Project 465500

Childhood cancer while rare is nevertheless responsible for the most disease-related deaths in childhood. For children whose cancers relapse after treatment or for whom those initial treatments are unsuccessful, there are often few treatment options. Over recent years there has been growing interest in identifying specific genetic abnormalities in individual cancers with the aim of then targeting these abnormalities to provide a new treatment approach. The current Terry Fox PROFYLE initiative is a pan-Canadian research project that provides comprehensive genetic sequencing for children, adolescents and young adults with hard-to-cure cancers. Building on this foundation, we now propose to launch (in collaboration with colleagues in Australia), a therapeutic trial that will bring targeted therapies to those patients with relevant genetic abnormalities identified through PROFYLE sequencing. The OPTIMISE trial will allocate participants to individual treatment arms depending on the genetic sequencing results and will provide access to novel targeted agents and/or combinations with the aim of expanding therapies available for these patients and ultimately using the information obtained from genetic sequencing to provide better, more targeted therapies.