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.
“It’s simple, effective, and potentially broadly applicable—we can modify the container for the drug, and by adding the NT-lipidoid, it’s like attaching an address label for delivery into the brain” said Feihe Ma, post-doctoral scholar in the Xu lab at Tufts. “We envision that a wide range of neurological therapeutics could eventually be tried that were previously thought to be impractical due to limitations in delivery,” said Liu Yang, graduate student in the Xu lab. Ma and Yang are co-first authors of the study.
The study authors made use of the fact that certain neurotransmitters have the chemical “passport” required to gain access throughout the brain. By attaching a lipid (fat-like) molecule to the neurotransmitter, the resulting NT-lipidoid can be doped into lipid nanoparticles (LNPs) - tiny bubbles of lipid that can encapsulate other molecules within, in particular therapeutic drugs. The LNPs can be injected intravenously, and carry the drugs to the blood-brain barrier, while the NT-lipidoid helps the LNPs to carry the drugs across the barrier. The LNPs can then fuse with neurons and other cells in the brain to deliver their therapeutic payload.
Obviously it will be a long time before this is used in humans. However it’s a huge step and very good news. Some therapies for a number of different brain illnesses could be tried without the risk of injecting something into the brain, it would just be an IV.