Paediatric cancer is still a major public health issue, despite high survival rates compared to adult cancers:
- Despite improving survival rates, cancer is still the first cause of death by disease beyond one year of age in 6,000 young people die prematurely each year due to cancer in Europe.
- Each year there are 35,000 new cases of cancer in children and adolescents in Europe (15,000 in children below the age of 15 and 20,000 in those aged 15-24). In the US, 10,000 children younger than 15 and 5,000 aged 15 – 24 years are diagnosed annually with cancer.
- 1 out of 600 newborns will develop cancer before turning 20 years of age
- 80% are disease-free after 5 years from diagnosis, thanks to the currently available multidisciplinary treatments. However:
- Today there are approximately 300,000 to 400,000 childhood cancer survivors both in Europe and the United States.
- Two-thirds of survivors have late side effects of treatment, which are severe and impact on the daily life of half of those affected
- Beyond 5 years from diagnosis, disease-free survivors have a higher mortality rates than their non-affected peers
Cancers in children differ from cancers in adults
The most frequent childhood cancers are leukaemias, tumours of the central nervous system (CNS), lymphomas and neuroblastomas. They occur from birth to adolescence, with 35% of the typical childhood cancers occurring before the age of five years. CNS tumours (33%), leukaemias (29%) and neuroblastoma (8%) are responsible for 60% of cancer deaths amongst children aged 0 to 14 years.
Considering epidemiology and outcomes, there are 3 main groups of paediatric cancers:
- Those with a good prognosis (with higher than 85% chance of survival after five years) when treated with current standard multidisciplinary treatments, using cytotoxic drugs in often an intensive mode (acute lymphoblastic leukaemia, lymphomas, retinoblastoma and renal tumours). Over the last five years, the survival rates have plateaued for patients suffering from these malignancies, while treatment intensity has been reduced for some patients in order to decrease the risk of long-term side-effects;
- Those with a poor prognosis (~50% or less 5 year survival) such as acute myeloid leukaemia, several CNS tumours, neuroblastoma, bone and soft tissue sarcomas. Among these diseases, some have a very poor prognosis such as diffuse intrinsic pontine glioma, high-risk neuroblastoma and metastatic sarcomas;
- The extremely rare tumours, for which there is a lack of information on their real incidence and survival. They include paediatric malignancies which occur extremely rarely and adult cancers occurring extremely rarely in children and adolescents, such as breast cancer, colon cancer and melanoma.
Teenagers and Young Adults (TYA) aged 15 to 24 years have very specific needs and there are still differences in their 5 year survival when compared to younger children with the same malignancy.
Little Progress in the Last 5 Years
There has been little progress in the poor prognosis malignancies-over the last five years.
Progress has been made during the last 50 years by using intensive chemotherapy regimens (combined with surgery and/or radiotherapy in solid tumours). This includes improved outcomes in some cancers with poor prognosis such as high-risk neuroblastoma (40% survival with highly intensive chemotherapy regimens including immunotherapy) and acute myeloid leukaemia (60% survival with intensive chemotherapy and allogeneic hematopoietic stem cell transplantation).
Patient survival has plateaued over the last five years or more for the poor prognosis malignancies. This plateau calls for innovative treatments with new mechanisms of action to eradicate resistant disease.
Innovative oncology drugs with new mechanisms of action are available today for adult cancers and are more effective than traditional drugs in several refractory malignancies. The field of drug development is currently rapidly expanding beyond well-studied areas like signalling pathways, to target patient’s immune system, as well as their unique genetic profile and metabolism.
However, access to innovative therapies remains insufficient and slow for children and adolescents with cancer. It has been estimated that only about 10% of children with non-curable relapsed or refractory malignancies have access to new, experimental drugs in clinical trials from which they could benefit.
“Development of Innovative Treatments” is one of the 7 key objectives of the SIOPE Strategic Plan, a European Cancer Plan for Children and Adolescents that was launched in the European Parliament in November 2015. In the US, the Cancer Moonshot aimed at accelerating research on pediatric malignancies and making more therapies available for children and adolescents.
This is why introducing safe and innovative treatments into standard care is a priority for everyone involved, or working in the field of childhood cancers, to ensure the best possible care and outcome for all children and adolescents with cancer.