Gene Therapy May Be Used to Treat Alcohol Use Disorder


Alcohol use disorder, also known as alcoholism, affects roughly 30 million people 12-years-old and older in the U.S. alone. However, it impacts each person in different ways—with varying triggers and mental and physical responses to alcohol. As such, treating it can be a bit of a moving target. While there have been effective and promising treatments developed in recent years, it’s still a growing public health crisis that requires more research to tackle.

A new paper published Monday in the journal Nature Medicine sheds light on a surprising new treatment for the disorder: gene therapy. The study, which involved nonhuman primates, showed that the treatment could drastically reduce alcohol use in chronic heavy drinkers.

However, the authors note that due to the complex nature of the treatment—which involves brain surgery—it would likely be reserved only for the most severe cases of alcohol use disorder.

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“This was incredibly effective,” co-senior author Kathleen Grant, a neuroscientist at Oregon Health & Science University, said in a statement.

The brains of animals that chronically binge drink typically struggle to produce dopamine, the feel-good reward chemical produced by the body. In healthy bodies, even low doses of alcohol releases dopamine. However, those with alcohol use disorder require more and more alcohol in order to feel the same effects.

The new gene therapy treatment involves injecting a harmless virus into the brain to target this problem. The virus contains a gene that contains a protein called glial-derived neurotrophic factor (GDNF) that effectively resets the brain’s dopamine pathway in primates with alcohol use disorder. This allows the brain to produce dopamine in a more healthy and natural way.

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“This gene-therapy approach targets changes in dopamine function in the brain’s mesolimbic reward pathway that are caused by chronic alcohol use,” co-author Krystof Bankiewicz, and professor of neurosurgery at Ohio State University, said in a statement. “Our findings suggest that this treatment can prevent relapse without requiring long-term treatment adherence by patients.”

The trials involved eight rhesus macaque monkeys that heavily drank water-diluted ethanol. Four of the monkeys were given the treatment while the other half stood as the control. The study’s authors observed that alcohol consumption dropped by more than 90 percent amongst the gene therapy group when compared to a control group.

Blood alcohol levels were also undetectable amongst the gene therapy group. Meanwhile, the control group showed elevated levels of alcohol consumption as the trial went on.

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“Drinking went down to almost zero,” Grant explained. “For months on end, these animals would choose to drink water and just avoid drinking alcohol altogether. They decreased their drinking to the point that it was so low we didn’t record a blood-alcohol level.”

The authors note that the procedure also holds a lot of promise to treat issues beyond alcohol use disorder since the dopamine pathway is heavily involved in other forms of addiction as well.

It’s still not clear when the treatment will move to human trials. However, between psychedelics like magic mushrooms, hormone therapies, and now gene therapy, the world is becoming better armed to fight against alcohol use disorder and addictions in general—and that’s something we can all toast to (with non-alcoholic drinks of course).

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