Target intelligence / Profile preview

Cell-based therapy

Molecular classification
Other
01

Overview

Cell-based therapy is a therapeutic modality involving the administration of live cells to a patient to achieve a clinical effect, rather than targeting a specific molecular entity like a receptor or enzyme (NCI, 2024). This approach encompasses diverse treatments such as chimeric antigen receptor (CAR) T-cell therapies for cancer and mesenchymal stem cell (MSC) therapies for regenerative medicine (June et al., 2018). These therapies operate through broad cellular and paracrine interactions, meaning they utilize both direct cell-to-cell contact and the secretion of various bioactive factors—including cytokines, chemokines, and exosomes—to influence the host environment (Gnecchi et al., 2016). This multi-pronged mechanism allows cell therapies to perform complex functions such as immunomodulation, tissue repair, and targeted cytotoxicity that are difficult to achieve with single-target drugs. Because the therapeutic agent is a living cell, the biological response is dynamic and dependent on the cellular microenvironment. Safety monitoring is critical due to risks like cytokine release syndrome and potential long-term persistence or transformation of the administered cells (Abou-El-Enein et al., 2021).

Other names
Not applicable – cell-based therapy with broad cellular and paracrine interactionsCellular therapyCytotherapyAdoptive cell transferRegenerative medicine
02

Mechanism of action

Cell-based therapies exert their effects through direct cellular interactions and the secretion of paracrine factors (cytokines, growth factors, exosomes) that modulate the host immune system and promote tissue repair (Gnecchi et al., 2016; June et al., 2018).

03

Biological functions

Immune responseCell proliferationTissue repairParacrine signalingImmunomodulationApoptosis induction
04

Disease associations

CancerInflammationAutoimmune diseaseNeurodegenerative diseaseCardiovascular diseaseGraft-versus-host disease
05

Safety considerations

Cytokine release syndrome (CRS)Immune effector cell-associated neurotoxicity syndrome (ICANS)Graft-versus-host disease (GvHD)On-target off-tumor toxicityTumorigenicityImmunogenicity
06

Interacting drugs

Tisagenlecleucel

6 more in the full profile.

07

Biomarkers

Cell surface markers (e.g., CD19, CD34)Cytokine levels (e.g., IL-6, CRP)Cell expansion and persistence levels

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