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Evolving paradigms for new agent development in pediatric oncology

Andrea Flynn1, Elizabeth Fox

  • 1Division of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Colket Translational Research Building, Philadelphia, Pennsylvania, USA.

Current Opinion in Pediatrics
|November 28, 2017
PubMed
Abstract

Insights

Pediatric cancer drug development is evolving with genomic insights and immunotherapies. New clinical trial designs are crucial for evaluating targeted therapies and improving outcomes for children with cancer.

Area of Science:

  • Oncology
  • Pediatric Medicine
  • Genomics
  • Immunotherapy

Background:

  • Childhood cancer, though rare, has a profound long-term impact.
  • Multimodality therapy and risk stratification have improved cure rates to over 80% for pediatric cancers.
  • Cancer genomics has shifted drug development from cytotoxic agents to targeted therapies.

Purpose of the Study:

  • To review new paradigms in pediatric oncology drug development.
  • To incorporate advances in molecular alterations, cancer immunology, and cellular therapy.
  • To address new anticancer drugs, therapeutic approaches, combinations, and regulatory initiatives.

Main Methods:

  • Reviewing advances in cancer genomics for risk stratification.
  • Evaluating novel approaches in cancer immunology, including immune checkpoint blockade and cellular therapy.
  • Integrating preclinical findings with efficient clinical trial designs, including biomarker selection and response assessment.

Main Results:

  • Genomic understanding has transformed pediatric cancer drug development towards targeted approaches.
  • Immunotherapy and cellular therapy offer novel strategies to leverage T cells against cancer.
  • Improved clinical trial designs are essential for accelerating drug development and enhancing patient outcomes.

Conclusions:

  • Pediatric cancer drug development requires adapting to molecular complexity and therapeutic advancements.
  • Efficient clinical trial frameworks incorporating biomarkers, toxicity, and response are vital.
  • Continued innovation in therapeutic strategies and trial design is necessary to improve outcomes for children with cancer.

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