Seminars in cancer biologyJul 14, 2020

Updates on immune checkpoint blocker treatments for solid tumors in adults and children

Circadian Biology Weekly Brief ↗PubMed ↗DOI ↗

Updated Mar 29, 2026

Abstract

Immune checkpoint inhibitors (ICIs) may provide benefits in pediatric malignancies, building on their success in adult cancers.

Current response rates to ICIs as standalone treatments in pediatric cancers are disappointing.
Combination therapies may yield better results compared to individual ICI agents.
Only a subset of patients experiences prolonged clinical effects from ICIs, highlighting the need for predictive biomarkers.
The review discusses various immune checkpoints, including PD-1, PD-L1, and CTLA-4, as well as emerging targets like LAG-3 and TIM-3.
Prognostic value of these immune checkpoints is evaluated in both adult and childhood tumors.
The overview includes ongoing preclinical models and clinical trials aimed at enhancing immunotherapy effectiveness for young patients.

Simplified

Immune checkpoint inhibitors (ICIs) have shown unprecedented benefits in various adult cancers, and this success has prompted the exploration of ICI therapy even in childhood malignances. Although the use of ICIs as individual agents has achieved disappointing response rates, combinational therapies are likely to promise better results. However, only a subset of patients experienced prolonged clinical effects, thus suggesting the need to identify robust bio-markers that predict individual clinical response or resistance to ICI therapy as the main challenge. In this review, we focus on how the use of ICIs in adult cancers can be translated into pediatric malignances. We discuss the physiological mechanism of action of each IC, including PD-1, PD-L1 and CTLA-4 and the new emerging ones, LAG-3, TIM-3, TIGIT, B7-H3, BTLA and IDO-1, and evaluate their prognostic value in both adult and childhood tumors. Furthermore, we offer an overview of preclinical models and clinical trials currently under investigation to improve the effectiveness of cancer immunotherapies in these patients. Finally, we outline the main predictive factors that influence the efficacy of ICIs, in order to lay the basis for the development of a pan-cancer immunogenomic model, able to direct young patients towards more specific immunotherapy.

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Updates on immune checkpoint blocker treatments for solid tumors in adults and children

Abstract

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