Dr Nick Gottardo

Dr Nick Gottardo

MBChB (University of Leeds), FRACP (Paeds) and PhD (University of Western Australia)
Lillie Research Fellow & PMH Senior Paediatric Oncologist
Phone: 08 9340 8234
Email: nick.gottardo@health.wa.gov.au or Nicholas.Gottardo@telethonkids.org.au

What makes Dr Nick Gottardo so dedicated to his profession is his belief that it's unacceptable for children to die from brain tumours.


Nick's exuberant urgency represents a man on a constant deadline, and with such important work to be done, it's no surprise.


Along with Dr Peter Dallas, Nick leads the Brain Tumour Program team in the Division of Children's Leukaemia and Cancer Research, headed by Professor Ursula Kees. The team's essential work is striving for a future where brain tumours are completely preventable and their causes well understood.


"In conjunction with Peter, I have two research projects investigating two different types of brain tumours: one is Ependymoma (the 3rd most common brain tumour) and the other is Medulloblastoma (the most common malignant brain tumour)," Nick explains.


"My area of expertise is developing mouse models of these diseases, in an effort to have models where we can test new therapies and try and identify areas of weaknesses in the tumour that might be suitable drug targets. With these models, we can then test new drugs or previously developed drugs which have never been used for these kinds of tumours."


Nick's medical career began at Leeds University with a Bachelor of Medicine, Bachelor of Surgery/Chirurgery. He worked for two and a half years as a doctor in the UK, before heading to Australia in 1996, for what was initially to be a 'working holiday.' He soon found himself settled in Perth, working for Princess Margaret Hospital for Children.


"It was during that time I did my PhD with Professor Kees, in T-cell acute lymphoblastic leukaemia," Nick says.


"During my PhD studies I conducted gene expression profiling studies, using a technology known as microarrays. I measured the level of activity (the expression) of thousands of genes at once, to obtain a global picture of the cellular function of leukaemia specimens."


While working with children with tumours, it became apparent to Nick that with leukaemia, research had really transformed a disease which 40 or 50 years ago was essentially fatal, where now up to 80 per cent of children are cured.


"The same cannot be said for brain tumours," acknowledges Nick.


"Many brain tumours remain incurable and the ones that we can cure, often leave the children with a lot of long-term side effects, as a result of current therapies. So, childhood brain tumours appeared to me to be an under-researched area and one in which I could make a difference, if I was equipped with the necessary skills."


In searching the world for places to train, Nick settled on St Jude Children's Research Hospital in Memphis, Tennessee, one of the world's premier childhood cancer institutes. He spent three years at St Jude and gained additional experience in the lab as a post-doctoral brain tumour fellow as well as experience in the clinic.


"My aim was to come back to Perth as a dual trained clinician/scientist," Nick explained. "To be able to not only look after the children, but also use the knowledge I have in the clinical setting to finding new therapies or new ways of trying to cure children with brain tumours.


"I treat all childhood cancers, but really it's brain tumours that I want to make a difference in."


Nick discovered fantastic opportunities at St Jude, both in terms of research project options and his clinical mentor Amar Gajjar, who is now the chair of the Brain Tumour Committee at the international Children's Oncology Group.


"When I arrived in Memphis, my research boss Dr Richard Gilbertson - an incredible person and a recognised world leader in childhood brain tumour research - offered me numerous projects that I could work on and I chose one of the riskier ones," Nick says.


"My aim was to develop a mouse model of Ependymoma, which had never been done before. So I genetically engineered a mouse and eventually the mice developed these tumours, so it was a completely new discovery."


Nick joined the Telethon Institute on his return from the United States in August 2008, having created a mouse from scratch that actually develops Ependymomas. This was the project he brought with him to the Telethon Institute to try and develop further, as a model to test new therapies.


Funding from the Lillee Bequest Fellowship allowed Nick to establish his lab on his return to Perth.


"Without start-up funds I would not have been able to continue my brain tumour research in Perth - it was really nice that I was given this chance," Nick says. "Ultimately, all research boils down to funding. You may have great ideas and be well-trained, but if you don't have some money to back you up, well…"


Nick says the Lillee Bequest Fellowship was instrumental in allowing him to return to Perth and establish his lab. "Essentially I came into a ready-established lab and was able to establish my own small lab within the bigger infrastructure."


From his time in the United States, Nick has seen the benefits of adequate funding first hand.


"A very wealthy gentleman donated around USD$12m to our lab at St Jude to research one specific tumour. It's not often someone comes along and says 'Here's $12 million, go and find a cure for this disease.'


I'm not sure why we don't have that in Australia, given this is such a wealthy country and we're now one of the strongest economies, as shown by the recent global financial crisis," Nick explains.


"The hospital where I trained never charged the patient, which is quite unique in America. It was irrelevant whether you were the son of a millionaire or the son of a pauper, you got the same treatment and the same standard of care.


"To me, St Jude is a utopia of medical practice and research, a place where researchers and clinicians all work together with the common goal of curing childhood cancer. St Jude strives to attract the best researchers and clinicians from around the world. They provide generous start-up funds and facilities for five years in order for you to establish your research laboratory. They allow you that period of time to establish yourself and get some publications, which ultimately is how success is measured in research. After that period you are expected to attract your own funding - I think this approach is very fair and a good model," he says.


Nick explains the most difficult thing about research is constantly having to write grant proposals in order to try to obtain the funding to continue your research.

"I do tend to get frustrated, constantly writing about what I want to do and what I'm going to do, instead of getting out there and doing it," Nick says.


"I think it would be nice if there was more extensive and easier-to-obtain funding, especially for early career scientists who are trying to establish themselves. Obviously it needs to be peer-reviewed because you do not want to pour money into poorly thought-out research projects, but the current funding environment is so highly competitive that funding is very hard to obtain, and this can be quite demoralising," Nick admits.


A Fellow of the Royal Australasian College of Physicians, Nick is so inspired by the advances his predecessors made with similarly once fatal conditions such as leukaemia; he believes the same can be achieved with brain tumours.


"Working with children with brain tumours is not easy," Nick admits. "It is devastating when one of your patients dies or is severely affected in the long term by their illness and treatment. That is what drives me - the belief that we can improve on the current situation.


"If you came through the doors of PMH in the 70's or early 80's you would have most probably died from leukaemia. You come through the doors now and you've got an 80 per cent chance of being cured.


"That's because somebody visionary at the time said this is not acceptable, we need to do something and not accept that these children are going to die".


Nick believes we are now at the dawn of a new age of therapies for cancer.


"We have entered a new era of molecular targeted therapies - or 'silver bullets' if you like - where we try and target weaknesses or the Achilles heel in the cancer by specifically blocking a molecule or molecules that the cancer needs to grow and survive. There is great hope that these therapies might improve the outcomes for children with brain tumours."


When Nick is not at work, he tries to spend as much time as he can with his family.



"I have a 6-year-old daughter and a 12-week-old son, and at the moment I try and spend as much time as I can with them and my wife so that they get to know who their father and husband is," Nick says.


Although in possession of a black belt in Tai Kwon Do, Nick admits this interest has dropped by the wayside, with such an enormous amount of work still to be done.


"My aims are to take discoveries from the lab into the clinic. If I can make a discovery that can be translated into a new therapy for children with brain tumours, that would be my ultimate goal. That's the kind of contribution I would like to make to the field."


External Committees
Australian Children's Clinical Trials Group

The Cure Starts Now Foundation Medical Advisory Committee

Australian Children's Cancer Trials (ACCT) Principal Investigator.


Paediatric Brain Tumours. Royal Australasian College of Physicians Annual Scientific Meeting. Melbourne, March 2010.

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