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Genetic Condition to Drug Inspiration

Some researchers and drug companies are looking to people with essential fructosuria as inspiration for anti-obesity treatments, Bloomberg reports.

People with this rare genetic condition are unable to metabolize fructose, have an aversion to sweets, and appear to be at a decreased risk of developing obesity, type 2 diabetes, and some liver conditions, Bloomberg adds.

But finding people with it is difficult, the University of Denver's Richard Johnson tells Bloomberg. He first came up with the idea of affecting fructose metabolism to curb disease after he found that mice lacking the fructokinase enzyme gain less weight and had lower blood glucose and insulin levels than control mice when fed a high-fructose diet.

As Bloomberg notes, Johnson's work caught the attention of Pfizer and the pharmaceutical company is working on a drug based on the condition to treat nonalcoholic steatohepatitis (NASH), which is often preceded by fatty liver, which itself can be due to consuming too much fructose. By reducing fatty liver, the firm hopes to prevent NASH.

"What we're looking at is a medicine that prevents the development of obesity, prevents diabetes. My belief is this will have a major clinical impact," Johnson says.