Promising Gene Therapy for Severe Problem Drinking
Promising Gene Therapy for Severe Problem Drinking
Gene Therapy Offers Hope for Severe Alcohol Use Disorder

For people suffering from severe alcohol use disorder, a promising new gene therapy trial offers hope for an effective treatment by rebalancing the brain area associated with addiction. This groundbreaking research, conducted by a team at Oregon Health & Science University (OHSU) in Portland, aims to reset the brain’s dopamine reward pathways through gene therapy.
Dr. Tucker Woods, the associate medical director of Lenox Health Greenwich Village in New York City, expressed the potential of this therapy, stating, “With alcohol alone, there’s generally more than 100,000 deaths in the United States per year. And just think about the impacts on families when somebody’s addicted. The impact is not just to that patient, it’s also their family and friends. So if this pans out in humans, it could potentially be a game changer if they could fix that hijacked pleasure/reward pathway of the brain.”
Alcohol-related deaths rank as the fourth-leading preventable cause of death in the United States. The increasing number of individuals consuming alcohol, with the average American consuming over 500 standard drinks in a year, has led to a rise in health costs attributed to serious heart, liver, blood, and digestive problems. Additionally, alcohol consumption increases the risk of various cancers, weakens the immune system, and results in learning and memory problems, mental health issues, social problems, and dependency.
Understanding the underlying science behind addiction is crucial in combating the problem effectively. Dr. Woods emphasized the shift in understanding addiction, stating, “Up until 1980 or so, people thought that addiction was essentially a moral failure. Medicine then came around and said, ‘Not so fast.’ Medicine said that addictions are very much chronic relapsing illnesses like diabetes, high blood pressure, schizophrenia, depression, and other disorders. The true cause of addiction involves biological, psychological, and social forces that manifest changes in the brain.”
Dr. Kathleen Grant, a senior study co-author and professor at OHSU, expressed optimism about the potential applications of this treatment. The gene therapy utilizing glial-derived neurotrophic factor (GDNF) successfully reset the reward pathways in monkeys, reducing alcohol consumption by over 90%. However, this treatment would only be suitable for severe cases that have proven unresponsive to other therapeutic approaches.
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While the results are promising, caution is necessary before generalizing these findings to other types of addictions. Regarding stimulant addiction, which directly involves the dopamine pathways, further studies are required to identify potential adverse effects.
If the gene therapy proves successful in human trials, it could provide a breakthrough in the treatment of severe alcohol addiction and potentially open doors to addressing other types of addiction. However, additional research and testing are needed before this therapy becomes widely available.
The study was published in the journal Nature Medicine on August 14, 2023.

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Sources: – Tucker Woods, DO, Chair, Emergency Department, and Associate Medical Director, Lenox Health Greenwich Village, New York City – Kathleen Grant, PhD, Professor, Oregon National Primate Research Center, Professor, Behavioral Neuroscience, School of Medicine, and Chief, Division of Neuroscience, Oregon National Primate Research Center Behavioral Neuroscience Graduate Program – Nature Medicine, August 14, 2023