Therapeutic strategies for locally recurrent and metastatic de-differentiated liposarcoma with herpes simplex virus-thymidine kinase-expressing mesenchymal stromal cells

Cytotherapy. 2017 Sep;19(9):1035-1047. doi: 10.1016/j.jcyt.2017.05.008. Epub 2017 Jul 29.

Abstract

Background aims: Major challenges in de-differentiated liposarcoma (DDLPS) therapy are the high rate of sequential recurrence (>80%) and metastasis (20-30%) following surgical removal. However, well-defined therapeutic strategies for this rare malignancy are lacking and are critically needed.

Methods: We investigated a new approach to DDLPS therapy with mesenchymal stromal cells expressing herpes simplex virus-thymidine kinase (MSC-TK). In an effort to evaluate this efficacy, in vitro cytotoxicity of MSC-TK against DDLPS cells was analyzed using an apoptosis assay. For pre-clinical study, the MSC-TK-induced reduction in recurrence and metastasis was validated in a recurrent DDLPS model after the macroscopic complete resection and lung metastasis DDLPS model.

Results: MSC-TK induced apoptosis in DDLPS cells by bystander effects via gap junction intracellular communication (GJIC) of toxic ganciclovir (GCV). Recurrent DDLPS models following no residual tumor/microscopic tumor resection and lung metastasis DDLPS models were established, which suggested clinical relevance. MSC-TK markedly reduced locoregional recurrence rates and prolonged recurrence-free survival, thus increasing overall survival in the recurrent DDLPS model. MSC-TK followed by GCV treatment yielded a statistically significant reduction in early- and advanced-stage lung metastasis.

Discussion: This therapeutic strategy may serve as an alternative or additional strategy by applying MSC-TK to target residual tumors following surgical resection, thus reducing local relapse and metastasis in these patients.

Keywords: de-differentiated liposarcoma; herpes simplex virus–thymidine kinase; locoregional recurrence; mesenchymal stromal cells; metastasis.

MeSH terms

  • Administration, Intravenous
  • Animals
  • Apoptosis / drug effects
  • Bystander Effect
  • Cell Communication / drug effects
  • Ganciclovir / pharmacology
  • Genetic Therapy / methods
  • Humans
  • Liposarcoma / pathology
  • Liposarcoma / therapy*
  • Lung Neoplasms / secondary
  • Lung Neoplasms / therapy
  • Mesenchymal Stem Cell Transplantation / methods*
  • Mesenchymal Stem Cells / physiology*
  • Mice
  • Neoplasm Recurrence, Local
  • Simplexvirus / genetics*
  • Thymidine Kinase / genetics*
  • Tumor Cells, Cultured
  • Xenograft Model Antitumor Assays

Substances

  • Thymidine Kinase
  • Ganciclovir