Synthesis and enzymatic activation of N-[N(alpha)-(4-amino-4-deoxypteroyl)-N(delta)-hemiphthaloyl-L-ornithiny]-L-phenylalanine, a candidate for antibody-directed enzyme prodrug therapy (ADEPT)

Bioorg Med Chem. 2002 Mar;10(3):493-500. doi: 10.1016/s0968-0896(01)00298-x.

Abstract

N-[N(alpha)-(4-amino-4-deoxypteroyl)-N(delta)-hemiphthaloyl-L-ornithinyl]-L-phenylalanine (1), a carboxypeptidase A (CPA) cleavable prodrug was synthesized for use in an antibody directed strategy to improve the therapeutic selectivity of N(alpha)-(4-amino-4-deoxypteroyl)-N(delta)-hemiphthaloyl-L-ornithine (2), an extremely potent nonpoly-glutamatable DHFR inhibitor which is also highly cytotoxic. Compound 1 was shown by HPLC analysis to give a >99% yield of 2 upon incubation with bovine CPA (bCPA) for 20 min at 25 degrees C. In a spectrophotometric kinetic assay with 50 microM dihydrofolate as the competing substrate in the presence of 65 microM NADPH, 1+bCPA stoichiometrically inhibited recombinant human DHFR (rhDHFR) with a K(i) of 0.35 pM. In contrast, 1 without bCPA was a poor inhibitor of rhDHFR (K(i)>10 microM). In a 72 h growth inhibition assay against cultured CCRF-CEM human leukemic lymphoblasts, the growth inhibitory activities of 1+bCPA, 2+bCPA, and 2 alone were the same (IC(50) 1.3-1.4 nM), whereas 1 in the absence of bCPA was >100-fold less potent (IC(50) 155 nM).

Publication types

  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Alcohol Oxidoreductases / antagonists & inhibitors
  • Animals
  • Antineoplastic Agents / chemical synthesis
  • Antineoplastic Agents / metabolism
  • Antineoplastic Agents / pharmacology
  • Carboxypeptidases / metabolism*
  • Carboxypeptidases A
  • Cattle
  • Cell Division / drug effects
  • Dipeptides / chemical synthesis*
  • Dipeptides / metabolism
  • Dipeptides / pharmacology
  • Enzyme Inhibitors / chemical synthesis
  • Enzyme Inhibitors / metabolism
  • Enzyme Inhibitors / pharmacology
  • Humans
  • Inhibitory Concentration 50
  • Prodrugs / chemical synthesis*
  • Prodrugs / metabolism
  • Prodrugs / pharmacology
  • Structure-Activity Relationship
  • Tumor Cells, Cultured / drug effects

Substances

  • Antineoplastic Agents
  • Dipeptides
  • Enzyme Inhibitors
  • N-(N-(4-amino-4-deoxypteroyl)-N-hemiphthaloyl-ornithiny)-phenylalanine
  • Prodrugs
  • Alcohol Oxidoreductases
  • dihydroflavanol 4-reductase
  • Carboxypeptidases
  • Carboxypeptidases A