Has anyone around here had the HSCT treatment and not gotten better? It is kind of like the CCSVI board in that those with good results post the loudest.
I am really trying to figure out if either is worth the cost, and am in the modst of conversations on several other threads about benefits, but what about detriments?
Excellent question that I also look forward to anyone else adding info on this topic. This question of "did the treatment fail?" is very closely related to the question of "what is the definition of a cure?" Can't have one without the other.
There is no universal definition of what a cure for MS is. Even doctors and researchers cannot agree. So actually it is up to each individual to decide for themselves what constitutes a cure. For my own personal definition I consider a cure for MS as a "stopping" or "halting" of the progression of the disease. However, some people may not agree with my nomenclature and will insist that a cure be a complete reversal of all symptomatic manifestations of MS. On balance, the latter is unlikely to be acheived but in a few of individual cases utilizing HSCT.
Based upon the information available from the clinical trials and studies of HSCT as a treatment for MS (there is quite a bit of information), the results of the treatment are stratified primarily by where a person is in the MS disease lifecycle (relapsing vs. progressive) and by total disability (measured by EDSS) at the time of transplantation.
The earliest studies (phase I clinical trials were ten years ago) of HSCT for MS focussed on severely disabled cases of progressive MS patients. Additionally, the HSCT treatment protocol also incorporated a very aggressive ionizing radiation exposure as part of the treatment protocol. It turns out that the radiation caused some troubling complications and some patient deaths and is now-known to not be required to effect curative efficacy of the treatment. Many of these early-treated patients did not respond favorably to HSCT, in that 66% of the advanced primary progressive patients had their disease stopped, and the balance of this same population saw no benefit.
Fast forward to today. . . . since the early phase I clinical trial both the protocol (chemotherapy only, no radiation) and patient selection (excludes progressive patients) has evolved. So the curative results today cannot be directly compared with the early patient trial results.
The amazing thing about the current treatment result data (currently in phase III clinical trial) shows that early RRMS patients have essentially complete stopping of their disease in the entire population. And then on top of this greater then 80% of the population has "substantial" (and sometimes, but not always, complete) reversal (improvement) of existing symptomatic deficit. Additionally there have been no deaths. Absolutely none.
Scientists Reverse Early MS With Patients' Own Stem Cells
After an average follow-up of three years after receiving their transplants (which took place between January 2003 and February 2005), 17 patients (81 per cent) improved by at least one point on a [EDSS] disability scale. And for all [100%] patients, the disease had stopped progressing.
As for varied population morphology of HSCT-treated patient results, there is less data because once the researchers figured out that RRMS responds better to treatment as compared with PPMS, they then basically excluded most progressive patients from the trials. This is actually sad & unfortunate because the majority of progressive patients can also have their underlying MS disease activity stopped, albeit at a reduced population rate. Here is a summary. . .
RRMS = 100% population stopping of disease activity, >80% population with significant EDSS improvement
SPMS with low ambulation (EDSS >6.0) = 79% population stopping of disease activity, symptomatic reversal (improvement) variable (not quantified due to small study population)
PPMS with low ambulation (EDSS >6.0) = 66% population stopping of disease activity, symptomatic reversal (improvement) average not substantial
So treatment earlier in the disease cycle before it gets bad responds much better to the treatment. But also on the other hand that doesn't mean that progressive patients don't see any curative benefit. It's just impossible to predict for any specific individual what the result will be. Just like everything in nature; it's all about probabilities. Regardless of MS disease status, probably the underlying disease activity will be halted.
My own MS status following my HSCT (just as an example which doesn't mean that it translates to anyone else). . . . . I was diagnosed in 1995 with RRMS and then transitioned approximately 2006 into SPMS. I was EDSS 3.5 when I had my HSCT in December 2009 and today my MS disease progression is 100% stopped and my EDSS has improved to 2.0 today (16 months post-transplant). Methematically thats a 40% improvement following HSCT. Amazing this is all without any further treatment or drugs (the last time I had taken Avonex was November, 2009).
Going back to your original question. . . . for people that are progressive and treated by HSCT, clearly there must be some people that did not respond favorably to the treatment. I have not found anyone yet that fits this criteria, but I am still looking. The one big piece of positive news is that since HSCT has been perforemed since 2000, not a single person that had their underlying MS disease activity stopped has yet to progress even now. Positive indication that curative efficacy is long-lasting. Hopefully for lifetime. Time will tell. Sor far, so good.
As of today there have been approximately 600 people with MS treated worldwide with HSCT. So uncovering stories of unsuccessful HSCT treatment may be hard to come by. The good news is that the phase II and phase III clinical trials here in the US have shown virtually every single person to benefit from the treatment. Very compelling evidence that no other treatment can offer today.
http://themscure.blogspot.com/2010/06/s ... rence.html
Last note. . . is it worth it to seek (and pay for expensive) HSCT treatment to cure one's MS? That's definitely something that can only be answered by each individual but I would think it a strong piece of the decision factor as to what stage the existing disease is in.
Very best regards,