Opportunities and Challenges in Drug Development for Pediatric Cancers

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 c...

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Bibliographic Details
Published in:Cancer discovery 2021-03, Vol.11 (3), p.545-559
Main Authors: Laetsch, Theodore W, DuBois, Steven G, Bender, Julia Glade, Macy, Margaret E, Moreno, Lucas
Format: Article
Language:English
Subjects:
Age Factors
Animals
Antineoplastic Agents - pharmacology
Antineoplastic Agents - therapeutic use
Biomarkers, Tumor
Child
Clinical Trials as Topic
Combined Modality Therapy
Disease Susceptibility
Drug and Narcotic Control
Drug Development - legislation & jurisprudence
Drug Development - methods
Drug Evaluation, Preclinical
Genomics - methods
Humans
Molecular Targeted Therapy - adverse effects
Molecular Targeted Therapy - methods
Neoplasms - drug therapy
Neoplasms - etiology
Pediatrics
Treatment Outcome
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Summary: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 cancer by regulators and pharmaceutical companies holds the promise of accelerating the availability of new therapies for children with cancer, potentially improving survival and decreasing the acute and chronic toxicities of therapy. However, unique approaches are necessary to study novel therapies in children that take into account low patient numbers, the pediatric cancer genomic landscape and tumor microenvironment, and the need for pediatric formulations. It is also critical to evaluate the potential for unique toxicities in growing hosts without affecting the pace of discovery for children with these life-threatening diseases.
ISSN:2159-8274
2159-8290
DOI:10.1158/2159-8290.CD-20-0779