Progress & Directions

Progress & Directions
November 4, 2016
12:15p.m. - 1:30p.m.
1

HSG 2016: DISCOVERING OUR FUTURE
Agenda
• Thank you and Congratulations
• HSG Strategic Plan and Key Accomplishments
– HSG Overview Video
• Future of HD Care
• Future of HD Trials

Welcome
• 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

Founders Society
Platinum
HSG 2016: DISCOVERING OUR FUTURE 4

HSG 2016: DISCOVERING OUR FUTUREHSG 2016: DISCOVERING OUR FUTURE
Founders Society
Silver

Founders Society
Bronze

Make a Difference Society
Ambassador

Advocate

Associate

Thank you to our outgoing
Executive Committee members!
Sam Frank Greg Suter David Oakes

Joni Steinman
Board Member
Welcome to HSG New Board and
Executive Committee Members
Jenna Smith
Executive
Committee Member
Kevin Biglan
Executive
Committee Member
11

Our Leadership Team
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
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
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

Thank you to our Staff
Shari Kinel
Executive Director
Liz McCarthy
Director, Special Programs
Kristin Strazdins
Accounting & Office Manager
Stevan Ramirez
Director, Finance & Operations
Heather Hare
Director, Communications &
Outreach

Thank you to our Education Committee
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)

Congratulations
Jody Corey-Bloom
HSG Jack Penney
Investigator Award
2016
Paula Wasserman
Outstanding Coordinator
Award
2016
Elise Kayson
Lifetime Achievement
2016

Congratulations,
Shoulson Scholars!
• Nora Fritz, Wayne State
University
• Martin Kronenbuerger,
Johns Hopkins University

Congratulations, HD Human Biology
Project Scholars!
• Dawn Loh, University of
California, Los Angeles
• Regina Kim,
University of Iowa
• Sonia Podvin, University
of California, San Diego

Welcome New HSG Sites in 2016
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.

Overview Video

2016 Key Accomplishments: Trials/Studies

• 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

• Pride-HD Results
– Results recently released reporting slowing of the
decline in Total Functional Capacity

• 2Care Data publicly available
www.huntingtonstudygroup.org/2care/

• Launched Seed Fund
– Up to two $10,000 seed funds to support clinical
trials!
– www.huntingtonstudygroup.org/seed-fund-
projects

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

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

Outline
28
• Future of HD care
• Future of HD clinical trials

Outline
29

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

…and remote education
31
HSG produced informational videos to disseminate HD knowledge
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

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

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

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
Jack Griffin
The Griffin Foundation
Karen E. Anderson
MD

Outline
35

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

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

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

“I am the DIFFERENCE” Campaign expands HSG
engagement with families

Future of HD Trials
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

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
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.

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

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

Other HD biomarkers in CSF and blood have
investigative and therapeutic potential
Edward Wild
PhD
Steven M. Hersch
MD, PhD
Source: hdclarity.net and Rosas et al. Ann Clin Transl Neurol 2015;2:756-68

Source: Reilmann et al. Movement Disorders 2015;30:427-31
Objective tools for symptom assessment improve
certainty of results
Q-Motor
Ralf Reilmann
MD

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)

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

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

Keynote Speech
Dr. Bob Beall,
former CEO of the Cystic Fibrosis Foundation
5:00 pm Friday
Presidential AB
50

Keynote Reception
6:00 pm
Friday
Delta C
With special musical guests: Branch & Dean!
51

HSG 2016: DISCOVERING OUR FUTUREHSG 2016: DISCOVERING OUR FUTURE
HSG 2017
Nov. 1-4
Denver,
Colorado

Thank you!
Please fill out the
session survey in
Grupio.
53

Progress & Directions

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