Kyoto University researchers are set to begin the world’s first clinical trial of a drug identified using induced pluripotent stem (iPS) cells to treat a rare bone disease. 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.
The drug’s safety and effectiveness need to be tested in a clinical trial, which could begin as early as September, on 20 patients aged 6 or older. A review committee at Kyoto University Hospital has already approved the trial.
The team has confirmed the effectiveness of Rapamycin in experiments with mice. Researchers gave the agent to mice after transplanting FOP patients’ iPS cells into them, and found that the drug inhibited abnormal bone formation.
Shinya Yamanaka, professor at Kyoto University and a 2012 Nobel Prize winner in medicine for discovering iPS cells, said, “I hope the clinical trial will spur active research for drug development, and eventually lead to the discoveries of new treatment for various rare diseases.”
Any type of human body tissue can be formed using iPS cells. They are expected to be utilized for drug development as well as regenerative medicine.