p53-independent ibrutinib responses in an Eμ-TCL1 mouse model demonstrates efficacy in high-risk CLL

Blood Cancer J. 2016 Jun 10;6(6):e434. doi: 10.1038/bcj.2016.41.

Abstract

Deletion of the short-arm of chromosome 17 (17p-) is one of the most critical genetic alterations used in chronic lymphocytic leukemia (CLL) risk stratification. The tumor suppressor TP53 maps to this region, and its loss or mutation accelerates CLL progression, hampers response to chemotherapy and shortens survival. Although florescent in situ hybridization analyses for 17p deletions are routinely performed during clinical diagnoses, p53 mutational status is often unexamined. Given the limited clinical data that exists for frontline treatment of patients with CLL harboring TP53 mutations, there is a need to understand the biology of CLL with TP53 mutations and identify treatment strategies for this subset of patients. Herein, we used a CLL mouse model (Eμ-TCL1) harboring one of the most common TP53 hot-spot mutations observed in CLL (p53(R172H), corresponding to p53(R175H) in humans) to evaluate its impact on disease progression, survival, response to therapy and loss of the remaining wild-type Trp53 allele following ibrutinib treatment. We show that ibrutinib was effective in increasing survival, activating cellular programs outside the p53 pathway and did not place selective pressure on the remaining wild-type Trp53 allele. These data provide evidence that ibrutinib acts as an effective treatment for aggressive forms of CLL with TP53 mutations.

MeSH terms

  • Adenine / analogs & derivatives
  • Angiopoietin-1
  • Animals
  • Apoptosis / genetics
  • Cell Line, Tumor
  • Cell Proliferation
  • Cluster Analysis
  • Disease Models, Animal
  • Disease Progression
  • Gene Expression Profiling
  • Humans
  • Kaplan-Meier Estimate
  • Leukemia, Lymphocytic, Chronic, B-Cell / drug therapy
  • Leukemia, Lymphocytic, Chronic, B-Cell / genetics*
  • Leukemia, Lymphocytic, Chronic, B-Cell / mortality
  • Leukemia, Lymphocytic, Chronic, B-Cell / pathology
  • Loss of Heterozygosity
  • Mice
  • Mice, Knockout
  • Mutation
  • Piperidines
  • Prognosis
  • Protein Kinase Inhibitors / pharmacology*
  • Proto-Oncogene Proteins / genetics*
  • Pyrazoles / pharmacology*
  • Pyrimidines / pharmacology*
  • Signal Transduction
  • Tumor Suppressor Protein p53 / genetics*
  • Tumor Suppressor Protein p53 / metabolism
  • Xenograft Model Antitumor Assays

Substances

  • Angiopoietin-1
  • Angpt1 protein, mouse
  • Piperidines
  • Protein Kinase Inhibitors
  • Proto-Oncogene Proteins
  • Pyrazoles
  • Pyrimidines
  • TCL1A protein, human
  • Tcl1 protein, mouse
  • Tumor Suppressor Protein p53
  • ibrutinib
  • Adenine