Evidence map›Paper›PMID 31755996›Full record

ArticleEpilepsia2019

Personalized medicine: Vinpocetine to reverse effects of GABRB3 mutation.

Santoshi Billakota, J Michael Andresen, Bryant C Gay, Gregory R Stewart, Nikolai B Fedorov, Aaron C Gerlach, Orrin Devinsky

Abstract readCase Reports
In one paragraph

Article in Epilepsia, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.

0numbers the graph read from it
0cells of the map it votes in
15citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

15 citing papers in PubMed.

  1. Observational
  2. Review
  3. Infantile epileptic spasms syndrome: Mechanisms and therapeutic approaches.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2026
    Review
  4. Article
  5. Article
  6. Review
  7. Article
  8. Review
  9. Article
  10. Review
  11. Article
  12. Symptomatic and neurotrophic effects of GABAA receptor positive allosteric modulation in a mouse model of chronic stress.Neuropsychopharmacology : official publication of the American College of Neuropsychopharmacology · 2022
    Article
  13. Review
  14. Article
  15. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

7 authors.

Santoshi BillakotaNYU Langone Comprehensive Epilepsy Center, New York University Langone School of Medicine, New York, New York.ORCID 0000-0003-3610-5640
J Michael AndresenPairnomix, Plymouth, Minnesota.
Bryant C GayPairnomix, Plymouth, Minnesota.
Gregory R StewartPairnomix, Plymouth, Minnesota.
Nikolai B FedorovCharles River Discovery, Cleveland, Ohio.
Aaron C GerlachIcagen, Durham, North Carolina.
Orrin DevinskyNYU Langone Comprehensive Epilepsy Center, Department of Neurology, Neurosurgery, and Psychiatry, New York University Langone School of Medicine, New York, New York.ORCID 0000-0003-0044-4632

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectiveTo screen a library of potential therapeutic compounds for a woman with Lennox-Gastaut syndrome due to a Y302C GABRB3 (c.905A>G) mutation.

methodsWe compared the electrophysiological properties of cells with wild-type or the pathogenic GABRB3 mutation.

resultsAmong 1320 compounds, multiple candidates enhanced GABRB3 channel conductance in cell models. Vinpocetine, an alkaloid derived from the periwinkle plant with anti-inflammatory properties and the ability to modulate sodium and channel channels, was the lead candidate based on efficacy and safety profile. Vinpocetine was administered as a dietary supplement over 6 months, reaching a dosage of 20 mg three times per day, and resulted in a sustained, dose-dependent reduction in spike-wave discharge frequency on electroencephalograms. Improved language and behavior were reported by family, and improvements in global impression of change surveys were observed by therapists blinded to intervention. SIGNIFICANCE: Vinpocetine has potential efficacy in treating patients with this mutation and possibly other GABRB3 mutations or other forms of epilepsy. Additional studies on pharmacokinetics, potential drug interactions, and safety are needed.

Indexed as

AdultDose-Response Relationship, DrugElectroencephalographyFemalegamma-Aminobutyric AcidHEK293 CellsHumansLennox Gastaut SyndromeMutationNeuroprotective AgentsPrecision MedicineReceptors, GABA-AVinca AlkaloidsGABRB3 protein, humangamma-Aminobutyric AcidNeuroprotective AgentsReceptors, GABA-AVinca AlkaloidsvinpocetineepilepsyLennox-Gastautprecision medicinerefractoryvinpocetine

Identifiers

PMID31755996
PMCPMC7004153

What OpenQuestion holds

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LicenceCC BY-NC
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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.