International Neuroblastoma Research Initiative 2018
Solving Kids’ Cancer is leading an international competitive funding call commencing with a Request for Applications (RFA) issued in April 2018. Researchers are invited to submit applications for funding up to a specified maximum grant award value of $1,000,000.
The RFA is to conduct a novel international pilot study for newly diagnosed high-risk neuroblastoma, seeking to improve survival outcomes with less toxicity using innovative therapeutic approaches. Proposals must include recruiting centres in the United Kingdom, Europe, and North America as a minimum.
This is the first instance of our annual Competitive Funding Call process.
BEACON Beta (anti-GD2 Amendment)
The current BEACON study will complete its evaluation of Bevacizumab in the summer of 2018. An amendment to keep the study open to investigate the combination of the anti-GD2 monoclonal antibody dinutuximab beta in combination with chemotherapy (chemoimmunotherapy) was proposed in light of published studies from America, and awarded funding via our Exceptional Funding Requestprocess.
PTPP – ITCC-P4 Collaboration
Driven by Solving Kids’ Cancer’s deeply rooted belief in the benefits of collaborative research efforts and using our established international footprint, we played a pivotal role in bringing together American NCI PPTC and European ITCC-P4 paediatric preclinical research consortiums together for the first time ever via the Pediatric Cancer Working Group at the AACR Annual Meeting in Chicago in April 2018. co-sponsored with Solving Kids’ Cancer (US).
In Septmember 2018, alongside Solving Kids’ Cancer (US) and Neuroblastoma UK, we are also co-sponsoring a follow-up meeting in Amsterdam: ITCC-P4 International Workshop: Improving pediatric oncology drug development through preclinical research in Amsterdam.
Solving Kids’ Cancer Neuroblastoma Senior Trials Coordinator
We have funded a dedicated Senior Trials Coordinator for Neuroblastoma within the Children’s Cancer Trials Team (CCTU) of the Cancer Research UK Clinical Trials Unit (CRCTU), University of Birmingham. This is the first such position dedicated to a single childhood cancer and has responsibility for all neuroblastoma trials on the UK clinical trials portfolio, providing a single point of contact within CRCTU for investigators, from the UK and across Europe designing and developing clinical research projects that will benefit children with neuroblastoma in the UK.
By managing and coordinating the portfolio of neuroblastoma clinical trials from CRCTU, the new post will both increase the capacity of the clinical trials unit to deliver more trials for children with neuroblastoma in the UK and internationally, and ensure the process is as efficient as possible – thus allowing trials to be developed and conducted as quickly as possible.
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 (I-131 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.
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 lorlatinib (PF-06463922). 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.
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.
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.