Effective brain cancer therapies remain a critically unmet need in human healthcare. Advances in surgical, radiation and chemotherapeutic treatments have done little to change patients’ outcomes in the past 30 years. Clearly, new approaches are needed if we are to control these diseases. Many groups worldwide have been exploring the use of viruses that can selectively replicate in and kill tumor cells without harming normal cells; these so called “Oncolytic Viruses” (OV) have shown some remarkable results in late-stage clinical trials.  

Despite this clinical success most OVs are inherently neurotoxic and are not safe to use with brain cancer. Recently we have discovered a new OV variant (a Chimeric Maraba virus) that is non-neurotoxic when injected directly into the brain of mice and that is also effective in a rodent model of brain cancer in vivo and human glioblastoma tumor cells in vitro.  

A current impediment to further clinical development of this virus therapy is the manufacturing capability to supply this virus at a scale and quality suitable for requisite small rodent and non-human primate safety/toxicity studies, and to provide the eventual clinical stock that will be used in our upcoming phase I clinical trial.  

This Alliance for Cancer Gene Therapy funded grant will aid in the design and validation of a GMP-manufacturing process for this virus, and will provide the resources necessary to generate, characterize and validate the virus lots needed to supply the in vivo toxicity studies in support of our clinical trial application, and to conduct our clinical trial.  

Brain cancer continues to be an unmet clinical need requiring novel treatments that are not merely palliative (surgery, radiation and chemotherapy) but potentially curative. A potent OV that can safely target the brain can potentially treat primary, metastatic and recurrent brain cancer improving quality of life and extending survival rates for patients suffering from this deadly disease.   

This Alliance for Cancer Gene Therapy Research Fellow is funded in part by Swim Across America.