A sniff instead of a spinal tap: nose-delivered treatment delays ALS in mice

Getting medicine to the brain usually means going through the spine, because a filter called the blood-brain barrier keeps most drugs out. Researchers at the University of Georgia's Regenerative Bioscience Center, led by Yao Yao with Emory colleagues, tried the nose instead: they packed a gene-silencing drug (an antisense oligonucleotide) into tiny sacs made by neural stem cells and delivered them through the nasal passages of mice with ALS, the motor-neuron disease. Treated mice developed symptoms later, moved better, kept more of their motor neurons and showed less inflammation in the nervous system. It is a mouse study — doses and human testing come next — but a gentler route into the brain could change how several brain diseases are treated. The work appears in the Journal of Extracellular Vesicles.

SDG 3 · Good health and well-being — Mechanism: tech innovation