Researchers probe extreme gene therapy for severe alcoholism

Researchers probe extreme gene therapy for severe alcoholism

When all traditional therapeutic approaches fail for alcohol abuse disorder, a radical gene therapy might be something to try in the future.

Adobe Stock

Story byFreethink

A single shot — a gene therapy injected into the brain — dramatically reduced alcohol consumption in monkeys that previously drank heavily. If the therapy is safe and effective in people, it might one day be a permanent treatment for alcoholism for people with no other options.

The challenge: Alcohol use disorder (AUD) means a person has trouble controlling their alcohol consumption, even when it is negatively affecting their life, job, or health.


In the U.S., more than 10 percent of people over the age of 12 are estimated to have AUD, and while medications, counseling, or sheer willpower can help some stop drinking, staying sober can be a huge struggle — an estimated 40-60 percent of people relapse at least once.

A team of U.S. researchers suspected that an in-development gene therapy for Parkinson’s disease might work as a dopamine-replenishing treatment for alcoholism, too.

According to the CDC, more than 140,000 Americans are dying each year from alcohol-related causes, and the rate of deaths has been rising for years, especially during the pandemic.

The idea: For occasional drinkers, alcohol causes the brain to release more dopamine, a chemical that makes you feel good. Chronic alcohol use, however, causes the brain to produce, and process, less dopamine, and this persistent dopamine deficit has been linked to alcohol relapse.

There is currently no way to reverse the changes in the brain brought about by AUD, but a team of U.S. researchers suspected that an in-development gene therapy for Parkinson’s disease might work as a dopamine-replenishing treatment for alcoholism, too.

To find out, they tested it in heavy-drinking monkeys — and the animals’ alcohol consumption dropped by 90% over the course of a year.

How it works: The treatment centers on the protein GDNF (“glial cell line-derived neurotrophic factor”), which supports the survival of certain neurons, including ones linked to dopamine.

For the new study, a harmless virus was used to deliver the gene that codes for GDNF into the brains of four monkeys that, when they had the option, drank heavily — the amount of ethanol-infused water they consumed would be equivalent to a person having nine drinks per day.

“We targeted the cell bodies that produce dopamine with this gene to increase dopamine synthesis, thereby replenishing or restoring what chronic drinking has taken away,” said co-lead researcher Kathleen Grant.

To serve as controls, another four heavy-drinking monkeys underwent the same procedure, but with a saline solution delivered instead of the gene therapy.

The results: All of the monkeys had their access to alcohol removed for two months following the surgery. When it was then reintroduced for four weeks, the heavy drinkers consumed 50 percent less compared to the control group.

When the researchers examined the monkeys’ brains at the end of the study, they were able to confirm that dopamine levels had been replenished in the treated animals, but remained low in the controls.

The researchers then took the alcohol away for another four weeks, before giving it back for four. They repeated this cycle for a year, and by the end of it, the treated monkeys’ consumption had fallen by more than 90 percent compared to the controls.

“Drinking went down to almost zero,” said Grant. “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.”

When the researchers examined the monkeys’ brains at the end of the study, they were able to confirm that dopamine levels had been replenished in the treated animals, but remained low in the controls.

Looking ahead: Dopamine is involved in a lot more than addiction, so more research is needed to not only see if the results translate to people but whether the gene therapy leads to any unwanted changes to mood or behavior.

Because the therapy requires invasive brain surgery and is likely irreversible, it’s unlikely to ever become a common treatment for alcoholism — but it could one day be the only thing standing between people with severe AUD and death.

“[The treatment] would be most appropriate for people who have already shown that all our normal therapeutic approaches do not work for them,” said Grant. “They are likely to create severe harm or kill themselves or others due to their drinking.”

This article originally appeared on Freethink, home of the brightest minds and biggest ideas of all time.

Sign up for Big Think’s newsletter

Kristin Houser
Kristin Houser is a staff writer at Freethink, where she covers science and tech. Her written work has appeared in Business Insider, NBC News, and the World Economic Forum’s Agenda, among other publications, and Stephen Colbert once talked about a piece on The Late Show, to her delight. Formerly, Kristin was a staff writer for Futurism and wrote several animated and live action web series.
The flu shot looks different this year, thanks to the pandemic.

Thanks to safety cautions from the COVID-19 pandemic, a strain of influenza has been completely eliminated.

Getty Images

If you were one of the millions who masked up, washed your hands thoroughly and socially distanced, pat yourself on the back—you may have helped change the course of human history.

Scientists say that thanks to these safety precautions, which were introduced in early 2020 as a way to stop transmission of the novel COVID-19 virus, a strain of influenza has been completely eliminated. This marks the first time in human history that a virus has been wiped out through non-pharmaceutical interventions, such as vaccines.

Keep ReadingKeep Reading
Sarah Watts

Sarah Watts is a health and science writer based in Chicago.

After his grandmother’s dementia diagnosis, one man invented a snack to keep her healthy and hydrated.

Founder Lewis Hornby and his grandmother Pat, sampling Jelly Drops—an edible gummy containing water and life-saving electrolytes.

jellydrops.us

On a visit to his grandmother’s nursing home in 2016, college student Lewis Hornby made a shocking discovery: Dehydration is a common (and dangerous) problem among seniors—especially those that are diagnosed with dementia.

Hornby’s grandmother, Pat, had always had difficulty keeping up her water intake as she got older, a common issue with seniors. As we age, our body composition changes, and we naturally hold less water than younger adults or children, so it’s easier to become dehydrated quickly if those fluids aren’t replenished. What’s more, our thirst signals diminish naturally as we age as well—meaning our body is not as good as it once was in letting us know that we need to rehydrate. This often creates a perfect storm that commonly leads to dehydration. In Pat’s case, her dehydration was so severe she nearly died.

Keep ReadingKeep Reading
Sarah Watts

Sarah Watts is a health and science writer based in Chicago.