MUC1 is a potential target for the treatment of acute myeloid leukemia stem cells

Dina Stroopinsky, Jacalyn Rosenblatt, Keisuke Ito, Heidi Mills, Li Yin, Hasan Rajabi, Baldev Vasir, Turner Kufe, Katarina Luptakova, Jon Arnason, Caterina Nardella, James D. Levine, Robin M. Joyce, Ilene Galinsky, Yoram Reiter, Richard M. Stone, Pier Paolo Pandolfi, Donald Kufe, David Avigan

Research output: Contribution to journalArticlepeer-review

43 Scopus citations


Acute myeloid leukemia (AML) is a malignancy of stem cells with an unlimited capacity for self-renewal. MUC1 is a secreted, oncogenic mucin that is expressed aberrantly in AML blasts, but its potential uses to target AML stem cells have not been explored. Here, we report that MUC1 is highly expressed on AML CD34+/lineage-/ CD38- cells as compared with their normal stem cell counterparts. MUC1 expression was not restricted to AML CD34+ populations as similar results were obtained with leukemic cells from patients with CD34- disease. Engraftment of AML stem cell populations that highly express MUC1 (MUC1high) led to development of leukemia in NOD-SCID IL2Rgammanull (NSG) immunodeficient mice. In contrast, MUC1low cell populations established normal hematopoiesis in the NSG model. Functional blockade of the oncogenic MUC1-C subunit with the peptide inhibitor GO-203 depleted established AML in vivo, but did not affect engraftment of normal hematopoietic cells. Our results establish that MUC1 is highly expressed in AML stem cells and they define the MUC1-C subunit as a valid target for their therapeutic eradication.

Original languageEnglish (US)
Pages (from-to)5569-5579
Number of pages11
JournalCancer research
Issue number17
StatePublished - Sep 1 2013
Externally publishedYes

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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