Dr Andrew Munkacsi
School of Biological Sciences Victoria University of Wellington
Drugs known as histone deacetylase (HDAC) inhibitors show potential to treat many neurodegenerative diseases such as Parkinson’s and Alzheimer’s, yet these drugs are not efficient at crossing the membrane that covers the brain, the blood-brain barrier (BBB). We propose to administer an HDAC inhibitor (Vorinostat) via the nose to bypass the BBB, and deliver the drug directly to the brain. We will determine whether intranasal treatment inhibits neurodegeneration in a mouse model of Niemann-Pick type C disease, a paediatric neurodegenerative disease currently without treatment. This drug is already approved to treat cancer and positive results could quickly be translated into clinical practice which would provide immediate hope for the affected children as their lifespans are less than the time it takes to develop a new drug.