Related Experiment Video

Updated: Oct 30, 2025

Author Spotlight: Insight Into Advances in Prion Diseases Research
10:40

Author Spotlight: Insight Into Advances in Prion Diseases Research

Published on: August 11, 2023

629

Small molecule therapeutics for neuroinflammation-mediated neurodegenerative disorders

Silke Miller1, Maria-Jesus Blanco1

  • 1Sage Therapeutics, Inc. 215 First Street Cambridge Massachusetts 02142 USA maria-jesus.blanco@sagerx.com.

RSC Medicinal Chemistry
|July 5, 2021
PubMed

Insights

Chronically activated microglia drive neuroinflammation in neurodegenerative diseases. Targeting these immune cells with novel small molecules in clinical trials offers a promising therapeutic strategy to slow disease progression.

Area of Science:

  • Neuroscience
  • Immunology
  • Pharmacology

Background:

  • Microglia, the brain's innate immune cells, become chronically activated in neurodegenerative disorders.
  • Activated microglia release inflammatory factors, damaging neurons and astrocytes, exacerbating neurodegeneration.

Purpose of the Study:

  • To review recent advancements in targeting microglia for neurodegenerative diseases.
  • To highlight small molecule compounds in clinical trials for neuroinflammation.

Main Methods:

  • Literature review of recent scientific publications.
  • Analysis of therapeutic targets and clinical trial progress for microglia modulation.

Main Results:

  • Identified key molecular targets on activated microglia.
  • Cataloged small molecule compounds progressing through clinical trials.
  • Demonstrated the potential of microglia-modulating therapies.

Conclusions:

  • Modulating microglia activation is a viable therapeutic strategy for neuroinflammatory diseases.
  • Emerging small molecule compounds show promise in clinical development.
  • Further research into microglia-targeted therapies is warranted.

More Related Videos

Improved 3D Hydrogel Cultures of Primary Glial Cells for In Vitro Modelling of Neuroinflammation09:19

Improved 3D Hydrogel Cultures of Primary Glial Cells for In Vitro Modelling of Neuroinflammation

Published on: December 8, 2017

15.1K
Lipidomics and Transcriptomics in Neurological Diseases09:58

Lipidomics and Transcriptomics in Neurological Diseases

Published on: March 18, 2022

3.7K

Related Concept Videos

Alzheimer's Disease: Treatment01:22

Alzheimer's Disease: Treatment

Alzheimer's Disease (AD), a neurodegenerative disorder, is pathologically identified by amyloid plaques and neurofibrillary tangles composed of tau protein. AD pharmacotherapy aims to manage cognitive symptoms, delay disease progression, and treat behavioral symptoms. The treatment is primarily symptomatic and palliative, with no definitive disease-modifying therapy available. Cholinesterase inhibitors, including donepezil (Aricept), rivastigmine (Exelon), and galantamine (Razadyne), are...
428
siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the...
17.4K
Drugs Affecting Neurotransmitter Synthesis01:29

Drugs Affecting Neurotransmitter Synthesis

Drugs affecting neurotransmitter synthesis can impact the adrenergic neuron and the synthesis of neurotransmitters. For example, α-methyltyrosine and carbidopa target specific enzymes involved in catecholamine synthesis. α-methyltyrosine inhibits the enzyme tyrosine hydroxylase, which converts tyrosine into dopamine. By blocking this enzyme, α-methyltyrosine reduces dopamine production and other catecholamines. Carbidopa, on the other hand, inhibits the enzyme dopa decarboxylase,...
1.8K
Parkinson's Disease: Overview01:15

Parkinson's Disease: Overview

Neurodegenerative disorders are progressive diseases that cause irreversible damage and loss to neurons in specific brain areas. Examples of these disorders include Parkinson's disease, Alzheimer's disease, Multiple Sclerosis (MS), and Amyotrophic Lateral Sclerosis (ALS). These disorders share characteristics such as proteinopathies, selective neuronal vulnerability, and a complex interplay between genetic and environmental factors. The primary therapeutic goal for these conditions is...
995
Neurochemical Transmission: Sites of Drug Action01:26

Neurochemical Transmission: Sites of Drug Action

Neurochemical transmission, the conduction of electrical impulses between neurons mediated by neurotransmitters, plays a vital role in various physiological processes. Autonomic drugs exert their effects by modulating neurotransmission within the autonomic nervous system. For instance, drugs such as hemicholinium block the precursor uptake necessary for synthesizing acetylcholine, an essential autonomic neurotransmitter. Following synthesis, neurotransmitters are stored in vesicles. Metyrosine...
3.0K