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:
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 an
Send an Inquiry

1. Applications

• This therapeutic agent is originally designed to treat the remaining tumor masses after surgery in firstly diagnosed brain tumor patients.

• This therapeutic agent can be effective to treat recurrent patients who received a standard treatment [surgery, radiotherapy, and chemotherapy of temozolomide].

• This therapeutics can be utilized after removal of cancer mass by surgery in order to ensures the cell death of remaining micromass of cancers in other types of organs.

2. Market Feasibility

• This therapeutic agent can be given to patients while waiting for recovery from surgery, thus does not compete with standard therapy or treatment which are already in market.

• MSCs have strong tropism toward cancer cells and can serve as cellular vehicles to deliver therapeutic gene (CD) near to tumor sites.

• Approximately 20,000 new cases of primary brain tumors are diagnosed per in US. This therapeutics can be applied to treatment other types of cancers following surgery. Thus, more patients will get benefit from this therapeutic agent

• The master cell bank (MCB) of this therapeutic agent was already produced in a GMP-compliant facility.

• This therapeutic agent will be filed to Korean FDA in 2 years, after performing toxicity/safety tests in GLP laboratories.

• Large quantity of clinical lots will be produced by simple amplification of MCB since CD gene is integrated to MSC host genome. Consequently the cost of quality control (QC) of clinical lot is relatively easy.

• This therapeutic agent will be formulated for multi-center clinical trials.

3. Technical Advantages

• Compared to other companies which use replicating virus (Toca 511, Tocagen, CA, USA) or neural stem cells (City of Hope Hospital, CA, USA), the efficacy of this therapeutic agent is higher with 4-8 fold lower IC50 value for 5-FC MSCs have strong tropism toward cancers.

• Unlike replicating virus, MSC/CD cells undergo cell death via suicide effects after delivery of CD gene near cancer sites, which ensures the safety of this therapeutic agent and minimizes immune responses.

• Despite that it is allogeneic cells, this therapeutic agent can be repetitively utilized to patients without immune rejection, since MSCs have intrinsic immune suppressive functions. This agent also will benefit to alleviation of local brain edema after brain surgery.

• Additionally 5-FC is an antimycotic drug which will promote of healing after the surgery

• Since simple amplification ensures quality controlled production of large quantity, the cost of this agent will be competitive compared to other stem cell- and gene-based therapy.

4. Technical Highlighted Summary

• Clinical lot of MSC/CD is being produced by simple amplification of MCB that was already established. After performing safety/toxicity tests in GLP institutes, this therapeutic agent will be filed to Korean FDA for IND in 2 years.

• Figure 1. Anti-cancer effects in the brain. U87 glioma cells were modified to express reporter genes such as β-galactosidase (LacZ) or green fluorescence protein (GFP) and then stereotaxically transplanted into brain parenchyma. Next day MSC/CD was transplanted and 5-FC were systemically given for 5 days. Animals were sacrificed at indicated time and subject to histological analysis. Tumor volume was markedly reduced in animals treated with MSC/CD compared to the vehicle control (PBS).

Last Updated Jun 2016
Technology Type THERAPEUTIC
Phase of Development PRECLINICAL

Opportunity Contact