Opportunities and Challenges in Drug Development for Pediatric Cancers.

ABSTRACT: The use of targeted small-molecule therapeutics and immunotherapeutics has been limited to date in pediatric oncology. Recently, the number of pediatric approvals has risen, and regulatory initiatives in the United States and Europe have aimed to increase the study of novel anticancer therapies in children. Challenges of drug development in children include the rarity of individual cancer diagnoses and the high prevalence of difficult-to-drug targets, including transcription factors and epigenetic regulators. Ongoing pediatric adaptation of biomarker-driven trial designs and further exploration of agents targeting non-kinase drivers constitute high-priority objectives for future pediatric oncology drug development. SIGNIFICANCE: Increasing attention to drug development for children with ca

SUBMITTER: Laetsch TW 

PROVIDER: S-EPMC7933059 | biostudies-literature | 2021 Mar

REPOSITORIES: biostudies-literature