Reimagining the N-Of-1 Trial Within Pediatric Neuro-Oncology: A Shifting Paradigm

The field of neuro-oncology has seen significant advances that have allowed the expansion of the therapeutic armamentarium. Nevertheless, overall outcomes have not improved significantly particularly for high-grade tumors. The relative rarity of these pathologies in the pediatric population limits the capacity to design large-scale, multicenter, randomized clinical trials. The emergence of precision medicine as a direct result of better, more widespread genetic and molecular testing affords clinicians the possibility of envisioning new clinical trial paradigms. Each patient becomes their own singular trial receiving the most tailored treatment at every stage of their disease while serving as their own controls. Although limitations still exist for the widespread adoption of these technologies and incorporation into standard clinical care, the prospect of being able to offer directed therapies and monitor disease progression based on single-patient testing represents a much-needed paradigm shift in neuro-oncology.