HSCT Cure Rate?

Looking88 · Post by **Looking88** » Sun Jan 02, 2011 1:26 pm

Anyone have good information on the cure rate (halting MS) of different HSCT protocols?
I have seen the 85% cure rate listed, and near 100% for early RR, but am wondering if the cure rate is different between the (I will probably get this wrong) Lymphoablative (not totally rebooting immune system) versus Myoablative (total reboot requiring re-immunizations).

I read that so far the HALT-MS protocol is 23 for 23 in stopping progression.

georgegoss · Post by **georgegoss** » Sun Jan 02, 2011 11:21 pm

Hi Looking88,

You ask a great question! In fact one of the most important ones (in my own mind).

Both the myeloablative and lymphoablative protocols have so far in the clinical trials shown substantially similar (within statistical variation norms) the same cure (halting MS progression and existing-symptom improvement) rate at several years out from treatment. So when I was researching which HSCT protocol to go with for my own treatment (lymphoablative vs. myeloablative), I was actually asking the (yet unanswered) question of "How long will the cure last?"

Dr. Richard Burt's MIST treatment protocol is "Lymphoablative" (eliminates reactive T-cells but does not completely wipe out the bone marrow stem cells). The treatment I had was Dr. Richard Nash's HALT-MS "myeloablative" (completely wipes out the bone marrow stem cells prior to allograft re-infusion & engraftment). As mentioned, to date both protocols have shown substantially similar (very good) curative results at several years out from treatment. The open question in my own mind is "how long will these two different therapies result in stopping the progression of the MS disease activity?" Is it lifetime, or only temporary? Only the continuing population study over a longer period of time will actually tell us the answer with the greatest degree of confidence. However, I decided to go with a myeloablative therapy instead of the lymphoablative therapy for one simple reason. . . . myeloablation results in total loss of immune memory via the surrogate indicator that people lose childhood immunity and require re-vaccination. Dr. Burt's lymphoablative protocol does not require re-vaccination for childhood diseases. My own theory on this is that lymphoablation does not actually completely 100% reset the body's underlying defective autoreactivity that causes MS, and makes me fearful that the treatment "might" not result in a lifetime cure. But this is only my fear, as I have no data or evidence to suggest that this is actually the case as my assumption might very well be wrong. But for my own treatment I decided to be overly conservative to ensure the best possible chance (in my own mind) that I was receiving the best chance of a lifetime cure of my MS with a myeloablative (BEAM) protocol. Regardless of the final results with Dr. Burt's lymphoablative treatment, I believe that I have most likely achieved an enduring lifetime cure with the protocol I received. So far, so good.

Looking88 · Post by **Looking88** » Tue Jan 04, 2011 6:52 am

Thank you. Do you think it would be possible to get the less aggressive Lymphoablative treatment in Germany (or elsewhere?)?

georgegoss · Post by **georgegoss** » Tue Jan 04, 2011 8:27 am

Another great question Looking88. My simple answer is that I don't know.

The biggest problem with getting the lymphoablative treatment outside of a trial is that the protocol is unique since it is not used for cancer treatment and it is unlikely that that treating hospital will have any familiaraity with adminstering it. The BEAM protocol I received is commonly used for several forms of cancer so most treatment hospitals know how to adminster the therapy and know how to deal with side effects, ractions and follow-up care. Experience is they key here. And very simply, outside of a clinical trial it is unlikely that a treatment facility knows the best course for performing Dr. Burt's lymphoablativeprotocol.

With that said. . . . I don't want to dissuade anyone. I would bet that it is more likely than not that there are probably some treatment facilities somewhere around the globe that will do it anyway as a one-off treatment. Perhaps somewhere in India or China would do it? But would you really want to go somewhere that has no previous experience with it and have a complex treatment regimen that has the risk of srious adverse events for your health (or life)? For me personally, I wouldn't.

I did find Dr. Yvonne Loh at the Singapore General Hospital (I used to live in Singapore and they have good healthcare), and she used to work as a researcher on Dr. Burt's Northwestern team doing the Lymphoablative protocol. However, After communicating with her I quickly found that for some crazy reason she decided to apply the clinical trial inclusion/exclusion criteria in her clinical environment which will eliminate 99%+ of people that would want to get the lymphoablative protocol. (The unchanged study criteria has no business carrying over to a clinical treatment environment because the purpose of a clinical trial is to prove hypotheses' for a population, not specifically be a curative regimen for individuals.) I was very dissaappointed that Dr. Loh couldn't make the transition from researcher to clinician. Perhaps she just didn't want to perform the procedure? You could always give Dr. Loh a try. But I suspect you likely won't have any more success than I had.

Otherwise. . . From what the doctors in Heidelberg communicated to me the myeloablative BEAM treatment protocol I had has a death risk of somewhere around 1% for an otherwise healthy person at an experienced facility such as Heidelberg (they do over 300 transplants a year for cancer). That's reasonably low mortality for this treatment type and approaches what Dr. Burt has said publicly said about the NWU mortality rate of the lymphoablative protocol.

Looking88 · Post by **Looking88** » Tue Jan 04, 2011 8:41 am

Thank you for the detailed and kind response.

georgegoss · Post by **georgegoss** » Sat Feb 05, 2011 10:30 am

Looking88 wrote:Thank you. Do you think it would be possible to get the less aggressive Lymphoablative treatment in Germany (or elsewhere?)?

I just received a message from a very nice fellow MS'er reading my blog, and he had this relevant info that I was not previously aware of to add. I understand the cost for the procedure is approximately $75,000. Close to the 55,000 Euros I paid for my own procedure in Germany. . . . .

Hi George.

My name is [redacted] and I just now discovered your blog. I come from [redacted] and have (had?) MS. My compliments on your blog, everything is so precise and informing. It brings back memories on many sleepless nights that I spent on the internet, trying to find the solution that doctors were not able to find. It is nice to read about the people who just like me once decided to take things in their own hands.

In late 2006, I underwent a non-myeloablative HSCT, which is, as you explained, a lot les toxic and carries much less risk, with apparently the same outcome. The procedure was preformed at Hadassah clinic in Jerusalem, under the supervision of Prof. Shimon Slavin. I received my stem cells back on Christmas - talking about coincidence!? [I was also treated during the same time of year.] Ever since [the treatment I have had] no new symptoms of my MS, I am drug free, and some things with respectct to my condition have improved immensely, for example my strength.

Take care of you self, I am sure that this battle is yours!

Best regards

Lyon · Post by **Lyon** » Sat Feb 05, 2011 2:53 pm

packo · Post by **packo** » Sat Feb 05, 2011 4:12 pm

Greetings everyone, I am the guy that sent the cited e-mail to George today. Please tolerate occasional errors in my English, this is not my native language – I come from one European country.

Regarding the HSCT, as George pointed out, it is very important to do this procedure (myeloablative and non-myeloablative) in a medical center which has experience in those procedures. Experience doctors and stuff are the necessity, I strongly recommend everyone to think twice before doing this in some exotic locations.

With regards to the non-myeloablative protocol, George explained everything very precisely. And certainly it seems that myeloablative protocol theoretically offers the higher margin of safety with regards to the re-activation of MS, compared to the non-myeloablative protocol. But in the beginning, HSCT was performed with total body irradiation, and afterwards doctors came to the conclusion that this seems to be unnecessary and way to risky (who knows, maybe in the future it will turn out that only people that did the total body irradiation are “cured” from MS

). However, so far it seems that non-myeloablative protocol offers the same results, and is less toxic and risky.

Lyon · Post by **Lyon** » Sat Feb 05, 2011 4:49 pm

Looking88 · Post by **Looking88** » Tue Feb 15, 2011 11:59 am

Thank you George (and thank you Packo) for identifying a non-myeloablative option (assuming it is possible to still get the procedure at the Hadassah clinic in Jerusalem). It is good to have options.

Hello Packo: Please keep us posted on how you are doing if you can. Also, how you were doing before treatment.

Next challenge: Where can a person get the myeloablative treatment in North America. I am sure there will eventually be a way to get this done outside of a clinical trial.

Thanks,
Looking88

georgegoss · Post by **georgegoss** » Sat Feb 19, 2011 10:21 am

I personally know nothing regarding the treatment provider in Israel. It may be a good option. But I just received some additional info from a fellow MSer in Australia that heightens my sense of awareness when issues of money move to the forefront with a medical care provider. I sincerely hope its not just a money factory. So this is of course all just hearsay at this point. . . .

Ive had an initial email chat with Prof Slavin last year unfortunately he would not discuss my case without asking for money up front (no set figure either) I then came across a web page where a lady feels she had been ripped off...however I might revisit his services. A non-myeloablative HSCT AKA a mini-transplant, perhaps another option.

packo · Post by **packo** » Sun Feb 20, 2011 4:13 pm

I was intending to write a longer post about my experience in Israel and how I felt before the procedure and how I feel now, and I will do that when I find time-surely in the next 10 days. But right now I have duty before the God (maybe He has something to do with this – my zero day was Christmas, in Jerusalem, 10 min from Betlehem, if someone told me one year before HSCT that I would do this on that place at that exact time, wow! – I do not believe in coincidence) and myself to say something about Prof. Slavin, since I owe him so much, and we are talking about world class expert. I mean, he opened Israel’s first bone marrow transplantation unit, specialized at Stanford University and Fred Hutchinson’s Cancer Research Center, lectured all across the USA and world. So make no mistake, this is not a commercial and this guy does not need one. Here are just a few sites:

His short biography:

http://www.repairstemcells.org/Home/Sci ... lavin.aspx

How he became famous worldwide in medical community:

http://www.israelnationalnews.com/News/News.aspx/130335

Or how he did this (we are talking about PPMS!), with the comment of Dr Freedman from Canada:

http://www.ctv.ca/CTVNews/Health/200811 ... nt_081116/

hannakat · Post by **hannakat** » Tue Feb 22, 2011 3:48 pm

packo ~ I'm looking forward to hearing of your experience and outcome under Professor Slavin!

packo · Post by **packo** » Sun Feb 27, 2011 9:43 am

Disclaimer: The purpose of my comment is not to promote a certain person or idea. This is how I ended writing on this forum: When I was thinking about HSCT five years ago I came across two sites which were very useful to me:

http://www.ctv.ca/CTVNews/WFive/2005020 ... ms_050203/
My greatest admiration for all the people that went through this and for those that are still in this project – if I am not mistaking, somebody on this forum is in this procedure? And of course, thank you John McCleary (the guy who died in this project), every now and then I think of you, best wishes to you in heaven.

http://www.mult-sclerosis.org/craigsstory.html
This is an experience of Craig Garisson - this is how I discovered George Goss site: One evening two months ago I was on the internet and was wandering what happened to that guy Garisson after 2003. Then I typed “multiple sclerosis Craig Garisson” and one of the first sites that appeared was one created by George Goss, in which he mentioned this guy Craig. Then I noticed that George in his blog did not mentioned the possibility of doing the non-myeloablative HSCT outside clinical trials and decided to let him know that such an option exist. Consequently, he posted my mail here. I am writing all this so that nowbody would get the impression that I am trying to promote either this procedure or Prof. Slavin. But let us get back to my story:

In short, I was diagnosed with MS in 2002, and upon trying all sorts of medication (Rebif, Novantrone, steroids) was in such a shape that sometimes was barely able to walk with two crutches. By 2006, the idea of HSCT was already deep inside me, but no neurologist that I met knew nothing about it. Then fortunately I was introduced to a neurologist who knew much about MS, and was also acquainted with the attempts to stop MS with SHCT. She told me that this procedure is available outside clinical trials only on few places in the world, and among them in Jerusalem. She also told me that I should be aware of the fact that this procedure carries certain risk and that maybe it will not work on me, because in my case the irreversible damage already occurred.

But I decided that I have to do something with my condition, and if it does not work, at least I tried. Upon initial contacts with Prof. Slavin via mail, I came to Hadassah Hospital in Jerusalem. Prof Slavin looked at my MRI’s, my medical history and examined me, and told me:

“OK. boy, I am telling you this honestly, if we do the HSCT in your case it might not work due to the irreversible damage. I also notice that your latest MRI does not show any new lesions, and this may also be due to the fact that your disease entered the neurodegenerative phase. But this can also be because you were receiving Novantrone in the last 6 months prior to your arrival. It is up to you, but I am not promising anything”.

But I was determined I want to do this, so I took my chances. The procedure was not an easy one, though it cannot be compared to the myeloablative protocol. I still remember that cyclophosphamide shit (sorry for the term I used, but I cannot help myself) in my body, and I thought of all those people that have no option but to receive much more of this medicine. All in all, my HSCT went well and I returned to my country.

Today, more then four years from my HSCT I still walk with two crutches (though I strongly believe I could walk with one if it was not for my aitch bone fracture (steroids, be careful with them) – due to this, my left leg is 3 centimeters shorter then my right leg, so even a completely healthy person would experience difficulty walking in that situation. Some of the things did not improve at all, for example tremor in my hands – but the tremor is not getting stronger, which is a great thing!

But the think that did improve very much is my strength: I can walk on my crutches up to 45 minutes without stopping (so much that blisters on my hands can appear

), I exercise up to one and a half hour every day, can do up to 500 push-ups per day (right now I am laughing because recently I hurt my back a bit with this over-aggressive exercising – but it is hard for someone like me who used to be a professional athlete to stop when I smell that I have this much strength), I am completely drug-free, and finally I fell like a fully productive mmber of the society.

One last thing, I did the non-myeloablative HSCT in late 2006, back then Prof. Slavin was still at Hadassah. Right now he works in the newly established center in Tel Aviv. So I do not know whether the non-myeloablative HSCT can still be done at Hadassah or Tel Aviv, but if I would have to make an assumption – my answer to both of that questions would be: Yes.

georgegoss · Post by **georgegoss** » Sun Feb 27, 2011 11:13 am

Thank you very much for sharing your story, Packo. This is great information and perhaps a treatment possibility for anyone interested in stem cell transplantation utlizing the lymphoablative protocol. It's also great to know that overall your experience (and especially your health) was positive. Kudos to you for taking control of your disease!

Although you still have some residual disability following your transplant, it's wonderful to know that your underlying MS disease activity has completely stopped and you no longer have any progression (and some improvement). For me, I have really come to realize that stopping my MS disease process via HSCT has given back to me the ability to plan for the future with my family. That is something that MS often robs us of and I hope you have been able to derive as much appreciation as I have for regaining this intangible benefit.

And thanks for mentioning Kirk Garrison (a very good guy) that had HSCT ten years ago in 2001. I traded a few e-mails with Craig and he reports that today his MS progression is still 100% stopped. Another good indication for us that likely we will see a permanent stopping of our disease.

Sounds to me that Prof. Slavin is a very capable & accomplished clinician. I hope you have the opportunity to send him greeting cards every year as I do to the doctors that treated me.

An explanation of the clinical science for anyone that is not familiar. . .

http://themscure.blogspot.com/2010/06/s ... rence.html