Target intelligence / Profile preview

Immune checkpoint inhibitor

Molecular classification
Other
01

Overview

The term "checkpoint inhibitor" does not refer to a specific molecular target, but rather to a class of therapeutic agents—primarily monoclonal antibodies—that block immune checkpoint proteins on immune cells such as T lymphocytes or, less commonly, on tumor cells. The most widely targeted immune checkpoints in cancer therapy are PD-1, PD-L1, and CTLA-4. Checkpoint inhibitors prevent these proteins from inhibiting T cell activity, thereby boosting the immune system's ability to detect and destroy cancer cells. Drugs in this class have transformed the treatment landscape for multiple cancer types by providing durable responses for some patients, though their use is limited by serious immune-related side effects. Accurate description of a "checkpoint inhibitor" requires reference to its specific molecular target, such as "Programmed cell death protein 1 (PD-1)" or "Cytotoxic T-lymphocyte-associated protein 4 (CTLA-4)"[5][6][4][1][2][3]. Note: "Checkpoint inhibitor" is a class/therapeutic approach, not a single molecule or receptor—thus, marking as is_incorrect=true for a molecular target index. Specific molecular targets within this class include PD-1, PD-L1, CTLA-4, LAG-3, etc.

Other names
Checkpoint inhibitorImmune checkpoint blockadeICI
02

Mechanism of action

Blockade of immune checkpoint receptors or ligands (e.g., PD-1, PD-L1, CTLA-4) on T cells and/or tumor cells, thereby preventing negative regulation of the immune response and enabling enhanced T cell activation against cancer cells[5][6].

03

Biological functions

Immune responseSignal transduction
04

Disease associations

CancerInflammation (less commonly targeted, mostly cancer)
05

Safety considerations

Immune-related adverse events (irAEs) in multiple organs (e.g., colitis, pneumonitis, hepatitis, dermatitis, endocrinopathies)Risk of autoimmune toxicity requiring immunosuppressionNot all patients respond, thus requiring robust patient selection[4][5][6]
06

Interacting drugs

8 more in the full profile.

07

Biomarkers

PD-L1 expressionTumor mutation burden (TMB)Microsatellite instability (MSI)dMMR (deficient mismatch repair)Microbiome composition (emerging)Interferon-gamma gene signature[6]

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