Preclinical antitumor activity of a novel folate-targeted dual drug conjugate

Mol Pharm. 2007 Sep-Oct;4(5):659-67. doi: 10.1021/mp070049c. Epub 2007 Sep 18.

Abstract

We have designed a new type of tumor-targeted agent by tethering two different drug molecules, with distinct biological mechanisms of action, to the same ligand. This compound, named EC0225, represents the "first in class" multidrug, folate receptor (FR)-targeted agent to be disclosed. It was constructed with a single folate molecule, extended by a hydrophilic peptide-based spacer, which was in turn attached to mitomycin and Vinca alkaloid units via two separate disulfide-containing linkers. EC0225 produced potent, dose-responsive activity in vitro, and curative activity was observed against FR-positive syngeneic and xenograft tumors following the administration of well-tolerated dosing regimens. Multiple complete responses and cures were also noted when EC0225 was used to treat mice initially bearing tumors as large as 750 mm (3) in volume. Overall, EC0225's impressive preclinical activity allowed for its selection as a development candidate and for the start of Phase 1 clinical trials, which began in March of 2007, for the treatment of advanced malignancies.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Animals
  • Antineoplastic Agents / chemistry*
  • Antineoplastic Agents / pharmacology*
  • Antineoplastic Agents / toxicity
  • Cell Line, Tumor
  • Disease Models, Animal
  • Drug Evaluation, Preclinical*
  • Female
  • Folic Acid / chemistry
  • Folic Acid / metabolism*
  • Folic Acid / pharmacology
  • Folic Acid / toxicity
  • Humans
  • Mice
  • Mice, Nude
  • Molecular Structure
  • Neoplasms / metabolism*
  • Neoplasms / pathology
  • Substrate Specificity
  • Xenograft Model Antitumor Assays

Substances

  • Antineoplastic Agents
  • EC0225
  • Folic Acid