Genome BC : Regional Perspectives on Personalized Medicine

in British Columbia, Canada

EuroBioForum 2013 / MunichMay 28, 2013

• 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

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

Investment Sources

International, Industry &

Institutional30%

Provincial 25%

Federal45%

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

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

Discovery large scale Genome BCGenome 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

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

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)

Support for Personalized Medicine in British ColumbiaPersonalized 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

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 failure10

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

Genomics and Personalized Health – New Projects

12

Project Leader(s) Project TitleTotal

budgetJoseph ConnorsMarco MarraRandy Gascoyne

Personalized Treatment of Lymphoid Cancer: British Columbia as Model Province $10M

Richard HarriganJulio Montaner Viral and Human Genetic Predictors of Response to HIV Therapies $4.9M

Andrew PennChristoph BorchersShelagh Coutts

Reducing Stroke Burden with Hospital-Ready Biomarker Test for Rapid TIA Triage $9.8M

Don SinRaymond Ng

Clinical Implemetation and Outcomes Evaluation of Blood-Based Biomarkers for COPD Management $7.2M

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

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-win14

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

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

• Cohorts

• Biostatistics / Bioinformatics

• End-user buy-in

TOP 3 recommendations for achieving tangible results

17

For More Information:

Website:www.genomebc.ca

Contact:

THANK YOU!

Rachael Ritchie, Director of Business Developmentrritchie@genomebc.ca

18