Clinical research with targeted drugs in paediatric oncology

Paula Valle-Simón; Alberto M Borobia; Antonio Pérez-Martínez

doi:10.1016/j.drudis.2023.103672

Clinical research with targeted drugs in paediatric oncology

Drug Discov Today. 2023 Aug;28(8):103672. doi: 10.1016/j.drudis.2023.103672. Epub 2023 Jun 16.

Authors

Paula Valle-Simón¹, Alberto M Borobia², Antonio Pérez-Martínez³

Affiliations

¹ Clinical Pharmacology Department, La Paz University Hospital, Idipaz, Paseo de la Castellana 261, 28046 Madrid, Spain. Electronic address: pvalle@salud.madrid.org.
² Clinical Pharmacology Department, La Paz University Hospital, School of Medicine, Universidad Autónoma de Madrid (UAM) IdiPAZ, Paseo de la Castellana 261, 28046 Madrid, Spain.
³ Paediatric Haemato-Oncology Department, La Paz University Hospital, School of Medicine, Universidad Autónoma de Madrid (UAM), IdiPAZ, Paseo de la Castellana 261, 28046 Madrid, Spain.

PMID: 37330039
DOI: 10.1016/j.drudis.2023.103672

Abstract

The development of targeted drugs in paediatric oncology has been notoriously slow, in part due to the peculiarities of this rare and highly heterogeneous population. To provide therapeutic breakthroughs for the highest risk subgroups of childhood cancer, innovative research solutions have been implemented in the last several years by different international collaborative groups and regulators. Here, we discuss and summarise some of these approaches, as well as challenges and unmet needs that are still being addressed. A wide range of topics were covered in this review including molecular diagnosis optimisation, innovative research methodologies, big data approaches, trial enrolment strategies, and improvements in regulation and preclinical research platforms.

Keywords: clinical trials; paediatric oncology; personalised medicine; targeted drugs.

Publication types

Review

MeSH terms

Child
Drug Delivery Systems
Humans
Medical Oncology* / methods
Neoplasms* / drug therapy