Small-molecule multi-targeted kinase inhibitor RGB-286638 triggers P53-dependent and -independent anti-multiple myeloma activity through inhibition of transcriptional CDKs

Leukemia. 2013 Dec;27(12):2366-75. doi: 10.1038/leu.2013.194. Epub 2013 Jun 28.

Abstract

Small-molecule multi-targeted cyclin-dependent kinase (CDK) inhibitors (CDKIs) are of particular interest due to their potent antitumor activity independent of p53 gene alterations. P53 deletion is associated with a very poor prognosis in multiple myeloma (MM). In this regard, we tested the anti-MM activity of RGB-286638, an indenopyrazole-derived CDKI with Ki-nanomolar activity against transcriptional CDKs. We examined RGB-286638's mode-of-action in MM cell lines with wild-type (wt)-p53 and those expressing mutant p53. RGB-286638 treatment resulted in MM cytotoxicity in vitro associated with inhibition of MM tumor growth and prolonged survival in vivo. RGB-286638 displayed caspase-dependent apoptosis in both wt-p53 and mutant-p53 cells that was closely associated with the downregulation of RNA polymerase II phosphorylation and inhibition of transcription. RGB-286638 triggered p53 accumulation via nucleolar stress and loss of Mdm2, accompanied by induction of p53 DNA-binding activity. In addition, RGB-286638 mediated p53-independent activity, which was confirmed by cytotoxicity in p53-knockdown and p53-mutant cells. We also demonstrated downregulation of oncogenic miR-19, miR-92a-1 and miR-21. Our data provide the rationale for the development of transcriptional CDKIs as therapeutic agents, which activate p53 in competent cells, while circumventing p53 deficiency through alternative p53-independent cell death mechanisms in p53-mutant/deleted cells.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Animals
  • Apoptosis / drug effects
  • Cyclin-Dependent Kinases / antagonists & inhibitors*
  • Humans
  • Male
  • Mice
  • Multiple Myeloma / pathology*
  • Phosphorylation
  • Protein Kinase Inhibitors / pharmacology
  • Pyrazoles / pharmacology*
  • Real-Time Polymerase Chain Reaction
  • Transcription, Genetic / drug effects*
  • Tumor Suppressor Protein p53 / physiology*
  • Urea / analogs & derivatives*
  • Urea / pharmacology
  • Xenograft Model Antitumor Assays

Substances

  • Protein Kinase Inhibitors
  • Pyrazoles
  • RGB 286638
  • TP53 protein, human
  • Tumor Suppressor Protein p53
  • Urea
  • Cyclin-Dependent Kinases