A closed culture system for the ex vivo transduction and expansion of human T lymphocytes

J Hematother. 1998 Jun;7(3):205-15. doi: 10.1089/scd.1.1998.7.205.

Abstract

A phase I clinical trial is currently being performed at our institution, with the aim of evaluating the feasibility and toxicity related to the administration of herpes simplex thymidine kinase gene-expressing human primary T lymphocytes following allogeneic hematopoietic stem cell transplantation. The need for safe and standardized preparation conditions for gene-modified cells is crucial. We describe the closed culture system used in the current trial for ex vivo retroviral-mediated gene transfer and transduced cell selection. Cell handling is performed in closed systems using a sterile connection device that avoids opening the culture system. Cell numbers during the production process increased from 93 +/- 16 on day 0 to 440 +/- 92 x 10(6) on day 12 (7.2 +/- 1.4-fold increase) (n = 11). Transduction efficiency before and after G418 resistance-based selection was 13.5 +/- 3.8% and 90.0 +/- 1.4%, respectively. Safety and efficacy testing included a search for replication-competent retrovirus, endotoxins, Mycoplasma, and bacterial contamination (n = 0/9), PCR-DNA, % CD3+ cells (91 +/- 2%), and viability after thawing (82 +/- 3%). Effective working time from day 0 to day 12 is approximately 20 h. The closed system we developed allows for safe and reproducible ex vivo preparation of gene-modified primary T lymphocytes for clinical use.

Publication types

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

MeSH terms

  • Cell Culture Techniques / methods
  • Cell Survival / drug effects
  • Clone Cells
  • Feasibility Studies
  • Ganciclovir / toxicity*
  • Gene Transfer Techniques
  • Genetic Therapy* / standards
  • Graft vs Host Disease / prevention & control
  • Hematopoietic Stem Cell Transplantation* / adverse effects
  • Hematopoietic Stem Cell Transplantation* / standards
  • Humans
  • Lymphocyte Transfusion* / standards
  • Polymerase Chain Reaction
  • Quality Control
  • Simplexvirus / enzymology*
  • T-Lymphocytes / cytology*
  • T-Lymphocytes / drug effects
  • T-Lymphocytes / enzymology
  • Thymidine Kinase / biosynthesis*
  • Thymidine Kinase / genetics
  • Transplantation, Autologous
  • Transplantation, Homologous

Substances

  • Thymidine Kinase
  • Ganciclovir