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Research for diversity of treatment response for myeloid leukemia with Down syndrome

Research Project

Project/Area Number 17K08443
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeMulti-year Fund
Section一般
Research Field Medical pharmacy
Research InstitutionShiga University of Medical Science

Principal Investigator

Taga Takashi  滋賀医科大学, 医学部, 准教授 (30273410)

Project Period (FY) 2017-04-01 – 2021-03-31
Project Status Completed (Fiscal Year 2020)
Budget Amount *help
¥4,810,000 (Direct Cost: ¥3,700,000、Indirect Cost: ¥1,110,000)
Fiscal Year 2019: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2018: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
Fiscal Year 2017: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Keywords薬剤感受性 / 白血病 / 抗がん剤 / 小児 / ダウン症 / 臨床薬学
Outline of Final Research Achievements

Myeloid leukemia with Down syndrome is associated with good prognosis in general, however, few patients have very poor prognosis. To address for this diversity and to develop new treatment approach, drug sensitivity test for leukemic cells of ML-DS were conducted. Unfortunately, we could not evaluate leukemic cells of ML-DS, but we could these of eight patients with transient abnormality of myelopoiesis (TAM), which is considered pre-leukemic status of ML-DS. The leukemic cells with TAM had high sensitivity for cytarabine, dexamethasone and Trametinib (MEK inhibitor). This finding indicates TAM’s cells proliferate via RAS/MEK pathway and trametinib could be one of the promising drugs for treatment TAM and ML-DS.

Academic Significance and Societal Importance of the Research Achievements

近年、白血病を含むがん治療においては分子標的薬の開発ならびに導入が進んでいるが、TAMならびにML-DSに対してはほとんど手が付けられていない。この研究はTAMならびにML-DSの病的細胞の増殖機転の解明につながる可能性がある。また、これらの疾患はダウン症という基礎疾患に引き続き発症することから、従来のような全身に負担となる抗がん剤治療でなく、より特異性の高く副作用の少ない治療薬の開発が必要であり、この研究の成果ならびに発展が有力な治療方法の開発につながる可能性がある。

Report

(5 results)
  • 2020 Annual Research Report   Final Research Report ( PDF )
  • 2019 Research-status Report
  • 2018 Research-status Report
  • 2017 Research-status Report
  • Research Products

    (1 results)

All 2019

All Presentation (1 results)

  • [Presentation] In vitro Testing of Trametinib in Transient Abnormal Myelopoiesis2019

    • Author(s)
      Yokosuka T, Nakamura W, Hayashi A,Fujita S, Miyagawa N ,Iwasaki F, Hamanoue S, Goto S, Goto H, Taga T
    • Organizer
      第61回日本小児血液・がん学会
    • Related Report
      2019 Research-status Report

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Published: 2017-04-28   Modified: 2022-01-27  

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