Just to share some info from a technical paper on this same (primary progressive) subject from the early phase I HSCT clinical trials that focused on late-stage progressive MS. After the researchers compared treatment efficacy between progressive and relapsing forms of the disease, they quickly discovered that HSCT is "less effective" (but not completely ineffective) for PPMS cases as opposed to RRMS and SPMS cases. I refer you specifically to this paper titled "Hematopoietic Stem Cell Transplantation for Multiple Sclerosis" from 2005:
http://pegasus.fmrp.usp.br/projeto/arti ... igo113.pdf
And specifically this passage. . . .
In a European retrospective analysis of 85 patients,
the progression-free survival at 3 years was 78%
in secondary progressive MS and 66% in primary progressive
MS At Northwestern University, Chicago, Ill,
of 21 patients with secondary progressive MS treated using
a myeloablative HSCT regimen, disease progression
in more disabled patients with a pretreatment EDSS score
of 6.0 or higher was significantly worse compared with
those with an EDSS score below 6.0. In fact, none of
the 9 patients with an EDSS score below 6.0 had disease
progression worsening by 1.0 or more EDSS points after
more than 2 years of follow-up.
I find it interesting that there is a clinically-differentiated curative efficacy population result between PP and SP at equivalent EDSS levels. I have found no theories offered to explain this phenomenon. Clearly PP and SP disease activity are both dominated by axonal dystrophy. I can't imagine how the two cases would differ.
Bottom line. . . Clearly progressive (PP and SP) patients see less overall curative benefit from HSCT (but that does not mean “no” benefit.). Overall advanced PPMS cases (EDSS >6.0) still "stopped” or “halted” the underlying progression of the MS disease process in approximately two-thirds of this PP progressive population, and nearly 80% of the SPMS population. And although not clearly demonstrated (the clinical trial population was too small to draw such a final conclusion), there is indication from the SP population that being PPMS with an ambulatory EDSS bodes particularly well for achieving curative results as opposed to cases that are not ambulatory.
A couple other items to note regarding this paper. . . .
- TBI is no longer used in any HSCT protocol for treatment of MS because it has been found to be unhelpful under all circumstances. In fact, it is the most detrimental part of the initial study protocols, responsible for the deaths that had occurred at that early study stage. TBI is no longer used and there have been zero deaths in the phase II chemical-only protocols.
- The BEAM protocol is the gentlest & safest of the myeloablatiove chemo protocols while simultaneously being efficacious. A good regimen for this treatment.
Based on this info if I were PPMS, regardless of disability status, I personally might attempt to get HSCT to stop my own disease. There is a chance that my disease would be stopped, and a lesser chance that it would not. For me, better-than-even odds is superior to thinking there is nothing that will work. And being SPMS, that would likely fair even better.
I think there is an argument to be made that this treatment has a reasonable chance of achieving the objective of “stopping” or “halting” of the underlying MS disease activity & progression for PPMS and advanced SPMS cases. If that is achieved, it could at least bring a better degree of certaintly to the future of one’s life. And there’s also the possibility that some symptomatic improvement following HSCT could be seen. Only time would tell for any given individual in that area.
http://themscure.blogspot.com/2010/06/s ... rence.html