Latent Warheads for Targeted Cancer Therapy: Design and Synthesis of pro-Pyrrolobenzodiazepines and Conjugates

Bioconjug Chem. 2017 Dec 20;28(12):2921-2931. doi: 10.1021/acs.bioconjchem.7b00476. Epub 2017 Dec 6.

Abstract

Pyrrolobenzodiazepines (PBDs) and their dimers (bis-PBDs) have emerged as some of the most potent chemotherapeutic compounds, and are currently under development as novel payloads in antibody-drug conjugates (ADCs). However, when used as stand-alone therapeutics or as warheads for small molecule drug conjugates (SMDCs), dose-limiting toxicities are often observed. As an elegant solution to this inherent problem, we designed diazepine-ring-opened conjugated prodrugs lacking the imine moiety. Once the prodrug (pro-PBD) conjugate enters a targeted cell, cleavage of the linker system triggers the generation of a reactive intermediate possessing an aldehyde and aromatic amine. An intramolecular ring-closing reaction subsequently takes place as the aromatic amine adds to the aldehyde with the loss of water to give the imine and, as a result, the diazepine ring. In our pro-PBDs, we mask the aldehyde as a hydrolytically sensitive oxazolidine moiety which in turn is a part of a reductively labile self-immolative linker system. To prove the range of applications for this new class of latent DNA-alkylators, we designed and synthesized several novel latent warheads: pro-PBD dimers and hybrids of pro-PBD with other sequence-selective DNA minor groove binders. Preliminary preclinical pharmacology studies showed excellent biological activity and specificity.

MeSH terms

  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / metabolism
  • Antineoplastic Agents / pharmacology
  • Antineoplastic Agents / therapeutic use
  • Benzodiazepines / chemistry*
  • Benzodiazepines / metabolism*
  • Benzodiazepines / pharmacology
  • Benzodiazepines / therapeutic use
  • Chemistry Techniques, Synthetic
  • Drug Design*
  • Humans
  • KB Cells
  • Molecular Targeted Therapy*
  • Neoplasms / drug therapy*
  • Neoplasms / pathology
  • Prodrugs / chemical synthesis*
  • Prodrugs / chemistry
  • Prodrugs / metabolism*
  • Pyrroles / chemistry*
  • Pyrroles / metabolism*
  • Pyrroles / pharmacology
  • Pyrroles / therapeutic use

Substances

  • Antineoplastic Agents
  • Prodrugs
  • Pyrroles
  • pyrrolo(2,1-c)(1,4)benzodiazepine
  • Benzodiazepines