A new experiment has discovered that children with neuroblastomas, a rare kind of cancer that arises in nerve cells, may benefit from anti-tumor medications as well as chemotherapy.
The BEACON trial, conducted by the Cancer Research UK Clinical Trials Unit at the University of Birmingham, discovered that combining anti-angiogenic drugs, which prevent tumors from forming blood vessels, with various chemotherapy drugs resulted in more young people seeing their tumors shrink, from 18% in the control group to 26% among those on Bevacizumab.
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The findings were published in the Journal of Clinical Oncology. The experiment randomized 160 young people aged 1 to 21 from 43 hospitals in 11 European nations, with half getting the anti-angiogenic medication Bevacizumab in addition to standard care. Bevacizumab increased the odds of responding to treatment from 18% in those who only received the established medication to 26% in those who received the extra medicine. Patients who received Bevacizumab also exhibited higher one-year progression-free survival rates.
The trial was one of many cooperation between the University of Birmingham and European expert organizations SIOPEN (International Society of Paediatric Oncology European Neuroblastoma) and ITCC (Innovative treatments for children with cancer).
“We are delighted that the BEACON trial has helped to shape treatment for children with relapsed and refractory neuroblastoma going forward.” – Professor Simon Gates
Simon Gates, Professor of Biostatistics and Clinical Trials at the University of Birmingham and the paper’s senior author, stated:
“These are very exciting findings that will hopefully bring us closer to finding treatments for children with neuroblastomas.” Currently, the prognosis for children with this terrible cancer are quite bad, so even seemingly minor increases in the likelihood that a patient will be able to decrease their tumors are substantial.
“We are delighted that the BEACON trial has helped to shape treatment for children with relapsed and refractory neuroblastoma going forward.”
Dr. Lucas Moreno, Chief Investigator for the study and Head of Pediatric Hematology and Oncology at Vall d’Hebron University Hospital in Barcelona, Spain, stated:
“BEACON was a hypothesis-generating trial that identified active regimens that are now being investigated further.” We are overjoyed that the evidence collected has been integrated into the current UK Clinical Practice Guidelines, and that Bevacizumab is now standard treatment for relapsed neuroblastoma.”
Case Study: “It was our last hope”
Abdullah Mir’s parents claim he owes his life to a clinical study after being given a poor chance of survival after being diagnosed with a rare malignancy.
Bushra and Yasir Ali Mir recall being called aside and told that their four-year-old son Abdullah had neuroblastoma, a rare cancer that starts in early nerve cells.
After two rounds of chemotherapy treatments failed to work, Abdullah was invited to participate in the BEACON experiment.
“It was our last hope,” said Bushra, who had just graduated from primary school when her kid was diagnosed with leukemia in November 2017. “We were desperate because he’d had two rounds of chemotherapy that had done nothing to shrink his tumor.”
“There were no other options so we signed up thinking that, even if it didn’t benefit Abdullah, it might help someone else.”
For ten-year-old Abdullah, a keen player and Manchester United supporter who attends Shirelands Technology Primary School in Sandwell, the eight-month treatment nightmare is now a distant memory.
“Abdullah has always been very strong and positive but he does remember treatment and it was a very difficult time,” Bushra recounted. “He was very sick.” “Year three was his first full year at school due to COVID, but he’s doing really well.” He’s a football fanatic who really enjoys school.”
Dr. Laura Danielson, Children and Young People’s Research Lead at Cancer Research UK, which contributed to the trial’s funding, stated:
“There are few effective treatment options for children whose neuroblastoma has not responded or returned following initial treatment.” The BEACON trial results provide these families more hope by identifying a medicine combination that is more effective than regular chemotherapy alone in treating the cancer.
“These small advances in treatment can make a big difference for cancer patients, and it’s fantastic to see that the standard of care in the UK has already been updated based on these findings, giving children with neuroblastoma more treatment options.”
“Meanwhile, more work is still needed to achieve greater survival and long-term quality of life for children affected by neuroblastoma and this trial is helping to pave the way for studies to better understand the biology of this disease and further efforts to improve outcomes.”
Patricia Blanc, President of Imagine for Margo, and Gail Jackson, CEO of Solving Kids’ Cancer UK, made the following comments:
“We’re thrilled to have helped fund the BEACON trial, which has been a lifeline for children with relapsed and refractory neuroblastoma.” It’s quite gratifying to see such great results from the study that are now influencing clinical practice.”
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