Highlights on BioJapan 2018 – Updates on the Development of Cell Therapy and the Regenerative Medicine Ecosystem in Japan
Regenerative medicine is one of the highlights in BioJapan, this year’s regenerative medicine topic including Professor Shinya Yamanaka, the Nobel laureates on 2012, who shared the latest development in the field of iPS cell, the representatives from MEXT, MHLW, MEIT and AMED, shared four aspects from research, regulation, industry to budget review, to discuss the establishment of Japan regenerative medicine ecosystem.
iPS Cell Banks: Expecting to Cover over 50% Population of Japan by 2020
Professor Shinya Yamanaka said that many laboratories have differentiated iPS cells into myocardial cells, nerve cells, endothelial cells, etc., which are widely used in regenerative medicine research and pharmaceutical development. In terms of regenerative medicine, since autologous cell transplantation is not only expensive but time-consuming, Center for iPS Cell Research and Application (CiRA), Kyoto University, is establishing a local iPS cell bank, which is expected to effectively reduce the cost of cell therapy and benefit more patients. He also mentioned that people who had HLA-homozygous and obtained the donation consent through the Japanese Red Cross Society and Japan Marrow Donor Program would lead to the establishment of iPS cell bank, which is expected to cover the population of Japan for more than 50% by 2020. The ultimate goal is to be used by 80-90% of people in Japan.
However, it is a challenge for the establishment of the laboratories which met the good manufacturing practice (GMP) because the iPS cell stocks for clinical use must be high quality. With regard to this, CiRA established the iPS Facility for iPS Cell Therapy (FiT), which is responsible for the training of related personnel on quality control. Pharmaceuticals and Medical Devices Agency (PMDA), which is the main unit to examine and audit good clinical practice (GCP) in Japan, served as the consultant and assisted in the cellular material for medical use. In the aspects of the application results, including the research units and corporations, such as Kyoto University, RIKEN, Sumitomo Dainippon Pharma; all used iPS with CiRA to proceed 32 academic studies and 12 clinical research programs, and achieve the phased results in the clinical trials of age-related macular degeneration (AMD), heart disease, Parkinson’s disease. Moreover, CiRA has cooperated with Takeda to establish a massive cell bank for the treatment of heart disease and type 1 diabetes, which expected to expand the chance to collaborated with the international companies in the future, promote industrial development and benefit the public.
Academic Research, Industries, Regulations, and Budgets; the Ecosystem of Japan Regenerative Medicine Aspects
MEXT: Support the Basic Research and Young Entrepreneurs
Director Hideshi Semba from MEXT introduced that in order to advance the development of the biomedical field, Japan has promoted 9 Flagship Projects and Budgets (Sheet One). In the basic research and pharmaceutical development of regenerative medicine, it has invested 110 billion yen since 2012; CiRA is responsible for the cell bank stocks and cooperated with the academic institutions, which targeted on the different diseases to develop the related research and clinical trials of the iPS cell therapy. The academic institutes would give the feedback to CiRA, such as Kyoto University specialized in Parkinson’s disease, Institute of Physical and Chemical Research focused on the retina diseases, Osaka University concentrated on the heart disease, and Keio University engaged in the treatment of spinal cord injury. In the pharmaceutical development, which advanced 14 iPS cell research programs of the intractable diseases to develop the patient’s specificity iPS cell as the disease model, analyzed the potential molecular mechanism of diseases to proceed drug screening and discovery. The clinical trials of FOP and Pendred syndrome had respectively carried on this year and last year. Finally, he mentioned that MEXT would keep supporting the related research on iPS cell, ES and somatic stem cell, as well as the cross-industry cooperation with the MHLW, MEIT, and AMED. In addition to supporting the basic research, MEXT encourages the young entrepreneurs to activate the development of regenerative medicine industry by innovation.
MHLW: the New Law has Balanced Speed and Safety
For the regulations, manager Haruhiko Hakuno from research and development department of MHLW introduced the regenerative medicine background; there were only Medical Care Act and Pharmaceutical Affairs Law in Japan previously, which did not have the related regulations with the regenerative medicine. In order to react to the rapid emergence in this field, MHLW announced guideline for clinical research using human stem cell for the clinical research. As for the medical treatment, it did not have a clear definition. With regard to this, the government of Japan passed the Regeneration Medicine Promotion Law in 10th May 2013 to differentiated the applicants and the methods of gene therapy, which could decide the management methods and balance the treatment efficiency and safety. In the clinical treatment and research, the government of Japan announced Act on the Safety of Regenerative Medicine, which allowed the pharmaceutical organizations outsourcing the cell culture to the industries in accordance with the cell resources, processing methods, and the usage to classify the risk level. For the related products of cell therapy, it published the Revised Pharmaceuticals Affairs Act; according to the regenerative medicine products characteristics to provide the quick review mechanism and obtain the consent from patients before treatment and surveil when listing, which had 31 products carried on the clinical trial from 2013 to 2017, 4 drugs approved listing on April 2018, and 24 products achieved Sakigake Designation System.
MEIT: Advance the Development of Products and Improve the Entrepreneur Environment
Director Masahiro Uemura from MEIT talked in the industry way, mentioned that the most important is to build up the comprehensive industry chain, including the supply of material, cell processing, the related devices of cell culture, quality testing, shipment of the examples, the assessment system of pharmaceutical development, and the pharmaceutical development, etc. MEIT promote two programs, the first is to establish the supply units which could sustainably provide the cell material to help generate the products amount, such as CiRA, the other is to accelerate the business in regenerative medicine, which could help the startups investment on the program got more value, such as the establishment of the proceeding program. Furthermore, he especially pointed out 3 key reasons why Japan could harvest: increase the investment of startups, establish the connection between and startups, and establish the successful model, which could be followed by the research and development startups. He emphasized the importance of international cooperation; in addition to advancing the industry in Japan, it helps to expand the international market. Lastly, he mentioned that in his opinion, it is quite important to establish a cooperation platform with the industries, governments, and academia rather than concentrate on the process of individual programs.
The Budget has to be Spend in a Worthy Way: AMED One-stop Review Services
In order to accelerate the clinical application of medical basic research, the government of Japan established Japan Agency for Medical Research and Development (AMED) in 2015 to provide the financial assistance for medical research in Japan.
Yuka Suzuki, director of the regenerative medicine research division of AMED said that the research funding from the MEXT, MHLW, and MEIT would gather to AMED, and AMED would provide a one-stop service for the research program approved by AMED. At present, the government of Japan has spent 126.1 billion yen (about NT$34.8 billion) in 2018 to promote the 9 Flagship Projects in the biomedical field, including pharmaceutical development, medical devices, infrastructure of clinical research and trial, regenerative medicine, genomic medicine, cancer, mental and neurological diseases, emerging and re-emerging infectious diseases, and intractable diseases. The budget allocations are as shown in sheet one:
Sheet One. The 9 Flagship Projects and Budgets (Sort by the price)
|No.||Project type||billion Yen|
|7||Psychiatric and Neurological Disorders||11.6|
|8||Infrastructure of clinical research and trial||10.8|
|9||Emerging and re-emergeing Infectious diseases||6.4|
Source: AMED, Sort by GeneOnline
According to the sheet, pharmaceutical development, cancer research, and regenerative medicine are the top three key projects. Among them, regenerative medicine is expected to achieve the goal by 2020 to promote the clinical application of new drugs for iPS cells, increase the number of regenerative medicine products which approved by the review mechanism, improve the medical devices of regenerative medicine, and propose an international standard assessment of the safety on iPS cells heart disease treatment. In terms of the results, 31 projects have reached the clinical application stage from 2013 to 2017, and there are 4 cell treatment products approved by the review, and listed on market by April 2018, which shown in the following sheet:
Sheet Two. Approvals for Manufacturing and Marketing drugs
|Cell type||Name||Introduction||The approved year|
|Autologous cultured skin||JACE||Use the patient’s own healthy skin cells to culture the skin tissue and treat the burned patients.||2007|
|Autologous cultured cartilage||JACC||The cartilage tissue is cultured through the chondrocytes and replaced the damaged joint.||2012|
|Autologous Skeletal Myoblast Sheets||HeartSheet||Developed by professor Teruo Okano, a pioneer of cell layer, TERUMO CORPORATION, he used the discovery of cell layer to develop the treatment of severe heart failure on ischemic heart disease. Through the autologous thigh muscle cell to culture the myocardium layer with the cell layer technology and implant the myocardium layer to the patients’ heart.||2015|
|Allogenic human mesenchymal stem cell||TEMCELL® HS injection||Prochymal was first developed by Osiris, the allogenic human mesenchymal stem cell, which used for the implantation after hematopoietic stem cells GVHD. It listed in Canada and New Zealand by 2012, and Mesoblast from Australia bought the techniques and had an agreement with Japan JCR, which had the permission to list as TEMCELL in 2015.||2015|
reportFileDownload/C10503463/001, sort by GeneOnline
However, the development of regenerative medicine got questioned by the participants, because there were only four products approved for listing. In this regard, director Yuka Suzuki believes that iPS cell technology has only developed for ten years; there is still a lot of room for growth, but the emerging technologies have also brought many new business models. AMED also welcomes those who are interested to join, which made the system more perfect. After the conference, GeneOnline specially interviewed director Yuka Suzuki that we could have a deep understanding of AMED and the role AMED played in the field of regenerative medicine in Japan.
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