The novel AML stem cell associated antigen CLL-1 aids in discrimination between normal and leukemic stem cells

Blood. 2007 Oct 1;110(7):2659-66. doi: 10.1182/blood-2007-03-083048. Epub 2007 Jul 3.

Abstract

In CD34(+) acute myeloid leukemia (AML), the malignant stem cells reside in the CD38(-) compartment. We have shown before that the frequency of such CD34(+)CD38(-) cells at diagnosis correlates with minimal residual disease (MRD) frequency after chemotherapy and with survival. Specific targeting of CD34(+)CD38(-) cells might thus offer therapeutic options. Previously, we found that C-type lectin-like molecule-1 (CLL-1) has high expression on the whole blast compartment in the majority of AML cases. We now show that CLL-1 expression is also present on the CD34(+)CD38(-) stem- cell compartment in AML (77/89 patients). The CD34(+)CLL-1(+) population, containing the CD34(+)CD38(-)CLL-1(+) cells, does engraft in nonobese diabetic/severe combined immunodeficiency (NOD/SCID) mice with outgrowth to CLL-1(+) blasts. CLL-1 expression was not different between diagnosis and relapse (n = 9). In remission, both CLL-1(-) normal and CLL-1(+) malignant CD34(+)CD38(-) cells were present. A high CLL-1(+) fraction was associated with quick relapse. CLL-1 expression is completely absent both on CD34(+)CD38(-) cells in normal (n = 11) and in regenerating bone marrow controls (n = 6). This AML stem-cell specificity of the anti-CLL-1 antibody under all conditions of disease and the leukemia-initiating properties of CD34(+)CLL-1(+) cells indicate that anti-CLL-1 antibody enables both AML-specific stem-cell detection and possibly antigen-targeting in future.

MeSH terms

  • ADP-ribosyl Cyclase 1 / metabolism
  • Adolescent
  • Adult
  • Aged
  • Animals
  • Antigens, CD34 / metabolism
  • Bone Marrow / metabolism
  • Cell Transformation, Neoplastic / metabolism*
  • Cell Transformation, Neoplastic / pathology
  • Female
  • Hematopoietic Stem Cells / metabolism*
  • Humans
  • Lectins, C-Type / metabolism*
  • Leukemia, Myeloid, Acute / diagnosis
  • Leukemia, Myeloid, Acute / metabolism*
  • Male
  • Mice
  • Middle Aged
  • Neoplastic Stem Cells / metabolism*
  • Receptors, Mitogen / metabolism*

Substances

  • Antigens, CD34
  • CLEC12A protein, human
  • CLEC12A protein, mouse
  • Lectins, C-Type
  • Receptors, Mitogen
  • ADP-ribosyl Cyclase 1