Phosphonooxymethyl Prodrug of Triptolide: Synthesis, Physicochemical Characterization, and Efficacy in Human Colon Adenocarcinoma and Ovarian Cancer Xenografts

J Med Chem. 2015 Dec 10;58(23):9334-44. doi: 10.1021/acs.jmedchem.5b01329. Epub 2015 Nov 24.

Abstract

A disodium phosphonooxymethyl prodrug of the antitumor agent triptolide was prepared from the natural product in three steps (39% yield) and displayed excellent aqueous solubility at pH 7.4 (61 mg/mL) compared to the natural product (17 μg/mL). The estimated shelf life (t90) for hydrolysis of the prodrug at 4 °C and pH 7.4 was found to be two years. In a mouse model of human colon adenocarcinoma (HT-29), the prodrug administered intraperitoneally was effective in reducing or eliminating xenograft tumors at dose levels as low as 0.3 mg/kg when given daily and at 0.9 mg/kg when given less frequently. When given via intraperitoneal and oral routes at daily doses of 0.6 and 0.9 mg/kg, the prodrug was also effective and well tolerated in a mouse model of human ovarian cancer (A2780).

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adenocarcinoma / drug therapy*
  • Adenocarcinoma / pathology
  • Animals
  • Antineoplastic Agents / chemical synthesis
  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / therapeutic use*
  • Cell Line, Tumor
  • Colon / drug effects
  • Colon / pathology
  • Colonic Neoplasms / drug therapy*
  • Colonic Neoplasms / pathology
  • Diterpenes
  • Drug Stability
  • Epoxy Compounds
  • Female
  • HT29 Cells
  • Humans
  • Mice
  • Mice, Nude
  • Organophosphates / chemical synthesis
  • Organophosphates / chemistry
  • Organophosphates / therapeutic use*
  • Ovarian Neoplasms / drug therapy*
  • Ovarian Neoplasms / pathology
  • Ovary / drug effects
  • Ovary / pathology
  • Phenanthrenes / chemical synthesis
  • Phenanthrenes / chemistry
  • Phenanthrenes / therapeutic use*
  • Prodrugs / chemical synthesis
  • Prodrugs / chemistry
  • Prodrugs / therapeutic use*
  • Solubility

Substances

  • Antineoplastic Agents
  • Diterpenes
  • Epoxy Compounds
  • Organophosphates
  • Phenanthrenes
  • Prodrugs
  • 14-O-phosphonooxymethyltriptolide disodium salt