Translating discoveries into health benefits

Translating discoveries into health benefits

Illuminate newsletter index page, June 2018
June 2018
Our researchers are making basic discoveries that can be translated into the diagnosis, treatment and prevention of disease.

The Institute has a strong history of translating fundamental research discoveries into real benefits for the community. There are currently more than 100 clinical trials, underway all over the world, based on our research.

Ros Edmond and Professor Don Metcalf
(L-R) CSFs patient Roz Edmond with the late Professor Don
Metcalf who pioneered research to boost infection-fighting white
blood cells after chemotherapy.

An inspiring history of translation

One of the Institute’s great success stories was the 25-year quest to discover and purify colony stimulating factors (CSFs) as a supportive therapy for cancer patients, led by the late Professor Don Metcalf. In 1991, CSFs were approved for the clinic and are now used to boost infection-fighting white blood cells after chemotherapy.

Another significant milestone was in 2017 when Australians with certain forms of leukaemia gained access to venetoclax, a potent anti-cancer drug based on a discovery at the Institute in the late 1980s. Almost 30 years of Institute research by dedicated experts in cancer biology, structural biology, medicinal chemistry and clinical research underpinned the drug which was co-developed with companies AbbVie and Genentech, a member of the Roche Group.

Institute director Professor Doug Hilton said he was proud of the commitment Institute researchers had shown to seeing their discoveries impact the lives of patients.

“Both CSFs and venetoclax are great examples of the contribution Australia can make to globally significant translation.

"There is no question that investment in basic research is vital for the future of drug discovery and development,” Professor Hilton said.

Breast cancer breakthrough to clinical trial

For 20 years, Professor Jane Visvader and Professor Geoff Lindeman have been investigating the basic biology of breast cancer in the hope of developing new treatment and prevention strategies.

This year they will embark on a world-first trial that aims to prevent high-risk women carrying a ‘faulty’ BRCA1 gene from developing breast cancer. The trial is based on a landmark biology discovery the team that identified a critical cancer ‘trigger’ that could be targeted with existing medications to prevent breast cancer.

Professor Goeff Lindeman and Professor Jane Visvader
(L-R) Professor Geoff Lindeman and Professor Jane
Visvader are leading a world-first trial that aims to prevent
women from developing breast cancer.

Professor Lindeman, who is also a medical oncologist at the Royal Melbourne Hospital and Peter Mac, said the trial was a decade in the making.

“The BRCA-P randomised phase 3 clinical trial, run by Breast Cancer Trials Australia, will test whether existing medication denosumab can safely and effectively reduce the incidence of breast cancer in high-risk women with a faulty BRCA1 gene.

“We think this strategy could delay or prevent breast cancer in these women, who have few other options except radical surgery to remove their breasts and/or ovaries, usually at a relatively young age,"

"We are hopeful for the trial and excited about the potentially significant positive impact it could have,” Professor Lindeman said.

Pinpointing the genetics of stuttering

Professor Melanie Bahlo is working with a team of researchers from four Australian research organisations on Australia’s largest-ever research study to pinpoint the genes that predispose people to stuttering.

The project will recruit more than 3000 volunteers aged seven or older who stutter or have a history of stuttering. The Genetics of Stuttering Study is a collaboration between the Institute, Murdoch Children’s Research Institute, QIMR Berghofer Medical Research Institute, Griffith University and the University of Melbourne.

Professor Bahlo said her team was leading the DNA analysis of participants’ saliva to unravel the genes involved in stuttering.

“Very little is understood about why people stutter,” Professor Bahlo said.

“We are aiming to identify the genes associated with stuttering in the hope of revolutionising future research into the causes, treatment and prevention of the disorder.”

For more information visit the study's website.

Blood test diagnosis for acute rheumatic fever

Dr Laura Dagley is working to develop a blood test that will better detect acute rheumatic fever, a disease which predominantly affects young, Aboriginal children and if left untreated often results in life-threatening heart disease. Dr Dagley said that little has changed in the past 50 years in terms of how this serious condition was diagnosed.

“We are developing a blood-based test that will hopefully make it easier to properly diagnose acute rheumatic fever so it can be treated before it causes heart disease.

“We are also hoping to improve how samples are collected and stored in remote clinical settings where the disease is prevalent.”

The project involved measuring thousands of proteins in samples of tissues and blood to identify specific ‘biomarkers’ to help diagnose the disease.

Dr Dagley said rheumatic fever "continued to lead to early death and disability for many people, particularly in remote Aboriginal communities."

"A new test could have a significant impact on the length and quality of life for affected individuals."

In 2017 Dr Dagley received a $22,500 AMP Tomorrow Fund grant to help facilitate an expansion of the program developed at the Institute. Learn more about Dr Dagley's research:


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