Grant awarded to identify markers of poor survival in high-risk neuroblastoma

Solving Kids’ Cancer Europe, Solving Kids' Cancer in New York, Oscar Knox Fund, Smiles for Stanley Fund and Merryn Lacy Trust have awarded a grant of $420,000 to co-investigators Dr Lucas Moreno, Dr Daniel Morgenstern, Dr Meredith Irwin, Dr Gudrun Schleiermacher, and Dr Wendy London for their research proposal: International integrated analysis to identify markers of poor survival in high-risk neuroblastoma. 

Neuroblastoma is a disease that takes on many forms. There are various different sub-groups of children whose disease course is well-known clinically, but who currently can only be identified as belonging to a particular sub-group 'after the fact'. The aim of this research study that we have helped to commission is to try and change that.

Biomarkers

Over the course of many years, various researchers have looked at biomarkers (biological characteristics) that could potentially identify particular sets of patients with the poorest outcome right from the time of diagnosis, so that these patients can be offered novel therapies very early in their disease.

Despite hundreds of studies evaluating factors routinely used in the clinic (such as age, or number of metastatic sites), or more advanced technologies including complex genomic analyses, none of these biomarkers have been tested and validated, and none are currently available for clinical decision making. 

We believe all research must be in the pursuit of helping children with neuroblastoma. It is a travesty if potentially crucial information that might lead to advances in how to treat this disease remain unchallenged, unvalidated, and unused. We want to know if any of the research conducted in this area so far can be found to be reliably effective, or can a combination of more than one technique perform even better, or have the answers simply not been found yet?

The purpose of this new research is to integrate as many biomarkers as possible into the INRG (International Neuroblastoma Risk Group) database, populate it with data from all of the various independent studies and perform comprehensive statistical analyses on the full combined dataset. The aim is that a set of markers will be found that can very accurately predict at diagnosis those children for whom existing treatment will not work. If this proves to be possible it would be a major step towards identifying which children need new and more effective frontline therapy to treat their disease. These children could then be offered additional experimental therapies as part of new frontline clinical trials. 

An esteemed group of researchers

A feature of this study is that the co-investigators and list of collaborators represents arguably the most esteemed group of neuroblastoma researchers ever assembled. From Germany, Netherlands, France, UK, Ireland, America, Canada, and Australia. From SIOPEN, GPOH, COG, and MSK. For the study to succeed it requires widespread input and support at a senior level. If it succeeds it needs widespread adoption. We believe the investigators have addressed both aspects superbly. 

This study was awarded through Solving Kids’ Cancer’s Research Funding Process and supported by its Scientific Advisory Board led by Professor Andy Pearson. Our focus at Solving Kids’ Cancer is on those children whose disease is currently the most difficult to treat successfully. As well as finding more effective therapies for children our aim is to find more appropriate ones. If we can identify those children who need more treatments or different treatments we will be working not only to improve their outcomes but also move closer to a day where each child receives the treatment they need to cure their own neuroblastoma and nothing more in an effort to cure other children’s. 

Research Funding 

Solving Kids’ Cancer acknowledges all those children whose funds enable us to drive forward our research work in pursuit of more effective treatments to help children with neuroblastoma both now and in the future and extends its sincere gratitude to families for their ongoing support. We particularly remember those children who are very sadly no longer with us, our work and our steadfast commitment to research in order to find more effective treatments for children with neuroblastoma honours all their memories. 

Oscar Knox Fund is a Restricted Fund held by Solving Kids’ Cancer. It was established in memory of Oscar Knox who died of neuroblastoma in May 2014 by parents Stephen and Leona. All donations to the Oscar Knox Fund are held for the sole purpose of supporting research and research-related activities. 

The Smiles for Stanley Fund at Solving Kids’ Cancer is for anyone who wishes to donate in his memory. It was established by Stanley's loving parents Sarah and Paul Appleton. The money from this will be used to fund innovative neuroblastoma research to enable children to beat the disease in the future.

The Merryn Lacy Trust was set up to raise funds for specialist treatment for Merryn by her loving parents Jenny and Michael after her diagnosis in July 2013. They, together with Merryn's sister Cora and brother Olly, are determined to help other children in her name. The team at Solving Kids' Cancer is honoured to work with the Merryn Lacy Trust to improve outcomes for children and save more young lives.