Monday, November 23, 2015

Bits and Pieces: MS Diversions

Wow, I just checked and it’s been almost 7 months since I last did a “Bits and Pieces” post. These posts, compendiums of the latest and greatest MS news and info, have long been a regular feature on these pages. Somehow, I’ve really let things slide. Seems I’ve been a very naughty blogger. The blogging police are probably getting ready to mount a raid on my computer any day now, so I better get back on the beam, lest I be found guilty of blogging negligence and sentenced to reading the comments section of some celebrity gossip website until my brown eyes turn blue. For the record, Justin Bieber is NOT hotter than ANY of the guys in One Direction and anyone who thinks so is a big fat stupid loser!!!!! And the budding romance between Gwen and Blake is like the best thing that ever happened in the world ever ever ever!!!! I might have to cut down on watching of kitten videos so I can soak up every word being written about those two crazy love birds. OMG.

Just to refresh everybody’s memory, Bits and Pieces posts are collections of interesting items of MS related research, news, and other Internet MS flotsam and jetsam that have caught my attention in the recent past. Though there’s certainly been plenty of impactful MS info floating around the inter-webs during my long lapse in trying to anthologize it, I made the command decision to devote this post to a collection of MS related online communities, blogs, and other sites that I think might be of interest, places which can serve as welcome diversions in the online MS world. Personally, I can sometimes get so caught up in parsing through all the latest research that I neglect the touchy-feely human side of things. The sites that follow offer interesting twists on typical online forums, blogs, and whatnot. Especially the whatnot, along with the doohickeys and thingamajigs.

Please note, I know there are lots and lots of MS themed blogs and communities out there, and by calling attention to these few I don’t mean to slight any of the others. This isn’t a “best of” list, just a compendium of places that have stuck in my brain pan lately. So, without any further hemming and/or hawing, here are some suggested sites upon which to aim your eyeballs…

♦ Let’s start with a site that has long been near and dear to my heart, MS Kurmudgeons Korner (click here). MSKK, as it is affectionately known by its regulars, is an under the radar MS forum that is completely beyond the reach of any Internet search engines, making it accessible only to those in the know. The site adheres to a strict privacy policy, meaning that what is said in MSKK stays in MSKK upon pain of death. MS Kurmudgeons Korner is so named because it is a self-avowed rainbow and unicorn free zone where people are free to discuss the fact that MS sucks huge hairy monkey balls without fear of stepping on anybody's rose-colored glasses. Not that the place is without humor, as at it's often more fun than a night at the GiggleSnort motel, but it is definitely Nowheresville for those prone to utterances like “I have MS but MS doesn’t have me”.

Since MSKK is a closed community, it’s rare that the gates are thrown open in such a public fashion as this. However, due to the usual ebb and flow of the Internet, membership is down and an infusion of new blood will be welcomed. In all honesty, because of the progression of my illness and the incredible amount of time it takes just being sick (dammit), I haven’t been hanging out there nearly as much as I have in the past, but I guess that’s part of that whole ebb and flow thing.

So, if you are an MS cranky pants with a sense of humor, by all means mosey on up to MS Kurmudgeons Korner and knock on the door. You’ll be asked to sign a virtual privacy policy, and then introduce yourself with an opening post. Current members will then say hello and ask a few questions so that you can get a feel for them and them for you. After you tell a little something about yourself and prove that you’re not an undercover unicorn, you’ll soon be given free rein to join the community.

♦ Next up is a blog called Tripping on Air (click here), which is exceptionally well written by a classically trained singer with MS who lives in Toronto. The writer of Tripping on Air manages to blend her sardonic wit, keen sense of the absurd, and terrific observational skills into essays on everything from miracle cures to wetting the bed (click here) that have me laughing out loud and nodding in agreement, even though she sometimes talks about girly stuff that doesn’t quite jibe with my manly universe filled with bad zombie flicks and the Boston Red Sox. The blog’s only been around for about five months, so there’s not a huge amount of content, but what is there is well worth a look.

♦ My Counterpane (click here) takes a unique approach to building an online MS community. The site is built around members’ moods, as expressed by “moodifiers”, entries describing a person’s mood and what’s making them feel that way. What really sets this site apart is the ability for members to include videos in their moodifiers, so lots of folks choose to express themselves via short video clips rather than the written word. This all probably sounds kinda weird, but it’s actually pretty engaging. Posts can be sorted by mood, so whether you’re feeling angry or hopeful it’s easy to find others experiencing similar emotions, and members can build a little moodifier universes of their very own by following other members.

I personally feel much more comfortable behind the camera than in front of it, but the ability to just sit at of your WebCam and describe whatever emotions life and MS happen to be throwing at you at any particular moment does have its appeal. Unfortunately, these days I’m afraid I often look like one of the zombies in those bad flicks I love so much, and my WebCam would scurry off in horror if I ever turned it on when I’m in such a state. Good thing Rick from The Walking Dead hasn’t chanced upon me, otherwise I’d definitely have a knife sticking out of my head. Still, just having the option to communicate by video makes My Counterpane unique, and that in combination with its mood-centric emphasis has lots of people hooked.

♦ One of the most visually appealing MS blogs I’ve come across is Wheels & Red (click here), put together by an MSer in the Pacific Northwest who goes by the moniker "Wheels" (his wife is “Red”). Wheels (real name Kellen) was diagnosed in 2010 with progressive MS at the age of 25. His wife Meg is a photographer, and a quite talented one at that. Kellen is a very good writer, and his subject matter diverse. Now on full-time disability, Kellen uses his enforced freedom to explore his inner and outer worlds, and presents his viewers with an always interesting hodgepodge of visuals and essays that are both thought-provoking and entertaining.

The only thing I don’t like about Wheels & Red is that it makes me feel old. The writer and his wife are young and Bohemian in a very Pacific Northwest kind of way. Problem is that I’m now a 52-year-old fuddy-duddy who used live a Bohemian existence myself, back before our culture turned a generation of pierced and tattooed twentysomethings into a marketing demographic. My friends and I were slackers about a decade before slacking was a recognized social phenomenon (I believe the term was first brought into mainstream consciousness with the 1991 film “Slacker”). Wheels & Red reminds me of a time before I capitulated and allowed myself to slide down the slippery slope towards 9-to-5 normalcy – for a time I even wore a tie to work every day. Ack! Now, despite the fact that my own disease enforced freedom has returned me to a warped version of the slacker lifestyle, I still rue the fact that regardless of my loud and boasting youthful protestations that I would do no such thing, I sold out. And rather cheaply, at that.

Wow, where did that bit of existential angst come from? I guess a really good blog can do that to you, get your thought processes shooting off on unexpected tangential extracurricular activities, which I suppose is one of the great benefits of any successful creative effort. So, I guess the above paragraph marks Wheels & Red a success. Hopefully, whatever tangents it sends you off on won’t involve self chastisements decades in the making. May all your tangents be happy ones…

♦ Shift MS (click here) is a growing social network for MSers, founded by a young man in England with MS as a way of building connections with others who share the diagnosis. The site is very well put together, with lots of interesting features including a members’ forum, a variety of online groups to join, plenty of MS research info (including a large library of video interviews with MS researchers), and advice on lifestyle and medication choices. The site is pretty slick, so slick that at first I suspected it must have been created by one of the pharmaceutical companies as an undercover effort to collect valuable data on MS patients (I’m not being paranoid; some of the most popular MS and health-related websites are actually just such Trojan horses), but upon further investigation I discovered that Shift MS is a registered nonprofit in England, and appears to be run by a bunch of people with MS for the sole purpose of helping other people with MS. How refreshing! In spite of its numerous offerings, which could make for a cumbersome Internet experience, I found Shift MS to be quite easy to navigate and filled with interesting nooks and crannies. The site has a nice international flavor to it, also, with large groups of Brits, Americans, Canadians, and Aussies represented. Nothing like sharing a potentially crippling disease to break down international borders, huh?

♦ For those who eschew talking to real-life human beings and would rather converse with weird web-based artificial MS patients who hardly ever blink and could easily scare young children, do I have a site for you! The Consortium of Multiple Sclerosis Centers (CMSC) has created a bizarre little program called “My MS Conversation”, that you can download by (clicking here). If you are asked to enter a code for downloading, try 54S2. I promise, the program is absolutely safe, at least from a computer virus standpoint, though I can’t guarantee that using it won’t leave emotional scars.

Upon opening the program, you are “greeted” by four virtual animatronic “people” who move like they are inflated with helium and have big perfectly round largely unblinking eyeballs. One of the characters is a “nurse moderator”, and this jolly foursome is eager to engage you in “conversations” about managing multiple sclerosis. As you can see in the screenshot on the right, this is a very inviting bunch, so unnervingly bizarre that you might be fooled into thinking that you are interacting with actual animated virtual MS people, and not MS zombies just waiting for you to get close enough to your computer screen for them to reach out and EAT YOUR BRAINS! Lucky for me I'm hyper aware of the coming zombie apocalypse, and not easily fooled. This program would be better titled "The Limping Dead", except no matter how much I click on them or hover my face dangerously close to the screen just tempting them to reach out and EAT MY BRAINS these MS zombies won't get up off their seats, so I can't tell just how disabled any of them are. They look pretty healthy, though, which must mean that they've tricked plenty of other people into getting close enough to their computer screens for them to reach out and EAT THEIR BRAINS!

The creation of My MS Conversation was partially funded by two pharmaceutical companies, Teva and Mallincrodt. Teva is the manufacturer of Copaxone, the best-selling MS drug which last year brought in $4 billion in revenue. Yes, that’s “billion” with a “B”, which also just so happens to be the first letter in BRAINS! So, as you might guess, most of the “conversations” that the MS zombies engage in have to do with taking disease modifying treatments. But no matter, the entire experience of using the program is so otherworldly that I find it almost impossible to concentrate on anything the animated MS zombies are saying, as all other thoughts in my head are crowded out by one huge “WTF?”. As I might have mentioned before, I have a huge affinity for all things zombie, but this program could be the one exception. Still, I just can’t get myself to delete the damn thing from my computer, as every now and then I find myself clicking on its icon just to make sure I wasn’t imagining it all or that I didn’t conjure up My MS Conversation in a scary dream one night after eating a batch of bad enchiladas. But no, the program always turns out not to be a product of my warped imagination, but instead of somebody else’s. I definitely want no part of whatever enchiladas they were eating. Or were they maybe – EATING BRAINS!?!?

So go ahead, download My MS Conversation. I dare you.

Thursday, November 5, 2015

A Worthy Cause

(For those receiving this via email, the following post contains multiple videos which aren’t available for viewing in email format. To view the videos, please visit the Wheelchair Kamikaze website, by clicking here)

Okay, folks, I’m going to take the liberty of using my virtual soapbox here on Wheelchair Kamikaze to beat the drum for a worthy cause, an outside the box effort to make the lives of the disabled easier and maybe even a bit more joyful. I’ll include a bunch of videos which will help explain this project and the needs it addresses. If a picture is worth a thousand words, then a video must be worth several hundred thousand, and these short clips will save you the rigors of having to read yet another of my seemingly endless polemics.

I present to you the AXS Map project, which is the brainchild of filmmaker and fellow MSer Jason DaSilva. Jason recently won an Emmy for his documentary film When I Walk (click here), an eloquent and touching exploration of his journeys through the world of progressive MS. When I Walk received many well-deserved accolades at film festivals around the world, and was shown on US television as part of the PBS series POV. The film is available for instant viewing on a variety of streaming platforms, including Hudu, Vudu, iTunes, Google Play, and YouTube (click here).

If you haven’t seen the film, I highly recommend it. When I Walk offers an unvarnished portrayal of the realities of life with a chronic progressive disabling disease without dissolving into pathos, and also offers an uplifting love story to boot. There, now I’ve played film critic, testament to the teaching skills of my professors back in film school over 30 years ago. How nice that my degree hasn’t gone completely to waste…

Ah, but I digress. Back to the subject at hand, the AXS Map project (click here), which endeavors to create an online and mobile app based mapping tool that will allow disabled folks to readily identify accessible shops, restaurants, and other establishments in whatever city or neighborhood they happen to find themselves. The project is worldwide in scope, and its efforts to map locales both near and far involve interactive “mapathons”, organized mapping events that can be turned into fun little competitions. AXS Map project funding efforts are currently underway via a Kickstarter crowdfunding campaign intended to turn Jason’s vision into reality (click here). Here’s a video about AXS Map produced by Jason  that fully explains the project.

As most every disabled person can attest, the frustrations involved in simply trying to accomplish even the most mundane errand can often prove daunting and disheartening. Cities like New York, in which Jason and I both reside, are filled with obstacles that can go completely unnoticed by able-bodied folks, but are just about insurmountable to those of using canes, walkers, wheelchairs, or scooters. Here’s a great video Jason did for the New York Times that vividly illustrates the hassles routinely encountered by those whose legs are no longer their primary means of transportation…

Hey, it occurs to me that this would be a great place to dust off one of my old Wheelchair Kamikaze videos, which were the reason this blog got started in the first place. Way back in 2009 Wheelchair Kamikaze was born with the intention of it being a place to park the videos and photos I was shooting for my wheelchair, a spot on the Internet where friends and family could access my self-indulgent little projects. Among these self-indulgences was a video called “Searching for Audrey Hepburn”, in which I take a wheelchair journey through the streets of New York looking for a statue inspired by the late great actress’s indefatigable humanitarian work on behalf of the children of the world. Along the way I encounter quite a number of the obstacles that can make getting around New York in a wheelchair a genuine pain in the ass. Sadly, the friend I mention in the video, who inspired my search for the statue with his boundless affection for Audrey Hepburn, passed away about a year and a half ago. Rest in peace, Sir Brett, and thanks for the smiles…

And just for the heck of it, here’s a video of Jason receiving his Emmy award for When I Walk, which, even if you haven't seen the film, is poignant and touching in its own right…

Please support the AXS Map project, just waiting for your help to get kicked into high gear. If you’re not in a position to make a financial donation, consider organizing or taking part in a mapathon, thereby helping to make life that much easier for people with disabilities. For more info, just visit the AXS Map website (click here). Thanks.

Monday, October 19, 2015

Video: Promising FDA Approved Stem Cell Trial, Repeatedly Rejected for Funding by NMSS, Featured in TV News Segment

The only FDA approved Multiple Sclerosis stem cell trial currently underway in the US was featured last week in “The Big Idea”, a monthly segment seen on the local New York Fox Network affiliate’s evening newscast. The segment features the study’s lead researcher, Dr. Saud Sadiq (full disclosure: he’s my neuro), detailing the trial, and highlights a patient currently enrolled in the study who appears to be benefiting from the treatment.

The stem cell trial is being conducted by the Tisch Multiple Sclerosis Research Center of New York, and utilizes proprietary methodologies unlike those being used in any other stem cell study or treatment center in the world. As is detailed in the below video, raw mesenchymal stem cells are extracted from a patient’s own bone marrow and are then transformed in the laboratory into Neural Progenitor cells, stem cells specific to the central nervous system. The cells are then introduced directly into the patient’s central nervous system via intrathecal injection. Unlike HSCT, the type of stem cell therapy that seeks to stop or slow down MS disease progression by rebooting the immune system after ablating it using chemotherapy drugs, the Tisch Center’s trial is aimed directly at regenerating and restoring the damage done to brain and spinal cord tissues by the MS disease process.

Regular Wheelchair Kamikaze readers will recognize this as the very same trial that the National Multiple Sclerosis Society has repeatedly refused to fund for reasons that can only be deemed suspect, as I’ve detailed in several previous blog posts (click here and here). The Tisch Center’s trial is currently an early phase 1 study and is in urgent need of additional funding in order to expand into a larger and more comprehensive phase II trial.

As the Tisch Center is not affiliated with any academic institution or hospital and is a completely independent nonprofit organization, its research efforts are entirely reliant on private donations and institutional grants for continuation. Due to funding shortfalls, the trial is currently on precarious financial footing despite showing tremendous potential and promising early results. One would hope that the NMSS would see fit to reconsider its previous withholding of funds and do its part to help further the only FDA approved Multiple Sclerosis stem cell trial in the USA.

As is evidenced by this news segment, the Tisch Center stem cell trial has the potential to dramatically change the MS treatment landscape. I’d urge all who feel that the National Multiple Sclerosis Society should reverse course and support the Tisch Center to contact the Society and voice their opinions. The  email addresses of the Society’s executive staff can be accessed by (clicking here), and the phone number of the NMSS is 1-800-344-4867

Please keep all communications polite, as the NMSS may in this instance be misguided, but it is not an adversary. As my grandmother used to tell me, you can catch more flies with honey than you can with vinegar…

Thursday, October 15, 2015

Ocrelizumab Update – RRMS Trials Successful; PPMS Trial Shows Positive Effect, But Many Questions Left Unanswered

When I last posted to Wheelchair Kamikaze, I profiled the experimental MS drug Ocrelizumab, which was developed by Swiss drug giant Roche (and their US subsidiary Genentech). In press releases put out by the company two weeks ago (click here), Ocrelizumab was touted as the first MS drug shown capable of putting the brakes on Primary Progressive Multiple Sclerosis (PPMS) – a subtype of the disease which until now has been largely untreatable – as well as it being highly effective in treating the more common Relapsing Remitting Multiple Sclerosis (RRMS). These results were even more noteworthy because Ocrelizumab is the first MS drug to specifically target immune system B cells, while other currently available MS drugs (Tysabri, Gilenya) were developed to target T cells, which until now were believed by many to be the primary culprits driving the MS disease process.

Though the initial Ocrelizumab press releases were scant on details, the news certainly appeared promising, though reservations were expressed by many MS researchers based on Ocrelizumab’s development history and its close similarity with another Roche product, Rituxan, a much older drug that had gone through early trials in RRMS (very successful) and PPMS (largely a failure, but with some hints of success) back in 2005-2008. Rituxan’s development as an MS treatment was halted by Roche because of what many believe to be strictly financial concerns. Reading my previous essay (click here) will provide much background on Ocrelizumab’s development and the issues surrounding the drug.

Roche presented the details of the Ocrelizumab RRMS and PPMS trials this past weekend at the ECTRIMS conference in Barcelona, Spain. The RRMS trials were named OPERA I and OPERA II, and the PPMS trial was named ORATORIO. The results of the OPERA I and OPERA II RRMS studies were indeed impressive. These studies compared Ocrelizumab to Rebif, an interferon drug long used to treat RRMS, and found that treatment with Ocrelizumab resulted in a 50% reduction of annualized relapse rates, a 40% reduction in progression, and an 80% reduction in enhancing lesions as shown on MRI when compared to patients taking Rebif (click here – login required, but sign-up process is easy and well worth it). Adverse events were found to be similar in both groups, which is encouraging as Rebif is considered one of the safer MS medications currently available.

It should be noted, however, that the two-year length of these trials may be insufficient to detect longer-term problems that may develop as a result of the intense B cell suppression that is Ocrelizumab’s primary mechanism of action. Nevertheless, it does appear that Ocrelizumab will become a very valuable weapon in the growing arsenal of drugs used to combat RRMS, though, like all current MS drugs, it doesn’t do a thing towards actually curing the disease. But I’ll save that rant for another day…

(Please note, the following information and accompanying commentary were largely gleaned from materials contained in articles and interviews that can be accessed by clicking here, here, here, and here. I would urge all to read them for greater context and background)

Results of the ORATORIO PPMS study, while initially impressive, still remain riddled with questions. Despite the screaming headlines hailing Ocrelizumab as a “revolutionary new MS drug” by the – as usual – hyperbolic mainstream medical media (click here), the actual picture remains far more nuanced. The ORATORIO trial design was heavily influenced by the results of the failed Rituxan PPMS trial back in 2008. Though that trial was officially deemed unsuccessful, subsequent examination of the data revealed that a subset of PPMS patients in the study were seemingly helped by the drug. These patients were generally younger, had shorter disease duration, were less disabled, and had enhancing lesions on their MRIs. It’s thought that patients who fall within these parameters make up about 15% of the overall PPMS population.

It appears that in designing the ORATORIO study, Roche chose trial subjects who were most likely to benefit from Ocrelizumab treatment, specifically those who fit the demographic and disease parameters outlined above. As presented at ECTRIMS, the results of the ORATORIO study are indeed impressive, demonstrating a marked slowing of disease progression, a reduction in MS enhancing lesions, a reduction in brain volume loss, and a stable time to walk 25 feet when compared to trial subjects given a placebo. Of course, these results also mean that many of the ORATORIO trial subjects were patients with enhancing lesions (only a fraction of PPMSer’s have such lesions) and who were relatively less disabled (as evidenced by the fact that they could still walk). The majority of trial subjects also appear to have been relatively recently diagnosed, with a disease duration of approximately 3-5 years, and were under 50 years old.

Unfortunately, when Roche presented the ORATORIO results at ECTRIMS, they declined to detail the breakdown of results by patient subgroups, claiming that such subgroup analysis had not yet been satisfactorily completed. This beggars belief, as one would think that examining the data in such a way as to determine which patient groups benefited the most from treatment would be among the highest research priorities, especially given the mixed results of the previous Rituxan PPMS trials. Instead, Roche chose to give a broad overview of favorable trial results without answering some of the most important questions about those results, primary among them whether or not the patients who most benefited were those with enhancing lesions. Indeed, in the Q&A period following Roche’s presentation, presenters were questioned on just this issue by conference attendees, who were given no suitable answers other than promises that the data would be forthcoming. Roche's presentation led several experts to say the primary progressive data were less impressive than those seen with Ocrelizumab in relapsing remitting disease.

Although Roche portrayed the safety profile of Ocrelizumab in the ORATORIO trial to be excellent, they did note that there were 11 cancers detected during the ORATORIO trial on patients taking Ocrelizumab, versus only two in the placebo group.

The results of the Ocrelizumab RRMS and PPMS studies have not yet been published in any scientific journals, and it is expected that much additional data will need to be provided to publishers before the trial results will be deemed fit for publication. Getting the drug through the regulatory approval process required by government agencies such as the FDA will also necessitate a much greater detailing of trial results, including subgroup analysis which will shed light on just which patient groups were treatment responders. While it seems that the RRMS results will likely stand up to more intense scrutiny, the PPMS results may prove to be less dramatic than originally portrayed when subjected to the spotlight of journal publication and regulatory approval.

That said, even in a worst-case scenario in which only a small subgroup of PPMS patients, those with enhancing lesions,  prove to benefit from Ocrelizumab treatment, the fact that any headway at all has been made in treating PPMS is a significant step forward. If in fact the drug’s efficacy is limited to patients with enhancing lesions, and such lesions are most likely to be found in patients in the earliest stages of the disease, the importance of early diagnosis will be brought to the forefront. Given current diagnostic tools and techniques, many patients with PPMS aren’t correctly diagnosed until the disease has long since taken hold, beyond the period when enhancing lesions are likely to be present. Additionally, the fact that Ocrelizumab targets B cells should open up new avenues of investigation for MS researchers worldwide, as this potential mechanism of MS disease action has until now been largely discounted by most mainstream researchers. Perhaps these investigations might finally unravel some of the deepest mysteries of the disease, and (crossing the fingers I can still cross) eventually lead to a cure for all forms of the disease.

At this point, only time will provide us with the answers we seek. The next chapters of the Ocrelizumab saga will be getting these studies peer-reviewed and published in medical journals, a process which will shed much light on issues still in need of clarification. Roche is targeting the first quarter of 2016 for regulatory approval in both RRMS and PPMS. It will be very interesting to see whether or not Ocrelizumab is approved for the PPMS population as a whole, or, as many researchers suspect, for only a subgroup of progressive MS patients. Again, even if the latter case holds true, we will have at least made some headway in the fight against an insidious form of Multiple Sclerosis that until now has defied efforts to combat it.

At long last, it appears that the attentions of Multiple Sclerosis researchers are finally focusing on progressive MS, an area of investigation which until very recently had largely been neglected. The confounding problems represented by progressive MS will never be solved until well-funded trials are directed specifically at this disease subtype, a circumstance that finally seems to be coming to fruition. And that’s no small thing…

Sunday, October 4, 2015

Experimental MS Drug Ocrelizumab Deemed A Success In Progressive MS Trial, But Questions Remain

Last week, the drug company Genentech (a subsidiary of Swiss drugmaker Roche Pharmaceuticals) announced that its Phase III trials for treating Primary Progressive Multiple Sclerosis with the experimental MS drug Ocrelizumab had met with success. According to a press release titled “Roche’s Ocrelizumab First Investigational Medicine to Show Efficacy in People with Primary Progressive Multiple Sclerosis in Large Phase III Study”, the drug met the trial’s primary endpoint, “showing treatment with Ocrelizumab significantly reduced the progression of clinical disability sustained for at least 12 weeks compared with placebo, as measured by the Expanded Disability Status Scale” (click here). Earlier this summer two previous trials had shown Ocrelizumab to be more effective than the beta interferon drug Rebif for treating RRMS, resulting in reduced relapse rates, number of MS lesions, and a reduction in progression of disability (click here). Ocrelizumab is an intravenous drug that is administered twice over a two-week period, and then not again for six months.

While this would all appear to be great news, especially for people with PPMS who currently have no approved treatment options, just how great this news actually is remains to be seen as the press release provided absolutely no details on the study results, which will only be revealed on October 9 and 10th at the annual European MS conference ECTRIMS, being held in Barcelona, Spain. Understandably, news of Ocrelizumab’s success in treating PPMS has created quite a buzz in the MS community as well as in the financial pages of publications around the world, as the drug stands a good chance of becoming the latest multibillion-dollar blockbuster MS treatment (click here).

There is reason to believe, however, that Ocrelizumab may only be effective in treating a subset of patients suffering from Primary Progressive MS, specifically those who are less disabled (still walking), younger, and who display enhancing lesions on their MRIs. And though the press release states that the side effects experienced by study subjects taking Ocrelizumab were not much different than those who were given a placebo, back in 2010 trials testing Ocrelizumab for the treatment of Lupus and Rheumatoid Arthritis had to be abandoned because of an unacceptable number of serious and sometimes fatal opportunistic infections seen in trial subjects (click here).

How can I make any assumptions on Ocrelizumab’s efficacy at all based on the scant details divulged in Roche’s press release, you may ask? Well, it turns out that Ocrelizumab’s mechanism of action is nearly identical to that of a much older drug also manufactured by Roche, called Rituxan (Rituximab), which has been used for decades to treat cancers of the blood and for the last seven or eight years, on an off label basis, to successfully treat Relapsing Remitting Multiple Sclerosis.

In fact, back in 2008 Rituxan completed early stage trials (phase I and phase II) investigating its use in treating both RRMS and PPMS. In these earlier trials, Rituxan was deemed successful in treating RRMS (click here), but the drug failed in its much-anticipated trial for PPMS (click here). Subsequent to the PPMS trial’s completion, however, researchers combing through the data discovered that Rituxan did appear to have a beneficial effect on a subset PPMS patients, specifically those who were less disabled, were 50 years old or younger, and who had enhancing lesions, (click here). Ocrelizumab’s mechanism of action is so nearly identical to Rituxan’s that within the industry the drug has been nicknamed “brother of Rituxan”.

Why then was Rituxan not further investigated for the treatment of RRMS and PPMS, Roche and Genentech instead shifting their all of their attentions to costly process of developing a brand new drug that works in a very similar fashion, Ocrelizumab? Well, this is where things get interesting and more than a little bit curious. If I were a cynical man I might conclude that purely financial considerations were involved, but far be it for me to be so cynical. Instead, I’ll present you with the evidence as I know it, from which you may draw whatever conclusions you wish.

As I mentioned earlier, Rituxan is an older drug, and it is scheduled to lose its patent protection this year. Once the Rituxan comes off patent, Roche will lose its exclusive rights to the drug and thus the ability to generate tremendous profits from marketing it (click here). This fact made the return on investment of proceeding with very costly late stage MS trials in 2008 decidedly negative even if they proved wildly successful, because after 2015 another company could step in and market a generic version of Rituxan, thereby killing the drug’s profit making potential for Roche.

Both Rituxan and Ocrelizumab are in a class of drug called monoclonal antibodies, which are manufactured antibody cells that work by targeting specific cells in the human body. Rituxan and Ocrelizumab work by attacking B cells, one of the two primary types of cells that comprise the human immune system (these two types of cells are called T cells and B cells). They both seek out the same protein expressed on the cell walls of B cells, CD 20, and both drugs ultimately destroy almost all of the B cells in the human body using nearly identical mechanisms.

This anti-B cell approach is not taken by any other current MS drug, as until very recently it was believed that the MS disease process was driven primarily by T cells, which are the focus of drugs such as Tysabri and Gilenya (the latter of which recently failed in a recent PPMS trial). The fact that Rituxan and Ocrelizumab – which exclusively target B cells – are proving to be so effective in battling MS is forcing researchers to completely rethink much of what has been assumed in the understanding of the disease. Roche’s recent Ocrelizumab press release also contains hints that the drug’s action on B cells may have some neuroprotective properties, which if true would be truly remarkable. Of course, since Rituxan has already been used successfully to treat RRMS for quite a few years, the knowledge that a B cell immunosuppressant was capable of dramatically impacting the Multiple Sclerosis disease state was readily available, but also easy to ignore given the somewhat rogue nature of treating MS with Rituxan.

The difference between Ocrelizumab and Rituxan is the fashion in which they are manufactured. All monoclonal antibodies are “biologic” drugs, meaning they are derived from living animal cells, usually from a combination of mouse and human tissues. Most older monoclonal antibody drugs, such as Rituxan, are what is known as “chimeric monoclonal antibodies” (click here), meaning they contain both mouse and human DNA. Newer drugs such as Ocrelizumab are “humanized monoclonal antibodies” (click here), and have their DNA structure manipulated to more fully resemble completely human cells.

Although one might assume that humanized monoclonal antibodies would always be preferable to those that contain non-human DNA, Rituxan has been used for decades and has shown itself to be a very safe and effective drug. Although some cases of opportunistic infections, including PML, have been associated with Rituxan, they’ve appeared far less frequently than those seen with Tysabri (click here). Rituxan has proven itself successful in treating RRMS both in trials and in real life practice (the drug is FDA approved for the treatment of Rheumatoid Arthritis, and is used off label to treat a variety of other autoimmune diseases).

The Rituxan PPMS trials, though at first deemed a failure, did reveal a subset of PPMS patients on whom there was evidence of efficacy, and it appears that the Ocrelizumab PPMS trial was designed to capture as many of this type of patient as possible (click here). Had Roche pushed ahead with its Rituxan MS trials even in the face of a looming patent expiration we might have had another very effective treatment for RRMS, and perhaps even PPMS, for at least the last five years. Alas, ‘twas not to be, quite likely because Rituxan was scheduled to soon lose its patent protection.

I truly hope that when the full details of the Ocrelizumab trials are revealed at the upcoming European MS conference they exceed all expectations and make Rituxan look like a rusty old Model T compared to Ocrelizumab’s shiny brand new Maserati. If the new drug is in fact capable of stabilizing the disease progression of the majority of PPMS patients we will have a true game changer on our hands. Even if, as I suspect, the drug is shown to be effective on just a subset of PPMS patients – those who are younger, less disabled, and have enhancing lesions – it will represent a tremendous leap forward in the treatment of what has up until now been an untreatable disease. The fact that the drug exclusively targets B cells and benefits both the relapsing remitting and progressive forms of Multiple Sclerosis should send researchers scrambling to develop an entirely new model of the MS disease process, and, who knows, may lead to an eventual understanding of the ever elusive underlying cause of Multiple Sclerosis.

Here’s to hoping that the new drug proves to be as safe and reliable as the old one, Rituxan, and Ocrelizumab will become not only a powerful new weapon in the fight against MS, but also a catalyst for an entirely new way of thinking about the disease. For people with PPMS, this successful drug trial, even if limited, finally opens a door to treatment options and allows rays of hope to filter in through the clouds. We’ll begin to get our answers in just about a week, and I for one can’t wait for the data to be released.

Geez, that last line may be just about the wonkiest sentence I’ve ever written. I guess I just have to own up to the fact that I’m now a full-fledged MS research nerd…

Wednesday, September 23, 2015

Last Week’s Webinar on Complementary Medicine and Multiple Sclerosis Now Available Online

For those who missed the live webinar I participated in last week, it’s now available online for your listening pleasure. Okay, maybe "pleasure" is taking it a little too far, but the webinar is packed with lots of useful information. I was joined on the iConquerMS sponsored webinar by Dr. Deneb Bates, a naturopathic physician who I’ve worked with for over seven years. As is in ample evidence during the webinar, Dr. Bates really knows her stuff.

Topics covered include the doctor-patient connection, biotin, vitamin D, natural remedies for spasticity, oxidative stress, and more. There’s much material about progressive MS, along with actionable info that you can discuss with your treating physicians.

I believe the webinar can only be viewed on a desktop or laptop computer, not on a mobile device. I’d love to hear opinions from those who give the webinar a listen, so please make use of the comments section of this post to leave your thoughts. All critiques, both positive and negative, are welcome, so please don’t be shy…

Click the link below to view the webinar.

Thursday, September 17, 2015

Reminder-Live Webinar Tonight, Thursday, September 17 at 8 PM Eastern Time

Just wanted to remind everybody about tonight’s iConquerMS webinar, in which I’ll be joined by naturopathic physician Dr. Deneb Bates and the Accelerated Cure Project’s President Robert McBurney for a 60 minute discussion on “Besides My MS Medications, What Else Can I Do?” We’ll be exploring complementary and alternative medical approaches to treating MS and its symptoms, and topics will include biotin, diet, vitamins and supplements, and other integrative approaches.

In order to attend the webinar, please register (click here) to receive an email with instructions on how to connect to the event Thursday evening. During the registration process you’ll be able to submit questions of your own. There are already several hundred people registered, but don’t worry, there will be plenty of room for everyone. Please don’t forget to use deodorant, though, it could get pretty crowded and I'm sure you wouldn't want to offend.

I told the organizers that I thought it would be cool if I did the entire webinar in a fake French accent because, you know, it’s classy. Sadly, they said no, and attendees will instead be subject to my glorious native New York intonations. So come on, youse mugs, register now or I’ll hit you so hard that your kids will come out shaking…

UPDATE: here are a list of the links that were put up on the screen at the end of the webinar. Several folks have asked that they be made available…

American Association of Naturopathic Physicians


International Multiple Sclerosis Management Practice

Accelerated Cure Project

Tisch MS Research Center

Saturday, September 12, 2015

Please Join Me for a Live Webinar on Complementary and Alternative Medicines In the Treatment of MS, This Thursday Night, September 17, 2015, at 8 PM Eastern Time.

This coming Thursday, September 17, 2015, I’ll be participating in a live 60 minute webinar discussing the topic of complementary and alternative medicines in the treatment of MS, held by the iConquerMS initiative (click here). I’m a big supporter of iConquerMS, a patient driven research project, and I urge everybody to visit their site, join up, and participate in helping to rid the planet of this terrible disease.

During this Thursday’s webinar I’ll be joined by Dr. Deneb Bates, the Naturopathic Doctor who works at the MS clinic at which I am a patient, the International Multiple Sclerosis Management Practice here in New York City (click here). Dr. Bates is among the sharpest doctors I’ve ever worked with, and believe me, I’ve worked with a lot of them given the wild and wacky nature of the Three-Headed Beast of an illness that is trying to have its way with me (click here). Moderating the event will be Robert McBurney, the President and CEO of the Accelerated Cure Project, the extraordinary nonprofit MS research group that is facilitating the iConquerMS project.

I expect the webinar to include a wide range of topics, including the use of diet and supplements in the treatment of MS, many of the popular alternative Multiple Sclerosis treatment methodologies being utilized by the MS patient population, and the importance of taking an integrative medical approach to treating the disease. There will be ample time for questions and answers, and by registering for the webinar online (click here) – be sure to hit the "register" link on the webinar info website – you’ll have the opportunity to submit questions on your own particular topic of interest. There will also be an interactive chat box included on the webinar screen, allowing attendees to submit questions during the live event.

Unfortunately, my request that Dr. Bates and I get to sing some of my favorite tunes from “West Side Story” and then maybe reenact the “I Coulda Been a Contender” scene from On the Waterfront (click here) during the webinar were respectfully denied, but I I agreed to do the event anyway. Hey, no sacrifice is too great for the cause, so please register and actively participate by submitting your questions…

UPDATED 9/13/2015: For those who cannot attend the live webinar, it will be archived for later viewing/listening. I'll post a link to the archived webinar as soon as it's posted to the net.

Wednesday, September 2, 2015

Thoughts Formed While Staring At The Ceiling

I’m baaaaack! Due to circumstances beyond my control, it’s been quite a while since I posted anything to this blog. My long absence from the blogosphere (is there an uglier word in the English language?) was not due to alien abduction, time travel, or simple lack of interest, but rather because I was, in a word, sick. Sicker than normal, that is. So sick that I could barely get out of bed for about six weeks, feeling, as they say in the hallowed halls of medicine, horrendously yucky.

Turns out that my case of the horrendous yuckies was caused not so much by my mysterious and ever progressing neurologic crap, but rather an obstinate sinus infection and – mostly – my completely messed up endocrine system, which decided to go utterly out of whack. Take it from me; having an utterly out of whack endocrine system is definitely not more fun than a barrel of monkeys, or even one monkey with or without a barrel. After two rounds of antibiotics and much tinkering with my various endocrine meds, I’m finally feeling halfway human. Since there are strong suspicions that I may only be half human anyway, things are definitely looking up.

Speaking of looking up, my six weeks spent in bed doing my best impersonation of an area rug gave me plenty of time to do just that, look up and study the intricacies of the ceiling over my bed. Since that pastime got old pretty fast, instead I couldn’t help but start peering deep within my own brainpan, an activity that comes dangerously easy to me anyway, given my lifelong proclivity for introspection. While there are plenty of interesting nooks and crannies inside that creaky old noggin of mine, there are also all kinds of surplus flotsam and jetsam in there as well, along with some scary beasties that are better left undisturbed lest they wrap their tentacles around your naughty bits and begin taunting you with really bad impersonations of Ernest Borgnine in “Marty” – a wonderful movie which if you haven’t seen already I highly recommend you watch right this second (click here).

Also found stuffed in the neglected dusty and cobwebed steamer trunks of my mind were memories, lots and lots of memories of people, places, and things, some of which hadn’t seen the light of day in decades. It’s amazing how many seemingly trivial moments remain captured in crystal clarity in our heads long, long after they occurred. Some were a treat to revisit, others not so much. Nevertheless, all of this mental poking, prodding, and reflecting done during my enforced period of respite resulted in some observations on life with and without chronic illness, some of which I thought I’d share in an attempt to make my time spent infirm not a complete waste. Some of the these may be trite and/or unoriginal, others a bit more significant, but I think all are true. Do with them what you will, but please keep your Ernest Borgnine impersonations to yourself…

♦ Time flies even when you’re not having fun. I’ve been diagnosed with “atypical” MS for over 12 years, and have been dealing with various troubling symptoms for longer than that, during which time I’ve lost the use of the entire right side of my body and an increasing amount of my left, and still it feels like all those years went by in the blink of an eye. Time is every person’s most precious possession, and each passing moment diminishes our allotment of this priceless endowment, never to be recovered. Time spent wallowing and worried over things inconsequential is time indeed wasted, and in the grand scheme of things almost all circumstances and situations except the absolutely most dire turn out to be inconsequential. With few exceptions, lost loves give way to new romances, lost jobs to fresh opportunities, lost monies to refilled coffers. As long as a person is of sound mind and body the power to reshape their destiny is entirely theirs to command. Once recognized, past mistakes and self-destructive patterns can serve as illuminating roadmaps to a more fulfilling future. Not that the losses shouldn’t be felt, but letting those hurts linger and poison future moments, moments filled with endless possibility, amounts to time spent in a prison of one’s own making. Regrettably, a lesson I learned far too late in life. Another lesson I learned far too late in life is that trying to put your underpants on over your head is but one more terrible waste of time. Go figure.

♦ Judging people by their outward appearance is about as foolhardy as it gets, unless they are blood spattered and brandishing weapons. As I’ve previously recounted on these pages, at one point in my life I found myself an unwilling resident of the state of Florida, very much a stranger in a strange land. It seems I shared little by way of aesthetic sensibilities with most of the other residents of The Sunshine State, and for quite some time I felt extremely isolated. As I got to know people, though, most of whom looked and thought very differently than the people I was used to socializing with in New York or Boston, I came to realize that many of them were full of delightful surprises. One very burly and proudly blue-collar man, who at first glance would seem to be the antithesis of the artsy types I hung out with up north, turned out to be one of the most interesting and entertaining friends I’ve ever known. One thing I realized we shared early on was a taste for booze and bars, and on one of our first nights out on the town together, after downing a few, he confounded me by suddenly spouting by rote memory verse after verse of Rudyard Kipling poetry, recited with a heartfelt vigor that left me dazzled. “If you can make one heap of all your winnings, and risk it on one turn of pitch and toss, and lose and start again at your beginnings, and never breathe a word about your loss…”. During the rest of my Florida years we would have quite a few inebriated adventures together, and through them all I’m sure I learned more from him than him from me. I haven’t seen or spoken to my friend in at least 16 years. Denny, if you’re out there, thanks for the surprises and all the good times.

♦ On that note, it’s vitally important to let the people who brighten your world know just how much they mean to you. No need to get all gushy and maudlin, but acknowledging the good people now will save you regret later on. I had the good fortune of working closely with two of the most brilliant people I ever met, at a major audio and video production studio here in New York City. When I first received my MS diagnosis they were both visibly upset and tried to express their concern and sympathy as best they could, genuinely chagrined at the bad break I’d received. Well, 12 years later I’m still here and they’ve both since passed away, one with shocking suddenness and the other after a long illness. I never really expressed to either one how special I thought they were, at least not directly. We cry at funerals not for the departed but for ourselves, for everything left unsaid and for the part of us the lost take with them. I have an awful lot of phone calls I need to make.

♦ While we are still on the subject of people, although it is foolish to judge folks by their outward appearance, judging them by the company they keep can save you a whole lot of trouble. Birds of a feather, as they say. I once had a long-term relationship with a woman who, on paper, seemed almost perfect. She was extremely easy on the eyes, had an interesting, even noble profession, was possessed of a sharp mind and a keen sense of humor, owned two great dogs (a big plus in my book), and had wonderful parents. The only thing that bothered me about her from the outset was her friends. Though this may sound impolite, they all made my skin crawl and seemed to me on the whole to be a thoroughly distasteful bunch. The passage of time would confirm my instincts about pretty much the entire crew, and despite any initial illusions otherwise, my ex turned out to be a great fit among them. Of course, in retrospect, early on I chose to willfully ignore copious signs of this woman’s toxicity, for I was truly a fool for beauty. Unless extraordinarily motivated, people generally stay true to their nature and if you think that you are somehow going to change them you’re cruisin’ for a bruisin’… Years later I married a woman with a kind heart and genuinely good soul, beautiful both inside and out. I suppose the universe taught me a lesson I desperately needed to learn, and boy, I sure am glad I learned it.

♦ Chance plays a tremendous role in shaping the contours of a life, but there are key moments when decisions made or not made, or actions taken or not taken, can change the whole ballgame. How important it is to recognize those moments and in doing so bend the fates to your favor. As a young man I suffered greatly from HUMA (Head Up My Ass) syndrome, and lived my life with all the conviction of a fart in a windstorm, mindlessly rather than mindfully. Though I harbored grand dreams of fame and fortune, I spent far too much time dreaming rather than doing. I did manage to eventually find success in what many people consider a “glamour” industry – video and television production – but I always had gnawing within me the feeling that I was meant for other things. When my illness forced me to “retire” and eventually start this blog, I discovered a much truer me, a me that had been MIA for decades. Fear, I now realize, played a tremendously negative role in the direction of my life, fear of both success and failure, playing itself out in too many ways to count. Lying sick in bed this last month and a half I sifted through all of the chances missed and all of the roads not taken, almost all due to lapses in confidence, neurotic apprehensions, or sometimes simply the weight of inertia, and I was overwhelmed by the sheer magnitude of all of the collected “what if’s”, a latticework of paths left unexplored. Who knows if any of them any might have led to a place without MS? In a weird twist of fate my illness has resulted in some of my dreams finding realization, but geez, the price has been steep. I suppose such things don’t come cheap, though. Unless, of course, you’re a Kardashian.

Okay, with that I’ll leap off of my soapbox (as if I could even get on a soapbox, or leap). Thanks to all of you for taking the time to read Wheelchair Kamikaze, and for commenting, sending notes, and breathing life into this thing. You’ve given method to the madness of my getting sick, and for that I am eternally grateful. When they find a cure for this whole MS thing you’re all invited over to my place for a shindig that will leave my neighbors pissed off for years…

Wednesday, July 29, 2015

Moments of Clarity – Repost

(Sorry I've been scarce lately, been fighting a really bad case of the creeping cruds that has kept me in bed the last few weeks. Fever, no appetite, weakness, all on top of the progressive MS stuff. Needless to say, not much fun at all. Just started my second round of antibiotics, so I expect things will get better soon. In the meantime, lest you think I simply vanished into the ether, here's a favorite old Wheelchair Kamikaze post of mine, back from December 2009, when this blog was not yet one year old. Rereading this essay after all these years reminded me that I really need to get back to my meditation routine and Zen Buddhist leanings, as I've let them slip these last few years. Anyway, hope you enjoy, and I'll  post something new just as soon as I'm feeling up to it…)

Receiving a diagnosis of MS or any serious illness is a reality shattering event. There you are, going about the business of day-to-day life often as if on autopilot, when a huge hairy beast steps into your path, grabs you by the ankles, and drags you kicking and screaming into some strange new dimension. Suddenly, the world is a different place, everything that you took for granted now imperiled, your expectations for the future warped beyond recognition. What once may have seemed quite orderly has now fallen victim to chaos, and however composed one might appear on the outside, on the inside pandemonium has gained a foothold.

Even after many years of dealing with chronic illness, the chasm between what was expected and what has been received can be tremendously disorienting. When dealing with a progressively disabling illness like MS, reality is a moving target. It seems that just when you acclimate to your current condition, some new symptom or event crops up to tear apart even that impermanent reality. The human mind craves at least a minimal amount of order, but in the life of a chronically ill patient, disorder often rules the day.

Lately I've been awash in a surplus of muddle and inner tension. The relentless progression of my MS, my dissatisfaction with the results of my cataract surgeries (which I'd had such high hopes for), and a bunch of other nagging health issues have combined to turn the universe into an unfriendly swirl of doubt and confusion. The coming of another holiday season, which starkly marks just how far my disease has progressed compared to holidays past, has also taken its toll. What is this treacherous path that I've been forced to follow? Is there some purpose that can be divined from it? And can this path possibly lead to a destination less ugly than the one I anticipate?

As threatening skies have gathered, I've managed to find some solace by taking shelter in a moment I experienced many years ago, a fleeting instant of understanding, a flash of insight that was gone before I could grasp it, a momentary comprehension that there are indeed patterns and reason and logic that lie just beyond the abilities of our puny, inadequate brains to realize. 

I think I've experienced such a moment only once. 

In late November, 1993, I was driving back from Key West to Miami in the company of my extremely difficult but very attractive girlfriend, after spending a slightly debauched but life-affirming weekend at the southernmost town in the United States. Back then Key West still had the vestigial feel of the Bohemian town of last resort it once was, a feel which I understand is tragically now lost. I drove in the early dusk of one of those perfect tropical winter afternoons, the sky an endless blue, the balmy air tender as a gentle peck on the cheek. The convertible top of my little red sports car was down, its engine singing its satisfying throaty growl, King Pleasure's "Moody's Mood for Love" pouring from the speakers which lay embedded in the car's head rests, cleverly placed there so the music could be heard above the noise of the open road. 

The late afternoon sun infused everything with a glowing pink and gold. The road we followed was on one of the smaller islands that make up the Florida Keys, just a little spit of land less than a half-mile across, the Gulf of Mexico a few hundred yards to my left and the Atlantic Ocean the same distance to my right. The air whipping around us tasted like the ocean, and with one hand on the steering wheel and the other feeling the vibrations of the motor through the stick shift, all of my senses were full. 

I started to say something to the girl, and glanced over at her sitting to my right. Though she wore big dark sunglasses, I knew in an instant she was dozing, her head cocked gently to one side, her long strawberry blonde hair playing with the wind. In the amber warmth of the setting sun, the sight of the girl and the feel of the car and the sound of the music stirred some secret part of my soul. 

I took a breath, and quite suddenly everything stopped. The music and the car and the road around me fell silent, I didn't exhale, I couldn't exhale, the girl and her tousled hair and the automobile and the sky and the world around us frozen for a pregnant instant. In that momentary pause, that crack in time, I flashed upon the unexpected understanding that I might have a chance at figuring it all out, that there could be significance and purpose to the teeming chaos that makes up a life, that the path upon which I tread might actually have some direction. 

And then it was gone, everything set back in motion. King Pleasure sang another note, my heart registered a beat, the girl stirred, and the march of life continued once again, just beyond the reach of comprehension. I exhaled. 

Well, the car was sold about a year later, and the girl and I didn't last half that long. I’ve heard that Key West has since succumbed to the inevitable rot of commercialism and is no longer the wonderfully strange little place I so loved back then. But for one single transcendent moment, all of these elements came together in a way that still keeps me pondering, in a tableau forever imprinted in my minds eye. In times of sadness, or trouble, or remorse, or confusion, I often retreat to that moment and somehow find comfort in the wisp of insight into the wholeness of all things that it provided. 

Zen Buddhists refer to these brief moments of understanding as "kensho". They call a deeper, more lasting enlightenment "satori". Whatever its name, I'm grateful to have experienced my moment as its impact has served me as an anchor through troubled times. Whenever the burden seems too great, the road too twisted, when I start getting lost in abstractions like "fair" and "not fair", I can slip back into that moment, and though I can't re-create the experience, the simple knowledge of it and the sense that there are indeed unseen patterns within all of the seeming randomness grants me at least a few measures of serenity.

I guess some would call it faith...

Here's King Pleasure and Annie Ross, singing the song that accompanied my moment of clarity all those years ago… BTW, the title of the track is "Moody's Mood For Love", just for the record...

Monday, July 6, 2015

Back from the NIH: Unfortunately, Nothing Dramatic to Report

Well, I’m back from my sojourn to the nation’s capital in search of answers regarding my ever progressing and relentlessly stubborn illness. Sorry it’s taken so long to report back, but the trip left me pretty exhausted, and for the last week or so I just haven’t been myself. Oddly, I think I’ve been Ethel Merman, but that’ll have to be a subject for another blog post…

On the evening of June 23, Karen and I packed ourselves into a rented wheelchair accessible van and hoofed it down to Bethesda, Maryland (just outside of Washington DC) for a visit to the National Institutes of Health, where I was examined by members of their neuroimmunology and endocrine teams. I haven’t yet received a final report, but it doesn’t look like any new discoveries or insights were made about my condition. Despite the fact that I try to keep my expectations in check, this time around I thought that maybe, just maybe, some aspect of my disease would raise its ugly little head high enough to be recognized by one of the doctors handling my case. I’ve been feeling particularly cruddy the last several months, so I thought that perhaps some kind of shift might have occurred that would lead to an “aha” moment. Alas, it appears ‘twas not to be…

The NIH is the US government’s primary medical research organization, and is staffed by some of the best minds in the business. As a taxpaying US citizen who also happens to suffer from a chronic illness (and who is also a bit of a medical research wonk) my visits to the NIH are always interesting, if only to see firsthand our tax dollars being put to good use. For those qualifying for the NIH’s many ongoing studies, treatment is absolutely free, and the NIH Clinical Center, located on a sprawling campus packed with research facilities, is top-notch. Though I’m no longer part of an NIH study, once you’re in you’re in, and I’ve been making semi regular visits to Bethesda ever since 2009 in my quixotic quest for answers to my medical conundrum. Hey, I used “quixotic” and “conundrum” in the same sentence! Bonus points!

This time around I saw the MS neuro who is in charge of my case, Dr. Irene Cortese, as well as a member of the NIH’s endocrine staff. I underwent a full neurologic exam, had MRIs done of my brain, spine, and pituitary gland, lots of blood taken, and had few functionality tests thrown in just for good measure. All of this over the course of two and half days, which made for a pretty grueling schedule. Especially since the NIH doesn’t keep to my nocturnal ways, forcing me to acknowledge that yes, there is such a thing as morning. As a matter of fact, on day two of our visit I had to get up at the ungodly hour of 5 AM to be ready for an early morning MRI appointment, and getting up at 5 AM is NOT in my repertoire of favorite activities. As a matter of fact, going to bed at 5 AM is more my style. Maybe the NIH should have tested me for vampirism. Just call me Count Gimpula.

As I mentioned, I have yet to receive a final report, but I do know that my MRI images were once again unchanged, which is no surprise since they haven’t changed at all since I was diagnosed over 12 years ago. Same two lesions as always, one tiny spot in my brain (again termed “insignificant” and perhaps not even related to my illness), and a big juicy one right at the top of my spinal cord, where it connects to the brainstem. Not a great spot for a very invasive lesion, as there’s lots of important stuff going on in that area with little extra real estate available for any rewiring or other physiological workarounds. My neurologic exam confirmed that I have gotten significantly weaker over the last year and that my muscles have noticeably atrophied. Some of this atrophy might be attributed to all the weight I’ve lost, which in turn might be attributed to my endocrine problems. No surprises there, either.

Speaking of my endocrine problems, I was seen by a member of the NIH’s endocrine staff, a young “fellow” (this particular fellow happened to be a woman), who while quite diligent didn’t seem to want to even try to integrate my myriad endocrine dysfunctions with my neurologic problems. This is one of my pet peeves with modern medicine – it’s become so damned specialized that each individual specialist views the patient through their own very narrow lens, making it almost impossible to find someone to put together all the pieces of the puzzle. The situation is kind of like that old parable about three blind men trying to describe an elephant. One feels the trunk and thinks the beast is a very large snake. Another bumps up against the elephant’s leg and declares that he’s dealing with a tree. The third reaches out and touches the animal’s side and says he’s come across a wall. All very reasonable assumptions, but all quite wrong.

And here I am, presenting with a wide assortment of debilitating symptoms that to me scream “weird systemic disease”, but each specialist I see concentrates only on one very specific aspect of whatever it is that ails me. Sure, I suppose it’s possible that my neurologic symptoms are completely divorced from my endocrine symptoms which are completely divorced from some of my other strange findings, but common sense would seem to dictate that there must be some common thread running through all of them. But I guess I’m just the Count Gimpula Elephant Man who every now and finds himself turned into Ethel Merman, so why look any further? Shouldn’t I at least have my own reality show?

My pituitary MRI showed that I do not have a pituitary tumor, which counterintuitively might actually be bad news, since pituitary tumors are almost always benign and very treatable, so if I did have one something could be done about it. I do suffer from something called Empty Sella Syndrome (click here), meaning that the space in my skull that should be taken up by my pituitary gland is largely vacant due to the fact that my pituitary has shrunk or become flattened. Why? Who knows? I’ve never gotten an answer. I’ve known about this condition since around the year 2000, but somehow this further physical weirdness never seems to get fully factored in to my complicated diagnostic mess. Really though, since it's so evident that I'm the Ethel Merman Count Gimpula Elephant Man, why bother?

So, there you have it, a nutshell summary of my trip to the NIH. The actual trip itself was okay, aside from the fact that the hotel in Maryland decided to forget that I’d reserved a handicapped accessible room so I could barely get into the bathroom the first night of our stay (we didn’t arrive until after 1 AM and had to be out and about by midmorning the next day, so the mistake wasn’t calamitous), and getting back was a slow 7 ½ hour crawl through Northeast corridor traffic, which was none too kind to my crumbling hips. Still, nothing ventured, nothing gained, even if it doesn’t look like very much was gained. Who knows, maybe once the NIH doctors convene and go over all of my test results something will have revealed itself and I’ll be pleasantly surprised with the final report, but I’m keeping my expectations to a minimum.

Much thanks to everybody who sent me well wishes and notes of support, all of them greatly appreciated. In return, I give you this very private video of me recovering during the week after my return from my visit to Bethesda, when I still wasn’t quite myself…

Tuesday, June 23, 2015

I’m Off to the National Institutes Of Health…

Just wanted to check in lest anybody think I vaporized or otherwise met with misfortune. I’ll have to keep this relatively short, as I’m scrambling to get ready to go to the National Institutes of Health (NIH) Clinical Center, in Bethesda Maryland. The NIH is the US government’s primary medical research organization, and they maintain a state-of-the-art clinical complex on a sprawling campus just outside of Washington DC.

I’ve been seen by the doctors/researchers at the National Institutes of Health (NIH) three times since 2009, when I was part of an NIH study that sought to identify patients with clinically definite multiple sclerosis so that these patients could be used in future MS trials. The NIH conducted this study because they had found the misdiagnosis rate of MS so high in some of their previous research efforts that the number of wrongly diagnosed patients were skewing the results of those trials. In some cases up to 15% of trial subjects were ultimately found to have diseases other than multiple sclerosis. Yes, this number is alarmingly high, and if you think you might be misdiagnosed I wrote an action-packed Wheelchair Kamikaze essay on the subject a few years ago, which you can read by (clicking here).

Being nothing if not pure of soul and kind of heart, I of course had only the noblest intentions for joining the trial, wanting simply to help further the exalted cause of science. Okay, before you’re overcome with nausea I’ll admit to other, less altruistic motives which might have played just a teensy weensy role in my schlepping the five and half hours from New York City to the wilds of the nation’s capital for a medical workup. I’d long suspected that I had, in fact, been misdiagnosed and I knew that as a subject in a study designed specifically to identify clinically definite MS patients I’d be getting a thorough going over by the world-class neuroimmunology team at the NIH, the prospect of which my obsessive impulses quickly latched onto, like a lonely nebbish wrapped around the finger of a femme fatale. Jeez, what an awful metaphor…

Okay, so maybe I’m not so pure of soul and kind of heart, but a selfish bastard interested in nothing more than me me me! Holy Mother of Moses it feels good to finally come clean! By 2009, just six years after my initial diagnosis, I had already been in a wheelchair for a year and despite undergoing all kinds of treatments both mainstream and alternative I was only getting worse. So, yes, I was desperately seeking answers, dammit, and I was dead set on getting them come hell or high water. There, I said it, and I’m glad I said it! Hey, stop looking at me that way or your face will freeze.

After several days of intense poking, prodding, zapping, and scanning, the assembled big brains at the NIH concluded that indeed I did not fit the diagnostic criteria for any form of multiple sclerosis, though they couldn’t come up with any reasonable alternative diagnosis either. Drats! I floated the idea that I might just have a severe case of the cooties, but the esteemed physicians quickly shot down that notion, too. Double drats! An unsatisfying result, to say the least, though the doctors did state that some weird form of MS certainly couldn’t be ruled out. So, as I’ve written previously, my doctors and I have agreed to go with an unofficial diagnosis of PPMS, which in my case stands for the Peculiar Paralysis of Marc Stecker. And, no, I won’t stop making that joke, because it’s just so damned clever.

Now, six years after our first journey to Bethesda, Karen and I will be packing up a rented wheelchair friendly van on Tuesday for yet another sojourn down I-95 to the NIH. Well, actually, Karen will be doing most of the packing since I'm, you know, a complete freaking gimp. I will, though, be driving my wheelchair with reckless abandon around the van as she packs it, in an attempt to keep away the roving hoards of New York City's notorious ne'er-do-wells, these days composed primarily of Wall Street bankers looking to suck every last dime from an almost extinct middle-class…
As I recently detailed in “Attacked by a Three-Headed Beast” (click here) my disease has been giving me quite the thorough ass whupping for the last five or six months, with my neurologic problems compounded immensely by an endocrine system gone berserk and a degenerative bone condition that delights in making my hips and shoulders feel like they are made out of red hot razor blades and shards of glass. Last month I contacted the good doctors at the NIH and told them of the situation, and they invited me to come down for another good going over. This time around I’ll be seeing not only the neuroimmunology team but also their endocrine doctors, and I’ll be getting an extensive series of MRIs and whatever other medical tests are deemed necessary. We’re scheduled to return to NYC Friday evening.

I’m hoping that now with all of the elements of my physical decrepitude apparently coming to a head, something will finally show itself that will elicit a flicker of recognition in at least one of the assembled NIH doctors and researchers and we’ll finally start to unravel the mystery of me. My hunch is that my endocrine problems may be playing a much larger role in my physical mess than was previously thought, a possibility that both my top notch New York City neurologist and endocrinologist agree needs to be explored. The general consensus now seems to be that I probably do have some form of progressive MS, but also some widespread systemic dysfunction that is making my body go kablooey.

Yes, in my case kablooey is an official medical term. In all probability my pituitary gland and/or hypothalamus has gone completely kablooey, which has in turn made the rest of my endocrine system absolutely whopperjawed, leading to a mishmash of messeduppedness that has left me feeling like absolute dreck, weak as a batch of watered-down hooch, muscles as wasted as Dean Martin on a bad night, and down about 25 pounds in only the last three or four months. I have lodged an official protest about all of this with the Cosmic Complaint Department, and if I don’t get some satisfaction soon somebody is going to get a severe tongue lashing, the likes of which hasn’t been seen since Groucho Marx made his introduction to Margaret Dumont in “Duck Soup”:

Yes, if my disease doesn’t want to leave in a taxi, it can leave in a huff. If that’s too soon it can leave in a minute and a huff… One way or another, though, it’s gotta go. If it’s one thing I can’t stand it’s a horrendous, crippling illness that overstays its welcome like a houseguest who’s searching the cupboards for more Cheez Whiz while you’re putting on your pajamas. Jeez, what an obvious metaphor…

I’ll report back upon my return from the hallowed halls of the NIH…

Friday, June 5, 2015

Recent Research on HSCT, the Stem Cell Therapy That May Soon Change the MS Treatment Landscape.

(Please note: the following article is quite long. Though it covers a very important topic and I’ve tried to make it as accessible as possible, I’d suggest getting comfy and grabbing a nice beverage before diving in…)

Over the last six months or so, there has been a wave of new and encouraging research published regarding HSCT (Hematopoietic Stem Cell Therapy) for the treatment of multiple sclerosis. HSCT is the type of stem cell therapy in which a patient’s existing immune system is completely eradicated through the use of powerful chemotherapy drugs and is then rebooted via an infusion of their own stem cells. Some of the results reported have been nothing short of astounding, and even previously skeptical neurologists are now being forced to consider HSCT as a potential game changer that may significantly impact the MS treatment landscape sometime in the not-too-distant future.

So that this article doesn’t take on the length of a Russian novel, rather than getting into the detailed specifics of what HSCT is and isn't I’d like to concentrate on the recent HSCT published research and its implications. For a comprehensive overview of HSCT, please read my previous post on the subject by (clicking here). For a rundown on the different types of stem cell therapies currently being trialed in MS, please (click here).

Just a few things before delving into the research. It’s vitally important that patients not confuse HSCT with regenerative stem cell therapy, which attempts to directly repair the central nervous system damage done by MS. HSCT is a completely different approach with a completely different goal. Regenerative stem cell therapy, such as that being trialed at the Tisch Center in New York (click here) generally does not involve any direct manipulation of patients’ immune systems. HSCT, on the other hand, is entirely directed at providing patients with a new immune system after completely wiping out their presumably defective existing set of immune cells. The two treatment approaches couldn’t be any more different; their only similarity is that they both use stem cells in an attempt to combat multiple sclerosis, albeit in very different ways.

There are currently two forms of HSCT being trialed on MS patients, termed the myeloblative and non-myeloblative treatment protocols. The myeloblative protocol uses a combination of very strong chemotherapy drugs to completely ablate (a fancy word for “destroy”) patients’ immune systems as well as their bone marrow (immune system cells are manufactured in the bone marrow). Non-myeloblative HSCT uses a gentler chemotherapy regimen that takes down the immune system but leaves bone marrow intact. There is some debate among researchers as to which treatment protocol is best suited for combating MS, an issue that is still being sorted out.

There’s also the question of which patients are most likely to respond to HSCT. Because HSCT is directed entirely at replacing a patient’s presumably defective immune system with a new one that doesn’t attack the body’s own nervous system cells, the prevailing thinking is that only patients displaying the hallmarks of immune system driven multiple sclerosis would likely respond well to the treatment. With this in mind, most HSCT research and treatment centers restrict the treatment to patients with active inflammatory MS, meaning that eligible patients must have recently experienced multiple MS relapses and/or displayed enhancing lesions on their MRI images (enhancing lesions are indicators of immune activity within the central nervous system). Since this excludes many secondary progressive (SPMS) and most primary progressive (PPMS) patients, who experience increasing disability in the complete absence of relapses or enhancing lesions, these requirements are understandably the source of much consternation among this patient population (myself included), who are currently presented with no effective treatment options. More on this later.

With these issues in mind, let’s take a look at some of the recently published HSCT research and explore the issues they raise…

A study out of Chicago that used the gentler, non-myeloblative form of HSCT on 151 MS patients (123 RRMS, 28 SPMS) resulted in some startling results on the RRMS patients taking part in the trial (click here). Not only did the vast majority of these patients see their disease stop in its tracks, but a large proportion also saw their levels of disability improve. Four years after treatment, 80% of trial subjects remained relapse free, and 87% showed no signs of disease progression. Furthermore, and perhaps even more astounding, there was a decrease in the disability scores in 50% of patients two years after treatment, and in 64% of patients four years after undergoing the treatment protocol without any further follow-up treatment (click here). Given that a reduction in disability scores is almost unheard of when treating MS patients, these are eye-popping results, to say the least, tempered only by the fact that the SPMS patients included in the trial were reported to have shown no benefit.

This trial was headed up by Dr. Richard Burt of Northwestern University, a leading HSCT researcher. I tried to reach out to Dr. Burt via email with several questions before writing this piece, but unfortunately received no reply. A very informative interview with Dr. Burt is available by (clicking here). If anybody out there knows Dr. Burt, please tell him I’m still patiently waiting for his email reply, fully understanding that he’s a very busy man. In the meantime, if Dr. Burt should find himself in New York City and happens to get run over by a speeding wheelchair, I’ll swear on a stack of research papers that I had nothing to do with it.

A smaller, 25 patient study, called HALT-MS, which used a harsher myeloblative chemotherapy regimen on patients with highly active relapsing disease who had failed to see benefit from at least two previous MS drugs reported impressive interim results (click here). Though study subjects are scheduled to be tracked for five years, the results reported were a snapshot of these patients at the three year mark after treatment. After three years, 78.4% of trial subjects were reported to be free of any disability progression, relapses, or new lesions. Digging deeper into the results revealed that 90.9% of subjects were progression free and 86.3% were relapse free after 36 months. Very impressive.

I managed to get hold of the full published paper of this study, which indicated that although the numbers had not yet been fully crunched, it did appear that the effect of the treatment lessened with time, as fewer patients were reported to be free of signs of the disease at the four and five year marks (68.6% and 58.8% respectively). Again, though, these four and five year numbers were very preliminary, as not all patients had reached this duration after treatment.

A friend of mine, Dave Bexfield, who runs the terrific MS website/forum Active MSers (click here) was one of the participants in the HALT-MS trial. Five years ago Dave was getting absolutely slammed by his disease despite having been on a series of MS drugs. He qualified for the HALT-MS trial and received myeloblative HSCT. His tremendously aggressive RRMS was put entirely into remission for several years, but unfortunately, after five years, Dave recently reported that his disease has shown signs of reawakening (click here) and he is now planning on going on one of the newer, more powerful MS drugs. Dave told me that even though his disease has returned, he has no regrets about undergoing HSCT back in 2010. Without the treatment he is sure the disease would have by now left him completely disabled, and HSCT bought him time at a point when it looked like multiple sclerosis would get the better of him. There are highly effective treatment options available today that weren’t available five years ago, and despite the reemergence of some of his MS symptoms, Dave is very grateful to have taken part in the study.

An Italian team did a head-to-head test of the effectiveness of HSCT versus mitoxantrone (also known as Novantrone) on SPMS patients who had enhancing lesions on their MRIs (click here), the presence of which indicated that they still had an active inflammatory component to their disease.

Currently, mitoxantrone is the only approved drug for use on patients suffering from SPMS. It’s a fairly wicked drug, so toxic to the heart that patients can only stay on it for a limited amount of time. Additionally, mitoxantrone has been linked to a dangerous increase in specific forms of leukemia and lymphoma. As a result of this daunting side effect profile, the use of mitoxantrone has largely fallen out of favor with most MS neurologists.

This phase 2 study used an intensive myeloblative form of HSCT on a small group of study subjects (21, 17 of whom completed the study), and the primary endpoint of the study was a reduction in the number of enhancing lesions detected by MRI. Four years after treatment, HSCT resulted in a 79% reduction in new lesions as compared to those treated with mitoxantrone Unfortunately, no difference in disease progression was reported, but the study was not ideally set up to detect changes in disease progression, a metric which has proven to be very difficult to track even in studies specifically designed to detect disability progression. An editorial accompanying the study, which adds commentary to the statistics provided in the study abstract, should be of interest to all readers (click here).

A very small study out of Hamburg, Germany attempted to directly address the difference in effectiveness of HSCT between RRMS patients with enhancing lesions and progressive MS patients not displaying lesion enhancement (click here). This very small 10 patient trial (four progressive MSers, six with RRMS) compared the results between the two groups two and half years after treatment. The researchers found that although five of the six RRMS patients did show benefit from treatment, none of the progressive patients followed suit. It should be noted that the RRMS patients were considerably less disabled at the start of the study than the progressive test subjects (EDSS 4.25 versus EDSS 6.25).

Previous studies have found that approximately 40% of patients with early PPMS display enhancing lesions on their MRIs (click here) and the presence of such lesions early in the disease may be an indicator of a more aggressive disease course. Patients undergoing the transition from RRMS to SPMS frequently continue to have enhancing lesions for some time after their disease has gone mostly progressive. Remember, enhancing lesions are a sign of active inflammation in the central nervous system, an indicator that the immune system is playing a primary role in driving the disease forward. Since HSCT directly attacks the peripheral immune system, it stands to reason that the treatment would be most effective on patients with active immune system involvement. In the later stages of SPMS, and in the majority of cases of PPMS, it appears that some other as yet undiscovered neurodegenerative process is at work, a process which seems impervious to treatments targeting the peripheral immune system. Studies testing some of the latest potent immunosuppressive drugs – which are highly effective in treating RRMS – on progressive patients have repeatedly failed, the latest such study involving the drug Gilenya (click here). For those of us with progressive MS such failures are very discouraging, and it is with great hope that I’ve looked for hard data supporting the notion that HSCT may benefit even hard-core progressive MSers, which leads us to the final study to be discussed…

Lastly, a study out of Moscow, Russia (click here) provided data suggesting that HSCT might be beneficial to progressive patients who don’t have enhancing lesions (as stated earlier, this includes many SPMS patients and the majority of PPMS patients). Unlike most HSCT treatment centers, the center in Moscow accepts patients with all types of MS (RRMS, SPMS, PRMS, and PPMS), regardless of whether or not they have enhancing lesions on their MRIs or have recently experienced relapses.. A total of 99 patients were included in the study, 43 with RRMS and 56 with various forms of progressive MS.

The summary of this study revealed that 49 months after undergoing treatment, 83.3% of RRMS patients and 75.5% of progressive patients exhibited no signs of relapses or disease progression. After eight years, 45% of treated patients exhibited an improvement in mobility scores, while 45% remained stable. These are compelling numbers, to say the least, particularly for someone like me who has never had enhancing lesions but has nevertheless experienced a constant progression of disability. So compelling, in fact, that I asked a physician friend of mine to provide me the full paper (I could have bought it myself for $40 but most doctors can get research papers for free, and, hey, that’s what friends are for) so that I could dig deeper into this apparently promising research.

Upon reading the full paper (sorry, I can’t link to it because of copyright laws) my enthusiasm was tempered a bit. Although the paper states that only 40% of study subjects had exhibited enhancing lesions before undergoing HSCT, it turns out that this statistic was based on a single MRI done soon before the patients underwent therapy. A single MRI can easily miss enhancing lesions, especially if patients had been on disease modifying drugs, which is why most HSCT centers require patients to have exhibited enhancing lesions and/or relapses (which are almost universally accompanied by enhancing lesions) at some point during the full year prior to treatment.

Additionally, the scope of the disease histories of the patients treated in Moscow varied widely and in many cases was severely deficient. The full paper clearly states this, saying that “patients enrolled in the study previously have been treated in different centers, and the information about disease activity prior in the disease course and its treatment was inhomogeneous and in some cases quite scarce.” I found this sentence to be disquieting, as without comprehensive disease and treatment histories it’s almost impossible to accurately assess the disease state of any individual patient suffering from an illness as complicated as multiple sclerosis. In fact, the abstract of the study, which is available to the general public, even warns that because of this dearth of patient info “comparison with the results in the literature should be done with caution.” This line throws up a big red flag, as the ability to compare and replicate results from study to study is among the foundational basics of the scientific method.

Furthermore, despite publishing statistics that appear to indicate the treatment as practiced in Moscow was effective across a wide spectrum of MS patients, the authors state that “the best candidates for transplantation seem to be relatively young patients with active inflammatory lesions of relatively short duration and rapidly progressive disease, but still low disability scores, unresponsive to conventional therapy.” This was among a number of apparently contradictory elements contained in this research that left me with more questions than answers. While these results certainly can’t be discounted entirely, without a comprehensive, detailed data set it’s impossible to properly discern their value and validity when compared to other studies, a point the authors themselves reiterate on several occasions.

Based on this accumulated HSCT research, certain trends seem clear. HSCT treatment definitely seems remarkably effective on patients with active inflammatory relapsing remitting multiple sclerosis, in the majority of cases resulting in complete remission of the disease for five or more years, sometimes accompanied by a reduction in disability as well. This is far more effective than any of the currently approved disease modifying drugs with the possible exception of Lemtrada, whose mechanism of action in many ways mirrors that of HSCT (for more information on Lemtrada, which was recently approved by the FDA and is covered by most insurance companies, please click here and here).

The safety profile of HSCT as now practiced seems quite robust. None of the above studies reported any HSCT related mortalities, although some serious adverse events above and beyond those that would be considered normal for patients undergoing chemotherapy were described (most in the form of opportunistic infections). Clearly, the decision to undergo HSCT should not be taken lightly, as at its heart the treatment involves using strong chemotherapy drugs to completely eradicate a patient’s existing immune system. Multiple sclerosis is serious business, though, and many patients would gladly accept this risk/reward scenario for a chance at complete remission lasting years or even decades.

Despite the fact that there are credible anecdotal reports on various HSCT websites from progressive MS patients who state that they’ve been helped by the treatment, and even though I’ve desperately wanted – for my own selfish reasons – to find research to back up such statements, I’ve yet to come across any hard data to back up these claims. Research has shown that the treatment can be effective on progressive patients showing signs of active inflammatory disease (enhancing lesions), but there is little hard objective evidence to support the notion that HSCT benefits patients experiencing progression in the absence of active inflammation.

Because the rate of disability progression varies widely from patient to patient suffering from progressive MS, and can sometimes temporarily “plateau” or even cease entirely, predicting disease progression can be quite difficult, even for patients themselves. Using myself as an example, my wife likes to chide me that I’ve been telling her that I’ll probably be bedridden in six months for the last five years. In order to properly assess the effectiveness of any treatment for progressive MS using disability scores as markers, studies would need to last much longer than the 2-3 years typical MS trials generally run, which is among the reasons why so few trials on progressive MS have been conducted. Even in the recent, much talked about successful study involving the use of biotin to treat progressive MS patients (click here), it was shown that after one year only 13% of patients taking placebo showed evidence of disease progression (compared to 4% taking biotin). I certainly hope that the patients with nonenhancing progressive MS reporting benefit do turn out to be success stories and that some form of HSCT does demonstrably prove effective on noninflammatory multiple sclerosis, but so far, at least, the data is lacking.

Also lacking is long-term data on the durability of HSCT derived benefits on patients of all stripes. One older study (click here) looked at patients with aggressive multiple sclerosis 15 years after treatment and found that 44% of patients who had exhibited enhancing lesions before treatment and 10% who didn’t remained progression free. It should be noted that the patients included in this study were treated with earlier, much harsher forms of chemotherapy, and one would hope that the refinements made in the technique more recently would increase durability.

Although all of the studies reported on in this article are considered early to mid-stage trials, they do suggest that HSCT is an extremely promising and potentially game changing treatment for multiple sclerosis. Further trials will be necessary before HSCT receives any kind of official approval for the treatment of multiple sclerosis. Although the treatment is being offered to patients in quite a few clinics around the world, the cost is quite high and most insurance companies will not cover the expense. Given the exorbitant cost of multiple sclerosis drugs, though, as more reliable data is released this situation should change. Paying $100,000 for a one-time treatment that keeps the disease at bay for even 5-7 years is far more cost-effective than paying the roughly $50,000-$60,000 per year currently charged for the FDA approved disease modifying drugs, a point that I’m sure will not be lost on the insurance company bean counters. It should be interesting to see how the pharmaceutical companies react to the HSCT threat to their bottom lines.

Those patients currently considering HSCT therapy should limit their choice of treatment centers to those which have extensive experience administering HSCT, as study after study has shown that safety and efficacy increases exponentially with the number of procedures done. This treatment is not to be taken lightly, and cutting corners for cost or convenience could prove quite the egregious mistake.

Given the current quickening pace of HSCT research, I would guess that the treatment could approach widespread acceptance sometime within the next five years, as long as the financial considerations of the pharmaceutical companies don’t get in the way (and that may prove to be a mighty big if). Based on the latest data, once approved HSCT should prove to be the most effective treatment available for patients with very aggressive active inflammatory multiple sclerosis, but will likely be reserved for those patients who have first failed current frontline therapies. As for patients with nonenhancing progressive disease, though objective hard data supporting the use of HSCT is lacking, given the anecdotal evidence I hope and expect expect future research will be done on such patients in an attempt to clarify the matter. The stakes are quite high for these patients, and even if the odds of success seem slight, the potential gains warrant further investigation. With neuroregenerative and neuroprotective compounds now in the research pipeline, and with HSCT offering so much promise, there is much reason for MSers to harbor hope for a brighter future.

Stay strong, my friends…

Geez, what was that I said about this article not becoming as long as a Russian novel? Maybe I should change my name to Tolstoy (many apologies to Tolstoy)…