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The CFF Story: Innovating for a Cure

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The CFF Story: Innovating for a Cure, presented by Robert Beall, PhD, Cystic Fibrosis Foundation, HSG 2016

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The CFF Story: Innovating for a Cure

  1. 1. The CFF Story: Innovating for a Cure Huntington Study Group November 4, 2016 Robert J. Beall, Ph.D. Former President and CEO, Cystic Fibrosis Foundation
  2. 2. The CFF Story: Innovating for a Cure  Community  Clinical Care  Research  Discovery & Development  Treatment
  3. 3. Median Predicted Survival Age of US Patients with Cystic Fibrosis Source: Cystic Fibrosis Foundation, National Patient Registry 41 0 5 10 15 20 25 30 35 40 45 1940 1950 1960 1970 1980 1990 2000 2010 2015
  4. 4. As of 2015 There Are More Adults With CF Than Children With CF in the U.S. Cystic Fibrosis Foundation. Patient Registry Annual Report. Bethesda, MD: Cystic Fibrosis Foundation. 2015 %ofCFindividualsovertheageof18 1965 1970 1975 1980 1985 1990 1995 2000 2005 2010 2015 0 10% 20% 30% 40% 50%
  5. 5. 46% Other Indications of Success 22% CF Adults Employed CF Adults Attending College
  6. 6. Innovating Community
  7. 7. Academics • Research • Care • Training Biopharmaceutical • Translation • Commercialization CF Foundation • Care • Research • Education Ecosystem for Cystic Fibrosis Foundation Success FDA NIH
  8. 8. Innovating Care
  9. 9. Care Center Network Care Center Network Peer accreditation • 119 care centers, • 107 adult programs, • 51 affiliates
  10. 10. Quality Improvement Program • Web-based registry • Learning and Leadership Collaboratives • Leadership development • Benchmarking - identify and enable “best practices” • Discipline-specific mentoring programs • Patient/family engagement and partnership • Transparency of center-level outcomes
  11. 11. 2002 50 60 70 80 90 100 110 20 25 30 35 40 45 50 55 60 65 70 FEV1PercentPredicted BMI Percentile FEV1 vs. BMI Percentile for Patients 6 to 17 Years, 2002 vs. 2012 32.2%17.5% 18.9% 2002 Median Values 31.5%
  12. 12. 2012 50 60 70 80 90 100 110 20 25 30 35 40 45 50 55 60 65 70 FEV1PercentPredicted BMI Percentile 2012 FEV1 vs. BMI Percentile for Patients 6 to 17 Years, 2002 vs. 2012 13.4% 76.4% 4.5% 5.7% 2002 Median Values
  13. 13. CFF Recognized as Model
  14. 14. 2015 Annual Data Report
  15. 15. Patient Registry: Uses
  16. 16. Innovating Research & Development
  17. 17. RDP Centers
  18. 18. Research Publications 0 500 1000 1500 2000 2500 New Publications 1,993
  19. 19. Working Hypothesis
  20. 20. Danny Then and Now
  21. 21. Cystic Fibrosis Transmembrane Conductance Regulator • Acts as a Chloride Channel • Controls Salt and Water Balance in the Airways
  22. 22. Therapeutics Development Program (TDP) Started in 1998 • Created to encourage industry and academia to focus on CF and CFTR as drug target • Components of TDP – Financial assistance – Research tools and scientific advice – Well organized clinical trial network Venture Philanthropy
  23. 23. TDP Process Available to CF Patients DISTRIBUTION FDA P P R O V A L DEVELOPMENT TDN Bring Existing Drugs for CF Indication NDA Clinical Dosage and Efficacy IND Preclinical Safety Testing Therapeutics Development Awards DISCOVERY Basic Research • CFTR Corrector Consortium • CFTR Structural Consortium • Mucociliary Clearance Consortium High-throughput Screening The CF Foundation has “really paved the way for other small disease nonprofits to take drug discovery into their own hands.” -Harvard Business School Professor Robert Higgins
  24. 24. Therapeutics Development Network
  25. 25. Highlights of CF TDN Studies (1998-2016) • Network supports 25-35 studies annually: – 75% industry-based therapeutic trials – 25% PI-initiated • Initial studies were early Phase (1 and 2) • Transition to larger Phase 3 trials – First CFFT Phase 3 study in 2001 – Industry moved to predominantly Phase 3 by 2009 • PI initiated studies have focused on outcome measure development • In total, over 150 studies involving thousands of individuals with CF have been successfully completed.
  26. 26. Venture Philanthropy in Action High-throughput Screening
  27. 27. 2012 – FDA Approves Ivacaftor
  28. 28. Ivacaftor Improved FEV1 McKone, Borowitz….Davies et al, NACFC 2013. Poster 207 Ivacaftor vs Placebo Everyone on Ivacaftor
  29. 29. Effect of Decreased Rate of Decline in FEV1 20 30 40 50 60 70 80 90 100 6 16 26 36 46 56 66 76 86 G551D With Ivacaftor F508del/F508del
  30. 30. FDA Approves Lumacaftor/Ivacaftor Combination for F508del Homozygotes
  31. 31. Venture Philanthropy: It is not just the money! It also involves access to: • Expertise • Patients • Clinical Trial Network • Data • Specialized Resources • Committed Community Venture Philanthropy is not for the faint of heart…
  32. 32. Innovating For the Future
  33. 33. A Lifelong Cure For All CF Patients Gene (DNA) CF Mutation Continuous Therapy Infection Mucus Inflammation Nutrition…. RNA Transcription Daily Therapy CFTR modulation Potentiators Correctors Symptoms Lungs liver pancreas gut Protein Translation CFTR Periodic Therapy Transcription Translation (PTCs) RNA replacement** RNA editing Replication Permanent Repair Gene editing Gene delivery Stem cell biology
  34. 34. Innovating for the Future February 2016
  35. 35. Dream Big Continuously refine and replace existing therapies until a safe, effective, permanent cure is available for all individuals with CF.
  36. 36. Thank you.

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