An Improved PDE6D Inhibitor Combines with Sildenafil To Inhibit KRAS Mutant Cancer Cell Growth

J Med Chem. 2024 Jun 13;67(11):8569-8584. doi: 10.1021/acs.jmedchem.3c02129. Epub 2024 May 17.

Abstract

The trafficking chaperone PDE6D (or PDEδ) was proposed as a surrogate target for K-Ras, leading to the development of a series of inhibitors that block its prenyl binding pocket. These inhibitors suffered from low solubility and suspected off-target effects, preventing their clinical development. Here, we developed a highly soluble, low nanomolar PDE6D inhibitor (PDE6Di), Deltaflexin3, which has the lowest off-target activity as compared to three prominent reference compounds. Deltaflexin3 reduces Ras signaling and selectively decreases the growth of KRAS mutant and PDE6D-dependent cancer cells. We further show that PKG2-mediated phosphorylation of Ser181 lowers K-Ras binding to PDE6D. Thus, Deltaflexin3 combines with the approved PKG2 activator Sildenafil to more potently inhibit PDE6D/K-Ras binding, cancer cell proliferation, and microtumor growth. As observed previously, inhibition of Ras trafficking, signaling, and cancer cell proliferation remained overall modest. Our results suggest reevaluating PDE6D as a K-Ras surrogate target in cancer.

MeSH terms

  • Animals
  • Antineoplastic Agents / chemical synthesis
  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / pharmacology
  • Cell Line, Tumor
  • Cell Proliferation* / drug effects
  • Cyclic Nucleotide Phosphodiesterases, Type 6* / antagonists & inhibitors
  • Cyclic Nucleotide Phosphodiesterases, Type 6* / metabolism
  • Humans
  • Mutation
  • Phosphodiesterase Inhibitors / chemical synthesis
  • Phosphodiesterase Inhibitors / chemistry
  • Phosphodiesterase Inhibitors / pharmacology
  • Proto-Oncogene Proteins p21(ras)* / antagonists & inhibitors
  • Proto-Oncogene Proteins p21(ras)* / genetics
  • Proto-Oncogene Proteins p21(ras)* / metabolism
  • Sildenafil Citrate* / pharmacology
  • Structure-Activity Relationship

Substances

  • Cyclic Nucleotide Phosphodiesterases, Type 6
  • Sildenafil Citrate
  • Proto-Oncogene Proteins p21(ras)
  • KRAS protein, human
  • PDE6D protein, human
  • Antineoplastic Agents
  • Phosphodiesterase Inhibitors