Identification of Wee1 as a novel therapeutic target for mutant RAS-driven acute leukemia and other malignancies

Leukemia. 2015 Jan;29(1):27-37. doi: 10.1038/leu.2014.149. Epub 2014 May 5.

Abstract

Direct targeting of rat sarcoma (RAS), which is frequently mutated, has proven to be challenging, and inhibition of individual downstream RAS mediators has resulted in limited clinical efficacy. We designed a chemical screen to identify compounds capable of potentiating mammalian target of rapamycin (mTOR) inhibition in mutant RAS-positive leukemia, and identified a Wee1 inhibitor. Synergy was observed in both mutant neuroblastoma RAS viral oncogene homolog (NRAS)- and mutant kirsten RAS viral oncogene homolog (KRAS)-positive acute myelogenous leukemia (AML) cell lines and primary patient samples. The observed synergy enhanced dephosphorylation of AKT, 4E-binding protein 1 and s6 kinase, and correlated with increased apoptosis. The specificity of Wee1 as the target of MK-1775 was validated by Wee1 knockdown, as well as partial reversal of drug combination-induced apoptosis by a cyclin-dependent kinase 1 (CDK1) inhibitor. Importantly, we also extended our findings to other mutant RAS-expressing malignancies, including mutant NRAS-positive melanoma, and mutant KRAS-positive colorectal cancer, pancreatic cancer and lung cancer. We observed favorable responses with combined Wee1/mTOR inhibition in human cancer cell lines from multiple malignancies, and inhibition of tumor growth in in vivo models of mutant KRAS lung cancer and leukemia. The present study introduces for the first time Wee1 inhibition combined with mTOR inhibition as a novel therapeutic strategy for the selective treatment of mutant RAS-positive leukemia and other mutant RAS-expressing malignancies.

Publication types

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

MeSH terms

  • Animals
  • Antineoplastic Agents / pharmacology
  • Antineoplastic Agents / therapeutic use
  • Base Sequence
  • Cell Cycle Proteins / drug effects*
  • Cell Cycle Proteins / genetics
  • Cell Line, Tumor
  • Gene Knockdown Techniques
  • Leukemia, Myeloid, Acute / drug therapy*
  • Leukemia, Myeloid, Acute / genetics
  • Leukemia, Myeloid, Acute / pathology
  • Mice
  • Nuclear Proteins / drug effects*
  • Nuclear Proteins / genetics
  • Protein-Tyrosine Kinases / drug effects*
  • Protein-Tyrosine Kinases / genetics
  • Proto-Oncogene Proteins p21(ras) / genetics*
  • Pyrazoles / pharmacology
  • Pyrazoles / therapeutic use
  • Pyrimidines / pharmacology
  • Pyrimidines / therapeutic use
  • Pyrimidinones
  • RNA, Small Interfering / genetics

Substances

  • Antineoplastic Agents
  • Cell Cycle Proteins
  • Nuclear Proteins
  • Pyrazoles
  • Pyrimidines
  • Pyrimidinones
  • RNA, Small Interfering
  • Protein-Tyrosine Kinases
  • WEE1 protein, human
  • Proto-Oncogene Proteins p21(ras)
  • adavosertib