Document Type
Article
Publication Date
12-1-2024
Original Citation
Smith M,
Houghton P,
Lock R,
Maris J,
Gorlick R,
Kurmasheva R,
Li X,
Teicher B,
Chuang J,
Dela Cruz F,
Dyer M,
Kung A,
Lloyd M,
Mossé Y,
Stearns T,
Stewart E,
Bult C,
Erickson S.
Lessons learned from 20 years of preclinical testing in pediatric cancers. Pharmacol Ther. 2024;264:108742.
Keywords
JGM, JMG, Humans, Animals, Neoplasms, Antineoplastic Agents, Child, Drug Evaluation, Preclinical, Disease Models, Animal, Mice, Drug Development
JAX Source
Pharmacol Ther. 2024;264:108742.
ISSN
1879-016X
PMID
39510293
DOI
https://doi.org/10.1016/j.pharmthera.2024.108742
Abstract
Programs for preclinical testing of targeted cancer agents in murine models of childhood cancers have been supported by the National Cancer Institute (NCI) since 2004. These programs were established to work collaboratively with industry partners to address the paucity of targeted agents for pediatric cancers compared with the large number of agents developed and approved for malignancies primarily affecting adults. The distinctive biology of pediatric cancers and the relatively small numbers of pediatric cancer patients are major challenges for pediatric oncology drug development. These factors are exacerbated by the division of cancers into multiple subtypes that are further sub-classified by their genomic properties. The imbalance between the large number of candidate agents and small patient populations requires careful prioritization of agents developed for adult cancers for clinical evaluation in children with cancer. The NCI-supported preclinical pediatric programs have published positive and negative results of efficacy testing for over 100 agents to aid the pediatric research community in identifying the most promising candidates to move forward for clinical testing in pediatric oncology. Here, we review and summarize lessons learned from two decades of experience with the design and execution of preclinical trials of antineoplastic agents in murine models of childhood cancers.
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