Nanoparticle Drug-Delivery System Developed to Aid Treatment of Brain Disorders/Diseases; Use in Mouse Models Results In Unprecedented Penetration of siRNA Across Intact Blood-Brain Barrier; System Could Be “Game-Changer” in Treatment of CNS Diseases

In the past few decades, researchers have identified biological pathways leading to neurodegenerative diseases and developed promising molecular agents to target them. However, the translation of these findings into clinically approved treatments has progressed at a much slower rate, in part because of the challenges scientists face in delivering therapeutics across the blood-brain barrier (BBB) and into the brain. To facilitate successful delivery of therapeutic agents to the brain, a team of bioengineers, physicians, and collaborators at Brigham and Women's Hospital and Boston Children's Hospital created a nanoparticle platform, which can facilitate therapeutically effective delivery of encapsulated agents in mice with a physically breached or intact BBB. In a mouse model of traumatic brain injury (TBI), the scientists observed that the delivery system showed three times more accumulation in brain than conventional methods of delivery and was therapeutically effective as well, which could open possibilities for the treatment of numerous neurological disorders. Findings were published online on January 1, 2021 in Science Advances. The article is titled “BBB Pathophysiology Independent Delivery of siRNA in Traumatic Brain Injury.” Previously developed approaches for delivering therapeutics into the brain after TBI rely on the short window of time after a physical injury to the head, when the BBB is temporarily breached. However, after the BBB is repaired within a few weeks, physicians lack tools for effective drug delivery. "It's very difficult to get both small and large molecule therapeutic agents delivered across the BBB," said corresponding author Nitin Joshi, PhD, an associate bioengineer at the Center for Nanomedicine in the Brigham's Department of Anesthesiology, Perioperative and Pain Medicine.
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