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Chemotherapy Protocol Proves Worth for High Risk Children’s Cancer



Monday 4 July 2011



High risk Neuroblastoma is one of the hardest cancers to treat in children.  An 18 country Europe-wide study has demonstrated the benefit of the new European high dose chemotherapy protocol.


The trial was funded by Cancer Research UK, the Children’s Cancer Research Institute in Vienna, and also by the European Commission Framework Five. The research was led by Great Ormond Street Hospital, The Royal Marsden and The Institute of Cancer Research (ICR) in the UK, and international collaborators.

As reported to this month’s American Society of Clinical Oncology conference (ASCO), the European protocol offered 16 per cent better disease free survival at three years compared with a treatment based on the current American protocol.  It has also proved to have fewer toxic side effects.  From the end of last year, when these figures were validated, the randomised controlled trial was halted on ethical grounds so all European patients could receive the better treatment.

Figures reported were 49 per cent v 33 per cent Event Free Survival and 61 per cent v 48 per cent overall survival at three years.

Peppy Brock, UK lead for the trial, consultant paediatric oncologist at Great Ormond Street Hospital and President of SIOPEN (European Neuroblastoma Group of the International Society for Paediatric Oncology) said:

“It has taken since 2002 to prove the advantages of the European protocol.  It could never have been achieved without pan European co operation, since no one country has large enough numbers to run an effective trial in a realistic time frame. The high dose regime was pioneered in France, the study chair is Austrian, and the UK enters the largest number of patients onto this trial.

“This trial predates the introduction of immunotherapy which this group of children are also now benefiting from. We are now beginning to see real improvements in survival, even in this very high risk group of children, right across the UK and Europe. Funding is now needed to ensure that we can develop this work and continue to make progress against this particularly difficult cancer in children.”


Senior trial investigator Professor Andrew Pearson, from the ICR and The Royal Marsden, added: "Neuroblastoma is one of the most common types of childhood cancer in the UK and high risk Neuroblastoma is one the major causes of death in children from malignancy.  

"In this study, we found that the combination of chemotherapy drugs given for consolidation therapy used in Europe led to children living longer on average than a regimen based on the US standard. This very conclusive result will alter the way that doctors treat their patients world wide, and will mean fewer children die from this disease each year. 

“These chemotherapy drugs - busulphan and melphalan - were actually discovered and developed at The Institute of Cancer Research in the 1950s, so it is especially pleasing that drugs discovered here in the UK so many decades ago may now make a difference to the lives of children all over the world."

Julie Hearn, head of clinical trials at Cancer Research UK, said: “The results of this study offer new hope for children diagnosed with high-risk Neuroblastoma. It shows how drugs Cancer Research UK were at the forefront of developing 30 or 40 years ago have stood the test of time and are still proving useful today. But while we are making progress in treating childhood cancers, today almost 80 per cent survive, there is an urgent need to develop new treatments for hard to treat cancers.”




For further information, including case studies, please contact Hayley Dodman, Great Ormond Street Hospital press office on 0207 239 3126 or email [email protected], or ICR Science Communications Manager Jane Bunce on 0207 153 5106


Notes to editors

The control group received the current American high dose protocol but had different initial treatments.  We note that the control group had worse outcomes than those reported on the complete American regime. The detailed analysis of these differences is being undertaken on both sides of the Atlantic and will be taken into consideration in the design of further trials. There are many aspects which need to be taken into consideration but it is clear that in European hands the better regimen is now being taken forward for all children.


Thanks to: Children’s Cancer Research Institute in Vienna, the SIOPEN (European Neuroblastoma Group of the International Society for Paediatric Oncology) Cancer Research UK, all UK Paediatric Oncology Centres, the Birmingham Clinical trials centre.


Great Ormond Street Hospital is, with the UCL Institute of Child Health, the largest centre for paediatric research outside North America, and a crucial centre for the training and education of paediatric staff  It has the largest range of paediatric specialists in one site in the UK.


The Institute of Cancer Research (ICR)  

•       The ICR is Europe’s leading cancer research centre.

•       The ICR has been ranked the UK’s top academic research centre, based on the results of the Higher Education Funding Council’s Research Assessment Exercise.

•       The ICR works closely with partner The Royal Marsden NHS Foundation Trust to ensure patients immediately benefit from new research. Together the two organisations form the largest comprehensive cancer centre in Europe.

•       The ICR has charitable status and relies on voluntary income, spending 90 pence in every pound of total income directly on research.

•       As a college of the University of London, the ICR also provides postgraduate higher education of international distinction.

•       Over its 100-year history, the ICR’s achievements include identifying the potential link between smoking and lung cancer which was subsequently confirmed, discovering that DNA damage is the basic cause of cancer and isolating more cancer-related genes than any other organisation in the world.

•       The ICR is home to the world’s leading academic cancer drug development team. Several important anti-cancer drugs used worldwide were synthesised at the ICR and it has discovered an average of two preclinical candidates each year over the past five years. 

For more information visit

The Royal Marsden Hospital
The Royal Marsden was the first hospital in the world dedicated to cancer diagnosis, treatment and research into the causes of cancer. Today, as The Royal Marsden NHS Foundation Trust, together with its academic partner The Institute of Cancer Research, form the largest comprehensive cancer centre in Europe with over 40,000 patients from the UK and abroad seen each year. It is a centre of excellence, and was the only NHS Trust to achieve the highest possible ranking in the Healthcare Commission’s Annual Health Check for four year in a row. Prince William became President of The Royal Marsden in 2007, following a long royal connection with the hospital.

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