Gene Therapy Used for Parkinson’s Reduces Alcohol Consumption by 90% in Addicted Primates

In the United States, alcoholism is recognized as a significant public health concern, and, in fact, statistics diagnose approximately 29.5 million people with this disorder. The complexity of treating alcoholism doesn’t just revolve around the reduction of alcohol consumption. Instead, effective treatment of this disorder necessitates a deeper understanding, addressing both the physiological dependencies that alcohol creates and the underlying psychological and sociocultural factors.

Alcohol Abuse

Alcohol Abuse

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To meet this challenge researchers are now testing the potential of a form of gene therapy developed to treat Parkinson’s disease to treat alcoholism. The results of a trial on monkeys were very impressive!

Researchers at Oregon Health & Science University have found another use for GDNF gene therapy already used to treat Parkinson’s disease: treating alcoholism. The treatment, injected directly into the brains of ethanol-dependent rhesus monkeys, reduced their alcohol consumption by 90%. “It’s incredibly effective,” says Kathleen Grant, a neuroscience researcher who took part in the trials.

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The gene therapy in question comes in the form of an adeno-associated virus serotype 2 encoding human glial-derived neurotrophic factor (AAV2-hGDNF) that carries the genes encoding the GDNF protein, or glial-derived neurotrophic factor. As a growth factor, this protein promotes the growth of neurons that synthesize dopamine, an important neurotransmitter in the reward system. “Alcohol consumption can increase dopamine production. But if you drink chronically, the brain adapts in such a way that it reduces the release of dopamine. So when people are addicted to alcohol, they don’t really feel the pleasure of drinking,” explains Kathleen Grant.

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Gene therapy brings dopamine to the place in the brain where it is produced and allows alcohol-dependent macaque monkeys to reduce their drinking dramatically. The animals managed to stay abstinent for 12 months, while the researchers introduced periods of abstinence followed by periods of alcohol exposure. These pre-clinical results, published in Nature Medicine, need to be confirmed in humans before this gene therapy can be used to treat alcoholism.

References

Ford, M.M., George, B.E., Van Laar, V.S. et al. GDNF gene therapy for alcohol use disorder in male non-human primates. Nat Med 29, 2030–2040 (2023). https://doi.org/10.1038/s41591-023-02463-9

Oregon Health & Science University. (2023, August 14). Study shows promise of gene therapy for alcohol use disorder. EurekAlert!. https://www.eurekalert.org/news-releases/998023

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