Exhaustive analysis for clinical application of anti-complement drugs
Project/Area Number |
15K09502
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Research Category |
Grant-in-Aid for Scientific Research (C)
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Allocation Type | Multi-year Fund |
Section | 一般 |
Research Field |
Hematology
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Research Institution | Osaka University |
Principal Investigator |
Ueda Yasutaka 大阪大学, 医学系研究科, 特任助教(常勤) (30533848)
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Co-Investigator(Kenkyū-buntansha) |
西村 純一 大阪大学, 医学系研究科, 助教 (80464246)
金倉 譲 大阪大学, 医学系研究科, 教授 (20177489)
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Project Period (FY) |
2015-04-01 – 2018-03-31
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Project Status |
Completed (Fiscal Year 2017)
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Budget Amount *help |
¥4,940,000 (Direct Cost: ¥3,800,000、Indirect Cost: ¥1,140,000)
Fiscal Year 2017: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2016: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2015: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
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Keywords | 発作性夜間ヘモグロビン尿症 / PNH / 補体 / 溶血性貧血 / 抗補体薬 / 血管内溶血 / 遺伝子多型 |
Outline of Final Research Achievements |
Eculizumab, an anti-C5 antibody, effectively blocks complement-mediated intravascular hemolysis in paroxysmal nocturnal hemoglobinuria (PNH), but some patients show poor response to the drug, and others show insufficient recovery from hemolytic anemia possibly due to extravascular hemolysis manifested by eculizumab. We obtained new candidate drugs for PNH including an anti-C5 recombinant protein secreted in tick saliva, an anti-C5 circular peptide, a Factor D inhibitor, C1-inactivator, and a C3 inhibitor, and tested, in vitro, those efficacy in inhibiting intravascular and extravascular hemolysis. Our data suggest that targeting a molecule in the alternative pathway may be promising in its potency and safety in treating PNH.
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Report
(4 results)
Research Products
(24 results)
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[Journal Article] Effects of eculizumab treatment on quality of life in patients with paroxysmal nocturnal hemoglobinuria in Japan2018
Author(s)
Y. Ueda, N. Obara, Y. Yonemura, H. Noji, M. Masuko, Y. Seki, K. Wada, T. Matsuda, H. Akiyama, T. Ikezoe, S. Chiba, Y. Kanda, T. Kawaguchi, T. Shichishima, H. Nakakuma, S. Okamoto, J. Nishimura, Y. Kanakura, H. Ninomiya
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Journal Title
International Journal of Hematology
Volume: epub
Issue: 6
Pages: 1-10
DOI
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Peer Reviewed
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[Presentation] Baseline Assessment of Patients with Newly-Diagnosed Acquired Bone Marrow Failure Enrolled in a Prospective Observational Study, “Search for Unidentified Links between PNH Clone Size and the Related Clinical Manifestations By High Precision Flow Cytometry (SUPREMACY)”2017
Author(s)
Yuji Yonemura, Tatsuya Kawaguchi, Ken Ishiyama, Chiharu Sugimori, Kohei Hosokawa, Naoshi Obara, Hideyoshi Noji, Yukari Shirasugi, Yasutaka Ueda, Hiroyuki Takamori, Tsutomu Shichishima, Shigeru Chiba, Haruhiko Ninomiya, Kiyoshi Ando, Jun-Ichi Nishimura, Yuzuru Kanakura, Masanori Takahashi, Shinji Nakao
Organizer
The American Society of Hematology 59th Annual Meeting
Related Report
Int'l Joint Research
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[Presentation] The First Follow-up Data Analysis of Patients with Acquired Bone Marrow Failure Harboring a Small Population of PNH-Type Cells in the Japanese, Multicenter, Prospective Study Optima2016
Author(s)
Ueda Y, Nishimura J, Sugimori C, Hosokawa K, Yonemura Y, Obara N, Noji H, Nakamura Y, Shirasugi Y, Ando K, Shichishima T, Ninomiya H, Chiba S, Kawaguchi T, Kanakura Y, Nakao S
Organizer
The American Society of Hematology 58th Annual Meeting
Place of Presentation
San Diego Convention Center, San Diego, USA
Year and Date
2016-12-03
Related Report
Int'l Joint Research
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[Presentation] Paroxysmal Nocturnal Hemoglobinuria Caused By Pigt Mutations; Atypical PNH2016
Author(s)
Murakami Y, Kwamoto M, Inoue N, Osato M, Murata S, Murase S, Yoshimura H, Ueda Y, Nishimura J, Kankura Y, Kohara N, Kinoshita T
Organizer
The American Society of Hematology 58th Annual Meeting
Place of Presentation
San Diego Convention Center, San Diego, USA
Year and Date
2016-12-03
Related Report
Int'l Joint Research
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[Presentation] A CLINICAL SIGNIFICANCE AND TIME-DEPENDENT CHANGE OF PNH CLONE SIZE IN PATIENTS WITH BONE BARROW FAILURE SYNDROME: JAPANESE MULTI-CENTRE PROSPECTIVE STUDY2016
Author(s)
Shirasugi Y, Sugimori C, Noji H, Shichishima T,Obara N, Chiba S, Ninomiya H, Nakamura Y, Ando K, Yonemura Y, Kawaguchi , Hosokawa K, Ueda Y, Nishimura J, Kanakura Y, Nakao S
Organizer
21st Congress of the European Hematology Association
Place of Presentation
Bella Center, Copenhagen, Denmark
Year and Date
2016-06-09
Related Report
Int'l Joint Research
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[Book] 中山書店2015
Author(s)
植田 康敬、西村 純一 (金倉 譲編)
Total Pages
560
Publisher
最新ガイドライン準拠 血液疾患 診断・治療指針
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