First of all my empathy for you and your sister. No one should have to endure the stress of having to deal with MS. I hope someday the world will be ridded (prevented) of this meanace.
Very sorry for my long-winded response. I'm still trying to get the nerd out of my head. He's not gone yet.
Also I'm glad Hud had a chance to add his comments. His specific MS case type (ambulatory PPMS with EDSS <6.0) is unusual in the sense that this is not a well-studied area for determining curative results of HSCT. His feedback regarding this is valuable information.
A little history. . . . all the early HSCT studies were performed on advanced MS cases with people that were generally not ambulatory. Nearly the entire population of this group were either PPMS or SPMS in the EDSS 6.0 - 8.0 range because it was thought at that time to perform HSCT on the most severely disabled patients. So the curative statistics are well understood for this group, which I list below.
But also in these early years there was one rapidly-evolving severely disabled RRMS patient (EDSS 8.0) in this group of treated patients that also underwent HSCT for his MS. And low-and-behold this patient eventually recovered to en EDSS of approximately 2.0 - 3.0 range. This almost fantasically unbeleivable anomolous result shocked the researchers and is what finally made them realize that HSCT can work more effectively on early relapsing cases. It has in fact been seen that three things "generally" indicate more favorable outcome. . . . 1) Relapsing (inflammatory) disease status, 2) Shorter duration of time from initial diagnosis, and 3) Ambulatory with EDSS <6.0. Having even one or two out of these three also bodes better for progressive (SP, PP) patients.
Here are the statistics of all the patient grouping populations:
For early RRMS cases
Stopping of underlying MS disease activity in virtually 100% of all patients
"Substantial" improvement/reversal of pre-existing symptoms
For later RRMS cases with EDSS >6.0
Excellent probability of stopping disease activity (my own guess is in 95% - 99% of the population)
"Substantial" improvement/reversal of pre-existing symptoms also likely for this population
For advanced SPMS cases with EDSS >6.0
Stopping of underlying MS disease activity in 78% of patients
Improvement/reversal of pre-existing MS symptoms range from none-to-moderate (impossible to predict) based on current data
For early SPMS cases (EDSS <6.0) [my case]
This not a widely studied area, but there is some data that indicates approximately 85-95% will have their MS stopped and are "likely" to experience none-to-substantial improvement/reversal of pre-existing symptoms, athough this magnitude of improvement is unpredicatable
For advanced PPMS cases (EDSS >6.0)
Stopping of underlying MS disease activity in 66% of patients
Improvement/reversal of pre-existing MS symptoms unlikely, but also not impossible.
For earlier PPMS cases (EDSS <6.0)
This is the most unstudied and not quantified population result that only has anecdotal patient outcomes.
There is early indication that PPMS patients with an EDSS <6.0 will do better both in terms of stopping of disease (I would expect a probability of better 66%) and potential improvement of disability. But there simply is no population data to make an accurate probability prediction. Certainly I would expect the statistics to be better for this group as opposed to the advanced PPMS cases, but can't say precisely what that is.
When taking into account PPMS patient types, the picture is not wholly complete. However, the main takeaway lessons from this data are 1) Treatment earlier in the disease lifecycle with lower EDSS is better, and 2) "Most" (but not all) patients will see a beneficial result (stopping of underlying disease activity and possible symptomatic improvement) regardless of the type of disease they have, although this is less than 100% of all people treated.
Cutting to the present day. . . . the researchers running the clinical trials (such as Dr. Burt in Chicago and Dr. Nash in Seattle) have made the decision to limit treated patients to early RRMS / rapidly evolving patients because HSCT works "best" on such patients, not because the treatment cannot have beneficial effects for progressive patients as well. Basically, by limiting the patient inclusion criteria as they have, they get to look like medical superstars letting people get out of their wheelchairs. This is how they will acheive fame and glory. I don't know exactly what their personal motivations are.
I took the limited data points from all the trials over time and created an interpolated graph on this page that attempts to correlate HSCT efficacy with MS disease type (which clearly indicates that "most" MS patients benefit from HSCT, regardless of disease type):http://themscure.blogspot.com/2010/06/s ... rence.html
Going to the situation with your sister. . . . . . Of course there are no gaurantees in life, just probabilities. But I think it not reckless to make some general statements regarding your sister having reasonably good odds of beneficial effect from HSCT because of these factors that you describe of her status:
1) Relatively recent (2007) diagnosis
2) Ambulatory (EDSS <6.0)
So when you state "I believe that HSCT is potentially the best, and possibly only, option for my sister,"
I would agree with you for the simple fact that, although there is no gaurantee your sister would arrest her MS via HSCT, certainly she has far better-than-even odds of doing so. For myself, if I were in the same situation as you have described, likely I would seek HSCT.
I am really hoping that Hud will eventually be able to affirmatively report his underlying PPMS disease activity as stopped. At least the odds are also in his favor. He has given his disease a great shot across the chops and I applaud him for that!
Just to share one additional anecedotal case. . . . I started comminucating last year with a man from Seattle who's wife has PPMS (diagnosed 2002, EDSS 6.0). He also read my blog and we talked about her case and they decided to seek a myeloablative HSCT protocol (identical to my own) in Bangalore, India. For the approximate timeframe of two years prior to her HSCT she went from an EDSS 2.0 to 6.0. Clearly a very rapid deterioration (which is probably why they decided so expeditiously to seek a serious treatment like HSCT). Long story short, I spoke with him yesterday and his wife is currently four-and-a-half months post-transplant and still recovering. It is still early in her recovery and I would not expect to notice any specific benefit until at least the 6-12 month timeframe. But interestingly she claims to be convinced that her disease is now stopped (no further physical deterioration). Additionally, she says that she is no longer sensitive to heat (this was also my first symptom to dissappear immediately following my own HSCT so I actually beleive this is likely real and bodes well for her disease being stopped), and she claims that she has regained balance/coordination and speech impairment, although she is still working on physical therapy of her quite weak legs. From her report, it sounds like she may have "possibly" already seen some early indication that her underlying disease activity has been halted. Another six months should indicate for sure which side of the cure curve she has come down on. But so far so good with no bad news as of yet.
The point here is that individual cases cannot and do not specifically translate to anyone else. Only a population study can tell us for sure what the probability of outcome will be. But even then, predicting an individual's outcome is impossible. I would imagine that over time more MS'ers in this ambulatory PPMS population will receive HSCT treatment and that will slowly build up a better picture of what result is likely to occur. Until then, I hope to see more people fight their disease!