The rapidly expanding class of RNA-based drugs are breaking therapeutic barriers by making personalized medicine a reality. These promising drugs are cost-effective, easy to manufacture and have the potential to change the standard of care for many diseases. However, they have not been useful in getting through to the well-protected brain to treat tumors or other maladies—until now.
Researchers at the Georgia Institute of Technology in collaboration with Emory University, have developed a method to deliver RNA-loaded nanoparticles to the brain. To get through the protective blood-brain barrier, the multi-institutional team developed a technique using ultrasound to deliver precious payloads. In the study, researchers packaged siRNA, a drug that can block the expression of genes that drive tumor growth, in lipid-polymer hybrid nanoparticles, and combined that with the focused ultrasound technique in pediatric and adult preclinical brain cancer models. Using single-cell image analysis, the team demonstrated a more than 10-fold improvement in delivery of the drug, reducing harmful protein production and increasing tumor cell death in preclinical models of medulloblastoma, the most common malignant brain tumor in children. The study was published in the journal Science Advances.
'"This is completely tunable," said Costas Arvanitis, assistant professor at Georgia Tech. "We can fine tune the ultrasound pressure to attain a desired level of vibration and by extension drug delivery. It's non-invasive, because we are applying sound from outside the brain, and it's very localized, because we can focus the ultrasound to a very small region of the brain."'
Current standard treatments for brain tumors come with potentially awful side effects, Arvanitis said, "however, this technology can provide treatment with minimal side effects, which is very exciting. Now, we are moving forward to try and identify what components are missing to translate this technology to the clinic."’
Photo: Georgia Tech Mechanical Engineering PhD student Yutong Guo (left) and her mentor, Assistant Professor Costas Arvanitis, have developed a way to use ultrasonics to treat brain disease. Credit: Ashley Ritchey, Georgia Tech