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Anti-Tumor Therapy Using Mesenchymal Stem Cells as Targeting Vehicles for Suicide Genes Into Brain Tumors_Ajou Univ.
Korea Health Industry Development Institute (KHIDI) South Korea flag South Korea
Abstract ID:
A developed novel use of mesenchymal stem cells (MSCs) genetically modified to express a suicide gene for treating cancer
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Executive Summary

Dr. Haeyoung Suh, a professor of Ajou University, has developed novel use of mesenchymal stem cells (MSCs) genetically modified to express a suicide gene for treating cancer.

Glioblastoma is one of the most difficult cancers due to its metastasis to other sites and no effective treatments cannot be performed safely in most cases and the chemotherapy with anti-cancer medicines is not effective due to the brain-blood which blocks penetration of drugs into the brain parenchyma.

The Ajou University Industry-Academic Cooperation Foundation, a Technology Licensing Organization in Ajou University, intends to enter into a technology transfer or licensing transaction with regard to use of MSCs  genetically modified to express a suicide gene for treating cancer. Terms of the transaction are not set, and interested parties may further discuss the details if they wish to enter into an agreement.

Industry Sector:

1. Academic/Research: Animal health , 2. Animal health: Pharmaceutical, 3. Biotech: Gene therapy, 4. Diagnostic: Supplies-Biological materials, 5. Drug delivery: Parenteral - implant, 6. Drug discovery: Drug research platforms, 8. Non-profit org./Governmen: Drug Information, 9. Pharmaceutical: Personal Care, 10. Research tools: vectors

Therapeutic Target: 16. Oncology - Brain cancer (Glioblastoma)

Development phase: early stage

Type of business relationship sought (including licensing availability): development collaboration, or non-exclusive or exclusive licensing agreement

Key Technology Highlights

? Excellent Anti-tumor

MSCs (mesenchymal stem cell) expressing CD (cytosine deaminase) gene shows remarkable anti-tumor effects in the presence of a prodrug, 5-fluorocytosine, which were verified in the animal experiments.


? By-Stander Effects

MSCs expressing CD showed strong tropism toward tumors and exert significant bystander effects, so that specifically induced apoptosis in tumor cells. 


? Safety for Cyto/Immune-Toxicity and Biocompatibility

In animal tests, lower biological toxicity or no side effects were observed for animals injected both MSCs expressing suicide gene and a  produg 5-FC.


? Plentiful Animal Data

Many animal data for anti-tumor activities of the present technology have been already obtained, permitting the speed of the product development to increase.

Technology Overview

Technology Platform: The core technology of Ajou University is to provide promising MSCs expressing a suicide gene that show excellent and highly selective anticancer effects against cancer tissues through the selective conversion of a prodrug of an anticancer agent to the anticancer agent around the cancer.

Background and unmet needs: Gene therapy for treatment of glioblastoma by introducing viruses engineered to carry an anti-cancer gene directly into the tumor cells is not effective for highly metastatic cancers such as glioblastomas. Due to strong tropism to tumor cells, MSCs can serve as efficient vehicles for delivering suicide genes to cancers. Suicidal genes are capable of converting non-toxic prodrugs to corresponding anti-cancer drugs. Cytosine deaminse (CD) can convert 5-fluorocytosine (5-FC) to a cytotoxic anticancer agent, 5-fluorouracil (5-FU). The direct administration of 5-FU causes cytotoxicity and leads to adverse side effects, but the combined use of CD, and 5-FC in a specific way makes it possible to generate 5-FU selectively around tumor cells, which is called "bystander effect". Accordingly, it is necessary for anti-tumor agents without no side effect, immune rejection and cytotoxicity.

Discovery and Achievements: The present researchers have prepared a safe and effective anticancer agent using MSCs; and have found that a brain tumor can be efficiency cured when MSCs expressing a suicide gene are transplanted into the brain of a brain cancer animal model, followed by administering a prodrug of an anticancer agent.

Last Updated Sep 2013
Technology Type THERAPEUTIC
Phase of Development EARLY STAGE

Opportunity Contact

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