A type of drug used to treat cancers in adults may be useful in treating children with neuroblastoma

  • A type of drug used to treat some breast and ovarian cancers called a PARP inhibitor may prove useful for children with high-risk neuroblastoma, a common childhood tumour with a low survival rate (around 50%)
  • This is one of the first studies to show positive effects in neuroblastoma cells and the first time it has been shown in complex models of neuroblastoma
  • The study showed that PARP inhibitors kill neuroblastoma cells with MYCN in the laboratory
  • The study, funded by Children with Cancer UK, was recently published in Oncotarget. 
Neuroblastoma is a cancer which arises from nerve cells in either the chest or the abdomen. In some neuroblastoma cases there is an abnormal gene called MYCN that makes it particularly difficult to treat. Currently children receive intense chemotherapy, radiotherapy and surgery but despite this only about half of children with high-risk neuroblastoma will survive. Researchers at the University of Sheffield collaborated with The Institute of Cancer Research, London, to conduct research to find a new, more effective and less toxic way to target high-risk neuroblastoma tumours with increased levels of MYCN. MYCN affects how tumour cell cells grow and makes them more dependent on a protein called PARP for survival. This means that the tumour cells are specifically killed by a drug that stops PARP working. An advantage to this type of therapy is that normal non-tumour cells are not harmed so the damaging side-effects often seen with chemotherapy are reduced. The study, funded by childhood cancer research charity Children with Cancer UK, was led by Dr Helen Bryant and Dr David King in Sheffield, in collaboration with Professor Louis Chesler in London. They found that the use of PARP inhibitors alone, and alongside chemotherapy, was effective in killing neuroblastoma cells, leaving behind healthy cells. This is one of the first studies to show this in neuroblastoma cells and in tumour models. Dr David King, children’s doctor at Sheffield Children’s NHS Foundation Trust and Children with Cancer UK researcher, said:
Children with neuroblastoma and MYCN currently receive some of the most intense treatment used for any type of cancer. The harsh treatment causes numerous side effects and children often have long hospital stays. And despite the treatment, only about half of children with high-risk neuroblastoma will survive. PARP inhibitors are known to have very few side effects in adults and we have shown they may be an effective treatment for neuroblastoma. Their use in neuroblastoma could mean more children survive the disease and need less toxic treatment. We have shown that PARP inhibitors kill neuroblastoma cells with MYCN in the laboratory. The next step is to see whether they are beneficial in children with neuroblastoma, probably in combination with chemotherapy or radiotherapy. This would be done in a clinical trial.
Dr Helen Bryant, research group lead and senior lecturer at the University of Sheffield, said:
Our study has examined the way DNA is damaged and repaired – one of the features common in all cancers is that there are changes in that process. We were interested in how that process changes in neuroblastoma and whether we can exploit those changes to specifically kill cancer cells and leave the normal cells around the tumour unharmed. In this way we hope to develop kinder and less toxic therapies for children. PARP inhibitors are being used successfully in women with breast and ovarian cancer so we are optimistic that this can be translated fairly quickly into children with neuroblastoma.
Mark Brider, Chief Executive Officer, Children with Cancer UK, said:
Neuroblastoma is one of the most common childhood tumours with around 100 children, mostly under five years old, diagnosed every year in the UK. Yet it also has one of the lowest survival rates – in its high-risk form the survival rate is sadly around just 50%. It remains one of the most difficult childhood cancers to cure. It is crucial that we find more effective and personalised treatments for children with neuroblastoma. The findings of Dr Bryant, Dr King and the teams in Sheffield and London represent an important step towards the development of new and kinder treatments that reduce the burden of toxicity for young cancer patients and improve survival rates in this aggressive and hard-to-treat cancer.
Case study Alice Latham, 3, from Derbyshire, was diagnosed with high-risk neuroblastoma, with the MYCN gene, a week before her second birthday in January 2019. Her parents, Jamie and Helena, explain the harmful side-effects of Alice’s treatment.
Alice’s neuroblastoma treatment has been very aggressive and harsh on her body, she has experienced many side effects, including hair and weight loss. The treatment may also have an impact on her later in life – she may not grow to her full height and there is a chance she may not be able to carry children. If treatments could be developed that are less harmful to children’s growing bodies that would be incredible, said Helena.
Jamie added:
As a parent you know nothing about neuroblastoma – you understand when you get the diagnosis that the prognosis for the high-risk form is 40-50% survival after all of the treatment. The research for this type of cancer has really lagged behind and the treatment is very hard hitting. It is vital for charities like Children with Cancer UK step in and continue to fund this lifesaving research.
Editors’ Notes Children with Cancer UK’s press office E: media@childrenwithcancer.org.uk T: 0207 404 0808 M: 07 795 956 342 About Children with Cancer UK Children with Cancer UK is the leading national charity dedicated to research into childhood cancer. We fund research into the causes and treatment of childhood cancers and provide support for families affected by childhood cancer. We have accelerated breakthroughs to improve childhood cancer survival rates and find kinder, more effective treatments with fewer toxic side effects. This ground-breaking research, which would otherwise go unfunded, saves the lives of children with cancer. About childhood cancer and Children with Cancer UK’s impact Every day in the UK, 12 children and young people are diagnosed with cancer. Fifty years ago, only 30% of children with leukaemia survived, and for most other forms of childhood cancer survival rates were even lower. Today, thanks to our supporters and the dedication of visionary researchers like those we fund, more than 80% of young patients can be successfully treated. More vital research is needed though as there are still a number of cancers affecting children and young people with low survival rates and life-limiting side effects. Cancer remains the single largest cause of death from disease in children and young people in the UK.
Girl with no hair and feeding tube wearing a blue denim jacket
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