Musashi 2 influences chronic lymphocytic leukemia cell survival and growth making it a potential therapeutic target

Leukemia. 2021 Apr;35(4):1037-1052. doi: 10.1038/s41375-020-01115-y. Epub 2021 Jan 27.

Abstract

Progression of chronic lymphocytic leukemia (CLL) results from the expansion of a small fraction of proliferating leukemic B cells. When comparing the global gene expression of recently divided CLL cells with that of previously divided cells, we found higher levels of genes involved in regulating gene expression. One of these was the oncogene Musashi 2 (MSI2), an RNA-binding protein that induces or represses translation. While there is an established role for MSI2 in normal and malignant stem cells, much less is known about its expression and role in CLL. Here we report for the first time ex vivo and in vitro experiments that MSI2 protein levels are higher in dividing and recently divided leukemic cells and that downregulating MSI2 expression or blocking its function eliminates primary human and murine CLL and mature myeloid cells. Notably, mature T cells and hematopoietic stem and progenitor cells are not affected. We also confirm that higher MSI2 levels correlate with poor outcome markers, shorter time-to-first-treatment, and overall survival. Thus, our data highlight an important role for MSI2 in CLL-cell survival and proliferation and associate MSI2 with poor prognosis in CLL patients. Collectively, these findings pinpoint MSI2 as a potentially valuable therapeutic target in CLL.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antineoplastic Agents
  • Apoptosis / drug effects
  • Biomarkers, Tumor*
  • Caspase 3 / metabolism
  • Cell Cycle Checkpoints / drug effects
  • Cell Line, Tumor
  • Cell Survival / genetics
  • Cyclin-Dependent Kinase Inhibitor p27 / metabolism
  • Disease Models, Animal
  • Gene Expression
  • Gene Expression Profiling
  • Gene Expression Regulation, Leukemic
  • Gene Knockdown Techniques
  • Humans
  • Immunophenotyping
  • Leukemia, Lymphocytic, Chronic, B-Cell / genetics*
  • Leukemia, Lymphocytic, Chronic, B-Cell / metabolism
  • Leukemia, Lymphocytic, Chronic, B-Cell / mortality
  • Leukemia, Lymphocytic, Chronic, B-Cell / pathology
  • Mice
  • Molecular Targeted Therapy
  • Prognosis
  • RNA, Small Interfering
  • RNA-Binding Proteins / genetics*
  • RNA-Binding Proteins / metabolism
  • Tumor Suppressor Protein p53 / metabolism
  • Xenograft Model Antitumor Assays

Substances

  • Antineoplastic Agents
  • Biomarkers, Tumor
  • CDKN1B protein, human
  • MSI2 protein, human
  • RNA, Small Interfering
  • RNA-Binding Proteins
  • Tumor Suppressor Protein p53
  • Cyclin-Dependent Kinase Inhibitor p27
  • Caspase 3