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 and Productivity of 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 2 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 4 $ Research Community Research Community Publications Impact $ $ $ $ Publications Impact on Patients ? ? Accelerate by doing more of the same

No Increase in Traditional MS Research Support 5 Area inc. ARRA 2010 inc. ARRA 2011 est est. Multiple Sclerosis Neuroscience Schizophrenia NIH National MS Society Multiple Sclerosis ?? Numbers are $MM

How to Accelerate→Cure?: Translation 6 $ Research Community Publications Impact Focus on Enabling Translation Pharma/biotech’s primary area of focus

Increase in Pharma/Biotech’s Interest in MS Research 7 Numbers are $BN If R&D expenditure is 15-20% of sales (CBO, 2006), then an estimate of MS 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 21 st C E N T U R Y M S C A F E JOIN IN SHARE MS Discovery Forum 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 Hopkins Baltimore, MD Beth Israel Deaconess Boston, MA Shepherd Center Atlanta, GA UT Southwestern Dallas, TX Barrow Phoenix, AZ MSRCNY New York, NY Ohio State Columbus, OH University of Colorado Aurora, CO Stanford Stanford, CA UMass Memorial Worcester, MA The ACP Repository: Initiated in 2006 Subjects enroll at 10 participating MS clinics across the US, donating blood samples and data 14 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 15

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 Q 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 19

21 Thank You