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 GABA_B agonist medication used to treat muscle spasms, may have beneficial effects in encouraging abstinence, especially in alcoholic patients with cirrhosis.