General Public

Paediatric cancer research is a significant public health issue: some 15,000 children are diagnosed with cancer every year in Europe. Although approx. 80% can be cured today, thanks to the progresses made by the paediatric oncology research community, the remaining estimated 3,000 will not survive due to various hurdles and pitfalls in paediatric oncology.
The ENCCA network is dedicated to reducing these obstacles by laying the foundation for a virtual European institute for cancer research in children and adolescents, that will work in order to significantly improve cure and quality-of-cure in the future decades.


Main types of Paediatric Cancer

Paediatric cancer accounts for the second cause of death in children. As a parent, you need to be aware of the symptoms of childhood cancer: although your paediatrician is probably a very good doctor, he/she may have never diagnosed childhood cancer. You need to know to trust your own intuition when you feel that there is really something wrong with your child, even if the doctor initially interprets the symptoms as a common child ailment.

The main types of paediatric cancers encountered are leukaemias, nNeuroblastomas, Wilms’ tumors, brain cancers, rhabdomyosarcomas, lymphomas, retinoblastomas , osteosarcomas and Ewing's sarcomas.

  • Leukaemia is a cancer of the bone marrow, the spongy centre of the bones that makes blood cells. It accounts for approximately 35% of all childhood cancers. Although it is more common in children under the age of 10, approximately 1 in 1000 children are diagnosed with leukaemia by the age of 1.
  • Neuroblastoma is a disease in which malignant (cancer) cells form in nerve tissue of the adrenal gland, neck, chest or spinal cord. It accounts for 5%-7% of all childhood malignancies and usually occurs by the age of 5.
  • Wilms’ tumor is a type of kidney cancer also known as nephroblastoma. It accounts for 6-7% of childhood cancer cases. It occurs in about 8 in 1 million children under the age of 14, but is more common in children under the age of 5. Some children have a genetic tendency to develop Wilms’ tumour, usually because other family members have developed Wilms’ or there have been certain development problems from birth. 
  • Brain cancers account for 15% of paediatric cancers. The symptoms depend on the location of the tumour. The two main types of brain tumours that affect children are gliomas and medulloblastomas. Gliomas develop from the supporting cells of the brain, which hold the nerve cells in place, and brainstem gliomas occur almost only in children. The average age of development is about 6 years old.Medulloblastoma originates in the cerebellum or posterior fossa, and may spread to other parts of the brain or into the spinal cord. Most medulloblastomas occur before the age of 10.
  • Rhabdomyosarcoma is a fast-growing, highly-malignant soft-tissue sarcoma which develops in the muscle, mostly in the neck, head, bladder and testes. It accounts for 5-8% of childhood cancers. It usually affects children between the ages of 2 to 6 and 15 to 19.
  • Lymphomas are malignant cell infiltrations of the lymphatic system located in the neck, armpit, and groin. Lymphomas are broadly classified as Hodgkin's and non-Hodgkin's. The two are distinguished by cell type. Hodgkin's generally occurs in individuals between 15-40 years of age.
  • Retinoblastoma is a type of eye cancer that occurs in the retina of one, or both eyes. It is the most common eye tumour in children. Retinoblastoma accounts for 3-4% of all childhood cancers. It usually occurs before the age of 5
  • Osteosarcoma and Ewing's sarcoma are the most common malignancies of bone tissues in children. Osteosarcoma is usually presents in bones around the knee whereas Ewing's sarcoma may affect bones of the pelvis, thigh, upper arm, or ribs. Bone cancers are most common in ages 10-20 and they account for about 6% of all childhood cancers.

Current challenges of clinical research in children

Current issues in paediatric oncology:

  • Access to drugs developed by industry remains limited and difficult, even though the EU Paediatric Medicines Regulation (1901/2006) improved the framework for drug development in children
  • Clinical research still needs to improve its harmonisation across informal paediatric oncology groups in Europe
  • Biological knowledge is insufficiently linked to clinical research, despite the existence of internationally recognised research laboratories in the field of biology and pharmacology
  • Financial resources for running clinical trials are rather limited and fragmented among national research funding programmes, charity donations and private donors
  • Existence of major cultural differences in Europe in terms of ethics and attitudes towards experimental therapy
  • Although the Paediatric Medicines Regulation encourages pharmaceutical industry to develop paediatric drugs, it is unlikely that all these efforts can be funded by private industry alone

Importance of paediatric clinical trials in children

“It is clear that paediatric clinical research should not be considered a luxury but rather a necessary tool to combat the burden of cancer”

Paediatric clinical trials are essential to find a cure for sick children. Unfortunately, 50-90% of treatments prescribed today have not been tested and approved for children and, in such cases, clinicians are forced to extrapolate the treatment from studies' results in adults. This extrapolation is often inappropriate, as children have a different range of diseases and metabolise medications differently, resulting in responses to treatment that are unpredictably different to adults. In addition, because the tumour conditions is rare in children and parents make decisions about trial participation on behalf of their child, the pool of eligible children entering clinical trials is often small, and the procedure for gaining consent very complex.

On a more positive note, children enrolled in clinical trials with life-threatening diseases such as cancer have often a better outcome, as there is an increased adherence to the treatment protocol.

Paediatric oncology clinical trials are therefore key to encourage advances in treatments, quality-of-care and innovative practices for children with cancer. ENCCA is working to improve the current paediatric pitfalls and hurdles, towards an improved and harmonised standard of care and cure in Europe.


To these ends, ENCCA will take the following actions:

  • Structure and integrate clinical and translational research for children and young people with cancer on a European scale, to create a long-term ‘sustainable platform’ which will be a test-bed for clinical researchers
  • Promote innovative methodology and designs for clinical trials, taking into account specific needs of rare diseases
  • Raise the standards of care by increasing patients' access to information, by raising awareness schemes within all age-categories and reaching out to less advantaged areas, improving patients’ standard of care referral schemes and promoting healthy lifestyles for childhood cancers' survivors
  • Facilitate knowledge-sharing across disciplines and stakeholders, providing specialised training programmes to enhance existing paediatric oncology knowledge
  • Enhance information-flow and facilitate partnership with pharmaceutical companies and industry at large
  • Substantially improve the quality-of-life of children and adolescents with cancer, with a special emphasis on future less aggressive therapies and potentially reduced long-term side effects
  • Address and monitor all ethical issues related to the participation of children and adolescents with cancer in clinical research, with a view to bridging existing information gaps.