Protacs for treatment of cancer

Pediatr Res. 2010 May;67(5):505-8. doi: 10.1203/PDR.0b013e3181d35017.

Abstract

Protein degradation is the cell's mechanism of eliminating misfolded or unwanted proteins. The pathway by which proteins are degraded occurs through the ubiquitin-proteasome system. Ubiquitin is a small 9-kD (kDa) protein that is attached to proteins. A minimum of four ubiquitins are required for proteins to be recognized by the degradation machinery, known as the 26S proteasome. Defects in ubiquitination have been identified in a number of diseases, including cancer, neurodegenerative diseases, and metabolic disorders. We sought to exploit the delicate balance between protein synthesis and degradation to treat cancer by designing a chimeric molecule, known as Protac (Proteolysis Targeting Chimeric molecule). Protacs are heterobifunctional nanomolecules that are approximately 10 nm in size and can recruit proteins that cause cancer to the ubiquitin-proteasome machinery for degradation. In this review, we discuss the development of this novel technology for the treatment of cancer.

Publication types

  • Review

MeSH terms

  • Animals
  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / pharmacology*
  • Drug Design*
  • Humans
  • Nanomedicine*
  • Nanoparticles*
  • Nanotechnology*
  • Neoplasm Proteins / metabolism*
  • Neoplasms / drug therapy*
  • Neoplasms / metabolism
  • Neoplasms / pathology
  • Proteasome Endopeptidase Complex / metabolism
  • Protein Processing, Post-Translational
  • Ubiquitination

Substances

  • Antineoplastic Agents
  • Neoplasm Proteins
  • Proteasome Endopeptidase Complex