Clinical translation of stem cell transplantation in Parkinson's disease

Journal of Internal Medicine

Volumn 279, Issue 1, 2016, Pages 30-40

  Lindvall, O ^a  

^a LUND UNIVERSITY HOSPITAL   (Sweden)

Author keywords

Dopaminergic neurons; Parkinson's disease; Reprogramming; Stem cells; Transplantation

Indexed keywords

DOPAMINE;

ADRENAL MEDULLA; BRAIN DEPTH STIMULATION; BRAIN FUNCTION; CAUDATE NUCLEUS; CELL DIVISION; CLINICAL TRIAL (TOPIC); CONTROLLED CLINICAL TRIAL (TOPIC); CORPUS STRIATUM; DOPAMINE RELEASE; DOPAMINERGIC NERVE CELL; EMBRYONIC STEM CELL; FIBROBLAST; GENETIC TRANSCRIPTION; HUMAN; LEWY BODY; MESENCEPHALIC TISSUE; NONHUMAN; NUCLEAR REPROGRAMMING; PARKINSON DISEASE; PHENOTYPE; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; REVIEW; SOMATIC CELL; STEM CELL TRANSPLANTATION; SUBSTANTIA NIGRA; CYTOLOGY; FETAL TISSUE TRANSPLANTATION; MESENCEPHALON; TRANSLATIONAL RESEARCH; TRANSPLANTATION;

DOPAMINERGIC NEURONS; EMBRYONIC STEM CELLS; FETAL TISSUE TRANSPLANTATION; HUMANS; MESENCEPHALON; PARKINSON DISEASE; STEM CELL TRANSPLANTATION; TRANSLATIONAL MEDICAL RESEARCH;

EID: 84949625733     PISSN: 09546820     EISSN: 13652796     Source Type: Journal    
DOI: 10.1111/joim.12415     Document Type: Review

References (53)

1. Perlow MJ, Freed WJ, Hoffer BJ, Seiger A, Olson L, Wyatt RJ. Brain grafts reduce motor abnormalities produced by destruction of nigrostriatal dopamine system. Science 1979; 204: 643-7.
2. Bjorklund A, Stenevi U. Reconstruction of the nigrostriatal dopamine pathway by intracerebral nigral transplants. Brain Res 1979; 177: 555-60.
3. Backlund EO, Granberg PO, Hamberger B et al. Transplantation of adrenal medullary tissue to striatum in parkinsonism. First clinical trials. J Neurosurg 1985; 62: 169-73.
4. Lindvall O, Backlund EO, Farde L et al. Transplantation in Parkinson's disease: two cases of adrenal medullary grafts to the putamen. Ann Neurol 1987; 22: 457-68.
5. Madrazo I, Drucker-Colin R, Diaz V, Martinez-Mata J, Torres C, Becerril JJ. Open microsurgical autograft of adrenal medulla to the right caudate nucleus in two patients with intractable Parkinson's disease. N Engl J Med 1987; 316: 831-4.
6. Brundin P, Strecker RE, Widner H et al. Human fetal dopamine neurons grafted in a rat model of Parkinson's disease: immunological aspects, spontaneous and drug-induced behaviour, and dopamine release. Exp Brain Res 1988; 70: 192-208.
7. Lindvall O. Developing dopaminergic cell therapy for Parkinson's disease - give up or move forward? Mov Disord 2013; 28: 268-73.
8. Freed CR, Greene PE, Breeze RE et al. Transplantation of embryonic dopamine neurons for severe Parkinson's disease. N Engl J Med 2001; 344: 710-9.
9. Olanow CW, Goetz CG, Kordower JH et al. A double-blind controlled trial of bilateral fetal nigral transplantation in Parkinson's disease. Ann Neurol 2003; 54: 403-14.
10. Barker RA, Barrett J, Mason SL, Bjorklund A. Fetal dopaminergic transplantation trials and the future of neural grafting in Parkinson's disease. Lancet Neurol 2013; 12: 84-91.
11. 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: 504-6.
12. Kordower JH, Chu Y, Hauser RA, Olanow CW, Freeman TB. Transplanted dopaminergic neurons develop PD pathologic changes: a second case report. Mov Disord 2008; 23: 2303-6.
13. Kordower JH, Freeman TB, Chen EY et al. Fetal nigral grafts survive and mediate clinical benefit in a patient with Parkinson's disease. Mov Disord 1998; 13: 383-93.
14. Kordower JH, Freeman TB, Snow BJ et al. Neuropathological evidence of graft survival and striatal reinnervation after the transplantation of fetal mesencephalic tissue in a patient with Parkinson's disease. N Engl J Med 1995; 332: 1118-24.
15. Kordower JH, Rosenstein JM, Collier TJ et al. Functional fetal nigral grafts in a patient with Parkinson's disease: chemoanatomic, ultrastructural, and metabolic studies. J Comp Neurol 1996; 370: 203-30.
16. Li JY, Englund E, Holton JL et al. Lewy bodies in grafted neurons in subjects with Parkinson's disease suggest host-to-graft disease propagation. Nat Med 2008; 14: 501-3.
17. Li JY, Englund E, Widner H et al. Characterization of Lewy body pathology in 12- and 16-year-old intrastriatal mesencephalic grafts surviving in a patient with Parkinson's disease. Mov Disord 2010; 25: 1091-6.
18. Mendez I, Sanchez-Pernaute R, Cooper O et al. Cell type analysis of functional fetal dopamine cell suspension transplants in the striatum and substantia nigra of patients with Parkinson's disease. Brain 2005; 128: 1498-510.
19. Mendez I, Vinuela A, Astradsson A et al. Dopamine neurons implanted into people with Parkinson's disease survive without pathology for 14 years. Nat Med 2008; 14: 507-9.
20. Piccini P, Brooks DJ, Björklund A et al. Dopamine release from nigral transplants visualized in vivo in a Parkinson's patient. Nat Neurosci 1999; 2: 1137-40.
21. Politis M, Wu K, Loane C et al. Serotonin neuron loss and nonmotor symptoms continue in Parkinson's patients treated with dopamine grafts. Sci Transl Med 2012; 4: 128ra41.
22. Politis M, Wu K, Loane C et al. Serotonergic neurons mediate dyskinesia side effects in Parkinson's patients with neural transplants. Sci Transl Med 2010; 2: 38ra46.
23. Piccini P, Lindvall O, Björklund A et al. Delayed recovery of movement-related cortical function in Parkinson's disease after striatal dopaminergic grafts. Ann Neurol 2000; 48: 689-95.
24. Ma Y, Tang C, Chaly T et al. Dopamine cell implantation in Parkinson's disease: long-term clinical and (18)F-FDOPA PET outcomes. J Nucl Med 2010; 51: 7-15.
25. Piccini P, Pavese N, Hagell P et al. Factors affecting the clinical outcome after neural transplantation in Parkinson's disease. Brain 2005; 128: 2977-86.
26. Kefalopoulou Z, Politis M, Piccini P et al. Long-term clinical outcome of fetal cell transplantation for Parkinson disease: two case reports. JAMA Neurol 2014; 71: 83-7.
27. Lindvall O, Rehncrona S, Brundin P et al. Human fetal dopamine neurons grafted into the striatum in two patients with severe Parkinson's disease. A detailed account of methodology and a 6-month follow-up. Arch Neurol 1989; 46: 615-31.
28. Hagell P, Piccini P, Björklund A et al. Dyskinesias following neural transplantation in Parkinson's disease. Nat Neurosci 2002; 5: 627-8.
29. Graff-Radford J, Foote KD, Rodriguez RL et al. Deep brain stimulation of the internal segment of the globus pallidus in delayed runaway dyskinesia. Arch Neurol 2006; 63: 1181-4.
30. Herzog J, Pogarell O, Pinsker MO et al. Deep brain stimulation in Parkinson's disease following fetal nigral transplantation. Mov Disord 2008; 23: 1293-6.
31. Carlsson T, Winkler C, Lundblad M, Cenci MA, Bjorklund A, Kirik D. Graft placement and uneven pattern of reinnervation in the striatum is important for development of graft-induced dyskinesia. Neurobiol Dis 2006; 21: 657-68.
32. Maries E, Kordower JH, Chu Y et al. Focal not widespread grafts induce novel dyskinetic behavior in parkinsonian rats. Neurobiol Dis 2006; 21: 165-80.
33. Politis M, Oertel WH, Wu K et al. Graft-induced dyskinesias in Parkinson's disease: high striatal serotonin/dopamine transporter ratio. Mov Disord 2011; 26: 1997-2003.
34. Kurowska Z, Englund E, Widner H, Lindvall O, Li J-Y, Brundin P. Signs of dergeneration in 12-22-year old grafts of mesencephalic dopamine neurons in patients with Parkinson's disease. J Parkinsons Dis 2011; 1: 83-92.
35. Hallett PJ, Cooper O, Sadi D, Robertson H, Mendez I, Isacson O. Long-term health of dopaminergic neuron transplants in Parkinson's disease patients. Cell Rep 2014; 7: 1755-61.
36. Lindvall O, Barker RA, Brustle O, Isacson O, Svendsen CN. Clinical translation of stem cells in neurodegenerative disorders. Cell Stem Cell 2012; 10: 151-5.
37. Roy NS, Cleren C, Singh SK, Yang L, Beal MF, Goldman SA. Functional engraftment of human ES cell-derived dopaminergic neurons enriched by coculture with telomerase-immortalized midbrain astrocytes. Nat Med 2006; 12: 1259-68.
38. Kriks S, Shim JW, Piao J et al. Dopamine neurons derived from human ES cells efficiently engraft in animal models of Parkinson's disease. Nature 2011; 480: 547-51.
39. Kirkeby A, Grealish S, Wolf DA et al. Generation of regionally specified neural progenitors and functional neurons from human embryonic stem cells under defined conditions. Cell Rep 2012; 1: 703-14.
40. Steinbeck JA, Choi SJ, Mrejeru A et al. Optogenetics enables functional analysis of human embryonic stem cell-derived grafts in a Parkinson's disease model. Nat Biotechnol 2015; 33: 204-9.
41. Grealish S, Diguet E, Kirkeby A et al. Human ESC-derived dopamine neurons show similar preclinical efficacy and potency to fetal neurons when grafted in a rat model of Parkinson's disease. Cell Stem Cell 2014; 15: 653-65.
42. Doi D, Samata B, Katsukawa M et al. Isolation of human induced pluripotent stem cell-derived dopaminergic progenitors by cell sorting for successful transplantation. Stem Cell Rep 2014; 2: 337-50.
43. Hargus G, Cooper O, Deleidi M et al. Differentiated Parkinson patient-derived induced pluripotent stem cells grow in the adult rodent brain and reduce motor asymmetry in Parkinsonian rats. Proc Natl Acad Sci USA 2010; 107: 15921-6.
44. Kikuchi T, Morizane A, Doi D et al. Survival of human induced pluripotent stem cell-derived midbrain dopaminergic neurons in the brain of a primate model of Parkinson's disease. J Parkinsons Dis 2011; 1: 395-412.
45. Rhee YH, Ko JY, Chang MY et al. Protein-based human iPS cells efficiently generate functional dopamine neurons and can treat a rat model of Parkinson disease. J Clin Invest 2011; 121: 2326-35.
46. Sundberg M, Bogetofte H, Lawson T et al. Improved cell therapy protocols for Parkinson's disease based on differentiation efficiency and safety of hESC-, hiPSC-, and non-human primate iPSC-derived dopaminergic neurons. Stem Cells 2013; 31: 1548-62.
47. Swistowski A, Peng J, Liu Q et al. Efficient generation of functional dopaminergic neurons from human induced pluripotent stem cells under defined conditions. Stem Cells 2010; 28: 1893-904.
48. Theka I, Caiazzo M, Dvoretskova E et al. Rapid generation of functional dopaminergic neurons from human induced pluripotent stem cells through a single-step procedure using cell lineage transcription factors. Stem Cells Transl Med 2013; 2: 473-9.
49. Hallett PJ, Deleidi M, Astradsson A et al. Successful function of autologous iPSC-derived dopamine neurons following transplantation in a non-human primate model of Parkinson's disease. Cell Stem Cell 2015; 16: 269-74.
50. Caiazzo M, Dell'Anno MT, Dvoretskova E et al. Direct generation of functional dopaminergic neurons from mouse and human fibroblasts. Nature 2011; 476: 224-7.
51. Pfisterer U, Kirkeby A, Torper O et al. Direct conversion of human fibroblasts to dopaminergic neurons. Proc Natl Acad Sci USA 2011; 108: 10343-8.
52. Kim J, Su SC, Wang H et al. Functional integration of dopaminergic neurons directly converted from mouse fibroblasts. Cell Stem Cell 2011; 9: 413-9.
53. Dell'Anno MT, Caiazzo M, Leo D et al. Remote control of induced dopaminergic neurons in parkinsonian rats. J Clin Invest 2014; 124: 3215-29.