An Introduction to the Canadian OneMatch Public Cord Blood Bank

An Introduction to the Canadian OneMatch Public Cord Blood Bank Dr. Heidi Elmoazzen, Director, OneMatch Public Cord Blood Bank 2012-04-13

Overview Overview of the need for stem cells OneMatch Public Cord Blood Bank model Cord blood banking process Current activities and next steps 2

Stem Cells Two types of stem cells Embryonic Adult Three main sources of (hematopoietic) adult stem cells Bone marrow Peripheral blood mobilization Umbilical cord blood 3

Number of Patients Canadian Patient Searches Demand and Need Continues to Increase 1000 894 964 900 811 800 708 700 600 500 393 475 543 400 295 300 200 100 0 04/05 05/06 06/07 07/08 08/09 09/10 10/11 11/12 YTD Total 4

Canadian Stem Cell Recipients Canadian Unrelated Transplants 2002-2011 350 300 250 200 150 148 190 187 203 228 252 285 266 273 100 50 0 89 43 61 39 45 43 36 45 58 53 41 02/03 03/04 04/05 05/06 06/07 07/08 08/09 09/10 10/11 11/12 YTD International Donor Canadian Donor 5

Canadian Patients Transplanted Using Cord Blood Stem Cells 6

Need for Additional Stem Cells OneMatch is an active member of the International Network of stem cell registries and on behalf of all Canadian patients needing a stem cell match, we search and have access to: 18.9M potential adult donors from 66 registries in 49 countries; and, 523K public cords from 44 cord blood banks in 30 countries. source: BMDW, April 09, 2012 However, even with this access, we are still only able to find matches for about 50% of patients AND it is harder to find matches in many ethnic groups due to under-representation on registries worldwide. With the continued significant increase in demand and Canada s unique and large ethnic diversity, there is significant need for a national, public cord blood bank. 7

Need for a National Public Cord Blood Bank An increasing majority of all unrelated paediatric stem cell transplants in Canada are performed using cord blood and there has been and increase in the adoption of cord blood for adults (two cords used). Cord blood has distinct advantages over bone marrow or PBSCs: More lenient HLA matching requirements Units are available immediately Lower risk of graft-vs.-host disease (GvHD) Minimal risk to donor (mother and baby) All cord blood units supplied by Canadian Blood Services for unrelated transplant are imported from international sources. The cost of importing cord blood units continues to increase $42,000 ea. Uncoordinated, multiple banks cannot meet the need for a HLA diverse inventory that is required for Canadian patients. 8

Current Canadian Situation In Canada, there are 10 private cord blood banks and 3 public cord blood banks. The 3 public cord banks are: 1. Héma-Québec 2. Alberta Cord Blood Bank 3. The Victoria Angel Registry of Hope 9

OneMatch Public Cord Blood Bank 1) A centralized, national model for cord blood is best able to meet the needs of all Canadians by addressing the patient needs of each province, including ensuring local (hospital), timely access to this important stem cell product. 2) Canadian Blood Services is well-positioned to provide an HLAdiverse, searchable, accredited cord blood bank that reflects Canada's ethnic diversity. 3) By implementing a national, public cord blood bank, Canada would join all other countries in the G8 by becoming increasingly self-reliant with using this important resource domestically, while also supporting patients, internationally. 10

Benefits of a National Public Cord Blood Bank Reduce Canada s dependency on international sources for cord blood, and we will become a global supplier instead of simply a net importer Confidence and consistency in product quality and safety Leverages Canada s ethnic and racial demographics for the benefit of Canadians and international patients Stimulates stem cell research in Canada and provides support for translating Canada s leading edge research into cellular therapies Gain efficiencies by having one national bank for the country, avoiding the expense of duplicate effort and infrastructure A national, centralized model would provide a single point of access for physicians searching a cord blood unit and facilitate international exchanges of cord blood units 11

Official Announcement March 14, 2011 Ministers of Health announce Canada's first national, publiclyfunded umbilical cord blood bank $48 million investment over eight years. Please note: $12.5M of the $48M will be raised over three years by Canadian Blood Services fundraising campaign. April 1, 2011 OneMatch Public Cord Blood Bank project begins in Ottawa, as per approved model by the Ministers of Health April 2, 2013 Go live! 12

Guiding Principles Canadian Blood Services will establish and operate a national, public cord blood bank that will: be accessible to all Canadian patients, at no charge; be accessible to international patients; be a source for cord blood units in addition to international registries; operate in compliance with regulatory and accreditation standards, to ensure the safety and efficacy of the product for therapeutic use; be publicly funded through the provincial and territorial governments (excluding Québec); contain an HLA diverse inventory to reflect the ethnic make up of the Canadian population, to ensure that ethnic minorities have access to high-quality cord blood; promote research efforts that contribute to improved clinical care, by providing to Canadian researchers stem cells that cannot be used for transplantation; and, operate in the highest ethical manner, respecting both the rights and needs of Canadian infants and mothers, and the rights and needs of patients. In keeping with its mission, Canadian Blood Services will operate the cord blood bank in a manner that gains the trust, commitment and confidence of all Canadians by providing a safe, secure, costeffective, affordable and accessible supply of quality cord blood units. 13

OneMatch Public Cord Blood Bank Model Includes at least four collection cities and two manufacturing facilities for processing, storage and distribution. Please note: We will also be working on cord blood collection kits that can be sent to targeted areas outside of cities listed below to reach Aboriginal communities, for e.g. Canadian Blood Services OneMatch National Public Cord Blood Bank (inventory of 20,000 units within 8 years*) Medical and Scientific Advisory Committee Searching, Matching OTTAWA Manufacturing Facility (April 2, 2013) EDMONTON Manufacturing Facility (2014) CBS Support HLA Testing Transmissible Disease Testing CBS Research Ethics Committee (units unsuitable for transfusion) Marketing & Recruitment *Optimal size of a national cord blood inventory is currently estimated to be approximately 20,000 units, which can be achieved within eight years using this Model. Canadian Transplant Programs International Registries OTTAWA Collection Hospital(s) (April 2, 2013) TORONTO Collection Hospital(s) (2014) EDMONTON Collection Hospital(s) (2014) VANCOUVER Collection Hospital(s) (2014) Canadian Research Scientists 14

Cord Blood & Research Approximately 20% to 40% of the cord blood units collected are of sufficient volume and/or cell count to be used for transplantation. Thus, donors will have the option to designate their cord for research in Canada if it cannot be used for transplantation. To ensure a comprehensive system is in place for Canadian researchers to access cord units in a rational, fair and transparent manner, Canadian Blood Services will work with groups such as: Canadian Blood Services Research Ethics Board Canadian Stem Cell Network Canadian Blood and Marrow Transplant Group 15

Potential Uses of Stem Cells 16

Cord Blood Banking Process Seven key processes for cord blood will include capturing and centralizing data on a per unit basis for traceability, searching and matching, information management, ad-hoc queries, reporting, data export and modeling for optimization. Awareness Recruitment Collection Receiving, Assessing, Processing, Testing, Storage Searching, Matching Distribution Transplant, Follow-up Stem Cell Application Solution 17

Awareness Marketing Objectives 1. To develop strategy to raise awareness of the public cord blood bank among prospects to facilitate recruitment 2. To implement tactics to support recruitment 3. To develop strategy for donor recognition 18

Recruitment Our target group is specific and localized, i.e., healthy pregnant women who plan to deliver at the designated collection hospitals: Discussions begin during the antenatal period with their health care professional Education, information gathering, questions (becoming informed of process in advance of day of delivery) Arrive on special day with Permission to Collect form ready No advance registration 19

Collection Three Collection Methods 1. Ex-utero: Fully supported by CBS OMPCBB designated staff 2. In-utero (supported): All aspects of collection, labelling, packing/shipping by hospital staff CBS OMPCBB Nurse Specialist for maternal interview 3. In-utero(non-supported): Entire process completed by hospital staff. This model is intended for future remote sites e.g. northern Canada. The Ottawa Hospital April 2, 2013 General Campus: Ex-utero Civic Campus: In-utero (supported) 20

Manufacturing Current Resources Ottawa Stem Cell Manufacturing Facility (40 Concourse Gate): Peripheral Blood Apheresis / Bone Marrow: Processing, testing, storage, distribution New Manufacturing Area 2004 Four manufacturing areas 2000 sq. ft. FACT Accredited / GMP Guidelines Staffing: Charge Technologists and MLTs Manufacturing Equipment 21

Receiving & Assessing From collection site to manufacturing facility Process control / inventory control / tracking CBS Transport / selected courier Assessing: Acceptability criteria Volume, blood cell counts, visual inspection, pre- manufacturing sample Maternal blood samples: TD testing 22

Processing Sepax System Fully-automated, mobile, GMP compliant system for the efficient and reproducible processing of umbilical cord blood, bone marrow and peripheral blood Hands-free operation automated, closed, sterile system Quick, accurate data tracking Consistently high mononuclear cell recoveries 23

Quality Control Testing Blood cell counts (Pre and Post) Purity, Potency, Safety Purity: Microbiology Potency: Stem Cell Counts Flow Cytometer (CD34+ count) CFU Assay (stem cell culture) Safety: Genetic screening, infectious disease testing QC samples storage 24

Cryopreservation Volume reduced cord blood unit Cryo-protectant added (DMSO) Controlled rate freezing using ThermoGenesis BioArchive freezer Holds up to 3,636 units Liquid nitrogen storage (-196 C) Inventory controlled / tracked Must be stored within 48 hours of collection; can be stored indefinitely 25

Searching and Matching Listed on OneMatch Registry Banked inventory of cord blood units Cords listed, searched and requested Searched both nationally and internationally OneMatch Stem Cell National Systems Solution (IT system) Physicians / Clinical Transplant sites request OneMatch banked cord blood unit Build on current OneMatch process 26

Distribution Requested Cord Blood for Transplant Manufacturing selects, releases, packs and ships Transport courier 27

Transplant and Follow-up Transplant Follow-up: Outcome Measurement Build upon the current OneMatch process Infusion Reactions Engraftment Data 100 day follow-up Family History Genetic 28

Scope and Timelines The Canadian Blood Services OneMatch Public Cord Blood Bank will consist of collection sites (hospitals) in Ottawa, Toronto, Edmonton and Vancouver, with processing and storage sites at our two existing stem cell manufacturing facilities in Ottawa and Edmonton. The goal is to reach a minimum of 20,000 cord blood units in storage for transplantation by March 31, 2019 eight year period beginning April 1, 2011. Phase I of the cord blood bank will be operational in Ottawa in April 2, 2013, and in the remaining sites, Phase II, in 2014. FY 2011/12 FY 2012/13 FY 2013/14 FY 2014/15 Ottawa Collection & Manufacturing Edmonton Collection & Manufacturing Vancouver Collection Toronto Collection PHASE I PHASE II PHASE II PHASE II 29

Cord Blood Bank Site Visits and Key Learning Site Visits 2011-08-16: Cleveland Cord Blood Centre 2011-09-26: MD Anderson Cancer Center, Houston 2011-10-27: New York Blood Center 2011-11-01: LifeCord Cord Blood Bank, Gainesville FL 2012-01-31: Héma-Québec, Montreal 2012-02-23: St. Louis Cord Blood Bank, St. Louis, MI Key Learning Processes, Methods, Validation, Testing, Research, Shipping, Documentation Demonstrations (e.g., processing, cryopreservation) TNC Cut-off for storage most are targeting 1.4 X 10 9 cells Recruitment Collection Processing Storage 30

Manufacturing Facility Renovations 40 Concourse Gate Ottawa LN Auto-fill System Installed Storage Room Renovations Completed Equipment, room temperature/humidity monitoring implemented Receipt and installation of key pieces of equipment 31

Edmonton Facilities renovations Human resources Other Cord Blood Activities Regulation/Accreditation Health Canada Registration FACT/NetCord Accreditation AABB Accreditation Information Technology SAP: Selected Solution Customer Support Provided by our National Contact Centre and OMPCBB Team 32

Other Cord Blood Activities Fundraising First annual charity golf tournament held September 22, 2011 Second annual charity hockey game held March 19, 2012 Sponsorship engagement with key corporations and other organizations, ongoing Phase II Hospital Selection Planning Based on birth statistical data and geographic location, 13 hospitals in Vancouver, Edmonton and Toronto have been identified Ethnicity data on the population surrounding these hospitals will be collected to further narrow down the selection of potential collections sites for Phase II 33

Next Steps to Go-live, April 2, 2013 Infrastructure Ottawa Facility Renovations Equipment Installation & training Documentation Processes Data Requirements Procedures (ie, SOPs) Forms (ie, Consent) Hospital Readiness Agreement (MOU) Renovations Communications Validation Begin collecting in Ottawa to validate/test all processes and equipment 34

Cord Blood Project Team Sue Smith Executive Director, Stem Cells Dr. Heidi Elmoazzen Project Director Dr. David Allen Medical Director, Consultant Dr. Tanya Petraszko Medical Director, Consultant Mike Halpenny Sr. Project Manager, Manufacturing Karen Mostert Manager, Recruitment & Collections Todd Campbell Project Manager Cheryl Earl Laboratory Technologist Colleen Bruneau Project Coordinator Lily Pepper Activations Administrator 35

Cord Blood Project Support Team First Name Last Name Title First Name Last Name Title Lisa Martin Charge Technologist Donna Perron Stem Cell Technologist Berna Mendoza Technologist Lin Yang Senior Research Assistant Paul Waite IT Consultant Alison Baizana Director, Risk and Insurance Management Janet Parks Case Manager, OneMatch Cilla Perry Manager, Research & Development Sandra Balestra Senior Administrative Assistant Virginia Gaffney Marketing Mgr, OneMatch & Automated Collections Jennifer Biemans Director, Regulatory Affairs Yves Garcia Director, Stem Cells National Systems Solutions John Bromley National Manager, Public Affairs Gail Mingie Coordinator, Privacy and Access to Information Dr. Locksley McGann Scientific Advisor Wendy Lowthers Associate, Reporting, Regulatory Affairs Scott Burton Contract Coordinator Jennifer Philippe Director, OneMatch Stem Cell and Marrow Network Kathy Ganz Manager, Quality Assurance Lisa Potter Process and Risk Management Consultant Melanie Griffin Legal Counsel Shelley Price Manager, Submissions, Regulatory Affairs Robert Kelly Manager, Facilities Kathleen Priestman Associate, Privacy and Access to Information Todd Fenton Supervisor, Supply Services Dale Rosborough A/Project Manager, Business Continuity Jason Oliver Business Systems Analyst II Annie Barrette Communications Specialist Janet Parks OneMatch Program Integration Jeannette Venedam Subject Matter Expert, Business Lead Brian Wand Purchasing Lucie Sabourin A/Director, Records Management Rubina St. Louis Financial Analyst Margaret Miedema Director, Major Gifts and Campaign Michael den Admirant Associate, Validation Brenda Letcher Laboratory Technologist III, Supervisor Gilles Rancourt Director, Logistics Alexandra Wilgosh Project Manager, Donor Testing Gordon Sutherland Field Logistics Manager, NEON Dr. Antonio Giulivi Scientific Advisor Mary Mainella Manager, Human Resources Jean Landreville Manager, Distribution David Strachan Facilities Coordinator Sophie Charge Director, Research & Education Dr. Sofia Tavoularis Director, HLA Jim Jeang Interactive Marketing Manager Denny Michaud Manager, Customer Relations (NCC) Donna Killeen Director, Corporate Portfolio Management Christopher Mulders Acting NDAS Team Lead Ryan Melnyk Biomedical Technologist/NDAS Specialist Rosanne Dawson Legal Counsel Judie Leach Bennett Executive Director, Research & Education Steve Harding Executive Director, Development Margaret Miedema Director, Major Gifts & Campaign Ramin MaysamiAzad Biomedical Technologist Elaine Ashfield Executive Director, PRIMO & Chief Privacy Officer John Soucie Biomedical Technologist Nancy Robitaille Document Facilitator

Thank you heidi.elmoazzen@blood.ca 37

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