Birmingham schoolboy Abdullah Mir with his father at a wedding in 2018.
A groundbreaking clinical trial offers new hope for children battling a rare nerve cell cancer called neuroblastoma. Researchers from the University of Birmingham have developed a novel treatment combining anti-tumor drugs with chemotherapy, resulting in significant tumor shrinkage in a greater number of young patients.
The key lies in adding 'anti-angiogenic' drugs, which act like traffic cops, cutting off the tumor's blood supply and starving it. Compared to standard chemotherapy alone, this combined approach saw a 26% improvement in tumor shrinkage, offering a vital edge in the fight against this aggressive cancer.
The results are encouraging, with over one year of tumor-free survival observed in more patients receiving the new treatment. While further research is needed, this breakthrough offers a beacon of hope for families facing this devastating disease.
The findings are published in the Journal of Clinical Oncology.
As per a release, the trial saw 160 young people aged 1-21, from 43 hospitals in 11 European countries, randomised with half receiving the anti-angiogenic drug 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 Clinical 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 tumors are significant. We are delighted that the BEACON trial has helped to shape treatment for children with relapsed and refractory neuroblastoma going forward."