Alternate Approaches to Explore Multi-Dimensional Clinical ......research is about to fundamentally...

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Alternate Approaches to Explore Multi-Dimensional Clinical Space Accelerating open research through provenance and transparency Changing the roles of whom we learn what from when Catalyst March 24, 2015

Transcript of Alternate Approaches to Explore Multi-Dimensional Clinical ......research is about to fundamentally...

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Alternate Approaches to Explore

Multi-Dimensional Clinical Space Accelerating open research

through provenance and transparency Changing the roles of whom we learn what from when

Catalyst March 24, 2015

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maximizing the flow of relevant insights for the widest impact

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time of transition

symptoms to molecular components

molecular components to underlying networks

singular syndromes to variable presentations

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HDAC / AURORA KINASE / TELOMERASE / PD1

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NASCENT TOOLS OF THE LAST DECADE

1- Now possible to generate massive amount of human “omic’s” data 2-Network Modeling Approaches for Diseases are emerging 3- IT Infrastructure and Cloud compute capacity allows a generative open approach to biomedical problem solving 4- Uncoupling the linkage between those that generate data and those that analyze data 5-Nascent Movement for patients to generate and share their data 6- Open Social Media allows citizens and experts to use gaming to solve problems

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We focus on a world where biomedical research is about to fundamentally change. We think it will be often conducted in an open, collaborative way where teams of teams far beyond the current guilds of experts will contribute to making better, faster, relevant discoveries

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incentives and rewards

sharing among pre-defined researchers

Synapse- within defines Communities

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Two approaches to building common scientific knowledge

Text summary of the completed project Assembled after the fact

Every code change versioned Every issue tracked Every project the starting point for new work All evolving and accessible in real time Social Coding

github

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Goal is for Synapse is to function as a github for Biomedical Data

• Data and code versioned • Analysis history captured in real time • Work anywhere, and share the results with anyone • Social/Interactive Science

• Every code change versioned • Every issue tracked • Every project the starting point for new work • Social/Interactive Coding

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Currently >20KK datasets and ~1M models

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NIH AMP Program in Alzheimer’s All data shared quarterly and teams participating on bi-weekly calls

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CRC Subtyping Consortium

DREAM Challenges Progenitor Cell Biology Consortium

TCGA Pan-Cancer Consortium

CommonMind Consortium

PsychENCODE

Accelerating Medicines Partnership

Synodos / NF2

Influenza Vaccinations

Dengue Fever Psychosis Spectrum Disorder

Type II Diabetes

Next Gen Scientific Publishing

Mozilla Science Labs

sage bionetworks

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incentives and rewards

sharing beyond pre-defined researchers

Crowdsourcing using “Challenges”

empowered by Synapse

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Engaging New experts

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Incentivizing Continuous Participation

• Monthly leaderboard winners – Winner is highlighted within the

Challenge community – Winner posts a blog on winning model to Synapse

• Communities that link to the

Leaderboard – Stackoverflow: Q&A site with 1,000,000 users – Science Translational Medicine

community

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Predict cancer-associated mutations from whole-genome sequencing data. Continuous Challenges- ways to accelerate the adoption of a new standard

Develop predictive models to infer genes that are essential to cancer cell viability using gene expression and/or gene copy number features.

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DREAM 10 Challenge Planning Already Underway:

• Data set is ready: fully harmonized clinical data set of >9,000 ALS patients • Challenge objective: algorithms that can assist with stratification of ALS

patient populations to help improve individual patient-level prognosis and to improve the success rate of clinical trials

Project Datasphere Prostate Cancer DREAM Challenge Challenge Focus: predict survival for prostate cancer patients based on patients’ clinical variables using Comparative Arm data from Industry Trials

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searching for targets

looking beyond those that are sick

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The Resilience Project:

A Search for Unexpected Heroes

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power to diagnose yet lack power to fully treat

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AN OPEN FEDERATED COLLABORATION

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engaging the public as partners

expanding the dimensions of diseases

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BRIDGE

Incorporating open data, patient wisdom and public involvement into biomedical research

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A Research Study with Feedback Loops

Anecdotes into Signals

Partners Partners

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Tanner Kruger

Bloem Kieburtz

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Current measures of disease are often relatively insensitive, episodic, provider-centered, clinic-based … Parkinson disease outcome measures – MDS-UPDRS

Source: Movement Disorder Society – Unified Parkinson’s Disease Rating Scale

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Smartphones and wearable devices can greatly improve current measures of Parkinson disease Parkinson disease outcome measures

Current measures Smartphone measures

Insensitive

Subjective

Episodic

Provider-centered

In clinic

Uni-dimensional

Limited or absent feedback

Sensitive

Objective

Continuous

Individual-centered

Remote

Multi-dimensional

Real-time feedback

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TIMED TAPPING TASK

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Gait

Tremors

Mood

Cognition

Fatigue

Speech

Sleep

ANECDOTES INTO SIGNALS

STRESS SLEEP MEDICATION EXERCISE FOODS MEDITATION

FINDING WINDOWS FOR INTERVENTION Pre-enroll Clinical trials

Validated anchors with serious friction Structured Tests Moderate validity Friction Poorly validated Frictionless Passive measures

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OPPORTUNITIES FOR OPTIMIZING CLINICAL TRIALS

Parkinson’s Disease

Alzheimer’s Disease

MS

Diabetes

Rheumatoid Arthritis

(Breast Cancer Survivors) Etc….

(mood social behavior, cognition, movement)

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ResearchKit Apps enabled by Sage Bionetworks’ – Bridge Server Data stored at Institutions and on the Sage Bionetworks’ Synapse

PRIVACY AND SHARING

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Participant Centered Studies x

Broadly Shared Data x

New ResearchKit Modules

X

“ “

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combining the pieces

altering roles and responsibilities

a look at the process end-to-end of health care and drug discovery

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Arch2POCM

Engaging communities

Challenges- Crowdsourced Analyses and Targets

Engaging the public Defining dieases

Crowdsourced tools reagents

Crowdsourced POC Molecules

Engaging the public Stratified Clinical Trials

Sustainable pipeline for better care and treatments

Alternate Approaches to Explore Multidimensional Clinical Space

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maximizing the flow of relevant insights for the widest impact

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Q&A