F14512, a potent antitumor agent targeting topoisomerase II vectored into cancer cells via the polyamine transport system

Cancer Res. 2008 Dec 1;68(23):9845-53. doi: 10.1158/0008-5472.CAN-08-2748.

Abstract

The polyamine transport system (PTS) is an energy-dependent machinery frequently overactivated in cancer cells with a high demand for polyamines. We have exploited the PTS to selectively deliver a polyamine-containing drug to cancer cells. F14512 combines an epipodophyllotoxin core-targeting topoisomerase II with a spermine moiety introduced as a cell delivery vector. The polyamine tail supports three complementary functions: (a) facilitate formulation of a water-soluble compound, (b) increase DNA binding to reinforce topoisomerase II inhibition, and (c) facilitate selective uptake by tumor cells via the PTS. F14512 is 73-fold more cytotoxic to Chinese hamster ovary cells compared with CHO-MG cells with a reduced PTS activity. A decreased sensitivity of L1210 leukemia cells to F14512 was observed in the presence of putrescine, spermidine, and spermine. In parallel, the spermine moiety considerably enhances the drug-DNA interaction, leading to a reinforced inhibition of topoisomerase II. The spermine tail of F14512 serves as a cell delivery vehicle as well as a DNA anchor, and this property translates at the cellular level into a distinct pharmacologic profile. Twenty-nine human solid or hematologic cell lines were used to characterize the high cytotoxic potential of F14512 (median IC50 of 0.18 micromol/L). Finally, the potent antitumor activity of F14512 in vivo was evidenced with a MX1 human breast tumor xenograft model, with partial and complete tumor regressions. This work supports the clinical development of F14512 as a novel targeted cytotoxic drug and sheds light on the concept of selective delivery of drugs to tumor cells expressing the PTS.

MeSH terms

  • Animals
  • Binding, Competitive
  • Biogenic Polyamines / metabolism*
  • Biogenic Polyamines / pharmacology
  • Breast Neoplasms / drug therapy
  • Breast Neoplasms / genetics
  • Breast Neoplasms / metabolism
  • CHO Cells
  • Cell Line, Tumor
  • Cricetinae
  • Cricetulus
  • DNA Damage
  • DNA Topoisomerases, Type II / biosynthesis
  • DNA Topoisomerases, Type II / genetics
  • DNA Topoisomerases, Type II / metabolism
  • DNA, Neoplasm / metabolism
  • Drug Delivery Systems
  • Etoposide / pharmacology
  • Female
  • Humans
  • Leukemia L1210 / drug therapy
  • Leukemia L1210 / metabolism
  • Lung Neoplasms / drug therapy
  • Lung Neoplasms / genetics
  • Lung Neoplasms / metabolism
  • Mice
  • Mice, Nude
  • Neoplasms / drug therapy*
  • Neoplasms / enzymology
  • Neoplasms / genetics
  • Neoplasms / metabolism*
  • Podophyllotoxin / analogs & derivatives*
  • Podophyllotoxin / pharmacology
  • Topoisomerase II Inhibitors*
  • Xenograft Model Antitumor Assays

Substances

  • Biogenic Polyamines
  • DNA, Neoplasm
  • F14512
  • Topoisomerase II Inhibitors
  • Etoposide
  • DNA Topoisomerases, Type II
  • Podophyllotoxin