Research Projects

Biomarker evaluation of neuroblastoma mRNAs in children with relapsed and refractory neuroblastoma enrolled in the European BEACON-Neuroblastoma trial

When combinations of drugs are given to children, it is not always known which ones are providing benefit and which ones are contributing to toxicity alone. Biomarkers from blood tests can help predict which patients will respond to a particular therapy.

In this study, blood and bone marrow samples from 120 children treated in the BEACON-Neuroblastoma trial will be collected in order to analyse levels of neuroblastoma mRNAs – molecules that carry codes to make proteins – and the results will be evaluated looking at which children responded to therapy. If the biomarkers are validated in this research study, they can be used in future to predict which children are likely to respond to a therapy before they are treated.

Principal investigator: Sue Burchill, MD, University of Leeds

Project grant: £41,500

Date of award: October 2016

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MiNivAn: A phase I study of 131-1 MIBG, nivolumab and ch14.18/CHO in relapsed / refractory neuroblastoma

Neuroblastoma has a high rate of relapse (over 50% in high-risk cases), and after relapse, only 10% remain alive 5 years later. This therapy aims to improve the cure-rate of children with relapsed or refractory neuroblastoma by combining targeted radiotherapy (131-I MIBG) with the antibodies anti-GD2 (ch14.18/CHO) and anti-PD1 (nivolumab). Laboratory studies have shown that this combination could be a powerful way of eradicating tumours and producing strong protective immunity and long-term cure.

Principal investigator: Dr Juliet Gray, University Hospital Southampton

Co-investigators: Dr Paul Sondel, University of Wisconsin, USA; Prof Holger Lode, University Medicine, Greifswald, Germany; Dr Mark Gaze, University College Hospital, London

Project partners: Solving Kids’ Cancer (USA); J-A-C-K, Band of Parents

Our project grant: £96,000

Date of award: December 2015

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International clinical trial testing third-generation ALK inhibitor lorlatinib in children with neuroblastoma

This exciting new clinical trial provides 17 transatlantic centres in the UK, USA and Europe, recruiting patients simultaneously for an innovative new ALK-inhibitor agent PF-06463922 (lorlatinib). Aimed at targeting mutated ALK genes often indicative of lower survival rates in children with neuroblastoma, the drug has shown minimal toxicity in adults and presents a low treatment burden for children as an oral tablet taken daily. Throughout the trial, there will be regular monitoring to assess for adverse events and efficacy but the amount of time spent in hospital for patients will be significantly less than they will have experienced during prior treatments.

Principal investigators: Lou Chesler, MD, Institute of Cancer Research; Yael Mosse, MD, Children’s Hospital of Philadelphia, Araz Marachelian, Children’s Hospital Los Angeles.

Our grant award: £60,000

Project partners: Solving Kids’ Cancer USA; The Children’s Neuroblastoma Cancer Foundation; The Band of Parents; The Evan’s Victory Against Neuroblastoma Foundation; The Ronan Thompson Foundation; Wade’s Army; The Catherine Elizabeth Blair Memorial Foundation.

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National Neuroblastoma Genetics Reference Centre

Amplification of the MYCN gene and the presence of chromosome rearrangements are strongly linked to aggressive tumour behaviour; children possessing these indicators and deemed high-risk will require intensive treatment, whilst those without and classified lower risk will likely respond to less toxic therapies, sparing them the risk of long term side effects. The National Neuroblastoma Genetics Reference Centre aims to rapidly screen children to identify these indicators (or lack thereof) and thus determine the appropriate treatment.

Principal investigators: Prof Deborah A. Tweddle, University of Newcastle and Dr Nick Bown, Northern Genetics Service.

Project grant: £75,082

Date of award: August 2014

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