Catherine Cole, Professor, Stan Perron Chair of Paediatric Haematology and Oncology Princess Margaret Hospital

Professor Catherine Cole

Professor of Paediatric Haematology and Oncology

University of Western Australia, Princess Margaret Hospital for Children, PathWest and the Telethon Kids' Institute.

The improvements in outcomes for children with cancer and leukaemia in the last half century have been phenomenal. From almost universal fatality before 1965, now over 80% of children will be cured. This has come about purely and simply because they have been enrolled on clinical trials.

For the most part the improvement in outcome for children with cancer and leukaemia so far has not come from new drugs, but by better use of drugs we know well and better care of patients from the side effects of the drugs.

Because the various types of cancer and leukaemia in children are rare, the doctors treating these children co-operate with others around Australia and New Zealand and in many International centres in the USA, Canada, UK and Europe, so we can enrol enough children to answer a question of which is the better therapy within a reasonable time frame.  There have been few miraculous improvements in outcomes, rather we have seen a slow steady stepwise improvement in each successive trial-each one building on the last.

At Princess Margaret Hospital for Children we have trials available for about 65% of patients at any time and over 95% of these eligible patients agree to being enrolled on the trial.

At the same time as enrolling our patients on clinical trials we also seek permission to study and store part of their tumour or leukaemia in our research laboratories so we can learn more about the causes of childhood cancer and develop new and less toxic treatments. This close association with the basic scientists means that the knowledge gained at the research bench is translated to improvement at the bedside as quickly as possible.

Being on a trial means that the information gained from your child's treatment is prospectively collated with all the other similar patients for the benefit of future patients. It also gives us the means to see what happens many years into the future to our survivors to inform our future trials and avoid long-term side effects of treatment.

Australian of the Year and Child Health Researcher Fiona Stanley AM says: "Every clinical interaction with a patient is an opportunity for clinical research". She is teaching us to learn systematically from every patient to help our future patients. We do this by enrolling as many children as possible on clinical trials.

It is my hope that the improvements which have benefitted children and younger adolescents with cancer and leukaemia will be extended to older adolescents and young adults as our model of success is adopted by the doctors treating adults with these diseases.