Summary

Currently there is no effective therapeutics for Motor Neurone Disease (MND). One of the main hurdles to development of new drugs is achieving efficient delivery of drugs to the location in the brain and spinal cord where it is required.

This is highlighted by one of the most effective gene therapies trialed in MND mice that reaches only 8% of motor neurons in these mice. Our objective is to develop a therapeutic delivery system that will increase the success rate of targeting any drug to motor neurons in the brain and spinal cord.

Our drug delivery system can be formulated to carry a wide variety of therapeutic cargoes. This will have a major impact on the types of drugs that can be delivered to motor neurons opening up many new avenues of therapy.

The objective of the proposed research is to develop a targeted liposomal drug delivery system for delivery of therapeutics specifically to increase the efficiency of targeting motor neurons.

We will prepare biocompatible phospholipid vesicles (liposomes) that have been sterically stabilized (PEGylated) and surfaced functionalised with anti-transferrin receptor antibodies ( anti-TfR) to cross the blood brain barrier [1], enter neurons [2] and deliver DNA.

Model systems to be used include ALS mouse model spinal motor neurons in culture and murine models of ALS. This model will allow proof of concept of our gene-targeting drug delivery strategy both in vitro and in vivo.

Funding body

Motor Neurone Disease Australia

Scheme name

Grant-In-Aid

Years funded

2016 – 2018

Lead institution

University of Wollongong