It was only 10 years ago that Shinya Yamanaka, M.D., Ph.D., discovered how to convert a person’s skin cells into stem cells by tweaking just four genes. The historical breakthrough landed Dr. Yamanaka the 2012 Nobel Prize in Physiology-Medicine, because it meant that patients could be their own stem-cell donors. Known as induced pluripotent stem cells (iPSC), they are now being used to develop powerful therapies and drug-screening tools including those for the retina.
To the delight of nearly 300 retinal researchers from around the world attending the FFB-funded RD2016 meeting, September 19-24 in Kyoto, Japan, Dr. Yamanka discussed his early clinical trial for iPSC-derived retinal pigment epithelial (RPE) cells for a 78-year-old woman with advanced wet age-related macular degeneration (AMD). The study met its main goal – safety – and he and his collaborator, Masayo Takahashi, M.D., Ph.D., are planning to treat additional patients in the near future.
Therapy developers are excited about using iPSC, because: 1) They can be coaxed to become almost any cell type in the body, 2) They can be matched with the recipient’s immune system profile to minimize chances of rejection, and 3) They reduce the need to derive stem cells from blastocysts (often referred to as embryonic stem cells).
Dr. Yamanaka now heads one of the world’s largest and most innovative iPSC research centers based at Kyoto University. He and more than 500 investigators are working to use the highly promising technology to develop therapies including those for retinal diseases.
FFB-funded investigator David Gamm, M.D., Ph.D., University Wisconsin-Madison, in collaboration with stem-cell pioneer James Thomson, M.D., Ph.D., are also using iPSC to develop transplantation therapies for a variety of inherited retinal conditions, including those that replace lost photoreceptors, the cells that make vision possible, and RPE, which are a critical support system for photoreceptors. Dr. Gamm is working on a patch comprised of both photoreceptors and RPE to potentially restore vision to people with advanced retinal conditions.
“It was a major thrill to have a Nobel-prize-winning scientist speak to our community,” says Stephen Rose, Ph.D., FFB’s chief research officer. “Yamanaka is to stem cells like LeBron James is to basketball. He’s been a real game-changer, and most exciting, there is much more to come from him and his team in Kyoto, and other investigators leveraging his work.”