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National Institute on Alcohol Abuse and Alcoholism (NIAAA)

Current Research in Medications Development

Research on Promising Medications (not approved by the U.S. Food and Drug Administration [FDA]) to Treat Alcohol Use Disorder (AUD)

  • Varenicline (Chantix®), a medication approved for smoking cessation, was found in a recent 200-patient clinical trial conducted by NIAAA’s Clinical Investigations Group (NCIG) to reduce alcohol consumption and craving among people who are alcohol-dependent. Varenicline may work by partially stimulating receptors for nicotinic acetylcholine, a promising molecular target implicated in both nicotine and alcohol use disorders.
  • Gabapentin, a generic anticonvulsant used to treat pain conditions and epilepsy, has shown promise as an effective treatment for alcohol dependence, based on the results of a recent 150-patient clinical trial of the medication. The study found that alcohol dependent patients using gabapentin were more likely to stop drinking or refrain from heavy drinking than those taking placebo.
  • Topiramate, a generic anticonvulsant used to treat seizures and prevent migraine headaches, targets the neurotransmitters GABA and glutamate. Research has shown that it appears to be effective in reducing drinking in alcohol-dependent patients.
  • Ondansetron, a drug used to treat nausea and vomiting, has shown promise in reducing drinking in patients who developed alcohol dependence early in life. In addition, a recent 300-patient clinical trial has shown that ondansetron works better in individuals who possess specific combinations of genes that regulate the function and binding of serotonin, a brain chemical affected by the treatment. Ondansetron is thought to work by blocking serotonin-3 receptors.
  • Nalmefene (Selincro®), an opioid receptor antagonist, was recently approved by European Medications Agency (the FDA-equivalent in Europe) for use in the European Union to treat alcohol dependence. In two main studies involving 1,322 men and women with alcohol dependence, Selincro® was shown to reduce the number of heavy drinking days and daily alcohol consumption. 
  • Baclofen, a GABAB agonist medication used to treat muscle spasms, may have beneficial effects in encouraging abstinence, especially in alcoholic patients with cirrhosis. 
A more detailed review of the efficacy and safety of these (and other) medications can be found here:
NIAAA continues to support studies of other new and existing medications to treat AUD, to provide physicians with additional tools for treating their patients experiencing AUD, and to help match the best treatment to the patients most likely to respond to each medication.
Medications Development Branch (MDB) within the Division of Treatment and Recovery (DTR)  
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