2016 11-11 IHE jaarcongres 2016, Bussum, Alain van Gool

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

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

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

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 !

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

Emerging Personalized / Precision / Targeted Medicine

5% of drugs in pipeline had companion diagnostic biomarker test

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

Personalized health(care) in a systems view

Focus on the end user: the patient / citizen

Societal need in efficient personalized health(care)

{Source: prof Jan Kremer}

Towards cost effective care, less cure

Highest need in efficient personalized health(care)

It’s personal !

‘I want to stay healthy.’ ‘If not, how do I get healthy?’

Translating Personalized Health(care) in society

Metaphor: a route planner

Analogy: GPS route planner

GPS to a location

Amsterdam Amsterdam

Route 1 Route 2

= Default Traffic jam near Utrecht Alternative route

Personalized Health(care) model

GPS to health

Health

Route 1 Route 2

= Default First signs of disease risk

Alternative route

Health

We need a personalized data-driven GPS for health

• Monitor (biomarkers) on background

• Alert when you are at risk

• Advice what to do

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?

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

5 10 15 20 25 30 35 40 Time [min]

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

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 !?

System biology β-cell Pathology

gluc Risk factor

{Source:

Ben van Ommen

therapy

A changing world: Getting digital

New data ! (generators, owners)

Ongoing: translate laboratory to society

• Point-of-care analysis of few biomarkers

• 1.000.000 signals per proteomics analysis

and many many many more …

What does my DNA tell me?

23% chance blond hair 3.1% Neanderthaler DNA

Genetic risk lung cancer → don’t smoke !

What does my DNA tell me?

No expected adverse reaction to Warfarin

Demo room

… but not all data is useful data !

Data for personalized health(care)

time for quality, not quantity

Need for optimal quality in health biomarker analyses

Test, interpret, advice

“Post-traumatic Test Syndrome” ?

Need to change our focus

Biomarker innovation gaps !

Discovery Clinical

validation/confirmation

Diagnostic

Number of

biomarkers

• Too much biomarker discovery • Too little development to application

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

Number of

biomarkers

Choice for clinical researchers: discover or confirm?

Good example of multi-laboratory biomarker validation

3 biomarkers: • Aβ42 • T-Tau • P-Tau

Good example of multi-laboratory biomarker validation

Adoption of best biomarker practice ???

Share negative outcomes

Need to build biomarker validation pipelines

Standardisation, harmonisation, knowledge sharing needed in:

1. Assay development

2. Clinical validation

3. Application

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.

Critical component in personalized healthcare: Data

• Data capture • Data stewardship

{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

Open Science

Open Access

FAIR data

Open Science Conference

The Future of FAIR Data Stewardship

5% FAIR

Services

Exchange layer minimal protocols

FAIR data stewardship working model

Translation is key in Personalized Healthcare !

“I’m afraid you’re

suffering from an

increased IL-1β and

an aberrant miR843

expression”

Adapted from:

Think big, start ‘small’

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

Joining forces in Netherlands To drive personalized medicine & health research

https://youtu.be/MpMSkbu9YQg

11-11-2016 63

Health-RI Congress

1 December 2016 De Flint in Amersfoort Register: www.nlhealthresearch.nl

Closing remark

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

2016 11-11 IHE jaarcongres 2016, Bussum, Alain van Gool

Health & Medicine

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