Kyoto University researchers are set to begin the world's first clinical trial of a drug identified using iPS cells to treat a rare bone disease, the university said Tuesday.
A team led by Junya Toguchida, a professor at the university, used induced pluripotent stem cells to develop a potential drug for fibrodysplasia ossificans progressiva (FOP), a rare disorder in which muscle tissue is gradually replaced by bone, inhibiting body movement.
The researchers created iPS cells from FOP patients and replicated the symptom outside their bodies. After adding components to the cells with features of the disease, they found that — out of 6,800 substances — an immune-suppressive agent called Rapamycin was effective for preventing abnormal bone formation.