Experimental Drug Cures Osteoporosis In Mice

A team of international researchers led by scientists from the Columbia University Medical Center have reported that an experimental drug that inhibits the function of serotonin in the gut has been effective in curing osteoporosis in rats. This discovery is reported on the Feb 7 issue of Nature Medicine. The findings may someday be used to find better therapies to inhibit serotonin production as a means to treat osteoporosis.

Osteoporosis is a bone disease characterized by a reduction in bone mineral density and mass that leads to an increased risk of breaks or fractures. It is a common disease that affects women especially after menopause, which number in the tens of millions worldwide.

The study using the experimental drug known as LP533401 was preceded by a study by the same team of researchers headed by Dr. Gerard Karsenty, MD, PhD. and chair of the Department of Genetics and Development at Columbia University College of Physicians and Surgeons that discovered serotonin being released in the gut may be inhibiting bone formation. By regulating the production of serotonin within the gut, bone formation may also be affected. Prior to this, serotonin, which around 95 percent is found in the gut, was only known primarily as a neurotransmitter in the brain.

Armed with this knowledge, the researchers also found out about the investigational drug LP533401 acts as an effective inhibitor of serotonin in the gut. When we learned of this compound, we thought that it was important to test it as proof of principle that there could be novel ways to treat osteoporosis with therapies that can be taken orally and regulate the formation of serotonin, said Dr. Karsenty.

Based on the findings, inhibiting the production of serotonin has enabled the team to cure osteoporosis in mice that have gone through menopause. The experimental compound was administered orally at a small dose daily for up to 6 weeks. This prevented post-menopausal osteoporosis in the tested rodents from developing. It cured the disease in those rats already with osteoporosis. The serotonin production using the experimental drug was only inhibited in the gut and did not affect the serotonin found in the brain. This is of the utmost importance since it provided evidence that the use of the drug did not cross the blood-brain barrier.

Source: http://cumc.columbia.edu/news/press_releases/Inhibiting_Serotonin_Osteoporosis.html

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