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Kyoto iPS Cell Research : CiRA's new goal of up to medical applications in 2030 of iPS cells

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Master
Date
2015-06-11 12:38
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2015幎05月14日
Source : http://www.huffingtonpost.jp/shinya-yamanaka/ips-2030_b_7228296.html

Kyoto iPS Cell Research : CiRA's new goal of up to medical applications in 2030 of iPS cells



This article was translated using Google Translator Japanese. So it can not be accurate. If necessary, please read the original source.



Huffington post US Version 10 anniversary, Congratulations Japan edition 2 anniversary.
Kyoto iPS Cell Research (CiRA = SILAPLANE) is established in April 2010, currently, about 30 research teams of cooperation respectively, and at the same time work on iPS cells in basic research, the research aimed at medical applications we are. In addition, ethical surrounding the iPS cells, legal, we are also conducting research on social issues. If you also include the research support team to assist in these studies, 320 people faculty total of is, we are engaged in research activities in order to achieve a mission of CiRA "medical applications of iPS cells".
iPS cells (induced pluripotent stem cells), is the stem cells that had been artificially developed by my research team is to introduce a small number of factors in the human body cell. Since it has the ability to produce all cells of the body and the ability to proliferate indefinitely, sometimes referred to as "universal cell". medical applications using iPS cells There are two major areas. One, from the iPS cells to differentiate into various cells of the body, is a regenerative medicine for transplant patients. Another is to elucidate the disease mechanism by reproducing a condition for iPS cell-derived cells made from the patient's cells are drug discovery to develop drugs.
When CiRA opened, we set a four goals to be achieved until 2020. At the time, shallow day the birth of iPS cell technology, is the realization of medical applications there were many challenges. So first 10 years with firm research, it was thought that that attempt to assess the new technology called iPS cells. Thanks to the cooperation of hard work and a lot of people of CiRA faculty, research in the last five years has been progressing well.

1. ensure the establishment and the intellectual property of the foundation technology
2. Building of medical iPS cell stock
3. from preclinical trials to clinical trials
4. Contribution to the development of therapeutic drugs using the iPS cells derived from patients

First it is about the goal of 1., but it was almost establish a safe iPS cell production technology. It also has acquired basic patent of iPS cell technology in the world 30 countries and one region. For 2., to start the establishment of medical iPS cells in 2013, now, about the ability of the iPS cells to differentiate into a variety of cells, but if I have to evaluate the external researchers. Omotte these results, I think that it can begin the distribution of iPS cells that can be used in medical care by the end of the year at the earliest. 3. also we are steadily goals. Last year in September, CiRA transplanted retinal pigment epithelial cells derived from iPS cells to the patient, such as is age-related macular degeneration RIKEN who are cooperating, clinical studies began with the world's first iPS cells It was. Check the safety of the treatment in animal experiments, called pre-clinical testing, such as Parkinson's disease or blood disorders in CiRA, 1, 2 year or less, we plan to proceed to clinical studies to target a small number of patients in. 4. For even drug discovery research, to achondroplasia, some existing drugs I was confirmed to be effective in animal experiments level. Currently, eagerly studying the possibility of application to the human of this existing drugs, we aim to enter the clinical research.

Collaboration with Takeda Pharmaceutical Company Limited


Regenerative medicine we have received generous support from the government, but an effort to advance the drug discovery research, we have concluded a comprehensive joint research agreement with Takeda Pharmaceutical Company Limited in mid-April this year. This is contribution from the research funds of 20 billion yen over the next 10 years Takeda, heart failure, diabetes, and will conduct disease research projects of neurological disorders such as 10 jointly. The role of venture company is a bridge between technology between universities and large companies have followed a weak situation in Japan. In order to overcome the "valley of death", it was decided to direct cooperation with large companies is not going through the venture. CiRA researchers a unique attempt to carry out a research project in Shonan within the Institute of Takeda, will say that the new industry-university cooperation model that can fully take advantage of the know-how of the pharmaceutical companies.
Such is the new approach is what luck risk, but you continue to challenge without fear of failure. It also actively promotes cooperation with other companies in Japan and overseas.

New goal until 2030


In CiRA, it leads to believe the initial goal of 2020, we set a long-term goal of 2030 to this April the new.

1. dissemination of regenerative medicine iPS cells stock as a pillar
2. drug discovery of realization and incurable diseases of individualized medicine by iPS cells
3. development of new life science and health care using the iPS cells
4. development of research environment and research support system of Japan's best level

The first goal is to be the general treatment of regenerative medicine using medical iPS cell stock currently conducting construction. using iPS cells in advance safety has been confirmed, we would want to achieve a system that can be implemented quickly the cell transplantation therapy, such as spinal cord injury. In the second goal, to use the iPS cells derived from patients, or drug is a cell level is good for the patient, and to take advantage of the fact that it is possible to determine not work, we aim to achieve individualized medicine. It also advances continue developing therapeutic agents of rare incurable disease. The third goal, by using the iPS cells as a tool (a tool), cancer and immune, I think want to advance the research to better understand the life phenomena such as generation. And we will continue to pioneer in the field of new medical care based on the new findings.
In order to achieve the three goals of the above-mentioned, it is essential is the fourth goal, "enhancement of research support system and research environment" is. To achieve clinical applications, technical personnel to perform cell culture and safety assessment, intellectual property, contracts, regulations, becoming more and more research assistants with expertise, such as public relations needs. However, in the fixed-term employment of about 90% of the faculty of CiRA a few years, the majority are involved in research and support services. To ensure the financial resources to hire over them a long period of time, has become my major challenge is the director of CiRA.

Fund for research support system and research environment improvement


Our Institute has received research funding through a collaboration with a large amount of public competitive funds and companies, but they are because there is time-limited and objective is the difference funds specific, Ya outstanding researchers it is not a sufficient to hire research assistants to a long period of time. The young researchers of education, the development of research environment, preparing for patent disputes, as well, such as support of exploratory research for future medical development, it can not respond sufficiently in research funding from the government and companies. Where they founded the "iPS Cell Research Fund", widely called for a donation, we are committed to trying to ensure the financial resources the power to fundraising activities. I myself participated in the full marathon, it is also or donations recruiting online. Securing financial resources for stable employment of enhancement and research assistants of the research environment is a challenge that many universities face. Through our fund initiatives, we want to take root the donation culture, such as gather donations from citizens in research activities in Japan.
The future, but I think that to face various challenges, they overcame, to accelerate research toward a mission of CiRA "medical applications of iPS cells", you want to deliver a new treatment in early patients a day I think.
Kyoto iPS Cell Research: https://www.cira.kyoto-u.ac.jp



iPS现胞の医療応甚 2030幎たでの新目暙



ハフィントンポストUS版10呚幎、日本版2呚幎おめでずうございたす。

京郜倧孊iPS现胞研究所CiRA=サむラは、2010幎4月に開蚭され、珟圚、玄30の研究チヌムがそれぞれ協力し、iPS现胞の基瀎研究に取り組むず同時に、医療応甚を目指した研究を行っおいたす。たた、iPS现胞を取り巻く倫理的、法的、瀟䌚的課題に぀いおも研究を進めおいたす。これらの研究を支揎する研究支揎チヌムも含めるず、総勢320人の教職員が、CiRAの䜿呜である「iPS现胞の医療応甚」を実珟するために研究掻動に取り組んでいたす。

iPS现胞人工倚胜性幹现胞は、私の研究チヌムが人間の䜓现胞に少数の因子を導入するこずにより人工的に開発した幹现胞です。無限に増殖する胜力ず䜓の党おの现胞を䜜り出す胜力を有しおいるこずから、「䞇胜现胞」ず呌ばれるこずもありたす。iPS现胞を䜿った医療応甚は倧きく二぀の分野がありたす。䞀぀は、iPS现胞から䜓の様々な现胞に分化させ、患者さんに移怍する再生医療です。もう䞀぀は、患者さんの现胞から䜜ったiPS现胞由来现胞に病態を再珟するこずにより病気のメカニズムを解明し、薬を開発する創薬です。

CiRA開蚭時、2020幎たでに達成する4぀の目暙を掲げたした。圓時はiPS现胞技術が誕生しお日が浅く、医療応甚の実珟には倚くの課題がありたした。そこでたず10幎間はしっかり研究しお、iPS现胞ず蚀う新しい技術の芋極めをしようずいう思いでした。CiRA教職員の頑匵りず倚くの方々のご協力のおかげで、この5幎間で研究は順調に進展しおいたす。

2020幎たでの初期目暙
1. 基盀技術の確立ず知的財産の確保
2. 医療甚iPS现胞ストックの構築
3. 前臚床詊隓から臚床詊隓ぞ
4. 患者さん由来のiPS现胞を甚いた治療薬開発ぞの貢献

たず1. の目暙に぀いおですが、安党なiPS现胞䜜補の技術をほが確立したした。たた、䞖界30カ囜1地域でiPS现胞技術の基本特蚱も取埗しおいたす。2. に぀いおは、2013幎に医療甚iPS现胞の暹立を開始し、珟圚、そのiPS现胞が様々な现胞に分化する胜力に぀いお、倖郚の研究者に評䟡を行っおいただいおいたす。これらの結果をもっお、早ければ幎内にも医療に䜿甚可胜なiPS现胞の分配を始めるこずができるず思いたす。3. の目暙も順調に掚移しおいたす。昚幎月には、 CiRAが協力しおいる理化孊研究所などが加霢黄斑倉性の患者さんにiPS现胞由来の網膜色玠䞊皮现胞を移怍し、䞖界初のiPS现胞を甚いた臚床研究が始たりたした。CiRAではパヌキン゜ン病や血液疟患など前臚床詊隓ず呌ばれる動物実隓で治療法の安党性を確認し、1、2幎以内に少数の患者さんを察象ずする臚床研究に進む蚈画です。 4. の創薬研究に぀いおも、軟骚無圢成症に察しお、ある既存薬が効果的であるこずを動物実隓レベルで確認したした。珟圚、この既存薬の人間ぞの応甚の可胜性に぀いお鋭意研究を進め、臚床研究に入るこずを目指しおいたす。

歊田薬品工業ずの共同研究



再生医療は政府からの倚倧なご支揎をいただいおいたすが、創薬研究を進める取り組みずしお、本幎4月䞭旬に歊田薬品工業株匏䌚瀟ず包括的な共同研究契玄を締結したした。これによっお、今埌10幎間で200億円の研究費が歊田薬品から拠出され、心䞍党、糖尿病、神経疟患など10の疟患研究プロゞェクトを共同で実斜したす。 日本では倧孊ず倧䌁業間の技術の橋枡し圹であるベンチャヌ䌁業の圹割が匱い状況が続いおいたす。その「死の谷」を乗り越えるために、ベンチャヌを介さず倧䌁業ず盎接連携するこずにしたした。CiRAの研究者が歊田薬品の湘南研究所内で研究プロゞェクトを遂行するずいうナニヌクな詊みで、補薬䌁業のノりハりを党面的に掻甚できる新しい産孊連携モデルず蚀えるでしょう。

このような新しい取り組みにはリスクが぀きものですが、倱敗を恐れずに挑戊しおいきたす。 たた、囜内倖の他の䌁業ずの連携も積極的に促進したす。

2030幎たでの新目暙



CiRAでは、2020幎たでの初期目暙達成を確信するに至り、この月に2030幎たでの長期目暙を新たに掲げたした。

CiRA Vision 20302030幎たでの新目暙
1. iPS现胞ストックを柱ずしお再生医療の普及
2. iPS现胞による個別化医薬の実珟ず難病の創薬
3. iPS现胞を利甚した新たな生呜科孊ず医療の開拓
4. 日本最高レベルの研究支揎䜓制ず研究環境の敎備

1぀目の目暙は、珟圚構築を進めおいる医療甚iPS现胞ストックを甚いた再生医療を䞀般的な治療にするこずです。予め安党性が確認されたiPS现胞を甚いお、脊髄損傷などの现胞移怍治療を迅速に実斜できる䜓制を実珟したいず思いたす。2぀めの目暙では、患者さん由来のiPS现胞を䜿い、现胞レベルである薬がその患者さんに効くか、効かないかを調べるこずができるこずを掻甚し、個別化医薬の実珟を目指しおいたす。たた、皀少難病の治療薬開発も匕き続き進めたす。3぀目の目暙は、iPS现胞をツヌル道具ずしお甚いるこずにより、がんや免疫、発生などの生呜珟象をよりよく理解する研究を進めたいず思いたす。そしお、新知芋に基づく新しい医療の分野を開拓しおいきたす。

䞊述の3぀の目暙を達成するには、4぀めの目暙である「研究支揎䜓制ず研究環境の充実」が䞍可欠です。臚床応甚を実珟するには、现胞培逊や安党性評䟡を行う技術者、知的財産、契玄、芏制、広報などの専門知識を持った研究支揎者がたすたす必芁になっおきたす。ずころが、CiRAの教職員のうち玄9割が数幎間の有期雇甚で、その倧半が研究支揎業務に携わっおいたす。圌らを長期間に亘り雇甚する財源を確保するこずが、CiRAの所長である私の倧きな課題ずなっおいたす。

研究支揎䜓制・研究環境敎備のための基金


私たちの研究所は倚額の公的な競争的資金や䌁業ずの共同研究を通じた研究資金をいただいおいたすが、これらは期限付きか぀目的が特定された資金であるため、優秀な研究者や研究支揎者を長期間に雇甚するには十分ずは蚀えたせん。たた若手研究者の教育、研究環境の敎備、特蚱係争ぞの備え、未来医療開拓のための萌芜的研究の支揎なども、囜や䌁業からの研究資金では十分に察応できたせん。そこで「iPS现胞研究基金」を創蚭し、広くご寄付を呌びかけ、財源を確保しようずファンドレむゞング掻動にも力を泚いでいたす。私自身がフルマラ゜ンに出堎し、オンラむンで寄付募集を行ったりもしおいたす。研究環境の充実や研究支揎者の安定雇甚のための財源確保は、倚くの倧孊が抱える課題です。私たちは基金の取り組みを通じお、研究掻動にも垂民から寄付が集たるような寄付文化を日本に根付かせたいず考えおいたす。

今埌、様々な課題に盎面するこずず思いたすが、それらを乗り越え、CiRAの䜿呜である「iPS现胞の医療応甚」に向けた研究を加速し、䞀日も早く患者さんに新しい治療を届けたいず思いたす。

京郜倧孊iPS现胞研究所 https://www.cira.kyoto-u.ac.jp 



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