Medications Development for Alcohol Use Disorder | NIAAA

Treatment for alcohol use disorder (AUD) includes behavioral treatments such as mutual support groups and individual therapy, as well as pharmaceutical treatments. Often these are used in combination.

Current Medications

Currently, there are three U.S Food and Drug Administration (FDA)-approved medications for the treatment of AUD.

  • Disulfiram (Antabuse®): In 1949, disulfiram became the first drug approved to treat alcoholism. The drug works by increasing the concentration of acetaldehyde, a toxic byproduct that occurs when alcohol is broken down in the body. Excess amounts of this byproduct cause unpleasant symptoms, such as nausea and flushing of the skin. The anticipation of these effects can help some people avoid drinking while taking disulfiram. For over 40 years, disulfiram was the only medication physicians could offer to their patients who were battling alcoholism.
  • Naltrexone (oral medication: Revia® and injectable: Vivitrol®): However, the FDA approved naltrexone as an oral medication in 1994 and as an extended-release injectable in 2006. Naltrexone blocks opioid receptors involved in the pleasant sensations associated with drinking and can reduce alcohol craving.
  • Acamprosate (Campral®): In 2004, 10 years after the FDA approved naltrexone, acamprosate was approved. Acamprosate is thought to ease the negative effects related to quitting drinking by dampening glutamate activity and reducing some of the brain’s hyperexcitability associated with alcohol withdrawal.

In addition, benzodiazepines have shown efficacy in treating acute alcohol withdrawal symptoms. Benzodiazepines target the gamma aminobutyric acid (GABA) neurotransmitter to curb excitability in the brain during alcohol withdrawal, allowing the brain to restore to its natural balance.

Current Research in Medications Development

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.
 

NIAAA's Medications Development Branch (MDB) within the Division of Treatment and Recovery (DTR) supports the development of medications for AUD through grants and contracts to academic sites and small businesses, as well as partnerships with pharmaceutical companies. The goal of the MDB is to improve the care and treatment of those affected by AUD through supporting the evaluation of promising safe and effective medications.

NIAAA’s Clinical Investigations Group (NCIG) consists of an NIAAA steering committee and a network of clinical sites with the charge of testing medications in a clinical setting. NCIG works closely with the phar/node/29233maceutical industry in the development of drugs to treat alcoholism and helps speed the process for getting these medications on the market.

Research on promising medications (not FDA-approved) to treat AUD includes:

  • 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. 
 

Sponsored Studies

Several NIAAA-sponsored pharmacotherapy databases are available to researchers per request and approval.