https://www.nature.com/articles/s41573-020-0090-8
https://pubmed.ncbi.nlm.nih.gov/26451106/
https://advances.sciencemag.org/content/6/30/eabb4429
“The power of our method is that it is extremely versatile and relatively non-disruptive,” said Qiaobing Xu, PhD, associate professor of biomedical engineering at Tufts University and corresponding author of the team’s published paper in Science Advances. “We can deliver a wide range of molecules by packaging them into the lipid-based nanoparticles without chemically modifying the drugs themselves. We can also achieve delivery across the blood-brain barrier without disrupting the integrity of the barrier.” Xu and colleagues described the technology in a paper titled, “Neurotransmitter-derived lipidoids (NT-lipidoids) for enhanced brain delivery through intravenous injection.”
The blood-brain barrier comprises a layer of endothelial cells that line the blood vessels in the brain, which allows only select types of molecules to pass from the bloodstream into the fluid surrounding the neurons and other cells of the brain. The BBB prevents the transfer of most small-molecule drugs and macromolecules, such as peptides, proteins, and gene-based drugs, which has limited the treatment of CNS diseases, such as neurodegenerative disorders, brain tumors, brain infections, and stroke, the authors noted. “Safe and efficient delivery of BBB–impermeable cargos into the brain through intravenous injection remains a challenge.”
Current strategies for delivering drugs into the brain, such as direct injection, or disruption of the BBB to make it “leaky,” are fraught with risks, including infection, tissue damage, and neurotoxicity. “Although extensive efforts have been undertaken to enhance brain delivery efficiency, each method has both advantages and disadvantages; thus, the development of safe and efficient delivery of BBB-impermeable cargos into the brain through intravenous injection remains a big challenge,” the investigators noted.