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Background <p>Rhabdomyosarcoma, the most common pediatric soft tissue sarcoma, remains a therapeutic challenge due to its aggressive nature and poor outcomes in high-risk patients. Herein, we explore the potential of CAR-T cell therapy as a transformative approach for rhabdomyosarcoma, focusing on target antigens such as HER2, CD276, FGFR4, PDGFR-α, and others.</p> Main body <p>While preclinical studies are promising, antitumor activity in clinical studies to date has been limited. Significant clinical hurdles include antigen heterogeneity, immunosuppressive tumor microenvironments, and logistical barriers in manufacturing. Innovative strategies like dual-targeting CARs, combinatorial checkpoint blockade, and allogeneic platforms are discussed to address these limitations. Additionally, we highlight emerging modalities such as immune checkpoint inhibitors, oncolytic viruses, and small molecule inhibitors that may synergize with CAR-T cell therapy.</p> Conclusions <p>By integrating these advances, rationally designed multimodal therapies could overcome resistance mechanisms and improve outcomes for refractory rhabdomyosarcoma, offering hope for a historically incurable malignancy.</p>

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CAR-T cell immunotherapy in rhabdomyosarcoma

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2026年2月18日

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  • Pooria Safarzadeh Kozani,
  • Setareh Dashti Shokoohi,
  • Pouya Safarzadeh Kozani

摘要

Background

Rhabdomyosarcoma, the most common pediatric soft tissue sarcoma, remains a therapeutic challenge due to its aggressive nature and poor outcomes in high-risk patients. Herein, we explore the potential of CAR-T cell therapy as a transformative approach for rhabdomyosarcoma, focusing on target antigens such as HER2, CD276, FGFR4, PDGFR-α, and others.

Main body

While preclinical studies are promising, antitumor activity in clinical studies to date has been limited. Significant clinical hurdles include antigen heterogeneity, immunosuppressive tumor microenvironments, and logistical barriers in manufacturing. Innovative strategies like dual-targeting CARs, combinatorial checkpoint blockade, and allogeneic platforms are discussed to address these limitations. Additionally, we highlight emerging modalities such as immune checkpoint inhibitors, oncolytic viruses, and small molecule inhibitors that may synergize with CAR-T cell therapy.

Conclusions

By integrating these advances, rationally designed multimodal therapies could overcome resistance mechanisms and improve outcomes for refractory rhabdomyosarcoma, offering hope for a historically incurable malignancy.