A wide range of familial syndromes are associated with an increased risk of developing childhood cancer (see molecular biology and genetics section).1

Genetic testing for retinoblastoma has been adopted as the standard procedure for families with a history of the disease.2 Genetic tests are also available for some inherited conditions such as Neurofibromatosis type 1 (NF1) and Multiple Endocrine Neoplasia type 2 (MEN2); once diagnosed, children can then be monitored for the development of specific cancers such as CNS and soft tissue sarcomas in the case of NF1, and medullary thyroid carcinomas for MEN2). 1

Population screening of infants for neuroblastoma began in parts of Japan in 1984, but was later discontinued after studies in Germany and Canada showed little beneficial effect on the mortality of the disease. 3

Over the last four decades there have been major advances in the development of successful treatment strategies for childhood cancers, and much of this has been due to the use of standardised protocols in clinical trials and centralisation of care.

The first UK multi-centre childhood cancer clinical trial was set up for acute lymphoblastic leukaemia (ALL) in 1969 by the Medical Research Council Childhood Leukaemia Working Party (CLWP). The International Society of Paediatric Oncology (SIOP) was also established in Europe in 1969, and several childhood cancer clinical trials organisations were set up in North American and it became clear that a new national organisation trials was needed to bring together expertise and resources in the UK. The UK Children’s Cancer Study Group (UKCCSG) was established in 1977 and by 2003 around 30 working groups had been set up for specific tumour types and other disciplines.

Together the UKCCSG and CLWP enabled a comprehensive portfolio of national and international clinical trials to be developed. The recruitment of children into clinical trials increased substantially since the 1970s with around 70% of UKCCSG patients being entered into a trial by the late 1990s; 4,5 nowadays the proportion is likely to be higher.

The Children’s Cancer and Leukaemia Group (CCLG) was established in 2006 following the merger of the UKCCSG and the CLWP. The CCLG currently has more than 700 members from many disciplines including clinicians, pathologists, epidemiologists and scientists; these members have been instrumental in improving the survival rates of children with cancer and are continuing to drive forward research in this area.

Until recently, the CCLG Data Centre was co-ordinating around 30 clinical trials, observational studies and audits. From April 2010, the responsibility for running clinical trials within the CCLG network and developing new trials transferred to the newly formed Children’s Cancer Trials Team (CCTT), part of the Cancer Research UK Clinical Trials Unit (CTU) at the University of Birmingham. 6 The CTU has a wealth of expertise in designing and running clinical trials in adults, particularly innovative trials for rare tumours and setting up international collaborations(both of which are key areas for CCLG trials).

The CCTT is currently co-ordinating 10 clinical trials and has recently secured funding for a further four; more trials are also being developed. The recently formed NCRI Children’s Cancer and Leukaemia Clinical Study Group also aids the development of a new trial and study ideas and oversees the portfolio of clinical research into children’s cancer. 7

The establishment of the UKCCSG in 1977 brought about a big shift towards treating childhood cancers in specialist centres, and this has benefitted children with cancer enormously. There are a currently 21 such specialist centres forming a close-knit network across the UK and Ireland;each is recognised as a centre of excellence and children are treated by multidisplinary team of cancer specialists with experience of treating the very specific tumour types that occur in children.8

The children’s cancer treatment centres in the UK and Ireland are: Royal Aberdeen Children’s Hospital; Royal Hospital for Sick Children, Belfast; Birmingham Children’s Hospital; Royal Hospital for Sick Children, Bristol; Addenbrooke’s Hospital, Cambridge; Children’s Hospital for Wales, Cardiff; Royal Hospital for Sick Children, Edinburgh; Royal Hospital for Sick Children, Glasgow; St.James’s University Hospital, Leeds; Leicester Royal Infirmary; Alderhey Children’s Hospital, Liverpool; Royal Manchester Children’s Hospital; Great Ormond Street Hospital, London; University College London Hospital; Royal Victoria Infirmary, Newcastle; Queen’s Medical Centre, Nottingham; John Radliffe Hospital, Oxford; Sheffield Children’s Hospital; Southampton General Hospital; Royal Marsden Hospital, Sutton; Our Ladies’ Hospital for Sick Children, Dublin.

The mainstay of treatment for most childhood cancers is surgery, chemotherapy, radiotherapy and combinations of any or all of these (Table 6.1).

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High-dose chemotherapy or total body irradiation is often accompanied by stem cell or bone marrow transplantation. Other new therapies, such as immunotherapy for neuroblastoma, are being developed.8-10

The Cancer Reform Strategy clearly acknowledges that survivors of cancer (of all ages) have particular needs, 11 and one of the key initiatives of the strategy is the National Cancer Survivorship Initiative (NCSI). 12 The NCSI aims to improve the ongoing services and support for those living with, and beyond, cancer and has a specific work stream focussing on the unique needs of survivors of childhood and young people’s cancer.

Dramatic improvements in the treatment and management of childhood cancer since the 1960s mean that many children diagnosed with cancer today have an excellent chance of being cured of their disease. Currently more than seven out of ten children diagnosed with cancer are successfully treated, compared with fewer than three in ten in the late 1960s. However, it is not forgotten that more than two out of ten children do not survive, and despite best efforts, a small number of tumour types with poor prognosis (such as certain types of brain tumours and metastatic neuroblastoma) still remain. Developing treatment strategies for these tumours continues to be a major challenge for researchers. As more and more people are surviving their childhood cancers, the long-term effects of treatments suffered by a significant proportion of survivors are also becoming increasingly important. Developing treatments with minimal negative effects (to achieve “cure at least cost” rather than “cure at any cost” 13) is also an important research focus for the future.