Tuesday, November 24, 2009

Introduction to the voyage

If you have come to this blog for the first time, you may find reading the following page first will be useful. . . . .


Greetings, I have created this blog both to update my family & friends on the status of my journey through this trying process, and also to share with all others about the exciting prospect of a cure (really!) for Multiple Sclerosis. I will start this blog with some background, then a technical description of what I am doing, followed by a step-by-step description of the journey of treatment as I progress through it. I plan to follow-up after the treatment and report on the Treatment result and efficacy, either way it turns out (but I expect that I will eventually be "cured" of MS). I will periodically include some video posts to simplify and clarify the information that I will plan to share.

Make no mistake about it. . . . I fully expect that in a matter of ten years, or less, this treatment regimen will become the standard treatment to cure MS. I consider myself among one of several hundred people so far to be pioneer patients to prove this therapy works.

A little about myself. . . . I am 45 years old and was diagnosed with clinically-definite MS when I was 31 (the average age of MS diagnosis at 30 years for the total population). The presenting symptom at the time of my diagnosis was optic-neuritis (as is the case with approximately 60% of all people first diagnosed with MS). Up until a few years ago I followed a course of relapsing-remitting (RR) type of MS, but am now essentially secondary-progressive (SP). So to explain the overall idea and plan of what I am doing, here is a letter I wrote to my good friend that explains what's going on. . . .

Hi Yokota-san, I wanted to update you on my plans that we have discussed regarding my seeking a cure for my Multiple Sclerosis of which I was diagnosed in 1996. It turns out that contrary to classical beliefs, there is a "probable" cure for Multiple Sclerosis. In this case probable means 80% chance of positive outcome and cure means a "halting" of progression (not total reversal, although there may be a 30% reversal of existing physical deficit). Another good point is that all the research to date indicates that it works on all forms of MS.

I entered a secondary progressive phase a few years ago and continue to have a slow progression of continual deficit, which is quite common for the long-term course of the disease. So although I no longer have specific relapses (flare-ups), I continue to slowly degrade. It is already becoming rather fatiguing to walk several hundred meters. So although I still function relatively well, I can guess where my status will be in ten years if the course of my MS disease is not altered.

The cure? It's an autologous hematopoietic stem cell transplant (historically referred to as a bone marrow transplant, although that term is not commonly used now). I will spare you the technical details for now, but suffice it to say that it turns out to be an amazingly statistically effective treatment. So much so that I am going to undergo the procedure this coming December. And as a result I fully expect to be cured of any further MS disease progression. [Post transplant note: End result: It worked!] And on top of that I can discontinue my current interferon injections which is standard therapy for many people with MS. Yippee! No more chronic injections after that.

I honestly believe that in the future this treatment protocol will become the standard treatment as a cure for MS. Interesting that the reason that it currently is not is because it is still in phase II [now phase III as of 2011] clinical trials around the world. [Post transplant note: All the phase II trial patients have been treated with all having demonstrated successful curative results and are being monitored during long-term follow up. The procedure is currently into phase III randomized trial starting in 2010.] Also because it is a very aggressive medical treatment there is some mortality associated with it (perhaps somewhere around 5%. . . . I figure a 1 in 20 chance of death is not as good as a Space Shuttle ride, but it’s a hell of a lot better than a game of Russian Roulette with a revolver!). [Note added post-treatment: The mortality rate with this treatment at Heidelberg University Hospital is actually closer to a low 1% for an otherwise healthy individual. Not nearly as scary as I originally thought.] In addition, the treatment has a high probability to cause irreversible sterility due to the strong chemo. As far as other common side effects go, I also get to temporarily lose all my hair. (Not such a bad thing. It's practically all gray anyway. I wonder if it will come back brown again?) However, the main thing is that this procedure is also sure to be a damn uncomfortable procedure, just as the same procedure is often used to treat many forms of cancer such as leukemia, multiple myeloma and Hodgkin's & non-Hodgkin's lymphoma. Something like a living hell for a few weeks [Post-transplant note: OK. . . it's definitely uncomfortable. But it's not actually a "hell" experience.] while getting the high dose chemo and being in a clean environment while the stem cells repopulate and reactivate the immune system. This procedure is like hitting the "reset" button on the body's immune system. Taking it to zero and then bringing it back up to life again. [As a curative treatment HSCT works by erasing the body's immune system memory, changing the body's overall T-cell epitope (antigen binding) repretoire, inactivating autoimmunity (making the immune system "antigen naive") restoring immune self tolerance. This is often referred to as "resetting" the immune system which stops the underlying disease activity and progression of MS. Once achieved, the body then has a chance to repair (or compensate for) some of the existing neural damage that is not undermined by further MS disease progression.] In fact, even childhood and lifetime immunity goes away following the procedure. It becomes necessary to be re-vaccinated against childhood diseases such as tetanus, polio, measles, etc because my body will no longer recognizes them as foreign invaders. Presumably the same reason that my body stops attacking itself as is the case in an autoimmune disorder like MS. Problem for me (and anyone else seeking the treatment) is that within the United States [& Canada] it is currently impossible to receive this treatment for MS outside of a clinical trial. [Post transplant note: This is no longer true. See this web page: http://themscure.blogspot.com/2011/06/getting-into-hsct-treatment-if-you-have.html ] I tried to enter a trial in which I thought I was an ideal candidate in the following program:

But alas I was rejected from this trial for some technicality of documentation or paperwork, or something like that. I searched in vain for a US facility that would perform the procedure for me, but there are absolutely none in the US [or Canada] that will do it for MS outside of a clinical trial because US facilities are overly-paranoid of lawsuits, which is a shame because it is a treatment with excellent clinical outcomes. And even if I did find one in the US that would be willing to perform the procedure, the price tag is around $200K. Outside of the budget I had in mind and not a single penny covered by insurance because it is still considered "experimental" (even though the overwhelming data suggests otherwise).

So I started looking overseas for a facility that would agree to perform this treatment. The usual suspects were first on my list. . . India, China, Brazil and others. These locations will perform bone marrow transplants cheaply, and no questions asked. And indeed these places may even do a terrifically good job for this type of procedure. But frankly, I personally don’t know enough about these locations to know if they would be good treatment options. I just don't want to experiment with my life to find out with a possibly risky procedure like this. So with further investigation on the international bone marrow donor [EBMT] site I started checking out facilities in Europe. I figure the Germans are a good bet because of both the scientific excellence, and also because they have medical regulations that are on-par with the FDA here in the US.

I quickly learned about Heidelberg University Medical Center where they perform over 300 transplants a year. I contacted them and found out that they have even previously done some transplants for MS. They seem to me to be an exceptionally capable and experienced organization. So I've decided to go with Heidelberg this coming December. I'll have to be in the hospital a little less than a month. Then an outpatient checkup ten days after discharge and then I can return to California and be cared for by my own Kaiser doctors for the monitoring period lasting several more months, to a year.

And the great news. . . total cost in Germany is about 50,000 Euros, plus travel and housing costs. That comes out to approximately [less than] half of what it would cost in the US. Much more affordable. And for me, personally, a reasonable price tag to cure my MS. Of course with having the option, it's difficult to imagine anyone "choosing" to go through this treatment living hell if they didn't have to. (Most cancer patients have only the alternative option of death, so it’s a no-brainer decision for people in that situation. With MS patients, death is not the option. Just further potential disability.) But I'm going to do it for my wife and my son. I don't want to miss out on participating in the future growth of my son's physical activities. Or at least I don't want to be completely excluded from them. I think this procedure will enable me to accomplish this goal.

So just a final note. . . . I believe that I very well may be one of the first, if not THE first person in the United States to seek this treatment outside of a clinical trial. (My hat’s off to the Germans for recognizing the importance of helping people with treatment, as opposed to just doing research for the sake of research!) So to share the experience and the details of the process and outcome with friends and interested parties, I am doing a blog about the whole experience. I just set up the site so it may take a few days to start posting the info & videos. Here’s the blog site:


I’m looking forward to seeing you upon my return from Germany, Yokota-san. You are a true friend!

Best regards,


So there you have the summary of what this is all about. There are currently two similar, but different forms of curative treatment regimes of the bone marrow transplant type undergoing phase II clinical trials. The goal is the same for both treatments (a halting or reversal of disease progression). However, these two treatment protocols differ in one substantial manner.

The treatment that I will be receiving in Germany follows the HALT-MS study protocol under the direction of Dr. Richard Nash at the Fred Hutchinson Cancer Research Center which is a fully ablative regimen. That is, the bone marrow is completely destroyed by high-dose chemotherapy (myeloablative) prior to the re-infusion of my own stem cells used to reconstitute the immune system that will have been collected prior to the high dose chemo.

The other program, called the MIST trial pioneered by Dr. Richard Burt at Northwestern University recognizes that the fully myeloablative therapy is risky with approximately a 5% mortality. [Post transplant note: BEAM myeloablative treatment at Heidelberg University Hospital is actually closer to a low 1% mortality rate.] So Dr. Burt developed a regimen that is not so aggressively myelotoxic, but is instead predominantly & aggressively lymphoablative which does not kill off 100% of the bone marrow, reducing the period of time a patient must survive with degraded (not ablated) immune function. This results in reduced risk of treatment complications (infections) with an associated mortality rate of well under 1%. All of the initial indications are that this non-myeloablative protocol has good clinical benefit at several years out, albeit it is still not established for how long this benefit will ultimately last. (Is it lifetime, or is it temporary? Only time and the ongoing population study will provide the answer.)

A graphical comparison of the immune function recovery for the two protocols (click to enlarge):

The unfortunate part of the MIST study is that this therapy is difficult to find outside of a study trial [Post transplant note: Please see the following page for a list of facilities that will provide HSCT today: http://themscure.blogspot.com/2011/06/getting-into-hsct-treatment-if-you-have.html ] because it makes use of a very unique chemotherapy protocol not used for cancer treatment consisting of either (Fludarabine + either Cyclophosphamide or Campath-1h) or (Cyclophosphamide + rATG) that rapidly ablates in-vivo lymphocytes while only mildly (and incompletely) ablating the hematopoietic stem cells in the bone marrow. I contacted Dr. Yvonne Loh at Singapore General Hospital (she previously worked with Dr. Burt's team with MIST) to inquire about treatment availability in SGH's BMT unit where they use a Flu/Cy (Fludarabine + Cyclophosphamide) protocol. Singapore law does not disallow the treatment to be given to patients outside of a trial, but unfortunately Dr. Loh has been unable to make the transition from researcher to clinician and applies the overly-restrictive (and in my opinion, clinically ridiculous) inclusion and exclusion criteria from the MIST trial that eliminates 99%+ of people that might want this treatment. So even if one wanted this alternate therapy, it is virtually unavailable. It's too bad that Dr. Loh lost sight of helping people in a clinical environment as she is still blinded by the quest for proving a hypothesis in the context of a study program.
Regardless, for me the main determinant for which therapy I prefer revolves around a simple concept that I believe is based upon solid science. Following the MIST protocol, treated individuals need not be re-vaccinated as is the case following a fully myeloblative regimen. (Dr. Burt has published his reasoning for why the MIST protocol does not require re-vaccination as follows: "[For the non-myeloablative MIST protocol] Autoreactive memory cells survive and autoantibodies, while significantly diminished, generally remain positive.") This tells me that the body's immune system is not completely reset [for the MIST non-myeloablative protocol] and still recognizes points of attack of the human body. To me this seems like the "possible" (but not definite) Achilles heel of MIST. So in the end, I think the HALT-MS protocol might possibly have a better chance of lifetime cure success [since BEAM treatment also requires the surrogate indicator of disease re-vaccination not required in MIST], albeit with a (only slightly) higher probability of mortality. This is why I have chosen the HALT-MS protocol, which utilizes a BEAM protocol (BEAM an acronym for the four major types of chemo drugs used for the ablative process extensively used to treat certain forms of cancer). If interested you can read about BEAM here:


Before signing off today I should mention regarding other forms of stem cell therapy that are offered in an overseas unregulated market environment. There are many companies offering stem cell therapies that collect stem cells from adipose fat tissue, bone marrow and other sources, and then do an IV infusion and/or intrathecal injections. The theories cited for these treatments are usually valid, but from what I have learned not a single such "stem cell procedure" of this type has shown any reproducible clinical evidence for curing MS. Probably the downside risk is small for these therapies, but the upside usually fails to materialize and can be drowned out by the marketing hype of these companies (which is why these therapies are not available in the United States due to FDA restrictions). I feel sorry for people that get sucked into these programs that offer little clinical benefit beyond a placebo effect. And I feel especially bad for anyone seeking such a treatment in lieu of the true cure of a stem cell (bone marrow) transplant. [Post transplant note: Another way to think about this is "No chemo = No cure." This is because just injecting stem cells into one's body does nothing to correct the underlying defective autoreactivity of the body's immune cells that are causing the MS damage. MS disease progression needs to first be halted before meaningful nerve repair/compensation can take place.]

For now I'm getting ready to spend time with my wonderful relatives for a nice Thanksgiving and will depart for Germany next Monday, November 30. In my next post I'll let you know more about what I've done to prepare for my trip and the procedure. Getting phsyched up!

Thanks so much for following along with me!

- George


  1. Do U have a contact email?
    Thanks~ Tricia

  2. Hi Tricia,

    My e-mail is. . . .