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Updated: May 11, 2026

Isolation of Cortical Microglia with Preserved Immunophenotype and Functionality From Murine Neonates
Published on: January 30, 2014
Emerging Microglial Therapies and Targets in Clinical Trial
1Neuroscience Translational Medicine, Takeda Pharmaceutical Co. Ltd., Tokyo, Japan.
Abstract:
Modulation of microglia function for treatment of neurodegenerative and neuropsychiatric disorders is an emerging field of neuroscience drug development. This is largely attributed to human genetic association studies combined with biological evidence indicating that the innate immune system acts as a causal contributor superimposed on the reactive component of neuronal loss in neurological dysfunction. The identification of disease risk gene variants that encode immune-modulatory proteins in microglia provides tools to evaluate how microglia cellular function or dysfunction affect neuronal health. The development of clinical stage therapeutic compounds that modify myeloid cell function enables us to investigate how modulating microglia function could become a transformational approach to mitigate neurological disorders. Improving our ability to boost microglia-promoting homeostatic and reparative functions hopefully will translate into achieving a better outcome for patients affected by neurological diseases. In this chapter, we aim to provide an overview of the microglial emerging therapies and targets being studied in current clinical trials.
Insights
Emerging neuroscience therapies focus on modulating microglia function to treat neurodegenerative and neuropsychiatric disorders. Targeting these innate immune cells offers a promising approach to improve patient outcomes in neurological diseases.
Area of Science:
- Neuroscience
- Immunology
- Drug Development
Background:
- Human genetic studies and biological evidence implicate the innate immune system, specifically microglia, in neurological dysfunction.
- Microglia's role in neuronal health is increasingly recognized, with genetic variants linked to disease risk.
- The innate immune system is a key factor in neurodegenerative and neuropsychiatric disorders.
Purpose of the Study:
- To provide an overview of emerging therapies targeting microglia function for neurological disorders.
- To highlight current clinical trials investigating microglial modulation as a therapeutic strategy.
- To explore the potential of boosting microglia's homeostatic and reparative functions.
Main Methods:
- Review of human genetic association studies linking immune-modulatory proteins in microglia to disease risk.
- Analysis of biological evidence supporting the innate immune system's causal role in neurological dysfunction.
- Examination of clinical-stage therapeutic compounds designed to modulate myeloid cell function.
Main Results:
- Identification of disease risk gene variants provides tools to assess microglia's impact on neuronal health.
- Development of clinical-stage therapeutics enables investigation into modulating microglia function.
- Emerging therapies aim to enhance microglia's beneficial homeostatic and reparative functions.
Conclusions:
- Modulating microglia function is a promising therapeutic avenue for neurodegenerative and neuropsychiatric disorders.
- Targeting microglia offers a transformational approach to mitigate neurological diseases.
- Boosting beneficial microglia functions may lead to improved patient outcomes.
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