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A new module in the drug development process: preclinical multi-center randomized controlled trial of R-ketamine on alcohol relapse.

Marcus W Meinhardt, Ivan Skorodumov, Jérôme Jeanblanc, Federica Benvenuti, Fahd François Hilal, Esi Domi, Camille André, Sandra Bodeau, Virginie Jeanblanc, Kevin Domanegg, Roberto Ciccocioppo, Mickaël Naassïla, Rainer Spanagel

Neuropsychopharmacology : official publication of the American College of Neuropsychopharmacology May 1, 2025 DOI: 10.1038/s41386-025-02071-w via PubMed

Summary

AI-generated from the abstract

A new approach in psychiatric drug development, the preclinical randomized controlled trial (preRCT), was tested across three European centers using a rat model of alcohol relapse. Ketamine (20 mg/kg) reduced relapse, while R-ketamine worked only in females at the same dose; a higher dose (40 mg/kg) was effective in males. The sex-dependent effects were linked to plasma R-ketamine levels, which were twice as high in females. R-ketamine produced a lasting reduction in alcohol consumption without adverse effects. The findings support moving to a clinical trial that accounts for sex differences.

Study at a glance

Characteristics Preclinical randomized controlled trial Peer reviewed
Population Rats
Interventions Ketamine R-ketamine
Dose 20 mg/kg, 40 mg/kg
Keywords Addiction treatment Ketamine therapy Alcohol dependence Gender medicine Substance abuse recovery
Citations 8
Key finding R-ketamine reduces alcohol relapse in rats with sex-dependent effects linked to higher plasma levels in females.

Abstract

The drug development process in psychiatry faces significant challenges due to low reproducibility rates in animal testing, which often leads to translation failures. To address this issue, we introduce a new approach in psychiatric drug development: a preclinical randomized controlled trial (preRCT). To demonstrate its potential utility, we conducted a multi-center preRCT using the alcohol deprivation effect (ADE) model to assess the impact of ketamine and R-ketamine on alcohol relapse across three European research centers. Ketamine (20 mg/kg) significantly reduced relapse, while R-ketamine showed efficacy only in females. A higher dose of R-ketamine (40 mg/kg) was also effective in males. These sex-dependent effects were linked to plasma R-ketamine levels, which were two-fold higher in female compared to male rats. Notably, R-ketamine demonstrated a lasting reduction in alcohol consumption without adverse effects. In conclusion, our preRCT demonstrates R-ketamine's effectiveness in reducing alcohol relapse and supports translation to a clinical RCT that accounts for sex-dependent effects.

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