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Low dose valproate
Low-dose valproate inhibits histone deacetylases (HDACs) and enhances histone acetylation, leading to altered gene expression and potential anti-inflammatory or anti-proliferative effects.
Low-dose valproate inhibits histone deacetylases (HDACs) and enhances histone acetylation, leading to altered gene expression and potential anti-inflammatory or anti-proliferative effects. Used for Cancer (various types, under investigation), Neurological and psychiatric disorders (off-label/investigational).
At a glance
| Generic name | Low dose valproate |
|---|---|
| Also known as | Low valproate |
| Sponsor | Mario Negri Institute for Pharmacological Research |
| Drug class | Histone deacetylase (HDAC) inhibitor |
| Target | Histone deacetylases (HDACs) |
| Modality | Small molecule |
| Therapeutic area | Oncology; Neurology; Immunology |
| Phase | FDA-approved |
Mechanism of action
Valproic acid is a short-chain fatty acid that acts as a histone deacetylase inhibitor at therapeutic doses. At lower doses, it may preferentially modulate specific signaling pathways and gene expression patterns relevant to various disease states. The mechanism at low doses is thought to involve epigenetic modifications that can reduce inflammation, enhance differentiation, or inhibit proliferation depending on the target tissue.
Approved indications
- Cancer (various types, under investigation)
- Neurological and inflammatory conditions (under investigation)
Common side effects
- Nausea and gastrointestinal upset
- Tremor
- Weight gain
- Hepatotoxicity
- Thrombocytopenia
Key clinical trials
- Ketogenic Diet for New-Onset Absence Epilepsy (PHASE3)
- Low vs. Standard Daily Doses of Antiepileptic Drugs in Newly Diagnosed, Previously Untreated Epilepsy(STANDLOW) (PHASE4)
- Fast Acute Sedation at Intensive Care vs. High-dose i.v. Anti-seizure Medication for Treatment of Non-convulsive Status Epilepticus (FAST-trial) (PHASE3)
- Sodium Valproate in Patients With Acute Ischemic Stroke (PHASE2)
- MAGE-C2 TCR T Cell Trial to Treat Melanoma and Head and Neck Cancer (PHASE1, PHASE2)
- FX-322 in Sensorineural Hearing Loss (PHASE1, PHASE2)
- TELSTAR: Treatment of ELectroencephalographic STatus Epilepticus After Cardiopulmonary Resuscitation (NA)
- add-on Low Dose Memantine in Middle-to-old Aged Bipolar II Disorder Patients (PHASE2, PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Low dose valproate CI brief — competitive landscape report
- Low dose valproate updates RSS · CI watch RSS
- Mario Negri Institute for Pharmacological Research portfolio CI