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Kyoto Univ. Hospital starts trial treating rare disease that turns tissue to bone

(Mainichi)

KYOTO -- Kyoto University Hospital on Oct. 5 began its first full-scale clinical trial using medication that surfaced in induced pluripotent stem (iPS) cell research to treat a rare disease that causes tissue to turn into bone.

    The trial will test a treatment for fibrodysplasia ossificans progressiva (FOP), an intractable disease that causes bones to form in the muscles and tendons. Ikumi Yamamoto, a 19-year-old patient with the condition, took part, receiving a checkup.

    The trial was launched by a team including Junya Toguchida, a professor at Kyoto University's Center for iPS Cell Research and Application (CiRA). Researchers will divide roughly 20 patients across Japan into two groups, with one receiving the medication and the other not receiving it, to test the medicine's effectiveness and safety over a six-month period. After that, all of the patients will be given the medication to record its effectiveness.

    The team created iPS cells from samples provided by Yamamoto and the other patients. They then replicated FOP in culture dishes and searched for substances that could inhibit the formation of bones. They found that the existing immunosuppressant drug rapamycin was most effective in preventing the spread of bones.

    Clinical trials will also be conducted at the University of Tokyo, Nagoya University, and Kyushu University hospitals.

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