Why not stem cell treatments in MS? #ShiftMS #MSBlog #ClinicSpeak
“The following is the next MS Report as part of our collaboration with Shift.ms. The topic addresses the issue of stem cell treatments in MS.”
CoI: this work has been generously funded by the Wellcome Trust, Thank You.
Is the following is missing?
Inserted ProfG missed it off
Prof G,Slightly depressing that you don't think stem cells will repair damage. Is there anything else that might provide some repair in the no too distant. Living with some fixed disability is hard enough. Knowing you have it until the day you dies is unbearable. I do wonder if MS neuros really appreciate this.
We have to keep it in prospective stems cells have huge potential but based on studies in animals i would say we are running before we can walk.In animals the repair that stems do is limited at present. I think the more likely chance of success are drug that stimulate the bodies own stem cells to start to repair. These trials are already ongoing
That was a nicely presented explanation. It will be handy when friends and family tell me about something they read about stem cells.
The likelyhood of death is 0.5 to 2%,in your own literature you say the risk of PML on natalizumab at 2+ years if JCV positive is 1/118 which is 1.18%. I know you will be quick to point out that PML is not death but I'm pretty sure that nearly all PML suffers are serverly disabled (EDSS 7 to use a crude, poorly relevant scale like EDSS) Also isn't the prelim outcomes of HSCT more efficient than all the other drugs we have? So slightly more risk for higher reward? Any of the above incorrect?
1 divided by 118 is 0.85 I think the rate of PML is about 1 in 90 at 2 years
The 1.5 to 2% mortality is the estimated mortality risk for myeloablative procedures. Non myeloablative mortality risk is much less – i think it was estimated at about 0.5% at less by various centres, including Dr Burt.
The reparative stem cell study currently underway at the Tisch MS Research Center of New York is showing promising results, with a relatively high number of test subjects demonstrating some level of measurable physical improvement. Though this is a small phase 1 study (currently comprised of 20 test subjects), it is FDA approved, and uses mesenchymal derived neural progenitor cells injected intrathecally that, as stated above, do appear to have some appreciable reparative effect. It is a very early study, though, and the center is currently facing funding shortfalls that are making transitioning into a larger phase 2 study more difficult than anticipated. Here's a link to some of the preliminary data provided by the researchers. At the very least, reason for hope:http://tischms.org/news/june-29-2015-tisch-ms-research-center-new-york-delivers-additional-encouraging-results-phase-i
Reason for hope but the funding shortfalls is not a good thing to know as results such as these can sound like a sales pitch.A phase II needs predefined endpoints otherwise they will be acussed of p-hackingBut lets hope this is good news
The optimism and wishful thinking for this type of trial is so intense that I wouldn't regard any trial as "promising" unless it is placebo controlled with blinded patients and blinded raters. Any open label "stem cell" trial is going to show "positive" results based on patient optimism alone