In April 2013, in order to tackle issues of bioethics, we set up the Uehiro Research Division for iPS Cell Ethics with the assistance of a generous donation from The Uehiro Foundation on Ethics and Education.
Since it opened in 2010, CiRA has been eagerly engaged in areas from fundamental research to research into clinical application with the objective of attaining the four goals set out below by the year 2020.
Goals for 2020:
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 of therapeutic drugs using patient-derived iPS cells
In fiscal 2013, the fourth year of its operation, our research center achieved steady progress toward all four of these goals. Toward the first goal, we established an evaluation method for iPS cells by discovering marker genes that will allow us to separate out low-quality iPS cells. We also established a method for iPS cell generation and maintenance culture that will allow us to generate cells efficiently without the use of animal-derived constituents. These advances represent a major contribution to our second goal of building a stock of iPS cells for medical use. In fiscal 2013, we also started work on the generation of iPS cells for the cell stock.
Regarding our third goal, we are now close to completing the establishment of a protocol on which to base clinical research into transplantation therapy for Parkinsonís disease using dopamine-producing neurons induced from iPS cells. We are also engaged in the development of methods for large-scale production of iPS cell-derived platelets and red blood cells. In both cases, we envisage advancing to the clinical research stage in the near future.
Coming to our fourth goal, we successfully replicated the pathological conditions of muscular dystrophy using muscle cells induced from iPS cells generated from patients with Miyoshi muscular dystrophy, thus creating a platform for drug discovery. In addition, we were able to report success in the partial replication of the pathological conditions of fibrodysplasia ossificans progressiva (FOP) using iPS cells generated from patients, taking us one step further toward screening for therapeutic drugs. We are also progressing with research into the possibility of using iPS cells derived from Alzheimerís disease patients to predict the development of the disease and take appropriate therapeutic action. We have thus begun to progress with a wide-range of therapeutic drug development based on iPS cells.
In fiscal 2014, we aim to reinforce initiatives oriented toward clinical research and to further enhance research support systems. We ask for your continued support and guidance.
Director, Center for iPS Cell Research and Application