Sunday, March 16, 2014

Pioneering MS Stem Cell Trial Needs Your Help!

I've very rarely used Wheelchair Kamikaze to directly appeal for charitable donations to help any individual research project, as I've always been extremely wary of abusing the trust that's been built up between me and my readers. Very recently, though, I've been alerted to a cause that I feel is so fundamentally worthwhile that I decided to devote this entire essay to making such an appeal.

It is my most fervent desire to see crippling neurologic diseases wiped from the face of the planet, and to put the nightmarish world of such diseases firmly in the realm of history. There is a long-awaited research project currently in need of funding that has the potential to radically change the MS treatment landscape. Before I get into the dollars and cents of things, though, let me first provide a little background.

One of the most cherished dreams of every patient suffering from the ravages of multiple sclerosis is to see their losses reversed, to one day triumphantly trash their canes, walkers, and wheelchairs; to at long last find eyesight restored, withered limbs strengthened, and numbed minds sharpened. For those whose lives have been victimized by MS, this is the stuff of reverie, a hope so precious that it can sometimes feel taboo to speak of for fear it may be crushed.

It is the power of such dreams that make the use of stem cells to treat multiple sclerosis one of the most tantalizing areas of study currently underway in the world of MS research. Stem cell therapy offers the hope of repairing damaged tissues in the central nervous system, thereby restoring function lost to the scourge of disease. All currently available MS therapies seek, at best, to put the brakes on disease progression. None are targeted at or capable of neural regeneration, the process by which damaged nerve cells might be fixed and lost function thus regained.

Despite exciting headlines and overhyped internet buzz, the reality is that research into the use of stem cells to repair MS damage is only now taking its first baby steps. Like most paradigm shifting scientific breakthroughs, the realization of the dreams for stem cell therapy will take time, the effort of brilliant minds, and money. There are currently only two FDA approved trials of the use of reparative stem cells to treat MS, one at the Cleveland Clinic and the other at the Tisch Multiple Sclerosis Research Center of New York (click here), which is associated with the clinic at which I am a patient.

Both studies intend to use stem cells derived from a patient’s own bone marrow in an attempt to repair the damage done by the disease. While the two studies each use a type of cell called mesenchymal stem cells, or MSCs, the study at the Tisch Center uses a more complex but also potentially more powerful approach. After more than a decade’s research by a team of scientists dedicated solely to stem cell therapies, led by Dr. Saud A. Sadiq, researchers at the Tisch Center have developed a proprietary method for turning mesenchymal stem cells into neural progenitor (NP) cells, a type of stem cell specific to the central nervous system (CNS) that, in theory, should be extremely effective at repairing CNS damage at the source of the problem.

The process begins by extracting bone marrow from each patient; the patient's mesenchymal stem cells are then separated out from this material. The Tisch Center then takes those MSCs, and, using a recently patented process, inducing them to transform into potentially more potent neural progenitor cells, which are then multiplied over several months in the laboratory. These NP cells will be injected directly into the spinal fluid of trial subjects, in three individual treatments, each given at three-month intervals. Preliminary studies using animals have provided very encouraging results, and nervous system damage has actually been reversed. The Holy Grail of MS research may finally be within sight.

The Tisch Center, which is an independent facility unaffiliated with any university or hospital, had been applying to get FDA approval of their proposed trial for many years. During this time, federal regulators diligently insisted time and time again that more evidence be provided and that further animal studies be done. Throughout this arduous process the staff of researchers at Tisch worked hard to refine their techniques and methodology, and produced increasingly convincing data. The Tisch Center finally received their long-awaited FDA approval in August, 2013. That glow you see coming off of your screen may be the first light of a radical new age in MS therapy creeping over the horizon.

Unfortunately, one major roadblock stands in the way of launching the now FDA approved Tisch Center MS stem cell trial: funding. Though the Tisch Multiple Sclerosis Research Center of New York is a registered nonprofit organization, almost all of its fundraising efforts thus far have gone into the research that has made this trial possible. The cost of the initial 20 patient trial will be about $600,000. Towards reaching that end, the Tisch Center has set up a funding page at the crowdfunding website Indiegogo, where people from all over the world can contribute any amount from one dollar to fifty thousand dollars or more to help set the trial in motion (click here). For those in the US, the Tisch Center’s foundation is fully 501(c)(3) compliant, so any donations made are completely tax-deductible. The Indiegogo campaign has a set goal of raising $300,000 by April 14, 2014 at 11:59 PM PT. The campaign has already collected over $100,000 in donations, so as of today there’s about $200,000 to go. Detailed information on how these funds will be spent is available on the Tisch Center’s Indiegogo funding page (click here).

It may seem strange that an FDA approved trial should lack sufficient funds to get started. The Tisch MS Research Center of New York is an independent research organization funded solely through charitable donations, a status that has allowed it to pursue audacious research goals, but which also means that it doesn’t have the deep-pocketed resources that a research group affiliated with a well-endowed university or hospital might. Medical insurance does not cover the costs incurred by patients undergoing trials, and researchers at Tisch consider it unethical to require trial subjects to pay for unproven treatments.

Traditionally, medical research has been funded by the federal government or, increasingly, by the big pharmaceutical companies. Ongoing battles over the US federal budget have resulted in the (in my opinion unconscionable) slashing of medical and scientific research funds to paltry levels (click here, here, and here), and thus far no pharmaceutical company has funded any trials using a patient’s own stem cells, quite likely because the success of such a trial could have a tremendously negative impact on the mega-profits many companies are making selling MS disease modifying drugs.

It is a matter of record that the pharmaceutical industry concentrates tremendous resources on influencing the FDA (click here, here, and here), and is actively lobbying to have the FDA declare that a patient's own stem cells are pharmaceutical products (click here, here, and here), an outrageous claim (again, my opinion) which would effectively shut down independent stem cell research organizations like the Tisch Center and place adult stem cell research firmly in the hands of Big Pharma. It is always vitally important to remember that these are public companies whose legal mandate is to continually expand their bottom lines, a motivation that sometimes puts shareholder interests at odds with those of the patient. Big Pharma innovations have dramatically increased the quality of life for some MS patients, but the reality is that conflicts of interest arise when there are billions to be made treating a disease, not curing it. Yes, I’m fully aware that to some the previous sentences may read like the rantings of a paranoid schizophrenic, but if I’m completely nuts, I’m in good company. Please read the material linked to above, which includes an opinion piece from the bastion of anti-capitalist radicalism that is the Wall Street Journal, and connect the dots…

As stated earlier, I’ve rarely used this blog as a platform to solicit donations for any cause or organization, but I feel funding the Tisch Center MS trial is tremendously important to people with MS and all those who love them. I’m sick of being sick, and I’m sick to death of watching many of my MS friends slip ever further into the clammy grasp of this horrendous disease. Because of the atypical nature of my illness, I very likely will not be part of the 20 person trial, so I’m not making this appeal on my own behalf. At least not directly on my own behalf, as the success of this trial stands to benefit all patients suffering from not only MS but a wide variety of other neurodegenerative disorders, and traumatic brain and spinal injuries as well.

So, from the bottom of my Wheelchair Kamikaze heart, I ask all of my readers to do themselves or their loved ones a favor and (click here) to donate even the smallest amount to move the Tisch Center’s stem cell trial forward. And please, please, please pass the word along (or forward this blog post) to family and friends using email, Facebook, Twitter, or any other newfangled thingamajig or whatchamacallit to help make this cause go viral, as every donation great or small could very well take us that much closer to the stuff that dreams are made of…

Sorry, couldn’t resist conjuring up a little Humphrey Bogart there at the end. I was a film major, after all. Speaking of film, the below video detailing the Tisch Multiple Sclerosis Research Center of New York’s stem cell trial and fundraising effort is also available on their Indiegogo fundraising page (click here).


Tremendous thanks in advance to all who find it in their hearts to make a donation.

52 comments:

  1. Marc I'm in! Tisch is amazing and they deserve our help.

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    1. Thanks, Debbie. The Tisch Center is doing some remarkable stuff, not only with stem cells but also with biomarkers and other important research. They are definitely the "good guys"…

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  2. Just contributed. I trust and admire Dr. Sadiq and his entire team. I've often wondered if there would ever again be anyone as fearless, committed and purely humanitarian as Jonas Salk. The world doesn't seem to condone heroes like that anymore, not when there are billions to be made. The NY Times ran an interesting article recently about today's philanthropists stepping into vital research that governments and universities no longer fund. My chump change is what I can afford - but it is my vote, and also my (agnostic) prayer.

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    1. Thanks so much for your contribution. Dr. Sadiq and the entire staff at the center are remarkable folk. It's rare to see that level of dedication, and the facility is unlike any other I've had experience with in my 11 year MS "career". Wishing you the best…

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  3. Done! Thanks for drawing my attention to this Marc.

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    1. Thanks, can't wait to see the trial up and running, and the trial subjects well, if not running, then at least walking…

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  4. Made a small donation from my disability pay, and posted a link to the Tisch Research Center on my Facebook page.

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    1. Debbie, that was wonderful of you. Thanks a bunch…

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  5. You are doing a great thing by spreading the word about this research and contributions. Thanks!

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    1. Just doing my part, Melinda. All too often this disease leaves us feeling helpless, at least this gives us a chance to poke MS in the eye… Thanks for your help…

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  6. Done. : ) Don't forget employer charitable matching programs.

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    1. Thanks. The fundraisers at the Tisch Center are very much aware of charitable matching programs, and are hard at work getting things like that set up. If you have any more thoughts, please pass them along…

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  7. Done, and fingers quickly and unquicky crossed due to spasms when I cross my fingers. All good intentions. :-) I also shared your blog on my wall.

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    1. Thanks very much, Donna. I can only cross the fingers on one hand, but I guess we all do the best we can physically, socially, financially, etc. Let's hope more people will jump on this trials bandwagon, and the study can get started quickly…

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  8. Done! Thank you for drawing attention to this.

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  9. I just donated. I wish that I could do more, but it's the best I can manage. Really hope this takes off.

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    1. Jonathan, every little bit counts. We all wish we could do more, but hopefully, through the power of social media, the trial will be "go for launch" as quickly as possible. Thanks for your contribution…

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  10. I wish I could give more, I share your heartfelt desire and I thank you for sharing

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    1. Thanks very much for your donation, Steve. I'm sure we all gave as much as we were able. Remember what they say about how a journey of 1000 miles begins…

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  11. I too wish I could contribute more. I hope they exceed their goals; first, in fundraising; then, in finding solutions.

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    1. Judy, we all wish we could contribute more. Unfortunately, none of us are billionaires (at least I don't think any of us are billionaires – if there are any billionaires out there reading this, well, go to the Indiegogo site and pony up some big bucks, pretty please). You've contributed to the community in so many ways through your writing, let's just hope that the trial gets going and the results turn out to be useful for us all… Thanks for your help.

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  12. Good stuff! I just wish that we could get someone to look at PANDAS disease. I no in my heart this is the start of MS! Diana

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    1. Diana, I'm not sure if PANDAS (Pediatric Autoimmune Neuropsychiatric Disease Associated with Streptococcal) is directly associated with MS, but it sure looks like the mounting evidence points to some sort of a viral trigger for MS and many other autoimmune diseases. My favorite theory these days involves human endogenous retroviruses. I wrote an essay on the blog about this about a year ago. If you search for the post "Can Anti-HIV Drugs Stop MS", I think you'll find it interesting reading…

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  13. Great idea to highlight this Marc, thanks for the heads up!

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    1. Well, I just feel that this is very important research. And it's research that I've been aware of for the past several years, since it's being spearheaded by the people who run my MS clinic. Well, not my MS clinic, but the MS clinic at which a patient. Let's hope that this research eventually puts an end to the need for such places.

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  14. Though I come from Czech Republic and not US thus even if this trial succeeds the treatment will be here 2-3 years later than in US, I have donated at least something. I wish I could give more but the salaries in academics where I work are not so big in our country and I am glad that I can still work somewhere even though I have MS. I will try to popularize the donations for this stem cell research on the forums in our country, hope someone will contribute too.

    Petr

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    1. Thanks so much, Petr. The impact of this research will indeed be worldwide, but this is an early-stage trial, that if successful will need to be pursued in bigger and more elaborate studies. But we have to start someplace.

      It would be great if you could get this as much publicity as possible in the Czech Republic. I visited Prague back in 2000, and thought it was a wonderful city. Had a great time, and as this was about three years before my MS diagnosis, I sampled much of the fantastic Prague nightlife.My friends and I tried to see as much of the city as possible, and not just stay in the typical tourist areas. Great city, nice people. Thanks again for your help…

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    2. So the word spread on our forums. Sure this is an early-stage trial but as you say, one has to start from somewhere. Of course when there will be some phase II, III trial I will contribute as well if I will have any money. Donations to this and to dog shelters are anyway the only ones I contribute to.

      Anyway, your blog great and I look forward to every post you write. It is almost every time enlightening and informative. But maybe the most important thing is that you are telling the complete truth about MS, not like neurologists. So thank you very much.

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  15. Hello Marc
    i have made my donation, spread the word and I am keeping my finger crossed. If the study works it will be an amazing result for all patients around the world. Thanks for your explanation and for putting the divulgative power of wheelchairkamikaze in the project. Wishing good luck to all of us
    Ciao
    Alessandra

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    1. Hi Alessandra, thank you so much for your donation, and for spreading the word. I know that you are writing this from Italy, so between you and the previous commentor, Petr, we have two countries in Europe covered. I know I have lots of British readers, it'd be great to hear from them as well. I've even seen some of my posts translated into French and German. The power of the Internet is incredible, really.

      Thanks again, this is really a chance for us to pull together as a community and make something happen. Sure beats sitting around and waiting for word of a miracle…

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  16. I'm in - this looks like it could be a major breakthru. I understand that Richard Cohen ( Merideth Vieira's husband) is in this study, and you can follow his blog at richardmcohen.com.

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    1. Yes, Richard Cohen will be in the trial, and his blog makes for good reading. Thanks so much for your support…

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  17. friend of a friendMarch 19, 2014 at 12:41 PM

    Thank you for all the information, Marc.

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  18. I just donated 30$.
    Even none Americans want to help, too bad I had to choose an American state to be able to donate...

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    1. Thank you very much for your donation. It's strange that you had to choose a state here in the US in order to donate, I was under the impression that Indiegogo was set up to take international donations. I'll have to look into this…

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  19. Thanks for this, Marc. I'm in the study, and can't believe we still need funding. I posted this on my FB page, too....Hope you're doing okay. I think of you whenever I'm barreling down the streets of the city, cussing at the absence of decent cut-outs! Wasn't this a hell of a winter?

    Best as always,
    Terri

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    1. Hi Terri, yes, this winter was really something else. Reminded me of winters way back in the 70s, when I was growing up. Seems like the winters were much colder back then.

      Wishing you great things in the study. Don't know what the deal is with the curb cuts, but it does seem like the harsh weather only made things worse, creating huge potholes at the base of many curb cuts. Makes for quite the bumpy ride! Who knows, maybe we'll crash into each other one of these days…

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    2. Hope so, Marc! We could tear ass through Central Park when/if it ever gets warm! ; ) My pup loves a good wc run!

      I just saw Dr. Sadiq today. The whole office is grateful for your excellent blog, and for this article. Dr. Sadiq himself seemed to be somewhat harried - maybe cuz the office was crazy busy today. Sometimes, we forget to simply say 'thank you'' even for the chocolates on his desk! : )

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  20. Done - Donated as "www.wheelchairkamikaze.com follower, $100 USD"
    Keep up the good work!

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    1. Thank you so much, both for the donation and the shout out. Much appreciated.

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  21. Mark,

    I just got an update from Tisch's donation page.
    "Tisch MSRCNY Campaigner of the Week!

    THANK YOU to Marc Stecker a.k.a. Wheelchair Kamikaze for the incredible article about the Tisch MS Research stem cell trial campaign on Indiegogo.com. You can read the post and follow Marc's blog at: http://www.wheelchairkamikaze.com/"

    Congratulations, man! Maybe this will help you get the procedure.

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    1. Yes, I got an email this morning telling me I was the "campaigner of the week". I'll now be able to wear a sash and a tiara when I'm out and about in the wheelchair, at least for a week. I just hope they don't ask me to participate in a swimsuit competition…

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  22. Just called to see if they are accepting patients for their trial and was told that the ONLY patients for their trial are their own current patients. SOOO, the bottom line is, if you aren't one of their own patients you will not be considered.

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    1. Hi Barbara, yes, only patients currently being seen at the clinic are eligible for the trial. This is understandable, though, as the clinic sees well over 1000 patients, and the researchers need to be well informed about each patient's clinical history and the intricacies of their disease in order to properly conduct the trial. Keep in mind, this is an early-stage trial limited to 20 test subjects, so space is extremely limited.

      However, the benefits of this trial, if successful, will extend way beyond the confines of the clinic walls, and certainly has the potential of changing the way MS medicine is practiced.

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  23. Longtime reader, first time commenting. Ex-Brooklynite, SPMS, first RRMS diagnosed in '91. Just finished donating. Can't see nothing but good things from this trial. Thanks for all your information, Marc.
    Ed

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  24. Done - donating and sharing. I love that place although if I was fixed and never had to go back that would be great. Hope they exceed their goal!

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  25. They're around $1500 shy of the 50% point. I feel pretty confident that they will exceed their goal by the time this campaign runs its course.

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  26. Do you mind if I ask why you're not in the trial, Marc?

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    1. My disease is very atypical, so I doubt I'll meet the inclusion criteria. The price I pay for being a weirdo…

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  27. Awesome, the indiegogo donation site is only $6,015 away from being fully funded. And that's with 6 days remaining on the campaign.

    Looks like it'll be fully funded now.

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