Melanocyte destruction after antigen-specific immunotherapy of melanoma: direct evidence of t cell-mediated vitiligo

J Exp Med. 2000 Dec 4;192(11):1637-44. doi: 10.1084/jem.192.11.1637.

Abstract

Current strategies for the immunotherapy of melanoma include augmentation of the immune response to tumor antigens represented by melanosomal proteins such as tyrosinase, gp100, and MART-1. The possibility that intentional targeting of tumor antigens representing normal proteins can result in autoimmune toxicity has been postulated but never demonstrated previously in humans. In this study, we describe a patient with metastatic melanoma who developed inflammatory lesions circumscribing pigmented areas of skin after an infusion of MART-1-specific CD8(+) T cell clones. Analysis of the infiltrating lymphocytes in skin and tumor biopsies using T cell-specific peptide-major histocompatibility complex tetramers demonstrated a localized predominance of MART-1-specific CD8(+) T cells (>28% of all CD8 T cells) that was identical to the infused clones (as confirmed by sequencing of the complementarity-determining region 3). In contrast to skin biopsies obtained from the patient before T cell infusion, postinfusion biopsies demonstrated loss of MART-1 expression, evidence of melanocyte damage, and the complete absence of melanocytes in affected regions of the skin. This study provides, for the first time, direct evidence in humans that antigen-specific immunotherapy can target not only antigen-positive tumor cells in vivo but also normal tissues expressing the shared tumor antigen.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Antigens, Neoplasm / biosynthesis
  • Antigens, Neoplasm / immunology*
  • Female
  • Humans
  • Immunotherapy, Adoptive / adverse effects*
  • Immunotherapy, Adoptive / methods
  • MART-1 Antigen
  • Melanocytes / cytology
  • Melanocytes / immunology*
  • Melanoma / complications
  • Melanoma / immunology
  • Melanoma / pathology
  • Melanoma / therapy*
  • Middle Aged
  • Neoplasm Proteins / biosynthesis
  • Neoplasm Proteins / immunology*
  • Skin / cytology
  • Skin / immunology
  • Skin / pathology
  • Skin Neoplasms / complications
  • Skin Neoplasms / immunology
  • Skin Neoplasms / pathology
  • Skin Neoplasms / therapy*
  • T-Lymphocytes, Cytotoxic / classification
  • T-Lymphocytes, Cytotoxic / immunology
  • Vitiligo / etiology
  • Vitiligo / immunology*
  • Vitiligo / pathology

Substances

  • Antigens, Neoplasm
  • MART-1 Antigen
  • MLANA protein, human
  • Neoplasm Proteins