Landmark Transatlantic Trial for Children with High-Risk Neuroblastoma Funded by Parent-Led Charities
In a landmark in pediatric 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 Gustave Roussy in Paris. They will be 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 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.
The grant award was driven by collaborative funding from seven parent-led neuroblastoma research charities; Band of Parents, Joining Against Cancer in Kids (J-A-C-K), Ronan Thompson Foundation, Solving Kids’ Cancer UK, Solving Kids’ Cancer (US), Wade’s Army and Zoé4life.
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.
“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 pediatric 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.”
Marc Winthrop, Chairman of the Band of Parents and Dad to Talia said, “Although we have made great strides in the fight against this insidious disease here at home, by being a part of this global initiative we look forward to levelling the playing field across the world so someday no family has to face this disease with no hope.”
Principal investigators: Dr. Yael Mossé of Children’s Hospital of Philadelphia and Dr Dominique Valteau-Couanet of Gustave Roussy in Paris.TITAN FUNDER LOGOs
Award for selected project: $1.3M
Band of Parents Contribution: $200,000
Date of award: November 2020