CONQUERING ALZHEIMER’S DISEASE HOPE ON THE HORIZON Howard Fillit, MD | Executive Director & Chief Science Officer All Slides © 2015 Alzheimer’s Drug Discovery Foundation. Not for distribution.
WHAT WE ARE FACING
2
$226 billion annual cost in U.S.
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28 million Baby Boomers expected to develop Alzheimer’s
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ADDF’S APPROACH
5
HOW ADDF IS UNIQUE
% RESEARCH FUNDING BY AREA
Drug Discovery & Clinical Trials Molecular Pathogenesis Diagnosis & Assessment Epidemiology Care & Support
6
HOW ADDF IS UNIQUE % of total budget dedicated to drug discovery, clinical trials & diagnostics
ADDF 95%
$
7.3
MILLION
Alzheimer’s Association 5.6%
$
6.8
MILLION
Cure Alzheimer’s Fund 10.2%
$
650,000 7
WHAT WE KNOW: Our Strategy
8
AGING
IS THE LEADING RISK FACTOR FOR ALZHEIMER’S DISEASE
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Genetics & Epigenetics
Neuronal Energy Failure
Vascular Disease
Oxidation
Inflammation
NEURONAL DYSFUNCTION AND DEATH
Damaged Proteins
ALZHEIMER’S DEMENTIA
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PORTFOLIO OF DRUG TARGETS
2014 ADDF Investments
EPIGENETICS & OTHER
11
12
+
YEARS
$
2
BILLION
12
REASONS FOR HOPE: Clinical Trials
ADDF FUNDING BY STAGE OF DISCOVERY & DEVELOPMENT
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ADDF FUNDING BY STAGE OF DISCOVERY & DEVELOPMENT
Preclinical
Clinical
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1999-2009
86.6%
13.4%
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2015 YTD
54.6%
45.4%
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120
+
Treatments Now in Clinical Trials
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20
%
ADDF Supported
19
AMYVID ACCELERATING TRIALS
PLACEBO TREATMENT
VACCINE TREATMENT
Has enabled more scientifically rigorous clinical trials • Disease starts ~20 years before symptoms begin • Anti-amyloid prevention clinical trials are underway
from Biogen study of Aducanumab 20
ADDF NOVEL DRUGS IN CLINICAL TRIALS
Neuroprotection Oxidation
Neuroendocrine aging
Systemic Inflammation
Failed Mitochondrial Energetics dysfunction
Epigenetic changes
Hypoxia
Neuroinflammation
BBB dysfunction
CELL DEATH
CELL SURVIVAL Misfolded Proteins
Neuroprotection
Frank Longo, MD, PhD Drug: LM11A-31 Phase 2 Clinical Trial
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ADDF FUNDING OF PHARMATROPHIX
2000-2004 $521,517
2004-2005 $160,177
2005-2009 $195,000
2008-2009 $300,000
2013-2014 $250,000
2015 $500,000
2009-2010 $180,000 24
Neuroinflammation
Healthy Control
2007
Alzheimer’s
Neuroinflammation
Philip Haydon, PhD
Paolo Pevarello, PhD
GliaCure, Boston
Axxam SpA, Milan
Drug: GC021109 Phase 1 Clinical Trial
Drug: AXX00179871 Preclinical 26
Epigenetics 27
Epigenetics
Tamara Maes, MD Oryzon Genomics, Barcelona Drug: ORY-2001 Preparing for Phase 1
28
REPURPOSING FDA-APPROVED DRUGS
3
+
YEARS
$
2
MILLION
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Neuroprotection
Ana Pereira, MD
Michela Gallagher, PhD
The Rockefeller University
John Hopkins University & Agenebio
Drug: Riluzole Phase 2 Clinical Trial
Drug: AGB10 Phase 3 Clinical Trial 30
Neuroinflammation
Clive Holmes, PhD University of Southampton, UK Drug: Etanercept Phase 2 Clinical Trial
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Neuronal Energy Failure
Paul Edison, MD, PhD
Jeffrey L. Cummings, MD
Imperial College London
The Cleveland Clinic
Drug: Liraglutide Phase 2 Clinical Trial
Drug: Rasagiline Phase 2 Clinical Trial 32
TOOLS FOR PREVENTION
Preventing Alzheimer’s Disease Genetics & Gender
Environment
Substance Abuse
Head Trauma
Exercise
Medical Risk Factors
Major Surgery
Psychological Factors
Diet
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Rating the Evidence for:
• Nutrition, Natural Products, and Supplements • Health Management & Drugs • Potential Risks
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WE CAN CONQUER ALZHEIMER’S
Howard Fillit, MD
HFillit@AlzDiscovery.org
212.901.8000
All Slides © 2015 Alzheimer’s Drug Discovery Foundation. Not for distribution.