The Accelerated Cure Project for MS

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

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

3. 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.

4. How to Accelerate→Cure?: Research ? Research Community $ Impact Publications Accelerate by doing more of the same Research Community $ $ Publications $ $ ? Impact on Patients

5. No Increase in Traditional MS Research Support NIH National MS Society Numbers are $MM

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

7. Increase in Pharma/Biotech’s Interest in MS Research Numbers are $BN MS market has grown by ~80% in last 5 years 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

10. INNOVATION

12. T H E 21st C E N T U R Y M S C A F E JOIN IN SHARE MS Discovery Forum open since 2012

13. “Never doubt that a small group of thoughtful, committed citizens can change the world. Indeed, it is the only thing that ever has.”

14. The ACP Repository: Initiated in 2006 Subjects enroll at 10 participating MS clinics across the US, donating blood samples and data Beth Israel Deaconess Boston, MA UMass Memorial Worcester, MA University of Colorado Aurora, CO Stanford Stanford, CA Ohio State Columbus, OH MSRCNY New York, NY Johns Hopkins Baltimore, MD UT Southwestern Dallas, TX Barrow Phoenix, AZ Shepherd Center Atlanta, GA Bio-samples and data currently available from ~2800 subjects; each sample can support hundreds of different studies

15. 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

16. Efficiently Enabling “Squeaky Mice*” to ROAR EUREKA! ACP REPOSITORY *Small groups with novel ideas for research in MS

17. COLLABORATION MS Clinical Network OneMind Bionetworks

19. 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

21. Thank You