What is PD-1 (Programmed Death-1) targeted therapy?

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Last updated: February 10, 2025View editorial policy

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From the Guidelines

PD-1 targeted therapy is a type of immunotherapy that works by blocking the PD-1 receptor, preventing it from binding to its ligand, PD-L1, and thereby disrupting the signaling that inhibits the immune response against cancer cells.

Key Points

  • PD-1 targeted therapy is used to treat various types of cancer, including non-small cell lung cancer (NSCLC), renal cell carcinoma (RCC), and squamous cell lung cancer (SqCLC) 1.
  • The therapy involves the use of monoclonal antibodies, such as pembrolizumab and nivolumab, that target the PD-1 receptor or its ligand, PD-L1 1.
  • The goal of PD-1 targeted therapy is to enhance the body's immune response against cancer cells, leading to improved overall survival and progression-free survival rates 1.
  • The therapy has been shown to be effective in patients with high PD-L1 expression, and its use has been integrated into treatment guidelines for various types of cancer 1.

Examples of PD-1 Targeted Therapies

  • Pembrolizumab: a PD-1-blocking antibody approved for the first-line treatment of metastatic NSCLC in patients with high PD-L1 expression 1.
  • Nivolumab: a PD-1 immune checkpoint inhibitor monoclonal antibody approved for the treatment of advanced RCC 1.

Important Considerations

  • PD-1 targeted therapy can be associated with immune-related adverse events (irAEs), which can involve any organ or system of the body 1.
  • The incidence and onset of irAEs vary based on the specific therapy and patient population, and a high level of suspicion is required to diagnose and manage these events 1.

From the Research

Overview of PD-1 Targeted Therapy

  • PD-1 (Programmed Death-1) is an inhibitory receptor expressed on activated T cells, which can reverse immune suppression and release T cell activation 2.
  • PD-1 targeted therapy, also known as immune checkpoint inhibitors, has shown unprecedented success in a wide variety of human cancers, including non-small-cell lung cancer (NSCLC), melanoma, renal cell carcinoma (RCC), and lymphoma 2, 3, 4.

Mechanism of PD-1 Targeted Therapy

  • PD-1 is activated by its ligands, mainly PD-L1, which recruits the phosphatase SHP-2 and leads to inhibition of T cell proliferation, activation, cytokine production, and cytotoxic T lymphocytes (CTLs) killer functions 3.
  • Tumor cells can hijack the PD-1/PD-L1 pathway to escape host immune surveillance by overexpressing PD-L1, providing a scientific rationale for clinical application of immune checkpoint inhibitors in oncology 3.

Clinical Applications of PD-1 Targeted Therapy

  • Antibodies targeting PD-1/PD-L1, such as nivolumab, pidilizumab, and pembrolizumab, have shown remarkable objective response and durable remission with acceptable toxicity profile in various types of cancer 2, 3, 4.
  • PD-1 targeted therapy has been effective in treating melanoma, lymphoma, and mismatch-repair deficient tumors, with ongoing clinical trials evaluating its role in other types of cancer 3, 4.

Challenges and Future Perspectives

  • Despite the success of PD-1 targeted therapy, most patients are still refractory to anti-PD-1/PD-L1 therapy, highlighting the need for identifying predictive biomarkers and designing rational combination therapies 3.
  • Advanced nanotechnology has been developed to enhance the efficiency of PD-1 targeted therapy, including nanoprobe-assisted analysis of PD-L1 and functional nanocarriers for targeted delivery of PD-L1 immune checkpoint inhibitors 5.

References

Guideline

Guideline Directed Topic Overview

Dr.Oracle Medical Advisory Board & Editors, 2025

Research

Roles of PD-1/PD-L1 Pathway: Signaling, Cancer, and Beyond.

Advances in experimental medicine and biology, 2020

Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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