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Progress & Directions

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Progress & Directions, HSG 2016, presented by Ray Dorsey, chair of HSG

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Progress & Directions

  1. 1. Progress & Directions November 4, 2016 12:15p.m. - 1:30p.m. 1
  2. 2. HSG 2016: DISCOVERING OUR FUTURE Agenda HSG 2016: DISCOVERING OUR FUTURE • Thank you and Congratulations • HSG Strategic Plan and Key Accomplishments – HSG Overview Video • Future of HD Care • Future of HD Trials
  3. 3. HSG 2016: DISCOVERING OUR FUTURE Welcome HSG 2016: DISCOVERING OUR FUTURE • Thanks to you, HSG 2016 has record breaking attendance: • 452 registrants – 72 CME4HD registrants – 39 Scholarship recipients – 19 Advocacy Organization Representatives – 66 Industry Sponsors and Partners Representatives – 120 HD Community Members
  4. 4. Founders Society Platinum HSG 2016: DISCOVERING OUR FUTURE 4
  5. 5. HSG 2016: DISCOVERING OUR FUTUREHSG 2016: DISCOVERING OUR FUTURE Founders Society Silver
  6. 6. Founders Society Bronze HSG 2016: DISCOVERING OUR FUTURE 6
  7. 7. HSG 2016: DISCOVERING OUR FUTURE Make a Difference Society Ambassador HSG 2016: DISCOVERING OUR FUTURE
  8. 8. Make a Difference Society Advocate HSG 2016: DISCOVERING OUR FUTURE 8
  9. 9. Make a Difference Society Associate HSG 2016: DISCOVERING OUR FUTURE 9
  10. 10. HSG 2016: DISCOVERING OUR FUTURE Thank you to our outgoing Executive Committee members! HSG 2016: DISCOVERING OUR FUTURE Sam Frank Greg Suter David Oakes
  11. 11. Joni Steinman Board Member HSG 2016: DISCOVERING OUR FUTURE Welcome to HSG New Board and Executive Committee Members Jenna Smith Executive Committee Member Kevin Biglan Executive Committee Member 11
  12. 12. HSG 2016: DISCOVERING OUR FUTURE Our Leadership Team HSG 2016: DISCOVERING OUR FUTURE Ray Dorsey, MD Chair University of Rochester Blair Leavitt, MD, CM, FRCP(C) Co-Chair University of British Columbia Julie Stout, PhD Treasurer Monash University Shari Kinel, JD Secretary Huntington Study Group Joni Steinman Board Member Huntington Study Group David Oakes, PhD University of Rochester Mike Poole, MD Bill & Melinda Gates Foundation Ralf Reilmann, MD, PhD George-Huntington-Institute Jenna Smith University of Alabama Birmingham Greg Suter, BA Hereditary Neurological Disease Center Kevin Biglan, MD University of Rochester Sam Frank, MD Beth Israel Deaconess Medical Center Jack Griffin Griffin Foundation Elise Kayson, MS, RNC Center for Human Experimental Therapeutics Martha Nance, MD University of Minnesota/ Struthers Parkinson’s Center
  13. 13. HSG 2016: DISCOVERING OUR FUTURE Thank you to our Staff HSG 2016: DISCOVERING OUR FUTURE Shari Kinel Executive Director Liz McCarthy Director, Special Programs Kristin Strazdins Accounting & Office Manager Stevan Ramirez Director, Finance & Operations Heather Hare Director, Communications & Outreach
  14. 14. HSG 2016: DISCOVERING OUR FUTURE Thank you to our Education Committee HSG 2016: DISCOVERING OUR FUTURE Karen Anderson Daniel Claassen Mary Edmondson Jody Goldstein Lisa Hale Bonnie Hennig Liz McCarthy Martha Nance Jenna Smith Victor Sung Lauren Seeberger (Chair) Elise Kayson (Co-Chair)
  15. 15. HSG 2016: DISCOVERING OUR FUTURE Congratulations HSG 2016: DISCOVERING OUR FUTURE Jody Corey-Bloom HSG Jack Penney Investigator Award 2016 Paula Wasserman Outstanding Coordinator Award 2016 Elise Kayson Lifetime Achievement 2016
  16. 16. HSG 2016: DISCOVERING OUR FUTURE Congratulations, Shoulson Scholars! HSG 2016: DISCOVERING OUR FUTURE • Nora Fritz, Wayne State University • Martin Kronenbuerger, Johns Hopkins University
  17. 17. HSG 2016: DISCOVERING OUR FUTURE Congratulations, HD Human Biology Project Scholars! HSG 2016: DISCOVERING OUR FUTURE • Dawn Loh, University of California, Los Angeles • Regina Kim, University of Iowa • Sonia Podvin, University of California, San Diego
  18. 18. HSG 2016: DISCOVERING OUR FUTURE Welcome New HSG Sites in 2016 HSG 2016: DISCOVERING OUR FUTURE 1. Sanford Health • Tanya Harlow 2. Banner Sun Health Institute • David Shprecher 3. Methodist Neurological Institute • Tetsuo Ashizawa • William Ondo 1. 2. 3. 4. University of Buffalo • Kelly Andrzejewski 4.
  19. 19. HSG 2016: DISCOVERING OUR FUTURE Overview Video
  20. 20. HSG 2016: DISCOVERING OUR FUTURE 2016 Key Accomplishments: Trials/Studies HSG 2016: DISCOVERING OUR FUTURE
  21. 21. HSG 2016: DISCOVERING OUR FUTURE 2016 Key Accomplishments: Trials/Studies HSG 2016: DISCOVERING OUR FUTURE • Together with Vaccinex, Dr. Andrew Feigin launched the SIGNAL trial. – Enrolled prodromal patients – First monoclonal antibody and first infusion study for HD – Enrollment in Cohort A was completed two months early – Cohort B completed one month early, and enrollment is complete – Retention remains high
  22. 22. HSG 2016: DISCOVERING OUR FUTURE 2016 Key Accomplishments: Trials/Studies HSG 2016: DISCOVERING OUR FUTURE • Pride-HD Results – Results recently released reporting slowing of the decline in Total Functional Capacity
  23. 23. HSG 2016: DISCOVERING OUR FUTURE 2016 Key Accomplishments: Trials/Studies HSG 2016: DISCOVERING OUR FUTURE • 2Care Data publicly available www.huntingtonstudygroup.org/2care/
  24. 24. HSG 2016: DISCOVERING OUR FUTURE 2016 Key Accomplishments: Trials/Studies HSG 2016: DISCOVERING OUR FUTURE • Launched Seed Fund – Up to two $10,000 seed funds to support clinical trials! – www.huntingtonstudygroup.org/seed-fund- projects
  25. 25. HSG 2016: DISCOVERING OUR FUTURE o Develop and evaluate sensitive objective measures of HD o Expand capabilities of the HSG research network o Improve efficiency of conducting clinical trials HSG Strategic Plan 2015 - 2019 o Investigate new therapies for HD o Expand the pipeline of therapies in development for HD o Investigate, communicate and educate about HD research and care Families Members To be an invaluable research partner to families, sponsors and members o Identify & develop the next generation of investigators o Build and develop existing human capital o Foster community Sponsors
  26. 26. HSG 2016: DISCOVERING OUR FUTURE
  27. 27. HSG 2016: DISCOVERING OUR FUTURE Make HD an Increasingly Treatable Condition Objectives for the next 5 years: 1) Support the development of 2 FDA approved treatments for HD 2) Engage 1-2 additional sponsors per year in the development of novel therapies for HD 3) Train 20-50 investigators and clinicians per year in the care of HD 4) Foster the development of objective measure(s) of disease progression for us in clinical trials 5) Create scholarship program for junior investigators, including HDSA Human Biology recipients 6) Create new opportunities for professional development for all our members
  28. 28. HSG 2016: DISCOVERING OUR FUTURE Outline 28 • Future of HD care • Future of HD clinical trials HSG 2016: DISCOVERING OUR FUTURE
  29. 29. HSG 2016: DISCOVERING OUR FUTURE Outline 29 • Future of HD care • Future of HD clinical trials HSG 2016: DISCOVERING OUR FUTURE
  30. 30. HSG 2016: DISCOVERING OUR FUTURE We are increasingly making HD education available for clinicians 30 Continuing Medical Education (CME) aims to increase clinician knowledge of HD Mary Edmondson MD, HD Reach HSG 2016: DISCOVERING OUR FUTURE
  31. 31. HSG 2016: DISCOVERING OUR FUTURE …and remote education 31 HSG produced informational videos to disseminate HD knowledge HSG 2016: DISCOVERING OUR FUTURE Video link: https://goo.gl/FZdttL 0 500 1000 1500 2000 2500 3000 3500 4000 4500 5000 Family Planning (Martha Nance) HSG's Role in Research (Lavonne Goodman) New Treatments (Ira Shoulson) HD Research Progress (Anne Young) HD Niche (Daniel Claassen) Late Stage (Victor Sung) Facebook Views
  32. 32. HSG 2016: DISCOVERING OUR FUTURE Good care can make a difference Source: Modified from The Cystic Fibrosis Foundation Annual Report, 2006 and CFF website Robert Beall PhD, CFF 1 10 16 18 29 32 35.1 36.9 37.4 0 5 10 15 20 25 30 35 40 1950 1960 1970 1980 1990 2000 2004 2006 2007 Median Predicted Age of Survival for Individuals with Cys tic Fibrosis, 1950-2007 5HSG 2016: DISCOVERING OUR FUTURE
  33. 33. HSG 2016: DISCOVERING OUR FUTURE Dr. Kevin Biglan has crafted a novel care model for Parkinson Disease Supported by: Greater Rochester Health Foundation & the Safra Foundation Who: Any New Yorker with Parkinson disease What: Multidisciplinary care + optional use of smartphone to track disease When: May 2016 Where: New York state, especially the 9 counties surrounding Rochester Why: To provide comprehensive care to residents of New York state, especially the underserved How much: Free 33 To learn more: Visit www.pdcny.org HSG 2016: DISCOVERING OUR FUTURE
  34. 34. HSG 2016: DISCOVERING OUR FUTURE To learn from others & you, Jack Griffin is supporting an initiative to improve HD care Goal - To assess, develop, and execute a set of initiatives to improve quality of care, access to care, and patient and caregiver outreach Phase I: Assess • Understand current care delivery by HD Centers of Excellence (COEs) • Identify means to increase access to care for families affected by HD • Identify funding sources to deploy new care models Phase II: Plan and Execute • Help stakeholders evaluate and prioritize alternatives and costs • Define ideal coordinated care model for HD COEs and expand care capacity • Tighten linkages between clinical trials and care HSG 2016: DISCOVERING OUR FUTURE Jack Griffin The Griffin Foundation Karen E. Anderson MD
  35. 35. HSG 2016: DISCOVERING OUR FUTURE Outline 35 • Future of HD care • Future of HD clinical trials HSG 2016: DISCOVERING OUR FUTURE
  36. 36. HSG 2016: DISCOVERING OUR FUTURE Future trials will incorporate new tools that can extend the research of academic centers 36 Source: JAMA Neurol. 2015 72(5):582-8. Characteristics of 20th vs 21st century clinical trials HSG 2016: DISCOVERING OUR FUTURE
  37. 37. Need for new HD recruitment model Industrial Model Post-Industrial Model Objective: Recruit in a Day - Innovating trial recruitment process Subjects Pharma CRO Site Site Site Pharma, HSG, and Investigators Community and Participants HSG 2016: DISCOVERING OUR FUTURE 37
  38. 38. Targeted recruitment strategies directly engage the community Streamlined methodology: • Social targeted study recruitment for LRRK2 genetic mutation 33% increase in volunteers enrolled in PPMI studies • Recruitment cost per individual dropped from $800 to $7-$24 • Make traditionally limited tools freely available to researchers 38 Michael J. Fox Foundation accelerates recruitment for PPMI clinical studies HSG 2016: DISCOVERING OUR FUTURE
  39. 39. HSG 2016: DISCOVERING OUR FUTURE “I am the DIFFERENCE” Campaign expands HSG engagement with families HSG 2016: DISCOVERING OUR FUTURE
  40. 40. Future of HD Trials HSG 2016: DISCOVERING OUR FUTURE 2006: Formed first study steering committee with HD family member (2CARE) 2007: Shared study results with participants (TREND) 2010: Formed focus group of patients and caregivers 2010: Surveyed >600 patients and caregivers for Project AWARE 2016: Conducted family advisory board with Pharmaceutical Company Sponsor (RAPTOR) to provide input on protocol 2017: Recruit in a Day
  41. 41. Emergence of quantitative disease models augment clinical trial planning Source: Holford et al., Venuto et al., Atchison et al., summary by Charles Venuto 41 0 2 4 6 8 10 12 1990 - 1999 2000-2009 2010-2016 #ofmodels Years Epilepsy HD PD AD HSG 2016: DISCOVERING OUR FUTURE Modeling and simulation offers the ability to test, modify, and re-test design choices Charles Venuto PharmD Karl D. Kieburtz MD, MPH If you have interest and statistical expertise, we need your help.
  42. 42. HSG 2016: DISCOVERING OUR FUTURE Virtual visits can bring trials to the participants instead of participants to the trials Map of participants reached via virtual research visits 42 Source: Journal of Parkinson's Disease, 2015; 5: 505-515 HSG 2016: DISCOVERING OUR FUTURE
  43. 43. HSG 2016: DISCOVERING OUR FUTURE Perhaps most important is the need for objective measures for HD TRACK-HD demonstrates longitudinal changes in HD biomarkers 43 Source: Tabrizi et al. The Lancet Neurology 2012;10:31-42 Sarah Tabrizi PhD, FRCP HSG 2016: DISCOVERING OUR FUTURE
  44. 44. HSG 2016: DISCOVERING OUR FUTURE Other HD biomarkers in CSF and blood have investigative and therapeutic potential Edward Wild PhD HSG 2016: DISCOVERING OUR FUTURE Steven M. Hersch MD, PhD Source: hdclarity.net and Rosas et al. Ann Clin Transl Neurol 2015;2:756-68
  45. 45. Source: Reilmann et al. Movement Disorders 2015;30:427-31 Objective tools for symptom assessment improve certainty of results Q-Motor Ralf Reilmann MD HSG 2016: DISCOVERING OUR FUTURE 45
  46. 46. HSG 2016: DISCOVERING OUR FUTURE We also need a new class of measurements that can provide high frequency data 46 Source: Modified from Social Physics: How Social Networks can Make Us Smarter, Alex Pentland (2015) HSG 2016: DISCOVERING OUR FUTURE
  47. 47. HSG 2016: DISCOVERING OUR FUTURE Novel digital health measures can assess the 99% of time patients are not in the clinic 47 ObjectiveHD sensor data reveals significance in at-home data not seen in clinic Source: Andrzejewski et al. Journal of Huntington’s Disease 2016;5:199-206 HSG 2016: DISCOVERING OUR FUTURE
  48. 48. HSG 2016: DISCOVERING OUR FUTURE The signal from the smartphone sensors could be quite sensitive in response to drugs Sample accelerometry tracings in an individual with Parkinson disease pre- and post-levodopa 48 Withdrawn participant Active participant Confidential HSG 2016: DISCOVERING OUR FUTURE
  49. 49. Keynote Speech HSG 2016: DISCOVERING OUR FUTURE Dr. Bob Beall, former CEO of the Cystic Fibrosis Foundation 5:00 pm Friday Presidential AB 50
  50. 50. Keynote Reception HSG 2016: DISCOVERING OUR FUTURE 6:00 pm Friday Delta C With special musical guests: Branch & Dean! 51
  51. 51. HSG 2016: DISCOVERING OUR FUTUREHSG 2016: DISCOVERING OUR FUTURE HSG 2017 Nov. 1-4 Denver, Colorado
  52. 52. Thank you! Please fill out the session survey in Grupio. 53

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