 2010 about one of the best friends I've ever had. Thanks for reading, and a new essay will hopefully be up soon…)

My best buddy Stella passed away just a little over three years ago. She was a faithful friend with a huge heart who always knew just how to make me smile and often had me laughing riotously out loud. Stella was compassionate and sweet, and knew exactly how to live in the moment and seize every day. She was faithful, devoted and I knew that I could trust her entirely with my deepest darkest secrets. About the worst thing I can say about her is that she had the unshakable habit of loudly and vigorously chewing her paws in the middle of the night, while emitting strange noises that were impossible to sleep through.

stella%20action%20cu[1]Stella was, of course, my furry best friend, a yellow Labrador Retriever who came into my life in 1998, while I was still living in Fort Lauderdale. A coworker had just given birth to a baby girl and no longer had the time to care for Stella. I'd recently moved into a charming little 1940s Florida cottage with my then girlfriend, and was jonesing for a dog. So, the timing was perfect, and after two "meet and greets", during which Stella gave me the thumbs up, I was a happy new doggy daddy. Stella had just turned three years old when she came to me, and I was five years away from developing MS.

I hadn't had my own dog since I was a kid, but I had bonded with the canine companions of several friends and lovers that I'd met along the journey of my adult life. I was particularly close with a Dalmatian named Briar, whose owner unfortunately turned out to be a pathologically lying serial cheater who delighted in using my heart like a roll of Charmin. Quick life lesson: if you find out that your lover has cheated on every person they've ever been with, don't fool yourself into thinking you can somehow change them. Simply open your eyes to the truth, realize that once a person accepts such behaviors in themselves they will never change, and get as far away as possible, even if they have an adorable spotted four-legged creature with the most haunting eyes you've ever seen...

But, I digress. Stella and I quickly bonded, even as my girlfriend and I quickly unbonded. Turned out my Labrador friend enjoyed spending Sundays sprawled on the couch watching NFL games as much as I did, as long she could watch them while laying between my legs with her head nestled on my belly. We took long walks around the neighborhood together, although she wasn't much for jogging. The one time I took her out for a run, she made it about a block before squatting in the middle of the road and doing what dogs generally do when they squat. After completing that most natural of acts, Stella let me know that jogging just wasn't her thing. No harm, really, because jogging wasn't really my thing, either.

About six months after Stella joined me, the girlfriend and I decided to call it quits, and I decided to get the hell out of Florida, a place I never much cared for, even though I spent 10 years there. I think the tropical sun beating on your noggin causes some kind of dementia, because even though I felt like a stranger in a strange land the entire time I lived down there, for some reason I could never formulate actionable plans to leave. It was like, "gee, this place royally sucks, ooh, I think I'll go for a swim..."

Anyway, Stella and I were soon back in my hometown of New York City, living in a section of the city known affectionately as "Hells Kitchen". For a dog that was born and raised in Florida, Stella took to city life like a socialite. For some reason, she naturally curbed herself (if only the same could be said for socialites), and she loved the wonderful sniffing opportunities that the city streets offered up in droves. She also loved the take-out Chinese joint around the corner from our apartment, which always had partially eaten chicken wings discarded on the sidewalk in front of it. One of the few arguments Stella and I ever had were over her insistence on insanely gobbling down as many of those gnawed on chicken bones as quickly as she possibly could, but a few rounds of very stern "bad girls" helped her kick the habit. You see, she really was a "good girl", and my disapproval trumped the irresistible gristly remains of chicken wings, true testament of her feelings for me.

For about a year, Stella and I were strictly a duo, spending lots of time at neighborhood dog runs and in Central Park, where she'd occasionally take an ecstatic jump in the lake. She absolutely lost her mind during that winter's first snowfall, which was the first snow the native Floridian had ever seen. If pure joy could be embodied in flesh and blood, it would be Stella burying herself in mounds of freshly fallen snow and then wriggling on her back to make canine snow angels. Her glee was infectious, and soon I too was a snow-covered whirling dervish, joining Stella in her carefree frolicking, covered head to toe in the powdery white stuff blanketing the fields of Central Park.

After about a year back in the city, late one night in a neighborhood bar I met a girl named Karen, who, despite my best efforts, didn't seem very interested until I mentioned the fact that I had to get home to walk my Labrador Retriever. Turned out that Karen had grown up with Labradors, and, figuring that a single guy with a Labrador couldn't be all that bad, she gave me her number. Just about two years later, we were married. At first, Stella didn't exactly welcome Karen with open paws; after all, Karen had supplanted her place on the couch. But the two soon became buddies, and Karen even succeeded in getting Stella to lose a little weight (for a while, we referred to her as "Jabba the Pup"), much to the veterinarians delight.

For a year, everything was hunky dory, until one very cold day in March 2003, when I took Stella for a very long walk along the Hudson River. About 2 miles into our trek, I noticed that I'd started limping. I didn't think much of it, but in the following weeks, the limp in my right leg grew worse, and I felt my right arm starting to weaken. Several doctors visits and an MRI later, and I found myself sitting in a doctor's office listening to words like "multiple sclerosis" and "progression" and "spinal tap" somehow become associated with the words "me" and "holy shit".

Strangely enough, just about the same time, Stella also started having all kinds of health problems. I honestly believe that she was so empathic that she somehow shared my distress and manifested physical illnesses of her own. Between 2003 and 2006, Stella developed mast cell cancer and autoimmune hepatitis. She had multiple surgeries to get rid of the cancer, and was put on a variety of medications and a special diet to address the hepatitis. For a while, we were actually both on the same immunosuppressants, bought from the same pharmacist. On several occasions, it appeared that Stella was on death's door, but she always managed to somehow pull through, often to the veterinarian's surprise. He'd smile, shrug his shoulders, and offer the only explanation he could, "She's Stella..."

Through it all, Stella stayed Stella. Though she would suffer a while from her painful surgeries, and the hepatitis would sometimes rob her of strength and appetite, as soon as she felt a little bit better, her tail was wagging, her eyes were bright, and she was ready to embrace whatever joy that the day had to offer. In so many ways, she taught me how to deal with my own illness, which progressed continuously through the ensuing years.

Stella didn't waste any time bemoaning her fate, or thinking about what might have been, because she was blessed to simply not have the capacity to do so. As my condition has continued to worsen, I've often thought of Stella, and have realized just how right she had it. Feeling sorry for yourself or worrying about future calamity only serve to poison the present, and the present, the now, and our place in it, is the only thing in the entire universe that we have any real control over. Endeavor to live your life like a Labrador, attack each day like it's a great big rawhide bone sent from the heavens.

Eventually, Stella's illnesses and advancing years got the upper hand; the cancer returned, and my sweet little girl started slipping away. Over the Thanksgiving weekend of 2006 we boarded her at the veterinarians while we visited my mom in Florida, and when we returned the vet told us that Stella's condition had worsened, and he recommended we put her down. He brought her out to us with an IV already inserted into her leg, but upon seeing us I could see that familiar spark in her eye, and she started eating the treats I tried to hand feed her. We decided to bring Stella home, to give her the chance to make one more rally.

By this time I was no longer able to walk Stella, and most of her caregiving fell to Karen. Stella actually did rebound a bit for the first few weeks, but I guess the power of love can only go so far. A few days after Christmas, we brought Stella back to the vet one last time, held her, and said goodbye. Those weeks between Thanksgiving and Christmas became one extra month of bonus life for Stella, during which Karen took Stella to Central Park almost every day, and Stella ate all of the chicken and turkey she wanted.

Stella saw me through many transitions; from Fort Lauderdale to New York, from single to married, from well to Multiple Sclerosis. Aside from my wife, there is no being I have ever felt closer to, or more intimate with. I miss her still, and will for the rest of my days. Karen and I now live in a building that is wheelchair friendly, but doesn't allow dogs. If I somehow beat this thing, first thing we're doing is moving out of this place and getting ourselves a great big pooch, who will take Stella’s space, but surely not her place.

Here's my favorite photo of my pal Stella...

stella door effect

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Friday, September 20, 2013

Stem Cell Treatments for Multiple Sclerosis

Stem cell division and differentiation
For patients, physicians, and researchers alike, stem cells hold the tantalizing potential of turning back the tide of multiple sclerosis, repairing damaged brains and spinal cords, and perhaps even offering something approaching a cure. There is plenty of hype surrounding stem cells, and they provide much reason for hope, but what is the reality of the current state of stem cell research for the treatment of MS?

As all patients with MS are aware, the currently available treatments do nothing to cure the disease or repair the damage that it does. At their best, today’s crop of disease modifying drugs (DMDs) quiet the disease, thereby improving the quality of life for many of the patients taking them, especially those suffering from relapsing remitting multiple sclerosis. However, many of these drugs carry with them risky side effect profiles, and though the newest compounds represent advances over their predecessors, patients are crying out for revolution, not evolution.

Stem cells could represent the revolution patients so fervently desire. Because of their ability to transform into almost any type of cell in the human body, stem cells may hold the key to achieving one of the holy grails of modern medicine, the regeneration and repair of damaged tissues. For MS patients, this could potentially mean the reversal of disability, and with it the long dreamt of disposal of wheelchairs, walkers, and canes. We are still a long way from that lofty goal, however, but the first few steps along the path to that salvation are currently being taken.

Though stem cell research is advancing in laboratories worldwide, the science of using stem cells to treat diseases in humans is still in its infancy. Because multiple sclerosis is a neurodegenerative disease, and its most prominent feature is the damage the disease does to the central nervous system, it is hoped that stem cells may hold the key to reversing the carnage wrought by the disease by facilitating the repair of damaged nerve cells. Furthermore, research has provided hints that stem cells may modulate the abnormal immune response seen in MS patients, and some researchers are even using stem cells to completely reboot the human immune system, a process that in some cases appears to stop the disease dead in its tracks.

It’s important to understand that there are two very different approaches to using stem cells in the treatment of multiple sclerosis. One approach hopes to use the cells to repair damaged nervous systems; the other uses stem cells to provide the patient with a brand-new immune system, one that theoretically will not turn against a patient’s own body. The latter approach is known as hematopoietic stem cell transplant, or HSCT, and has been used on patients in trial settings for almost two decades.

HSCT involves ablating (destroying) a patient’s existing immune system through the use of powerful chemotherapy drugs, and then intravenously infusing a patient’s own stem cells back into their body, a process depicted in the below diagram:

Once infused back into a patient’s body, the stem cells go about reconstituting their immune cells, effectively providing them with a brand-new immune system that in theory shouldn’t go to war against the patient’s own brain and spinal cord. In practice, this type of therapy has proven to be quite effective, particularly among patients with aggressive relapsing remitting disease who display a high amount of inflammation in their central nervous systems, as are evidenced by enhancing lesions seen on MRI imaging.

As you might imagine, using powerful chemotherapy drugs to destroy a patient’s immune system is not without its dangers, and early attempts at this therapy had mortality rates as high as 10%. As researchers perfected their methodology and began using less dangerous chemotherapy agents, though, the risks associated with HSCT dropped dramatically. Today, most patients undergoing HSCT are subjected to chemotherapy and immunosuppressive agents that do not completely destroy their bone marrow, and the safety profile of the procedure has improved impressively. The results achieved by this HSCT can be dramatic. In one study (click here) that looked at the long-term outcomes of HSCT, after 11 years 44% of patients who had started out with aggressive relapsing remitting disease were free from disability progression. By comparison, only 10% of those who did not display signs of active inflammation before HSCT remained stable.

One of the primary proponents of HSCT therapy for MS patients, Dr. Richard Burt of Northwestern University, stresses that the proper selection of patients is the key to the success of the treatment. In fact, the title of the paper he recently published (click here) includes the phrase “if no inflammation, no response”. “It’s the only therapy to date that has been shown to reverse neurologic deficits,” said Dr. Burt, “But you have to get the right group of patients.” In a study published by Dr. Burt in 2009, 17 out of 21 relapsing remitting patients improved after HSCT, and after three years all patients were free from progression (click here). Dr. Burt is currently heading up the HALT-MS trial for HSCT (click here). There are several centers around the world offering HSCT therapy, and there is a Worldwide HSCT Facebook group (click here) that contains information on all of the legitimate HSCT facilities worldwide. The group is populated by many folks who have undergone HSCT therapy. Be aware that it’s a private group, and you must request membership before being given access to all of the available information.

While HSCT holds much promise for putting the brakes on very aggressive relapsing remitting multiple sclerosis, it unfortunately has little to offer those with progressive disease, and does nothing to directly repair the damage done to the central nervous system by MS. Fortunately, another form of stem cell therapy proposes to do just that. Researchers in two centers in the US have received FDA approval to use bone marrow derived mesenchymal stem cells (MSCs) to repair nervous system damage, thereby possibly reversing the effects of the disease. There are additional trials using MSCs to treat MS underway internationally. Mesenchymal stem cells have the ability to transform (differentiate) into many different cell types, and could prove to be the building blocks necessary for repairing damage to the central nervous system as well as other organs and tissues. Experiments using MSCs to treat animal models of MS have been very encouraging (click here), demonstrating the cells’ abilities to modulate the immune system and spur the repair of damaged nervous system tissues. It remains to be seen whether the same effects can be achieved when using the cells to treat human beings.

The two FDA approved studies both use MSCs harvested from a patient’s own bone marrow, but employ them in very different ways. One study, currently underway at the Cleveland Clinic (click here), infuses mesenchymal stem cells intravenously into the patient, in the expectation that the cells will modulate the immune system and also initiate the regeneration of damaged tissues in the central nervous system. This study, which will eventually use MSCs to treat 24 patients, is proceeding slowly, but as the above linked to article details, one of the first patients treated is already reporting encouraging results.

The second FDA approved trial, to be conducted by the Tisch MS Research Center of New York (which just so happens to be my MS clinic), will use mesenchymal stem cells that have been transformed through a proprietary laboratory process into neural progenitor (NP) cells, injected directly into the spinal fluid (intrathecally)) of the patient (click here). Neural progenitor cells are a specialized type of stem cell specific to the nervous system that have the ability to transform into the various types of tissues damaged and destroyed by the MS disease process. Researchers at the Tisch Center have developed a way to get mesenchymal stem cells to differentiate into neural progenitor cells, and hope that by injecting these cells directly into the spinal fluid the NP cells will directly target the regenerative mechanisms of the central nervous system (click here). The stem cells themselves may act to repair damaged tissues, but they’ve also been shown to have the ability to recruit existing stem cells within the brain and spinal cord to jumpstart the body’s own repair mechanisms.

It’s important to remember that both of these studies represent a very different approach to stem cell therapy for MS than HSCT. The primary goal of HSCT is to reboot a patient’s immune system; HSCT does nothing to directly address the damage that has already been caused by the disease, but rather seeks to disrupt the disease process. Taking a different approach, the trials being conducted at the Cleveland Clinic and the Tisch MS Center seek to effect repairs on the damaged brains and spinal cords of MS patients, albeit through two different methodologies. HSCT and the reparative therapies being tested in the FDA trials have little in common other than the fact that they both use stem cells in an attempt to treat MS.

I’m sure that many patients reading this are aware that there are clinics in Central America, Asia, and Europe offering regenerative stem cell therapy to patients at hefty price tags. Some of these clinics aggressively market their services, and typically charge $20,000-$40,000 for a single round of stem cell therapy. Various Facebook pages, blogs, websites and posts on MS Internet forums extol the virtues of the treatments these clinics provide, often offering glowing testimonials from patients they have purportedly treated. Although I don’t want to disparage any patient relating their genuine experiences with these clinics, I’ve known several MSers that have traveled to a variety of these clinics and undergone stem cell treatments, and unfortunately none of them have experienced anything in the way of significant or lasting benefit.

I would caution anybody considering treatment in Panama, Costa Rica, Germany, India, or any of the other clinics offering stem cell therapy without any published scientific proof of the effectiveness of their treatments to think long and hard before committing substantial amounts of money for a therapy that, according to the experiences of people that I actually know, has very little chance of working. The two legitimate trials I outlined above both involve multiple treatments given over an extended period of time, using cells that have undergone lengthy (months long) processes of multiplication and/or differentiation in the laboratory before being transplanted back into the patient. Such regimens are not followed by the “pay to play” clinics; instead, they generally infuse stem cells back into the patient soon after they are harvested, and offer extremely limited, if any, follow-up care.

Additionally, some of these clinics don’t use a patient’s own stem cells for treatment, but rather umbilical cord cells, on which far less research has been done. The use of stem cells not derived from the patient themselves opens up all kinds of questions regarding safety and efficacy, as the cells are genetically different from the tissues they are meant to repair. If any of these clinics regularly achieved anything close to the number of successful outcomes that they claim, they would surely publish their results in legitimate scientific journals and reap the personal and professional accolades that would follow. Can you say Nobel Prize? Instead, they publish marketing materials and partner with travel agencies. Reason enough for skepticism. In short, let the buyer beware.

Stem cell therapy holds tremendous potential for the treatment of multiple sclerosis, and provides much reason for hope. The efficacy of HSCT for treating very aggressive relapsing remitting multiple sclerosis is well documented, and the safety of this treatment regimen has increased dramatically as practitioners have perfected the process. Regenerative stem cell therapy, of the type currently being trialed at the Cleveland Clinic and Tisch MS Research Center of New York, is still in its infancy, but is bursting with promise, possibly holding the key to repairing the damage done by multiple sclerosis and restoring function robbed by the disease. As with all new therapies, though, it is vitally important to not let hope eclipse reason, or let hype cloud judgment. We are at the dawn of a new age, and I fully believe that the use of stem cells will revolutionize the practice of medicine. Research into the use of stem cells to treat MS is quickly picking up steam, and in combination with other emerging therapies, rays of hope are finally being shone upon the disease and those afflicted with it. It’s about time, don’t you think?

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Sunday, September 1, 2013

A Half-Century on Planet Earth

Birthday cake
This past Tuesday, August 27, I joined the half-century club, celebrating my 50th birthday. It doesn’t seem all that long ago that 50 sounded pretty goddamned old, and now I somehow find myself eligible for membership in AARP (the American Association of Retired People). For someone who is still very much in touch with their inner 12-year-old, there is something a bit surreal about the fact that I am entering my sixth decade. At least now I can qualify for discounted movie tickets and hotel rooms. Huzzah.

Naturally, dealing with a progressively disabling disease for the last 10 years has impacted the way I think about age and aging. I read somewhere that inside every 50-year-old there is an 18-year-old screaming "what the hell happened?", and though I’m sure that sentiment is true for just about everybody, it’s especially resonant for those dealing with something as completely unexpected (and dreaded) as a debilitating chronic illness. Experiencing my 50th birthday while sitting in a wheelchair is definitely not something I ever pictured back when I was dreaming of rock 'n roll glory and earning a degree in film. Polishing my prolific collection of well-earned Oscars or platinum records, yes; firmly planted half paralyzed in a mechanical monster, certainly not. Better, though, to be planted in a wheelchair than planted in the ground. As Dr. Einstein said, it’s all relative.

Way back in the late summer of 1963, I was born three weeks prematurely via cesarean section, a circumstance necessitated by the type I diabetes that struck my mom while she pregnant with me. Unlike the majority of gestational diabetes cases, my mother’s never resolved, and she’s been injecting herself with insulin multiple times a day ever since. When I was surgically snatched from the womb it was discovered that my lungs were completely filled with fluid, a situation quite dire. Just a few weeks earlier, President Kennedy’s wife Jackie had given birth to a baby boy suffering from the exact same condition. Little Patrick Kennedy died two days later.

My parents were acutely aware of the tragic circumstances of the Kennedy baby’s death when the doctors informed them of my condition, stating that I had only a 50-50 chance of surviving my first 48 hours. I was whisked away and placed in an incubator before my mom even had a chance to hold me. My dad, on leave from a training stint in the National Guard, was ordered to return to his base that day, despite not knowing whether or not his firstborn would make it through the night. A grim circumstance for sure, but I came out on top of my very first scrap, beating back an early demise by fighting for my first breaths. Three days later I was out of the incubator and finally placed in my mom’s warm embrace. Such is the randomness of the universe; a President’s son dies, and an anonymous little Jewish kid in the Bronx survives. It’s nice to know that I have a history of beating the odds, and leave it to me to make such a dramatic entrance onto the stage of this great big theater of the absurd.

In a sense, getting diagnosed with MS led me to a sort of rebirth, as the course of my life was altered so dramatically as to cleave it in two. There was part one, which spanned the time from my birth until my disease put the brakes on the running narrative of my existence, around the time I was forced to “retire” and go on long-term disability. Thus started part two, a reality that grows increasingly divorced from that previous incarnation, so much so that I can now look back on part one as an entity in and of itself, a story with a beginning, middle, and end. As such, from my new and somewhat unfortunate vantage point – a view filled with unexpected perspective – I can examine my old life like a biologist probing a particularly enigmatic specimen, teasing it apart in an attempt to discover the mysteries held within. I can trace the complicated web of experiences, circumstances, decisions, and happenstance that coalesced to form the story of my life, the subtle twists of mind and fate that led me to travel one path while bypassing an infinite number of others. If I had made a different decision here or there, if I had perhaps not lingered for one more drink or to furtively admire a pretty girl, or had not allowed fears of failure and success to exert their undue influence, might the path then taken have led to an entirely different destination, or did all roads invariably lead to Rome? Was I at the helm of the ship of destiny, or at the mercy of the cosmic winds?

In steadily untangling the jumbled knot of fate and self-determination which comprised that now extinct existence, I can in retrospect readily recognize the all too many wasted moments pregnant with possibility, can identify errors great and small made along the way, and take satisfaction in the many things that went right. The one thing I cannot do is change any of it; I can roll it around and dissect it ad infinitum, but the circumstances and outcomes of my old life will always remain frozen in time, like 200 million-year-old insects visible in pieces of amber, fascinating to gaze at but impossible to resurrect.

And, now, what to make of this new incarnation, this part two, so unwanted but also filled with its own peculiar brand of wonder and surprise. Certainly, many aspects of it are excruciating: the disease itself, the gradual loss of physical function, the sheer helplessness in the face of this progressive beast that gnaws away at me, the frustrations with a medical establishment that is shockingly ill-equipped to slay it. Despite these negatives, in a bizarre twist of fate the disease has bestowed upon me a freedom few adults ever experience. I am no longer bound by the shackles of work (I guess you can tell how much I loved working), and because of this I've been granted the gift of time, most of my days spent in a manner of my own choosing. Certainly, the disease imposes limits on my menu of choices, but even within those boundaries, whose borders are ever contracting, I’ve been able to pursue long sublimated passions, passions that had fallen victim to the realities of the workaday world. Writing, photography, a fascination with science and research, a need to communicate, all of which have gratefully come together on these virtual pages, reflections of parts of me that I had almost forgotten existed.

To think that people actually read these words and appreciate my photographs, well, it just about defies belief. This part two, this second act brought about by the realization of some of my worst fears, has graced me with the privilege of making friends in faraway places, of expressing thoughts and emotions that I’m told bring comfort to many and thus bring comfort to me, of hopefully helping to empower and offer distraction to my fellow wanderers along this road that none of us would’ve ever chosen to follow . Can this curse then, at times, be seen as something other than a vulgarity? Kipling wrote that triumph and disaster are both impostors, two sides of the same coin, and a keener observation was never made.

I look back on my 50 years and acknowledge my regrets while also celebrating my achievements. I revel in the rich tapestry of experiences and episodes I was lucky enough to be part of that will always make me smile. I’ve flown in the Goodyear blimp, come face-to-face with an apparently not very hungry 10 foot bull shark while snorkeling, won $14,000 in a state lottery, hit a hole-in-one in golf. Far more important than any of those moments, though, are the friends that I’ve made along the way, a precious few that have been part of my life for decades, others that have come and gone, but all of them more dear than any fleeting moment of experience ever could be. I thank the heavens for a wife who is the sweetest soul I’ve ever known. I mourn the friends and family that have passed, from 18-year-old Kimberly, her life cut obscenely short so many years ago, to David, the smartest man I’ve ever known, to The Greek from Detroit, my comrade in arms, to my grandmother, who even at 97 years old could make me laugh like no other. I miss them all, and will for all my days.

My 50th birthday provoked in me more introspection then any of the other milestone birthdays I’ve passed along the way, none of which ever really fazed me. Being afflicted with an unrelenting illness makes pondering the future a daunting proposition, and yet within me still resides a bubbling fount of hope. One of my oldest friends once described me as the most optimistic pessimist he’d ever known, and I think he got it right. Though I can often be a glass half empty guy, I’ve always expected to find that other half glass somewhere just over the horizon.

The disease that sliced my life in two has taught me that no matter how astute you fancy yourself, you never know what’s just around the next bend, and whatever comes into view is neither good nor bad but what you make it. As any good poker player knows, the key to winning big is not the hand you’re dealt but how it's played. There is infinite wonder in the world and in the people who occupy it. In one hundred years, the world will still be here, but all of us will be gone. No sense taking yourself too seriously, then, because we are, in the end, all just ephemera. Rejoice in that notion; nothing about us is ever written in stone, except our name on a marker that we’ll never see. Even after 50 years I’m still a work in progress, and in that sense, perhaps there's still a little part of me left in that incubator I was placed in all those years ago.

Here's a great old song that expresses one of the most important keys to contentment that the past 50 years have taught me: Be thankful for what you've got… I love the photos in the video, too, which remind me of the good old/bad old New York City that I grew up in. For those who may not be aware, the subways in NYC haven't been covered in graffiti for about 25 years.

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