World's 1st trial of drug developed from iPS cells to begin

Japan Economic Newswire |

Japanese researchers are set to begin the world's first clinical trial of a drug developed from iPS stem cells to treat a rare bone disease, Kyoto University said Tuesday.

A team led by Junya Toguchida, professor at the university, used so-called induced pluripotent stem cells to develop a potential drug for fibrodysplasia ossificans progressiva, or 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 an immune-suppressive agent called Rapamycin is 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 stem cells into them and found out that the drug inhibited abnormal born formation.

"Rapamycin is a drug already used (for treatment of other diseases) so I expect patients will welcome" its use in the clinical trial, Toguchida said at a press conference.

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."

The disease is caused by a mutation of a gene called ACVR1. Bones are formed in muscles, tendons and ligaments, hindering the movements of joints. Patients may experience difficulty in breathing if their respiratory muscles are affected.

iPS cells can grow into any type of human body tissue. They are expected to be utilized for drug development as well as regenerative medicine.

==Kyodo

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