A dual-action therapy that may eliminate existing tumors and prevent future tumors from occurring has been developed, according to new research. Targeting glioblastoma, an aggressive and deadly form of brain cancer, the therapy is designed to stop reoccurrence in mouse models, marking an important milestone in both treatment and prevention. 

“Our team has pursued a simple idea: to take cancer cells and transform them into cancer killers and vaccines,” said corresponding author Khalid Shah in a statement. 

“Using gene engineering, we are repurposing cancer cells to develop a therapeutic that kills tumor cells and stimulates the immune system to both destroy primary tumors and prevent cancer.” 

The treatment is a simple idea – but, as with all science plans, not as easy in practice. Most cancer vaccines use inactivated tumor cells, much like a virus vaccine uses inactivated virus particles, to help the immune system identify any living tumors and destroy them. However, this research planned to transform living tumor cells into anti-cancer agents that will both destroy active tumor cells and allow the immune system to memorize how to combat them next time. To do so, Shah and a team of scientists looked towards CRISPR-Cas9 genetic editing.  

The living tumor cells are to be repurposed to release immunomodulators, which help the immune system destroy brain tumor cells, while also expressing proteins on their surface that make them very obvious to immune cells. This helps them remember how to destroy similar cells next time. 

What makes this approach so promising is that these living tumor cells, as opposed to inactivated cells, can home in on other tumor cells within the brain, taking the anti-cancer agents directly to the target. 

Once packaged into a vaccine, the team delivered it to mouse models with immune systems that mimic a human’s, making the results as close to human trials as possible without actually using humans. The therapy proved to be safe and effective in the models, providing a long-lasting immune response. 

According to Shah, this approach is not specific to glioblastoma, but to other solid tumors too.  

“Our goal is to take an innovative but translatable approach so that we can develop a therapeutic, cancer-killing vaccine that ultimately will have a lasting impact in medicine," Shah continued.

They now believe there is a possible route to human trials once further safety trials can be conducted. 

The research was published in the journal Science Translational Medicine.