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Neuroblastoma: developing a new treatment for young children with cancer

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What did the project achieve?

Researchers have identified new drug combinations that could lead to a safer, more effective treatment for a devastating childhood cancer.

In this three year study researchers led by Professor Robert Mairs tested a number of new drug combinations to help treat children with high-risk neuroblastoma. Their aim was to reduce the suffering associated with the side effects of existing treatments, and try and save more young lives.

At present, around a third of children lose their lives within five years of being diagnosed with neuroblastoma, like Felix who sadly died aged just six after enduring two years of gruelling treatment.

Professor Mairs, who is based at the Institute of Cancer Sciences in Glasgow, set out to enhance a treatment known as targeted molecular radiotherapy. In this approach, radioactive drugs which seek and destroy cancer cells are injected into the bloodstream. It can be used to treat cancers that have spread through the body and even destroy tumours that are too small to detect on scans.

Researchers tested new combinations of drugs, designed to make cancer cells more susceptible to radiation-induced damage. They found two drugs, known as radiosensitisers, which work well in combination with a radioactive drug.

Professor Mairs comments: “This holds promise as another component in the arsenal for the treatment of high-risk neuroblastoma.”

It is anticipated that clinical trials in children with this devastating disease will take place within two years.

This research was completed on

Around 100 children are diagnosed with a cancer called neuroblastoma each year in the UK.1,2 Most are young – less than five years old.3,4 Although treatment often proves life-saving, it has side effects and doesn’t always work. Sadly, around a third of children lose their lives within five years of being diagnosed with neuroblastoma.4 Professor Robert Mairs, of Glasgow’s Institute of Cancer Sciences, is developing a new combination treatment in the hope of reducing the suffering that’s associated with side effects and saving more children’s lives.

Action Medical Research and Neuroblastoma UK are together funding this study

How are children’s lives affected now?

“Neuroblastoma is normally diagnosed in babies and very young children,” explains Professor Mairs. “It is a cancer that starts in specialised nerve cells, often within the abdomen. First symptoms can be vague, so the cancer has often spread by the time it is diagnosed, meaning children then need immediate and intensive treatment.”

Standard treatment options include surgery, radiotherapy and chemotherapy. Children can experience unpleasant side effects during treatment, including nausea, tiredness and hair loss, as well as longer-term problems such as hearing loss, lowered fertility and heart problems.

A diagnosis of cancer can be devastating and those affected often worry about the future. With neuroblastoma, children’s chances of making a good recovery vary considerably. “Children with low-risk disease have an excellent chance of being cured,” says Professor Mairs. “Sadly, though, children with high-risk disease who do not respond to treatment or whose cancer recurs after treatment have a much worse outlook, with many going on to lose their lives.”


How could this research help?

“We aim to improve the treatment of children who are suffering from neuroblastoma,” says Professor Mairs. “We are combining a radioactive drug called 177Lu-DOTATATE – which seeks out and destroys cancer cells – with other drugs that make cancers more susceptible to radiation-induced damage.”

This type of treatment is called targeted molecular radiotherapy. It can be used to treat cancers that have spread through the body, even destroying tumours that are too small to detect by scanning.

“We are testing several different drugs with 177Lu-DOTATATE in the laboratory to find out which combination is the safest and the most effective,” says Professor Mairs. “The next step will be clinical trials in children with neuroblastoma.”

Neuroblastoma is the second most common solid tumour of childhood, after brain tumours.3,5 It makes up around eight per cent of children’s cancers.5 A safer and more effective treatment is urgently needed.



1. NHS Choices. Neuroblastoma. http://www.nhs.uk/conditions/neuroblastoma/Pages/Introduction.aspx Website accessed 25 June 2014.

2. The Neuroblastoma Society. Home page. http://neuroblastoma.org.uk/ Website accessed 25 June 2014.

3. The Neuroblastoma Society. Neuroblastoma FAQs. http://neuroblastoma.org.uk/families/neuroblastoma-faqs/ Website accessed 30 June 2014.

4. Cancer Research UK. Childhood cancer survival statistics. http://www.cancerresearchuk.org/cancer-info/cancerstats/childhoodcancer/... Website accessed 16 September 2014.

5. Macmillan. Neuroblastoma in children. http://www.macmillan.org.uk/Cancerinformation/Cancertypes/Childrenscance... Website accessed 26 June 2014.


Project Leader Professor Robert J Mairs DSc FRCR
Project Team Dr Mark Gaze MD FRCP FRCRDr Colin Rae PhD
Project Location Institute of Cancer Sciences, University of Glasgow
Project Location Other Department of Oncology, University College London Hospitals
Project duration 36 months
Date awarded 9 June 2014
Project start date 1 July 2014
Project end date 30 June 2017
Grant amount £179,874
Grant code GN2196


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