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First stage study finds potential new drugs for childhood cancer


A video summary accompanies this story.


20 June 2013


Scientists at The Institute of Cancer Research, London have made a step towards new, targeted drugs for children with the highest-risk form of a childhood cancer called neuroblastoma.

In a pre-clinical study, published in journal Cancer Cell today (20 June), researchers successfully used two potential drugs to target cancerous neuroblastoma cells with an overactive gene called MYCN. The gene plays a major role in most cases of aggressive and treatment-resistant neuroblastoma in children, as well as in some adult cancers.

In the study, scientists showed that two drugs, from a class called Aurora kinase inhibitors, disrupted growth in laboratory cultures of human cancer cells with overactive MYCN. They then showed that the drugs were effective against neuroblastoma tumours in mice with tumours caused by increased activity of MYCN. The drugs had a significant therapeutic effect against rapidly growing, aggressive neuroblastomas, as a result of the destruction of N-Myc, the protein produced by the MYCN gene.

The study was funded by the charity Christopher’s Smile, the Neuroblastoma Society, the Sparks Medical Charity, and the Medical Research Council.

Neuroblastoma is a cancer of developing nervous tissue which almost always begins very early in life. Usually causing tumours to develop in the chest or abdomen, it affects around 100 children in the UK each year (1), accounting for about 6 per cent of childhood cancers. It disproportionately affects very young children, making up more than one in five cancers diagnosed in infants under one year of age (2). Around half of children with neuroblastoma have a high-risk type which is resistant to all current treatments. Almost all high-risk patients have overactive MYCN.

The drugs are called MLN8054 and MLN8237. They work by causing the breakdown of the protein produced by the MYCN gene, called N-Myc, in cancer cells. Over-production of N-Myc drives other, pro-cancer genes to produce proteins which make tumours particularly resistant to current treatments.

MYCN has been known to scientists as a potentially important cancer target, but after many early, unsuccessful attempts it was widely considered to be undruggable. In this study, scientists targeted MYCN in an indirect way: MLN8054 and MLN8237 prevent N-Myc from binding to a stabilising protein called Aurora-A. Without this interaction, N-Myc is targeted for destruction in cancer cells.

Dr Louis Chesler, Head of the Paediatric Solid Tumour Biology and Therapeutics team at The Institute of Cancer Research, who co-led the study, said:

“High-risk neuroblastoma is very frequently fatal, even after the most intensive possible treatment with our most powerful drugs. It’s essential to find more effective and curative therapies.

“Our small but promising study has made a step forwards by finding a practical way to target the MYCN cancer-causing protein in children with this devastating disease. This has been a long-sought after, but so far unachieved goal.

“However, much work remains to establish that MYCN targeted drugs are safe and effective for children in the clinic. Our next step will be to trial these drugs in children who aren’t responding to conventional treatments, and those whose disease has relapsed.

“We are very hopeful that our work will help towards a new clinical strategy to directly target cancer-causing genes in childhood cancer, using drugs designed specifically for children.”

Karen Capel, co-founder and trustee of Christopher’s Smile, which co-funded the study, said:

“Christopher’s Smile is pleased to fund cutting edge research which utilises targeted agents to treat cancers such as high risk neuroblastoma. Our strategy is to fund projects that will enable personalised medicine for children with cancer to save the 25 per cent of children who do not survive their disease.”

Dr Guy Blanchard, Research Trustee at The Neuroblastoma Society, which co-funded the study, said:

“We welcome this exciting new development in the hunt for a treatment for MYCN-amplified neuroblastoma, one of the most aggressive and untreatable forms of the disease. We congratulate Dr Chesler and hope that Aurora-A inhibitors can be brought to trial as soon as is reasonably practicable.”

John Shanley, chief executive of Sparks, which co-funded the study, said:

“Funding research that helps prevent, diagnose, treat and cure conditions affecting the health of children is at the heart of what Sparks does.

“Neuroblastoma is a very unpredictable form of childhood cancer which can be difficult to treat, many of the current treatments are not effective and aggressive therapies can cause serious side effects for the children.

“We welcome this new research, which should help researchers to develop more effective, targeted therapies – meaning that the harsh side effects of more aggressive treatments are minimised.”



Notes to editors

For more information contact the ICR press office on 020 7153 5312 / [email protected]. For enquiries out of hours, please contact Claire Bithell, ICR’s Head of Media Relations, on 07969 082 520.

  1. University of Oxford Childhood Cancer Research Group: Childhood Cancer Registrations, Great Britain, 1971-2005 (2001-5). Accessed online 19 June 2013.
  2. Cancer Research UK Childhood cancer incidence statistics: 2008-10. Accessed online 18 June 2012.


The Institute of Cancer Research, London, is one of the world’s most influential cancer research institutes.

Scientists and clinicians at The Institute of Cancer Research (ICR) are working every day to make a real impact on cancer patients’ lives. Through its unique partnership with The Royal Marsden Hospital and ‘bench-to-bedside’ approach, the ICR is able to create and deliver results in a way that other institutions cannot. Together the two organisations are rated in the top four cancer centres globally.

The ICR has an outstanding record of achievement dating back more than 100 years. It provided the first convincing evidence that DNA damage is the basic cause of cancer, laying the foundation for the now universally accepted idea that cancer is a genetic disease. Today it leads the world at isolating cancer-related genes and discovering new targeted drugs for personalised cancer treatment.

As a college of the University of London, the ICR provides postgraduate higher education of international distinction. It has charitable status and relies on support from partner organisations, charities and the general public.

The ICR’s mission is to make the discoveries that defeat cancer. For more information visit  


Christopher’s Smile

Christopher’s Smile was set up in October 2008 after the death of Karen and Kevin Capel’s only child, Christopher in June the same year to an aggressive brain tumour after a 21 month battle with the disease. He was 9 days away from his 6th birthday. The charity funds the testing and development of new targeted drugs to treat medulloblastoma and other childhood cancers without devastating side effects. The charity supports world class UK research centres such as The Institute of Cancer Research, London.


The Neuroblastoma Society

The Neuroblastoma Society was formed in October 1982 by a group of parents whose children were suffering from or had died from Neuroblastoma. The Trust Deed formally creating the charity was signed on 20 May 1983. The Society is registered as a national charity 326385 with the Charity Commission for England and Wales.

The charity's sole purpose is the relief of children suffering from Neuroblastoma, and to achieve this it raises funds for medical research into improving both diagnosis and treatment of the disease. The Society also offers an opportunity for parents to give each other mutual help, support and comfort.


About Sparks

As a leading children’s medical research charity we are dedicated to funding and promoting pioneering research into a wide range of conditions and disabilities affecting babies, children and mums-to-be.

Sparks has now funded over 250 research projects in more than 80 hospitals and universities across the UK. We have committed over £24 million into pioneering research projects across a wide spectrum of medical conditions relating to problems in pregnancy, premature birth, the natal period and the early years of life. The charity also supports research into rare childhood diseases.

Through our research, we aim to improve the quality of life for children and families affected by serious illness or disability today, whilst seeking ways to better diagnose, treat and prevent these conditions in the future.

Our working is making a difference for thousands of children and their families, not only in the UK but globally too.

For more information visit

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