Breakthrough new pill cuts alcohol consumption by over 50%

Researchers from Oregon Health & Science University and institutions across the country have identified a pill used to treat a common skin disease as an “incredibly promising” treatment for alcohol use disorder, according to a study published in the Journal of Clinical Investigation.

The medication, called apremilast, reduced alcohol intake by more than half among the people who received it, from an average of five drinks per day to two.

“I’ve never seen anything like that before,” said co-senior author Angela Ozburn, Ph.D., associate professor of behavioral neuroscience in the OHSU School of Medicine and a research biologist with the Portland VA Health Care System.

The study began in 2015, when Ozburn and collaborators searched a genetic database looking for compounds likely to counteract the expression of genes known to be linked to heavy alcohol use. Apremilast, an FDA-approved anti-inflammatory medication used to treat psoriasis and psoriatic arthritis, appeared to be a promising candidate.

They then tested it in two unique animal models that have a genetic of risk for excessive drinking, as well as in other strains of mice at laboratories across the country. In each case, apremilast reduced drinking among a variety of models predisposed to mild to heavy alcohol use. They found that apremilast triggered an increase in activity in the nucleus accumbens, the region of the brain involved in controlling alcohol intake.

Researchers at the Scripps Research Institute in La Jolla, California, then tested apremilast in people.

The Scripps team conducted a double-blind, placebo-controlled clinical proof-of-concept study involving 51 people who were assessed over 11 days of treatment.

“Apremilast’s large effect size on reducing drinking, combined with its good tolerability in our participants, suggests it is an excellent candidate for further evaluation as a novel treatment for people with alcohol use disorder,” said co-senior author Barbara Mason, Ph.D., Pearson Family professor in the Department of Molecular Medicine at Scripps.

The clinical study involved people with alcohol use disorder who weren’t seeking any form of treatment, and Mason predicts that apremilast may be even more effective among people who are motivated to reduce their alcohol consumption.

“It’s imperative for more clinical trials to be done on people seeking treatment,” Ozburn said. “In this study, we saw that apremilast worked in mice. It worked in different labs, and it worked in people. This is incredibly promising for treatment of addiction in general.”

Alcohol use disorder is a serious public health problem in the United States, with an estimated 95,000 people dying every year from alcohol-related deaths, according to the National Institute on Alcohol Abuse and Alcoholism.

Currently, there are three medications approved for alcohol use disorder in the United States: Antabuse, which produces an acute sensitivity akin to a hangover when alcohol is consumed; acamprosate, a medication thought to stabilize chemical signaling in the brain that is associated with relapse; and naltrexone, a medication that blocks the euphoric effects of both alcohol and opioids.

Ozburn conducted the study as part of the Integrative Neuroscience Initiative on Alcoholism-Neuroimmune, or INIA-Neuroimmune, a multidisciplinary, collaborative consortium funded by the National Institute on Alcohol Abuse and Alcoholism that studies the underlying biology of alcohol use disorder.

In her role with INIA, Ozburn works collaboratively with others in both the INIA-Neuroimmune and the INIA-Stress consortia to identify and test promising drug candidates in animal models. The INIA mechanism is an example of collaborative science, and has been essential for identifying immune targeting drugs and for advancing basic science research to improve human health.

According to Ozburn, the INIA-Neuroimmune consortium brings together experts in various fields, including neuroscience, immunology, and pharmacology, to study the mechanisms underlying alcohol use disorder and develop new treatments.

"This collaboration allows us to bring together researchers with different expertise and perspectives to tackle complex problems," Ozburn said. "It's been essential for identifying immune targeting drugs and for advancing basic science research to improve human health."

The consortium has been successful in identifying several potential treatments for alcohol use disorder, including the anti-inflammatory medication apremilast. Other drugs being studied by the INIA-Neuroimmune consortium include those that target the immune system, as well as those that modulate brain circuits involved in addiction.

The success of the apremilast study has generated excitement among researchers in the field of addiction medicine. While current treatments for alcohol use disorder are effective for some individuals, there is a need for new and more effective treatments that can help a wider range of patients.

"The fact that apremilast worked in both animal models and in people is very promising," said Dr. Bankole Johnson, a neuroscientist and addiction medicine specialist at the University of Maryland School of Medicine. "It's still early days, but this could be a game-changer for the treatment of alcohol use disorder."

Dr. Johnson noted that while the study involved a relatively small number of participants, the results were encouraging and warrant further research. He also pointed out that apremilast is already an FDA-approved medication, which could expedite the development of a new treatment for alcohol use disorder.

"It's always exciting when we can repurpose an existing medication for a new indication," Dr. Johnson said. "It could mean that we're able to get a new treatment to patients much more quickly than if we had to develop a completely new medication from scratch."

Despite the promising results, there are still many questions that need to be answered before apremilast can be considered a viable treatment option for alcohol use disorder. For example, it is not yet clear how long patients would need to take the medication to see sustained reductions in alcohol consumption, or whether it would be effective for individuals with severe alcohol use disorder.

Additionally, it is important to determine whether there are any side effects associated with long-term use of apremilast. The medication is generally well-tolerated, but it can cause side effects such as nausea, diarrhea, and headaches.

Dr. Mason and Dr. Ozburn both emphasized the need for further research to determine the safety and effectiveness of apremilast as a treatment for alcohol use disorder.

"We're excited about the results of this study, but we recognize that there is still much work to be done," Dr. Mason said. "We need to continue studying this medication to determine its potential as a treatment option for individuals with alcohol use disorder."

Despite the challenges that lie ahead, researchers are hopeful that apremilast could eventually become an important tool in the fight against alcohol use disorder.

"Alcohol use disorder is a complex condition that requires a multifaceted approach to treatment," Dr. Ozburn said. "Apremilast could be one important piece of that puzzle, and we're excited to continue exploring its potential."

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