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Tumor Immunotherapy

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Immunotherapy is a treatment that boosts or manipulates the immune system to fight diseases, including cancer. For instance, by stimulating an immune response through vaccinations against viruses that cause cancers, like hepatitis B virus and human papillomavirus, these diseases can be prevented. Nonetheless, some cancer cells can avoid the immune system due to their rapid mutation and division. The immune response to many cancers involves three phases: elimination, equilibrium, and escape.
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The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
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Cancer is the second leading cause of death in the United States. A cancer cell is genetically unstable and hence can mutate faster. They can also modify their microenvironment and escape immune surveillance. The difficulties in treating cancer are further compounded by the emergence of rapid resistance to anticancer drugs. The most common ways to attain resistance in cancer cells include alteration in drug transport and metabolism, modification of drug target, elevated DNA damage response, or...
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Cancer treatment vaccines are a rapidly evolving field that offers a promising approach to immunotherapy. Unlike traditional vaccines that prevent diseases, cancer treatment vaccines are designed to treat existing cancers by stimulating the immune system to recognize and attack cancer cells.
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CAR T cell therapy for central nervous system solid tumors: current progress and future directions.

Yaroslav Kaminskiy1, Vitaly Degtyarev2, Alexey Stepanov3

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Frontiers in Immunology
|September 2, 2025
PubMed
Summary

CAR T cell therapy shows promise for treating pediatric central nervous system (CNS) tumors, offering new hope where standard treatments fail. This review explores its efficacy, safety, and challenges for improving outcomes in pediatric CNS cancers.

Keywords:
CAR TCAR T solid tumorsCNS tumorsDIPGDMGbrain cancerglioblastomaglioma

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Area of Science:

  • Pediatric Oncology
  • Neuro-oncology
  • Immunotherapy

Background:

  • Central nervous system (CNS) tumors are the second most common pediatric cancer and the leading cause of cancer-related death in children.
  • High-risk pediatric CNS tumors have poor prognoses with current standard treatments, necessitating novel therapeutic strategies.
  • CAR T cell therapy is an emerging immunotherapy with early reported clinical responses in this patient population.

Purpose of the Study:

  • To review the clinical efficacy and toxicity of CAR T cell therapy for pediatric CNS tumors.
  • To analyze antigen heterogeneity and challenges in the clinical implementation of CAR T cell therapy.
  • To provide a broader perspective on immunotherapies, including TILs, NK, and LAK cells, for pediatric CNS malignancies.

Main Methods:

  • Comprehensive analysis of reported clinical trials and literature on CAR T cell therapy for pediatric CNS tumors.
  • Evaluation of systemic versus locoregional delivery methods for CAR T cells.
  • Inclusion of studies on other immunotherapeutic approaches like tumor-infiltrating lymphocytes (TILs), NK cells, and lymphokine-activated killer (LAK) cells.

Main Results:

  • CAR T cell therapy has demonstrated early objective clinical responses in pediatric CNS tumors.
  • Systemic and locoregional delivery methods present distinct toxicity profiles.
  • Antigen heterogeneity poses a significant challenge for effective CAR T cell targeting in CNS tumors.

Conclusions:

  • CAR T cell therapy represents a promising alternative for high-risk pediatric CNS tumors.
  • Addressing challenges in delivery, antigen targeting, and clinical implementation is crucial for maximizing therapeutic potential.
  • A broader view of immunotherapies offers a comprehensive strategy for pediatric CNS malignancies.