Biomedical engineers at the Tufts University School of Engineering have developed tiny lipid-based nanoparticles that incorporate neurotranmitters to help carry drugs, large molecules, and even gene editing proteins across the blood-brain barrier and into the brain in mice. The innovation, published today in Science Advances, could overcome many of the current limitations encountered in delivering therapeutics into the central nervous system, and opens up the possibility of using a wide range of therapeutics that would otherwise not have access to the brain.
“The power of our method is that it is extremely versatile and relatively non-disruptive,” said Qiaobing Xu, associate professor of biomedical engineering at Tufts University and corresponding author of the study. “We can deliver a wide range of molecules by packaging them into the lipid-based nanonparticles without chemically modifying the drugs themselves. We can also achieve delivery across the blood-brain barrier without disrupting the integrity of the barrier.”
Xu cautioned that more studies and clinical trials are needed to determine the efficacy and safety of the delivery method in humans.
The blood-brain barrier consists of a layer of endothelial cells that line the blood vessels in the brain and allows only a highly select set of molecules to pass from the bloodstream into the fluid surrounding the neurons and other cells of the brain.The ability to safely and efficiently deliver therapeutic molecular cargos across the barrier and into the brain has been a long-standing challenge in medicine.
The treatment of neurodegenerative disorders, brain tumors, brain infections and stroke has been limited by the difficulty in safely delivering small molecule drugs and macromolecules, such as peptides and proteins, into the brain. Current approaches, such as direct injection or disruption of the barrier to make it “leaky,” are fraught with risks, including infection, tissue damage and neurotoxicity. The use of carriers, such as modified viruses and monoclonal antibodies to ferry cargo into the brain, has limitations, including production cost and safety. Other carriers, such as nanoparticles, nanocapsules and polymers, have shown promise but the modifications required to ensure delivery can be complicated.
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Thanks to Heinz V. Hoenen. Follow him on twitter: @HeinzVHoenen