TR-16PERSONALIZED TARGETED THERAPY IN REFRACTORY OR RELAPSED CANCER IN CHILDHOOD (TRICEPS STUDY).

Despite all recent advances in cancer's treatment, 18% of children will die from their cancer. New tools that enable better individual tumor characterization and classification are required to improve patient care and outcomes on children. Personalized targeted therapy has proven to be a promising strategy for first line treatment in adult cancer. Given the high heterogeneity in childhood cancers, access to histopathology-based clinical trials for targeted therapy is challenging and facilitated by the capacity to sequence individual childhood tumors in real time. A feasibility study is currently underway at the CHU Sainte-Justine, Montreal, where 30 relapse or refractory cancer patients between 0 and 21 years, are being offered in-depth genomic and transcriptomic investigation to identify patient-specific alterations that may be targetable with alternative therapies. This pilot study will allow us to address the following questions: 1) determine the number of children with cancer who are suitable candidates for targeted therapy at our institution each year, 2) determine the number and type of driver mutation(s) found in our population of recurrent or refractory cancers, 3) determine the number of cancer patients who harbour actionable driver mutation(s) that can be targeted with a Health Canada approved targeted drug, 4) assess the feasibility of obtaining high quality/quantity biospecimen, performing genomic sequencing and data analysis, identifying a drug based on the genomic data and offering this information to the medical team, the patient and the family within a 10-week time frame. The following step toward translating our results into novel personalized therapeutic approaches will then be the development of phase II clinical trials to evaluate the efficacy of these targeted therapies in terms of therapeutic response, survival, and overall quality of life. Our overreaching goal is to be able to offer personalized targeted therapy options to all patients upon primary diagnosis of their cancer.