Stem cells-derived suicide gene exosomes: a promising platform for innovative cancer therapy

Dajana Vanova1,2, Michal Andrezal2, Ursula Altanerova2

  • 1Cancer Research Institute, Biomedical Research Center of the Slovak Academy of Sciences, Bratislava, Slovakia.

Neoplasma
|December 18, 2025
PubMed

Insights

Suicide gene exosomes, engineered from modified stem cells, offer a novel cancer therapy. These exosomes deliver therapeutic genes to tumors, converting prodrugs into cancer-killing agents for improved treatment outcomes.

Area of Science:

  • Oncology
  • Biotechnology
  • Nanomedicine

Background:

  • Metastatic tumors often lack effective treatment options.
  • Small extracellular vesicles, particularly exosomes, are emerging as novel drug delivery vehicles.
  • Current cancer therapies require innovative approaches for improved efficacy.

Purpose of the Study:

  • To review recent advancements in suicide gene exosome therapy for cancer.
  • To explore the potential of engineered exosomes as intracellular anti-cancer drugs.
  • To highlight therapeutic outcomes and future directions for exosome-based cancer treatments.

Main Methods:

  • Production of suicide gene exosomes from genetically modified human mesenchymal stem cells (MSCs) expressing yeast cytosine deaminase::uracil phosphoribosyl transferase (yCD::UPRT).
  • Utilizing tumor-targeting exosomes to convert the prodrug 5-fluorocytosine (5-FC) into the cytotoxic compound 5-fluorouracil (5-FU) within the tumor microenvironment.
  • Investigating suicide gene exosomes derived from Herpes Simplex Virus thymidine kinase and ganciclovir.

Main Results:

  • Engineered exosomes effectively convert prodrugs into cytotoxic agents specifically within tumor cells.
  • Combined therapy using suicide gene exosomes from MSCs and cancer-associated fibroblasts (CAFs) shows promise for aggressive tumors.
  • In vitro and in vivo studies demonstrate therapeutic potential of suicide gene exosomes.

Conclusions:

  • Suicide gene exosomes represent a promising platform for intracellular anti-cancer drug delivery.
  • Exosome-based therapies can be enhanced with additional drugs and customized for targeted delivery.
  • Further research into therapeutically beneficial exosomes holds significant potential for future cancer treatment strategies.

Related Concept Videos

Cancer Stem Cells and Tumor Maintenance02:40

Cancer Stem Cells and Tumor Maintenance

Early diagnosis and treatment can often cure cancer. However, even with treatment, residual cells called cancer stem cells (CSC) might remain, often causing tumor recurrence. These cancer stem cells possess the potential for self-renewal and multi-lineage differentiation and are often responsible for the therapeutic resistance displayed in most cancers.
Cancer stem cells are thought to originate from tissue-specific normal stem cells or progenitor cells. The normal stem cells usually reside in...
5.8K
Stem Cell Therapy for Tissue Regeneration01:21

Stem Cell Therapy for Tissue Regeneration

Stem cell therapy is a method used in regenerative medicine to repair and restore function to damaged tissues and organs. Stem cells have the potential to proliferate and differentiate into various tissue types, making them ideal candidates for tissue regeneration. For example, hematopoietic stem cell transplants are commonly used in blood cancer treatment to replenish damaged bone marrow and restore healthy blood cells.
Types of Stem Cells used in Stem Cell Therapy
The two main cell...
4.6K
Overview of Exosomes01:36

Overview of Exosomes

Exosomes are stable, lipid bilayer-enclosed vesicles capable of crossing biological barriers. They can carry a wide range of molecules required for intercellular communication. Once exosomes are released from the cell where they originated, they enter a recipient cell through various pathways such as fusion, receptor-mediated endocytosis, macropinocytosis, and phagocytosis.
Stahl et al. discovered exosomes in 1983, but the exosomes were initially considered waste products released from the...
3.4K
Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
27.3K
Induced Pluripotent Stem Cells01:06

Induced Pluripotent Stem Cells

Stem cells are undifferentiated cells that divide and produce different cell types. Ordinarily, cells that have differentiated into a specific cell type are terminally differentiated; however, scientists have found a way to reprogram these mature cells so that they dedifferentiate and return to an unspecialized, proliferative state. These cells are pluripotent like embryonic stem cells—able to produce all cell types—and are called induced pluripotent stem cells (iPSCs).
Somatic...
5.3K