This website is intended for healthcare professionals only.

Hospital Healthcare Europe
Hospital Pharmacy Europe     Newsletter    Login            

Press Releases

Take a look at a selection of our recent media coverage:

Bevacizumab plus chemotherapy shown to improve paediatric neuroblastoma outcomes

9th January 2024

Combining the antiangiogenic agent bevacizumab with chemotherapy improves outcomes in children with relapsed and refractory high-risk neuroblastoma (RR-HRNB), according to a recent trial conducted by the Cancer Research UK Clinical Trials Unit at the University of Birmingham, UK.

Published in the Journal of Clinical Oncology, the BEACON Neuroblastoma trial evaluated three backbone chemotherapy regimens and the addition of bevacizumab.

The primary endpoint was best overall response (complete or partial) rate (ORR) during the first six courses, by RECIST or International Neuroblastoma Response Criteria for patients with measurable or evaluable disease, respectively.

Secondary endpoints were safety, progression-free survival (PFS) and overall survival time.

A total of 160 young people with RR-HRNB aged 1-21, from 43 hospitals in 11 European countries, were randomly assigned in a 3 × 2 factorial design to temozolomide, irinotecan-temozolomide, or topotecan-temozolomide with or without bevacizumab.

The researchers found that the addition of bevacizumab led to more young people seeing their tumours shrink, with the ORR at 26% (95% CI, 17 to 37) compared with 18% (95% CI, 10 to 28) without bevacizumab (risk ratio [RR], 1.52 [95% CI, 0.83 to 2.77]; P = .17).

The one-year PFS in the bevacizumab arms was 0.46 (95% CI, 0.34 to 0.56) compared with 0.38 (95% CI, 0.27 to 0.49) in the non-bevacizumab arms.

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 tumours is significant.

‘We are delighted that the BEACON trial has helped to shape treatment for children with RR-HRNB 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.’

GD2-CART01 effective for R/R high-risk neuroblastoma 

18th April 2023

GD2-CART01 has been found in a phase 1-2 clinical trial to be both safe and effective for children with heavily pretreated neuroblastoma.

GD2-CART01 is a safe and feasible therapy for children with relapsed or refractory (R/R) high-risk neuroblastoma according to a phase 1 – 2 trial by Italian researchers.

Neuroblastoma is responsible for 11% of paediatric cancer deaths and these cancer cells express high levels of disialoganglioside GD2.

Moreover, targeting this protein, while a valid and safe strategy, requires further modification to promote CAR-T cell longevity.

In the current trial, researchers used a third-generation CAR-T cell therapy in patients with R/R high-risk neuroblastoma. The therapy included the inducible caspase 9 suicide gene, designed to kill cells in the presence of dangerous toxic effects.

GD2-CART01 outcomes

The trial enrolled 27 children with heavily pretreated neuroblastoma.

The overall response rate was 63%; 9 children had a complete response and 8 a partial response. The three-year overall survival and event-free survival were 60% and 36%, respectively.

Overall, after infusion 33% of patients (eight patients) had a complete response or maintained a complete response (one patient).

Cytokine release syndrome occurred in 74% of patients but was of mild severity in the majority (95%). The suicide gene activation occurred in only one patient. GD2-targeted CAR T cells were detectable in peripheral blood in 26 of 27 patients up to 30 months after infusion.

The authors concluded that GD2-CART01 may induce sustained eradication of disease in a proportion of patients with R/R neuroblastoma.

Del Bufalo F et al. GD2-CART01 for Relapsed or Refractory High-Risk Neuroblastoma. N Eng J Med 2023.