This year was a major turning point for me with the award of the Nobel Prize in Physiology or Medicine. The receipt of the award brought home to me the strong reputation enjoyed by iPS cell technology and the great expectations it inspires. At the same time, it reminded me that iPS cells are the product of long years of research into nuclear reprogramming, for which I would like to express my admiration and respect to my joint Nobel Prize recipient, Dr. Sir John Gurdon, and the many other scientists who have preceded me in this research field. I would also like to express my heartfelt gratitude for the many gestures of support, including public research grants and donations from the public to the iPS Cell Research Fund.
In 2010, when the Center for iPS Cell Research and Application was established, I set its four goals to fulfill over the first 10-year period. These goals reflect our commitment to steadily rolling out iPS cell technology and presenting the results of our research internationally.
Goals for our First Ten Years
1) Establish basic iPS cell technology and secure the associated intellectual property rights
2) Build a stock of iPS cells for use in regenerative medicine
3) Carry out preclinical studies and work toward clinical studies
4) Contribute to the development fo therapeutic drugs using patient-derived iPS cells
In fiscal 2012, our third year, the first goal of establishing basic iPS cell technology came close to complete realization, while in the securing of intellectual property, which is essential to allow researchers to engage with confidence in iPS cell research, we were able to obtain new basic technology patents in Japan and the United States.
For our second goal, which envisages the creation of an iPS cell stock with an advance guarantee of quality for use in regenerative medicine, our aim within five years is to build a cell stock that covers 30 to 50 percent of the Japanese population. As part of our work toward reaching this goal, we discussed regulatory issues with the Ministry of Labour, Health and Welfare and the Pharmaceutical and Medical Devices Agency (PMDA) and build a cooperative relationship with the Japanese Red Cross Society for recruitment of HLA-homozygous donors. We are also engaged in discussions on joint research with associations involved in umbilical cord blood bank operations.
Regarding the third goal, we have now carried out the laboratory animal experiments which represent the last stage before starting clinical research into Parkinson's disease. For the near future, after submitting an iPS cell-based therapy research plan to an institutional review board at Kyoto University for inspection, we will aim to present a clinical research protocol for approval to the Ministry of Labour, Health and Welfare in three years from now at the earliest. We have also succeeded in developing a highly efficient method of differentiating human intermediate mesoderm from iPS cells and reproducing the three-dimensional structure of renal tubules, thus taking the first step toward the regeneration of the kidney.
Coming to the fourth goal, some research groups at CiRA have published papers on studies that showed the successful creation of disease models of chronic infantile neurological cutaneous and articular (CINCA) syndrome, amyotrophic lateral sclerosis (ALS), and Alzheimer's disease, and published relevant reports. In the case of ALS, the research group has discovered drug candidate substance.
In the coming fiscal year, we are committed to continuing with steady and focused work to bring us little toward realizing our 10-year goals. We ask for your continued understanding and support.
Director, Center for iPS Cell Research and Application
© Nobel Foundation 2012, Ulla Montan