Targeting acute myeloid leukemia by dual inhibition of PI3K signaling and Cdk9-mediated Mcl-1 transcription

Blood. 2013 Aug 1;122(5):738-48. doi: 10.1182/blood-2012-08-447441. Epub 2013 Jun 17.

Abstract

Resistance to cell death is a hallmark of cancer and renders transformed cells resistant to multiple apoptotic triggers. The Bcl-2 family member, Mcl-1, is a key driver of cell survival in diverse cancers, including acute myeloid leukemia (AML). A screen for compounds that downregulate Mcl-1 identified the kinase inhibitor, PIK-75, which demonstrates marked proapoptotic activity against a panel of cytogenetically diverse primary human AML patient samples. We show that PIK-75 transiently blocks Cdk7/9, leading to transcriptional suppression of MCL-1, rapid loss of Mcl-1 protein, and alleviation of its inhibition of proapoptotic Bak. PIK-75 also targets the p110α isoform of PI3K, which leads to a loss of association between Bcl-xL and Bak. The simultaneous loss of Mcl-1 and Bcl-xL association with Bak leads to rapid apoptosis of AML cells. Concordantly, low Bak expression in AML confers resistance to PIK-75-mediated killing. On the other hand, the induction of apoptosis by PIK-75 did not require the expression of the BH3 proteins Bim, Bid, Bad, Noxa, or Puma. PIK-75 significantly reduced leukemia burden and increased the survival of mice engrafted with human AML without inducing overt toxicity. Future efforts to cotarget PI3K and Cdk9 with drugs such as PIK-75 in AML are warranted.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antineoplastic Agents / pharmacology
  • Antineoplastic Agents / therapeutic use
  • Cells, Cultured
  • Cyclin-Dependent Kinase 9 / antagonists & inhibitors*
  • Gene Expression Regulation, Leukemic / drug effects
  • HEK293 Cells
  • HL-60 Cells
  • Humans
  • Hydrazones / therapeutic use
  • Leukemia, Myeloid, Acute / drug therapy*
  • Leukemia, Myeloid, Acute / pathology
  • Mice
  • Mice, Inbred NOD
  • Mice, Knockout
  • Mice, SCID
  • Molecular Targeted Therapy / methods*
  • Myeloid Cell Leukemia Sequence 1 Protein
  • Phosphoinositide-3 Kinase Inhibitors*
  • Protein Kinase Inhibitors / pharmacology
  • Protein Kinase Inhibitors / therapeutic use*
  • Proto-Oncogene Proteins c-bcl-2 / genetics*
  • Signal Transduction / drug effects
  • Signal Transduction / genetics
  • Sulfonamides / therapeutic use
  • Transcription, Genetic / drug effects
  • Xenograft Model Antitumor Assays

Substances

  • Antineoplastic Agents
  • Hydrazones
  • Mcl1 protein, mouse
  • Myeloid Cell Leukemia Sequence 1 Protein
  • PIK 75
  • Phosphoinositide-3 Kinase Inhibitors
  • Protein Kinase Inhibitors
  • Proto-Oncogene Proteins c-bcl-2
  • Sulfonamides
  • CDK9 protein, human
  • Cyclin-Dependent Kinase 9