Download Open Science accelerating drug discovery

Open Science accelerating
drug discovery
Dr Wen Hwa Lee
Director, Patient & Disease Foundations
Strategic Alliances
SGC, University of Oxford
The (un)targeted cancer kinome.
Fedorov O, Müller S, Knapp S.
Nat Chem Biol. 2010 Mar;6(3):166-169.
Too many roads not taken.
Edwards AM, Isserlin R, Bader GD, Frye SV, Willson TM, Yu FH.
Nature. 2011 Feb 10;470(7333):163-5.
www.thesgc.org
[email protected]
Open Science can reduce duplication, improve efficiency and
accelerate discoveries
CREATIVE COMMONS
Public-Private
Partnership
Tools & Basic Knowledge
NOVEL Proteins only!
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Structure
Chemistry
Antibodies
Screening
Cell Assays
PROPRIETARY
Public Domain
Discovery and Exploration
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No patent
No restriction on use
Open access to tools and data.
Target identification &
validation
Commercial
Drug Discovery and Development
Facilitated by access to increased amount of information in the public
domain
-
(re)Screening
Lead Optimisation
Pharmacology
Metabolism
Pharmacokinetics
Toxicology
Chemical development
Clinical development
Edwards, Bountra, Kerr, Willson – Open access chemical and
clinical probes to support drug discovery. Nat Chem Biol 2009
www.thesgc.org
[email protected]
SGC at a glance
• Operations started in June 2004
• Government agencies, Wellcome Trust, charities & leading
pharma companies
• +300-strong team in Oxford, Toronto, Stockholm, Campinas,
Chapel Hill & Frankfurt (plus extended network)
• Open Access Policy:
- Promptly placing results, reagents and know-how in the
public domain
- SGC scientists never file patents
Bromodomains: Open Access probe drives discovery of an entire new target class
2 Years and 8 months
6 Months
July 2009
11 Months
Jan 2010
Dec 2010
3 Months
6 Months
1 Month
Jan 2011
July 2011
Oct 2011
Mar 2012
GSK carries out
first in man
(for open access discovered
indication!)
GSK informs SGC
about Mitsubishi
compound
Oxford and Harvard
start collaboration
Booming interest in
Academia and
Industry
Co -publication of JQ1
probe (SGC; cancer) and
I-BET probe (GSK;
inflammation)
JQ1 distributed to
100+ labs
Pfizer BET probe
announced
Brd4 linked to
AML (Nature)
MM (Cell)
Filippakopoulos et al, Nature 2010 (Dec)
Patients and Disease Organisations
Sharp focus on specific disease areas
Primary objective: discover/ improve therapies
Sophisticated strategies
Novel targets
Excellent network of
• Clinicians
• Disease experts
Participatory and Open
Access to patients
www.thesgc.org
[email protected]
Much more than just data and trials
‘remove hurdles to their widespread use’
“CHDI recognizes that the discovery
of treatments for HD is dependent on
the research scientists’ unobstructed
access to quality-controlled and
reliable research tools.”
www.thesgc.org
[email protected]
Fibrodysplasia Ossificans Progressiva (FOP):
From gene to clinical candidate
Apr 2006
Gene causing FOP was
discovered
Mechanism
3 years
Apr 2009
Structure solved;
mutations mapped
Pre-clinical
3 years
2012-2013
Clinical candidate
2 years
2015
Clinical study being
designed
New tool compounds & animal
models
6 years
Tool compounds
Potent and selective
Kinase gain of function
% activity
Gene
Alex N Bullock
I’M a PATIENT
I’MPATIENT
Open access enabling early-stage
drug discovery by patient &
disease foundations
I’MPATIENT programme partnerships
I’MPATIENT
Nature Medicine
5 June 2014
Huntington’s Disease
+
Role of epigenetic regulation in HD
Novel proteins and strategies identified by
CHDI network (eg. clearance, DNA repair,
autophagy, energetics)
Real-time open lab notebook
I’MPATIENT
Huntington’s Disease
+
http://labscribbles.com/
Real-time open
lab notebook
Tapping into disease experts and patients
www.thesgc.org
[email protected]
Open sharing and caring for each other
www.thesgc.org
[email protected]
“Science knows no country, because
knowledge belongs to humanity, and
is the torch that illuminates the world.”
Louis Pasteur
Definition of Open Science in 1800s
I’MPATIENT calls for speed-dating!
http://www.thesgc.org/speed-dating2015
Acknowledgements
Aled Edwards
Chas Bountra
Cheryl Arrowsmith
Michael Sundstrom
Opher Gileadi
Tim Willson
Stefan Knapp
Alex Bullock
Paul Brennan
Takis Prinos
ABOUT THE SGC
Arij Al Chawaf
Amy Donner
Rachel Harding
Roslin Adamson
Sergio Bartual
Andrew Lewis
Jenny Ward
Mizio Matteucci
FOP Friends
CHDI
Myeloma UK
Brain Tumour Charity
Chris Bedford-Gay
Richard Simcox
Leticia Toledo-Sherman
Eric Low
Lindsay Perera
Simon Ridley
Sarah Lindsell
Erica Moyes
David Jenkinson
SGC
SGC
SGC
SGC
SGC
SGC
SGC
SGC
Oxford
Toronto
UNICAMP
UNC
Stockholm
Frankfurt
Board
+ ULTRA PIs
The SGC is an international public-private partnership (UK charity number 1097737) that aims to carry out basic science of relevance
to drug discovery, placing all information, reagents and know-how into the public domain without restriction.
Contact
Wen Hwa Lee, PhD
Director
Disease Foundations Network,
Strategic Alliances
Structural Genomics Consortium
[email protected]
ABOUT THE SGC
The SGC is an international public-private partnership (UK charity number 1097737) that aims to carry out basic science of relevance
to drug discovery, placing all information, reagents and know-how into the public domain without restriction.
www.thesgc.org
I’MPATIENT programme partnerships
“Stone-Man Syndrome”
+
Multiple Myeloma
+
Brain Tumour Charity
+
Huntington’s Disease
+
I’MPATIENT
Ultra-rare disease
No treatment
SGC co-dev. compounds.
assays & models
Relapses
Need to identify novel targets for new
treatments
Myeloma UK already established Translational
Research Programme & Clinical Trials Network
Novel tissue platform and follow-up
med chem to generate open clinical
compounds for DIPG
Role of epigenetic regulation in HD
Novel proteins and strategies identified by
CHDI network (eg. clearance, DNA repair,
autophagy, energetics)
ZMPSTE24 and Progeria
Liz Carpenter
www.thesgc.org
[email protected]