Scientists from Kyoto University and Hyogo College of Medicine have found that cholesterol-fighting statins might help treat people with rare bone and cartilage diseases via iPS cells made from their own bodies.
Their research, published Wednesday in the online edition of the British science journal Nature, could help people with skeletal dysplasia, a group of rare diseases that affect skeletal growth through abnormalities in bone and cartilage, particularly types known as thanatophoric dysplasia and achondroplasia.
The study is remarkable in the sense that, by using iPS cells to re-create abnormal body cells, researchers have found the possibility that an existing drug could be used to treat another disease.
The diseases, caused by genetic mutations, can cause respiratory problems among infants and lead to death, while others suffer from dwarfism and other complications throughout their lives.
There are no treatments are available.
According to the study, the researchers created induced pluripotent stem (iPS) cells from six patients' skin cells. The scientists tried to turn the stem cells into cartilage, but confirmed that they could not make normal cartilage cells.
In experiments using mice, however, the scientists found that adding statins, widely used to treat people with high cholesterol levels, led to the formation of normal cartilage cells, and that transgenic mice given statins grew in height and weight.
Kyoto University professor Noriyuki Tsumaki, who was involved in the research, said his team will examine the safety and efficacy of using statins further for these diseases and that he wants to start a clinical study within two years.
But he remained cautious, saying: "Simply taking statins will not work and will be dangerous because doing so would lower the levels of cholesterol, which is essential for the growth and development (of children.) We urge that patients not take statins on their own."
