Charities fund new clinical trial

Solving Kids’ Cancer, with Neuroblastoma UK, has awarded a grant of £609,762.40 to enable the SIOPEN High-Risk Neuroblastoma Clinical Trial 2 (HR-NBL2) to open in the UK, it is anticipated to open in early 2021. This Phase 3 clinical trial will offer children in the UK, with a diagnosis of high-risk neuroblastoma, to have the same opportunities as children across Europe and take part in this pan-European study, enabling a series of important randomised questions to be answered within the overall framework of the trial.

“As the principal funder of clinical trials for children with neuroblastoma in the UK, we are wholly committed to funding pioneering research that provides the greatest benefit and opportunities for children facing diagnosis.

During the Covid-19 crisis, our priority remains on supporting families affected by neuroblastoma and ensuring vital research can continue. We have continued to work with our neuroblastoma community and clinicians and collaborated with our colleagues at Neuroblastoma UK to award this important grant.”

Gail Jackson, CEO of Solving Kids' Cancer UK

Solving Kids’ Cancer is working with Neuroblastoma UK, and have funded the trial in the respective amounts of £434,762.40 and £175,000, to enable this trial to take place. Without this commitment of funding there would not be any available upfront clinical trials in the UK for children diagnosed with high-risk neuroblastoma and the treatment pathway would remain ‘as is’ over the next five years.

“We are delighted to join forces with Solving Kids Cancer in funding this crucial clinical research trial. As a charity, we receive no government funding - it is thanks to our generous supporters that we can provide funding of £175,000 to support this vital research, which offers real potential to develop more effective and kinder treatment for children with high-risk neuroblastoma in the UK.” Commented Tony Heddon, Chair of Neuroblastoma UK, “We are committed to ensuring that research into neuroblastoma continues beyond the coronavirus pandemic. International collaboration to understand more about this rare disease is hugely important and can help to ensure that children in the UK can have equal access to new treatment. By funding this ten-year project, we can give children across the UK a chance to access treatment which could potentially save their lives.”

"The international community is exceptionally grateful to Solving Kids’ Cancer and Neuroblastoma UK for funding SIOPEN High Risk Neuroblastoma Trial 2 in the United Kingdom. The participation of the United Kingdom in this trial is absolutely crucial for children with high-risk neuroblastoma in the UK and ultimately the inclusion of the United Kingdom in this study will have a very positive benefit for children in Europe and globally."

Professor Andy Pearson, MD, Chair of the Solving Kids’ Cancer UK Scientific Advisory Board

The trial builds on the successes of the previous High-Risk Neuroblastoma Trial 1 (HR-NBL1) pan-European study developed by SIOPEN and provides a clinical trial that ensures equal access to the very best treatments and care across the UK, and a pathway for future innovative therapies to be safely administered and studied.

UK patients accounted for 20% of patients recruited to the SIOPEN HR-NBL1 and hence made a major contribution to international research. Opening of the SIOPEN HR-NBL2 trial in the UK will offer children newly diagnosed with high-risk neuroblastoma in the UK the same opportunity as children across Europe:

  • To take part in a clinical trial with the potential to significantly improve their chance of survival
  • To benefit from ongoing plans to incorporate clinical trials of targeted agents into upfront treatment for high-risk neuroblastoma, most notably by the addition of an ALK inhibitor for patients whose tumours harbour aberrations in the ALK gene.
  • To participate in research looking at how the presence and levels of tumour markers in blood, circulating tumour DNA (ctDNA), might be used at diagnosis and for ongoing disease response assessments instead of existing more invasive imaging and biopsy techniques.
  • To benefit from ongoing plans to study whether adding anti-GD2 monoclonal antibody therapy alongside induction chemotherapy will improve survival for children with high-risk neuroblastoma.
  • To contribute to further improvements in clinical outcomes in neuroblastoma by participation in and be able to benefit from the introduction of future innovations over the course of the next 5-year cycle.

“As the UK lead investigator of the new European High-Risk Neuroblastoma 2 trial, I am extremely grateful to Solving Kids’ Cancer and Neuroblastoma UK for funding the opening and running of the trial in the UK allowing UK patients and families to actively contribute to future improvements in treatment and survival of children with neuroblastoma through participation in high quality, collaborative international research and build on the advances in treatment and in survival of patients, resulting from the previous High-Risk Neuroblastoma 1 Trial, which have now been incorporated in to standard treatment in the NHS.” writes Dr Martin Elliot, Consultant Paediatric Oncologist, “The funding will secure the ability for UK clinicians to offer high-risk neuroblastoma patients and their families access to a clinical trial, which I am certain will be highly appreciated by families recruited to the trial and even more so by patients and families that will benefit from the potential positive outcomes of this research, which will hopefully lead to further improvements in cure rates of children with this devastating cancer.  As an investigator I am excited to work with doctors and research teams in all UK paediatric oncology centres, to enable this trial to be an option for newly diagnosed patients and families across the UK in the near future.”

The trial is anticipated to open in the UK in early 2021, as part the wider SIOPEN pan-European trial. It is a ten-year longitudinal study that includes five years of clinical investigation and five years of corresponding data collection and research analysis. The trial is currently open in France, the sponsor nation, and the UK will be joining in 2021. The University of Birmingham Cancer Research UK Clinical Trials Unit, where Solving Kids’ Cancer funds a dedicated senior Trials Coordinator for Neuroblastoma, is the UK national coordinator and will have the responsibility for delivering the trial across UK sites.

I’m proud that we have come together with Neuroblastoma UK to fund this pivotal study, the largest and most significant neuroblastoma trial in Europe, so that it will now open in the UK. On behalf of the Board of Trustees I’d also like to thank Charlie and Corey’s families, whose benevolence in donating their funds to Solving Kids’ Cancer will enable us to not only fund this trial, but also continue our vital work initiating and funding other clinical trials to help children with neuroblastoma.

Current standard of care for children diagnosed with neuroblastoma in the UK is a result of the SIOPEN high-risk 1 study, and to not participate in its successor would have been enormously detrimental to our children. This isn’t only about the trial in its current form, but also the framework that it provides for assessing future benefits of those innovative and targeted therapies that we know are coming down the line.

If we had not funded the study there was every chance that the UK would be left behind, leading to the unthinkable yet real prospect of our children missing out on potentially life-saving new treatments."

Nick Bird, Chair of Trustees

Around 100 children a year in the UK are diagnosed with neuroblastoma, of which approximately half will be classified as high-risk. It is a rare and aggressive childhood cancer and the vast majority of children diagnosed are under 5, despite intensive multi-modal therapy long-term survival from the high-risk form of the disease remains around 40-50%. Solving Kids’ Cancer supports families and their children throughout their cancer journey and is committed to funding research to bring better and less toxic treatment options to the UK for children fighting this deadly disease. The charity receives no statutory funding to deliver its vital work and relies on the generosity of its passionate supporters to ensure research to inform future treatments can continue. 

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