Jenkinson Memorial Lecture: Recent progress in induced pluripotent stem cell research and application

Professor Shinya Yamanaka
CiRA & Kyoto University; Nobel Laureate 2012

Professor Shinya Yamanaka is most recognized for his discovery of induced pluripotent stem (iPS) cells, which are differentiated cells that have been reprogrammed to the pluripotent state. He is Director of the Center for iPS Cell Research and Application (CiRA), Kyoto University, and Senior Investigator at the Gladstone Institutes.

Since his breakthrough finding, he has been the recipient of many prestigious awards, including the Nobel Prize in Physiology or Medicine (2012). Professor Yamanaka’s primary vision is to bring iPS cell technology to human health care.

Therefore, at CiRA, he has recruited a mixture of scientists conducting basic research and clinicians applying this research to disparate diseases. He also advocates iPS cell science regularly to the general public.

 

Abstract

Induced pluripotent stem cells (iPSCs) can proliferate almost indefinitely and differentiate into multiple lineages, giving them wide medical application. As a result, they are being used for new cell-based therapies, disease models and drug development around the world.

In 2014, the world’s first clinical study using iPSC-derived RPE (retinal pigment epithelium) sheets began for the treatment of age-related macular degeneration (AMD). iPSCs can be used for regenerative medicine to restore organ function. To push these efforts, we are proceeding with an iPSC stock project in which clinical-grade iPSC clones are being established from “super” donors with homologous HLA haplotypes. Homologous HLA haplotypes are associated with decreased immune response and therefore less risk of transplant rejection. In 2015, we started distributing an iPSC stock clone to organizations in Japan. The aim of the stock is to hold iPS cells of guaranteed quality which can be supplied quickly to medical care institutions and research institutions in Japan and overseas when required. In 2017, clinical study using the iPSC stock began for AMD patients, and the transplantation of 5 cases have completed. Additionally, clinical trial for Parkinson’s disease has started using the iPSC stock-originated neurons in this August, and the surgery to transplant dopaminergic progenitors into the patient’s brain has been just conducted at Kyoto University Hospital. 

Other applications of iPSCs include drug screening, toxicity studies and the elucidation of disease mechanisms using disease-specific iPSCs from patients with intractable diseases. In addition, iPSCs may be resourceful for preventative measures, as they make it possible to predict the patient condition and provide a preemptive therapeutic approach to protect against the onset of the disease or to establish personalized medicine. We reported a new drug screening system using iPSCs derived from fibrodysplasia ossificans progressiva (FOP) patients, revealing one drug candidate, Rapamycin. Based on these findings, we have achieved to initiate a clinical trial to treat FOP patients in 2017. This clinical trial is the world's first for a drug that is based on results from an iPS cell-based model.

Over the past decade iPSCs research made a great progress. However, there are still various hurdles to be overcome, iPSC-based science is certainly moving forward for delivering innovative therapeutic options to the patients with intractable diseases.

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This seminar will take place in the Oxford University Museum of Natural History. Following this lecture you are welcome to join Professor Yamanaka for drinks beneath the dinosaurs

Prof yamanaka