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Rare but not forgotten: Therapeutic advancements for rare childhood cancers

Jacquelyn N. Crane,Urania Dagalakis,Robyn Gartrell,K. A. Schultz,T. Laetsch

Published 2025 in Molecular therapy. Oncology

ABSTRACT

Cancers in children represent less than 1% of all new cases of cancer. While all cancers are rare during childhood, certain types are particularly rare. Multi-institutional pediatric trials and coordinated efforts between pediatric and adult groups have resulted in recent key advances in the treatment of certain rare pediatric cancers. This article reviews these advances with a focus on immunotherapies, molecularly targeted therapies, and radioligands. This includes work that led to the FDA approvals of immune checkpoint inhibitors in multiple rare pediatric tumor types, the NTRK inhibitors larotrectinib, entrectinib, and repotrectinib for children and adults with solid tumors with NTRK fusions, the ALK inhibitor crizotinib in children and adults with ALK-positive inflammatory myofibroblastic tumors, and the radioligand LUATHERA for adolescents and adults with somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors. Additionally, key data from adult studies which have informed the management of pediatric rare cancers are reviewed. Despite these advances, the study of rare pediatric cancers faces multiple challenges including a limited number of patients for efficient and well-powered clinical trials and a dearth of financial incentives. Ongoing, coordinated efforts are needed to continue the advancement of novel treatments and improve survival and minimize late effects.

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