Dialog Box


Paediatric Oncology, From Bench to Bed 

Lead PI: Dr Nick Gottardo, Telethon Kid's Research Institute, WA

 Dr Nick Gottardo

Research Idea 

Our idea is that we can identify new therapies to be tested in clinical trials by using high-throughput robotics to screen thousands of drugs for the ability to inhibit the growth of childhood brain cancer cell lines in test tubes and then subsequently validate these drugs using novel sophisticated laboratory models of childhood brain cancer.


Childhood brain cancers are often incurable. In Australia, brain cancer is the second most common cause of death in childhood, after accidents. Even when brain cancer can be cured, the treatment often results in severe long-term side effects, such as learning disabilities and movement problems, because the central nervous system (i.e. the brain and spine) is still developing in children. We are attempting to find new, more specific therapies to increase cure rates and reduce treatment side effects to improve the quality of life of survivors.


One of our laboratory’s areas of expertise is in generating sophisticated laboratory models using brain cancer cells obtained from patients at the time of surgery. These models closely mimic the characteristics and behaviour of tumours in children and provide the ideal platform in which to test new therapies. We search for 'vulnerabilities' in the tumours and use our models to test new drugs that target these. We also use our models to test anti-cancer drugs that have been developed to treat other cancer types to see whether these drugs might be useful for treating children with brain cancer. 

Why Now?

There are two major advances that make our project feasible now. The first advance is the availability of high-throughput robotics. This technology allows us to rationally screen thousands of drugs, in an unbiased manner, for their ability to kill brain cancer cells. The second advance is that during my fellowship at St. Jude Children’s Research Hospital in Memphis, Tennessee, I was the first to generate a sophisticated model of ependymoma, the third most common brain tumour that affects children. After returning to Australia, I established my own brain tumour research laboratory, and since then my team has generated several other childhood brain cancer models. These models now allow us to more accurately assess new drugs in model systems that faithfully reflect the disease in children. Our project will harness the power of both robotic high-throughput drug screening and innovative models of childhood brain tumours to robustly test the efficacy of newly identified drugs, in order to select the treatments that are most likely to be successful in clinical trials.


Our research strategy has been designed specifically to speed up the translation of drugs that are discovered in our program into new treatments for childhood brain cancer in the clinic. Utilising high-throughput screening of approved and well-characterised drugs, we will identify several drugs that have anti-tumour activity on brain tumour cells. We propose that our studies will reveal that combining new drugs with each other and with conventional chemotherapy will kill brain tumour cells more effectively, improving cure rates and potentially allowing a reduction in the dose of the more toxic components of current therapy. Our study will identify a small number of drug combinations that warrant evaluation in our sophisticated laboratory models, an essential step to proving the efficacy of treatment before implementation in a clinical trial.


Our ultimate goal is to design new clinical trials using preclinically proven treatments and knowledge to appropriately select the patients who are most likely to benefit. These trials will be implemented nationally through collaboration with the Australian Children’s Cancer Trials (ACCT) group and internationally through the Children’s Oncology Group (COG). Our findings will help to personalise treatment for particular childhood brain cancers, leading to more cures and a better quality for life for children who are cured. 

Team & Partners 

Dr Raelene Endersby was recruited in 2011 from St. Jude Children’s Research Hospital, where she gained expertise in childhood brain cancer modelling and testing new drugs. She leads our preclinical testing program. Dr Tobias Schoep was recruited at the end of 2012 from the Department of Chemical Engineering at the University of California Santa Barbara in California, where he worked on developing strategies to improve the specificity and reduce the toxicity of anti-cancer therapeutics. His research focuses on drug screening and testing drugs in combinations to increase their effectiveness. Our brain tumour research program is also part of the Brain Cancer Discovery Collaborative (BCDC), and we will use our models to assess the effectiveness of potential treatments generated by other BCDC researchers.


The BCDC brings together eminent members of the scientific and medical research community across Australia and is committed to finding treatments and a cure for brain cancer.