Genetically Targeted Medication Shows Great Promise in Treating Alcohol Addiction

For the first time in alcohol addiction research, UVA investigators have successfully treated alcohol-dependent individuals with medication that is tailored specifically to match their genetic profile.

"Our findings suggest a new paradigm for the treatment of alcoholism, as well as a major breakthrough in individualized medicine for predetermined genotypes," says Bankole Johnson, MD, PhD, study leader and professor and chair of the UVA Department of Psychiatry and Neurobehavioral Sciences.

The study, to be published in the March issue of the American Journal of Psychiatry and now available online, tested 283 genetically profiled alcoholics for the efficacy of ondansetron, a serotonin antagonist drug.

Previous research by UVA scientists has found that specified variations in the serotonin transporter gene, SLC6A4, play a significant role in influencing drinking intensity. Furthermore, past UVA research has identified ondansetron as a likely pharmaceutical target for serotonin-related genes.

Serotonin is a brain chemical that is involved in the regulation of pain perception, sleep, mood and other psychological processes. Studies have shown that serotonin mediates the rewarding effects of alcohol.

In this latest study, UVA researchers randomized alcoholics by genotype (LL vs. Sx and TT vs. Gx) in a controlled, double-blind clinical trial. Subjects received either ondansetron or placebo for 11 weeks, and all received standard cognitive behavioral therapy. A majority of subjects were white males, and more than 65 percent of subjects completed the study in its entirety. The study was funded by the National Institute on Alcohol Abuse and Alcoholism (NIAAA) of the National Institutes of Health (NIH). (View NIAAA press release).

Study findings show that ondansetron is indeed a promising therapeutic agent for the treatment of severe alcohol consumption among alcohol-dependent individuals with the predicted genetic marker - a marker that's responsible for the amount of pleasure certain people may perceive while drinking or may perceive as a craving when they stop drinking, Johnson explains.

"The treatment response among those who received ondansetron was remarkable," says Johnson. "What this tells us is that we have measurable evidence that personalized medicine is indeed a viable treatment for alcohol dependence.

An animation of how the medication works

The primary outcome tested by researchers involved ondansetron's effects on the severity of alcohol consumption in selected alcohol-dependent individuals. The study was confined to one secondary variable - the percentage of days abstinent, in order to provide clinicians additional efficacy information. Study findings demonstrated a predicted therapeutic response to ondansetron, which increased the percentage of days abstinent relative to placebo for predetermined genotypes.