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The CFF Story:Innovating for a Cure
Huntington Study GroupNovember 4, 2016
Robert J. Beall, Ph.D.Former President and CEO, Cystic Fibrosis Foundation
The CFF Story:Innovating for a Cure
Community Clinical Care Research Discovery & Development Treatment
Median Predicted Survival Age of US Patients with Cystic Fibrosis
Med
ian
Pre
d. S
urvi
val A
ge
Source: Cystic Fibrosis Foundation, National Patient Registry
1940 1950 1960 1970 1980 1990 2000 2010 20150
5
10
15
20
25
30
35
40
45 41
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
% o
f CF
indi
vidu
als
over
the
age
of 1
8
1965 1970 1975 1980 1985 1990 1995 2000 2005 2010 2015
0
10%
20%
30%
40%
50%
46%
Other Indications of Success
22%
CF Adults Employed
CF Adults Attending College
InnovatingCommunity
Academics• Research• Care• Training
Biopharmaceutical• Translation• Commercialization
CF Foundation• Care• Research• Education
Ecosystem for Cystic Fibrosis Foundation Success
FDANIH
InnovatingCare
Care Center Network
Care Center Network
Peer accreditation• 119 care centers,
• 107 adult programs,
• 51 affiliates
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
2002
20 25 30 35 40 45 50 55 60 65 7050
60
70
80
90
100
110
BMI Percentile
FEV1
Per
cent
Pre
dict
ed
FEV1 vs. BMI Percentile for Patients 6 to 17 Years, 2002 vs. 2012
32.2%17.5%
18.9%
2002 Median Values
31.5%
2012
20 25 30 35 40 45 50 55 60 65 7050
60
70
80
90
100
110
2012
BMI Percentile
FEV1
Per
cent
Pre
dict
ed
FEV1 vs. BMI Percentile for Patients 6 to 17 Years, 2002 vs. 2012
13.4%
76.4%
4.5% 5.7%
2002 Median Values
CFF Recognized as Model
2015 Annual Data Report
Patient Registry: Uses
InnovatingResearch & Development
RDP Centers
Research Publications
20142013
20122011
20102009
20082007
20062005
20042003
20022001
20001999
19981997
19961995
19941993
19921991
19901989
19881987
19861985
19841983
19821981
0
500
1000
1500
2000
2500
New Publications 1,993
Working Hypothesis
Danny Then and Now
Cystic Fibrosis Transmembrane Conductance Regulator
• Acts as a Chloride Channel
• Controls Salt and Water Balance in the Airways
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
TDP Process
AvailabletoCF
Patients
DISTRIBUTION
FDAPPROVAL
DEVELOPMENT
TDN
Bring Existing Drugs for CF
Indication
NDAClinical
Dosageand
Efficacy
INDPreclinica
lSafetyTesting
TherapeuticsDevelopment
AwardsDISCOVERY
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
Therapeutics Development Network
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.
Venture Philanthropy in Action
High-throughput Screening
2012 – FDA Approves Ivacaftor
Ivacaftor Improved FEV1
McKone, Borowitz….Davies et al, NACFC 2013. Poster 207
Impr
ovem
ent i
n Lu
ng F
unct
ion
(FE
V1
%)
Ivacaftor vs Placebo
Everyone on Ivacaftor
Effect of Decreased Rate of Decline in FEV1
6 16 26 36 46 56 66 76 8620
30
40
50
60
70
80
90
100
G551D With Ivacaftor
F508del/F508del
FDA Approves Lumacaftor/IvacaftorCombination for F508del Homozygotes
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…
InnovatingFor the Future
A Lifelong Cure For All CF Patients
Gene (DNA)
CF Mutation
ContinuousTherapyInfectionMucusInflammationNutrition….
RNA
Transcription
DailyTherapy CFTR modulationPotentiatorsCorrectors
Symptoms
Lungs
liver
pancreas
gut
Protein
Translation
CFTR
PeriodicTherapy TranscriptionTranslation (PTCs)RNA replacement**RNA editing
Replication
PermanentRepair Gene editingGene deliveryStem cell biology
Innovating for the Future
February 2016
Dream Big
Continuously refine and replace existing therapies until a safe, effective, permanent cure is available for all
individuals with CF.
Thank you.