Screening of drugs to treat 8p11 myeloproliferative syndrome using patient-derived induced pluripotent stem cells with fusion gene CEP110-FGFR1

PLoS ONE

Volumn 10, Issue 3, 2015, Pages

  Yamamoto, Shohei ^a   Otsu, Makoto ^a   Matsuzaka, Emiko ^a   Konishi, Chieko ^a   Takagi, Haruna ^a   Hanada, Sachiyo ^a   Mochizuki, Shinji ^a   Nakauchi, Hiromitsu ^a   Imai, Kohzoh ^b   Tsuji, Kohichiro ^a,c   Ebihara, Yasuhiro ^a  

^a UNIVERSITY OF TOKYO   (Japan)

^b THE UNIVERSITY OF TOKYO   (Japan)

^c Shinshu Ueda Medical Center   (Japan)

Author keywords

[No Author keywords available]

Indexed keywords

CD34 ANTIGEN; CYTARABINE; DOVITINIB; IDARUBICIN; IMATINIB; MIDOSTAURIN; PONATINIB; 4-AMINO-5-FLUORO-3-(5-(4-METHYLPIPERAZIN-1-YL)-1H-BENZIMIDAZOL-2-YL)QUINOLIN-2(1H)-ONE; BENZIMIDAZOLE DERIVATIVE; CEP110-FGFR1 FUSION PROTEIN, HUMAN; FIBROBLAST GROWTH FACTOR RECEPTOR 1; IMIDAZOLE DERIVATIVE; ONCOPROTEIN; PROTEIN KINASE INHIBITOR; PYRIDAZINE DERIVATIVE; QUINOLONE DERIVATIVE; STAUROSPORINE;

8P11 MYELOPROLIFERATIVE SYNDROME; ACUTE GRANULOCYTIC LEUKEMIA; ADOLESCENT; ALLOGENIC BONE MARROW TRANSPLANTATION; ANIMAL CELL; ARTICLE; CASE REPORT; CELL DIFFERENTIATION; CEP110 FGFR1 GENE; COCULTURE; CONCENTRATION RESPONSE; CONTROLLED STUDY; DRUG EFFECT; DRUG SCREENING; EMBRYO; EMBRYONIC STEM CELL; ERYTHROID CELL; FUSION GENE; HEMATOPOIETIC CELL; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; HUMAN TISSUE; MACROPHAGE; MALE; MOUSE; MYELOPROLIFERATIVE DISORDER; NONHUMAN; PLURIPOTENT STEM CELL; ANALOGS AND DERIVATIVES; CELL CULTURE; DRUG EFFECTS; GENE TRANSLOCATION; GENETICS; HEMATOPOIESIS; INDUCED PLURIPOTENT STEM CELL; METABOLISM; MYELOPROLIFERATIVE DISORDERS; PATHOLOGY; PRECLINICAL STUDY;

IPS; MURINAE;

ADOLESCENT; BENZIMIDAZOLES; CELLS, CULTURED; DRUG EVALUATION, PRECLINICAL; HEMATOPOIESIS; HUMANS; IMATINIB MESYLATE; IMIDAZOLES; INDUCED PLURIPOTENT STEM CELLS; MALE; MYELOPROLIFERATIVE DISORDERS; ONCOGENE PROTEINS, FUSION; PROTEIN KINASE INHIBITORS; PYRIDAZINES; QUINOLONES; RECEPTOR, FIBROBLAST GROWTH FACTOR, TYPE 1; STAUROSPORINE; TRANSLOCATION, GENETIC;

EID: 84925688219     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0120841     Document Type: Article

References (33)

1. Macdonald D, Aguiar RC, Mason PJ, Goldman JM, Cross NC. A new myeloproliferative disorder associated with chromosomal translocations involving 8p11: a review. Leukemia. 1995; 9(10):1628-30. Epub 1995/10/01. PubMed PMID: 7564500.
2. Macdonald D, Reiter A, Cross NC. The 8p11 myeloproliferative syndrome: a distinct clinical entity caused by constitutive activation of FGFR1. Acta haematologica. 2002; 107(2):101-7. Epub 2002/03/29. doi: 46639. PubMed PMID: 11919391.
3. Jackson CC, Medeiros LJ, Miranda RN. 8p11 myeloproliferative syndrome: a review. Human pathology. 2010; 41(4):461-76. Epub 2010/03/17. doi: 10.1016/j.humpath.2009.11.003. PubMed PMID: 20226962.
4. Hu S, He Y, Zhu X, Li J, He H. Myeloproliferative disorders with t(8;9)(p12;q33): a case report and review of the literature. Pediatric hematology and oncology. 2011; 28(2):140-6. Epub 2011/01/11. doi: 10.3109/08880018.2010.528170. PubMed PMID: 21214407.
5. Guasch G, Mack GJ, Popovici C, Dastugue N, Birnbaum D, Rattner JB, et al. FGFR1 is fused to the centrosome-associated protein CEP110 in the 8p12 stem cell myeloproliferative disorder with t(8;9) (p12;q33). Blood. 2000; 95(5):1788-96. Epub 2000/02/26. PubMed PMID: 10688839.
6. Ramos-Mejia V, Montes R, Bueno C, Ayllon V, Real PJ, Rodriguez R, et al. Residual expression of the reprogramming factors prevents differentiation of iPSC generated from human fibroblasts and cord blood CD34+ progenitors. PloS one. 2012; 7(4):e35824. doi: 10.1371/journal.pone.0035824. PubMed PMID: 22545141; PubMed Central PMCID: PMC3335819.
7. Okabe M, Otsu M, Ahn DH, Kobayashi T, Morita Y, Wakiyama Y, et al. Definitive proof for direct reprogramming of hematopoietic cells to pluripotency. Blood. 2009; 114(9):1764-7. doi: 10.1182/blood-2009-02-203695. PubMed PMID: 19564635.
8. Loh YH, Agarwal S, Park IH, Urbach A, Huo H, Heffner GC, et al. Generation of induced pluripotent stem cells from human blood. Blood. 2009; 113(22):5476-9. doi: 10.1182/blood-2009-02-204800. PubMed PMID: 19299331; PubMed Central PMCID: PMC2689048.
9. Yamamoto S, Ebihara Y, Mochizuki S, Kawakita T, Kato S, Ooi J, et al. Quantitative polymerase chain reaction detection of CEP110-FGFR1 fusion gene in a patient with 8p11 myeloproliferative syndrome. Leukemia & lymphoma. 2013; 54(9):2068-9. Epub 2013/01/19. doi: 10.3109/10428194.2013.767455. PubMed PMID: 23327291.
10. Chase A, Grand FH, Cross NC. Activity of TKI258 against primary cells and cell lines with FGFR1 fusion genes associated with the 8p11 myeloproliferative syndrome. Blood. 2007; 110(10):3729-34. Epub 2007/08/19. doi: 10.1182/blood-2007-02-074286. PubMed PMID: 17698633.
11. Chen J, Deangelo DJ, Kutok JL, Williams IR, Lee BH, Wadleigh M, et al. PKC412 inhibits the zinc finger 198-fibroblast growth factor receptor 1 fusion tyrosine kinase and is active in treatment of stem cell myeloproliferative disorder. Proceedings of the National Academy of Sciences of the United States of America. 2004; 101(40):14479-84. Epub 2004/09/28. doi: 10.1073/pnas.0404438101. PubMed PMID: 15448205; PubMed Central PMCID: PMC521956.
12. Chase A, Bryant C, Score J, Cross NC. Ponatinib as targeted therapy for FGFR1 fusions associated with the 8p11 myeloproliferative syndrome. Haematologica. 2013; 98(1):103-6. Epub 2012/08/10. doi: 10.3324/haematol.2012.066407. PubMed PMID: 22875613; PubMed Central PMCID: PMC3533670.
13. Wasag B, Lierman E, Meeus P, Cools J, Vandenberghe P. The kinase inhibitor TKI258 is active against the novel CUX1-FGFR1 fusion detected in a patient with T-lymphoblastic leukemia/lymphoma and t (7;8)(q22;p11). Haematologica. 2011; 96(6):922-6. Epub 2011/02/19. doi: 10.3324/haematol.2010.036558. PubMed PMID: 21330321; PubMed Central PMCID: PMC3105656.
14. Ren M, Qin H, Ren R, Cowell JK. Ponatinib suppresses the development of myeloid and lymphoid malignancies associated with FGFR1 abnormalities. Leukemia. 2013; 27(1):32-40. Epub 2012/07/12. doi: 10.1038/leu.2012.188. PubMed PMID: 22781593; PubMed Central PMCID: PMC3629706.
15. Ory DS, Neugeboren BA, Mulligan RC. A stable human-derived packaging cell line for production of high titer retrovirus/vesicular stomatitis virus G pseudotypes. Proceedings of the National Academy of Sciences of the United States of America. 1996; 93(21):11400-6. Epub 1996/10/15. PubMed PMID: 8876147; PubMed Central PMCID: PMC38069.
16. Takahashi K, Tanabe K, Ohnuki M, Narita M, Ichisaka T, Tomoda K, et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell. 2007; 131(5):861-72. Epub 2007/11/24. doi: 10.1016/j.cell.2007.11.019. PubMed PMID: 18035408.
17. Ma F, Wang D, Hanada S, Ebihara Y, Kawasaki H, Zaike Y, et al. Novel method for efficient production of multipotential hematopoietic progenitors from human embryonic stem cells. Int J Hematol. 2007; 85(5):371-9. PubMed PMID: 17562610.
18. Takayama N, Nishimura S, Nakamura S, Shimizu T, Ohnishi R, Endo H, et al. Transient activation of c-MYC expression is critical for efficient platelet generation from human induced pluripotent stem cells. The Journal of experimental medicine. 2010; 207(13):2817-30. doi: 10.1084/jem.20100844. PubMed PMID: 21098095; PubMed Central PMCID: PMC3005234.
19. Ma F, Ebihara Y, Umeda K, Sakai H, Hanada S, Zhang H, et al. Generation of functional erythrocytes from human embryonic stem cell-derived definitive hematopoiesis. Proceedings of the National Academy of Sciences of the United States of America. 2008; 105(35):13087-92. PubMed PMID: 18755895. doi: 10.1073/pnas.0802220105
20. Gutierrez-Aranda I, Ramos-Mejia V, Bueno C, Munoz-Lopez M, Real PJ, Macia A, et al. Human induced pluripotent stem cells develop teratoma more efficiently and faster than human embryonic stem cells regardless the site of injection. Stem cells (Dayton, Ohio). 2010; 28(9):1568-70. doi: 10.1002/stem.471. PubMed PMID: 20641038; PubMed Central PMCID: PMC2996086.
21. Hiramoto T, Ebihara Y, Mizoguchi Y, Nakamura K, Yamaguchi K, Ueno K, et al. Wnt3a stimulates maturation of impaired neutrophils developed from severe congenital neutropenia patient-derived pluripotent stem cells. Proceedings of the National Academy of Sciences of the United States of America. 2013; 110(8):3023-8. Epub 2013/02/06. doi: 10.1073/pnas.1217039110. PubMed PMID: 23382209; PubMed Central PMCID: PMC3581925.
22. Xu MJ, Tsuji K, Ueda T, Mukouyama YS, Hara T, Yang FC, et al. Stimulation of mouse and human primitive hematopoiesis by murine embryonic aorta-gonad-mesonephros-derived stromal cell lines. Blood. 1998; 92(6):2032-40. Epub 1998/09/10. PubMed PMID: 9731061.
23. Ma F, Gu Y, Nishihama N, Yang W, Ebihara Y, Tsuji K. Differentiation of human embryonic and induced pluripotent stem cells in coculture with murine stromal cells. In: Marton P, Ye K, Jin S, editors. Human Embryonic and Induced Pluripotent Stem Cells Lineage-Specific Differentiation Protocols. New York: Humana Press; 2011. p. 321-35.
24. Nakahata T, Ogawa M. Hemopoietic colony-forming cells in umbilical cord blood with extensive capability to generate mono- and multipotential hemopoietic progenitors. The Journal of clinical investigation. 1982; 70(6):1324-8. Epub 1982/12/01. PubMed PMID: 7174797; PubMed Central PMCID: PMC370352.
25. Kumaki Y, Oda M, Okano M. QUMA: quantification tool for methylation analysis. Nucleic acids research. 2008; 36(Web Server issue):W170-5. doi: 10.1093/nar/gkn294. PubMed PMID: 18487274; PubMed Central PMCID: PMC2447804.
26. Ramos-Mejia V, Fraga Mf Fau-Menendez P, Menendez P. iPSCs from cancer cells: challenges and opportunities. Trend Mol Med. 18(5):245-7. doi: 10.1016/j.molmed.2012.04.001 PMID: 22521522
27. Ye Z, Zhan H, Mali P, Dowey S, Williams DM, Jang YY, et al. Human-induced pluripotent stem cells from blood cells of healthy donors and patients with acquired blood disorders. Blood. 2009; 114(27):5473-80. Epub 2009/10/03. doi: 10.1182/blood-2009-04-217406. PubMed PMID: 19797525; PubMed Central PMCID: PMC2798863.
28. Hu K, Yu J, Suknuntha K, Tian S, Montgomery K, Choi KD, et al. Efficient generation of transgene-free induced pluripotent stem cells from normal and neoplastic bone marrow and cord blood mononuclear cells. Blood. 2011; 117(14):e109-19. Epub 2011/02/08. doi: 10.1182/blood-2010-07-298331. PubMed PMID: 21296996; PubMed Central PMCID: PMC3083304.
29. Carette JE, Pruszak J, Varadarajan M, Blomen VA, Gokhale S, Camargo FD, et al. Generation of iPSCs from cultured human malignant cells. Blood. 2010; 115(20):4039-42. Epub 2010/03/18. doi: 10.1182/blood-2009-07-231845. PubMed PMID: 20233975; PubMed Central PMCID: PMC2875096.
30. Kumano K, Arai S, Hosoi M, Taoka K, Takayama N, Otsu M, et al. Generation of induced pluripotent stem cells from primary chronic myelogenous leukemia patient samples. Blood. 2012; 119(26):6234-42. Epub 2012/05/18. doi: 10.1182/blood-2011-07-367441. PubMed PMID: 22592606.
31. Bedel A, Pasquet JM, Lippert E, Taillepierre M, Lagarde V, Dabernat S, et al. Variable Behavior of iPSCs Derived from CML Patients for Response to TKI and Hematopoietic Differentiation. PloS one. 2013; 8(8):e71596. Epub 2013/09/24. doi: 10.1371/journal.pone.0071596. PubMed PMID: 24058405; PubMed Central PMCID: PMC3751925.
32. Agerstam H, Jaras M, Andersson A, Johnels P, Hansen N, Lassen C, et al. Modeling the human 8p11-myeloproliferative syndrome in immunodeficient mice. Blood. 2010; 116(12):2103-11. Epub 2010/06/18. doi: 10.1182/blood-2009-05-217182. PubMed PMID: 20554971.
33. Ren M, Qin H, Kitamura E, Cowell JK. Dysregulated signaling pathways in the development of CNTRL-FGFR1-induced myeloid and lymphoid malignancies associated with FGFR1 in human and mouse models. Blood. 2013; 122(6):1007-16. Epub 2013/06/20. doi: 10.1182/blood-2013-03-489823. PubMed PMID: 23777766; PubMed Central PMCID: PMC3739028.