2 Years Post HSCT And MS Still Stopped

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Re: 2 Years Post HSCT And MS Still Stopped

Postby Pesho » Mon Jun 18, 2012 12:59 pm

Guys, I have a dilemma. I can probably find 40 000$ for HSCT in Russia or India, probably Russia because it is closer and cheaper transportation and so on. The thing is, that first I'm not feeling very good for asking for this amount of money from my brother in law. Second I'm not that bad at the moment EDSS 2, but I feel very, very insecure. I don't know how long will I be well, I don't feel like creating a family, even getting a new girlfriend. Second I will probably have to quit my job, which is pretty good for this moment. But they cannot wait for me to be gone for 2-3 months, I have no backup person on the job. So, should I give up on everything, probably give up my career and my pride? Also no doctor here would recommend this procedure, and convincing people this is the right way might be tricky.
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Re: 2 Years Post HSCT And MS Still Stopped

Postby Asher » Mon Jun 18, 2012 3:19 pm

Pesho, I can't possibly comment on your personal considerations and circumstances. All I can say is that I went into my HSCT treatment with EDSS 4.5 and will have to come to terms with irreversible damage for the rest of my life. If only I knew of the treatment when I was EDSS 2, I would beg, steal or borrow to have it. For MS the maxim is: Time equals brain mass. At EDSS 2 you've not past the point of no return yet.
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Re: 2 Years Post HSCT And MS Still Stopped

Postby georgegoss » Tue Jun 19, 2012 4:40 pm

Hi everyone,

Stopping in to say Hello and see what's been going on. I like everyone's comments, especially with my absolute full agreement with what Asher has said. If I knew then what I know now, and if it were available (it was not at that time), I would have had HSCT the day after I was diagnosed with MS. Asher's right that improvement (reversal) of accumulated disability is better when HSCT is performed earlier before more substantial symptomatic accumulation manifests. And if the underlying MS disease activity and progression can be stopped (which HSCT does in the vast majority of cases), it returns a predictable future for people. That alone was worth it for me going through the procedure. All of my symptomatic improvements have just been a bonus. But I'm not looking a gift-horse in the mouth. I'm happy (as eventually are virtually all other HSCT-for-MS recipients I have met).

Sincerely wishing everyone well!

- George

HSCT curative efficacy statistics:

The early phase I HSCT clinical trials for MS focussed on treatment of severely disabled progressive patients that were primarily in the range of 6.0 - 8.0. Although the underlying MS disease activity can likely be stopped in this group, "improvement" of pre-existing sysmptoms is not spectacularly good. This is why today the clinical trials focuss on ambulatory episodic patients in which HSCT works best. Here is a general description of HSCT efficacy level based upon morphological disease type: As a general rule, treatment earlier in the MS disease lifecycle is more effective than later.

The specific statistical "probability" that has been demonstrated for each morphological evolution groups (RR, SP, PP) for halting of underlying disease activity is as follows:

Ealy RRMS-treated patients

Complete stopping of the disease process & progression in virtually 100% of treated patients.

81% of the same group experiences "significant" improvement (>1.0 point EDSS) reduction of existing symptomatic EDSS. Many HSCT-treated patients in this group report their symptoms completey dissappearing, 100%.

During the RRMS phase of the disease I would expect very good beneficial results following HSCT regardless of the EDSS-measured symptomatic disability at the time of treatment (highly effective in the entire EDSS range).

Late SPMS (non ambulatory) treated patients

Stopping of MS disease progression in 78% of patients in this group.

EDSS improvement not quantified in this population and varies by individual.

Curative efficacy is not population-quantified in other patient EDSS stratum. So for SPMS patients with an ambulatory status (EDSS <6.0), the cure rate (stopping of disease progression) is not well established, but certainly I would expect it to be greater than 78%. Probably in the range of 85% - 95%.

Just my own personal expeience data point (that does not necessarily translate to any other specific individual) that appears to be consistent and fairly representative of other (ambulatory) SPMS patients so far. . . . . I was RRMS for 11 years and then went SPMS for 4 years (EDSS 3.5) before my HSCT procedure. As of today (2 years post-transplantation) my disease is 100% stopped and my pre-existing symptomatic deficit (as measured by EDSS) has improved (reversed) 50%. So clearly it is possible that SPMS cases (especially those that are ambulatory) can experience good beneficial improvement from HSCT.

Late PPMS (non ambulatory) treated patients

Stopping of MS disease progression in 66% of of patients in this group.

EDSS improvement likely poor, if at all

So for the PPMS patients with an ambulatory status (EDSS <6.0), the cure rate (stopping of disease progression) is not well studied and not well established, but certainly I would expect it to be greater than 66% people in the PPMS population.

Also, for ambulatory PPMS cases, EDSS improvement is possible, but not gauranteed. This is a very un-studied and unknown area. I would expect this number of people in this poulation to show improvement somewhere in the chasm-like range of 1% - 65% of treated patients. Perhaps someday as more ambulatory PPMS patients are treated then this can be better quantified. For now, the treated population is so small its impossible to predict with any confidence.

But no matter what, HSCT has a far better chance of having a positve beneficial effect for MS patients of any type as compared to any (every) other therapy anywhere in the world. So far, no other curative therapy can even come close in accomplishing what HSCT has already (scientifically and repeatably) demonstrated.

Here is a single graph that I created that provides a comparative overview of all the aforementioned data:

http://2.bp.blogspot.com/-PvejGH-NIG4/T ... al%2B2.jpg
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Re: 2 Years Post HSCT And MS Still Stopped

Postby shucks » Tue Jun 19, 2012 6:36 pm

Thanks for the response George and Asher. I am trying to figure out how to come up with 150k now based on the info you guys supply. I only have 6 k saved up and am brainstorming. I have a tax exempt 501c3 set up now and just trying to figure out how to pit the word out. You guys are always inspirational i have even pit my wedding on hold to figure it out. Thanks guys.
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Re: 2 Years Post HSCT And MS Still Stopped

Postby georgegoss » Tue Jun 19, 2012 8:20 pm

shucks wrote:Thanks for the response George and Asher. I am trying to figure out how to come up with 150k now based on the info you guys supply. I only have 6 k saved up and am brainstorming. I have a tax exempt 501c3 set up now and just trying to figure out how to pit the word out. You guys are always inspirational i have even pit my wedding on hold to figure it out. Thanks guys.


150K is nearly the most expensive treatment with Dr. Burt in Chicago. Every other facility is less expensive.

And if you want substantially similar HSCT treatment protocol that Dr. Burt offers, CTCI with Prof. Slavin (inventor of non-myeloablative HSCT for autoimmune diseases) in Israel can be had for $94K.

The least expensive HSCT being available for $40K, all of which are listed here:

http://themscure.blogspot.com/2011/06/g ... -have.html
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Re: 2 Years Post HSCT And MS Still Stopped

Postby merlin26 » Tue Jun 19, 2012 8:40 pm

georgegoss wrote:
shucks wrote:Thanks for the response George and Asher. I am trying to figure out how to come up with 150k now based on the info you guys supply. I only have 6 k saved up and am brainstorming. I have a tax exempt 501c3 set up now and just trying to figure out how to pit the word out. You guys are always inspirational i have even pit my wedding on hold to figure it out. Thanks guys.


150K is nearly the most expensive treatment with Dr. Burt in Chicago. Every other facility is less expensive.

And if you want substantially similar HSCT treatment protocol that Dr. Burt offers, CTCI with Prof. Slavin (inventor of non-myeloablative HSCT for autoimmune diseases) in Israel can be had for $94K.

The least expensive HSCT being available for $40K, all of which are listed here:

http://themscure.blogspot.com/2011/06/g ... -have.html



Or you can enlist to be part of Dr. Burt's trial in Chicago and I believe they are going to be restarting a PhaseII/PhaseIII trial in Seattle as part of the HALT-MS program which is a BEAM protocol similar to what George undertook. These of course are free.
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Re: 2 Years Post HSCT And MS Still Stopped

Postby georgegoss » Tue Jun 19, 2012 9:05 pm

I don't intentiontionally mean to be contradictory. Actually all the trials must be externally patient-funded since the trials don't have a funding source (even though approved by the NIH). Dr. Burt's MIST phase III trial currently underway must be funded by either the patients (majority of cases) or patient's-insurance (minority of cases) and is currently approximately $150K. The HALT-MS phase III trial out of Fred Hutchinson in Seattle scheduled to begin enrollment in later 2013 (or 2014 at the latest) is running approximately $215K. Another motivating factor for many to seek a more economical HSCT treatment overseas.
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Re: 2 Years Post HSCT And MS Still Stopped

Postby merlin26 » Tue Jun 19, 2012 11:57 pm

georgegoss wrote:I don't intentiontionally mean to be contradictory. Actually all the trials must be externally patient-funded since the trials don't have a funding source (even though approved by the NIH). Dr. Burt's MIST phase III trial currently underway must be funded by either the patients (majority of cases) or patient's-insurance (minority of cases) and is currently approximately $150K. The HALT-MS phase III trial out of Fred Hutchinson in Seattle scheduled to begin enrollment in later 2013 (or 2014 at the latest) is running approximately $215K. Another motivating factor for many to seek a more economical HSCT treatment overseas.



Do you happen to know if Medicare has covered any of the trial participants?
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Re: 2 Years Post HSCT And MS Still Stopped

Postby shucks » Wed Jun 20, 2012 4:50 am

I was accepted to burts trial and have just exhausted my last insurance appeal. I am now appeal my one an only chance to the state insurance board,which in my case, is the last option. Dr Burt actually lowered the price some to 135k to help me out after insurance denied me. They seem like great folks. I Always appreciate you guys input on this stuff and thanks for all of your insight. 2 year without a relapse would be awesome.
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Re: 2 Years Post HSCT And MS Still Stopped

Postby Pesho » Wed Jun 20, 2012 11:09 am

Asher wrote:Pesho, I can't possibly comment on your personal considerations and circumstances. All I can say is that I went into my HSCT treatment with EDSS 4.5 and will have to come to terms with irreversible damage for the rest of my life. If only I knew of the treatment when I was EDSS 2, I would beg, steal or borrow to have it. For MS the maxim is: Time equals brain mass. At EDSS 2 you've not past the point of no return yet.

Thank you. You know, sometimes you just have to hear a good word to get you going. I will probably have the talks this week, hope things go well. I'm also wondering about asking for money from big companies I worked for/with, like a charity. It is very popular in Bulgaria, just HSCT is not officially approved and there will be negative comments 100%. I'm also wondering, why is the procedure so expensive in the US? I can understand maybe some sort of liability and insurance against lawsuits, but it is two times more expensive than in Germany? Even more expensive than Israel, and both countries are more expensive to live in than the USA?
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Re: 2 Years Post HSCT And MS Still Stopped

Postby CVfactor » Wed Jun 20, 2012 2:40 pm

Why will some insurance companies cover it while others wont? It seems like all insurance companies would be doing everything they could to deny coverage.
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Re: 2 Years Post HSCT And MS Still Stopped

Postby shucks » Wed Jun 20, 2012 5:32 pm

Bcbs always denies it but will approve it on appeal in certain states. Sc is self insured but has the bcbs company manage the fund. Sc is a state where they don't approve it.
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Re: 2 Years Post HSCT And MS Still Stopped

Postby Liberation » Fri Jul 06, 2012 12:46 pm

Hi guys,

I am also interested in hsct with ppms.

I have found the following:

"Mancardi et.al along with Italian BMT Study Group reports the long term follow-up findings from the data obtained from the European Group for Blood and Marrow Transplantation (EBMT) registry. Six centres come along to organize a phase I/II study, aimed to evaluate the outcomes of AHSCT on MRI markers. Later, the patients were subjected to BEAM/ATG conditioning regimen and outcomes were reported to EBMT registry and two leading haemato-neurological centres in Italy. The study was approved by local ethics committee and based upon EBMT guidelines.

After the transplant procedure, all patients were examined every 3 months for first two years and then at least once a year in subsequent period. The study constituted of 74 patients and the median follow-up period of 48.3 (range 0.8 – 126) months. The evaluation is based upon the clinical and MRI outcomes.

Transplant related mortality include early toxic effect found in 80% of total cases, which include neuropenic fever (70%), sepsis (30%), UTI (25%) and diarrhea and severe mucositis (15%). During neuropenic period related fever, transient worsening of neurological symptoms with headache, fatigue, deterioration of motor and sensory symptoms were found in 50% of cases.

The study reports that, after 5 years, two-third remained stable and shows improved outcomes. With follow up time more than 1 yr, 31% of patients with relapsing-remitting course confirmed EDS scale improvement >1 point after AHSCT when compared to 3% which have secondary progressive disease course. In seven year long follow-up of 18 subjects, 44% remained stable and sustained improvement while rest of the subjects after initial stabilization or improvement with mean period of 3.5 shows slow disability progression.

There are currently several clinical trials testing the potential of Stem Cells in MS patients around the world, including a phase I trial at the Cleveland Clinic in Ohio that emerged from the work in Miller’s lab which is based upon hMSCs. The large cohorts like EBMT consistently evaluate the outcomes of hematopoietic stem cell transplantation"

http://blog.uberbrain.net/2012/06/stem- ... rosis.html

Could it be that results vary significantly among hospitals?

I have not been here lately, so I am just wondering if there is any development with other stem cell types like MSC and neuro stem cells. A few months ago lots of promising news came out on them. For me HSCT is too expensive, but if it is worth it for ppms and if there is any way to collect money from companies or charities I would consider it.
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Re: 2 Years Post HSCT And MS Still Stopped

Postby georgegoss » Fri Aug 03, 2012 12:22 pm

Sorry, I just wanted to share a posting that was entered today on our HSCT FB forum. Because these are comments made by an individual that received HSCT outside of a controlled study, this does not specifically translate to any other individual. I'm really just sharing this to show "what's possible." This patient is actually experiencing better post-HSCT results than I have. Most likely because she had an especially agressive form of malignant MS, and I did not. At least the vast majority of MS'ers are able to have their disease stopped via HSCT, and the majority experience improvements. . .

"I am only a little more than 4months post [HSCT] transplant right now, but I have seen improvements already with my walking, and the MS fatigue. My MRI has confirmed that my MS has been STOPPED because this MRI showed NO ACTIVE lesions in my brain or spinal cord. I have ALWAYS shown new and ACTIVE lesion with every new MRI, so it is amazing that the MRI performed 4 months post transplant has shown my MS is stable and inactive now. One brain lesion that was a threat to my life is now shrinking, and my neurologist says that the HSCT is amazing him with these results. My neurologist really thought I would lose the battle with MS and die very soon because my MS was very aggressive and progressing very quickly despite my efforts with all the traditional MS therapies available. I can tell you the sooner you can have the HSCT the better off you will be in the long run. I wish I could have had it done in 2008 when I was diagnosed because I had less damage to my brain and spinal cord then, but I am so thankful I had it done this year on March 27!!!!!! HSCT has given me a future that I wouldn't have without it."
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Re: 2 Years Post HSCT And MS Still Stopped

Postby georgegoss » Fri Aug 03, 2012 12:34 pm

BTW Lib. . . . good posting of that report. Very relevant data.

When you ask "Could it be that results vary significantly among hospitals?," The answer is "not really." The overwhelming factor here that accounts for the variation of the patient clinical outcomes over time is the patient selection criteria. Relapsing patients fare much better, on average, as compared to advanced progressive patients.

All the early (phase I) clinical trial work focussed on advanced progressive SPMS and PPMS (in the range of EDSS 6.0-8.0) and accounts for the reports of early HSCT clinical trial work not looking so good. However, current trial data clearly indicates that virtually 100% of relapsing MS patients have the underlying MS disease activity and progression stopped. And then on top of this greater than >80% of this same population experiences "significant" (greater than >1.0) improvement of EDSS. Unfortunately advanced progressive patients don't fare as well, on average.
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