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Studying 830 pre-B ALL cases from four clinical trials, we found that human ALL can be divided into two fundamentally distinct subtypes based on pre-BCR function. While absent in the majority of ALL cases, tonic pre-BCR signaling was found in 112 cases (13.5%). In these cases, tonic pre-BCR signaling induced activation of BCL6, which in turn increased pre-BCR signaling output at the transcriptional level. Interestingly, inhibition of pre-BCR-related tyrosine kinases reduced constitutive BCL6 expression and selectively killed patient-derived pre-BCR(+) ALL cells. These findings identify a genetically and phenotypically distinct subset of human ALL that critically depends on tonic pre-BCR signaling. In vivo treatment studies suggested that pre-BCR tyrosine kinase inhibitors are useful for the treatment of patients with pre-BCR(+) ALL.

Original publication

DOI

10.1016/j.ccell.2015.02.003

Type

Journal article

Journal

Cancer Cell

Publication Date

09/03/2015

Volume

27

Pages

409 - 425

Keywords

Basic Helix-Loop-Helix Transcription Factors, Clinical Trials as Topic, DNA-Binding Proteins, Gene Expression Regulation, Neoplastic, Humans, Intracellular Signaling Peptides and Proteins, Molecular Sequence Data, Phosphatidylinositol 3-Kinase, Pre-B-Cell Leukemia Transcription Factor 1, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Precursor Cells, B-Lymphoid, Protein-Tyrosine Kinases, Proto-Oncogene Proteins, Proto-Oncogene Proteins c-bcl-6, Signal Transduction, Syk Kinase, Up-Regulation, src-Family Kinases