The mesenchymal stem cells in multiple sclerosis (MSCIMS) trial protocol and baseline cohort characteristics: an open-label pre-test: post-test study with blinded outcome assessments - PubMed (original) (raw)

Controlled Clinical Trial

doi: 10.1186/1745-6215-12-62.

Madhan Kolappan, Rickie Patani, Michael A Scott, Charles Crawley, Xiao-Ling He, Karen Richardson, Kelly Barber, Daniel J Webber, Claudia A M Wheeler-Kingshott, Daniel J Tozer, Rebecca S Samson, David L Thomas, Ming-Qing Du, Shi L Luan, Andrew W Michell, Daniel R Altmann, Alan J Thompson, David H Miller, Alastair Compston, Siddharthan Chandran

Affiliations

Controlled Clinical Trial

The mesenchymal stem cells in multiple sclerosis (MSCIMS) trial protocol and baseline cohort characteristics: an open-label pre-test: post-test study with blinded outcome assessments

Peter Connick et al. Trials. 2011.

Abstract

Background: No treatments are currently available that slow, stop, or reverse disease progression in established multiple sclerosis (MS). The Mesenchymal Stem Cells in Multiple Sclerosis (MSCIMS) trial tests the safety and feasibility of treatment with a candidate cell-based therapy, and will inform the wider challenge of designing early phase clinical trials to evaluate putative neuroprotective therapies in progressive MS. Illustrated by the MSCIMS trial protocol, we describe a novel methodology based on detailed assessment of the anterior visual pathway as a model of wider disease processes--the "sentinel lesion approach".

Methods/design: MSCIMS is a phase IIA study of autologous mesenchymal stem cells (MSCs) in secondary progressive MS. A pre-test : post-test design is used with healthy controls providing normative data for inter-session variability. Complementary eligibility criteria and outcomes are used to select participants with disease affecting the anterior visual pathway.

Results: Ten participants with MS and eight healthy controls were recruited between October 2008 and March 2009. Mesenchymal stem cells were successfully isolated, expanded and characterised in vitro for all participants in the treatment arm.

Conclusions: In addition to determining the safety and feasibility of the intervention and informing design of future studies to address efficacy, MSCIMS adopts a novel strategy for testing neuroprotective agents in MS--the sentinel lesion approach--serving as proof of principle for its future wider applicability.

Trial registration: ClinicalTrials.gov (NCT00395200).

PubMed Disclaimer

Figures

Figure 1

Figure 1

Recruitment and retention to treatment cohort.

Similar articles

Cited by

References

    1. Compston DAS, Coles AJ. Multiple Sclerosis. Lancet. 2008;372:1502–1517. doi: 10.1016/S0140-6736(08)61620-7. - DOI - PubMed
    1. Kappos L, Freedman MS, Polman CH, Edan G, Hartung HP, Miller DH, Montalbán X, Barkhof F, Radü EW, Bauer L, Dahms S, Lanius V, Pohl C, Sandbrink R. BENEFIT Study Group. Effect of early versus delayed interferon beta-1b treatment on disability after a first clinical event suggestive of multiple sclerosis: a 3-year follow-up analysis of the BENEFIT study. Lancet. 2007;370:389–97. doi: 10.1016/S0140-6736(07)61194-5. - DOI - PubMed
    1. CAMMS223 Trial Investigators. Coles AJ, Compston DA, Selmaj KW, Lake SL, Moran S, Margolin DH, Norris K, Tandon PK. Alemtuzumab vs. interferon beta-1a in early multiple sclerosis. N Engl J Med. 2008;359:1786–801. doi: 10.1056/NEJMoa0802670. - DOI - PubMed
    1. Rivera FJ, Couillard-Despres S, Pedre X, Ploetz S, Caioni M, Lois C, Bogdahn U, Aigner L. Mesenchymal stem cells instruct oligodendrogenic fate decision on adult neural stem cells. Stem Cells. 2006;24:2209–19. doi: 10.1634/stemcells.2005-0614. - DOI - PubMed
    1. Bai L, Lennon DP, Eaton V, Maier K, Caplan AI, Miller SD, Miller RH. Human bone marrow-derived mesenchymal stem cells induce Th2-polarized immune response and promote endogenous repair in animal models of multiple sclerosis. Glia. 2009;57:1192–203. doi: 10.1002/glia.20841. - DOI - PMC - PubMed

Publication types

MeSH terms

Grants and funding

LinkOut - more resources