The melanosomal protein PMEL17 as a target for antibody drug conjugate therapy in melanoma

J Biol Chem. 2012 Jul 13;287(29):24082-91. doi: 10.1074/jbc.M112.361485. Epub 2012 May 21.

Abstract

Melanocytes uniquely express specialized genes required for pigment formation, some of which are maintained following their transformation to melanoma. Here we exploit this property to selectively target melanoma with an antibody drug conjugate (ADC) specific to PMEL17, the product of the SILV pigment-forming gene. We describe new PMEL17 antibodies that detect the endogenous protein. These antibodies help define the secretory fate of PMEL17 and demonstrate its utility as an ADC target. Although newly synthesized PMEL17 is ultimately routed to the melanosome, we find substantial amounts accessible to our antibodies at the cell surface that undergo internalization and routing to a LAMP1-enriched, lysosome-related organelle. Accordingly, an ADC reactive with PMEL17 exhibits target-dependent tumor cell killing in vitro and in vivo.

MeSH terms

  • Animals
  • Antibodies / chemistry
  • Antibodies / therapeutic use*
  • Cell Line
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Flow Cytometry
  • Humans
  • Immunohistochemistry
  • In Vitro Techniques
  • Melanocytes / metabolism*
  • Melanoma / drug therapy*
  • Melanosomes / metabolism*
  • Mice
  • Mice, Inbred BALB C
  • Microscopy, Confocal
  • Microscopy, Fluorescence
  • Oligopeptides / chemistry
  • Oligopeptides / therapeutic use
  • Xenograft Model Antitumor Assays
  • gp100 Melanoma Antigen / genetics
  • gp100 Melanoma Antigen / metabolism*

Substances

  • Antibodies
  • Oligopeptides
  • PMEL protein, human
  • gp100 Melanoma Antigen
  • monomethyl auristatin E