RNA offers hope for new Parkinson’s treatment

An RNA-based gene therapy treatment for Parkinson’s disease that could slow and possibly even stop the condition is now a big step closer.

Professor Vincent Harley at Hudson Institute of Medical Research is investigating the potential for mRNA technology to target the processes of cell death that characterise the disease.

Prof Harley is one of two Hudson Institute researchers to receive grants worth $100,000 from the mRNA Victoria Research Acceleration Fund (mVRAF) to further their research and bring potential treatments closer to human trials.

The other is Dr Jim Vadolas, who researches iron dysregulation in the blood and the health impacts that can result from it.

They are among 16 projects sharing in a total of $2.7 million funding, announced today by Premier Daniel Andrews and Minister for Industry and Innovation Ben Carroll.

Potential Parkinson’s treatment

Prof Harley’s work stems from his discovery of the sex determining gene, SRY, and its contribution to sex (male) bias in certain neurological disorders, such as Parkinson’s disease, ADHD and schizophrenia.

He has established that in men with Parkinson’s disease there is an excess of SRY, which contributes to the death of brain cells responsible for dopamine production.

“We have created an RNA/DNA hybrid molecule that binds to the SRY and degrades it,” Prof Harley said.

“This molecule is a disease modifier in that the aim is to stop the neurodegeneration that is a characteristic of Parkinson’s.”

“We hope this drug will stop it, but we know it should slow the progression of Parkinson’s.” he said.

This form of treatment has been successfully trialled in rats, and this grant will help researchers to develop a human version.

“This funding allows us to evaluate the drug through screening and testing – modifying which part of the SRY molecule to target and how to do so.” Prof Harley said.

Iron dysregulation and blood diseases

Dr Vadolas said his grant will allow him to advance his work to address the consequence of iron dysregulation in Myelodysplastic Syndromes (MDS).

“In collaboration with clinical colleagues Dr George Grigoriadis and Dr Shahla Vilcassim, this project aims to inform on clinically relevant strategies that can be used to better manage MDS patients who are at risk of developing iron overload and acute leukaemia.” he said.