Rational development of gemcitabine-based nanoplatform for targeting SERPINB9/Granzyme B axis to overcome chemo-immune-resistance

Nat Commun. 2025 May 5;16(1):4176. doi: 10.1038/s41467-025-59490-y.

Abstract

SERPINB9, an endogenous inhibitor of granzyme B (GzmB), has emerged as a critical factor in the resistance to immunotherapy by protecting cancer cells from GzmB-induced cytotoxicity. However, its role in chemosensitivity remains unknown. In this study, we show that gemcitabine (GEM) treatment upregulates SERPINB9 through transcription factor ATF-3. Interestingly, GEM also induces the expression of GzmB and knockout or knockdown of SERPINB9 results in enhanced response of tumor cells to GEM, suggesting a role of GzmB/SERPINB9 axis in regulating chemosensitivity. To facilitate the therapeutic translation of these findings, we engineer POEM nanocarrier (consisting of lipid-derivatized polylysine (PEG-PLL-Oleic acid, PPO), and GEM-conjugated polylysine (PEG-PLL-OA-GEM, PPOGEM), PPO/PPOGEM (POEM)) that is highly effective in codelivery of built-in GEM and loaded SERPINB9 short interfering RNA (siSPB9). GEM conjugation introduces an additional mechanism of carrier/siRNA interaction in addition to charge-mediated interaction and enables efficient i.v. delivery at lower N/P ratios. Here, we show that co-delivery of GEM and siSPB9 significantly improves antitumor efficacy and remodels the tumor immune microenvironment in pancreatic cancer models, supporting a promising therapeutic strategy.

MeSH terms

  • Animals
  • Antimetabolites, Antineoplastic / pharmacology
  • Cell Line, Tumor
  • Deoxycytidine* / administration & dosage
  • Deoxycytidine* / analogs & derivatives
  • Deoxycytidine* / chemistry
  • Deoxycytidine* / pharmacology
  • Drug Resistance, Neoplasm* / drug effects
  • Gemcitabine
  • Granzymes* / antagonists & inhibitors
  • Granzymes* / genetics
  • Granzymes* / metabolism
  • Humans
  • Mice
  • Nanoparticles / chemistry
  • Pancreatic Neoplasms / drug therapy
  • Pancreatic Neoplasms / immunology
  • RNA, Small Interfering / genetics
  • Serpins* / genetics
  • Serpins* / metabolism
  • Xenograft Model Antitumor Assays

Substances

  • Gemcitabine
  • Deoxycytidine
  • Serpins
  • Granzymes
  • SERPINB9 protein, human
  • Antimetabolites, Antineoplastic
  • RNA, Small Interfering