TOKYO – Japanese researchers are set to begin the first clinical trials of a drug developed from induced pluripotent stem cells (iPS), making it the world’s first clinical trial, Kyoto University said on Tuesday.
The trials of the drug, developed to treat an extremely rare and genetic bone disease, are set to begin in September on a group of 20 patients to test its effectiveness and safety after receiving a go-ahead by the review committee at Kyoto University Hospital, according to a statement by the university.
The drug is based on an agent capable of inhibiting the immune system called Rapamycin, found by Japanese researchers, thanks to the development of iPS cells that simulated progressive ossifying fibrodysplasia (FOP) symptoms, and were taken from patients suffering from this disease.
The condition, with only 1,200 cases worldwide and no known treatment so far, causes muscle tissue to be gradually replaced by bone, inhibiting movement of patients’ joints or even breathing.
The team has already tested the Rapamycin’s effectiveness in experiments with mice transplanting FOP patients’ iPS stem cells into them and found out that the drug inhibited abnormal bone formation.
This agent is already used in treatment of other diseases, prompting the Japanese researchers to believe in its positive results in humans, Junya Toguchida, professor at the Kyoto University and head of the team, said during a press conference, according to Japanese news agency Kyodo.
Shinya Yamanaka, a professor at the same center and considered the father of iPS cells, said he hoped that the clinical trials encourage the development of other drugs and help with the discovery of new treatments for rare diseases.
Yamanaka won the 2012 Nobel Prize in Medicine thanks to his discovery of a method to create iPS – which has the ability to transform into any type of cell – by reprogramming mature cells.