Tuesday, November 22, 2011

A Few Bits, a Couple of Pieces, and a Happy Thanksgiving to All…

The First Thanksgiving, painted by Jean Leon G...

With Thanksgiving week upon us, I'm going to keep this one relatively short. Stay tuned, though, as next week I plan on posting a review of the latest in MS research, as there's been lots of important info released over the past six weeks or so. I'll cover the latest news on CCSVI, disease modifying drugs, and other MS research findings, along with the usual few dollops of opinion tossed in.

Please, don't let the inevitable tremendous anticipation of next week's post distract you from enjoying the holiday festivities. As difficult as it may be, try to stay focused on the turkey.

For those readers outside of the United States, who don't celebrate Thanksgiving this week, you'll just have to find something else to occupy your time. My European friends can busy themselves with happy thoughts about figuring out how to survive the impending collapse of the continent's economic system. Actually, we all can probably occupy our minds with such thoughts, as things aren't so hot in the US or Asia either.

WK readers in Canada, whose relatively sane economic policies have spared that country much of the turmoil roiling the rest of the world, will have to find some other distraction. Since Canadian Thanksgiving was last month, perhaps thoughts of hockey will have to suffice, or finding new and entertaining uses for maple syrup. It'll be hard to beat Sortilege (click here), though, which for the uninitiated is some pretty strong hooch made with maple syrup. BTW, kudos on the Canadian national anthem. As far as national anthems go, "Oh Canada" kicks major booty…

Okay, I said I was going to keep this short, and it's already getting long, so on with the show…

♦ A neuroimmunology researcher from the Scripps Institute sent me a note alerting me to an online petition she has started (click here), urging the US government to devote more funds to basic biomedical research at the National Institutes of Health.
I agree with her sentiments wholeheartedly, as the NIH is a precious resource that can't be left to wither on the vine, and is one of the last bastions of unbiased large-scale medical research left in the USA. The vast majority of our medical research is funded by for-profit pharmaceutical and medical device companies, who naturally devote their hard-earned bucks to research that stands a chance of turning them a huge profit. Don't get me wrong, I'm a big fan of capitalism, but money and medicine often make for terrible bedfellows. Even in this time of looming budget deficits, squeezing the NIH of funds will do nothing to solve our economic problems. Consider the following numbers:

For 2011 budget, U.S. spending on:
Social security was $2564 per citizen (20.8% of the budget)
Defense was $2203 per citizen (18% of the budget)
Medicare was $1569 per citizen (12.8% of the budget)
Medicaid was $1172 per citizen (7.8% of the budget)
NIH was $99 per citizen (0.8% of the budget)

Certainly, the funding of medical research is one of the last places we should be looking for savings. Finding newer and more effective treatments and even cures for dread diseases would pay huge dividends in the long run, both in human capital and reduced health care costs. I would think this issue is something that rational citizens across the political spectrum should be able to agree on. So please sign the petition…

♦ I recently came across an interesting new medical information website, called Medify.com (click here). The site offers links to lots of research abstracts and papers, along with patient to patient communities. A wonderful source of medical info, some of it otherwise hard-to-find, well worth checking out.

♦ Saving the best bit of medical info for last, here's some extremely exciting news: one of the nation's first multiple sclerosis stem cell therapy trials has been given the green light (click here), and should be soon underway. The Multiple Sclerosis Resource Center of New York (MSRCNY), in conjunction with the International Cellular Medicine Society (ICMS), will be conducting a 20 subject trial on patients with a definitive diagnosis of progressive MS, using mesenchymal stem cell derived neural progenitor cells, harvested from the patients' bone marrow, in an attempt to regenerate damaged nervous system tissues.

The director of the MSRCNY, Dr. Saud Sadiq, is my personal neurologist, and I know firsthand that his research facility, staffed with world-class scientists from around the world, has been hard at work for many years doing groundbreaking research in preparation for this trial. Stem cell therapy offers tremendous hope for MS patients, as it holds the promise of actually repairing the damage done by MS and restoring function lost to the disease. Let's all hope this first trial is a resounding success, one from which we all may reap tremendous benefit in the years to come. The trial is set to run for three years.

♦ I leave you with the following piece of eye candy (it's not hard on the ears, either), which is simply breathtaking. It takes 30 seconds or so to really get going, but be patient, and you'll be amazed by a stunning natural phenomenon, created by nothing more exotic than a flock of starlings…

Murmuration from Sophie Windsor Clive on Vimeo.


Happy Thanksgiving! Gobble, gobble…
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Thursday, November 17, 2011

Mind the Gap

When riding on the London Underground (subway), passengers are warned to "mind the gap". This is a reminder to beware the space between the subway car and the platform, which can pose a hazard to unwary folks entering or exiting the train. Having never been to England, I know this only from the movies, but I would imagine that nothing has changed and passengers are still reminded to take care to avoid any accidents. You've got to love the polite but direct way the British deliver this warning. Mind The Gap. Here in New York City, a similar official admonition would probably be something like "Look Alive, Jackass".

Be that as it may, in the course of our lives we are all confronted with gaps, both physical and existential, and, as with so many things, dealing with a chronic illness only magnifies these breaches. Perhaps the most difficult gap people come to grapple with is the sometimes gaping chasm between what they want and what they receive. We all start out with minds filled with dreams of who and what we'll be, a little preview clip of our own lives playing repeatedly in our heads. Despite ambitions that differ from individual to individual, the scenes playing out in our imaginations generally include joyful images of success, romance, and immense satisfaction. Aside from a very fortunate few, though, more often than not, as the paths of our lives unfold these imaginings divert increasingly from reality, and our expectations adapt to fit the circumstances in which we find ourselves.

When I was a little boy I wanted to be either a paleontologist or a veterinarian. I loved dinosaurs and dogs, so the future seemed clear. When I got older, in adolescence and early adulthood, I fancied myself a writer or rock star, in either case living large and thoroughly enjoying it. For a while I did front a rock band, and in some dusty folder packed away in a box whose location is now forgotten lives the yellowing, long untouched beginnings of a novel, so old it was written on a typewriter. I did the starving artist thing for a while, but found I could only run away from responsibility for so long, and eventually had to (gulp) cave in and take a "real job", to my absolute horror and chagrin. Surprisingly, after the initial shock wore off, my ambitions adapted and fell in line with my new reality, and I found myself thirsting for advancement in my new field of employment (TV and video production). Still, though, I was regularly haunted by the remnants of my old dreams, and despite a career that turned out to be relatively successful, somewhere deep within my soul flickered embers of disappointment, a feeling that I had somehow sold myself short.

When healthy, though, it was easy enough to placate myself with thoughts of the future. Even though I hadn't achieved the summits for which I had originally set out, the future was still an open book, and I could sit at my desk and pleasantly daydream about a tomorrow that was quite different from today. During the course of my life, I had jobs I liked and jobs I hated, periods filled with romance and also times of intense loneliness, episodes of mile a minute excitement punctuated by stretches of stone cold boredom. Through all of these ups and downs was always the promise of the future, an intensely strong motivational force that perpetually fueled the desire to plow onward. The gap between what I wanted and what I received could be papered over and camouflaged by the anticipation of a future that might still hold some magical surprises.

Living with a progressively disabling illness completely changes the equation, though. Instead of a future holding visions of grand dreams fulfilled, now the thought of days to come holds quite a bit of trepidation, the clouds coming over the horizon looking threatening indeed. I've watched the right side of my body slowly become essentially useless, and now the left is proceeding down a similar path. Of course, there is always hope, in the form of stem cells, CCSVI, neuroprotective and neuroregenerative therapies, and any one of a number of alternative treatments, but I'll not kid myself. As a man who likes to gamble, I have a good understanding of odds relative to eventual outcomes, and if this was a horserace, the pony named "Wheelchair Kamikaze Gets Cured" would be a definite longshot. Still, longshots do occasionally come in, and when they do the payoff is large and sweet. I've always been a guy to back the underdog, and I've cashed in on my share of longshots, so I am by no means counting myself out, but my situation has certainly put a damper on my proclivity to daydream about the future.

Of all the things that MS robs from those that it afflicts, perhaps it is this pilfering of a future filled with promise that has the potential to wound the most. Without it, the gulf between "wanted" and "received" can be viewed with crystal clarity. When healthy, it's easy enough to leave the mistakes of the past behind, because the activities of the present and anticipation of the future serve as kinetic distractions. Life proceeds like a twig caught in a flowing river, the headlong rush leaving little time to ruminate over the treacherous rapids or dangerous waterfalls that have already been navigated. Now, though, life has been cleaved in two along a fissure called illness, leaving me plenty of time to examine the intricacies of my existence before I got sick and pick apart the tangle of circumstances, some intended and some coincidental, that littered the path I followed.

Though there is much wisdom that can be gained from such retrospective examination, there's also plenty of room for regret. I certainly made my share of mistakes, some of them doozies, and now that my past existence has for all intents and purposes been separated from my current narrative, it's far too easy to get lost in a rush of "I should haves" and "I could haves". Some of these are relatively petty, like why the hell didn't I learn to scuba dive during the 10 years I lived in Florida, but others are more profound, "should haves" that had the potential to fundamentally impact my existence, lost opportunities that loom especially large now that the timeline of my life no longer runs in a continuous fashion, but rather as a whole broken into pieces. Might there have been a choice along the way that would have spared me my current predicament?

As a person dealing with a progressively disabling disease, losing the previously unappreciated luxury of burying the past in anticipation of the future leaves one to either flounder mournfully and angrily at the cruelty of the fates, or to consciously glean whatever positives can be plucked from the past and concentrate on making the most of the present. For all people, sick and healthy alike, the past has only as much influence on the present as it is allowed to have, although this can be a difficult concept to grasp and act upon. Those of us who have seen our lives divided by unfortunate circumstance, who have received a future none of us would have ever wanted, are forced to reconcile ourselves to this most unfortunate gap and consciously make the choice to maximize the present as an entity unto itself, divorced of the past and independent of whatever the future may hold.

Thus, we are impelled to mind the gap, and make the most of every day. The future may be frightening and the past of little consequence, leaving this day a standalone vessel which we can fill with purpose and contentment, paying little mind to desires left  unrealized, instead focusing on appreciating that which we have and not lamenting our losses. By consciously taking it day by day, hour by hour, minute by minute, and second by second, fulfillment can be found in spite of this infernal illness, the gap between wanted and received reduced to an inconsequential sliver.

So, mind the gap, lest the gap mind you…

(Not sure what that last line actually means, but it sure sounds good, kinda spooky, even…)

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Wednesday, November 9, 2011

Greed Trumps Common Sense Once Again, Courtesy Big Pharma


Image by Images_of_Money via Flickr

The mix of money and medicine often makes for a strange brew. Far too often, conflicts arise between what is best for the patient and what is best for the bottom line. Over the last several decades, treating chronic illness has mushroomed into a worldwide multibillion dollar industry. To the megacorporations reaping these profits, patients are seen first as consumers, rather than sick individuals needing to be healed. This truth is often camouflaged with warm and fuzzy programs designed for patient outreach and education, but CEOs of publicly traded medical corporations, as mandated by law, are beholden to their shareholders, not to the patients consuming their company's products, a mission which is sometimes at odds with what should be the goal of all involved in the healing professions: the curing of illness and the alleviation of suffering.

We see this unfortunate circumstance play out time and time again in the world of multiple sclerosis. Since Big Pharma finances the vast majority of medical research done in the USA, promising therapies with little profit-making potential are left to wither on the vine. Thus, medical research increasingly involves only therapies that stand to attain blockbuster status. We therefore have very little scientifically reliable data on the effectiveness of Low Dose Naltrexone, dietary supplements, acupuncture, naturopathic remedies, and other largely benign practices and substances. Alternative theories about the disease, such as CCSVI, are met with a fusillade of negativity instead of intellectual curiosity, as might be expected in the case of a disease as intractable as multiple sclerosis.

One of the oddest examples of the profit motive trumping common sense involves the drug Rituxan (click here), a compound first formulated to battle B cell non-Hodgkin's lymphoma, for which it was approved by the FDA in 1997. Rituxan was the first monoclonal antibody used to fight cancer, and proved to be both safe and effective in that role. The compound works by destroying B cells, one of the major components of the human immune system. Therefore, in addition to its lymphoma fighting abilities, Rituxan is a powerful immunosuppressant. Due to these immunosuppressive properties, the drug was tried with varying degrees of success on a number of autoimmune diseases, and has been approved for use in patients suffering from rheumatoid arthritis.

Several years ago, clinical trials were started testing Rituxan's efficacy in fighting multiple sclerosis. These trials included not only RRMS patients, as is typical of MS trials, but also PPMS patients, a population for which there are no approved therapies. Phase 1 and 2 trials showed the drug to be extremely effective, dramatically reducing the amount of enhancing lesions seen on patient MRIs, and cutting by half the number of relapses experienced by RRMS trial subjects (click here). The trial results were at least equal to those seen with the drug Tysabri, which to date had been the most effective MS drug on the market. Rituxan had the added advantage of having a long history of use, which showed it to have a much lower incidence than Tysabri in expexposing patients on the therapy to to the possibility of developing PML , a sometimes fatal brain infection. Trials for PPMS were not as successful, although analysis of the data did seem to indicate that a subgroup of PPMS patients did appear to benefit from Rituxan therapy (click here).

So, it would seem that all systems were "go", and that Rituxan would be quickly shepherded into phase 3 multiple sclerosis trials, the final step needed before FDA approval to go to market, right? Well, this is where things go a little bonkers. It turns out that Rituxan's patent is due to expire in 2015, meaning that the company that makes it, Genentech, will no longer have exclusive rights to the drug, and generic versions of it could come on the market, esseessentially stripping Rituxan of its profit-making potential. Because of the complexity involved in making monoclonal antibodies, it was at first thought that the production of generics would be too costly to be seriously considered, but other companies, sensing opportunity, did indeed step into the arena (click here). Given this situation, despite the great promise shown in the earlier trials, Genentech pulled the plug on further MS Rituxan trials. Nevermind that Rituxan appeared to be safe and effective in alleviating some of the suffering caused by a dread disease, there was no money to be made from it, so any further development hit a brick wall.

Instead, Genentech did some tinkering with the production methods used to make the drug, and came up with a compound called Ocrelizumab (click here), another monoclonal antibody that destroys B cells, which was quickly put into clinical trials for rheumatoid arthritis, lupus, multiple sclerosis, and hematological cancers. By developing this new compound, so similar to Rituxan, Genentech was insured of maintaining exclusive rights to the drug for several decades, with no threat to the tremendous profits a drug equally as effective as Rituxan could generate. Ah, but the best laid plans of man sometimes go awry, and things didn't work out quite the way Genentech intended.

In 2010, Genentech was forced to suspend Ocrelizumab trials in rheumatoid arthritis and lupus due to deaths resulting from opportunistic infections attacking trial participants (click here). Of course, this was a tremendous blow to Genentech's wily plan to circumvent Rituxan's patent issues (click here), and a serious kick in the bottom line. But, alas, all was not lost, as trials continued testing Ocrelizumab's use in multiple sclerosis. Recently released phase 2 clinical trial results have shown Ocrelizumab to be highly effective in reducing enhancing lesions and relapses in RRMS patients (click here), and Genentech is now in the process of recruiting patients for phase 3 trials for both RRMS and PPMS (click here, here, and here). Apparently, the perception is that tolerance for risk is higher in the MS population than it is among lupus or RA patients, so it's full speed ahead, torpedoes be damned.

Rituxan is currently still on the market, often used off label for the treatment of MS, and has proven to be very effective in relieving some of the suffering of RRMS patients. Unfortunately, since it is not FDA approved for use in MS, many insurance companies refuse to pay for it, as it is an extremely expensive therapy (over $40,000 per year). Once the generics do hit the market in several years, the price of the drug should plummet. Should Ocrelizumab pass its phase 3 trials for MS, and be approved by the FDA, rest assured that Genentech's marketing machine will use every trick in the book to get this newer, less proven, and possibly more dangerous drug given preferential treatment over its low-rent cousin.

Of course, neither of these drugs does one whit to cure MS, but why try to cure something when treating it is so immensely profitable? Rituxan has proven to be quite effective and relatively safe (the PML rate is in the range of 1 in 100,000) when used to treat MS, and the fact that it will never even be given the chance to get FDA approval as an MS therapy simply because its power to generate millions of dollars in profits will soon disappear is nothing short of a travesty, further compounded by the emergence of Ocrelizumab, whose sole reason for existence is to sop up the profits that will be lost when Rituxan goes off patent. Might not the money used to develop and test Ocrelizumab, a figure undoubtedly in the millions of dollars, have been better spent on research that might further our knowledge of how to combat MS, rather than simply finding a way to mimic the actions of an already existing drug in a form conveniently different enough to be patentable (and, apparently, more dangerous)?

Unlike some other MS advocates, who label all of the available mainstream disease modifying drugs nothing more than snake oil, I recognize their value in improving the quality of life of many of the patients taking them. Certainly, even if they do nothing to halt the progression of the disease, dramatically reducing the amount of relapses suffered by RRMS patients has great value, and I know of many patients whose lives have been tremendously improved through the use of today's DMDs. What I can't stomach, though, is the blatant profiteering practiced by the big pharmaceutical companies, as is so clearly illustrated in the Rituxan/Ocrelizumab saga. People's lives are at stake, but I suppose in the world of big money modern medicine that concern pales in comparison with the chase for the almighty dollar.

I say shame on all involved…

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