Development of Replicons as Brain Tumor Therapies

DESCRIPTION (PROVIDED BY APPLICANT): Reports from the US indicate that more than 8 million people in this country are currently battling or have had cancer. Replicon Technologies, Incorporated (RTI) has developed a unique gene therapy strategy based on RNA vectors (replicons) with the potential to overcome limitations in the treatment of malignancies of the CNS, including primary cancers of the brain. In the Phase I project, we used a xenogeneic intracranial glioma mouse model to evaluate the feasibility of replicons for the treatment of malignant brain tumors. We have demonstrated that the treatment of tumor-bearing mice with replicons results in a significant enhancement of survival. In the Phase II project, RTI will optimize manufacturing protocols which will be suitable for large-scale production under GMP conditions. Methods for quality control testing wil also be developed. These parameters will be implemented in a pilot feasibility study with a viral vector manufacturing company for small-scale production of GMP-quality replicon preparations that will then be evaluated for safety and toxicity in two appropriate animal species. Data generated from these studies will be used to support an Investigational New Drug (IND) application filing with the FDA to allow for a Phase I clinical study of the replicons. PROPOSED COMMERCIAL APPLICATION: Not Available