Dialog Box


Circulating microRNA as a biomarker in brain cancer 

Lead PI: Dr Andrew Morokoff, Royal Melbourne Hospital, VIC

Dr Andrew Morokoff 

Research idea

The team want to develop micro RNA detection in blood as a simple, rapid, cheap and accurate biomarker for brain cancer diagnosis and prediction.


The problem is that the current method of diagnosing and subtyping brain cancer, especially in lower grade gliomas is inadequate. Secondly, that the only current method of following tumours and diagnosing recurrence is by contrast enhanced MRI, which gives very good anatomical and spatial resolution, but poor biological information. For instance, in low grade gliomas, change is often subtle and non-contrast enhancing. In high grade tumours, it is impractical and expensive to perform multiple MR scans. There is the need for new more accurate, and more 'quick and easy' ways such as a blood test, to assess tumour status in brain cancer patients. 


The team’s approach seeks to validate a biological marker using a simple, cheap blood test and the advantages of microRNA detection. No biomarker has yet been validated as clinically useful in brain cancer, but various options are being actively pursued. Their advantage is that they have a unique situation of having been able to build an extremely large biobank of tumour and blood specimens over the last 4 years due to their coordinated, integrated research program linking the operating theatre and the research laboratory. Using the latest miRNA screening technology, they can use this biobank to validate this concept for rapid translation into the clinic. 

Why now?

There are 4 major factors that make this project possible at this time:

  • They have now established a large biobank of 200+ patients with matched tumour and multiple blood samples.
  • Their clinical database has now 'matured' to included valuable outcomes and survival data that can be correlated with biological variables.
  • They have access to the NanoString technology and local and international expertise in next generation genomics and bioinformatics for screening miRNA expression.
  • They have developed overseas collaborations that are critical to the project's success and will situate our work in the international brain tumour arena.

"Things have accelerated a lot in the last couple of years. I think we have a much better understanding of the human genome now and we now for the first time in history have technology that can actually screen the whole genome and can do this kind of project. So we can screen hundreds or thousands of genes in just a few hours for about $1000. Whereas just five years ago that was almost impossible. So it really is technology driven, a lot of the research."

- Dr Andrew Morokoff 


If they can identify particular miRNAs or a panel of miRNA that correlate with tumour grade, prognosis or progression, this scientific information can be rapidly translated to a pilot clinical trial or this biomarker in a hospital neuro-oncology setting. Their institution is well set up to do this, because they are currently running multiple clinical trials and have a large cohort of patients and a well-established brain cancer treatment platform. Further validation of the miRNA biomarker will involve prospective assessment in a cohort of brain cancer patients with particular treatment outcomes. Potential commercialisation of the test is possible.


A validated biomarker would be an enormous advance for brain cancer patients. Imagine if we could accurately predict how each person's tumour is going to behave and how it is going to respond to which particular targeted treatment. Furthermore, imagine if a simple blood test could be done that could monitor response to treatment and indicate when a therapy was failing or the tumour recurring. The big advantage of a circulating blood biomarker is that a blood sample is quick to take in the clinic and can be done multiple times to follow a patient's progress, something which is difficult, costly and inconvenient for patients with MRI.   

Team & Partners

CIs Morokoff and Drummond together with AI Kaye (Head of Dept) have driven the basic science, biobanking, translational and clinical database capabilities at Royal Melbourne Hospital over the last 5 years creating a platform for projects like this to now progress. Luwor is a senior scientist and lab head with expertise in cancer genomics. AI Siegal is a world renowned neuro-oncologist who they have collaborated with over the last 4 years to investigate circulating biomarkers. AI Koldej is a local expert in miRNA screening in cancer. AIs Johnson and Roncaroli are experts in brain genomics. They also collaborate with experts from Walter and Eiza Hall Institute & Florey institutes and Peter Mac.


  • A/Prof Kate Drummond, Royal Melbourne Hospital
  • Dr Rodney Luwor, University of Melbourne 


  • Prof Tali Siegal and Dr Rachel Koldej, University of Melbourne
  • Dr Michael Johnson and Dr Federico Roncaroli, Imperial College London
  • Prof Andrew Kaye Royal Melbourne Hospital and University of Melbourne 


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