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Published on: February 24, 2023
Translational considerations for immunotherapy clinical trials in pediatric neuro-oncology
Jessica B Foster1, Marta M Alonso2, Elias Sayour3
1Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA USA.
Abstract:
While immunotherapy for pediatric cancer has made great strides in recent decades, including the FDA approval of agents such as dinutuximab and tisgenlecleucel, these successes have rarely impacted children with pediatric central nervous system (CNS) tumors. As our understanding of the biological underpinnings of these tumors evolves, new immunotherapeutics are undergoing rapid clinical translation specifically designed for children with CNS tumors. Most recently, there have been notable clinical successes with oncolytic viruses, vaccines, adoptive cellular therapy, and immune checkpoint inhibition. In this article, the immunotherapy working group of the Pacific Pediatric Neuro-Oncology Consortium (PNOC) reviews the current and future state of immunotherapeutic CNS clinical trials with a focus on clinical trial development. Based on recent therapeutic trials, we discuss unique immunotherapy clinical trial challenges, including toxicity considerations, disease assessment, and correlative studies. Combinatorial strategies and future directions will be addressed. Through internationally collaborative efforts and consortia, we aim to direct this promising field of immuno-oncology to the next frontier of successful application against pediatric CNS tumors.
Insights
Immunotherapy offers new hope for pediatric central nervous system (CNS) tumors, a rare success in pediatric cancer treatment. This review explores current and future clinical trials, addressing challenges and combinatorial strategies for immuno-oncology in CNS tumors.
Area of Science:
- Pediatric Neuro-Oncology
- Cancer Immunotherapy
- Central Nervous System (CNS) Tumors
Background:
- Immunotherapy has advanced pediatric cancer treatment, but impact on pediatric CNS tumors remains limited.
- Recent progress in understanding CNS tumor biology fuels development of novel immunotherapeutics.
- Emerging successes include oncolytic viruses, vaccines, adoptive cellular therapy, and immune checkpoint inhibitors.
Purpose of the Study:
- To review the current and future landscape of immunotherapy clinical trials for pediatric CNS tumors.
- To focus on clinical trial development and address unique challenges in this field.
- To discuss combinatorial strategies and future directions for pediatric immuno-oncology.
Main Methods:
- Review of recent therapeutic trials in pediatric CNS tumors.
- Analysis of challenges in immunotherapy clinical trial development, including toxicity, disease assessment, and correlative studies.
- Discussion of combinatorial strategies and future research directions.
Main Results:
- Significant progress is being made in developing immunotherapeutics for pediatric CNS tumors.
- Key challenges include managing toxicity, accurate disease assessment, and conducting correlative studies.
- Collaborative efforts are crucial for advancing the field.
Conclusions:
- Immunotherapy holds significant promise for treating pediatric CNS tumors.
- Addressing specific clinical trial challenges is essential for successful translation.
- International collaboration and innovative strategies will drive future advancements in pediatric immuno-oncology.
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