Targeting these master genes with anti-cancer therapy in laboratory tests resulted in significant tumor reduction, indicating that depleting a few key genes could effectively eliminate the cancer.

Dr. David Tran, the principal investigator, highlighted the challenges of personalized treatment for glioblastoma, noting that rapid tumor mutation complicates effective intervention.

Dr. Tran expressed optimism about advancements in glioblastoma treatment over the next five to ten years, contrasting the past grim prognoses with today's potential for improved survival outcomes.

The research team has developed a delivery mechanism for the therapy utilizing a common adeno-associated virus (AAV), screening a library of ten billion AAV variants to specifically target glioblastoma cells without affecting healthy brain tissue.

Among the AAV variants, T6 has emerged as a promising candidate, demonstrating cure rates of 70 to 90 percent in mouse models with human glioblastoma tumors.

The research team utilized AI technology to identify nine 'master regulators' crucial for glioblastoma survival, with seven of these being developmental genes that are typically dormant but reactivated by tumor cells.

Further preclinical testing is planned to validate the outcomes observed in laboratory models, ensuring the therapy's effectiveness before clinical trials.

The grant will enable USC to collaborate with the University of Florida’s Zolotukhin Lab to progress the treatment through clinical trials.

On February 4, 2025, the University of Southern California (USC) received a $6 million grant from the California Institute for Regenerative Medicine to advance a groundbreaking gene therapy aimed at treating glioblastoma, an aggressive brain cancer with a five-year survival rate of only 5%.

This innovative delivery system overcomes the non-specific effects of traditional viral gene therapies, aiming for precision in targeting cancer cells.

Funding from the grant will assist in preparing the new viral delivery system and drug targets for clinical trials, while also refining conduction-enhanced delivery, a surgical method for administering gene therapy.

To enhance treatment efficacy, USC investigators are creating a computational method to map tumor flow patterns, improving catheter placement and drug diffusion during treatment.