Japanese researchers have won permission to embark on a pioneering clinical study of a stem cell therapy for age-related macular degeneration, a major cause of vision loss in aging patients. The planned trial is being hailed as the first human test of a stem cell therapy made from patients' own cells.
Japan provides an appropriate proving ground for induced pluripotent stem (iPS) cell therapies. Last year professor Shinya Yamanaka of Kyoto University won a piece of a Nobel Prize for his work on converting adult cells into stem cells. The stem cells have broad potential in medicine because they can evolve into a variety to cell types with the potential to repair diseased or damaged organs, and they skirt the political quagmire of harvesting stem cells from human embryos.
As AFP reported, researchers in the Japanese study plan to take skin cells from AMD patients, coax the cells into a stem-like state, and then reintroduce them to the patients. The study calls for testing the therapeutic approach in 6 patients, with the trial set to begin next year in Kobe. Applications to conduct the trial came from the Riken Center for Developmental Biology and the Institute of Biomedical Research and Innovation.
Stem cells offer a whole new way to treat vision problems. Today, AMD patients take injected therapies such as Eylea and Lucentis to combat the disease by inhibiting VEGF to stymie the formation of leaky blood vessels, which lead to deterioration of the eye tissue that is key to central vision.
However, with pioneering clinical trials comes great risk. As professor Chris Mason of University College London noted to the BBC, researchers lack evidence on the safety of iPS cells in humans. The hope is that using patients' own cells to generate the therapies could reduce the risk of their bodies rejecting the stem cells.