Gene therapy clinical trials in the human brain

Frontiers of Neurology and Neuroscience

Volumn 25, Issue , 2009, Pages 180-188

  Kaplitt, Michael G ^a  

^a WEILL CORNELL MEDICAL COLLEGE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

4 AMINOBUTYRIC ACID; ACICLOVIR; CARBOXYLYASE; GANCICLOVIR; GLUTAMATE DECARBOXYLASE; HERPESVIRUS VECTOR; LEVODOPA; LIPOSOME; NAKED DNA; NERVE GROWTH FACTOR; NEURTURIN; ONCOLYTIC VIRUS; ONYX 015; PARVOVIRUS VECTOR; RETROVIRUS VECTOR; THYMIDINE KINASE;

ABSENCE OF SIDE EFFECTS; ALZHEIMER DISEASE; BRAIN HEMORRHAGE; BRAIN TUMOR; CELL CULTURE; CENTRAL NERVOUS SYSTEM DISEASE; CLINICAL PROTOCOL; CLINICAL TRIAL; DNA MODIFICATION; DRUG MEGADOSE; EX VIVO GENE TRANSFER; GENE TARGETING; GENE THERAPY; GENE TRANSFER; GLIA CELL; GLIOBLASTOMA; HUMAN; IN VIVO GENE TRANSFER; LIPOSOMAL GENE DELIVERY SYSTEM; LOW DRUG DOSE; NERVE DEGENERATION; NEUROLOGIC DISEASE; ONCOLYTIC VIROTHERAPY; PARKINSON DISEASE; PRIORITY JOURNAL; REVIEW; TREATMENT RESPONSE; UNSPECIFIED SIDE EFFECT; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS GENE; BRAIN DISEASE; METHODOLOGY; STANDARD;

BRAIN DISEASES; CLINICAL PROTOCOLS; CLINICAL TRIALS AS TOPIC; GENE THERAPY; HUMANS;

EID: 67649822974     PISSN: 16604431     EISSN: 16622804     Source Type: Book Series    
DOI: 10.1159/000209498     Document Type: Review

References (47)

1. Culver K W, Van Gilder J, Link CJ, Carlstrom T, Buroker T, Yuh W, Koch K, Schabold K, Doombas S, Wetjen B: Gene therapy for the treatment of malignant brain tumors with in vivo tumor transduction with the herpes simplex thymidine kinase gene/ganciclovir system. Hum Gene Ther 1994;5:343-379.
2. Oldfield EH, Ram Z, Culver LW, Blaese RM, DeVroom HL, Anderson WF: Gene therapy for the treatment of brain tumors using intra-tumoral transduction with the thymidine kinase gene and intravenous ganciclovir. Hum Gene Ther 1993;4:39-69.
3. van Dillen IJ, Mulder NH, Vaalburg W, de Vries EFJ, Hospers GAP: Influence of the bystander effect on HSV-tk/GCV gene therapy. A review. Curr Gene Ther 2002;2:307-322.
4. Pope IM, Poston GJ, Kinsella AR: The role of the bystander effect in suicide gene therapy. Eur J Cancer 1997;33:1005-1016.
5. Kun LE, Gajjar A, Muhlbauer M, Heideman RL, Sanford R, Brenner M, Walter A, Langston J, Jenkins J, Facchini S: Stereotactic injection of herpes simplex thymidine kinase vector producer cells (PA317-G1Tk1SvNa.7) and intravenous ganciclovir for the treatment of progressive or recurrent primary supratentorial pediatric malignant brain tumors. Hum Gene Ther 1995;6:1231-1255.
6. Packer RJ, Raffel C, Villablanca JG, Tonn JC, Burdach SE, Burger K, LaFond D, McComb JG, Cogen PH, Vezina G, Kapcala LP: Treatment of progressive or recurrent pediatric malignant supratentorial brain tumors with herpes simplex virus thymidine kinase gene vector-producer cells followed by intravenous ganciclovir administration. J Neurosurg 2000;92:249-254.
7. Prados MD, McDermott M, Chang SM, Wilson CB, Culver K W, Van Gilder J, Keles G, Spence A, Berger M: Treatment of progressive or recurrent glioblastoma multiforme in adults with herpes simplex virus thymidine kinase gene vector-producer cells followed by intravenous ganciclovir administration: a phase I/II multi-institutional trial. J Neurooncol 2003;65:269-278.
8. Ram Z, Culver K W, Oshiro EM, Viola JJ, DeVroom HL, Otto E, Long Z, Chiang Y, McGarrity GJ, Muul LM, Katz D, Blaese RM, Oldfield EH: Therapy of malignant brain tumors by intratumoral implantation of retroviral vector-producing cells. Nat Med 1997;3:1354-1361.
9. Shand N, Weber F, Mariani L, Bernstein M, Gianella-Borradori A, Long Z, Sorensen AG, Barbier N: A phase 1-2 clinical trial of gene therapy for recurrent glioblastoma multiforme by tumor transduction with the herpes simplex thymidine kinase gene followed by ganciclovir. GLI328 European-Canadian Study Group. Hum Gene Ther 1999;10:2325-2335.
10. Klatzmann D, Valery CA, Bensimon G, Marro B, Boyer O, Mokhtari K, Diquet B, Salzmann JL, Philippon J: A phase I/II study of herpes simplex virus type 1 thymidine kinase 'suicide' gene therapy for recurrent glioblastoma. Study Group on Gene Therapy for Glioblastoma. Hum Gene Ther 1998; 9:2595-2604.
11. Rainov, NG: A phase III clinical evaluation of herpes simplex virus type 1 thymidine kinase and ganciclovir gene therapy as an adjuvant to surgical resection and radiation in adults with previously untreated glioblastoma multiforme. Hum Gene Ther 2000;11:2389-2401.
12. Eck SL, Alavi JB, Alavi A, Davis A, Hackney D, Judy K, Mollman J, Phillips PC, Wheeldon EB, Wilson JM: Treatment of advanced CNS malignancies with the recombinant adenovirus H5.010RSVTK: a phase I trial. Hum Gene Ther 1996;7:465-1482.
13. Germano IM, Fable J, Gultekin SH, Silvers A: Adenovirus/herpes simplex-thymidine kinase/ganciclovir complex: preliminary results of a phase I trial in patients with recurrent malignant gliomas. J Neurooncol 2003;65:279-289.
14. Immonen A, Vapalahti M, Tyynelä K, Hurskainen H, Sandmair A, Vanninen R, Langford G, Murray N, Ylä-Herttuala S: AdvHSV-tk gene therapy with intravenous ganciclovir improves survival in human malignant glioma: a randomised, controlled study. Mol Ther 2004;10:967-972.
15. Trask T W, Trask R P, Aguilar-Cordova E, Shine HD, Wyde PR, Goodman JC, Hamilton WJ, Rojas-Martinez A, Chen SH, Woo SLC, Grossman RG: Phase I study of adenoviral delivery of the HSV-tk gene and ganciclovir administration in patients with current malignant brain tumors. Mol Ther 2000; 1:195-203.
16. Fulci G, Chiocca EA: The status of gene therapy for brain tumors. Expert Opin Biol Ther 2007;7:197-208.
17. Pulkkanen KJ, Yla-Herttuala S: Gene therapy for malignant glioma: current clinical status. Mol Ther 2005;12:585-598.
18. Russell SJ,. Peng KW: Viruses as anticancer drugs. Trends Pharmacol Sci 2007;28:326-333.
19. Vaha-Koskela MJ, Heikkila JE, Hinkkanen AE: Oncolytic viruses in cancer therapy. Cancer Lett 2007;254:178-216.
20. Markert KM, Parker JN, Buchsbaum DJ, Grizzle WE, Gillespie G Y, Whitley RJ: Oncolytic HSV-1 for the treatment of brain tumours. Herpes 2006;13:66-71.
21. Harrow S, Papanastassiou V, Harland J, Mabbs R, Petty R, Fraser M, Hadley D, Patterson J, Brown SM, Rampling R: HSV1716 injection into the brain adjacent to tumour following surgical resection of highgrade glioma: safety data and long-term survival. Gene Ther 2004;11:1648-1658.
22. Markert JM, Medlock MD, Rabkin SD, Gillespie GY, Todo T, Hunter WD, Palmer CA, Feigenbaum F, Tornatore C, Tufaro F, Martuza RL: Conditionally replicating herpes simplex virus mutant, G207 for the treatment of malignant glioma: results of a phase I trial. Gene Ther 2000;7:867-874.
23. Chiocca EA, Abbed KM, Tatter S, Louis DN, Hochberg FH, Barker F, Kracher J, Grossman SA, Fisher JD, Carson K, Rosenblum M, Mikkelsen T, Olson J, Markert J, Rosenfeld S, Nabors LB, Brem S, Phuphanich S, Freeman S, Kaplan R, Zwiebel J: A phase I open-label, dose-escalation, multi-institutional trial of injection with an E1B-Attenuated adenovirus, ONYX-015, into the peritumoral region of recurrent malignant gliomas, in the adjuvant setting. Mol Ther 2004;10:958-966.
24. Freeman AI, Zakay-Rones Z, Gomori JM, Linetsky E, Rasooly L, Greenbaum E, Rozenman-Yair S, Panet A, Libson E, Irving CS, Galun E, Siegal T: Phase I/II trial of intravenous NDV-HUJ oncolytic virus in recurrent glioblastoma multiforme. Mol Ther 2006;13:221-228.
25. Lang AE, Lozano AM: Parkinson's disease. First of two parts. N Engl J Med 1998;339:1044-1053.
26. Samii A, Nutt JG, Ransom BR: Parkinson's disease. Lancet 2004;363:1783-1793.
27. Kaplitt MG, Leone P, Samulski RJ, Xiao X, Pfaff DW, O'Malley KL, During MJ: Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat Genet 1994;8:148-154.
28. Luo J, Kaplitt MG, Fitzsimons HL, Zuzga DS, Liu Y, Oshinsky ML, During MJ: Subthalamic GAD gene therapy in a Parkinson's disease rat model. Science 2002;298:425-429.
29. Erlander, Tillakaratne NJ, Feldblum S, Patel N, Tobin AJ: Two genes encode distinct glutamate decarboxylases. Neuron 1991;7:91-100.
30. Carbon M, Eidelberg D: Modulation of regional brain function by deep brain stimulation: studies with positron emission tomography. Curr Opin Neurol 2002;15:451-455.
31. Fukuda MC, Edwards C, Eidelberg D: Functional brain networks in Parkinson's disease. Parkinsonism Relat Disord 2001;8:91-94.
32. Obeso JA, Rodriguez-Oroz MC, Rodriguez M, Macias R, Alvarez L, Guridi J, Vitek J, DeLong MR: Pathophysiologic basis of surgery for Parkinson's disease. Neurology 2000;55(suppl 6):S7-S12.
33. Kaplitt MG, Feigin A, Chengke T, Fitzsimons HL, Mattis P, Lawlor PA, Bland RJ, Young D, Strybing K, Eidelberg D, During MJ: 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;369:2097-2105.
34. Feigin A, Kaplitt MG, Tang C, Lin T, Mattis P, Dhawan V, During MJ, Eidelberg D: Modulation of metabolic brain networks after subthalamic gene therapy for Parkinson's disease. Proc Natl Acad Sci USA 2007;104:19559-19564.
35. Fjord-Larsen L, Johansen JL, Kusk P, Tornøe J, Grønborg M, Rosenblad C, Wahlberg LU: Efficient in vivo protection of nigral dopaminergic neurons by lentiviral gene transfer of a modified Neurturin construct. Exp Neurol 2005;195:49-60.
36. Gasmi M, Brandon EP, Herzog CD, Wilson A, Bishop KM, Hofer EK, Cunningham JJ, Printz MA, Kordower JH, Bartus RT: AAV2-mediated delivery of human neurturin to the rat nigrostriatal system: long-term efficacy and tolerability of CERE-120 for Parkinson's disease. Neurobiol Dis 2007;27:67-76.
37. Kordower JH, Herzog CD, Dass B, Bakay RAE, Stansell J III, Gasmi M, Bartus RT: Delivery of neurturin by AAV2 (CERE-120)-mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTP-treated monkeys. Ann Neurol 2006;60:706-715.
38. Sánchez-Pernaute R, Harvey-White J, Cunningham J, Bankiewicz KS: Functional effect of adeno-associated virus mediated gene transfer of aromatic L-amino acid decarboxylase into the striatum of 6-OHDA-lesioned rats. Mol Ther 2001;4:324-330.
39. Bankiewicz KS, Forsayeth J, Eberling JL, Sanchez-Pernaute R, Pivirotto P, Bringas J, Herscovitch P, Carson RE, Eckelman W, Reutter B, Cunningham J: Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC. Mol Ther 2006;14:564-570.
40. Forsayeth JR, Eberling JL, Sanftner LM, Zhen Z, Pivirotto P, Bringas J, Cunningham J, Bankiewicz KS: A dose-ranging study of AAV-hAADC therapy in Parkinsonian monkeys. Mol Ther 2006;14:571-577.
41. Sanftner LM, Sommer JM, Suzuki BM, Smith PH, Vijay S, Vargas JA, Forsayeth JR, Cunningham J, Bankiewicz KS, Kao H, Bernal J, Pierce GF, Johnson KW: AAV2-mediated gene delivery to monkey putamen: evaluation of an infusion device and delivery parameters. Exp Neurol 2005;194:476-483.
42. Bankiewicz KS, Eberling JL, Kohutnicka M, Jagust W, Pivirotto P, Bringas J, Cunningham J, Budinger TF, Harvey-White J: Convection-enhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach. Exp Neurol 2000;164:2-14.
43. Shah S, Reichman WE: Treatment of Alzheimer's disease across the spectrum of severity. Clin Interv Aging 2006;1:131-142.
44. Herrmann N, Lanctot KL: Pharmacologic management of neuropsychiatric symptoms of Alzheimer disease. Can J Psychiatry 2007;52:630-646.
45. Tuszynski MH, Roberts J, Senut MC, U HS, Gage FH: Gene therapy in the adult primate brain: intraparenchymal grafts of cells genetically modified to produce nerve growth factor prevent cholinergic neuronal degeneration. Gene Ther 1996;3:305-314.
46. Tuszynski MH, Thal L, Pay M, Salmon D P, U HS, Bakay R, Patel P, Blesch A, Vahlsing HL, Ho G, Tong G, Potkin SG, Fallon J, Hansen L, Mufson EJ, Kordower JH, Gall C, Conner J: A phase 1 clinical trial of nerve growth factor gene therapy for Alzheimer disease. Nat Med 2005;11:551-555.
47. Tuszynski MH: Nerve growth factor gene delivery: animal models to clinical trials. Dev Neurobiol 2007;67:1204-1215.