Engraftment of gene-marked hematopoietic progenitors in myeloma patients after transplant of autologous long-term marrow cultures

Human Gene Therapy

Volumn 10, Issue 12, 1999, Pages 1953-1964

  Stewart, A Keith ^a,b,f   Sutherland, D Robert ^a,b   Nanji, Shaherose ^c   Zhao, Yongjun ^c   Lutzko, Carolyn ^c   Nayar, Rakash ^a   Peck, Brian ^a   Ruedy, Christine ^b   McGarrity, Gary ^d   Tisdale, John ^e   Dubé, Lan D ^b,c  

^a UNIVERSITY HEALTH NETWORK   (Canada)

^b UNIVERSITY OF TORONTO   (Canada)

^c SUNNYBROOK RESEARCH INSTITUTE   (Canada)

^d NOVARTIS PHARMA AG   (Switzerland)

^e NATIONAL INSTITUTES OF HEALTH   (United States)

^f PRINCESS MARGARET HOSPITAL   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

MYELOABLATIVE AGENT; VIRUS DNA;

ARTICLE; AUTOLOGOUS BONE MARROW TRANSPLANTATION; CLINICAL ARTICLE; CLINICAL TRIAL; COLONY FORMING UNIT; DNA DETERMINATION; EPSTEIN BARR VIRUS; GENE TRANSFER; GENETIC TRANSDUCTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HUMAN CELL; LYMPHOCYTE TRANSFORMATION; MARKER GENE; MULTIPLE MYELOMA; PHASE 1 CLINICAL TRIAL; POLYMERASE CHAIN REACTION; SOUTHERN BLOTTING;

BONE MARROW CELLS; BONE MARROW TRANSPLANTATION; CELLS, CULTURED; DNA, VIRAL; GENE TRANSFER TECHNIQUES; GENETIC MARKERS; GENETIC VECTORS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HUMANS; KANAMYCIN KINASE; MULTIPLE MYELOMA; POLYMERASE CHAIN REACTION; PROVIRUSES; RETROVIRIDAE; TRANSPLANTATION, AUTOLOGOUS;

HUMAN HERPESVIRUS 4; UNIDENTIFIED RETROVIRUS;

EID: 0033543096     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/10430349950017310     Document Type: Article

References (51)

1. ABKOWITZ, J.L., LINENBERGER, M.L., NEWTON, M.A., SHELTON, G.H., OTT, R.L., and GUTTORP, P. (1990). Evidence for the maintenance of hematopoiesis in a large animal by the sequential activation of stem-cell clones. Proc. Natl. Acad. Sci. U.S.A. 87, 9062-9066.
2. ALLAN, D.S., DE KOVEN, A., WILD, A., KAMEL-REID, S., and DUBE, I.D. (1996). Endogenous murine leukemia virus DNA sequences in murine cell lines: Implications for gene therapy safety testing by PCR. Leukemia Lymph. 23, 375-381.
3. BENDER, M.A., GELINAS, R.E., and MILLER, A.D. (1989). A majority of mice show long-term expression of human beta-globin gene after retrovirus transfer into hematopoietic stem cells. Mol. Cell. Biol. 9, 1426-1434.
4. BIENZLE, D., ABRAMS-OGG, A.C., KRUTH, S.A., ACKLANDSNOW, J., CARTER, R.F., DICK, I.E., JACOBS, R.M., KAMELREID, S., and DUBÉ, I.D. (1994). Gene transfer into hematopoietic stem cells: Long-term maintenance of in vitro activated progenitors without marrow ablation. Proc. Natl. Acad. Sci. U.S.A. 91, 350-354.
5. BODINE, D.M., MORITZ, T., DONAHUE, R.E., LUSKEY, B.D., KESSLER, S.W., MARTIN, D.I., ORKIN, S.H., NIENHUIS, A.W., and WILLIAMS, D.A. (1993). Long-term in vivo expression of a murine adenosine deaminase gene in rhesus monkey hematopoietic cells of multiple lineages after retroviral mediated gene transfer into CD34+ bone marrow cells. Blood 82, 1975-1980.
6. Bowtell, D.D., Johnson, G.R., Kelso, A., and Cory, S. (1987). Expression of genes transferred to haemopoietic stem cells by recombinant retroviruses. Biol. Med. 4, 229-250.
7. BRENNER, M.K., RILL, D.R., HOLLADAY, M.S., HESLOP, H.E., MOEN, R.C., BUSCHLE, M., KRANCE, R.A., SANTANA, V.M., ANDERSON, W.F., and IHLE, J.N. (1993). Gene marking to determine whether autologous marrow infusion restores long-term haemopoiesis in cancer patients. Lancet 342, 1134-1137.
8. CAPEL, B., HAWLEY, R., COVARRUBIAS, L., HAWLEY, T., and MINTZ, B. (1989). Clonal contributions of small numbers of retrovirally marked hematopoietic stem cells engrafted in unirradiated neonatal W/Wv mice. Proc. Natl. Acad. Sci. U.S.A. 86, 4564-4568.
9. CARTER, R.F., ABRAMS-OGG, A.C., DICK, J.E., KRUTH, S.A., VALLI, V.E., KAMEL-REID, S., and DUBÉ, I.D. (1992). Autologous transplantation of canine long-term marrow culture cells genetically marked by retroviral markers. Blood 79, 356-364.
10. CASHMAN, J., EAVES, A.C., and EAVES, C.J. (1985). Regulated proliferation of primitive hematopoietic progenitor cells in long-term human marrow cultures. Blood 66, 1002-1005.
11. CASHMAN, J.D., EAVES, A.C., RAINES, E.W., ROSS, R., and EAVES, C.J. (1990). Mechanisms that regulate the cell cycle status of very primitive hematopoietic cells in long-term human marrow cultures. I. Stimulatory role of a variety of mesenchymal cell activators and inhibitory role of TGF-beta. Blood 75, 96-101.
12. CASHMAN, J.D., LAPIDOT, T., WANG, J.C., DOEDENS, M., SHULTZ, L.D., LANSDORP, P., DICK, J.E., and EAVES, C.J. (1997). Kinetic evidence of the regeneration of multilineage hematopoiesis from primitive cells in normal human bone marrow transplanted into immunodeficient mice. Blood 89, 4307-4316.
13. DAO, M.A., HASHING, K., KATO, I., and NOLTA, J.A. (1998). Adhesion to fibronectin maintains regenerative capacity during ex vivo culture and transduction of human hematopoietic stem and progenitor cells. Blood 92, 4612-4621.
14. DEISSEROTH, A.B., ZU, Z., CLAXTON, D., HANANIA, E.G., FU, S., ELLERSON, D., GOLDBERG, L., THOMAS, M., JANICEK, K., ANDERSON, W.F. (1994). Genetic marking shows that Ph+ cells present in autologous transplants of chronic myelogenous leukemia (CML) contribute to relapse after autologous bone marrow in CML. Blood 83, 3068-3076.
15. DEXTER, T.M., ALLEN, T.D., and LAJTHA, L.G. (1977). Conditions controlling the proliferation of haemopoietic stem cells in vitro. J. Cell. Physiol. 91, 335-344.
16. DUBÉ, I.D., KRUTH, S., ABRAMS-OGG, A., KAMEL-REID, S., LUTZKO, C., NANJI, S., RUEDY, C., SINGARAJA, R., WILD, A., KRYGSMAN, P., CHU, P., MESSNER, H., REDDY, V., MCGARRITY, G., and STEWART, A.K. (1996). Preclinical assessment of human hematopoietic progenitor cell transduction in long-term marrow cultures. Hum Gene Ther. 7, 2089-2100.
17. DUNBAR, C.E., COTTLER-FOX, M., O'SHAUGHNESSY, J.A., DOREN, S., CARTER, C., BERENSON, R., BROWN, S., MOEN, R.C., GREENBLATT, J., STEWART, P.M., LEITMAN, S.F., WILSON, W.H., COWAN, K., YOUNG, N.S., and NIENHUIS, A.W. (1995). Retrovirally marked CD34-enriched peripheral blood and bone marrow cells contribute to long-term engraftment after autologous transplantation. Blood 85, 3048-3057.
18. EAVES, C.J., CASHMAN, J.D., KAY, R.J., DOUGHERTY, G.J., OTSUKA, T., GABOURY, L.A., HOGGE, D.E., LANSDORP, P.M., EAVES, A.C., and HUMPHRIES, R.K. (1991). Mechanisms that regulate the cell cycle status of very primitive hematopoietic cells in long-term human marrow cultures. II. Analysis of positive and negative regulators produced by stromal cells within the adherent layer. Blood 78, 110-117.
19. GAN, O.I., MURDOCH, B., LAROCHELLE, A., and DICK, J.E. (1997). Differential maintenance of primitive human SCID-repopulating cells, clonogenic progenitors, and long-term culture-initiating cells after incubation on human bone marrow stromal cells. Blood 90, 641-650.
20. GERARD, C.J., ARBOLEDA, M.J., SOLAR, G., MULE, J.J., and KERR, W.G. (1996). A rapid and quantitative assay to estimate gene transfer into retrovirally transduced hematopoietic stem/progenitor cells using a 96-well format PCR and fluorescent detection system universal for MMLV-based proviruses. Hum. Gene Ther. 7, 343-354.
21. JORDAN, C.T., and LEMISCHKA, I.R. (1990). Clonal and systemic analysis of long-term hematopoiesis in the mouse. Genes Dev. 4, 220-232.
22. KALEKO, M., GARCIA, J.V., OSBORNE, R.A., and MILLER, A.D. (1990). Expression of human adenosine deaminase in mice after transplantation of genetically-modified bone marrow. Blood 75, 1733-1741.
23. KANTOFF, P.W., GILLIO, A.P., McLACHLIN, J.R., BORDIGNON, C., EGLITIS, M.A., KERNAN, N.A., MOEN, R.C., KOHN, D.B., YU, S.F., KARSON, E. (1987). Expression of human adenosine deaminase in nonhuman primates after retrovirus-mediated gene transfer. J. Exp. Med. 166, 219-234.
24. KIEM, H.P., HEYWARD, S., WINKLER, A., POTTER, J., ALLEN, J.M., MILLER, A.D., and ANDREWS, R.G. (1997). Gene transfer into marrow repopulating cells: Comparison between amphotropic and gibbon ape leukemia virus pseudotyped retroviral vectors in a competitive repopulation assay in baboons. Blood 90, 4638-4645.
25. KIEM, H.P., ANDREWS, R.G., MORRIS, J., PETERSON, L., HEYWARD, S., ALLEN, J.M., RASKO, J.E.J., POTTER, J., and MILLER, A.D. (1998). Improved gene transfer into baboon marrow repopulating cells using recombinant human fibronectin fragment CH-296 in combination with interleukin-6, stem cell factor, FLT-3 ligand, and megakaryocyte growth and development factor. Blood 92, 1878-1886.
26. KOHN, D.B., HERSHFIELD, M.S., CARBONARO, D., SHIGEOKA, A., BROOKS, J., SMOGORZEWSKA, E.M., BARSKY, L.W., CHAN, R., BUROTTO, F., ANNETT, G., NOLTA, J.A., CROOKS, G., KAPOOR, N., ELDER, M., WARA, D., BOWEN, T., MADSEN, E., SNYDER, F.F., BASTIAN, J., MUUL, L., BLAESE, R.M., WEINBERG, K., and PARKMAN, R. (1998). T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates. Nature Med. 4, 775-780.
27. LEMISCHKA, I.R., RAULET, D.H., and MULLIGAN, R.C. (1986). Developmental potential and dynamic behavior of hematopoietic stem cells. Cell 45, 917-927.
28. LI, C.L., DWARKI, V.J., and VERMA, I.M. (1990). Expression of human alpha-globin and mouse/human hybrid beta-globin genes in murine hemopoietic stem cells transduced by recombinant retroviruses. Proc. Natl. Acad. Sci. U.S.A. 87, 4349-4353.
29. LIM, B., APPERLEY, J.F., ORKIN, S.H., and WILLIAMS, D.A. (1989). Long-term expression of human adenosine deaminase in mice transplanted with retrovirus-infected hematopoietic stem cells. Proc. Natl. Acad. Sci. U.S.A. 86, 8892-8896.
30. LOTHROP, C.D., AL-LEBBAN, Z.S., NIEMEYER, G.P., JONES, J.B., PETERSON, M.F., SMITH, J.R., BAKER, H.J., MORGAN, R.A., EGLITIS, M.A., and ANDERSON, W.F. (1991). Expression of a foreign gene in cats reconstituted with retroviral vector infected autologous bone marrow. Blood 78, 237-245.
31. LUTZKO, C., KRUTH, S., ABRAMS-OGG, A.C.G., RUEDY, C., NANJI, S., LIHENG, L., LAU, K., CLARK, B.R., FOSTER, R., KOHN, D., SHULL, R.M., and DUBE, I.D. (1999). Genetically corrected autologous stem cells engraft, but host immune responses and vector silencing limit their utility in canine α-L-iduronidase deficiency. Blood 93, 1895-1905.
32. MARSH, J.C.W., SUTHERLAND, D.R., DAVIDSON, J., MELLORS, A., and KEATING, A. (1992). Retention of progenitor cell function in CD34+ cells purified using a novel O-sialoglycoprotease. Leukemia 6, 926-934.
33. MILLER, D.G., ADAM, M.A., and MILLER, A.D. (1990). Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Mol. Cell. Biol. 10, 4239-4242.
34. MILLER, D.G., EDWARDS, R.H., and MILLER, A.D. (1994). Cloning of the cellular receptor for amphotropic murine retroviruses reveals homology to that for gibbon ape leukemia virus. Proc. Natl. Acad. Sci. U.S.A. 91, 78-82.
35. MOORE, K.A., DEISSEROTH, A.B., READING, C.L., WILLIAMS, D.E., and BELMONT, J.W. (1992). Stromal support enhances cell-free retroviral vector transduction of human bone marrow long-term culture initiating cells. Blood 79, 1393-1399.
36. MORITZ, T., PATEL, V.P., and WILLIAMS, D.A. (1994). Bone marrow extracellular matrix molecules improve gene transfer into human hematopoietic cells via retroviral vectors. J. Clin. Invest. 93, 1451-1457.
37. NOLTA, J.A., SMOGORZEWSKA, E.M., and KOHN, D.B. (1995). Analysis of optimal conditions for retroviral-mediated transduction of primitive human hematopoietic cells. Blood 86, 101-110.
38. ORLIC, D., GIRARD, L.J., JORDAN, C.T., ANDERSON, S.M., CLINE, A.P., and BODINE, D.M. (1996). The level of mRNA encoding the amphotropic retrovirus receptor in mouse and human hematopoietic stem cells is low and correlates with the efficiency of retrovirus transduction. Proc. Natl. Acad. Sci. U.S.A. 93, 11097-11102.
39. ORLIC, D., GIRARD, L.J., ANDERSON, S.M., PYLE, L.C., YODER, M.C., BROXMEYER, H.E., and BODINE, D.M. (1998). Identification of human and mouse hematopoietic stem cell populations expressing high levels of mRNA encoding retrovirus receptors. Blood 91, 3247-3254.
40. ROBBINS, P.B., SKELTON, D.C., YU, X.J., HALENE, S., LEONARD, E.H., and KOHN, D.B. (1998). Consistent, persistent expression from modified retroviral vectors in murine hematopoietic stem cells. Proc. Natl. Acad. Sci. U.S.A. 95, 10182-10187.
41. SCHUENING, F.G., STORE, R., STEAD, R.B., GOEHLE, S., NASH, R., and MILLER, A.D. (1989). Improved retroviral transfer of genes into canine hematopoietic progenitor cells kept in long-term marrow culture. Blood 74, 152-155.
42. SCHUENING, F.G., KAWAHARA, K., MILLER, A.D., TO, R., GOEHLE, S., STEWART, D., MULLALLY, K., FISHER, L., GRAHAM, T.C., APPELBAUM, F.R. (1991). Retrovirus-mediated gene transduction into long-term repopulating marrow cells of dogs. Blood 78, 2568-2576.
43. SIMONARO, C.M., HASKINS, M.E., ABKOWITZ, J.L., BROOKS, A.D., HOPWOOD, J.J., ZHANG, J., and SCHUCHMAN, E.H. (1999). Autologous transplantation of retrovirally transduced bone marrow or neonatal blood cells into cats can lead to long-term engraftment in the absence of myeloablation. Gene Ther. 6, 107-113.
44. STEAD, R.B., KWOK, W.W., STORE, R., and MILLER, A.D. (1988). Canine model for gene therapy: Inefficient gene expression in dogs reconstituted with autologous marrow infected with retroviral vectors. Blood 71, 742-747.
45. STEWART, A.K., DUBÉ, I.D., KAMEL-REID, S., and KEATING, A. (1995a). A phase I study of autologous bone marrow transplantation with stem cell gene marking in multiple myeloma. Hum. Gene Ther. 6, 107-119.
46. STEWART, A.K., IMRIE, K., KEATING, A., ANANIA, S., NAYAR, R., and SUTHERLAND, D.R. (1995b). Optimizing the CD34+ and CD34+Thy-1+ stem cell content of peripheral blood collections. Exp. Hematol. 23, 1619-1627.
47. STEWART, A.K., PRINCE, H.M., CHU, P., SUTHERLAND, D.R., and DUBE, I.D. (1996). In vitro maintenance and transduction of human myeloma cells in long term marrow cultures. Cancer Gene Ther. 4, 148-156.
48. SUTHERLAND, D.R., KEATING, A., NAYER, R., ANANIA, S., and STEWART, A.K. (1994). Sensitive detection and enumeration of CD34+ cells in peripheral and cord blood by flow cytometry. Exp. Hematol. 22, 1003-1010.
49. TISDALE, J.F., HANAZONO, Y., SELLERS, S.E., AGRICOLA, B.A., METZGER, M.E., DONAHUE, R.E., and DUNBAR, D.E. (1998). Ex vivo expansion of genetically marked rhesus peripheral blood progenitor cells results in diminished long-term repopulating ability.
50. TOKSOZ, D., DEXTER, T.M., LORD, B.I., WRIGHT, E.G., and LAJTHA, L.G. (1980). The regulation of hemopoiesis in long term-bone marrow cultures. II. Stimulation and inhibition of stem cell proliferation. Blood 55, 931-936.
51. VAN BEUSECHEM, V.W., KUKLER, A., HEIDT, P.J., and VALERIO, D. (1992). Long-term expression of human adenosine deaminase in rhesus monkeys transplanted with retrovirus-infected bone marrow-cells. Proc. Natl. Acad. Sci. U.S.A. 89, 7640-7644.