Unlocking the Potential of Regenerative Medicine

The future will almost certainly bring new and unexpected challenges. However, as we continue to add outstanding researchers and staff along with increasing support from the public and private sectors, I am determined to succeed in our mission, with the ultimate goal being to use iPS cell technology for patient care.
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The Center for iPS Cell Research and Application (CiRA) at Kyoto University was established in April 2010 to advance iPS (induced pluripotent stem) cell technology for clinical application. Currently, we have about 30 research teams conducting basic research on iPS cells as well as investigating their use for new medical treatments. CiRA also has several members devoted to studying the ethical, legal and social issues related to iPS cells. In total, CiRA has 320 members including research support staff.

iPS cells are stem cells that my research team first developed by introducing a small number of transcription factors into human somatic cells. Because they have the ability to multiply infinitely and to differentiate into any cell type in the body, iPS cells have tremendous potential for health care. Medical applications using iPS cells can be mainly divided into two fields. The first field is regenerative medicine, where iPS cells are differentiated into specific cell types that are transplanted into the patient for curative purposes. The second field is disease modeling, where patient-specific iPS cells are differentiated into cells of the diseased tissue. Using these cells, researchers can study the mechanism of the disease progression for the development of new drugs.

When CiRA was founded, we presented four goals to achieve by 2020, as shown below.

1) Establish basic iPS cell technology and secure the associated intellectual property rights
2) Build an iPS cell stock for use in regenerative medicine
3) Carry out preclinical studies and clinical research
4) Contribute to the development of therapeutic drugs using patient-derived iPS cells

The first goal was to establish safe iPS cells for patient care. Not all iPS cells made in the lab are suitable for the clinic. We have therefore worked hard at preparing iPS cells that can be used in patients. With these advances have come patents on iPS cell technologies in over 30 countries. These advances have also been the basis of our second goal, the generation of clinical-grade iPS cells, which we commenced in 2013. Currently, we are working with other researchers who are evaluating the quality of these cells. Assuming quality is confirmed, we anticipate the distribution of our iPS cells by the end of this year for use in regenerative medicine.

There is already much excitement about goal three. Last September, a team led by a Riken Center for Developmental Biology researcher, with whom CiRA is collaborating, prepared retinal pigment epithelial cells (RPE cells) from iPS cells that were made using skin cells from a patient with age-related macular degeneration. These RPE cells were then transplanted back to the patient. This study is the world's first clinical research using iPS cells. In the next few years, CiRA plans additional clinical research on Parkinson's disease and blood diseases.

Finally, regarding our fourth goal, the development of new drugs, researchers at CiRA have confirmed through animal research drug repositioning to treat achondroplasia. Currently, we are investigating the possibility of this repositioning in humans and progressing towards clinical research here as well.

At the time we announced our 2020 goals, iPS cell technology was less than 5 years old. Thanks to the hard work by CiRA faculty members and many others, the progress of iPS cell research has been quite remarkable.

The Japanese government has provided a great deal of support for regenerative medicine. However, to further our goal of drug discovery we formed a comprehensive partnership last April with Takeda Pharmaceutical Company Limited. Over the next 10 years, they will contribute 20 billion yen ($166 million as of May 2015) in research funding and work with us on research projects that focus on various diseases, including heart failure, diabetes, and neurological disorders.

In Japan, the role of venture businesses, which serve to build bridges between universities and large companies, continues to be weak. In order to avoid the so-called "valley of death," we chose to work directly with a large company. This unique program, in which CiRA researchers work within Takeda Pharmaceutical's Shonan Research Center, is a new model of cooperation between industry and academia.

Because this partnership is a new model, there are many unknowns. Nevertheless, we are excited about building new ways in which industry and academia can work together overcoming new hurdles. We are proactively seeking similar partnerships with other companies, both foreign and domestic.

New Goals for 2030
Seeing how we have progressed on our 2020 aims, last April CiRA announced four new long-term goals for 2030.
1) Promote iPS cell stock and regenerative medicine
2) Develop drugs for rare diseases and realize personalized medicine using patient iPS cells
3) Create new frontiers in life and medical sciences with iPS cell technology
4) Build Japan's best support team and environment for excellent research and development

Our first of these new goals is to bring iPS cell-based regenerative medicine to the bed by utilizing the iPS cell stock we initiated in 2013. We would like to create a system in which iPS cells can be quickly used in cell transplants to treat diseases and injuries such as spinal cord injury. For our second goal, we seek to realize personalized medicine using patient-specific iPS cells. For example, these cells can be used to study the effectiveness of a drug for the patient. We will also continue to work on development of medicines for rare diseases. As for goal three, we would like to use iPS cells as a tool to understand the mechanisms of development and diseases such as cancer. I expect this research to cultivate new fields in the life sciences.

In order to achieve the above-mentioned three goals, our fourth goal, the establishment of exceptional research support and research environment, is indispensable. In order to carry out clinical applications, staff with expertise in cell cultivation, safety evaluation, intellectual property, collaboration agreements, medical regulations, and public communications will become more and more important.

Because of the importance of all staff for CiRA's success, it might be surprising to learn that around 90% of CiRA staff is hired on limited term contracts that expire in a few years. While CiRA receives a large amount of research grants from the government as well as funds through collaboration with businesses, conditions of these funds prevent their use for retaining support staff. Therefore, raising funds to hire staff long term is one of the major issues I have been working on as director of CiRA.

Such funding is also to be used to train students and young researchers, settle patent disputes, and support us on other matters that current funding cannot. For these reasons, we have established the iPS Cell Research Fund. As an example of fundraising, I run full marathons across Japan. The establishment of a stable research environment and stable funding for hiring are problems that many universities grapple with. Through our iPS Cell Research Fund, we aim to promote a culture in Japan in which ordinary citizens can demonstrate their support to research institutes directly.

The future will almost certainly bring new and unexpected challenges. However, as we continue to add outstanding researchers and staff along with increasing support from the public and private sectors, I am determined to succeed in our mission, with the ultimate goal being to use iPS cell technology for patient care.

This post was originally published on HuffPost Japan and was translated into English.

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