Sequential Targeting TGF-β Signaling and KRAS Mutation Increases Therapeutic Efficacy in Pancreatic Cancer

Small. 2019 Jun;15(24):e1900631. doi: 10.1002/smll.201900631. Epub 2019 Apr 29.

Abstract

Pancreatic cancer is a highly aggressive malignancy that strongly resists extant treatments. The failure of existing therapies is majorly attributed to the tough tumor microenvironment (TME) limiting drug access and the undruggable targets of tumor cells. The formation of suppressive TME is regulated by transforming growth factor beta (TGF-β) signaling, while the poor response and short survival of almost 90% of pancreatic cancer patients results from the oncogenic KRAS mutation. Hence, simultaneously targeting both the TGF-β and KRAS pathways might dismantle the obstacles of pancreatic cancer therapy. Here, a novel sequential-targeting strategy is developed, in which antifibrotic fraxinellone-loaded CGKRK-modified nanoparticles (Frax-NP-CGKRK) are constructed to regulate TGF-β signaling and siRNA-loaded lipid-coated calcium phosphate (LCP) biomimetic nanoparticles (siKras-LCP-ApoE3) are applied to interfere with the oncogenic KRAS. Frax-NP-CGKRK successfully targets the tumor sites through the recognition of overexpressed heparan sulfate proteoglycan, reverses the activated cancer-associated fibroblasts (CAFs), attenuates the dense stroma barrier, and enhances tumor blood perfusion. Afterward, siKras-LCP-ApoE3 is efficiently internalized by the tumor cells through macropinocytosis and specifically silencing KRAS mutation. Compared with gemcitabine, this sequential-targeting strategy significantly elongates the lifespans of pancreatic tumor-bearing animals, hence providing a promising approach for pancreatic cancer therapy.

Keywords: KRAS; TGF-β; pancreatic cancer; sequential therapy.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Apolipoprotein E3 / genetics
  • Benzofurans / administration & dosage*
  • Drug Administration Schedule
  • Drug Carriers / administration & dosage
  • Gene Expression Regulation, Neoplastic / drug effects
  • Humans
  • Mice
  • Mice, Nude
  • Molecular Targeted Therapy / methods*
  • Mutation / drug effects
  • NIH 3T3 Cells
  • Nanoparticles / administration & dosage
  • Pancreatic Neoplasms / drug therapy*
  • Pancreatic Neoplasms / pathology
  • Peptide Fragments / administration & dosage
  • Proto-Oncogene Proteins p21(ras) / antagonists & inhibitors*
  • Proto-Oncogene Proteins p21(ras) / drug effects
  • Proto-Oncogene Proteins p21(ras) / genetics
  • RNA, Small Interfering / administration & dosage*
  • Signal Transduction / drug effects
  • Transforming Growth Factor beta / drug effects*
  • Transforming Growth Factor beta / metabolism
  • Tumor Cells, Cultured
  • Xenograft Model Antitumor Assays

Substances

  • Apolipoprotein E3
  • Benzofurans
  • Drug Carriers
  • KRAS protein, human
  • Peptide Fragments
  • RNA, Small Interfering
  • Transforming Growth Factor beta
  • fraxinellone
  • Proto-Oncogene Proteins p21(ras)