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Drug prescribed to millions over decades revealed to have unexpected impact on the brain

Home> Science> News

Published 11:03 27 Mar 2026 GMT

Drug prescribed to millions over decades revealed to have unexpected impact on the brain

It is prescribed to over 120 million people worldwide

Harry Boulton

Harry Boulton

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Featured Image Credit: Scott Olson/Getty Images
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One of the most popular forms of medication used by millions across the world has been revealed to have an unexpected impact on the brain of those that take it, marking a significant discovery that could spark further research.

While there are countless new drugs that become available each year, one of the biggest question marks for many health experts is the long-term effects of these medications as it's impossible to properly analyze that within the testing period.

It's why there remains a lot of skepticism surrounding weight loss drugs like Ozempic, as while they're largely fine for people to take in the short term, we know little about how it'll affect people's bodies over time.

One incredibly popular drug that has been used by millions for decades has only just been subject to a major new discovery, as scientists have revealed exactly how it impacts and interacts with our brain to work its magic.

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As reported by ScienceAlert, a new study published by researchers at Baylor College of Medicine has identified a brain pathway that popular diabetes medication Metformin works through, illuminating why it has proven to be so effective across the years.

Scientists have made a major new discovery surrounding metformin — a drug millions take every year (Scott Olson/Getty Images)
Scientists have made a major new discovery surrounding metformin — a drug millions take every year (Scott Olson/Getty Images)

Metformin primarily is prescribed to individuals either suffering from type 2 diabetes or at risk of developing it, and it is even given to people with polycystic ovary syndrome (PCOS) to improve fertility.

It works by lowering blood sugar levels by decreasing the amount of glucose produced by the liver, but it is only now that scientists have been able to understand where that reaction occurs, looking towards the brain for answers.

"Scientists at Baylor College of Medicine and international collaborators have discovered a previously unrecognized new player mediating clinically relevant effects of metformin: the brain," the university outlines in a statement.

"By uncovering a brain pathway involved in metformin's anti-diabetic action, researchers have discovered new possibilities for treating diabetes more effectively and precisely."

Metformin interacts with Rap1 within the brain in order to lower blood sugar levels at a far higher sensitivity level than the rest of the body (Scott Olson/Getty Images)
Metformin interacts with Rap1 within the brain in order to lower blood sugar levels at a far higher sensitivity level than the rest of the body (Scott Olson/Getty Images)

This research focuses specifically on a small protein known as 'Rap1', which is found within the brain's ventromedial hypothalamus (VMH), as metformin effectively 'turns off' this protein in order to lower blood sugar levels throughout the body.

Rap1 is still needed in order for metformin to do its job, as was discovered through clinical tests on mice lacking the protein, and Dr. Makoto Fukuda – one of the study's authors – has revealed how this makes an impact on the future of the drug.

"This discovery changes how we think about metformin. It's not just working in the liver or the gut, it's also acting in the brain," Fukuda illustrates. "We found that while the liver and intestines need high concentrations of the drug to respond, the brain reacts to much lower levels.

"These findings open the door to developing new diabetes treatments that directly target this pathway in the brain," the researcher continues, suggesting that they could become far more efficient in the near future.

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