CD99 is a therapeutic target on disease stem cells in myeloid malignancies Journal Article


Authors: Chung, S. S.; Eng, W. S.; Hu, W.; Khalaj, M.; Garrett-Bakelman, F. E.; Tavakkoli, M.; Levine, R. L.; Carroll, M.; Klimek, V. M.; Melnick, A. M.; Park, C. Y.
Article Title: CD99 is a therapeutic target on disease stem cells in myeloid malignancies
Abstract: Acute myeloid leukemia (AML) and the myelodysplastic syndromes (MDS) are initiated and sustained by selfrenewing malignant stem cells; thus, eradication of AML and MDS stem cells is required for cure. We identified CD99 as a cell surface protein frequently overexpressed on AML and MDS stem cells. Expression of CD99 allows for prospective separation of leukemic stem cells (LSCs) from functionally normal hematopoietic stem cells in AML, and high CD99 expression on AML blasts enriches for functional LSCs as demonstrated by limiting dilution xenotransplant studies. Monoclonal antibodies (mAbs) targeting CD99 induce the death of AML and MDS cells in a SARC family kinase-dependent manner in the absence of immune effector cells or complement, and anti- CD99 mAbs exhibit antileukemic activity in AML xenografts. These data establish CD99 as a marker of AML and MDS stem cells, as well as a promising therapeutic target in these disorders. 2017 © The Authors, some rights reserved; exclusive licensee American Association for the Advancement of Science.
Journal Title: Science Translational Medicine
Volume: 9
Issue: 374
ISSN: 1946-6234
Publisher: American Association for the Advancement of Science  
Date Published: 2017-01-25
Start Page: eaaj2025
Language: English
DOI: 10.1126/scitranslmed.aaj2025
PROVIDER: scopus
PUBMED: 28123069
PMCID: PMC5624309
DOI/URL:
Notes: Article -- Export Date: 2 March 2017 -- Source: Scopus
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MSK Authors
  1. Virginia Klimek
    147 Klimek
  2. Stephen Shiu-Wah Chung
    61 Chung
  3. Ross Levine
    776 Levine
  4. Wenhuo Hu
    60 Hu
  5. Christopher Yongchul Park
    90 Park
  6. William S Eng
    5 Eng
  7. Mona   Khalaj
    9 Khalaj