Alternate Approaches to Explore Multi-Dimensional Clinical ......research is about to fundamentally...
Transcript of Alternate Approaches to Explore Multi-Dimensional Clinical ......research is about to fundamentally...
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
maximizing the flow of relevant insights for the widest impact
time of transition
symptoms to molecular components
molecular components to underlying networks
singular syndromes to variable presentations
HDAC / AURORA KINASE / TELOMERASE / PD1
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
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
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
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
Currently >20KK datasets and ~1M models
NIH AMP Program in Alzheimer’s All data shared quarterly and teams participating on bi-weekly calls
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
Engaging New experts
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
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.
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
The Resilience Project:
A Search for Unexpected Heroes
power to diagnose yet lack power to fully treat
AN OPEN FEDERATED COLLABORATION
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engaging the public as partners
expanding the dimensions of diseases
BRIDGE
Incorporating open data, patient wisdom and public involvement into biomedical research
A Research Study with Feedback Loops
Anecdotes into Signals
Partners Partners
Tanner Kruger
Bloem Kieburtz
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
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
TIMED TAPPING TASK
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
OPPORTUNITIES FOR OPTIMIZING CLINICAL TRIALS
Parkinson’s Disease
Alzheimer’s Disease
MS
Diabetes
Rheumatoid Arthritis
(Breast Cancer Survivors) Etc….
(mood social behavior, cognition, movement)
ResearchKit Apps enabled by Sage Bionetworks’ – Bridge Server Data stored at Institutions and on the Sage Bionetworks’ Synapse
PRIVACY AND SHARING
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
maximizing the flow of relevant insights for the widest impact
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Q&A