Scientists in Newcastle have contributed to a breakthrough in research that could save the lives of thousands of young people affected by a cruel childhood cancer.
Neuroblastoma is a cancer of the nervous system that mainly affects children under the age of five. By the time it is discovered, it has often spread throughout the body and in such cases, nearly half of children die within five years of diagnosis.
Now an international team of researchers say they have discovered an important genetic marker in neuroblastoma tumors that is associated with worse outcomes for these young patients.
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The research, published in the Journal of Clinical Oncology, shows that changes in the ALK (anaplastic lymphoma kinase) gene of neuroblastoma are associated with a significantly worse prognosis for children with high-risk disease.
The discovery could represent a much-needed cure for such children, as the ALK can be attacked directly. It is hoped that this, in addition to chemotherapy, immunotherapy and radiotherapy, can help more of the highest-risk children survive.
Professor Deborah Tweddle, from the Newcastle University Center for Cancer and honorary advisor to the Newcastle Hospitals NHS Foundation Trust, led the UK portion of the study and said identifying the genetic marker could mean the children at greatest risk would be different. can be treated beyond the time of diagnosis.
She added: “The most exciting thing is that there are new treatments that target the ALK protein itself, which is used in other cancers that can now be used for patients with high-risk neuroblastoma with ALK gene abnormalities.
“This research is an excellent example of personalized medicine. By treating those patients with a genetic abnormality of ALK with an ALK inhibitor, we tailor the treatment to the individual tumor type of the patient.
“By combining an ALK inhibitor with the other treatments we currently provide for high-risk neuroblastoma, we hope to be able to cure more patients with this aggressive childhood cancer.”
(LR) Nathaniel, Alexander, Elish and Raphael Mohammedel
The breakthrough will be welcomed by families with first-hand experience of the devastation neuroblastoma can wreak.
Among them is Raphael Mohammed, 43, whose son Alexander was diagnosed with cancer five years ago.
Now 10 and in remission under the care of Prof Tweddle, the Gosforth schoolboy is living life to the fullest while enjoying fishing and outdoor activities with his family.
But for nearly two years, the Alexander had to battle through intensive treatment, including many aggressive cycles of chemotherapy at Great North Children’s Hospital.
Raphael, a doctor, said: “When Alexander was diagnosed with cancer it was such a shock and not something we expected because he didn’t have the classic symptoms of neuroblastoma.
“It was hard to watch him go through the intensive treatment but he was so brave and to see how well he is doing now is fantastic as he has been in remission for four years now.
“As a parent, you have concerns in the back of your mind that his condition could relapse and so it’s great to see neuroblastoma research being done at Newcastle University.
“Hearing about this major breakthrough in treating the condition for children offers real hope for personalized treatment options in the future so that more young people’s lives can be saved.”
Alexander Mohammed, 10 years old
Experts performed ALK testing on as many neuroblastomas as possible from patients treated in the high-risk study and pooled this data with those from national genetic reference labs from 19 other countries.
The study showed that ALK mutations are present in approximately 14% of patients newly diagnosed with high-risk neuroblastoma, and nearly 4% have amplification of this gene.
More importantly, these abnormalities are associated with a lower survival rate and are therefore important prognostic markers for targeting treatment.
Professor Tweddle added: “As part of the next European clinical trial for high-risk neuroblastoma, we will treat all patients with an ALK abnormality in their tumor with an ALK inhibitor in addition to standard treatment.”
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