Scientists at Newcastle have helped breakthrough research that could save the lives of thousands of young people affected by cruel childhood cancer.
Neuroblastoma is a cancer of the nervous system affecting mainly 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.
Today, an international team of researchers claims to have discovered a key 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 neuroblastoma ALK (anaplastic lymphoma kinase) are associated with a markedly poorer prognosis in children with high-risk disease.
The discovery could mean a much needed cure for these children, as ALK can be targeted directly. It is hoped that this, along with chemotherapy, immunotherapy and radiation therapy, could help more at-risk children survive.
Professor Deborah Tweddle, of the Newcastle University Center for Cancer and honorary consultant to the Newcastle Hospitals NHS Foundation Trust, led the UK part of the study and said identifying the genetic marker could mean children most at risk could be treated differently from the time of diagnosis.
She added: “Most exciting is that there are new treatments that target the ALK protein itself, used in other cancers that can now be used for high-risk neuroblastoma patients with gene abnormalities. ALK.
“This research is a great example of personalized medicine. By treating these patients with a ALK genetic abnormality with an ALK inhibitor, we tailor the treatment to the individual tumor type of patients.
“By combining an ALK inhibitor with the other treatments we are currently giving for high-risk neuroblastoma, we hope to be able to cure more patients with this aggressive childhood cancer.”
The breakthrough will be welcomed by families with first-hand experience of the devastation neuroblastoma can cause.
Among them is Raphael Mohammed, 43, whose son Alexander was diagnosed with cancer five years ago.
Now 10 years old and in remission in the care of Professor Tweddle, the Gosforth schoolboy is living his life to the fullest as he enjoys fishing and the outdoors with his family.
But for nearly two years, the Alexander had to struggle with intensive treatment that included numerous 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 as he did not have the classic symptoms of neuroblastoma.
“It was hard to see him undergo intensive treatment but he was so brave and to see how well he is now is fantastic as he has been in remission for four years.
“As a parent you have in mind the concern that his condition may relapse and that’s why it’s great to see neuroblastoma research being done at Newcastle University.
“Hearing about this major breakthrough in treating the disease in children offers real hope for personalized treatment options in the future so that the lives of more young people can be saved. “
Experts have realized ALK testing as many neuroblastomas as possible in patients treated in the high-risk trial and pooling this data with that from national genetic reference laboratories in 19 other countries.
The study showed that ALK mutations are present in about 14% of newly diagnosed patients with high-risk neuroblastoma, and almost 4% have this gene amplified.
More importantly, these abnormalities are associated with a lower survival rate and therefore are important prognostic markers that could be targeted by treatment.
Professor Tweddle added: “As part of the upcoming European clinical trial for high-risk neuroblastoma, we will be treating all patients with high risk neuroblastoma. ALK abnormality found in their tumor with an ALK inhibitor in addition to standard treatment.