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Japan Agency for Medical Research and Development (AMED)
Missions and Challenges
Makoto Suematsu, MD, PhDPresident
Japan Agency for Medical Research and Development
JSGT2015 and APGTC2015 in Osaka July 24, 2015
3 “MUST NOT” rules in AMED organization
A success of AMED (Japan Agency for Medical Research and Development) highly depends on alliance and cooperativity of three ministries (METI, MEXT, MHLW)
To this end, we need to challenge for an art of probability……..
AMED (1.4 billion US$/FY2015)
METI MEXT MHLW
Not only budgets but also officers and scientists with new mindsets
Our goal is to fast-track medical R&D that directly benefits people not only by extending lifespans but also by improving quality of life:supporting “life” as biological existance, diary living, and lifespans
Office of Audit
Au
dit
or
Department of General Affairs
Department of Industrial-Academic Collaboration
Department of Research Promotion
Department of Research InfrastructureDepartment of Clinical Research and Trials
Department of International Affairs
Department of Intellectual Property
Department of Research Integrity and Legal Affairs
Department of Innovative Drug Discovery and Development
Department of Financial Affairs
Man
age
me
nt
Sect
ion
Sup
po
rt
Sect
ion
Pro
ject
Se
ctio
n
Department of Planning and Management
Exec
uti
veD
irec
tor
Ad
viso
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oar
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Co
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cil o
f R
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an
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anag
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Au
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Permanent Staff Fixed-term Staff
102 Approx. 200
Organization of AMED
Drug Research
Regenerative Medicine Research
Cancer Research
Infectious Disease
Research
Neurological. Psychiatric and Brain Research
Rare/In-tractable Disease
Research
Emerging Research
Support for accommodating R&D platforms such as BioBank, etc.
Promotion of strategic international researches
Support through the Drug Discovery Support Network for realizing academia drug discovery
Support for practical application such as industrial-academic collaboration
Longitude/cross-sectional collaboration between “Department of Research PromotionMatrix structure for accelerating Medical R & D
Support for high-quality clinical studies/clinical trials
7 Research Projects
3
Undiagnosed Disease Program in NIHdiagnose (and treat) those undiagnosed ( from 2008, Dr William Gahl, NIH)
Undiagnosed Disease Patients
Home doctors Regional Core Hospitals
Collection of data and
examination
Genome analysis center
Medical Consultation
Local Specialists of Clinical Genetics
The Japan’s National Liaison Council for Clinical Sections of Medical Genetics etc.
G and M data
Genetic counselling
Diagnose known diseases
Assessment by specialist of clinical
genetics
Return of the Research Findings to Patients Individually
Known ones?
Analyses by physicians with
different specialties
Data banking
Judge treatable or not ?
Phenotyping
NGS (ISO, CLIA etc)
metabolomics
Strong team of physician scientists
4
Mutation of riboflabin receptor causes “ALS-like” severe phenotypes in a newborn baby.
Johnson et al Brain 2012:135;2875-2882
AMED will start✔ Joining in IRDiRC in this autumn✔ Organizing IRUD (Initiative for Rare and undiagnosed diseases)
under collaboration with NIH-UDP Program
Dr. William Gahl, MD, PhDFounder of undiagnosed Patients Program, NIH
“Not sequencers but real physicians” “ Phenotype is everything”
N Engl J Med. 2015 Jun 4;372(23):2235-42
In genome medicine, “likely pathogenic” variant now may turn out “likely benign” 5 years later;Necessity for real-time data sharing of clinical genetics through DB
IRUD診断連携・IRUD診断委員会は地域の拠点病院
等に配置し、全国配備を目指す
紹介受診
フォローアップ
エントリーシート(臨床・検査データ)
受診
IRUD診断委員会・幅広い診療科の医師で構成・臨床遺伝専門医を中心として臨床カンファレンスを開催・地域の臨床医と積極的に連携し地域をあげて取り組む
IRUD解析コンソーシアム
IRUD解析センター
・依頼された検体についてエクソーム解析等の遺伝学的検査を行う・遺伝学的検査以外の検査や遺伝子変異の機能解析も検討する
解析結果
検査依頼
診断困難な患者さん
AMED データセンター
・登録システムの構築・運営・データベースの構築・運営・国際ネットワークとの連携
IRUDデータネットワーク
データ
診断説明カウンセリング
IRUDに参加する拠点病院(総合病院)地域での
密接な連携
AMED has launched Initiative for Rare and Undiagnosed Diseases
(IRUD)
症例検討依頼 検討結果
フィードバック
IRUD臨床専門分科会
・疾患エキスパートによる専門分科会・IRUD診断委員会による依頼を受け、専門的な症例検討を追加する・必要に応じてIRUDに参加する拠点病院で直接診療
IRUDに参加する拠点病院
IRUD診断委員会
・幅広い診療科の医師で構成・臨床遺伝専門医を中心として臨床カンファレンスを開催・地域の臨床医と積極的に連携し、地域をあげて取り組む
IRUDに参加する
拠点病院が全国に設置されるよう体制整備を支援していく
IRUD constitutes a network consisting of multiple hospitals to save patients with RUD with equal opportunities all over Japan
Japan has joined IRDiRCin July 30, 2015
✔ Machine-readable consent
✔ microattribution
✔ data sharing for patients
AMED has participated in IRDiRC to share IRUD data for saving patients over the world
Advocacy activities by patients and their supporters;Patients constitutes a stakesholder that participates in designing clinical researches
Increase and influence government and private
funding for research
Promote running “smart” clinical trials
Heighten public awareness and visibility
Increase clinical trial enrollment rate
Identify the best practices and disseminate them across the
country
2015 2020
Permission by Julie Fleshman, President and CEO, PanCan
Discovery Innovation Screening Consortium: DISC
Purpose: Enrich “All Japan” screening library by integrating unique compounds ofpharmaceutical companies for use in HTS by the Drug Discovery Support Network
Univ. of TokyoLow MW compounds
AIST(N2PC)Natural products
NIBIOHNmAb-Phage/aptamer
AMED/iD3Low MW compoundspharma-companies
・Highly patentable, unique structures
Pharma A
Compound storage, assay plate preparation, HTS implementation and cherry picking to be outsourced to industry
・Tool compound discovery
・Lead discovery
・HTS by RIKEN
Impact of DISCWhole: create the first non-competitivecollaborative space in Japan and improve overallpharma R&D productivity
Academics: increase the chance to find qualitylead and tool compounds for their own newlydiscovered targets
Industry:maximize potential value of their own
compounds through increased chance to bescreened out against new targets discovered byuniversities, etc.
HTS
YCo. LtdPharma B
Pharma CPharma DPharma E
“All Japan” Screening Library
Storage assay-plate
preparation
XCo. Ltd
Already established:AIST, NIBIOHN, Univ.Tokyo
Pharma・・
Public-Private Partnership to take advantage ofunique compounds & HTS technology of pharma-industry for more effective translational research● iD3 to select targets & cover HTS cost● Pharma to provide access to premiumcompounds and HTS technology/equipment
DISC
CONFIDENTIAL
Input scaffold
information
Copyright © 2015 All Rights Reserved.
Liver tox risk
Side effect
Metabolizing
enzyme
Plasma stability
:0.002
:ILD
:CYP2C8
:Stable
structure information
X Tox DB
Physico-chem DB
ADME DB
DB integration
e.g. DISCHit candidates
True hit identification
eye ball
structure design
synthesis
Lead compound generation
Example ofthe system architecture
Schematic flow for the use of the system
Schematic diagram of the system
Output ADEM-T
information
Input scaffold
information
Output ADEM-T
information
To construct a system that can predict toxicological risk etc. with a certain significance levelby integrating various databases developed to date in our country, while identification of truehit compounds and generation of lead compounds have been done by eye ball of medicinalchemists with experience and expertise.
Constructing the system within 3 years, followed by the 2-year validation period
IT system for Supporting Development of Drug Discovery
Structure
optimization
unit
Platform for Drug Discovery, Informatics
and Structural life science
Toxicity・ADME information
AMED Mission1 Global Cooperation for Controlling Diseases:
①Infectious diseases②Regenerative medicine③Neuropsychiatric diseases④Rare diseases (and undiagnosed diseases)
and R/D for orphan drugs
2 Empowerment of Medical R&D in Japan① Opening resources of drug libraries② Accelerating nation-wide registry for optimizing cancer Tx③ Establishing infrastructure of clinical genetics (CLIA lab, etc)④ Fostering young physician scientists for medical R/D⑤ Utilization of private-public partnership to stimulate medical R/D
3 Renew selection processes and improve flexibility of budget systems4 Clinical database
(under supervision by Cabinet Secretariat, Office of Healthcare Policy)14
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