Clinical neurotransplantation: Core assessment protocol rather than sham surgery as control

Brain Research Bulletin

Volumn 58, Issue 6, 2002, Pages 547-553

  Boer, Gerard J ^a   Widner, Håkan ^b  

^a NETHERLANDS INSTITUTE FOR NEUROSCIENCE   (Netherlands)

^b LUND UNIVERSITY   (Sweden)

Author keywords

Core assessment protocol; Double blind; Ethics; Neurotransplantation; Parkinson's disease; Sham surgery

Indexed keywords

ADRENAL MEDULLA; BRAIN DEPTH STIMULATION; BRAIN TISSUE; BRAIN TRANSPLANTATION; CENTRAL NERVOUS SYSTEM; CLINICAL TRIAL; CONVALESCENCE; DEGENERATIVE DISEASE; DOPAMINE RELEASE; DOPAMINERGIC NERVE CELL; HUMAN; INFORMED CONSENT; MEDICAL ETHICS; NERVE GRAFT; NEUROSCIENCE; NONHUMAN; PARKINSON DISEASE; PREOPERATIVE PERIOD; PRIORITY JOURNAL; REVIEW; RISK BENEFIT ANALYSIS; TREATMENT OUTCOME;

ADRENAL MEDULLA; ANIMALS; BRAIN TISSUE TRANSPLANTATION; CLINICAL TRIALS; FETAL TISSUE TRANSPLANTATION; HUMANS; PARKINSON DISEASE; PATIENT SELECTION; PLACEBOS; TRANSPLANTATION, AUTOLOGOUS;

EID: 0037201104     PISSN: 03619230     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0361-9230(02)00804-3     Document Type: Review

References (63)

1. Abe K. Therapeutic potential of neurotrophic factors and neural stem cells against ischemic brain injury. J. Cereb. Blood Flow Metab. 20:2000;1393-1408.
2. Bachoud-Levi A.C., Remy P., Nguyen J.P., Brugieres P., Lefaucheur J.P., Bourdet C., Baudic S., Gaura V., Maison P., Haddad B., Boisse M.F., Grandmougin T., Jeny R., Bartolomeo P., Dalla Barba G., Degos J.D., Lisovoski F., Ergis A.M., Pailhous E., Cesaro P., Hantraye P., Peschanski M. Motor and cognitive improvements in patients with Huntington's disease after neural transplantation. Lancet. 356:2000;1975-1979.
3. Backlund E.-O., Granber P.-O., Hamberger B., Sedvall G., Seiger A., Olson L. Transplantation of adrenal medullary tissue to striatum in parkinsonism: First clinical trials. J. Neurosurg. 62:1985;169-173.
4. Benabid A.L., Pollak P., Gervason C.L., Hoffmann D., Gao D.M., Hommel M., Perret J.E., De Rougemont J. Long term suppression of tremor by chronic stimulation of the ventral intermediate nucleus thalamic nucleus. Lancet. 337:1991;403-406.
5. Boer G.J. Ethical guidelines for the use of human embryonic or fetal tissue for experimental and clinical neurotransplantation and research. J. Neurol. 242:1994;1-13.
6. Boer, G. J. The self-restraining ethical guidelines of NECTAR for the clinical neurotransplantation investigations. In: Hubig, C.; Poser, C., eds. Cognitio humana-Dynamik des Wissens und der Werte, XVII Deutscher Kongress für Philosophie, Leipzig; 1996:1420-1427.
7. Boer G.J. Ethical issues in neurografting of human embryonic cells. Theoret. Med. Bioethics. 20:1999;461-475.
8. Boer G.J. The Network of European CNS Transplantation And Restoration (NECTAR): An introduction on the occasion of its tenth meeting. Cell Transplant. 9:2000;133-137.
9. Brundin P., Karlsson J., Emgård M., Kaminski Schierle G.S., Hansson O., Petersen A., Castilho R.F. Improving the survival of grafted dopaminergic neurons: A review over current approaches. Cell Transplant. 9:2000;179-195.
10. Brundin P., Dunnett S., Bjorklund A., Nikkhah G. Transplanted dopaminergic neurons: More or less? Nat. Med. 7:2001;512-513.
11. Council of Europe. Convention for the protection of human rights and dignity of the human being with regard to the application of biology and medicine. European Treaty Series 164; 1997.
12. CPMP Working Party on Efficacy of Medicinal Products. EEC note for guidance: Good clinical practice for trials on medicinal products in the European community. Pharmacol. Toxicol. 67:361-372; 1990.
13. Defer G.L., Widner H., Marie R.M., Remy P., Levivier M. Conference participants, core assessment program for surgical interventional therapies in Parkinsons' disease (CAPSIT-PD). Mov. Disord. 14:1999;572-584.
14. Defer G.L., Geny C., Ricolfi F., Fenelon G., Monfort J.C., Remy P., Villafane G., Jeny R., Samson Y., Keravel Y., Gaston A., Degos J.D., Peschanski M., Cesaro P., Nguyen J.P. Long-term outcome of unilaterally transplanted parkinsonian patients. I. Clinical approach. Brain. 119:1996;41-50.
15. Dekkers W.J.M., Boer G.J. Sham surgery in patients with Parkinson's disease: Is it morally acceptable? J. Med. Ethics. 27:2001;151-156.
16. De la Fuente-Fernandez R., Ruth T.J., Sossi V., Schulzer M., Calne D.B., Stoessel A.J. Expectation and dopamine release: Mechanism of the placebo effect in Parkinson's disease. Science. 293:2001;1164-1166.
17. Dunnett S.B., Bjorklund A., Lindvall O. Cell therapy in Parkinson's disease - Stop or go? Nat. Rev. Neurosci. 2:2001;365-369.
18. During M.J., Samulski R.J., Elsworth J.D., Kaplitt M.G., Leone P., Xiao X., Li J., Freese A., Taylor J.R., Roth R.H., Sladek J.R., O-Malley K.L., Redmond D.E. In vivo expression of therapeutic human genes for dopamine production in the caudates of MPTP-treated monkeys using an AAV vector. Gene Ther. 5:1998;820-827.
19. Falci S., Holtz A., Akesson E., Azizi M., Ertzgaard P., Hultling C., Kjaeldgaard A., Levi R., Ringden O., Westgren M., Lammertse D., Seiger A. Obliteration of a posttraumatic spinal cord cyst with solid human embryonic spinal cord grafts: First clinical attempt. J. Neurotrauma. 14:1997;875-884.
20. Felten D.L. Cell transplantation and research design. Science. 263:1994;1546.
21. Freed C.R., Breeze R.E., Rosenberg N.L., Schneck S.A., Kriek E., Qi J.X., Lone T., Zhang Y.B., Snyder J.A., Wells T.H. Survival of implanted fetal dopamine cells and neurologic improvement 12 to 46 months after transplantation for Parkinson's disease. N. Engl. J. Med. 237:1992;1549-1555.
22. Freed C.R., Greene P.E., Breeze R.E., Tsai W.Y., DuMouchel W., Kao R., Dillon S., Winfield H., Culver S., Trojanowski J.Q., Eidelberg D., Fahn S. Transplantation of embryonic dopamine neurons for severe Parkinson's disease. N. Engl. J. Med. 344:2001;710-719.
23. Freeman, W. J. Neural transplantation: An introduction. Cambridge, MA: The MIT Press; 2000.
24. Freeman T.B., Vawter D., Goetz C.G., Leaverton P.E., Hauser R.A., Sanberg P.R., Godbold J.H., Olanow C.W. Towards the use of surgical placebo-controlled trials. Transplant. Proc. 29:1997;1925.
25. Freeman Th.B., Vawter D.E., Leaverton P.E., Godbold J.H., Hauser R.A., Goetz C.G., Olanow C.W. Use of placebo surgery in controlled trials of a cellular-based therapy for Parkinson's disease. N. Engl. J. Med. 341:1999;988-991.
26. Hauser R.A., Freeman T.B., Snow B.J., Nauert M., Gauger L., Kordower J., Olanow C.W. Long-term evaluation of bilateral fetal nigral transplantation in Parkinson's disease. Arch. Neurol. 56:1999;179-187.
27. Henderson B., Clough C., Hughes R., Hitchkock E., Keny B. Implantation of human ventral mesencephalon to the right caudate nucleus in advanced Parkinson's disease. Arch. Neurol. 48:1991;822-827.
28. Isacson O., Bjorklund L., Pernaute R.S. Parkinson's disease: Interpretations of transplantation study are erroneous. Nat. Neurosci. 4:2001;553.
29. Itakura T., Uematsu Y., Nakao N., Nakai E., Nakai K. Transplantation of autologous sympathetic ganglion into the brain with Parkinson's disease: Long-term follow-up of 35 cases. Stereotact. Funct. Neurosurg. 69:1997;112-115.
30. Johnson A.G. Surgery as placebo. Lancet. 344:1994;1140-1142.
31. Kopyov O.V., Jacques S., Lieberman A., Durna C.M., Eagle K.S. Safety of intrastriatal neurotransplantation for Huntington's disease patients. Exp. Neurol. 149:1998;97-108.
32. Kordower J.H., Freeman T.B., Chen E.Y., Mufson E.J., Sanberg P.R., Hauser R.A., Snow B., Olanow C.W. Fetal nigral grafts survive and mediate clinical benefit in a patient with Parkinson's disease. Mov. Disord. 13:1998;383-393.
33. Kordower J.H., Emborg M.E., Bloch J., Ma S.Y., Chu Y., Leventhal L., McBride J., Chen E.Y., Palfi S., Roitberg B.Z., Brown W.D., Holden J.E., Pyzalski R., Taylor M., Carvey P., Ling Z., Trono D., Hantraye P., Deglon N., Aebischer P. Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease. Science. 290:2000;767-773.
34. Latchman D.S., Coffin R.S. Viral vectors for gene therapy in Parkinson's disease. Rev. Neurosci. 12:2001;69-78.
35. Langston J.W., Widner H., Goetz C.G., Brooks D., Fahn S., Freeman T.et al. Core assessment program for intracerebral transplantation (CAPIT). Mov. Disord. 7:1992;2-13.
36. Levivier M., Dethy S., Rodesch F., Peschanski M., Vandesteene A., David P., Wikler D., Goldman S., Claes T., Biver F., Liesnard C., Goldmanb M., Hildebrand J., Brotchi J. Intracerebral transplantation of fetal ventral mesencephalon for patients with advanced Parkinson's disease: Methodology and 6-month to 1-year follow-up in 3 patients. Stereotact. Funct. Neurosurg. 69:1997;99-111.
37. Lindvall O. Cerebral implantation in movement disorders: State of the art. Mov. Disord. 14:1999;201-205.
38. Lindvall O., Brundin P., Widner H., Rehncrona S., Gustavii B., Frackowiak R., Leenders K.L., Sawle G., Rothwell J.C., Marsden C.D., Björklund A. Grafts of fetal dopamine neurons survive and improve motor function on Parkinson's disease. Science. 247:1990;574-577.
39. Lindvall O., Rehncrona S., Brundin P., Gutavii B., Åstedt B., Widner H.et al. Human fetal dopamine neurons grafted to the striatum in two patients with severe Parkinson's disease: A detailed account of methodology and a 6 months follow-up. Arch. Neurol. 46:1989;615-631.
40. López-Lozano J.J., Bravo G., Brera B., Millan I., Dargallo J., Salmeán J., Uria J., Insausti J. Long-term improvement in patients with severe Parkinson's disease after implantation of fetal ventral mesencephalic tissue in a cavity of the caudate nucleus: 5-Year follow up in 10 patients. J. Neurosurg. 86:1997;931.
41. Macklin R. The ethical problems with sham surgery in clinical research. N. Engl. J. Med. 341:1999;992-996.
42. Madrazo I., Drucker-Colin R., Diaz V., Martinez-Mata J., Torres C., Becerril J.J. Open microsurgical autograft of adrenal medulla to the right caudate nucleus in two patients with intractable Parkinson's disease. N. Engl. J. Med. 316:1987;831-834.
43. Madrazo I., Franco-Bourland M., Aguilera M., Ostrosky-Solis F., Cuevas C., Castregon H., Velazquez D., Grijalva E., Guizar-Sahagun G., Magallon E., Madrazo M. Fetal ventral mesencephalon brain homotransplantation in Parkinson's disease: The Mexican experience. Restor. Neurol. 4:1991;123-130.
44. Madrazo I., Franco-Bourland R.E., Castrejon H., Cuevas C., Ostrosky-Solis F. Fetal striatal homotransplantation for Huntington's disease: First two case reports. Neurol. Res. 17:1995;312-315.
45. Nishino H., Borlongan C.V. Restoration of function by neural transplantation in the ischemic brain. Prog. Brain Res. 127:2000;461-476.
46. Peschanski M., Defer G., N'Guyen J.P., Ricolfi F., Monfort J.C., Remy P., Geny C., Samson Y., Hantraye P., Jeny R. Bilateral motor improvement and alteration of L-dopa effect in two patients with Parkinson's disease following intrastriatal transplantation of foetal ventral mesencephalon. Brain. 117:1994;487-499.
47. Peschanski M., Césaro P., Hantraye P. Rationale for intrastriatal grafting of striatal neuroblasts in patients with Huntington's disease. Neuroscience. 68:1995;273-285.
48. Peschanski M., Césaro P., Hantraye P. What is needed versus what would be interesting to know before undertaking neural transplantation in patients with Huntington's disease. Neuroscience. 71:1996;899-900.
49. Philpott L.M., Kopyov O.V., Lee A.J., Jacques S., Duma C.M., Caine S., Yang M., Eagle K.S. Neuropsychological functioning following fetal striatal transplantation in Huntington's chorea: Three case presentations. Cell Transplant. 6:1997;203-212.
50. Piccini P., Brooks D.J., Björklund A., Gunn R.N., Grasby P.M., Rimoldi O., Brundin P., Hagell P., Rehncrona S., Widner H., Lindvall O. Dopaminergic release from nigral transplants visualized in vivo in a Parkinson's patient. Nat. Neurosci. 2:1999;1137-1140.
51. Quinn N., Brown R., Craufurd D., Goldman S., Hodges J., Kieburtz K., Lindvall O., MacMillan J., Roos R. (CAPIT-HD committee), core assessment programme for intracerebral transplantation in Huntington's disease (CAPIT-HD). Mov. Disord. 11:1996;143-150.
52. Spencer D.D., Robbins R.J., Naftolin F., Marek K.L., Vollmer T., Leranth C., Roth R.H., Price L.H., Gjedde A., Bunney B.S. Unilateral transplantation of human fetal mesencephalon tissue into the caudate nucleus of patients with Parkinson's disease'. N. Engl. J. Med. 327:1992;1541-1548.
53. Tasker R.R., Siqueira J., Hawrrylyshyn P., Organ L.W. What happened to VIM thalamotomy for Parkinson's disease. Appl. Neurophysiol. 46:1983;68-83.
54. Tremblay J.P., Malouin F., Roy R., Huard J., Bouchard J.P., Satoh A., Richards C.L. Results of a triple blind clinical study of myoblast transplantations without immunosuppressive treatment in young boys with Duchenne muscular dystrophy. Cell Transplant. 2:1993;99-112.
55. Tuszynski, M. H.; Connor, J.; Blesch, A.; Smith, D.; Merill, D.; Vahlsing, L. New strategies in neural repair. Prog. Brain Res. 138: in press; 2002.
56. United Nations, Universal declaration of human rights. Yearbook of the United Nations 1948-1949, Dept. of Public Information, United Nations. New York; 1948.
57. Vogel G. Parkinson's research, fetal cell transplant trial draws fire. Science. 291:2001;2060-2061.
58. Watts R.l., Freeman T.B., Hauser R.A., Bakay R.A., Ellias S.A., Stoessel A.J., Eidelberg D., Fink J.S. A double blind randomized, controlled, multicenter clinical trial of the safety and efficacy of stereotaxic intrastriatal implantation of fetal porcine ventral mesencephalic tissue (Neurocell-PD) vs. imitation surgery in patients with Parkinson's disease. Parkinsonims Relat. Disord. 7:2001;S87.
59. World Medical Association Declaration of Helsinki. Ethical principles for medical research involving human subjects. JAMA 284:3043-3045; 2000.
60. Widner H., Tetrud J., Rehncrona S., Snow B., Brundin P., Gustavii B., Björklund A., Lindvall O., Langston J.W. Bilateral fetal mesencephalic grafting in two patients with parkinsonism induced by 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine (MPTP). N. Engl. J. Med. 327:1992;1556-1563.
61. Widner H., Defer G.L. Dyskinesias assessment: From CAPIT to CAPSIT. Mov. Disord. 14(suppl. 1):1999;60-66.
62. Widner H. NIH neural transplantation funding. Science. 263:1994;737.
63. Zurn A.D., Tseng J., Aebischer P. Treatment of Parkinson's disease: Symptomatic cell therapies: Cells as biological minipumps. Eur. Neurol. 36:1996;405-408.