26 June 2020

WA led Myositis study attracts major federal grant

A global clinical trial led by consultant neurologist Professor Merrilee Needham has attracted a $1.88 million Federal Government grant to investigate use of a repurposed drug for Inclusion Body Myositis (IBM).

Professor Needham (The University of Notre Dame School of Medicine and Head of Neurology at Fiona Stanley Hospital) is a world leader in the Myositis field and heads the Myositis Research Group at the Perron Institute for Neurological and Translational Science.

As yet, there are no disease modifying treatments for IBM, a muscle wasting disease which causes weakness in the limbs and other issues such as difficulty with swallowing. About 60 per cent of those diagnosed lose independent mobility within seven to ten years of symptom onset.

Bringing hope to sufferers of this rare and relentlessly debilitating disease, this research will evaluate the effectiveness of an immunosuppressant drug, Sirolimus, also known as Rapamycin. This is a treatment primarily used to prevent organ rejection in post kidney transplant patients.

“The Federal Government’s announcement of the grant to fund this research was a landmark day for Inclusion Body Myositis patients, “Professor Needham said.

“With some encouraging results from a small pilot study in France, the hope is that the immunosuppressant drug we are investigating will stabilise or slow disease progression for people with IBM.

“The funding opens the way for an international study to validate the initial findings, bringing hope for a disease-targeting treatment, where currently there is none.”

Collaborators in the WA led study are the Perron Institute, University of Notre Dame, Murdoch University and Fiona Stanley Hospital.

IBM patients from seven sites across Australia will be recruited for the study and will receive either Sirolimus or a placebo over a period of 88 weeks. The study will also involve IBM sufferers from the USA, the UK and Europe.

Merrilee Needham Perron Institute