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 trans- Atlantic clinical trial led by Dr Yael Mosse of Children’s Hospital of Philadelphia and Dr Diminique Valteau-Couanet of Instituate Gustave Roussy in France, investigating the ALK inhibitor Loriatinib in the treatment of children with newly diagnosed high-risk Neuroblastoma, a deadly childhood cancer that mainly affects children under the age of five.
This ground-breaking research, known as TITAN, transatlantic Integration Targeting ALK in Neuroblastoma is being made possible by collaborative funding from seven parent-led neuroblastoma clinical research charities:
The proposal, “ Integration of Loriatinib to improve outcomes for patients with high-risk ALK-driven neuroblastoma: A COG and SIOPEN international collaboration” is 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 of the group of funding partners, Nick Bird Research Director at Solving Kids’ Cancer UK said “We are proud to be driving forward innovation to provide new hope to children and families shattered by a neuroblastoma diagnosis, and are thankful to members of the research community who stepped up and came together to meet the spirit of our challenge grant call.
“Making the first ever study of this kind a reality stands as a testament of what passionate like-minded charities and non-profit organisations can do if they come together for the greater
Richard Brown, [J-A-C-K], said, “A child diagnosed with high-risk neuroblastoma is a rare individual facing enormous battles; the cancer must not prosper as a consequence of their isolation. The collaboration of international teams to stand together and fund and deliver trials to the children diminishes isolation and accelerates an answer to their cancer.
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 and SIOPEN institutions across Europe and the UK having their tumours being tested for mutations in the ALK gene when they are first diagnosed. Such mutations are found in around 15% of patients and represents a group of children who currently have inferior outcomes with the existing multi-modal treatment regimen. 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.
Professor Andy Pearson, Chair of Solving Kids’ Cancer Scientific Advisory Board, said, “The SKC 2018 RFA has been an enormous catalyst for international collaboration. The Lorlatinib trial itself is a landmark in clinical research for children with neuroblastoma. The approach is a paradigm to individualise therapy and can be a prototype for other molecular sub-types which ultimately will result in widespread benefits for all children with this cancer.”
Principal investigators: Dr Yael Mossé of Children’s Hospital of Philadelphia and Dr Dominique Valteau-Couanet of Institute Gustave Roussy.
Total RFA award $1.4M
Award for selected project: $1.3M
J-A-C-K contribution: $200,000
Date of award: November 2020.