Autologous mesenchymal stem cells for the treatment of secondary progressive multiple sclerosis: an open-label phase 2a proof-of-concept study

The Lancet Neurology - Tập 11 - Trang 150-156 - 2012
Peter Connick1, Madhan Kolappan2, Charles Crawley3, Daniel J Webber4, Rickie Patani4, Andrew W Michell1, Ming-Qing Du5, Shi-Lu Luan6, Daniel R Altmann7, Alan J Thompson8, Alastair Compston1, Michael A Scott3, David H Miller2, Siddharthan Chandran9
1Department of Clinical Neurosciences, University of Cambridge, Cambridge, UK
2NMR Research Unit, Institute of Neurology, Department of Neuroinflammation, University College London, London, UK
3Blood and Marrow Transplant Unit, Addenbrooke's Hospital, Cambridge, UK
4Anne MacLaren Laboratory for Regenerative Medicine, University of Cambridge, Cambridge, UK
5Department of Pathology, University of Cambridge, Cambridge, UK
6Department of Veterinary Medicine, University of Cambridge, Cambridge, UK
7Medical Statistics Department, London School of Hygiene and Tropical Medicine, London, UK
8Department of Brain Repair and Rehabilitation, University College London, London, UK
9Centre for Clinical Brain Sciences, MRC Centre for Regenerative, Medicine University of Edinburgh, Edinburgh, UK

Tài liệu tham khảo

Pugliatti, 2006, The epidemiology of multiple sclerosis in Europe, Eur J Neurol, 13, 700, 10.1111/j.1468-1331.2006.01342.x Compston, 2008, Multiple sclerosis, Lancet, 372, 1502, 10.1016/S0140-6736(08)61620-7 Coles, 2008, Alemtuzumab vs interferon beta-1a in early multiple sclerosis, N Engl J Med, 359, 1786, 10.1056/NEJMoa0802670 Kappos, 2007, 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, 370, 389, 10.1016/S0140-6736(07)61194-5 Coles, 2006, The curious incident of disability in multiple sclerosis trials, Lancet Neurol, 5, 899, 10.1016/S1474-4422(06)70585-3 Pittenger, 1999, Multilineage potential of adult human mesenchymal stem cells, Science, 284, 143, 10.1126/science.284.5411.143 Arthur, 2009, The therapeutic applications of multipotential mesenchymal/stromal stem cells in skeletal tissue repair, J Cell Physiol, 218, 237, 10.1002/jcp.21592 Uccelli, 2011, Mesenchymal stem cells for the treatment of multiple sclerosis and other neurological diseases, Lancet Neurol, 10, 649, 10.1016/S1474-4422(11)70121-1 Payne, 2011, The prospect of stem cells as multi-faceted purveyors of immune modulation, repair and regeneration in multiple sclerosis, Curr Stem Cell Res Ther, 6, 50, 10.2174/157488811794480735 Zappia, 2005, Mesenchymal stem cells ameliorate experimental autoimmune encephalomyelitis inducing T-cell anergy, Blood, 106, 1755, 10.1182/blood-2005-04-1496 Zhang, 2006, Bone marrow stromal cells reduce axonal loss in experimental autoimmune encephalomyelitis mice, J Neurosci Res, 84, 587, 10.1002/jnr.20962 Bai, 2009, Human bone marrow-derived mesenchymal stem cells induce Th2-polarized immune response and promote endogenous repair in animal models of multiple sclerosis, Glia, 57, 1192, 10.1002/glia.20841 Gerdoni, 2007, Mesenchymal stem cells effectively modulate pathogenic immune response in experimental autoimmune encephalomyelitis, Ann Neurol, 61, 219, 10.1002/ana.21076 Kassis, 2008, Neuroprotection and immunomodulation with mesenchymal stem cells in chronic experimental autoimmune encephalomyelitis, Arch Neurol, 65, 753, 10.1001/archneur.65.6.753 Le Blanc, 2008, Mesenchymal stem cells for treatment of steroid-resistant, severe, acute graft-versus-host disease: a phase II study, Lancet, 371, 1579, 10.1016/S0140-6736(08)60690-X Sun, 2009, Mesenchymal stem cell transplantation reverses multiorgan dysfunction in systemic lupus erythematosus mice and humans, Stem Cells, 27, 1421, 10.1002/stem.68 Carrion, 2010, Autologous mesenchymal stem cell treatment increased T regulatory cells with no effect on disease activity in two systemic lupus erythematosus patients, Lupus, 19, 317, 10.1177/0961203309348983 Mohyeddin Bonab, 2007, Does mesenchymal stem cell therapy help multiple sclerosis patients? Report of a pilot study, Iran J Immunol, 4, 50 Yamout, 2010, Bone marrow mesenchymal stem cell transplantation in patients with multiple sclerosis: a pilot study, J Neuroimmunol, 227, 185, 10.1016/j.jneuroim.2010.07.013 Karussis, 2010, Safety and immunological effects of mesenchymal stem cell transplantation in patients with multiple sclerosis and amyotrophic lateral sclerosis, Arch Neurol, 67, 1187, 10.1001/archneurol.2010.248 Mehta, 2009, Proof of concept studies for tissue-protective agents in multiple sclerosis, Mult Scler, 15, 542, 10.1177/1352458508101939 Kolappan, 2009, Assessing structure and function of the afferent visual pathway in multiple sclerosis and associated optic neuritis, J Neurol, 256, 305, 10.1007/s00415-009-0123-z Connick, 2011, 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, Trials, 12, 62, 10.1186/1745-6215-12-62 Poser, 1983, New diagnostic criteria for multiple sclerosis: guidelines for research protocols, Ann Neurol, 13, 227, 10.1002/ana.410130302 Dominici, 2006, Minimal criteria for defining multipotent mesenchymal stromal cells. The International Society for Cellular Therapy position statement, Cytotherapy, 8, 315, 10.1080/14653240600855905 Johnson, 2006, Application of array CGH on archival formalin-fixed paraffin-embedded tissues including small numbers of microdissected cells, Lab Invest, 86, 968, 10.1038/labinvest.3700441 Oberg, 2007, Linear mixed effects models, Methods Mol Biol, 404, 213, 10.1007/978-1-59745-530-5_11 Lublin, 1996, Defining the clinical course of multiple sclerosis: results of an international survey. National Multiple Sclerosis Society (USA) Advisory Committee on Clinical Trials of New Agents in Multiple Sclerosis, Neurology, 46, 907, 10.1212/WNL.46.4.907 Barkhof, 2003, Remyelinated lesions in multiple sclerosis: magnetic resonance image appearance, Arch Neurol, 60, 1073, 10.1001/archneur.60.8.1073 Schmierer, 2004, Magnetization transfer ratio and myelin in postmortem multiple sclerosis brain, Ann Neurol, 56, 407, 10.1002/ana.20202 Franklin, 2008, Remyelination in the CNS: from biology to therapy, Nat Rev Neurosci, 9, 839, 10.1038/nrn2480 Rivera, 2006, Mesenchymal stem cells instruct oligodendrogenic fate decision on adult neural stem cells, Stem Cells, 24, 2209, 10.1634/stemcells.2005-0614 Bai, 2007, Human mesenchymal stem cells signals regulate neural stem cell fate, Neurochem Res, 32, 353, 10.1007/s11064-006-9212-x