A brief perspective on neural cell therapy
Abstract
For a range of nervous system disorders current treatment options remain limited. Focusing on Parkinson’s disease as a neurodegenerative entity that affects an increasing quantity of people in our aging societies, we briefly discuss remaining challenges and opportunities that neural stem cell therapy might be able to offer. Providing a snapshot of neural transplantation paradigms, we contemplate possible imminent translational scenarios and discuss critical requirements to be considered before clinical implementation.
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Politis M, Lindvall O: Clinical application of stem cell therapy in Parkinson’s disease. BMC Med. 2012, 10: 1-10.1186/1741-7015-10-1.
PubMedCentralCrossRefPubMedGoogle Scholar
Tsui A, Isacson O: Functions of the nigrostriatal dopaminergic synapse and the use of neurotransplantation in Parkinson’s disease. J Neurol. 2011, 258: 1393-1405. 10.1007/s00415-011-6061-6.
CrossRefPubMedGoogle Scholar
Bjorklund A, Kordower JH: Cell therapy for Parkinson’s disease: what next?. Mov Disord. 2013, 28: 110-115. 10.1002/mds.25343.
CrossRefPubMedGoogle Scholar
Barker RA, Barrett J, Mason SL, Björklund A: Fetal dopaminergic transplantation trials and the future of neural grafting in Parkinson’s disease. Lancet Neurol. 2013, 12: 84-91. 10.1016/S1474-4422(12)70295-8.
CrossRefPubMedGoogle Scholar
Freed CR, Greene PE, Breeze RE, Tsai WY, DuMouchel W, Kao R, Dillon S, Winfield H, Culver S, Trojanowski JQ, Eidelberg D, Fahn S: Transplantation of embryonic dopamine neurons for severe Parkinson’s disease. N Engl J Med. 2001, 344: 710-719. 10.1056/NEJM200103083441002.
CrossRefPubMedGoogle Scholar
Olanow CW, Goetz CG, Kordower JH, Stoessl AJ, Sossi V, Brin MF, Shannon KM, Nauert GM, Perl DP, Godbold J, Freeman TB: A double-blind controlled trial of bilateral fetal nigral transplantation in Parkinson’s disease. Ann Neurol. 2003, 54: 403-414. 10.1002/ana.10720.
CrossRefPubMedGoogle Scholar
Mendez I, Viñuela A, Astradsson A, Mukhida K, Hallett P, Robertson H, Tierney T, Holness R, Dagher A, Trojanowski JQ, Isacson O: Dopamine neurons implanted into people with Parkinson’s disease survive without pathology for 14 years. Nat Med. 2008, 14: 507-509. 10.1038/nm1752.
PubMedCentralCrossRefPubMedGoogle Scholar
Li JY, Englund E, Holton JL, Soulet D, Hagell P, Lees AJ, Lashley T, Quinn NP, Rehncrona S, Björklund A, Widner H, Revesz T, Lindvall O, Brundin P: Lewy bodies in grafted neurons in subjects with Parkinson’s disease suggest host-to-graft disease propagation. Nat Med. 2008, 14: 501-503. 10.1038/nm1746.
CrossRefPubMedGoogle Scholar
Kordower JH, Chu Y, Hauser RA, Freeman TB, Olanow CW: Lewy body-like pathology in long-term embryonic nigral transplants in Parkinson’s disease. Nat Med. 2008, 14: 504-506. 10.1038/nm1747.
CrossRefPubMedGoogle Scholar
Cooper O, Astradsson A, Hallett P, Robertson H, Mendez I, Isacson O: Lack of functional relevance of isolated cell damage in transplants of Parkinson’s disease patients. J Neurol. 2009, 256 (Suppl 3): 310-316.
CrossRefPubMedGoogle Scholar
Politis M, Wu K, Loane C, Quinn NP, Brooks DJ, Oertel WH, Björklund A, Lindvall O, Piccini P: Serotonin neuron loss and nonmotor symptoms continue in Parkinson’s patients treated with dopamine grafts. Sci Transl Med. 2012, 4: 128ra41-
CrossRefPubMedGoogle Scholar
Olanow CW, Isacson O: Stem cells for Parkinson’s disease: advancing science but protecting patients. Mov Disord. 2012, 27: 1475-1477. 10.1002/mds.25170.
CrossRefPubMedGoogle Scholar
Bianco P, Barker R, Brüstle O, Cattaneo E, Clevers H, Daley GQ, De Luca M, Goldstein L, Lindvall O, Mummery C, Robey PG, Sattler De Sousa E, Brito C, Smith A: Regulation of stem cell therapies under attack in Europe: for whom the bell tolls. EMBO J. 2013, 32: 1489-1495. 10.1038/emboj.2013.114.
PubMedCentralCrossRefPubMedGoogle Scholar
Smith Y, Wichmann T, Factor SA, DeLong MR: Parkinson’s disease therapeutics: new developments and challenges since the introduction of levodopa. Neuropsychopharmacology. 2012, 37: 213-246. 10.1038/npp.2011.212.
PubMedCentralCrossRefPubMedGoogle Scholar
Miocinovic S, Somayajula S, Chitnis S, Vitek JL: History, applications, and mechanisms of deep brain stimulation. JAMA Neurol. 2013, 70: 163-171. 10.1001/2013.jamaneurol.45.
CrossRefPubMedGoogle Scholar
Ramachandran PS, Keiser MS, Davidson BL: Recent advances in RNA interference therapeutics for CNS diseases. Neurotherapeutics. 2013, 10: 473-485. 10.1007/s13311-013-0183-8.
PubMedCentralCrossRefPubMedGoogle Scholar
Lindvall O: Developing dopaminergic cell therapy for Parkinson’s disease–give up or move forward?. Mov Disord. 2013, 28: 268-273. 10.1002/mds.25378.
CrossRefPubMedGoogle Scholar
Evans JR, Mason SL, Barker RA: Current status of clinical trials of neural transplantation in Parkinson’s disease. Prog Brain Res. 2012, 200: 169-198.
CrossRefPubMedGoogle Scholar
Murry CE, Keller G: Differentiation of embryonic stem cells to clinically relevant populations: lessons from embryonic development. Cell. 2008, 132: 661-680. 10.1016/j.cell.2008.02.008.
CrossRefPubMedGoogle Scholar
Ito D, Okano H, Suzuki N: Accelerating progress in induced pluripotent stem cell research for neurological diseases. Ann Neurol. 2012, 72: 167-174. 10.1002/ana.23596.
CrossRefPubMedGoogle Scholar
Turaç G, Hindley CJ, Thomas R, Davis JA, Deleidi M, Gasser T, Karaöz E, Pruszak J: Combined flow cytometric analysis of surface and intracellular antigens reveals surface molecule markers of human neuropoiesis. PLoS One. 2013, 8: e68519-10.1371/journal.pone.0068519.
PubMedCentralCrossRefPubMedGoogle Scholar
Yuan SH, Martin J, Elia J, Flippin J, Paramban RI, Hefferan MP, Vidal JG, Mu Y, Killian RL, Israel MA, Emre N, Marsala S, Marsala M, Gage FH, Goldstein LS, Carson CT: Cell-surface marker signatures for the isolation of neural stem cells, glia and neurons derived from human pluripotent stem cells. PLoS One. 2011, 6: e17540-10.1371/journal.pone.0017540.
PubMedCentralCrossRefPubMedGoogle Scholar
Pruszak J, Isacson O: Molecular and cellular determinants for generating ES-cell derived dopamine neurons for cell therapy. Adv Exp Med Biol. 2009, 651: 112-123. 10.1007/978-1-4419-0322-8_11.
CrossRefPubMedGoogle Scholar
Kriks S, Studer L: Protocols for generating ES cell-derived dopamine neurons. Adv Exp Med Biol. 2009, 651: 101-111. 10.1007/978-1-4419-0322-8_10.
CrossRefPubMedGoogle Scholar
Solozobova V, Wyvekens N, Pruszak J: Lessons from the embryonic neural stem cell niche for neural lineage differentiation of pluripotent stem cells. Stem Cell Rev. 2012, 8: 813-829. 10.1007/s12015-012-9381-8.
PubMedCentralCrossRefPubMedGoogle Scholar
Anisimov SV, Christophersen NS, Correia AS, Li JY, Brundin P: “NeuroStem Chip”: a novel highly specialized tool to study neural differentiation pathways in human stem cells. BMC Genomics. 2007, 8: 46-10.1186/1471-2164-8-46.
PubMedCentralCrossRefPubMedGoogle Scholar
Carson MJ, Doose JM, Melchior B, Schmid CD, Ploix CC: CNS immune privilege: hiding in plain sight. Immunol Rev. 2006, 213: 48-65. 10.1111/j.1600-065X.2006.00441.x.
PubMedCentralCrossRefPubMedGoogle Scholar
Zhao T, Zhang ZN, Rong Z, Xu Y: Immunogenicity of induced pluripotent stem cells. Nature. 2011, 474: 212-215. 10.1038/nature10135.
CrossRefPubMedGoogle Scholar
Nakatsuji N, Nakajima F, Tokunaga K: HLA-haplotype banking and iPS cells. Nat Biotechnol. 2008, 26: 739-740. 10.1038/nbt0708-739.
CrossRefPubMedGoogle Scholar
Liu GH, Yi F, Suzuki K, Qu J, Izpisua Belmonte JC: Induced neural stem cells: a new tool for studying neural development and neurological disorders. Cell Res. 2012, 22: 1087-1091. 10.1038/cr.2012.73.
PubMedCentralCrossRefPubMedGoogle Scholar
Gaj T, Gersbach CA, Barbas CF: ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering. Trends Biotechnol. 2013, 31: 397-405. 10.1016/j.tibtech.2013.04.004.
PubMedCentralCrossRefPubMedGoogle Scholar
Lancaster MA, Renner M, Martin CA, Wenzel D, Bicknell LS, Hurles ME, Homfray T, Penninger JM, Jackson AP, Knoblich JA: Cerebral organoids model human brain development and microcephaly. Nature. 2013, 501: 373-379. 10.1038/nature12517.
CrossRefPubMedGoogle Scholar
Adzick NS: Fetal surgery for spina bifida: past, present, future. Semin Pediatr Surg. 2013, 22: 10-17. 10.1053/j.sempedsurg.2012.10.003.
CrossRefPubMedGoogle Scholar
Bellenchi GC, Volpicelli F, Piscopo V, Perrone-Capano C, Di Porzio U: Adult neural stem cells: an endogenous tool to repair brain injury?. J Neurochem. 2013, 124: 159-167. 10.1111/jnc.12084.
CrossRefPubMedGoogle Scholar
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