Using patents to ensure access to pioneering cell technology

• Author: Shinya Yamanaka
• Position: M.D., Ph.D., Director of the Center for iPS Cell Research and Application, Kyoto University, Japan

The technology

My research focuses on pluripotent stem cells, which are cells capable of differentiating into any cell type within the adult body – nerve cells, muscle cells, lung cells, and so on.

Essentially, my colleagues and I managed to take mature cells and reprogram them into pluripotent cells called induced pluripotent stem cells (iPS cells). We first reported iPS cells from mouse skin cells in 2006 and from human skin cells in 2007. Since then, we have extended our research to iPS cells for new medical treatments. Almost all our research activities are based at the Center for iPS Cell Research and Application (CiRA) at Kyoto University in Japan.

Our success in generating iPS cells from human somatic cells (i.e. any cell in the body except sperm or egg cells) was a major breakthrough. It overcame the ethical concerns surrounding the use of human embryonic stem cells for medical research, because it made it possible to access pluripotent cells without the destruction of embryos. It also created many opportunities for medical research, particularly in the areas of diagnostics, drug screening and regenerative medicine.

To generate iPS cells, the information contained in somatic cells is reprogrammed through the addition of a small number of genes known as “nuclear reprogramming factors.”

iPS cells have two great advantages: they are (a) capable of differentiating into all cell types within the adult body and (b) they proliferate without limit. As a consequence, these cells have huge potential to treat a wide range of diseases for which effective therapies are not yet available.

Potential for regenerative medicine

One exciting application for which iPS cells hold great potential is in the area of regenerative therapy, where they can be used to repair or replace tissues. In 2014, the first clinical research using iPS cell transplantation was performed on a woman suffering from age-related macular degeneration by scientists at the RIKEN Center for Developmental Biology (CBD) and the Institute of Biomedical Research and Innovation Hospital. In this therapy, retinal tissues were created from iPS cells generated from her skin cells and transplanted into her eyes. Preparations are now underway to start similar iPS cell clinical research for Parkinson’s disease and other intractable conditions.

Potential for drug discovery

A second major application of iPS cells is in the area of drug discovery. Take, for example, a patient suffering...