Stem cells in Progressive MS

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#MSResearch #MSblog Stem cells in progressive MS 


Connick P, Chandran S. Autologous mesenchymal stem cells for the treatment of secondary progressive multiple sclerosis: an open-label phase 2a proof-of-concept study. J Neurol Neurosurg Psychiatry. 2013 Nov;84(11):e2. doi: 10.1136/jnnp-2013-306573.25.BACKGROUND:Multiple Sclerosis affects over 2.1 million people worldwide,with over half of the prevalent population characterised by progressive forms of disease that result in the steady accumulation of fixed disability.The absence of treatments for progressive MS therefore represents a major unmet clinical need. Based on increasing experimental evidence that mesenchymal stem cells (MSCs) have biological properties of potential therapeutic relevance, and beneficial effects across a range of disease models, we assessed the safety and efficacy of autologous MSCs as a potential neuroprotective therapy for secondary progressive MS.
METHODS: Ten patients with secondary progressive MS involving the visual pathways (EDSS 5.5 to 6.5), received intravenous infusion of autologous bone marrow-derived MSCs derived using the European Group for Blood and Marrow Transplantation ex-vivo expansion procedures in a prospective open label phase IIA proof of concept study, registered at ClinicalTrials.gov NCT00395200. Adverse events were compared between pre-treatment and post-treatment periods of up to 20 and 10 months respectively. Using the anterior visual pathway as a model of wider disease, efficacy outcomes were also chosen to assess change at the point of treatment in functional, structural, and physiological measures. Blinded endpoint analyses were used for electrophysiological and selected imaging outcomes.
RESULTS: MSCs were successfully isolated, expanded, characterised, and administered in all patients. The mean dose was 1·6 million cells (min-max range 1·1-2·0 millin) per kg bodyweight. One patient developed a transient rash not requiring treatment shortly after treatment; and two patients had self-limiting bacterial infections 3-4 weeks after treatment. No serious adverse events were observed. Improvement following treatment was seen in visual acuity and VER latency (difference in monthly rates of change: -0·02 LogMAR units [95% CI -0·03 to -0·01], p=0·003); and -1·33 ms [95% CI -2·44 to -0·21], p=0.020), with an increase in optic nerve area (difference in monthly rates of change 0·13 mm2 [95% CI 0·04 to 0·22], p=0·006). Exploratory analysis for non-linear effects showed cessation of treatment effects approximately six months after intravenous infusion.
CONCLUSIONS: Autologous MSCs can be safely administered in secondary progressive MS with evidence to suggest structural, functional and physiological improvement following treatment consistent with neuroprotection. The transient treatment response seen implied a likely requirement for repeat infusions to sustain benefit in the long-term.

This pioneering stem cell study reports some transient improvements in visual function, Vision was selected because the visual pathway can be used to monitor repair.  If the latency (loss of speed) of electrophysiology increases it can be associated with remyelination. However the treatment effects were not sustained. Whilst we should be encouraged we should also be realistic as this study indicates that stem cell transplantation may not yet be the miracle cure that the media hype is leading us to believe.

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MouseDoctor

4 comments

  • Its ok to take every month Tysabri, which may be very dangerous because of PML, but its completely wrong if stem cells would have to be administered every month. Makes sense.

  • I am not talking the rights and wrongs of repeated infusions and if we think monthly tysabri is expensive i suspect monthly infusions would be more so.
    We will need to see if benefit is sustained by repeated treatments afterall it is possible that the benefit was due to a placebo effect lasting 6months.
    The point i am making is that it is early days and shows we should not be expecting miracle cures just yet.
    The reality has yet to be the hype.

  • Stem cells are definitely a hot button topic, much more deserving than CCSVI. Interesting that the beneficial effect on SPMSers is transient. Is it possible to time-line the re-populating immune cells following HSCT and see what population of cells are associated with cessation of treatment effect? A guest blog on stem cell treatment would be a big plus.

  • I completely agree. Stem cells play an important role in new life growth such as for body parts, hairs etc. You might have often heard of stem cell therapy which is for multiple treatments such as hair loss treatment, blood circulation etc.

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