Intraspinal stem cell transplantation in amyotrophic lateral sclerosis: A phase i safety trial, technical note, and lumbar safety outcomes

Neurosurgery

Volumn 71, Issue 2, 2012, Pages 405-416

  Riley, Jonathan ^a   Federici, Thais ^a   Polak, Meraida ^a   Kelly, Crystal ^a   Glass, Jonathan ^a   Raore, Bethwel ^a   Taub, Jason ^a   Kesner, Vita ^a   Feldman, Eva L ^b   Boulis, Nicholas M ^a  

^a EMORY UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

Author keywords

Amyotrophic lateral sclerosis; Intraspinal microinjection; Stem cell

Indexed keywords

BASILIXIMAB; METHYLPREDNISOLONE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; TACROLIMUS;

ADULT; AGED; AMYOTROPHIC LATERAL SCLEROSIS; ARTICLE; BALLOON CATHETER; CERVICAL SPINE; DEATH; DEPRESSION; DRUG DOSE REDUCTION; EVOKED SOMATOSENSORY RESPONSE; FOOD AND DRUG ADMINISTRATION; HOSPITAL READMISSION; HUMAN; LIQUORRHEA; LUMBAR SPINE; MALE; MICROINJECTION; MICROINJECTOR; NEURAL STEM CELL TRANSPLANTATION; PEROPERATIVE COMPLICATION; PHASE 1 CLINICAL TRIAL; PRIORITY JOURNAL; SAFETY; STEM CELL TRANSPLANTATION; WOUND DEHISCENCE;

EID: 84929237446     PISSN: 0148396X     EISSN: None     Source Type: Journal    
DOI: 10.1227/NEU.0b013e31825ca05f     Document Type: Article

References (22)

1. Mandel RJ. CERE-110, an adeno-associated virus-based gene delivery vector expressing human nerve growth factor for the treatment of Alzheimer's disease. Curr Opin Mol Ther. 2010;12(2):240-247.
2. Kaplitt MG, Feigin A, Tang C, et al. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet. 2007;369(9579):2097-2105.
3. Marks WJ Jr, Ostrem JL, Verhagen L, et al. Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial. Lancet Neurol. 2008;7(5):400-408.
4. Janson C, McPhee S, Bilaniuk L, et al. Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain. Hum Gene Ther. 2002;13(11):1391-1412.
5. Worgall S, Sondhi D, Hackett NR, et al. Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA. Hum Gene Ther. 2008;19(5):463-474.
6. Raore B, Federici T, Taub J, et al. Cervical multilevel intraspinal stem cell therapy: assessment of surgical risks in Gottingen minipigs. Spine (Phila Pa 1976). 2011;36 (3):E164-E171.
7. Federici T, Riley J, Park J, Bain M, Boulis N. Preclinical safety validation of a stabilized viral vector direct injection approach to the cervical spinal cord. Clin Transl Sci. 2009;2(2):165-167.
8. Riley J, Butler J, Baker KB, et al. Targeted spinal cord therapeutics delivery: stabilized platform and microelectrode recording guidance validation. Stereotact Funct Neurosurg. 2008;86(2):67-74.
9. Riley J, Federici T, Park J, et al. Cervical spinal cord therapeutics delivery: preclinical safety validation of a stabilized microinjection platform. Neurosurgery. 2009;65(4):754-761; discussion 761-762.
10. Riley JP, Raore B, Taub JS, Federici T, Boulis NM. Platform and cannula design improvements for spinal cord therapeutics delivery. Neurosurgery. 2011;69(2 suppl operative):ons147-ons154; discussion ons155.
11. Xu L, Ryugo DK, Pongstaporn T, Johe K, Koliatsos VE. Human neural stem cell grafts in the spinal cord of SOD1 transgenic rats: differentiation and structural integration into the segmental motor circuitry. J Comp Neurol. 2009; 514(4):297-309.
12. Xu L, Shen P, Hazel T, Johe K, Koliatsos VE. Dual transplantation of human neural stem cells into cervical and lumbar cord ameliorates motor neuron disease in SOD1 transgenic rats. Neurosci Lett. 2011;494(3):222-226.
13. Xu L, Yan J, Chen D, et al. Human neural stem cell grafts ameliorate motor neuron disease in SOD-1 transgenic rats. Transplantation. 2006;82(7):865-875.
14. Yan J, Xu L, Welsh AM, et al. Extensive neuronal differentiation of human neural stem cell grafts in adult rat spinal cord. PLoS Med. 2007;4(2):e39.
15. Federici T, Boulis NM. Gene therapy for amyotrophic lateral sclerosis [published online ahead of print]. Neurobiol Dis. 2011. doi: 10.1016/j.nbd.2011.08.018.
16. Autologous Incubated Macrophages for Patients With Complete Spinal Cord Injuries. Available at: http://www.clinicaltrials.gov/ct2/show/NCT00073853? term=Proneuron&rank=1. Accessed September 1, 2011.
17. Safety Study of GRNOPC1 in Spinal Cord Injury. Available at: http://www.clinicaltrials. gov/ct2/show/NCT01217008?term=Geron&rank=9. Accessed September 1, 2011.
18. Glass JD, Boulis NM, Johe K, et al. Lumbar intraspinal injection of neural stem cells in patients with amyotrophic lateral sclerosis: results of a phase I trial in 12 patients. Stem Cells. 2012;30(6):1144-1151.
19. Nematalla AH, Bakr MA, Gheith OA, Elagroudy AE, Elshahawy el M, Aghoneim M. Steroid-avoidance immunosuppression regimen in live-donor renal allotransplant recipients: a prospective, randomized, controlled study. Exp Clin Transpl. 2007;5(2):673-679.
20. Blanquer M, Perez EspejoMA, Iniesta F, et al. Bone marrow stem cell transplantation in amyotrophic lateral sclerosis: technical aspects and preliminary results from a clinical trial [in Spanish]. Methods Find Exp Clin Pharmacol. 2010;32(suppl A):31-37.
21. Mazzini L, Ferrero I, Luparello V, et al. Mesenchymal stem cell transplantation in amyotrophic lateral sclerosis: a phase I clinical trial. Exp Neurol. 2010;223(1):229-237.
22. Karussis D, Karageorgiou C, Vaknin-Dembinsky A, et al. Safety and immunological effects of mesenchymal stem cell transplantation in patients with multiple sclerosis and amyotrophic lateral sclerosis. Arch Neurol. 2010;67(10):1187-1194.