Wheelchair Kamikaze: Pioneering MS Stem Cell Trial Needs …

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.

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 patients 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 decades 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 Centers 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 theres about $200,000 to go. Detailed information on how these funds will be spent is available on the Tisch Centers 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 doesnt 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 patients 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.

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Wheelchair Kamikaze: Pioneering MS Stem Cell Trial Needs ...