Stem cell therapy for neurological disorders

Walter de Gruyter GmbH - Tập 2 - Trang 319-324 - 2011
Koraljka Bačić Baronica1, Latica Friedrich1
1University Department of Neurology, University Hospital Sveti Duh, Zagreb, Croatia

Tóm tắt

Stem cells have long been in focus as potential therapy or even cure for a whole myriad of diseases. Many neurodegenerative disorders, both acute and chronic, are characterized by irreversible neuronal damage and loss, and only a few efficient treatment options exist. In contrast to many other tissues, the potential of self-regeneration of the central nervous system is highly limited. There is hope that stem cells could replace the damaged neuronal and glial cells, and provide biological and functional restoration based on their properties of self renewal and the ability to give rise to different cells. In recent years, the promising results of research on animal models has led to the establishment of the first clinical trials; although no clear evidence of therapeutic benefit for any of the conditions have been ascertained. Here we give a review of the current strategies of stem-cell based therapy for some of the more common neurological disorders, discussing the progress and current challenges, and giving an overview of future perspectives.

Tài liệu tham khảo

Park D. H., Eve D. J., Chung Y. G., Sanberg P. R., Regenerative medicine for neurological disorders, ScientificWorldJournal, 2010, http://www. tswj.com/2010/463612/abs/ Srivastava A. S., Malhotra R., Sharp J., Berggren T., Potentials of ES cell therapy in neurodegenerative diseases, Curr. Pharm. Design., 2008, 14, 3873–3879 Takahashi K., Yamanaka S., Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors, Cell, 2006, 126, 663–676 Baker M., Stem cells: Fast and furious, Nature, 2009, 458, 462–465 Mattis V. B., Svendsen C. N., Induced pluripotent stem cells: a new revolution for clinical neurology?, Lancet Neurol., 2011, 10, 383–394 Park I. H., Arora N., Huo H., Maherali N., Ahfeldt T., Shimamura A. et al., Disease specific induced pluripotent stem cells, Cell, 2008, 134, 877–886 Durnaoglu S., Genc S., Genc K., Patient-specific pluripotent stem cells in neurological diseases, Stem Cells Int., 2011, http://www.hindawi.com/journals/sci/2011/212487/ Doetsch F., Caille I., Lim D.A., Garcia-Verdugo J.M., Alvarez-Buylla A. Subventricular zone astrocytes are neural stem cells in the adult mammalian brain, Cell 97, 1999, 703–716 Reynolds B. A., Weiss S., Generation of neurons and astrocytes from isolated cells of the adult mammalian central nervous system, Science, 1992, 255, 1707–1710 Eriksson P. S., Perfilieva E., Bjork-Eriksson T., Alborn A. M., Nordborg C., Peterson D. A. et al., Neurogenesis in the adult human hippocampus, Nat. Med., 1998, 4, 1313–1317 Alvarez-Buylla A., García-Verdugo J. M., Tramontin A. D., A unified hypothesis on the lineage of neural stem cells, Nat. Rev. Neurosci., 2001, 2, 287–293 Kempermann G., Wiskott L., Gage F. H., Functional significance of adult neurogenesis, Curr. Opin. Neurobiol., 2004, 14, 186–191 Kempermann G. Seven principles in the regulation of adult neurogenesis. Eur J Neurosci., 2011, 33, 1018–24 Zhao C., Deng W., Gage F. H., Mechanisms and functional implications of adult neurogenesis, Cell, 2008, 132, 645–660 Jin K., Sun Y., Xie L., Peel A., Mau X. O., Batteur S. et al., Directed migration of neuronal precursors into the ischemic cerebral cortex and striatum, Mol. Cell. Neurosci., 2003, 24, 171–189 Emery D. L., Fulp C. T., Saatman K. E., Schutz C., Neugebauer E., McIntosh T. K., Newly born granule cells in the dentate gyrus rapidly extend axons into the hippocampal CA3 region following experimental brain injury, J. Neurotrauma, 2005, 22, 978–988 Picard-Riera N., Decker L., Delarasse C., Goude K., Nait-Oumesmar B., Liblau R. et al., Experimental autoimmune encephalomyelitis mobilized neuronal progenitors from the subventricular zone to undergo oligodendrogenesis in adult mice, Proc. Natl. Acad. Sci. U S A, 2002, 99, 13211–13216 Hattiangady B., Shuai B., Cai J., Coksaygan T., Rao M. S., Shetty A. K., Increased dentate neurogenesis after grafting of glial restricted progenitors or neural stem cells in the aging hippocampus, Stem cells, 2007, 25, 2104–2117 Munoz J. R., Stoutenger B. R., Robinson A. P., Spees J. L., Prockop D. J., Human stem/progenitor cells from bone marrow promote neurogenesis of endogenous neural stem cells in the hippocampus of mice, Proc. Natl. Acad. Sci. U S A, 2005, 102, 18171–18176, Erratum in: Proc. Natl. Acad. Sci. U S A, 2006, 103, 2000–2002 Martino G., Pluchino S., The therapeutic potential of neural stem cells, Nat. Rev. Neurosci., 2006, 7, 395–406 Lindvall O., Kokaia Z., Stem cells for the treatment of neurological disorders, Nature, 2006, 441, 1094–1096 Lindvall O., Kokaia Z., Stem cells in human neurodegenerative disorders—time for clinical translation?, J. Clin. Invest., 2010, 120, 29–40 Polgar S., Morris M. E., Reilly S., Bilney B., Sanberg P. R., Reconstructive neurosurgery for Parkinson’s disease: a systematic review and preliminary meta-analysis, Brain Res. Bull., 2003, 60, 1–24 Lindvall O., Björklund A., Cell therapy in Parkinson’s disease, NeuroRx., 2004, 1, 382–393 Brazzini A., Cantella R., De la Cruz A., Yupanqui J., León C., Jorquiera T. et al., Intraarterial autologous implantation of adult stem cells for patients with Parkinson disease, J. Vasc. Interv. Radiol., 2010, 21, 443–451 Venkataramana N. K., Kumar S. K., Balaraju S., Radhakrishnan R. C., Bansal A., Dixit A. et al., Open-labeled study of unilateral autologous bone-marrow-derived mesenchymal stem cell transplantation in Parkinson’s disease, Transl. Res., 2010, 155, 62–70 Lévesque M. F., Neuman T., Rezak M., Therapeutic microinjection of autologous adult human neural stem cells and differentiated neurons for Parkinson’s disease: five-year post-operative outcome, Open Stem Cell J., 2009, http://benthamscience.com/open/toscj/openaccess2.htm Hagell P., Piccini P., Björklund A., Brundin P., Rehncrona S., Widner H. et al., Dyskinesias following neural transplantation in Parkinson’s disease, Nat. Neurosci., 2002, 5, 627–628 Lang A. E., Obeso J. A., Challenges in Parkinson’s disease: restoration of the nigrostriatal dopamine system is not enough, Lancet Neurol., 2004, 3, 309–316 Behrstock S., Ebert A., McHugh J., Vosberg S., Moore J., Schneider B. et al., Human neural progenitors deliver glial cell line-derived neurotrophc factor to parkinsonian rodents and aged primates, Gene Ther., 2006, 13, 379–388 Bachoud-Lévi A. C., Gaura V., Brugières P., Lefaucheur J. P., Boissé M. F., Maison P. et al., Effect of fetal neural transplants in patients with Huntington’s disease 6 years after surgery: a long-term follow-up study, Lancet Neurol., 2006, 5, 303–309 Lescaudron L., Unni D., Dunbar G. L., Autologous adult bone marrow stem cell transplantation in an animal model of Huntington’s disease: behavioral and morphological outcomes, Int. J. Neurosci., 2003, 113, 945–956 McBride J. L., Behrstock S. P., Chen E. Y., Jakel R. J., Siegel I., Svendsen C. N. et al., Human neural stem cell transplants improve motor function in a rat model of Huntington’s disease, J. Comp. Neurol., 2004, 475, 211–219 Ebert A. D., Barber A. E., Heins B. M., Svendsen C. N., Ex vivo delivery ofGDNF maintains motor function and prevents neuronal loss in a transgenic mouse model of Huntington’s disease, Exp. Neurol., 2010, 224, 155–162 Silani V., Cova L., Corbo M., Ciammola A., Polli E., Stem-cell therapy for amyotrophic lateral sclerosis, Lancet, 2004, 364, 200–202 Borchelt D. R., Amyotrophic lateral sclerosis-are microglia killing motor neurons?, N. Engl. J. Med., 2006, 12, 1611–1613 Clement A. M., Nguyen M. D., Roberts E. A., Garcia M. L., Boillée S., Rule M. et al., Wild-type nonneuronal cells extend survival of SOD1 mutant motor neurons in ALS mice, Science, 2003, 302, 113–117 Lepore A. C., Rauck B., Dejea C., Pardo A. C., Rao M.S., Rothstein J. D. et al., Focal transplantation-based astrocyte replacement is neuroprotective in a model of motor neuron disease, Nat. Neurosci, 2008, 11, 1294–1301 Mazzini L., Ferrero I., Luparello V., Rustichelli D., Gunetti M., Mareschi K. et al., Mesenchymal stem cell transplantation in amyotrophic lateral sclerosis: A Phase I clinical trial, Exp. Neurol., 2010, 223, 229–237 Martinez H. R., Gonzalez-Garza M. T., Moreno-Cuevas J. E., Caro E., Gutierrez-Jimenez E., Segura J. J., Stem-cell transplantation into the frontal motor cortex in amyotrophic lateral sclerosis patients, Cytotherapy, 2009, 11, 26–34 Deda H., Inci M. C., Kürekçi A. E., Sav A., Kayihan K., Ozgün E., Treatment of amyotrophic lateral sclerosis patients by autologous bone marrow-derived hematopoietic stem cell transplantation: a 1-year follow-up, Cytotherapy, 2009, 11, 18–25 Karussis D., Karageorgiou C., Vaknin-Dembinsky A., Gowda-Kurkalli B., Gomori J. M., Kassis I. et al., Safety and immunological effects of mesenchymal stem cell transplantation in patients with multiple sclerosis and amyotrophic lateral sclerosis, Arch. Neurol., 2010, 67, 1187–1194 Lunn J. S., Sakowski S. A., Hur J., Feldman E. L., Stem cell technology for neurodegenerative diseases, Ann. Neurol., 2011, 70, 353–361 Martino G., Franklin R. J., Van Evercooren A. B., Kerr D. A., Stem Cells in Multiple Sclerosis (STEMS) Consensus Group. Stem cell transplantation in multiple sclerosis: current status and future prospects, Nat. Rev. Neurol., 2010, 6, 247–255 Scolding N., Adult stem cells and multiple sclerosis, Cell Prolif., 2011, 44(Suppl. 1), 35–38 Fassas A., Anagnostopoulos A., Kazis A., Kapinas K., Sakellari I., Kimiskidis V. et al., Peripheral blood stem cell transplantation in the treatment of progressive multiple sclerosis: first results of a pilot study, Bone Marrow Transplant., 1997, 20, 631–638 Fassas A., Passweg J. R., Anagnostopoulos A., Kazis A., Kozak T., Havrdova E. et al., Autoimmune disease working party of the EBMT (European Group for Blood and Marrow Transplantation). Hematopoietic stem cell transplantation for multiple sclerosis. A retrospective multicenter study, J. Neurol., 2002, 249, 1088–1097 Fassas A., Kimiskidis V. K., Sakellari I., Kapinas K., Anagnostopoulos A., Tsimourtou V. et al., Long-term results of stem cell transplantation for MS: a single-center experience, Neurology, 2011, 76, 1066–1070 Burt R. K., Loh Y., Cohen B., Stefoski D., Balabanov R., Katsamakis G. et al., Autologous non-myeloablative haemopoietic stem cell transplantation in relapsing-remitting multiple sclerosis: a phase I/II study, Lancet Neurol., 2009, 8, 244–253, Erratum in: Lancet Neurol., 2009, 8, 309 Cummings B. J., Uchida N., Tamaki S. J., Salazar D. L., Hooshmand M., Summers R. et al., Human neural stem cells differentiate and promote locomotor recovery in spinal cord-injured mice, Proc. Natl. Acad. Sci. U S A, 2005, 102, 14069–14074 Keirstead H. S., Nistor G., Bernal G., Totoiu M., Cloutier F., Sharp K. et al., Human embryonic stem cell-derived oligodendrocyte progenitor cell transplants remyelinate and restore locomotion after spinal cord injury, J. Neurosci., 2005, 25, 4694–4705 Lee S. H., Chung Y. N., Kim Y. H., Kim Y. J., Park J. P., Kwon D. K. et al., Effects of human neural stem cell transplantation in canine spinal cord hemisection, Neurol. Res., 2009, 31, 996–1002 Sharp J., Frame J., Siegenthaler M., Nistor G., Keirstead H. S., Human embryonic stem cell-derived oligodendrocyte progenitor cell transplants improve recovery after cervical spinal cord injury, Stem Cells, 2010, 28, 152–163 Pal R., Venkataramana N. K., Bansal A., Balaraju S., Jan M., Chandra R. et al., Ex vivo-expanded autologous bone marrow-derived mesenchymal stromal cells in human spinal cord injury/paraplegia: a pilot clinical study, Cytotherapy, 2009, 11, 897–911 Yoon S. H., Shim Y. S., Park Y. H., Chung J. K., Nam J. H., Kim M. O. et al., Complete spinal cord injury treatment using autologous bone marrow cell transplantation and bone marrow stimulation with granulocyte macrophage-colony stimulating factor: Phase I/II clinical trial, Stem Cells, 2007, 25, 2066–2073 Hofstetter C. P., Holmstrom N. A., Lilja J. A., Schweinhardt P., Hao J., Spenger C. et al., Allodynia limits the usefulness of intraspinal neural stem cell grafts; directed differentiation improves outcome, Nat. Neurosci., 2005, 8, 346–353 Macias M. Y., Syring M. B., Pizzi M. A., Crowe M. J., Alexanian A. R., Kurpad S.N. Pain with no gain: Allodynia following neural stem cell transplantation in spinal cord injury, Exp. Neurol., 2006, 201, 335–348 Thored P., Arvidsson A., Cacci E., Ahlenius H., Kallur T., Darsalia V. et al., Persistent production of neurons from adult brain stem cells during recovery after stroke, Stem Cells, 2006, 24, 739–747 Luo Y., Cell-based therapy for stroke, J. Neural. Transm., 2011, 118, 61–74 Shyu W. C., Lee Y. J., Liu D. D., Lin S. Z., Li H., Homing genes, cell therapy and stroke, Front. Biosci., 2006, 11, 899–907 Borlongan C. V., Hadman M., Sanberg C. D., Sanberg P. R., Central nervous system entry of peripherally injected umbilical cord blood cells is not required for neuroprotection in stroke, Stroke, 2004, 35, 2385–2389 Bliss T., Guzman R., Daadi M., Steinberg G. K., Cell transplantation therapy for stroke, Stroke, 2007, 38(Suppl. 2), 817–826 Kondziolka D., Steinberg G. K., Wechsler L., Meltzer C., Elder E., Gebel J. et al., Neurotransplantation for patients with subcortical motor stroke: a phase 2 randomized trial, J. Neurosurg., 2005, 103, 38–45 Savitz S. I., Dinsmore J., Wu J., Henderson G. V., Stieg P., Caplan L. R., Neurotransplantation of fetal porcine cells in patients with basal ganglia infarcts: a preliminary safety and feasibility study, Cerebrovasc. Dis., 2005, 20, 101–107 Bang O. Y., Lee J. S., Lee P. H., Lee G., Autologous mesenchymal stem cell transplantation in stroke patients, Ann. Neurol., 2005, 57, 874–882 Honmou O., Houkin K., Matsunaga T., Niitsu Y., Ishiai S., Onodera R. et al., Intravenous administration of auto serum-expanded autologous mesenchymal stem cells in stroke, Brain, 2011, 134, 1790–1807 Lee J. S., Hong J. M., Moon G. J., Lee P. H., Ahn Y. H., Bang O. Y. et al., A long-term follow-up study of intravenous autologous mesenchymal stem cell transplantation in patients with ischemic stroke, Stem Cells, 2010, 28, 1099–1106 Chopp M., Li Y., Treatment of stroke and intracerebral hemorrhage with cellular and pharmacological restorative therapies, Acta Neurochir. Suppl., 2008, 105, 79–83 Shyu W. C., Lin S. Z., Lee C. C., Liu D. D., Li H., Granulocyte colony-stimulating factor for acute ischemic stroke: a randomized controlled trial, CMAJ, 2006, 174, 927–933 Tang K. C., Trzaska K. A., Smimov S., Kotenko S. V., Schwander S. K., Ellner J. J. et al., Down-regulation of MHC-II in mesenchimal stem cells at high IFN can be partly explained by cytoplasmic retention of CIITA, J. Immunol., 2008, 180, 1826–1833