Professor Junya Toguchida has found a candidate drug for treating fibrodysplasia ossificans progressiva, or FOP -- a condition in which bone can grow in muscle tissue and elsewhere -- using induced pluripotent stem cells, the university's Center for iPS Cell Research and Application said Tuesday. Clinical trials are to start in September at the earliest. There is no effective remedy yet for the rare condition, from which around 80 Japanese are said to suffer.
A Kyoto University Hospital review committee has given the go-ahead for the trial, and the university has given notice to the Pharmaceuticals and Medical Devices Agency. Once all the necessary approvals are granted, safety and effectiveness trials can commence on a total of 20 test patients, aged 6 to 59. Plans call for the trials to be conducted at the University of Tokyo, Nagoya University and Kyushu University as well.
An iPS cell can be turned into any type of cell in the body. Shinya Yamanaka, director of Kyoto University's iPS cell research body, first induced them from mice in 2006, and from humans the following year. Injecting genes into, for instance, skin or blood cells rewinds them to a state akin to before they were differentiated into particular types, forming a sort of blank slate for creating human organ or tissue cells.
Regenerative medicine and drug discovery are the two main areas of application for these cells. There have already been a number of advances in tests of regenerative medicine, which can help restore bodies afflicted by illness or injury. "I hope drug discovery research will become active, leading to the development of new treatment methods for a range of conditions," Yamanaka said Tuesday.
In the FOP case, the research team made use of the special characteristics of iPS cells to accurately reproduce the diseased state in cells derived from patients. They tested around 7,000 potential treatments, efficiently narrowing them down to find one that checked the illness's progression.
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