In a landmark in paediatric oncology research, children with high-risk neuroblastoma across Europe and North America will be treated together for the first time, following the award of $1.3M to fund a new transatlantic clinical trial led by Dr Yael Mossé of Children’s Hospital of Philadelphia and Dr Dominique Valteau-Couanet of Institute Gustave Roussy in France, investigating the ALK inhibitor Lorlatinib in the treatment of children with newly diagnosed high-risk neuroblastoma, a deadly childhood cancer that mainly affects children under the age of five.

The challenge grant award was driven by collaborative funding from seven parent-led research charities; Joining Against Cancer in Kids (J-A-C-K),  Solving Kids’ Cancer UK, and Zoé4life in Europe alongside Band of Parents, Ronan Thompson Foundation, Solving Kids’ Cancer (New York), Wade’s Army in the United States.  

The study, known as TITAN – Transatlantic Integration Targeting ALK in Neuroblastoma – will see a targeted drug introduced to frontline treatment for children, with the hope of dramatically increasing survival rates. This landmark collaboration between the Children’s Oncology Group (COG) in North America and the SIOPEN research network in Europe represents the first time these pediatric consortia on both sides of the Atlantic have worked together to design and run a clinical trial. 

It is also the first International Neuroblastoma Research Initiative award to be funded under Solving Kids’ Cancer UK’s AMRC-accredited research funding call and follows an extensive and stringent scientific review process. 

Speaking on behalf Solving Kids’ Cancer UK, Research Director Nick Bird said, I’m thrilled that our ambitious call to action has resulted in the first ever joint SIOPEN/COG clinical trial. We are impatient for change because children with neuroblastoma do not have time on their side, and we’re determined to make bold and decisive moves that challenge the status quo in a bid for greater and more rapid progress. 

The new study will be integrated to form part of the ongoing COG and SIOPEN Phase 3 high-risk neuroblastoma trials, and will involve children at Children’s Oncology Group hospitals in North America and SIOPEN institutions across Europe and the UK having their tumours tested for mutations in the ALK gene when they are first diagnosed. Such mutations are found in around 14% of patients and represents a group of children who have inferior survival with current multi-modal treatment regimens. Children whose tumours are found to have ALK mutations will soon be treated with the addition of lorlatinib, a third-generation ALK inhibitor manufactured and supplied by the pharmaceutical company Pfizer.  

In Europe, TITAN is being integrated into the SIOPEN HR-NBL2 Clinical Trial that will run in over 20 countries, and is being funded to open throughout the United Kingdom by Solving Kids’ Cancer UK together with Neuroblastoma UK. 

“This is a landmark step in clinical research for children with neuroblastoma,” said Prof Andy Pearson, MD, Chair of the Solving Kids’ Cancer UK Scientific Advisory Board. “With paediatric cancer hugely underfunded in comparison to adult cancer, there is an urgent need for breakthrough treatments for the most difficult-to-cure childhood cancers like neuroblastoma. This trial has the potential to accelerate the discovery of a new treatment and lay the foundation for future collaborations of this nature.” 

Principal investigators: Dr. Yael Mossé of Children’s Hospital of Philadelphia and Dr Dominique Valteau-Couanet of Gustave Roussy in Paris. 

Project partners: Band of ParentsJoining Against Cancer in Kids (J-A-C-K)Ronan Thompson FoundationSolving Kids’ Cancer UKSolving Kids’ Cancer (US)Wade’s Army and Zoé4life. 

Total RFA award: $1.4M 

Award for selected project: $1.3M 

SKC UK Contribution: $200,000 

Date of award: November 2020