Oxford University scientists have developed a new method for delivering complex drugs directly to the brain, a necessary step for treating diseases like Alzheimer's, Parkinson's, Motor Neuron Disease and Muscular Dystrophy.
These diseases have largely resisted attempts to over the last 50 years develop new treatments, partly because of the difficulty of getting effective new drugs to the brain to slow or halt disease progression.
The team has successfully switched off a gene implicated in Alzheimer's disease in the brains of mice by exploiting exosomes -- tiny particles naturally released by cells. The exosomes, injected into the blood, are able to ferry a drug across the normally impermeable blood-brain barrier to the brain where it is needed.
Although this is a significant and promising result, there are a number of steps to be taken before this new form of drug delivery can be tested in humans in the clinic. The study, partly funded by the Muscular Dystrophy Campaign, is published in Nature Biotechnology.
These are dramatic and exciting results. 'It's the first time new "biological" medicines have been delivered effectively across the blood-brain-barrier to the brain,' says Dr. Matthew Wood of the Department of Physiology, Anatomy and Genetics at the University of Oxford, who led the work.
Exosomes are small capsules that are produced by most cells in the body in varying amounts. These natural nanoparticles are thought to be one fo the ways cells communicate with each other and the body's immune system. When exosomes break off from the outer walls of cells, they can take various cellular signals and genetic material with them, transporting this material between different cells.