The Accelerated Cure Project for MS 1 Suffolk University November 7 th, 2011 Robert N. McBurney, Chief Executive Officer A New Model to Speed the Pace.

The Accelerated Cure Project for MS

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Suffolk UniversityNovember 7th, 2011

Robert N. McBurney, Chief Executive Officer

A New Model to Speed the Pace and Productivityof Multiple Sclerosis Research

Multiple Sclerosis • Degenerative disease of the brain and spinal cord

• Relapsing-remitting initially, then progressive disability

Current Prospects for Patients

• Treatments can prevent relapses (8 drugs approved) at >$80,000 per relapse prevented

BUT

• Current drugs do not arrest progressive disability

• Treatment strategy for each patient involves trial and error

• Few guidelines to help physicians treat comorbidities

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We simply need to know more about the disease(s)

What’s Needed from Research • Comprehensive understanding of the causes and

mechanisms of the disease(s)

• Markers and mechanisms that define, or are associated with, different MS disease characteristics

• Understanding the relationships between MS disease mechanisms and current drug mechanisms

• Identification of novel targets for drug discovery that focus on specific MS disease characteristics

• Markers and methods for predicting the course of MS in an individual patient, including responses to treatments

• More, much more.3

How to Accelerate→Cure?: Research

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$ ResearchCommunity

ResearchCommunity

Publications Impact

$

$$

$

Publications

Impacton Patients

?

?

Accelerate by doing more of the same

No Increase in TraditionalMS Research Support

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Area 2007 20082009inc.

ARRA

2010inc.

ARRA

2011est.

2012est.

MultipleSclerosis

149 169 162 151 133 135

Neuroscience 5102 5224 6168 6309 5513 5612

Schizophrenia 220 249 350 339 276 282

NIH

National MS Society Multiple

Sclerosis51 45 36 37 ? ?

Numbers are $MM

How to Accelerate→Cure?: Translation

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$ ResearchCommunity Publications Impact

Focus onEnabling Translation

Pharma/biotech’s primary area of focus

Increase in Pharma/Biotech’sInterest in MS Research

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Numbers are $BN

If R&D expenditure is 15-20% of sales (CBO, 2006), then an estimate ofMS R&D in pharma/biotech in 2010 is $855-1,140MM - but mostly in D

MS market has grown by ~80% in last 5 years

INNOVATION

T H E 21T H E 21stst C E N T U R Y M S C A F E C E N T U R Y M S C A F E

JOIN IN

SHAREMS DiscoveryForum

open since 2012

“Never doubt that a small group of thoughtful, committed citizens can change the world.

Indeed, it is the only thing that ever has.”

Johns HopkinsBaltimore, MD

Beth Israel DeaconessBoston, MA

Shepherd CenterAtlanta, GA

UT SouthwesternDallas, TX

BarrowPhoenix, AZ

MSRCNYNew York, NY

Ohio StateColumbus, OH

University of ColoradoAurora, CO

StanfordStanford, CA

UMass MemorialWorcester, MA

The ACP Repository: Initiated in 2006Subjects enroll at 10 participating MS clinics across the US,

donating blood samples and data

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Bio-samples and data currently available from ~2800 subjects; each sample can support hundreds of different studies

The ACP Repository: Implementation

• Access given to scientists from any institution worldwide, academic or commercial - streamlined application process

• Benefits to scientists: speed, low cost, flexibility, wide range of subject types, access to other scientists’ results

• Results must be deposited for benefit of all (Open Access)

• 50+ studies supported, 15+ million datapoints returned

• 16 publications, 1 available Dx test, others in development

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EUREKA!EUREKA!

ACP REPOSITORY

*Small groups with novel ideas for research in MS

Efficiently Enabling “Squeaky Mice*” to ROAR

COLLABORATION

MS Clinical Network

OneMind Bionetworks

ACP’s 3-Year Goals

• Guaranteed funding to maintain and enhance MSDF for 5 years post launch

• Double the size of the MS Repository (to 6,000 samples) within 3 years– including multiple longitudinal outcome studies

• Introduce new database with patient portal in Q1 2012

• Broad molecular analyses of selected cohorts to provide comprehensive datasets for bioinformatics

• Catalyze and contribute to MS treatment optimization research towards Clinical Decision Support system

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Thank You