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Lumbar Intraspinal Injection of Neural Stem Cells in Patients with Amyotrophic Lateral Sclerosis: Results of a Phase I Trial in 12 Patients

dc.contributor.authorGlass, Jonathan D.en_US
dc.contributor.authorBoulis, Nicholas M.en_US
dc.contributor.authorJohe, Karlen_US
dc.contributor.authorRutkove, Seward B.en_US
dc.contributor.authorFederici, Thaisen_US
dc.contributor.authorPolak, Meraidaen_US
dc.contributor.authorKelly, Crystalen_US
dc.contributor.authorFeldman, Eva L.en_US
dc.date.accessioned2012-07-12T17:24:01Z
dc.date.available2013-08-01T14:04:40Zen_US
dc.date.issued2012-06en_US
dc.identifier.citationGlass, Jonathan D.; Boulis, Nicholas M.; Johe, Karl; Rutkove, Seward B.; Federici, Thais; Polak, Meraida; Kelly, Crystal; Feldman, Eva L. (2012). "Lumbar Intraspinal Injection of Neural Stem Cells in Patients with Amyotrophic Lateral Sclerosis: Results of a Phase I Trial in 12 Patients ." STEM CELLS 30(6): 1144-1151. <http://hdl.handle.net/2027.42/92063>en_US
dc.identifier.issn1066-5099en_US
dc.identifier.issn1549-4918en_US
dc.identifier.urihttps://hdl.handle.net/2027.42/92063
dc.description.abstractAdvances in stem cell biology have generated intense interest in the prospect of transplanting stem cells into the nervous system for the treatment of neurodegenerative diseases. Here, we report the results of an ongoing phase I trial of intraspinal injections of fetal‐derived neural stems cells in patients with amyotrophic lateral sclerosis (ALS). This is a first‐in‐human clinical trial with the goal of assessing the safety and tolerability of the surgical procedure, the introduction of stem cells into the spinal cord, and the use of immunosuppressant drugs in this patient population. Twelve patients received either five unilateral or five bilateral (10 total) injections into the lumbar spinal cord at a dose of 100,000 cells per injection. All patients tolerated the treatment without any long‐term complications related to either the surgical procedure or the implantation of stem cells. Clinical assessments ranging from 6 to 18 months after transplantation demonstrated no evidence of acceleration of disease progression due to the intervention. One patient has shown improvement in his clinical status, although these data must be interpreted with caution since this trial was neither designed nor powered to measure treatment efficacy. These results allow us to report success in achieving the phase I goal of demonstrating safety of this therapeutic approach. Based on these positive results, we can now advance this trial by testing intraspinal injections into the cervical spinal cord, with the goal of protecting motor neuron pools affecting respiratory function, which may prolong life for patients with ALS. S TEM C ELLS 2012;30:1144–1151en_US
dc.publisherWiley Subscription Services, Inc., A Wiley Companyen_US
dc.subject.otherMotor Neuron Diseaseen_US
dc.subject.otherSpinal Corden_US
dc.subject.otherStem Cellsen_US
dc.subject.otherAmyotrophic Lateral Sclerosisen_US
dc.subject.otherClinical Trialen_US
dc.titleLumbar Intraspinal Injection of Neural Stem Cells in Patients with Amyotrophic Lateral Sclerosis: Results of a Phase I Trial in 12 Patientsen_US
dc.typeArticleen_US
dc.rights.robotsIndexNoFollowen_US
dc.subject.hlbsecondlevelMolecular, Cellular and Developmental Biologyen_US
dc.subject.hlbtoplevelHealth Sciencesen_US
dc.description.peerreviewedPeer Revieweden_US
dc.contributor.affiliationumDepartment of Neurology, University of Michigan, Ann Arbor, Michigan, USAen_US
dc.contributor.affiliationotherDepartment of Neurology, 101 Woodruff Circle, Suite 6000, Atlanta, Georgia 30322, USAen_US
dc.contributor.affiliationotherDepartment of Neurology, Emory University School of Medicine, Atlanta, Georgia, USAen_US
dc.contributor.affiliationotherDepartment of Neurosurgery, Emory University School of Medicine, Atlanta, Georgia, USAen_US
dc.contributor.affiliationotherNeuralstem, Inc., Rockville, Maryland, USAen_US
dc.contributor.affiliationotherDepartment of Neurology, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, Massachusetts, USAen_US
dc.identifier.pmid22415942en_US
dc.description.bitstreamurlhttp://deepblue.lib.umich.edu/bitstream/2027.42/92063/1/1079_ftp.pdf
dc.description.bitstreamurlhttp://deepblue.lib.umich.edu/bitstream/2027.42/92063/2/SC_11-1235_sm_supplTable1.pdf
dc.description.bitstreamurlhttp://deepblue.lib.umich.edu/bitstream/2027.42/92063/3/SC_11-1235_sm_supplFigure1.pdf
dc.identifier.doi10.1002/stem.1079en_US
dc.identifier.sourceSTEM CELLSen_US
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dc.owningcollnameInterdisciplinary and Peer-Reviewed


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