Global Dementia Legacy Event: Dr Neil Buckholtz


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Session Two: Barriers to investment in research to find a disease modifying therapy or cure for dementia

Dr Neil Buckholtz , Director of Neuroscience, the National Institute on Aging (NIA), National Institutes of Health (NIH)

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Global Dementia Legacy Event: Dr Neil Buckholtz

  1. 1. Global Dementia Legacy Event Neil Buckholtz, Ph.D. Director, Division of Neuroscience National Institute on Aging June 18, 2014
  2. 2. International Alzheimer’s Disease Research Portfolio (IADRP) • Developed by NIA in collaboration with Alzheimer’s Association. • Will enable funders of Alzheimer’s research to coordinate planning, leverage resources, avoid duplication, and identify new opportunities. • Will give the public a full picture of the scale of ongoing research on AD both nationally and internationally.
  3. 3.  CADRO is a three-tier classification system created by NIA and the Alzheimer’s Association to capture the complete range of AD research  First level of classification consists of seven categories: 5 research and 2 research resources-related:  Category A – Molecular Pathogenesis and Physiology of Alzheimer’s Disease  Category B – Diagnosis, Assessment and Disease Monitoring  Category C – Translational Research and Clinical Interventions  Category D – Epidemiology  Category E – Care, Support and Health Economics of Alzheimer’s Disease  Category F – Resources for the Research Community  Category G – Consortia and Public Private Partnerships  Each category is divided into “research topics”; many topics further divided into “research themes”  Detailed classification will enable funders and researchers to identify research gaps, areas of overlap/duplication of effort, and opportunities for collaboration with much greater specificity Common Alzheimer’s Disease Research Ontology (CADRO) Structure
  4. 4.  Web-based portal developed by NIA and the Alzheimer’s Association: Alzheimer’s and related dementias research funded projects 2008 to 2012.  Currently includes: Federal agencies (including NIH, AHRQ, CDC, AoA, VA and DoD), Alzheimer’s Association, Alzheimer’s Drug Discovery Foundation, and Alzheimer’s Research UK, Alzheimer’s Society UK, Alzheimer’s Society of Canada, and Alzheimer’s Australia.  Summer 2014: Additional funders (10) and 2013 data will be available on IADRP.
  5. 5. 1. Amyloid 32% 2. Tau 6% 3. Presenilins 4% 4. ApoE and Lipids 9% 5. Brain Circuits and Synapses 7% 6. Neurogenesis Cell Death 5% 7. Immunity and Inflammation 10% 8. Bioenergetics 1% 9. Vascular/Metabolic Factors 5% 10. Hormones 3% 11. Genetics 11% 12. Other Pathogenic Mechanisms 7% Proportion of ‘Category A’ Projects by Topic across All Funding Organizations (2012)
  6. 6. 20% 30% 40% 50% 60% 70% 80% 90% 100% 2008 2009 2010 2011 2012 12. Other Pathogenic Mechanisms 11. Genetics 10. Hormones 9. Vascular/Metabolic Factors 8. Bioenergetics 7. Immunity and Inflammation 6. Neurogenesis Cell Death 5. Brain Circuits and Synapses 4. ApoE and Lipids 3. Presenilins 2. Tau 1. Amyloid Changes in the Proportion of ‘Category A’ Projects by Topic for All Funding Organizations (2008 - 2012)
  7. 7. Step 1 Funding organizations identify their respective AD and AD- related dementia projects Step 2 Funding organizations provide the IADRP Team with projects in a designated excel template or via links to projects online along with specified data elements and abstracts Step 3 The IADRP Team codes projects (via an automated coding portal) using the Common Alzheimer's Disease Research Ontology (CADRO) ( rch/dn/cadro-outline) Step 4 Funding organizations validate the coding via the coding portal Step 5 Coded and validated projects are uploaded into the IADRP online database ( Portfolio Analysis All users can systematically compare and analyze funding, composition of research, specific trends in funding over time, geographic distribution of projects, and overlapping funding priorities IADRP Submission Process
  8. 8. Accelerating Medicines Partnership Alzheimer's Disease Program 8
  9. 9. Why AMP? Why now? Developing effective new medicines takes too long, costs too much and fails too often.
  10. 10. AMP Pilots: Alzheimer’s disease Type 2 diabetes Rheumatoid arthritis/systemic lupus erythematosus
  11. 11. NIA Supported Secondary Prevention Trials Identify biomarkers correlated with therapeutic benefit in pre-symptomatic trials Proposal Description Principal investigator Dominantly Inherited Alzheimer Network Trials Unit (DIAN-TU) Trial • Phase II/III study to assess the safety, tolerability, and biomarker efficacy of gantenerumab and solanezumab in mutation carriers • Randall J. Bateman, WUSL • Collaborating companies: – Lilly – Roche – Alzheimer's Assoc. – Avid Radiopharm. – Cog State The Alzheimer's Prevention Initiative APOE4 Trial • Testing an anti-amyloid drug (TBD) in cognitively normal older volunteers who are at increased risk of developing late-onset Alzheimer’s (APOE-e4 homozygotes) • Eric Reiman, BANNER • Pierre Tariot, BANNER Alzheimer’s Disease Cooperative Study Anti- Amyloid Treatment in Asymptomatic AD Trial (A4) • Secondary prevention trial of solanezumab in clinically normal older people with biomarker evidence of brain amyloid. • Reisa Sperling, Harvard • Paul Aisen, UCSD © The Boston Consulting Group, Inc.
  12. 12. AMP Project A • Supplement the imaging and fluid biomarker panels already included in these three NIH-funded Phase II/III registration trials in presymptomatic Alzheimer’s through the addition of tau PET imaging, EEG measures, and novel fluid biomarkers.
  13. 13. Amyloid and Tau PET imaging K. Johnson HAI 2014
  14. 14. AMP Project B • Apply integrated network analyses (RNA, proteomic, other “omic” studies) in 2500 human AD brain samples to identify biologic nodes and networks that are linked to the development or progression of AD to identify new targets for drug development. • Create standardized open-source data structures and formats to aid the accessibility and ease of analysis of biological data in a manner not currently practiced in the AD field.
  15. 15. Pathway discovery, validation and compound identification for Alzheimer's disease* Phil De Jager (Contact PI) Broad Institute David Bennett Rush University Integrative Biology Approach to Complexity of Alzheimer's Disease* Eric Schadt (Contact PI) Icahn Institute for Multiscale Biology at Mount Sinai & Dept of Genetics and Genomics Sciences A System Approach to Targeting Innate Immunity in AD* Todd Golde (Contact PI) – University of Florida Nathan Price – Institute for Systems Biology, Seattle Nulifer Ertiken-Taner and Steven Younkin – Mayo Clinic Jacksonville Funded Projects Allan Levey (Contact PI) Emory University Discovery of Novel Proteomic Targets for Treatment of Alzheimer's Disease* Lara Mangravite and Stephen Friend –Sage Bionetworks Administrative Supplements for Data Enablement and Data Integration
  16. 16. Projected AMP funding contributions Disease area Total project funding ($M) Total NIH funding ($M) Total industry funding ($M) AD 135 67.6 67.4 T2D 58.4 30.4 28.0 RA/SLE 41.6 20.9 20.7 Total 235 118.9 116.1 Industry is also providing AMP with additional in-kind contributions, e.g., clinical trials, drug, tracer, databases, etc. © The Boston Consulting Group, Inc.
  17. 17. AMP Participation by Disease Area Alzheimer's disease Type 2 Diabetes RA, SLE & related Industry members Government members Non-profit members © The Boston Consulting Group, Inc.