Cationic liposome-mediated E1A gene transfer to human breast and ovarian cancer cells and its biologic effects: a phase I clinical trial

J Clin Oncol. 2001 Jul 15;19(14):3422-33. doi: 10.1200/JCO.2001.19.14.3422.

Abstract

Purpose: Preclinical studies have demonstrated that the adenovirus type 5 E1A gene is associated with antitumor activities by transcriptional repression of HER-2/neu and induction of apoptosis. Indeed, E1A gene therapy is known to induce regression of HER-2/neu-overexpressing breast and ovarian cancers in nude mice. Therefore, we evaluated the feasibility of intracavitary injection of E1A gene complexed with DC-Chol cationic liposome (DCC-E1A) in patients with both HER-2/neu-overexpressing and low HER-2/neu-expressing breast and ovarian cancers in a phase I clinical trial.

Patients and methods: An E1A gene complexed with DCC-E1A cationic liposome was injected once a week into the thoracic or peritoneal cavity of 18 patients with advanced cancer of the breast (n = 6) or ovary (n = 12).

Results: E1A gene expression in tumor cells was detected by immunohistochemical staining and reverse transcriptase-polymerase chain reaction. This E1A gene expression was accompanied by HER-2/neu downregulation, increased apoptosis, and reduced proliferation. The most common treatment-related toxicities were fever, nausea, vomiting, and/or discomfort at the injection sites.

Conclusion: These results argue for the feasibility of intracavitary DCC-E1A administration, provide a clear proof of preclinical concept, and warrant phase II trials to determine the antitumor activity of the E1A gene.

Publication types

  • Clinical Trial
  • Clinical Trial, Phase I
  • Research Support, U.S. Gov't, Non-P.H.S.
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adenovirus E1A Proteins / genetics*
  • Adult
  • Aged
  • Apoptosis
  • Breast Neoplasms / genetics
  • Breast Neoplasms / metabolism
  • Breast Neoplasms / pathology
  • Breast Neoplasms / therapy*
  • Cholesterol / analogs & derivatives
  • Cytokines / metabolism
  • Female
  • Gene Expression
  • Gene Transfer, Horizontal*
  • Genes, erbB-2
  • Genetic Therapy*
  • Humans
  • Immunohistochemistry
  • In Situ Nick-End Labeling
  • Injections
  • Ki-67 Antigen
  • Liposomes
  • Middle Aged
  • Ovarian Neoplasms / genetics
  • Ovarian Neoplasms / metabolism
  • Ovarian Neoplasms / pathology
  • Ovarian Neoplasms / therapy*
  • Peritoneal Cavity
  • Reverse Transcriptase Polymerase Chain Reaction
  • Thorax
  • Tumor Cells, Cultured

Substances

  • Adenovirus E1A Proteins
  • Cytokines
  • Ki-67 Antigen
  • Liposomes
  • 3-(N-(N',N'-dimethylaminoethane)carbamoyl)cholesterol
  • Cholesterol