[Professor Catherine Cole]
I'm a paediatric oncologist and haematologist, so that means I look after children with cancer and blood diseases. When I got to this ward and saw that the way of managing the children was on clinical trials, a very scientific approach to the therapy, it just clicked and was really where I wanted to spend the rest of my life. It is an emotional area to work in and everyone says, How can you possibly do the work? But the science of managing the children on clinical trials is the perfect foil for the emotional side of the work. It's much easier to cope with the fact that some children won't survive if we know that they've had the very best therapy in the world.
In the early eighties, most children with cancer and leukaemia died of their disease. We were lucky enough to have Michael Willoughby arrive from the UK, and he brought with him the philosophy of comparing one treatment with another. And over the next decade he was able to make our unit the first full member in the Children's Oncology group outside North America. But it brings with it not only the benefit of having the trials open for the children, but access to enormous numbers of incredibly wise people. To be able to say to a family, you don't have to go anywhere to get the best therapy in the world, your child's having the same treatment they would get at Sloan-Kettering in New York or MD Anderson in Houston or Great Ormond Street in London is fantastic.
Clearly, staying with your family is incredibly important in treating children. In doing a clinical trial, we randomly assigned children to one treatment or the other. Usually the standard arm of the trial is the best treatment we know to date and the other arm of the trial, the so-called experimental arm, is slightly different. And we're asking a question to see if that new treatment is better than, or less toxic than, or both, the standard treatments. If you look at the improvement in survival in childhood leukaemia trials over the last 50 years, there hasn't been any miraculous change it's been a slow, stepwise improvement from one study to the next, each improving on the last such that we've gone from perhaps 20% of children being cured when we began, or even fewer to now over 80, 90% depending on the type of leukaemia that you have.
The other strength is not only that we've cooperated with the other paediatric oncologists around the country and around the world, but that within our own teams we cooperate with our multidisciplinary team. So the patients are looked after by the oncologists, by the surgeon, by the radiation oncologists, we have all our specialized nurses and we have of equal importance a psychosocial team that actually keeps the kids lives moving ahead and keeps the families together in what is chaos for the family when your child is diagnosed with cancer. We couldn't do this without our X-ray department, the radiologists being on side because they have to fill out all the paperwork on all of the kids, X-rays and so on.
Our surgeons our pathologists, everyone is a member of our wider team. We now have a great relationship with the other specialists around the country in fields like immunology, because they will often diagnose diseases that respond well to similar sorts of treatment to the children with cancer. I'm very proud of the fact that successive hospital administrations here have supported us wholeheartedly. They haven't said, 'Oh, that's research, we're not going to fund it.' They've understood from the very beginning that this is the way to treat children's cancer. It's so much easier if you're collecting the data from all the patients prospectively and looking at it in real time. Not only because you see when things are going well, but you see when they're not going well. So it'll be small, stepwise improvements as we learn how to use these new drugs, how we use them with our traditional cytotoxic drugs and the platform for doing the investigation is the clinical trial.
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.