bill matthew director, office of translational research; ninds bill.matthew@nih
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U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
American Society for Experimental Therapeutics
Annual MeetingMarch 04-06, 2010
Therapeutic Development
Resources at NINDS
Bill MatthewDirector, Office of Translational Research; NINDS bill.matthew@nih.gov
1 -Matthew
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
Pipeline Principles: 101
Basic Research
Therapeutics
Raw Material & Pump
Block ValveStation Final Delivery
2 -Matthew
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
Translational Research: The process of using novel, basic research discoveries to develop clinical applications.
T1
T2
T1
bench
bedside
community3 -Matthew
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
Office of Clinical Research
Office of Translational Research
NINDS Extramural Research
4 -Matthew
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
Basic Research -Target ID
Assay Hit to LeadScreen for Hit
Proof of Principle
Lead Optimization
PreClincal Candidate – Safety/Tox
First in Human
Anti-Convulsant Screening Program
Exploratory/Developmental Projects in Translational ResearchCooperative Program in Translational Research
Translational SBIR
Blueprint Neurotherapeutics Grand Challenge
CounterACT
RAID: Rapid Access to
Interventional Development
Small Molecule
Optimization
Small Business Program: SBIR & STTR
Spinal Muscular Atrophy Project
Molecular Libraries
(Roadmap)
Assay Development (Roadmap)
Drug Discovery for
Nervous System
Disorders
NINDS/OTR Activities in Therapeutic Development: NINDS Multiple ICs
5 -Matthew
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
Anticonvulsant Screening Program (ASP)
Goal: To facilitate the development of drugs for patients with epilepsy, resistant epilepsy and related CNS conditions
• Screening performed via a contract mechanism using a battery of seizure models• Compounds are submitted by a broad base of Investigators: > 500 total
Industrial companies (~200) Academic institutions (~300)
• ASP Database > 29,000 small molecules – SAR chemistry & biology
• Compounds in clinical development: currently six
• Contributed to the development of ten marketed anti-convulsant drugs
Jim Stablesjs131e@nih.gov
www.ninds.nih.gov/research/asp/index.htm 6 -Matthew
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
Exploratory/Developmental Projects in Translational Research
www.ninds.nih.gov/research/translational/index.htm
Goal: Develop in vivo animal proof-of-concept for efficacy
•Program (R21) primarily targets four tasks:
• Creation and validation of screening assays
• Development of animal models of disease
• Identification of candidate therapeutics
• Development of preliminary animal efficacy data
• However, can be used for any preclinical development task
Tom Millertm208y@nih.gov
7 -Matthew
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
Cooperative Program inTranslational Research
www.ninds.nih.gov/research/translational/index.htm
Tom Millertm208y@nih.gov
Goal: Bring novel compounds and devices to the clinic
•Cooperative Agreement, Milestone-driven program• Significant staff involvement • Reviewed by therapy development experts
• IND/IDE by the end of the research project period
• 6 filed INDs and 3 Clinical Trials
8 -Matthew
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
Cooperative Program inTranslational Research U44 Small Business Awards
www.ninds.nih.gov/research/translational/index.htm
Goal: Facilitate Small Business development of neurotherapeutics
•Fast-Track (Phase I & II) and Phase II Applications accepted
• Phase I - identical scope as translational R21
• Phase II - identical scope as translational U01
Randy Stewartrs416y@nih.gov
Tom Millertm208y@nih.govFor Translational
9 -Matthew
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
THE SMA PROJECT
Goal: Develop a safe and effective drug for SpinalMuscular Atrophy
• Develop drugs that increase expression of SMN protein deficient in SMA patients
• A ‘Virtual Pharma’ network of contracts for industry-style drug development:
• medicinal chemistry • in vitro testing • pharmacological and toxicological studies • animal model testing
• Industry-seasoned consultants and advisors to oversee programs
www.smaproject.org
Jill Heemskerkjh440o@nih.gov
Coming Very Soon
10 -Matthew
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
Blueprint Neurotherapeutics Grand Challenge
•Goal: Provide Medicinal Chemistry and Pharm/tox expertise for developing new drugs for nervous system disorders
•Investigator-initiated drug starting points
•“Virtual Pharma” model of drug development
• Provide access to industry-standard services: medicinal chemistry, pharm/tox, phase I clinical trials
•www.grants.nih.gov/grants/guide/notice-files/NOT-NS-10-002.html
Jill Heemskerkjh440o@nih.gov
Coming Very Soon
11 -Matthew
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
Goal: To develop safe and effective therapies and diagnostic technologies for victims of emergency incidents involving exposure to highly toxic chemicals
CounterACT Countermeasures Against Chemical Threats
David Jett jettd@ninds.nih.gov
www.ninds.nih.gov/counteract
• Research Centers of Excellence
• Individual research projects at academic, government and industry laboratories
• Cooperative Agreements, Milestone-driven
• Efficacy screening and preclinical services contracts
• Interagency Agreements with DoD laboratories
12 -Matthew
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
www.ninds.nih.gov/research/molecular_libraries/index.htm
Mark Scheidelerscheidererm@ninds.nih.gov
Assay Development: Molecular Libraries and Imaging Roadmap Program
Goal: Develop assays to identify small molecule interactions via high-throughput screening of compound libraries, and to chemically optimize active compounds as molecular probes
13 -Matthew
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
NIH-RAIDNIH Rapid Access to Interventional Development
• Pre-IND studies
• All diseases
• Access to government expertise and contract resources – Production, bulk supply, GMP manufacturing– Pharmacokinetic/ADME studies– IND-directed toxicology, and more
• 6 successful IND submissions and 4 clinical trials to date
Tom Millertm208y@nih.gov
www.nihroadmap.nih.gov/raid
Goal: To execute IND-enabling studies for extramural projects
14 -Matthew
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
Basic Research -Target ID
Assay Hit to LeadScreen for Hit
Proof of Principle
Lead Optimization
PreClincal Candidate – Safety/Tox
First in Human
Anti-Convulsant Screening Program
Exploratory/Developmental Projects in Translational ResearchCooperative Program in Translational Research
Translational SBIR
Blueprint Neurotherapeutics Grand Challenge
CounterACT
RAID: Rapid Access to
Interventional Development
Small Molecule
Optimization
Small Business Program: SBIR & STTR
Spinal Muscular Atrophy Project
Molecular Libraries
(Roadmap)
Assay Development (Roadmap)
Drug Discovery for
Nervous System
Disorders
NINDS/OTR Activities in Therapeutic Development: NINDS Multiple ICs
15 -Matthew
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
Please Contact us…
www.ninds.nih.gov
Thank you
16 -Matthew
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICESNational Institutes of Health
National Institute of Neurological Disorders and
Stroke
Activity Code
R01 Research Project
R21 Exploratory/Developmental Grants
R41 Small Business Technology Transfer (STTR) Grants - Phase I
R42 Small Business Technology Transfer (STTR) Grants - Phase II
R43 Small Business Innovation Research Grants (SBIR) - Phase I
R44 Small Business Innovation Research Grants (SBIR) - Phase II
U01 Research Project--Cooperative Agreements
U44 Small Business Innovation Research (SBIR) Cooperative Agreements
U54 Specialized Center--Cooperative Agreements
X01 Resource Access Award
grants.nih.gov/grants/funding/funding_program.htm
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