iPS cells are artificially created pluripotent stem cells that can differentiate into cells of various tissues and organs by introducing specific genes into somatic cells. Like ES cells, they are expected to be useful in regenerative medicine and drug discovery research.
In 2006, Professor Shinya Yamanaka and his colleagues at Kyoto University succeeded for the first time in the world in producing iPS cells from mouse skin cells. Later, in 2007, they also succeeded in generating iPS cells from human skin cells. This was an epoch-making discovery that somatic cells were reprogrammed to a pluripotent state, and Professor Yamanaka was awarded the Nobel Prize in Physiology or Medicine in 2012.
One potential application of iPS cells is in the field of regenerative medicine, where they can be applied to cell transplantation therapy, in which tissue and organ cells produced by inducing differentiation from iPS cells are transplanted. In fact, clinical trials are underway to confirm the safety and efficacy of transplanting differentiated cells produced from iPS cells into patients with Parkinson’s disease (Note 5), age-related macular degeneration (Note 6) and corneal disease (Note 7). iPS cells can be produced from the patient’s own cells. The major advantage of using iPS cells is that they can be produced from the patient’s own cells, and transplantation of differentiated cells derived from the patient’s own (autologous) iPS cells is unlikely to cause rejection. Regenerative medicine using iPS cells is still in the research phase, although some clinical trials have already been completed, and there are no approved treatments available until now. Practical application is hoped for as soon as possible.
Another important application is “iPS drug discovery,” in which iPS cells are created from the somatic cells of patients with intractable diseases, and diseased cells such as neurons and cardiomyocytes are produced from these patient-derived iPS cells (disease-specific iPS cells), thereby elucidating the causes of diseases and developing new drugs. Until now, clinical trials have been conducted to confirm the safety and efficacy of new drug candidates discovered through the use of disease-specific iPS cells in patients with intractable diseases such as progressive ossifying fibrodysplasia (Note 8) and amyotrophic lateral sclerosis (ALS) (Note 9). For diseases other than these, the use of disease-specific iPS cells to reproduce cells with abnormalities could dramatically advance research to investigate disease mechanisms and find new drug candidates.
(Note 5)
Press Release from Center for iPS Cell Research and Application, Kyoto University (July 30, 2018)
