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Changing the future for children with cancer

A cancer diagnosis is a devastating time for people, their family and their friends.  It is particularly hard when the diagnosis is in a young child.  Here at Worldwide Cancer Research, we are always pleased to fund pioneering research projects looking to improve the lives of children with cancer to help reach our goal of no life cut short by cancer.

Do children really get cancer?

Each year in the UK 1,600 children are diagnosed with cancer.  Remarkable improvements in cancer medicine have more than doubled 10-year survival rates.  Despite this, more than 250 children die from cancer before their 15th birthday.  It is still a leading cause of childhood death in the developed world.

Worldwide Cancer Research has been funding research into childhood cancers for over 20 years.  We have already contributed to advances in some areas.  And we will continue to strive for better outcomes for children and their families.  This blog features some of these advances, but if you want to find out more about our current research projects, you can search for them using our interactive world map.


Leukaemia is the most common form of cancer in children under 14 years old.  Children almost always have one of the acute forms that progresses rapidly, requiring swift diagnosis and immediate treatment.  The good news is that most children with leukaemia are now cured outright. But this is the result of particularly harsh treatments.

Ten years ago, separate funding to Dr Aswin Menke, Radboud University, The Netherlands and Professor Hans Stauss, Imperial College London, helped them show that a protein produced inside leukaemia cells, WT1, could be used to direct the immune system to attack leukaemia cells.  WT1-targeted immunotherapy for leukaemia that has returned is now in the early phases of clinical trial.  The latest trial is enrolling children as well as adults.

Brain & Central Nervous System Tumours

Brain tumours are slightly less common in children, but are responsible for more deaths.  The most common types are glioma, which also affects adults, and medulloblastoma, which is almost always a childhood cancer.  There are also a number of very rare childhood brain tumours with some of the lowest survivability of all cancers.

Two successive grants in the early 2000s to Professor Paul Walker at the University of Geneva in Switzerland, helped make the case for immunotherapy against brain tumours.  By adapting a technique used previously to develop a vaccine against skin cancer, he was able to safely get immune cells to work against tumours in a mouse model of brain cancer. This work helped them design a clinical trial in glioblastoma, taking place in Geneva.  This has also, indirectly, led to a second trial, which started last year.  A range of other early clinical trials of immunotherapy for brain tumours is now also underway.  Although these initial clinical trials are largely recruiting adults it is expected that successful new therapies will ultimately be applied to children’s cancer too.

Inherited genetic disorders that cause cancer

Some inherited genetic diseases make children susceptible to many types of cancer.  Two of the most common are Fanconi’s Anaemia and Li-Fraumeni syndrome.  These disorders run in families.  It is important to identify the genes involved, so that family members can be screened.  This will allow those carrying the faulty gene to be monitored closely.

Grants given to Dr Niels Bentzen Atkin at Mount Vernon Hospital in London in the 1980s and early 1990s helped pin down the role of one of the most important genes in cancer, p53.  Without this understanding, we would not have genetic tests that diagnose Li-Fraumeni syndrome, in which p53 is defective.  And a grant awarded in 2004 to Professor Kevin Hiom at the University of Dundee discovered the FANCJ/BRIP1 gene, one of the genes responsible for Fanconi’s Anaemia, also now used to test for this condition.

Rare childhood cancers

There are also a number of rare childhood cancers that only affect a small number of children.  We have funded research into many of these, with some important results.

Neuroblastoma - a cancer of nerve cells that each year is diagnosed in around 100 children under five in the UK.  Over half of cases are the high risk, aggressive form, and many of these children relapse even after intensive treatment, in which case the outlook is poor.  Neuroblastoma is currently being featured on the popular UK soap Coronation Street, as a young girl called Hope was diagnosed this week.

Professor Martin Eilers at the University of Wurzburg, Germany identified the Aurora Kinase A (AURKA) enzyme as a highly promising drug target for high risk forms of neuroblastoma.  Drugs aimed at AURKA have been tested for other cancers, with disappointing results, but Professor Eilers showed that AURKA works quite differently in neuroblastoma.  He has uncovered strong evidence that AURKA drugs will work much better for neuroblastoma, and this is now being tested.

Wilms’ tumour - a rare type of kidney tumour usually diagnosed in children under 5.  Professor Michaela Aldred from the Cleveland Clinic, Ohio was able to narrow down the region on chromosome 2, which contributes to Wilms' tumour.  With this information, doctors can help parents, as they can give better informed advice about the possible risk of their child developing Wilms' tumour.

In addition, Professor Aldred identified the gene DIS3L2 to be within this region of chrosome 2.  At the same time, Professor Eamonn Maher at the University of Birmingham (he has since moved to the University of Cambridge), also funded by Worldwide Cancer Research, found that mutations to the DIS3L2 gene increased a child's risk of developing Wilms’ tumour.

Ewing’s sarcoma - a type of bone cancer predominantly affecting teenagers.  Professor Olivier Delattre’s work helped give us a better understanding of the cell that causes Ewing's sarcoma.  Since the end of our funding, his research has found a potential new way to treat this disease.

Retinoblastoma - a rare type of eye cancer that affects children under five.  Recent findings from Professor Sibylle Mittnacht’s grant at University College London has shown a new role for the RB1 gene in retinoblastoma.  Her results point to exciting prospects for new and more effective ways in which to treat this disease.

Malignant Rhabdoid Tumours - grants to Dr Agnes Klochendler at the Hebrew University, Israel and Professor Moshe Yaniv at Institut Pasteur, France in the early-mid 2000s confirmed that a mutation of the gene SNF5 drives rhabdoid tumour development.  Their findings make it possible to diagnose this disease by testing for gene mutations.  Swift and accurate diagnosis of these aggressive tumours is important to avoid delays in starting treatment.

Life is short, we never know how short, or who will be diagnosed next. But we do know that research is key to beating childhood cancer.  To help make a difference to children who are diagnosed in the future, please text WORLDWIDE to 70004 to donate £10.


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