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Data for personalized health(care): time for quality, not quantity
Prof Alain van Gool
IHE NL jaarcongres 2016 Bussum, 11 Nov 2016
Alain’s path 1989-now • Basis in molecular biology
• Academia, pharma, medical center, applied research institutes
• Biomarkers / Omics / technologies
• Mechanisms of disease
• Translational medicine
• Personalized healthcare
Senior Scientist Integrator Biomarkers
Scientific lead DTL-Technologies
Head EATRIS Biomarker Platform
Professor of Personalized Healthcare Head Radboud Center for Proteomics, Glycomics & Metabolomics Coordinator Radboud Technology Centers
2 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
3 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Alain’s path 1989-now • Basis in molecular biology
• Academia, pharma, medical center, applied research institutes
• Biomarkers / Omics / technologies
• Mechanisms of disease
• Translational medicine
• Personalized healthcare
Senior Scientist Integrator Biomarkers
Scientific lead DTL-Technologies
Head EATRIS Biomarker Platform
Professor of Personalized Healthcare Head Radboud Center for Proteomics, Glycomics & Metabolomics Coordinator Radboud Technology Centers
Consider individual differences in life science research
4 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Source: Chakma, Journal of Young Investigators, 16, 2009
Principle of Personalized Medicine
• The right drug for right patient at right dose at right time • = Precision medicine or Targeted medicine
5 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Personalized medicine in melanoma
B-RAFV600E mutation Strong growth of cell Growth of tumor
• B-RAFV600E cells always grow and become cancer cells
• RAF inhibitors will block pathway, block cell growth and inhibit cancers that have a B-RAFV600E mutation
• 60% of melanoma patients have B-RAFV600E mutation
• Basis for a personalized medicine !
*
6 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Personalized medicine in melanoma
Treat patients with
B-RAFV600E mutation Inhibit growth of cell
Patients live longer Tumors disappear Cells stop growing
B-RAF inhibitor
7 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Emerging Personalized / Precision / Targeted Medicine
2010:
5% of drugs in pipeline had companion diagnostic biomarker test
2015:
80%
50%
8 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Optimal Personalized / Precision / Targeted Medicine
Moving to personalized health(care)
{Source: Barabási 2007 NEJM 357; 4}
• People are more than linear pathways • Different systems and networks • Different risk factors • Different preferences
10 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Personalized health(care) in a systems view
11
11 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Focus on the end user: the patient / citizen
12 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Societal need in efficient personalized health(care)
{Source: prof Jan Kremer}
Towards cost effective care, less cure
13 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Highest need in efficient personalized health(care)
It’s personal !
‘I want to stay healthy.’ ‘If not, how do I get healthy?’
14 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
15
Translating Personalized Health(care) in society
Metaphor: a route planner
16 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Analogy: GPS route planner
GPS to a location
Amsterdam Amsterdam
Route 1 Route 2
= Default Traffic jam near Utrecht Alternative route
17 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Personalized Health(care) model
GPS to health
Health
Route 1 Route 2
= Default First signs of disease risk
Alternative route
Now
Health
Now
Health
18 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
We need a personalized data-driven GPS for health
• Monitor (biomarkers) on background
• Alert when you are at risk
• Advice what to do
19 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
3 key aspects of personalized health(care)
‘I want to stay healthy. If not, how do I get healthy?’
1. What to measure?
2. How much can it change?
3. What should be the follow-up for me?
20 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Exponential technology developments in laboratories
• Next generation sequencing
• DNA, RNA • Risk analysis and therapy selection
• Mass spectrometry • Proteins, metabolites • Monitoring of disease and treatment effects
• Imaging
• Non invasive images, real time
• Spatial view of intact organs and organisms
500
1000
1500
2000
m/z
5 10 15 20 25 30 35 40 Time [min]
21 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Advances in mass spectrometry • Mass spectrometry analysis of glycoproteins in human plasma • 0,05 microliter analysis: detection of 1.000.000 signals in one scan (1,4 Gb) • ~40.000 peptides of which >80% contain sugar modification • Diagnose patients and identify new biomarkers
500
1000
1500
2000
m/z
5 10 15 20 25 30 35 40 Time [min]
Proof of principle study:
{Hans Wessels, Monique van Scherpenzeel, Dirk Lefeber, Alain van Gool} Biomarkers !?
22 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
System biology β-cell Pathology
gluc Risk factor
{Source:
Ben van Ommen
TNO}
therapy
23 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
A changing world: Getting digital
24 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
25
New data ! (generators, owners)
25 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
26 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
27 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
28 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Ongoing: translate laboratory to society
• Point-of-care analysis of few biomarkers
• 1.000.000 signals per proteomics analysis
30 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
and many many many more …
31 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
What does my DNA tell me?
23% chance blond hair 3.1% Neanderthaler DNA
32 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Genetic risk lung cancer → don’t smoke !
What does my DNA tell me?
No expected adverse reaction to Warfarin
33 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
34 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
35 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Demo room
… but not all data is useful data !
Data for personalized health(care)
time for quality, not quantity
40 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Need for optimal quality in health biomarker analyses
Test, interpret, advice
“Post-traumatic Test Syndrome” ?
41 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Need to change our focus
42 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Biomarker innovation gaps !
Discovery Clinical
validation/confirmation
Diagnostic
test
Number of
biomarkers
Gap 1
Gap 2
• Too much biomarker discovery • Too little development to application
43 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Biomarker innovation gaps: some numbers
~ 5 biomarkers/ working day
1 biomarker/ 1-3 years
1 biomarker/ 2-10 years
Eg Biomarkers in time: Prostate cancer May 2011: 2,231 biomarkers Nov 2012: 6,562 biomarkers Oct 2013: 8,358 biomarkers Nov 2014: 10,350 biomarkers Oct 2015: 11,856 biomarkers 11 Nov 2016: 14,481 biomarkers
Discovery Clinical
validation/confirmation
Diagnostic
test
Number of
biomarkers
Gap 1
Gap 2
44 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Choice for clinical researchers: discover or confirm?
45 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Good example of multi-laboratory biomarker validation
3 biomarkers: • Aβ42 • T-Tau • P-Tau
46 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Good example of multi-laboratory biomarker validation
47
Adoption of best biomarker practice ???
Share negative outcomes
48 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Need to build biomarker validation pipelines
Standardisation, harmonisation, knowledge sharing needed in:
1. Assay development
2. Clinical validation
3. Application
49 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
The Good Biomarker Practice initiative
Join forces among Europe’s major academic infrastructures + industry to:
1. Establish “Good Biomarker Practice” guidelines
- on translational research, biomarker technologies, biobanking, data stewardship.
2. Efficiently execute high quality biomarker projects
- work together in clinical validation and development of probable biomarkers.
50 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Critical component in personalized healthcare: Data
• Data capture • Data stewardship
51 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
{Wilkinson et al, Nature Scientific Data, 2016}
FAIR data stewardship
Prof Barend Mons Leiden University Medical Center Dutch Techcenter for Life Sciences Netherlands eScience Center
52 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Open Science
Open Access
FAIR data
Open Science Conference
53 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
55 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
The Future of FAIR Data Stewardship
5% FAIR
Services
Data
Exchange layer minimal protocols
FAIR data stewardship working model
56 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Translation is key in Personalized Healthcare !
“I’m afraid you’re
suffering from an
increased IL-1β and
an aberrant miR843
expression”
Adapted from:
?
57 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Think big, start ‘small’
58 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Think big, start ‘small’ National Technology Infrastructure (collective of 40+ partners in DTL) www.dtls.nl
Ruben Kok
Barend Mons
Alain van Gool
Jaap Heringa
59 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Joining forces in Netherlands To drive personalized medicine & health research
60 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
https://youtu.be/MpMSkbu9YQg
61 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Health-RI Congress
1 December 2016 De Flint in Amersfoort Register: www.nlhealthresearch.nl
Closing remark
65 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
Acknowledgements
Hans Wessels Jolein Gloerich
Roel Tans Esther Willems
Maurice van Dael Jenneke Keizer
Dirk Lefeber Monique van
Scherpenzeel
Leo Kluijtmans Ron Wevers
Marcel Verbeek Lucien Engelen
Jan Kremer Bas Bloem
Nathalie Bovy Paul Smits
the Radboudumc Technology Centers
and many others
www.radboudumc.nl/personalizedhealthcare
www.radboudumc.nl/research/technologycenters
www.radboudresearchfacilities.nl
alain.vangool@tno.nl
alain.vangool@radboudumc.nl
www.linkedIn.com
www.slideshare.net/alainvangool
Many collaborators and funders
Jan van der Greef Ben van Ommen
Ivana Bobeldijk Hans Princen
Lars Verschuren Marjan van Erk
Suzan Wopereis Heleen Wortelboer
Wessel Kraaij Ronald Mooi
Peter van Dijken Cyrille Krul
and many others
CarTarDis
Ruben Kok Barend Mons
Jaap Heringa Merlijn van Rijswijk and
many others
Anton Ussi Florence Bietrix
Laura Bermejo Andreas Scherer
Sulev Koks Marian Hajduch
Giovanni Migliaccio
and many others
66 Alain van Gool, IHE jaarcongres, Bussum. 11 Nov 2016
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