"Our goal must be to ensure that all children with cancer have access to the best possible therapy"
Nicolas Waespe, MD
Every patient and every tumour is unique. Thanks to rapid advances in molecular biology, it is now possible to characterise the tumour of an individual person in detail. In addition to classic treatment methods such as surgery, radiation and chemotherapy, therapies tailored specifically to the patient are increasingly being used. An interview with Nicolas Waespe, MD, senior physician at Bern University Hospital, specialised in the treatment of children and adolescents.
Paediatric oncologists and parents of children with cancer are placing a lot of hope in novel therapeutic approaches, for example targeted therapies, which are also sometimes referred to as individual, personalised or tailor-made medicine. What exactly do these terms mean?
The targeted therapies that are currently revolutionising medicine each try to attack a specific mechanism that is only, or at least predominantly, active in cancer. This makes it possible to stop cancer cells from growing which means they die. These might be metabolic pathways that are active in the cancer cells or special surface proteins that are mainly produced by the cancer. This makes it possible to attack the cancer much more precisely than with conventional chemotherapy or radiation. CAR T cell therapy is one of these innovative, new therapeutic approaches. In this procedure, the body’s own defence cells are taken and modified in the laboratory so that they specifically attack cancer cells. These genetically modified cells are then administered to the patients again so they can unleash their effect. The advantage of these targeted therapies is that the cancer can be fought more efficiently and the side effects can in part be reduced whereas in classical chemotherapy the healthy cells are often also damaged. This leads to acute and long-term side effects, particularly in children who have not yet finished growing – something which can massively restrict their quality of life in the long term. And that’s what we are looking to avoid.
In adult cancer patients, these new treatment strategies with targeted drugs are already being used successfully for certain types of cancer. What is the situation in paediatric oncology?
Targeted therapies are already being used for children with certain cancers. This is taking place as part of clinical trials or often without special approval on a doctor’s prescription. Antibody therapies for leukaemia or lymphoma are examples of this. There, they are often used in combination with conventional chemotherapies and can thus also completely replace conventional chemotherapies in the case of certain cancers. Even though we still know very little about the long-term effects of these therapies in children, the success in treatment is sometimes very impressive. Especially for patients who do not respond to standard therapies or who suffer a relapse. However, in order to be able to make reliable statements that will help us to optimally develop these innovative therapies for children and adolescents and to incorporate them into therapy plans as early as possible, more extensive clinical trials are absolutely essential. They cost a lot of money, however, which is why financing them is an enormous challenge for us.
The pharmaceutical industry has been carrying out research into targeted cancer therapies for adults intensively and very successfully for years. Why do the young and vulnerable continue to be ‘forgotten’?
It is much less profitable for the pharmaceutical industry to develop drugs for childhood tumours because childhood cancer is so rare in comparison to cancer in adults. Around 350 children and adolescents are diagnosed with cancer in Switzerland every year, while more than 40,000 adults are affected by cancer. It is pretty obvious that there is little money to be made in childhood cancer medicine. Paediatric oncology thus often remains dependent on advances in the treatment of adult cancer patients before these then benefit young patients. The problem is that children are not just small adults and treatments developed for adults cannot always simply be directly applied to treat children’s conditions. More incentives have to be created for the pharmaceutical industry to develop drugs that are suitable and tested for the treatment of childhood tumours.
In your opinion, what exactly is needed for greater advances to be made in childhood cancer research?
First of all, I want to stress that the treatment of childhood cancer is an extraordinary success story thanks to the collaboration of researchers and paediatric oncologists worldwide. While 50 years ago less than half of all children with acute lymphoblastic leukaemia survived, the figure is now over 90 per cent. Nevertheless, there are still types of cancer that are very difficult to treat, such as certain brain tumours as well as soft tissue and bone tumours. In order for more children to be cured and suffer from fewer late effects, intensive research must continue. However, due to the small number of patients, progress in childhood cancer medicine is only possible in an international network and with the support of donors, as government funding is far from sufficient. Even though targeted therapies can result in a cure where conventional methods fail, childhood cancer can often only be cured with the interaction of the various therapies. Therefore, all available treatments are still needed: these then have to be adapted to the disease and the affected child.
Personalised medicine is expensive. And that means extremely high costs for the Swiss health system. Who then decides which children will be given these new drugs and which won’t?
The costs may seem great for just one child, but they can lead to great things. A child who has been cured with more gentle treatment grows up to be an important member of our society and can give back a productive contribution to the community throughout the rest of their life. And that is why, in my opinion, we should under no circumstances ration the medical treatment of children for financial reasons. On the contrary, our goal must be to ensure that all children with cancer have access to the best possible therapy. According to the National Health Report, in 2020 only around 12 per cent of health care costs in Switzerland were attributable to children, adolescents and young adults aged 0 to 25. The monthly per capita costs in this age group were less than half of the average per capita costs of the total population. In addition, only a few children are affected by cancer each year. So we are talking about very different case numbers than for adults. By ensuring that children and adolescents with cancer receive the best possible care, we are in fact investing in the future of our society.