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Prodrugs and prodrug-activated systems in gene therapy

Semira Sheikh1, Daniel Ernst2, Armand Keating3

  • 1Princess Margaret Cancer Centre, Toronto, ON, Canada; University of Toronto, Toronto, ON, Canada.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|April 8, 2021
PubMed

Insights

Gene therapy uses suicide genes to eliminate unwanted cells. This review examines prodrug systems, highlighting their limitations and potential for future cell therapies.

Area of Science:

  • Molecular Biology
  • Gene Therapy
  • Pharmacology

Background:

  • Gene therapy vectors can incorporate suicide genes for selective elimination of aberrant transfected cells.
  • Prodrug-activated systems, developed in the late 20th century, enable cell death via enzymatic conversion of prodrugs to active metabolites.

Purpose of the Study:

  • To review prodrug systems utilized in clinical gene therapy trials.
  • To analyze the efficacy and adverse effects of these prodrug systems.
  • To identify limitations and inform future advancements in cell therapies.

Main Methods:

  • Literature review of prodrug systems employed in clinical gene therapy trials.
  • Analysis of published data on prodrug efficacy, toxicity, biodistribution, and bystander effects.

Main Results:

  • Considerable effort has focused on vector design, with less attention paid to prodrug system efficacy and safety.
  • Potential drawbacks include systemic toxicity of activated compounds, limited biodistribution data, bystander effects, and destruction of genetically modified cells.

Conclusions:

  • Prodrug systems are crucial for the safety and efficacy of gene therapy but require further investigation.
  • Addressing identified drawbacks is essential for optimizing cell therapies and advancing the field of gene therapy.

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