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In a groundbreaking development, scientists have developed an mRNA vaccine that has shown efficacy in targeting glioblastoma, an aggressive form of brain cancer, in a small human clinical trial.

Conducted at the University of Florida, the trial involved four patients and demonstrated promising results that could revolutionize the treatment of this challenging cancer, which affects approximately 19,000 individuals annually in the EU.

Traditional treatments for glioblastoma, such as chemotherapy, radiotherapy, and surgery, have seen limited success, with patients facing a grim average survival duration of about 15 months.

Published in the peer-reviewed journal Cell, the study describes the mRNA vaccine’s innovative approach, utilizing the patient’s own tumor cells to create a personalized treatment. This vaccine, similar to those used in COVID-19 vaccines but with notable modifications, employs a newly developed delivery system to trigger a rapid immune response.

Lead author Elias Sayour explains that the vaccine utilizes clusters of particles wrapped around each other, enhancing immune system activation compared to single particles. By harnessing the patient’s immune system, the vaccine induces a shift in the tumor microenvironment from “cold” to “hot,” indicating a robust immune response against the cancer cells.

The study builds upon seven years of research, which initially involved experiments in preclinical mouse models and progressed to a clinical trial involving ten pet dogs with advanced brain cancer. Notably, the median survival of these dogs exceeded expectations, with a median survival of 139 days compared to the typical 30 to 60 days.

Professor Duane Mitchell emphasizes the significance of these results, highlighting the vaccine’s ability to generate rapid and significant immune responses across animals and humans. A Phase 1 clinical trial involving up to 24 adult and pediatric patients is planned to validate these findings and explore potential side effects.

While further research is warranted to optimize immune system activation and minimize adverse effects, the mRNA vaccine offers hope for a breakthrough in the treatment of glioblastoma, potentially improving outcomes for patients facing this devastating diagnosis.

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