EuroBioForum 2013 2nd Annual Conference 27-28 May 2013 - Hilton Munich City, Munich, Germany http://www.eurobioforum.eu/2013 ======================================= # REGIONAL PERSPECTIVES # Genome British Colombia, Canada: Regional Perspectives on Personalized Medicine in British Columbia, Canada Dr Rachael Ritchie Director Business Development Genome British Colombia ======================================= http://www.eurobioforum.eu

1. Genome BC : Regional Perspectives
on Personalized Medicine
in British Columbia, Canada
EuroBioForum 2013 / Munich
May 28, 2013

2. • A catalyst for the life sciences cluster on Canada's West Coast
• Cumulative portfolio of over $625M across 180 genomics
research projects and science and technology platforms
• Focusing on sectors of economic importance to BC and
Canada:
• Agriculture
• Aquaculture & Fisheries
• Forestry
• Mining, Energy & Environment
• Human Health
• To generate social and economic benefits
Genome British Columbia
2

3. Background
• Co-founded by the late Dr. Michael Smith, Nobel
Nobel Laureate, Genome British Columbia
was formed in July 2000.
• The initial strategic plan covered the period 2001
through 2005, with a $69M program.
• Genome BC has successfully implemented its second
strategic plan (2005-2010) and exceeded the $300M
research program.
• Genome BC has initiated its third strategic plan (2010-2015)
and plans a $340M research program. 3

4. Investment Sources
International,
Industry &
Institutional
30%
Provincial 25%
Federal
45%
Research Investment
Objectives
• Match BC’s key economic
concerns.
• Leverage contributions from
other organizations.
• Maintain partnerships with a
broad cross-section of
provincial institutions, and
locations.
4

5. Research Funding Continuum
Discovery Research Applied Translational
Genome BC invests in critical space along the research
funding continuum to bring genomics discoveries from
the bench into real world applications
5
Commercialization

6. Discovery large scale Genome BC
Genome BC & Genome
Canada
2002 2004 2005 2006 2007 2008 2009 2010 2011 20122003 2013 20142001
Translation – securing benefits
Application
The Evolution of Genome BC programs

7. Personalized Medicine : a New Paradigm
“One-size-fits-all” paradigm:
Same frequency of testing for large population (e.g.
mammograms)
Symptom-driven
diagnosis
Therapy based on
clinical symptoms
Monitoring based
on clinical
symptoms
Personalized medicine paradigm:
Test for markers
that risk (e.g.
genetic variants in
cancer)
Focused
prevention efforts
in people with
risk
Focused
monitoring in
people with
risk
Molecular
monitoring for
disease subtypes
Targeted therapy
based on disease
subtype, risk of
adverse response
Molecular
monitoring for
response to
therapy
Source: Personalized Medicine Coalition
7

8. British Columbia Health Care Context
• A population of 4.6 million
• Publically funded, single payer system
• delivery via regional & provincial authorities
• $15.51 billion health-care budget (+5.8%/yr2)
• $3,360 / per capita
• $180M in research spending (2011/12) 3
• BC Cancer Agency (BCCA)
• Michael Smith Genome Sciences Centre (GSC)
• BC Center for Disease Control (BCCDC)
• Center for Drug Research and Discovery (CDRD)
1 2012 year 2For the period 2000-2010.
3 www.phsa.ca/research report (2012)

9. Support for Personalized Medicine in British Columbia
Personalized Medicine Program (PMP) (Genome BC)
• investigator-driven larger-scale projects in human health
• outcomes are ready for clinic use and/or uptake into the health system
within 3 years of launch
• Must demonstrate the support from the payer (e.g. a regional health
authority) & the potential cost-effectiveness of the translation of the
proposed research to the healthcare system
• project budget up to $3 million
• similar to the Wellcome Trust “Health Innovation Challenge Fund”
Genomics and Personalized Health (GPH) (Genome Canada)
• in partnership with Canadian Institutes of Health Research (CIHR),
• outcomes concrete deliverables clinical utility and/or practical applicability
• project budget $10 million 9

10. Genomics Applied to the Management of High-risk
AML/Myelodysplastic syndromes – Karsan and Marra ($3.0M)
(BC Cancer Agency, BC Cancer Foundation; $3.0M)
Primary Research Objective
• Develop a genome-wide sequencing assay of RNA and/or DNA on a next-generation
sequencing platform and ensure that all the known mutations that predict outcome
of AML treatment are identified in a cost-effective manner
Implementation of a Pharmacogenetic ADR Prevention Program
in BC – Hayden and Carleton ($4.0M)
(Children’s and Women’s Health Centre and UBC; co-funding partners include Merck,
IBM, Pfizer; builds on earlier $12.4M investment in two projects)
Primary Research Objective
• Demonstrate the utility and cost-effectiveness of pharmacogenetic tests designed to
prevent cisplatin-induced deafness and anthracycline-induced heart failure
10

11. Clinical Implementation of Diagnostic Biomarker Assays in Heart
and Kidney Transplantation – McManus et al ($2.0M)
(St. Paul’s Hospital, Vancouver Coastal Heath, and UBC; builds on earlier $15.6M investment
in two projects)
Primary Research Objective
• To develop simple, safe, accurate, and cost-effective diagnostic tools that improve
transplant patient outcomes and reduce costs
Stratifying and Targeting Pediatric Medulloblastoma Through
Genomics - Marra ($9.9M)
(Hospital for Sick Children, Genome Canada, Terry Fox Research Institute, Ontario
Institute for Cancer Research et al.)
Primary Research Objective
• To develop markers that will more accurately classify the medulloblastomas, the
most common form of childhood brain cancer, to optimize treatments to improve
the quality of life of patients. 11

12. Genomics and Personalized Health – New Projects
12
Project Leader(s) Project Title
Total
budget
Joseph Connors
Marco Marra
Randy Gascoyne
Personalized Treatment of Lymphoid Cancer: British Columbia as Model Province $10M
Richard Harrigan
Julio Montaner
Viral and Human Genetic Predictors of Response to HIV Therapies $4.9M
Andrew Penn
Christoph Borchers
Shelagh Coutts
Reducing Stroke Burden with Hospital-Ready Biomarker Test for Rapid TIA Triage $9.8M
Don Sin
Raymond Ng
Clinical Implemetation and Outcomes Evaluation of Blood-Based Biomarkers for COPD
Management
$7.2M

13. The realityThe hope
50% 50% 80% 20%
• We are still doing research
• The transition to development not easy
• The shift to impact even more difficult
research researchdevelopment development
R & D in Personalized Medicine
13

14. Lessons Learned
• Success in translation varies by disease area
• Difficulty achieving milestones (e.g. ending research and
achieving clinical validation)
• Insufficient data provided to demonstrate achievement of
milestones
• Stratification reduces effective population sample sizes,
making replication difficult
• The pathway to translation envisioned may not be realistic
• Existing or emerging competition may limit potential for
translational success—need to figure out win-win-win
14

15. WHAT CAN WE OFFER?
• Publicly funded, single payer system
• Single medical school with strong ties to many communities and
healthcare organizations
• Critical mass of researchers, physicians and healthcare providers
• A networked, centralized system of care in key disease areas
(e.g., cancer, transplantation, pediatric ailments, cardiac, renal
and infectious diseases)
• Strong track record in supporting excellence in health research
• ongoing projects, funded projects
• Innovative organizations
• Genome Canada Science and Technology Innovation Centres (Proteomics,
Metabolomics & Michael Smith Genome Sciences Centre)
• BCCA – BC Cancer Agency
• BCCDC – BC Centre for Disease Control
• CDRD –Center for Drug Research and Discovery 15

16. WHAT ARE WE LOOKING FOR?
• International partnership and collaboration
• People we can work with
• Groups that can articulate what they need so we can
get a win-win
16

17. • Cohorts
• Biostatistics / Bioinformatics
• End-user buy-in
TOP 3 recommendations
for achieving tangible results
17

18. For More Information:
Website:
www.genomebc.ca
Contact:
THANK YOU!
Rachael Ritchie, Director of Business Development
rritchie@genomebc.ca
18