International neuroblastoma research initiative 2018 

Solving Kids’ Cancer, Band of Parents, Joining Against Cancer in Kids, Ronan Thompson Foundation, Wade’s Army, and Arms Wide Open Childhood Cancer Foundation initiated the 2018 International Challenge Grant Call. This grant call offered $1.4 million for research resulting in the improvement in survival and quality of cure for patients newly diagnosed with high-risk neuroblastoma or patients with relapsed or refractory neuroblastoma through an innovative pilot study that, if successful, would be moved quickly into frontline treatment.  

Three letters of intent were received in response to the revised RFA, all of which were assessed by the Scientific Advisory Board (SAB) of Solving Kids’ Cancer as meeting eligibility criteria and being scientifically strong enough to proceed to the next stage. To support the efforts of the research teams to overcome the significant and complex challenges involved in designing and delivering a transatlantic clinical trial that aligns with ongoing studies and future plans of COG (America) and SIOPEN (Europe), small feasibility grants were provided.

Significance of this grant call 

In many respects a transatlantic clinical trial has been something of a holy grail for the neuroblastoma research community, often discussed over the years to provide a clear path to benefit as many children as possible while moving the field of clinical research forward as quickly as possible.   

This initiative, led by a group of parent-led charities, aims to change the landscape of neuroblastoma research by bringing together international researchers together to develop studies that will impact frontline treatment of neuroblastoma simultaneously in Europe, America, and potentially beyond. This something that has never been achieved before.   

If this RFA ultimately translates into the first-ever transatlantic study in newly diagnosed neuroblastoma it will truly represent a paradigm shift and the opportunities going forward in the future will only become greater for it.

Outcomes of this grant call 

This international grant call resulted in the funding the Transatlantic Integration Targeting ALK in Neuroblastoma (TITAN) initiative. The TITAN initiative aims to introduce lorlatinib into frontline treatment for all children newly diagnosed with ALK+ high-risk neuroblastoma. This will be the first ever SIOPEN and COG international trial, meaning that this will happen simultaneously in Europe and the US.

Phase III TITAN trial

Phase III transatlantic trial of ALK inhibitor Lorlatinib.

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