6th DRUG DISCOVERY FOR  NEURODEGENERATION       CONFERENCE:An Intensive Course on Translating Research into Drugs February...
THANK YOU!Funding for this conference was made possible in part                                              by   Cooperat...
Thank You!MEETING SPONSORS   BRONZE SPONSORS
SCIENTIFIC ADVISORY COMMITTEE  SCIENTIFIC ADVISORY COMMITTEEKurt R. Brunden, PhD, University of PennsylvaniaNeil S. Buckho...
ADDF Staff• Diana Shineman, PhD – Assistant Director, Scientific Affairs• Rachel Lane, PhD – Scientific Program Manager• F...
NOTES Please remember to complete and submit the             meeting survey!  CME Certificates available at the Registrati...
SAVE THE DATE!13th International Conference on  Alzheimer’s Drug Discovery          September 10-11, 2012 • Jersey City, N...
Goals of the Meeting• Knowledge:  – The principles and practice of drug discovery, with a focus    on the unique aspects f...
Neurodegenerative Diseases         Affect >22 Million Worldwide       Some symptomatic agents, few disease modifying drugs...
Drug Discovery is a Vital Stage in Drug Development                  When Innovation is Created                        Pro...
Opportunity and Challenges for Success:A Perspective On The Origin of FDA Approved Drugs  20,000 human genes              ...
How a Biologist Thinks About Drug Discovery:           Many Targets for Neurodegeneration?• Deposits of Misfolded Protein ...
How a Chemist Thinks About Targets for Drug Discovery:           Success Rates of Target Types• Target types   –   GPCR (s...
Why A Biological Network Approach to Drug Discovery is    Needed: Signaling in the Synapse is Complex
How Were New Drugs Discovered?Phenotypic Screening Vs. Target-based             Screening              Swinney, et al, Nat...
Case Studies: Routes to Drug Discoverybeta-secretase inhibitors                                                           ...
Improving Success Rates?      Drug Discovery in Academia• Drug discovery is the interface between basic research  and clin...
Drug Discovery and Development Requires      Multidisciplinary Teams of Scientists        Clinical Trialists              ...
Feeding the Pipeline: The Alzheimer’s Drug Discovery Foundation     The ADDF has granted over $55 million to >370Alzheimer...
Drug Discovery: The “Valley of Death”?Or “Welcome to An Amazing Journey”!
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  • Figure 2 | The distribution of new drugs discovered between 1999 and 2008, according to the discovery strategy. The graph illustrates the number of new molecular entities (NMEs) in each category. Phenotypic screening was the most successful approach for first-in-class drugs, whereas target-based screening was the most successful for follower drugs during the period of this analysis. The total number of medicines that were discovered via phenotypic assays was similar for first-in-class and follower drugs — 28 and 30, respectively — whereas the total number of medicines that were discovered via target-based screening was nearly five times higher for follower drugs versus first-in-class drugs (83 to 17, respectively). Nature drug discovery july 2011
  • Sunday (1) fillet

    1. 1. 6th DRUG DISCOVERY FOR NEURODEGENERATION CONFERENCE:An Intensive Course on Translating Research into Drugs February 12-14, 2012 - New York City Howard Fillit, MDExecutive Director, Alzheimer’s Drug Discovery Foundation
    2. 2. THANK YOU!Funding for this conference was made possible in part by Cooperative Agreement U13AG031125-05 from the National Institute on Aging. The views expressed in written conference materials or publications and by speakers andmoderators do not necessarily reflect the official policies of the Department of Health and Human Services; nor does mention by trade names, commercial practices, or organizations imply endorsement by the U.S. Government.
    3. 3. Thank You!MEETING SPONSORS BRONZE SPONSORS
    4. 4. SCIENTIFIC ADVISORY COMMITTEE SCIENTIFIC ADVISORY COMMITTEEKurt R. Brunden, PhD, University of PennsylvaniaNeil S. Buckholtz, PhD, National Institute on AgingRebecca Farkas, PhD, National Institute of Neurological Disorders and StrokeHoward Fillit, MD, Alzheimer’s Drug Discovery FoundationBrian Fiske, PhD, Michael J. Fox Foundation for Parkinson’s ResearchMark Frasier, PhD, Michael J. Fox Foundation for Parkinson’s ResearchAbram Goldfinger, MBA, New York UniversityLorenzo Refolo, PhD, National Institute on AgingSuzana Petanceska, PhD, National Institute on AgingDiana Shineman, PhD, Alzheimer’s Drug Discovery FoundationEdward G. Spack, PhD, Fast Forward, LLCD. Martin Watterson, PhD, Northwestern University
    5. 5. ADDF Staff• Diana Shineman, PhD – Assistant Director, Scientific Affairs• Rachel Lane, PhD – Scientific Program Manager• Filomena Machleder – Assistant Director, Institutional Partnerships• Natalie Romatz – Partnerships Assistant, Institutional Partnerships• Niyati Thakker – Grants Assistant• World Events Forum – Conference Secretariat
    6. 6. NOTES Please remember to complete and submit the meeting survey! CME Certificates available at the Registration DeskA webcast of the conference will be available soon on our website: www.alzdiscovery.org
    7. 7. SAVE THE DATE!13th International Conference on Alzheimer’s Drug Discovery September 10-11, 2012 • Jersey City, NJ across from NYC on the Hudson River
    8. 8. Goals of the Meeting• Knowledge: – The principles and practice of drug discovery, with a focus on the unique aspects for neurodegenerative diseases• Network: – >190 attendees from 20 countries, ~40% from industry – Exchange ideas, foster alliances, partnerships and collaborations
    9. 9. Neurodegenerative Diseases Affect >22 Million Worldwide Some symptomatic agents, few disease modifying drugs Multiple Huntington’s, 30, sclerosis, 400,00 000 ALS, 30,000 0• WHO estimatesneurodegenerative disorders will Parkinson’sbe the major unmet disease, 1,000,00 0medical need of the 21stcentury,• surpassing cancer as the Alzheimer’s disease, 5,000,00worlds’ second leading cause of 0death by the year 2040
    10. 10. Drug Discovery is a Vital Stage in Drug Development When Innovation is Created Proof Safety and Proof Innovation of Mechanism Proof of Concept of Efficacy ANIMAL BIOLOGY STUDIES and AND CHEMISTRY HUMAN STUDIES PHARMACOLOGY10,000 to 1 FDA>1 million Approvedchemicals Drug Developing a Drug is Risky, Takes 12-15 years and Costs Over $1.2B
    11. 11. Opportunity and Challenges for Success:A Perspective On The Origin of FDA Approved Drugs 20,000 human genes ~50M compounds in Chem Abstracts; 100,000 proteins 1040-10100 possible small molecules ~10,000 approved drugs Most are variants on formulation and delivery Many anti-microbials Less than 500 distinct chemical entities Targeting ~266 human genome derived proteins Less than 50 unique chemical scaffolds From: T. Bartfai and GV Lees, Drug Discovery from Bedside to Wall Street, 2006; Le Couteur, et al 2011
    12. 12. How a Biologist Thinks About Drug Discovery: Many Targets for Neurodegeneration?• Deposits of Misfolded Protein – Β-Amyloid, tau, α-synuclein, TDP-43, poly-Q aggregates• Oxidative stress• Inflammation• Mitochondrial dysfunction• Synaptic and neuronal cell dysfunction• Vascular ischemia and damage• Other novel mechanisms (eg. epigenetics)
    13. 13. How a Chemist Thinks About Targets for Drug Discovery: Success Rates of Target Types• Target types – GPCR (small ligand) High – Enzyme (small ligand) – Ion channel – Nuclear receptor – Protease Success – Enzyme (large ligand) – GPCR (large ligand) – Cytotoxic (other) – Protein kinase – Protein-protein Low
    14. 14. Why A Biological Network Approach to Drug Discovery is Needed: Signaling in the Synapse is Complex
    15. 15. How Were New Drugs Discovered?Phenotypic Screening Vs. Target-based Screening Swinney, et al, Nature Reviews Drug Discovery, July, 2011
    16. 16. Case Studies: Routes to Drug Discoverybeta-secretase inhibitors gamma-secretase inhibitors Inhibitor Development Rational design approach Screening approach Assay development generation of protein High throughput screen (500,000 cpds.) Identification of hits Crystal Computer Structure M odel Selection of leads Focused Medicinal Chemistry Potency M edicinal Chemistry Specificity PK Test for in vivo activity
    17. 17. Improving Success Rates? Drug Discovery in Academia• Drug discovery is the interface between basic research and clinical development• Requires extensive resources and collaboration between teams of investigators• Increasingly requires partnerships between pharma, biotechs, non-profits, and government, especially for neurodegenerative diseases
    18. 18. Drug Discovery and Development Requires Multidisciplinary Teams of Scientists Clinical Trialists Clinical Development IND enabling studies: ADMET, Pharamaceutical Scientists formulation and scale-up chemistry Animal Trialists In vivo Testing and Biomarker Development Preclinical Proof of MechanismMedicinal Chemistry, Pharmacology Lead Identification and optimization Assay Development High Throughput Structure Based Chemical Libraries Screening Chemistry Computational Chemistry Basic Neurobiology Target identification
    19. 19. Feeding the Pipeline: The Alzheimer’s Drug Discovery Foundation The ADDF has granted over $55 million to >370Alzheimer’s drug discovery programs in academic centers and biotechnology companies in 20 countries ADDF funding has resulted in >$2 billion in follow-on commitments, and several novel drugs entering clinical trials www.AlzDiscovery.org
    20. 20. Drug Discovery: The “Valley of Death”?Or “Welcome to An Amazing Journey”!

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