Combining anti-tumour drugs with chemo may improve rare children’s cancer outcomes

• Case study: “It was our last hope. We were desperate because he’d had two lots of chemotherapy that hadn’t shrunk his tumour at all.” Parents of 10-year-old Abdullah say they owe his life to BEACON trial

Children who develop neuroblastomas, a rare form of cancer which develops in nerve cells, may benefit from receiving certain anti-tumour drugs as well as chemotherapy, a new trial has found.

The results of the BEACON trial conducted by the Cancer Research UK Clinical Trials Unit at the University of Birmingham found that combining anti-angiogenic drugs, which block tumours from forming blood vessels, alongside various chemotherapy drugs led to more young people seeing their tumours shrinking, from 18% in the control group to 26% among those on Bevacizumab.

The findings are published in the Journal of Clinical Oncology. The trial saw 160 young people aged 1-21, from 43 hospitals in 11 European countries, randomised with half receiving the anti-angiogenic drug called Bevacizumab on top of conventional therapy. The group who received Bevacizumab had an increase in the likelihood of responding to treatment, from 18% among those who only had the established therapy to 26% for those with the additional drug. Patients who received Bevacizumab additionally had better one year progression-free survival rates.

The trial constituted one of many collaborations between the University of Birmingham and European expert groups SIOPEN (International Society of Paediatric Oncology European Neuroblastoma) and ITCC (Innovative therapies for children with cancer).

Simon Gates, Professor of Biostatistics and Cli nical Trials at the University of Birmingham and senior lead author of the paper said:

“These are very exciting results that hopefully get us closer to finding treatments for children who develop neuroblastomas. Currently, the outcomes are really poor for children who get this horrible cancer and so even seemingly small increases in the chance that a patient is going to be able to shrink their tumours is significant.

“We are delighted that the BEACON trial has helped to shape treatment for children with relapsed and refractory neuroblastoma going forward.”

Dr Lucas Moreno, Head of Pediatric Hematology and Oncology at Vall d'Hebron University Hospital, Barcelona, Spain and Chief Investigator for the study said:

“BEACON was a hypothesis-generating trial that has served to identify active regimens that are now being further investigated. We are delighted that the data generated has been incorporated into the current UK Clinical Practice Guidelines and Bevacizumab is incorporated into standard treatment for relapsed neuroblastoma.”

Case study: “It was our last hope”

The parents of Birmingham schoolboy Abdullah Mir who was given a slim chance of survival after being diagnosed with a rare cancer say he owes his life to a clinical trial.

Bushra and Yasir Ali Mir vividly recall the day they were taken aside and told that their four-year-old son Abdullah had neuroblastoma – a rare cancer that develops in early nerve cells.

When two different rounds of chemotherapy drugs failed to work, Abdullah was offered the chance to join the BEACON trial.

“It was our last hope,” said Bushra, who had just qualified as primary school teacher before her son’s devastating diagnosis in November 2017. “We were desperate because he’d had two lots of chemotherapy that hadn’t shrunk his tumour at all.

“There were no other options so we signed up thinking that, even if it didn’t benefit Abdullah, it might help someone else.”

The eight-month treatment ordeal is now a distant memory for ten-year Abdullah, a keen footballer and Manchester United fan who attends Shirelands Technology Primary School in Sandwell.

“Abdullah has always been very strong and positive but he does remember treatment and it was a very difficult time,” recalled Bushra. “Due to COVID year three was his first full year at school but he’s doing really well. He’s crazy about football and he loves school.”

Dr Laura Danielson, Children and Young People’s Research Lead at Cancer Research UK who part funded the trial said:

“There are limited effective treatment options available for children whose neuroblastoma has not responded or come back after initial treatment. The results from the BEACON trial provide more hope to these families by finding a drug combination that is better at treating the disease than standard chemotherapy alone.

“These incremental improvements in treatment can make all the difference for cancer patients and it’s fantastic to see that the standard of care across the UK has already been updated based on these results 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, Imagine for Margo and Gail Jackson, CEO, Solving Kids’ Cancer UK commented:

"We're delighted to have contributed funding to the BEACON trial which has been a lifeline for children with relapsed and refractory neuroblastoma. It is incredibly encouraging to see such positive data from the study that is now impacting clinical practice."