Clinical and Research Update

One of the most important developments for UK children with neuroblastoma is the enlargement of SIOPEN, the umbrella organisation of neuroblastoma clinicians from mostly European countries. Germany and the Netherlands have now joined, which will benefit UK children in two ways:

•  it will significantly increase the number of children that enrol in SIOPEN trials, which means treatment comparisons can be resolved more quickly, speeding up improvements in standard care;  and
• it will also bring the significant German and Dutch research and clinical expertise more formally into SIOPEN decision-making.

A note on anti-GD2 immunotherapy provision in the UK is also in order. Anti-GD2 immunotherapy is available to UK children already enrolled in the SIOPEN high-risk trial, as long as they continue to respond to treatment adequately on the trial. In order for the anti-GD2 drug dinutuximab beta to become available as standard care, it now needs to be evaluated by NICE, having been approved by the European Medicines Agency. We fervently hope that this NICE process proceeds rapidly and in a way appropriate to the paediatric, rather than adult setting that NICE are more used to dealing with. On a related immunotherapy note, we hope to bring you a report on the novel CAR-T cell immunotherapy, developed by Dr. Martin Pule and Prof. John Anderson, in our next Newsletter. NBUK funded this work at its outset, which is currently the subject of a Phase I/II trial run by Cancer Research UK.

The most important recent research development of the last couple of years is probably the discovery of a common theme to the type of disease that older children present with, for whom the prognosis remains very poor. The ATRX gene and other molecular changes that are involved alter telomeres, the ‘plugs’ that protect the ends of chromosomes. We are glad to be supporting a meeting aimed at identifying new treatments in light of this new understanding of the changes in these tumours.  Read about it here.