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Parasite drug used to combat type 2 diabetes

Victor Shengkan Jin (Credit: Nick Romanenko/Rutgers University)
The drug they used is a modified form of a medication that the FDA already approved for human use.

Researchers from Rutgers Robert Wood Johnson Medical School, New Brunswick, NJ, have revealed that a modified form of a different drug, niclosamide, may hold the key to battling the type 2 diabetes at its source. Niclosamide is currently used to eliminate intestinal parasites.

The study, led by Dr Victor Shengkan Jin, an associate professor of pharmacology at Rutgers, is published online by the journal Nature Medicine.

"Our goal in this study was to find a safe and practical way of diminishing fat content in the liver. We used mice to perform proof-of-principle experiments in our laboratory," said Jin. "We succeeded in removing fat, and that in turn improved the animals' ability to use insulin correctly and reduce blood sugar."

The modified medication (niclosamide ethanolamine salt, NEN) burned the excess fat in liver cells, through a process known as mitochondrial uncoupling. Mitochondria are the microscopic energy source for each cell in the body, and ordinarily they burn fuels including fats and sugars in modest quantities to keep the cells functioning.

"The cell is like a car and the mitochondria are the engine," he added. "What we're doing inside cells is like putting the car's transmission into neutral by uncoupling it from the transmission. Then you step on the gas so the engine runs full throttle but the car doesn't move. If too much of the fuel in the cell is fat, you keep burning it until the fuel gauge reaches empty. Without the interference of fat, you hope that sugar will then enter the cell normally."

Getting rid of the interference of fat in liver and muscle tissue is the key to restoring the cells' ability to respond to insulin properly, which would allow the right amount of sugar to be taken up by cells and ultimately reverse the diabetes entirely. That outcome is far from certain, but the researchers claim the positive changes they saw in the mice are encouraging.

Jin said that the drug they used is a modified form of a medication that the FDA already approved for human use.

"We wanted a safe and practical compound to deplete fat inside cells. We went to the literature and found an approved drug that does in parasitic worms what we wanted to do in liver cells,” he added. “The modified form of the medication, although itself is not a drug used in humans, has an excellent safety profile in other mammal,  so very likely it would have a good safety profile in humans too."

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