Title:
CXCL12-RECEPTOR-EXPRESSING CHIMERIC ANTIGEN RECEPTOR (CAR)-T CELL
Document Type and Number:
WIPO Patent Application WO/2022/092277
Kind Code:
A1
Abstract:
As a treatment for curing hematological neoplastic diseases with poor prognosis, the present invention provides: a cell that co-expresses, on the cell membrane, a chimeric antigen receptor (CAR) protein and a CXCL12 receptor protein; and a drug and a pharmaceutical composition having antitumor activity containing said cell.
Inventors:
ISHIKAWA FUMIHIKO (JP)
SAITO YORIKO (JP)
ITOH ARI (JP)
SHULTZ LEONARD D (US)
SAITO YORIKO (JP)
ITOH ARI (JP)
SHULTZ LEONARD D (US)
Application Number:
PCT/JP2021/040069
Publication Date:
May 05, 2022
Filing Date:
October 29, 2021
Export Citation:
Assignee:
RIKEN (JP)
International Classes:
C12N5/10; A61K35/17; A61P35/02; C12N15/62; C12N15/90
Foreign References:
| US202762631070P | ||||
| JP2019529423A | 2019-10-17 |
Other References:
JAMALI AREZOO, HADJATI JAMSHID, MADJD ZAHRA, MIRZAEI HAMID REZA, THALHEIMER FREDERIC B., AGARWAL SHIWANI, BONIG HALVARD, ULLRICH E: "Highly Efficient Generation of Transgenically Augmented CAR NK Cells Overexpressing CXCR4", FRONTIERS IN IMMUNOLOGY, FRONTIERS MEDIA, LAUSANNE, CH, vol. 11, 28 August 2020 (2020-08-28), Lausanne, CH , pages 2028, 1 - 13, XP055926423, ISSN: 1664-3224, DOI: 10.3389/fimmu.2020.02028
YASUYUKI ARAI, UIMOOK CHOI, CRISTINA I. CORSINO, SHERRY M. KOONTZ, MASAKI TAJIMA, COLIN L. SWEENEY, MARY A. BLACK, STEVEN A. FELDM: "Enhanced Expression of CXCR4 Facilitates C-Kit-Targeted CAR-T Cell Trafficking to Bone Marrow and Enables Donor Stem Cell Engraftment", BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, vol. 24, no. 3, Suppl., 1 March 2018 (2018-03-01), US , pages S311, XP009536171, ISSN: 1083-8791, DOI: 10.1016/j.bbmt.2017.12.359
NGUYEN CHI HUU, SCHLERKA ANGELA, GRANDITS ALEXANDER M., KOLLER ELISABETH, VAN DER KOUWE EMIEL, VASSILIOU GEORGE S., STABER PHILIPP: "IL2RA Promotes Aggressiveness and Stem Cell-Related Properties of Acute Myeloid Leukemia", CANCER RESEARCH, AMERICAN ASSOCIATION FOR CANCER RESEARCH, US, vol. 80, no. 20, 15 October 2020 (2020-10-15), US , pages 4527 - 4539, XP009536174, ISSN: 0099-7374, DOI: 10.1158/0008-5472.CAN-20-0531
MICHAEL A. MORGAN, AXEL SCHAMBACH: "Engineering CAR-T Cells for Improved Function Against Solid Tumors", FRONTIERS IN IMMUNOLOGY, vol. 9, 1 January 2018 (2018-01-01), pages 2493, XP055669674, DOI: 10.3389/fimmu.2018.02493
ITOH-NAKADAI ARI, YORIKO SAITO, MARIKO MURASAWA-TOMIZAWA, HIROSHI KAJITA, TAKEHISA MATSUMOTO, MASASHI MATSUDA, TAKASHI WATANABE, M: "CXCR4-Expressing Anti-CD25 CAR T-Cells Effectively Eliminate Human AML Cells In Vivo", BLOOD, AMERICAN SOCIETY OF HEMATOLOGY, US, vol. 136, no. Suppl. 1, 5 November 2020 (2020-11-05), US , pages 35 - 36, XP055926448, ISSN: 0006-4971, DOI: 10.1182/blood-2020-142228
GRUPP, S.A.KALOS, M.BARRETT, D.APLENC, R.PORTER, D.L.RHEINGOLD, S.R.TEACHEY, D.T.CHEW, A.HAUCK, B.WRIGHT, J.F. ET AL.: "Chimeric antigen receptor-modified T cells for acute lymphoid leukemia", N. ENGL. J. MED., vol. 368, 2013, pages 1509 - 1518, XP055169041, DOI: 10.1056/NEJMoa1215134
LEE, D.W.KOCHENDERFER, J.N.STETLER-STEVENSON, M.CUI, Y.K.DELBROOK, C.FELDMAN, S.A.FRY, T.J.ORENTAS, R.SABATINO, M.SHAH, N.N. ET AL: "T cells expressing CD19 chimeric antigen receptors for acute lymphoblastic leukaemia in children and young adults: a phase 1 dose-escalation trial", LANCET, vol. 385, 2015, pages 517 - 528, XP055388598, DOI: 10.1016/S0140-6736(14)61403-3
PARK, J.H.RIVIERE, I.GONEN, M.WANG, X.SENECHAL, B.CURRAN, K.J.SAUTER, C.WANG, Y.SANTOMASSO, B.MEAD, E. ET AL.: "Long-Term Follow-up of CD19 CAR Therapy in Acute Lymphoblastic leukemia", N. ENGL. J. MED., vol. 378, 2018, pages 449 - 459, XP055849327, DOI: 10.1056/NEJMoa1709919
KENDERIAN, S.S.ROSADO, F.G.SYKES, D.B.HOYER, J.D.LACY, M.Q.: "Long-term complete clinical and hematological responses of the TEMPI syndrome after autologous stem cell transplantation", LEUKEMIA, vol. 29, 2015, pages 2414 - 2416, XP036972386, DOI: 10.1038/leu.2015.298
MARDIROS, A.FORMAN, S.J.BUDDE, L.E.: "T cells expressing CD123 chimeric antigen receptors for treatment of acute myeloid leukemia", CURR. OPIN. HEMATOL., vol. 22, 2015, pages 484 - 488
RITCHIE, D.S.NEESON, P.J.KHOT, A.PEINERT, S.TAI, T.TAINTON, K.CHEN, K.SHIN, M.WALL, D.M.HONEMANN, D. ET AL.: "Persistence and efficacy of second generation CAR T cell against the LeY antigen in acute myeloid leukemia", MOL. THER., vol. 21, 2013, pages 2122 - 2129, XP055187778, DOI: 10.1038/mt.2013.154
GONEN ET AL., BLOOD, 2012
SAITO ET AL., SCIENCE TRANSLATIONAL MEDICINE, 2010
"NCBI", Database accession no. NM_000734.3
JANG HWAN CHO, CELL, 2018
SAITO ET AL., SCI. TRANS. MED., 2010
NGUYEN ET AL., CANCER RESEARCH, 2020
MICHAEL C. MILONE ET AL., MOL. THERAPY, vol. 17, no. 8, 2009, pages 1453 - 64
SHULTZ ET AL., J IMMUNOL., 2005
ISHIKAWA ET AL., NATURE BIOTECHNOL., 2007
ISHIKAWA ET AL., BLOOD, 2005
J. ONCOL. PRACT., vol. 14, no. 11, November 2018 (2018-11-01), pages 665 - 671
YASUYUKI ARAI, UIMOOK CHOI, CRISTINA I. CORSINO, SHERRY M. KOONTZ, MASAKI TAJIMA, COLIN L. SWEENEY, MARY A. BLACK, STEVEN A. FELDM: "Enhanced Expression of CXCR4 Facilitates C-Kit-Targeted CAR-T Cell Trafficking to Bone Marrow and Enables Donor Stem Cell Engraftment", BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, vol. 24, no. 3, Suppl., 1 March 2018 (2018-03-01), US , pages S311, XP009536171, ISSN: 1083-8791, DOI: 10.1016/j.bbmt.2017.12.359
NGUYEN CHI HUU, SCHLERKA ANGELA, GRANDITS ALEXANDER M., KOLLER ELISABETH, VAN DER KOUWE EMIEL, VASSILIOU GEORGE S., STABER PHILIPP: "IL2RA Promotes Aggressiveness and Stem Cell-Related Properties of Acute Myeloid Leukemia", CANCER RESEARCH, AMERICAN ASSOCIATION FOR CANCER RESEARCH, US, vol. 80, no. 20, 15 October 2020 (2020-10-15), US , pages 4527 - 4539, XP009536174, ISSN: 0099-7374, DOI: 10.1158/0008-5472.CAN-20-0531
MICHAEL A. MORGAN, AXEL SCHAMBACH: "Engineering CAR-T Cells for Improved Function Against Solid Tumors", FRONTIERS IN IMMUNOLOGY, vol. 9, 1 January 2018 (2018-01-01), pages 2493, XP055669674, DOI: 10.3389/fimmu.2018.02493
ITOH-NAKADAI ARI, YORIKO SAITO, MARIKO MURASAWA-TOMIZAWA, HIROSHI KAJITA, TAKEHISA MATSUMOTO, MASASHI MATSUDA, TAKASHI WATANABE, M: "CXCR4-Expressing Anti-CD25 CAR T-Cells Effectively Eliminate Human AML Cells In Vivo", BLOOD, AMERICAN SOCIETY OF HEMATOLOGY, US, vol. 136, no. Suppl. 1, 5 November 2020 (2020-11-05), US , pages 35 - 36, XP055926448, ISSN: 0006-4971, DOI: 10.1182/blood-2020-142228
GRUPP, S.A.KALOS, M.BARRETT, D.APLENC, R.PORTER, D.L.RHEINGOLD, S.R.TEACHEY, D.T.CHEW, A.HAUCK, B.WRIGHT, J.F. ET AL.: "Chimeric antigen receptor-modified T cells for acute lymphoid leukemia", N. ENGL. J. MED., vol. 368, 2013, pages 1509 - 1518, XP055169041, DOI: 10.1056/NEJMoa1215134
LEE, D.W.KOCHENDERFER, J.N.STETLER-STEVENSON, M.CUI, Y.K.DELBROOK, C.FELDMAN, S.A.FRY, T.J.ORENTAS, R.SABATINO, M.SHAH, N.N. ET AL: "T cells expressing CD19 chimeric antigen receptors for acute lymphoblastic leukaemia in children and young adults: a phase 1 dose-escalation trial", LANCET, vol. 385, 2015, pages 517 - 528, XP055388598, DOI: 10.1016/S0140-6736(14)61403-3
PARK, J.H.RIVIERE, I.GONEN, M.WANG, X.SENECHAL, B.CURRAN, K.J.SAUTER, C.WANG, Y.SANTOMASSO, B.MEAD, E. ET AL.: "Long-Term Follow-up of CD19 CAR Therapy in Acute Lymphoblastic leukemia", N. ENGL. J. MED., vol. 378, 2018, pages 449 - 459, XP055849327, DOI: 10.1056/NEJMoa1709919
KENDERIAN, S.S.ROSADO, F.G.SYKES, D.B.HOYER, J.D.LACY, M.Q.: "Long-term complete clinical and hematological responses of the TEMPI syndrome after autologous stem cell transplantation", LEUKEMIA, vol. 29, 2015, pages 2414 - 2416, XP036972386, DOI: 10.1038/leu.2015.298
MARDIROS, A.FORMAN, S.J.BUDDE, L.E.: "T cells expressing CD123 chimeric antigen receptors for treatment of acute myeloid leukemia", CURR. OPIN. HEMATOL., vol. 22, 2015, pages 484 - 488
RITCHIE, D.S.NEESON, P.J.KHOT, A.PEINERT, S.TAI, T.TAINTON, K.CHEN, K.SHIN, M.WALL, D.M.HONEMANN, D. ET AL.: "Persistence and efficacy of second generation CAR T cell against the LeY antigen in acute myeloid leukemia", MOL. THER., vol. 21, 2013, pages 2122 - 2129, XP055187778, DOI: 10.1038/mt.2013.154
GONEN ET AL., BLOOD, 2012
SAITO ET AL., SCIENCE TRANSLATIONAL MEDICINE, 2010
"NCBI", Database accession no. NM_000734.3
JANG HWAN CHO, CELL, 2018
SAITO ET AL., SCI. TRANS. MED., 2010
NGUYEN ET AL., CANCER RESEARCH, 2020
MICHAEL C. MILONE ET AL., MOL. THERAPY, vol. 17, no. 8, 2009, pages 1453 - 64
SHULTZ ET AL., J IMMUNOL., 2005
ISHIKAWA ET AL., NATURE BIOTECHNOL., 2007
ISHIKAWA ET AL., BLOOD, 2005
J. ONCOL. PRACT., vol. 14, no. 11, November 2018 (2018-11-01), pages 665 - 671
Attorney, Agent or Firm:
HIRAKI & ASSOCIATES (JP)
Download PDF: