What is the mechanism of action of valproate (valproic acid)?

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Last updated: April 18, 2025View editorial policy

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From the Guidelines

Valproate primarily works by enhancing the inhibitory effects of gamma-aminobutyric acid (GABA) in the brain, increasing GABA levels, blocking voltage-gated sodium channels, and inhibiting T-type calcium channels, making it effective for treating epilepsy, bipolar disorder, and migraines. The mechanism of action of valproate involves multiple pathways, including the inhibition of GABA transaminase, the enzyme that breaks down GABA, and the stimulation of glutamic acid decarboxylase, which increases GABA synthesis 1. Additionally, valproate affects intracellular signaling pathways by inhibiting histone deacetylase, which influences gene expression and may contribute to its mood-stabilizing effects.

Key Mechanisms

  • Inhibition of GABA transaminase, increasing GABA levels
  • Stimulation of glutamic acid decarboxylase, increasing GABA synthesis
  • Blockage of voltage-gated sodium channels, reducing neuronal excitability
  • Inhibition of T-type calcium channels, important in controlling absence seizures
  • Inhibition of histone deacetylase, influencing gene expression and contributing to mood-stabilizing effects

The most recent evidence from 2024 supports the use of valproate in the management of adult patients presenting to the emergency department with seizures, highlighting its effectiveness in cessation of seizures in approximately half of all patients with benzodiazepine-resistant status epilepticus 1. Typical dosing ranges from 750-2000 mg daily for adults, divided into multiple doses, with therapeutic blood levels between 50-100 μg/mL. Regular monitoring of blood levels is necessary to ensure efficacy while avoiding toxicity, and liver function tests should be performed periodically as valproate can cause hepatotoxicity in some patients.

Clinical Considerations

  • Monitoring of blood levels to ensure efficacy and avoid toxicity
  • Periodic liver function tests to assess for hepatotoxicity
  • Consideration of potential adverse drug reactions
  • Importance of early treatment to reduce morbidity and mortality in status epilepticus 1

From the FDA Drug Label

The mechanisms by which valproate exerts its antiepileptic effects have not been established. It has been suggested that its activity in epilepsy is related to increased brain concentrations of gamma-aminobutyric acid (GABA) The mechanism of action of valproate is not established, but it is suggested to be related to increased brain concentrations of gamma-aminobutyric acid (GABA) 2.

From the Research

Mechanism of Action of Valproate

The mechanism of action of valproate is complex and involves multiple pathways. Some of the key mechanisms include:

  • Increasing GABA turnover and potentiating GABAergic functions in specific brain regions, such as substantia nigra, which are thought to be involved in the control of seizure generation and propagation 3, 4
  • Reducing the release of the epileptogenic amino acid gamma-hydroxybutyric acid 3, 4
  • Blocking cell firing induced by NMDA-type glutamate receptors 3, 4
  • Altering dopaminergic and serotonergic functions 3
  • Direct action on ion channels, limiting sustained repetitive neuronal firing 3, 4
  • Inhibiting histone deacetylases (HDACs) activity, leading to chromatin remodeling and regulation of gene expression 5

Neuroprotective Mechanisms

Valproate has also been shown to have neuroprotective effects, which may be related to its ability to:

  • Regulate gene expression at the molecular level, through epigenetic mechanisms 5
  • Affect neuronal survival/apoptosis and proliferation/differentiation balance 5
  • Modulate synaptic plasticity, by acting both directly on neurons and indirectly through glial cells 5

Clinical Implications

The multiple mechanisms of action of valproate may contribute to its broad spectrum of clinical effects, including:

  • Anticonvulsant activity against different seizure types 3, 6, 4
  • Antipsychotic and antidystonic efficacy 3
  • Treatment of bipolar disorder, migraines, and other conditions 6, 7

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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