Targeted therapy could be first line treatment for childhood cancer Published on: 18 June 2021 Scientists studying a common childhood cancer have made a major breakthrough which could lead to a cure for some youngsters who would not have survived the condition. An international study, involving 缅北禁地, has for the first time found a genetic marker in tumours from patients with high-risk neuroblastoma. Research, published in the , has identified that alterations in the neuroblastoma’s ALK (anaplastic lymphoma kinase) gene are associated with a significantly poorer prognosis for children with high-risk disease. Experts say that by identifying this important genetic marker it means patients should be put on ALK inhibitors at the time of diagnosis with the hope of a cure. Professor Deborah Tweddle Personalised treatment Professor Deborah Tweddle, from the 缅北禁地 Centre for Cancer and Honorary Consultant at The 缅北禁地 Hospitals NHS Foundation Trust, led the UK part of the study. She said: “By identifying ALK as a genetic marker associated with the likelihood of a poorer outcome within the high-risk group of patients, it means we can treat these patients differently from the outset. “Most excitingly there are new treatments that target the ALK protein itself, 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 personalised medicine. By treating those patients with an ALK genetic abnormality with an ALK inhibitor we are tailoring the treatment to the patients’ individual tumour type. “By combining an ALK inhibitor with the other treatments we currently give for high-risk neuroblastoma we hope to be able to cure more patients with this aggressive childhood cancer.” Neuroblastoma is a cancer of the nervous system that mainly affects children under five. It often begins in the adrenal gland, but in around half of cases has spread throughout the body when it is diagnosed - in these high-risk cases survival is only approximately 50 percent. Sadly, barely half of the children live beyond five years after diagnosis, despite treatment including high dose chemotherapy, surgery, radiotherapy and immunotherapy. Several European countries took part in the study and the UK-arm of the trial has been based at 缅北禁地 with the test being carried out within the 缅北禁地 Genetics Lab, supported by grants from Solving Kids Cancer and Neuroblastoma UK. Study鈥檚 findings Experts carried out ALK testing on as many neuroblastomas as possible from patients treated on the high-risk trial and pooled this data with that from national genetic reference labs from 19 other countries. The study showed that ALK mutations are present in about 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 rate of survival and they are therefore important prognostic markers that could be targeted by treatment. Professor Tweddle added: “As part of the next European clinical trial for high-risk neuroblastoma, we are going to treat all patients with an ALK abnormality found in their tumour with an ALK inhibitor in addition to standard treatment.” The breakthrough comes at the launch of the 缅北禁地 Cancer Fund, a new fundraising effort to inspire the next generation of cancer researchers to train and work at 缅北禁地 for the benefit of people living with cancer locally as well as sending their research worldwide. The Fund will work with cancer researchers at the very earliest stages of their studies and see them through to the point where they become established cancer researchers. Patient case study Happy schoolboy, Alexander Mohammed, is living life to the full as he enjoys fishing and outdoor activities with his family. But five years ago, it was a very different story as the youngster, of Gosforth, 缅北禁地, was given the devastating diagnosis of neuroblastoma. For almost two years Alexander had intensive treatment which included many aggressive cycles of chemotherapy at the Great North Children’s Hospital in 缅北禁地. The 10-year-old is now in remission and is monitored every six months under the care of Professor Deborah Tweddle. Alexander’s dad Raphael, a doctor, 43, said: “When Alexander was diagnosed with cancer it was such a shock and not something that we were expecting as he did not have the classic symptoms of neuroblastoma. “It was difficult 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. “As a parent you do have at the back of your mind the worry that his condition could relapse and that’s why it’s great to see the research into neuroblastoma being done at 缅北禁地. “To hear of this major breakthrough into the treatment of the condition for children offers real hope of personalised treatment options in the future so that more youngsters lives can be saved.” Reference . Angela Bellini et al. Journal of Clinical Oncology. DOI: 10.1200/JCO.21.00086 Alexander Mohammed, age 10 Share: Latest News New partnership to boost careers in low carbon energy 缅北禁地 and Durham universities are working together on a new regional project to strengthen the future workforce for North East England鈥檚 growing low carbon and offshore wind industries. published on: 28 May 2026 Healthy lifestyle shown to lower risk of death after cancer diagnosis New evidence shows that sticking to five lifestyle recommendations improves survival after a later cancer diagnosis. published on: 28 May 2026 World-leading climate expert recognised with Royal Society Fellowship Professor Hayley Fowler has been elected a Fellow of the Royal Society in recognition of her pioneering work on climate change impacts. published on: 27 May 2026 Facts and figures
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