The OPT-UP Study. Hollie Schmidt Accelerated Cure Project for MS November 29, 2012

Transcription

1 The OPT-UP Study Hollie Schmidt Accelerated Cure Project for MS November 29, 2012

2 Overview 1 study: OPT-UP 2 goals: Optimize Treatment, Understand Progression 10 lead partners: ACP and 9 clinical centers 2,500 subjects; 2.5 million beneficiaries $25M initial cost; several $B potential impact

3 Why MS?

4 MS is a lifelong disabling disease Most disabling neurological disease among young adults; affects 400K in the US, 2.5M worldwide Its effects are immune-mediated, but the causes and mechanisms are unknown Disability accrues both through relapses and gradual progression Treatments are available, but only for relapsing-remitting MS, and none are cures

5 The human costs of MS are massive MS causes blindness, paralysis, incontinence, depression, cognitive decline, fatigue, and more MS increases the likelihood of unemployment and divorce The effects of MS are unpredictable, resulting in anxiety and loss of control

6 The financial costs of MS are also great Estimated annual costs per person in US: $50K Estimated total annual costs in US: $28B Costs affected by #/severity of relapses, disability Professional home care, $883 Hospitaliza ons, $1,000 Informal care, $4,957 Other prescrip ons, $2,639 All other costs, $9,281 DMDs, $17,244 Early re rement, $14,704

7 What needs to be done right now? And why is now a good time to do it?

8 Unmet need #1: Tx optimization Prescription by trial and error; treatment failures and switches are common Personalized medicine = opportunity to preserve the CNS, prevent disability, and save jobs, money, and marriages Simultaneous opportunity to learn about MS; use treatments as perturbagens

9 Unmet need #2: Stopping progression Progressive MS is more disabling than RRMS and does not respond to RRMS therapies Why not? Two possibilities: Different disease from RRMS Loss of neuronal reserve + aging Knowing the basis of progressive MS would open the door to slowing/stopping it

10 Who is going to address these needs and how?

11 A cure-driven nonprofit Accelerated Cure Project is a nonprofit dedicated to accelerating advances toward a cure for MS In 10 years we have: Completed a comprehensive review of MS causes; Created a 3,000-subject, 65-study biorepository; and Launched an acclaimed information portal and online community for MS researchers (MS Discovery Forum)

12 A strong & focused partnership In 2011 we formed the Clinical Effectiveness Study Network with investigators at 9 leading MS clinics CESN s purpose is to conduct multi-site studies on critical MS topics beginning with the OPT-UP study Stanford Stanford, CA University of Colorado Aurora, CO UT Southwestern Dallas, TX UMass Memorial Worcester, MA Ohio State Columbus, OH Johns Hopkins Baltimore, MD Shepherd Center Atlanta, GA Beth Israel Deaconess Boston, MA MSRCNY New York, NY

13 The OPT-UP study CESN will address both unmet needs through a single prospective study involving: Enrollment of 2,500 people with relapsing and progressive MS Collection of biosamples and data over 5+ years Analysis of samples and data (within CESN and with defined external partners) Implementation of results for real-world impact

14 Sample/data collection plan 5 years + Enrollment: 2,500 PwMS -- Cohorts for each approved DMT -- PPMS and SPMS cohorts Clinical assessments of walking, cognition, dexterity, vision; record abstraction Imaging: MRI Biosample collection: Blood, urine, CSF Patient-reported outcomes: Symptoms, socioeconomic, disease impact, QoL, exercise, social network,

15 Sample/data collection plan 5 years + Enrollment: 2,500 PwMS -- Cohorts for each approved DMT -- PPMS and SPMS cohorts Treatment changes Relapses Development of SPMS Clinical assessments of walking, cognition, dexterity, vision; record abstraction Imaging: MRI Biosample collection: Blood, urine, CSF Patient-reported outcomes: Symptoms, socioeconomic, disease impact, QoL, exercise, social network, side effects, comorbidities

16 Sample/data analysis aims Multivariate predictors of treatment outcomes Biomarker analyses Clinical features Imaging measurements Treatment outcomes Data mining Multivariate analysis Bioinformatics Clinical assays Clinical decision support systems Validated biomarkers (or lack thereof) of progressive MS Drug discovery/treatment research for progressive MS

17 What do we need to succeed in our mission?

18 Partnerships are key to success Clinical research sites (more may be needed) Clinical research suppliers Biopharma companies (funding and expertise) MS patients (Community Advisors) & organizations Path to Impact partners: Bioanalysis Data analysis & interpretation Image analysis Assay development & commercialization Clinical support system development

19 Accomplishments & milestones Infrastructure partly in place Supplier relationships established Outcomes being evaluated Community advisory panel being established Path to Impact partners being enlisted Launch in 1H13 upon initial funding round

20 Budget and funding sources Estimated budget: $25M for five years Funding sources: MS biopharma companies Individual donors Foundations Government agencies (PCORI)

21 How will OPT-UP change the outlook for people with MS?

22 People with relapsing-remitting MS will get on the right treatment, right away. They will have better health and be better off. Some may go into long-term remission. People with progressive MS will finally have reason to hope, because we will have answered long-standing questions about the fundamental nature of their illness.

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