DUPA conjugation of a cytotoxic indenoisoquinoline topoisomerase I inhibitor for selective prostate cancer cell targeting

J Med Chem. 2015 Apr 9;58(7):3094-103. doi: 10.1021/jm5018384. Epub 2015 Mar 30.

Abstract

Prostate-specific membrane antigen (PSMA) is overexpressed in most prostate cancer cells while being present at low or undetectable levels in normal cells. This difference provides an opportunity to selectively deliver cytotoxic drugs to prostate cancer cells while sparing normal cells that lack PSMA, thus improving potencies and reducing toxicities. PSMA has high affinity for 2-[3-(1,3-dicarboxypropyl)ureido]pentanedioic acid (DUPA) (Ki = 8 nM). After binding to a DUPA-drug conjugate, PSMA internalizes, unloads the conjugate, and returns to the surface. In the present studies, an indenoisoquinoline topoisomerase I inhibitor was conjugated to DUPA via a peptide linker and a drug-release segment that facilitates intracellular cleavage to liberate the drug cargo. The DUPA-indenoisoquinoline conjugate exhibited an IC50 in the low nanomolar range in 22RV1 cell cultures and induced a complete cessation of tumor growth with no toxicity, as determined by loss of body weight and death of treated mice.

Publication types

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

MeSH terms

  • Animals
  • Antineoplastic Agents / chemical synthesis
  • Antineoplastic Agents / chemistry*
  • Antineoplastic Agents / pharmacology*
  • Chemistry Techniques, Synthetic
  • Drug Design
  • Glutarates / chemistry*
  • Humans
  • Inhibitory Concentration 50
  • Isoquinolines / chemistry*
  • Kallikreins / metabolism*
  • Male
  • Mice, Nude
  • Molecular Targeted Therapy
  • Prostate-Specific Antigen / metabolism*
  • Prostatic Neoplasms / drug therapy*
  • Prostatic Neoplasms / metabolism
  • Topoisomerase I Inhibitors / chemistry*
  • Topoisomerase I Inhibitors / pharmacology*
  • Urea / analogs & derivatives*
  • Urea / chemistry
  • Xenograft Model Antitumor Assays

Substances

  • 2-(3-(1,3-dicarboxypropyl)ureido)pentanedioic acid
  • Antineoplastic Agents
  • Glutarates
  • Isoquinolines
  • Topoisomerase I Inhibitors
  • Urea
  • KLK3 protein, human
  • Kallikreins
  • Prostate-Specific Antigen