Scientists at Kyoto University should start the first clinical trial in the autumn, testing out a medicinal product identified using induced pluripotent stem cells (iPS) to treat a rare bone disorder.
The team, led by Junya Toguchida, used iPS cells to model the target disease, namely progressive fibrodysplasia ossificans progressiva. To do this, they harvested and then reprogrammed patients’ cells. They then tested 6,800 substances on these cells and selected rapamycin which proved effective in vitro in preventing abnormal bone formation. Laboratory animal tests proved conclusive. The safety and efficacy of the medicinal product must now be tested in a clinical trial, which could start in September and include 20 patients. Initial authorisations have been granted. Since rapamycin is already used to treat other disorders, Junya Togushida is confident about the outcome of this first trial.
Shinya Yamanaka, a Professor at Kyoto University and the 2012 Nobel Prize Winner for Medicine, following the discovery of iPS cells, commented as follows: “I hope that this clinical trial will stimulate active research for the development of medicinal products and will initiate the discovery of new treatments for various rare diseases”.