Scientific Program JUNE 13-16, 2021 - ANNUAL CONFERENCE OF CELL THERAPY TRANSPLANT CANADA
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JUNE 13-16, 2021
Scientific
Program
ANNUAL CONFERENCE OF
CELL THERAPY TRANSPLANT CANADA
CTTC Head Office: 750 West Pender Street, Suite 301, Vancouver, BC, V6C 2T7 • T: 604-874-4944 F: 604-874-4378 E: info@cttcanada.org W: www.cvttcanada.org
SCIENTIFIC PROGRAM
JUNE 13-16, 2021
TABLE OF CONTENTS
Welcome Message 3
Board of Directors and Conference Planning Committee 4
Accreditation 4
Disclosures 5
Invited Speakers, Chairs, and Panelists 7
Conference Schedule 8
Session Summaries 11
Oral Abstract Guide and Summaries 23
Poster Abstract Guide 29
About CTTC 31
ANNUAL CONFERENCE OF CELL THERAPY TRANSPLANT CANADA 2
CTTC Head Office: 750 West Pender Street, Suite 301, Vancouver, BC, V6C 2T7 • T: 604-874-4944 F: 604-874-4378 E: info@cttcanada.org W: www.cttcanada.org
SCIENTIFIC PROGRAM
JUNE 13-16, 2021
A MESSAGE FROM THE CTTC PRESIDENT
Dear Colleagues, novel cell therapies into our activities, a continuation of our
On behalf of the Board of Directors, I welcome you to the CTTC 2021 discussion from 2019 on how we can leverage the CTTC expertise
Annual Meeting. This year will be our first virtual meeting, and we and membership resources from across Canada to successfully
look forward to engaging with our members and attendees through introduce CAR-T and other cell therapies into the clinic.
our online platform. Finally, I would like to thank our sponsors for their ongoing support
The planning committee, led by Dr. Guy Cantin and Dr. Wilson Lam, has of the CTTC and our mission of education in cell therapy and blood/
put together an exciting program that includes lectures, networking marrow transplantation. Without their support, meetings like this
opportunities, posters sessions, and corporate symposia. We hope would not be possible. We encourage you to visit the exhibit hall
you will take advantage of the meeting to share ideas and reconnect during breaks, learn about the companies, and find out what’s new
with colleagues from across Canada. and exciting in the treatment pipeline.
This year, the conference kicks off with a full pre-conference day on
June 13, including a Patient/Family/Caregiver-focused session and a
symposium on COVID-19. Please join us for a Presidential session on Kristjan Paulson, MD, FRCPC
the Sunday evening that will explore CTTC’s vision for incorporating CTTC President
A MESSAGE FROM THE CONFERENCE CHAIRS
Dear Colleagues, Outside of the symposia, we invite you to attend our Oral Abstract
On behalf of the 2021 conference planning committee, we are excited Presentations on June 15, which is followed by our poster presentation
to welcome you to the CTTC 2021 Annual Meeting! hour where attendees will have the opportunity to interact one-on-one
with our poster abstract presenters through our interactive Poster Hall.
It has been a long journey since early 2019, when our committee
began planning an in-person meeting in Québec City for May 2020. In We have some exciting networking opportunities this year. We have
February 2020, with the spread of COVID-19, this event was postponed. organized three types of networking sessions: informal general
Today, we are pleased to be able to offer a diverse scientific program on networking, profession-specific networking, and self-led hot topic
a fully virtual platform. We are proud to welcome speakers from across roundtable discussions. Please take a look at the networking times
Canada, as well as some international speakers, to share their expertise. listed in our program. You can take part in all of these sessions
through our virtual meeting platform.
Our program provides updates on novel aspects in the clinical care of
patients undergoing blood and marrow transplantation, future trends We wish to acknowledge all of our sponsors and supporters who made
in immune effector cellular therapy, innovations in genome editing, this conference possible. Please take a moment to visit our Exhibit Hall
and new trends in laboratory aspects that support hematopoietic cell and interact with our sponsors and exhibitors during the meeting.
transplantation. We would like to sincerely thank all members of the planning
We could not ignore the impact of COVID-19 this year on our clinical committee representing many BMT transplant units across Canada.
transplant activities, and invite you to join our COVID-19-focused Our special thanks goes to representatives from Héma-Québec, and
session that will be held on June 13, where we will discuss COVID-19 to Dr. John Kuruvilla who kindly organized the CAR-T and the Future
in HCT and CAR-T cell therapy recipients, vaccines in stem cell of Cell Therapy Symposium.
transplants, and the health and well-being of healthcare workers.
Our scientific program is enriched by our corporate symposia hosted
by industry. We encourage you to review our corporate program for Guy Cantin, MD, FRCPC Wilson Lam, MD, FRCPC
the specific titles and take part in these sessions. Co-chair, Conference Planning Co-chair, Conference Planning
Committee Committee
ANNUAL CONFERENCE OF CELL THERAPY TRANSPLANT CANADA 3
CTTC Head Office: 750 West Pender Street, Suite 301, Vancouver, BC, V6C 2T7 • T: 604-874-4944 F: 604-874-4378 E: info@cttcanada.org W: www.cttcanada.org
SCIENTIFIC PROGRAM
JUNE 13-16, 2021
CTTC BOARD OF DIRECTORS
PRESIDENT: Kristjan Paulson, MD, FRCPC
PRESIDENT-ELECT: Kirk R. Schultz, MD, FCAHS
PAST-PRESIDENT: Donna Wall, MD, CCPE
TREASURER: Mahmoud Elsawy, MD, MSc
SECRETARY: Mohamed Elemery, MD, MSC, PHD
DIRECTOR-AT-LARGE, EDUCATION: Wilson Lam, MD, BSc
DIRECTOR-AT-LARGE, RESEARCH: Jean-Sébastien Delisle, MD, PhD
DIRECTOR-AT-LARGE, QUALITY: Nicole Prokopishyn, PhD
DIRECTOR-AT-LARGE, PATIENT, FAMILY, & CAREGIVERS: Peter Malone
CONFERENCE PLANNING COMMITTEE
CHAIRS: Guy Cantin, MD, FRCPC, Wilson Lam, MD, FRCPC
Frédéric Barabé, MD, FRCPC Mike Halpenny, MLT (CMLTO)
Renée Bazin, PhD Susie Joron, BSc
Chris Bredeson, MD, FRCPC Gizelle Popradi, MD, FRCPC
Jean-Sébastien Delisle, MD, FRCPC, PhD Maryse Power, MD, FRCPC
Ronan Foley, MD, FRCPC Tal Schechter-Finkelstein, MD, FRCPC
Diane Fournier, PhD Craig Speziali, MD, MSc, FRCPC
Genevieve Gallagher, MD, FRCPC Pierre Teira, MD, MSc
ACCREDITATION
This program meets the accreditation criteria as defined by the Maintenance of Certification program of the Royal College of Physicians and
Surgeons of Canada and has been accredited by the Office of Continuing Professional Development, Faculty of Medicine and Health Sciences,
McGill University for up to 17 Section 1 credits/hours.
Through an agreement between the Royal College of Physicians and Surgeons of Canada and the American Medical Association, physicians
may convert Royal College MOC credits to AMA PRA Category 1 CreditsTM. Information on the process to convert Royal College MOC credit to
AMA credit can be found at www.ama-assn.org/go/internationalcme.
ANNUAL CONFERENCE OF CELL THERAPY TRANSPLANT CANADA 4
CTTC Head Office: 750 West Pender Street, Suite 301, Vancouver, BC, V6C 2T7 • T: 604-874-4944 F: 604-874-4378 E: info@cttcanada.org W: www.cttcanada.org
SCIENTIFIC PROGRAM
JUNE 13-16, 2021
DISCLOSURES
Harold Atkins, MD – Speaker Ronan Foley, MD, FRCPC – Speaker, Moderator, Planning
Committee
• ADVISORY BOARD/COMMITTEE – Children’s Hospital of
Eastern Ontario Research Institute • ADVISORY BOARD/COMMITTEE – Celgene, Janssen, Novartis
• CLINICAL TRIAL – Investigator-led CAR-T cell trial with no • SPEAKERS’ BUREAU – Celgene, Gilead, Novartis, Servier
industry involvement • Cinical Trial – CMRG
Julie Bergeron, MD, FRCPC – Speaker Genevieve Gallagher, MD, FRCPC – Moderator, Planning
Committee
• ADVISORY BOARD/COMMITTEE – Abbvie, Pfizer, BMS,
Amgen, Novartis, Astellas • CLINICAL TRIAL – Celgene, Genentech, GSK, Hoffman-La
Roche, Millenium Pharmaceutical, Merck Canada, Gilead
Christopher Bredeson, MD, FRCPC – Planning Committee Sciences Inc, Abbvie Inc, Acerta Pharma, Bayer, Pfizer, BMS,
ESAI Inc, Sanofi, Janssen R&D, Ozmosys Research, Sierra
• ADVISORY BOARD/COMMITTEE – CancerCare Ontario
Oncology Inc, BeiGene, GlyPharma
• GRANT/HONORARIA – Otsuka, Research Support: Novartis,
Kite, Otsuka Kevin Hay, MD, MSc, FRCPC – Speaker
• CLINICAL TRIALS – Kite
• ADVISORY BOARD/COMMITTEE – Celgene/BMS, Kite/Gilead,
Marina Cavazzana, MD – Speaker Novartis
• GRANT/HONORARIA – Jazz, Janssen, BC Cancer Foundation
• GRANT/HONORARIA – Cellectis
• CLINICAL TRIAL – Celgene/BMS
• INVESTMENTS – Smartimmune
Susie Joron, BSc – Moderator, Planning Committee
Sandra Cohen, MD, FRCPC – Speaker
• ADVISORY BOARD/COMMITTEE – Swab the World
• CONSULTANT – ExCellThera
• GRANT/HONORARIA – ExCellThera, Stem Cell Network, Donald Kohn, MD – Speaker
Canadian Cancer Society Research Institute, CIHR
• CLINICAL TRIAL – UM171 Expanded cord blood • ADVISORY BOARD/COMMITTEE – Orchard Therapeutics,
Allogene Therapeutics, ImmunoVec
Isabelle Fleury, MD, MSc, FRCPC – Speaker • PAYMENT – Orchard Therapeutics, Allogene Therapeutics, CSL
Behring, Bluebird bio
• ADVISORY BOARD/COMMITTEE – Abbvie, Astrazeneca, BMS,
• INVESTMENTS – Allogene Therapeutics, ImmunoVec
Celgene, Gilead, Janssen, Merck, Novartis, Roche, Seattle
• CLINICAL TRIAL – ADA SCID, XSCID Boston Children’s/NIAID,
Genetics
XCHD and Sickle Cell by CIRM Rocket and IMO, Rocket Pharma
• CLINICAL TRIAL – Abbvie, Astrazeneca, BMS, Celgene, Gilead,
Janssen, Merck, Novartis, Roche, Seattle Genetics
ANNUAL CONFERENCE OF CELL THERAPY TRANSPLANT CANADA 5
CTTC Head Office: 750 West Pender Street, Suite 301, Vancouver, BC, V6C 2T7 • T: 604-874-4944 F: 604-874-4378 E: info@cttcanada.org W: www.cttcanada.orgSCIENTIFIC PROGRAM
JUNE 13-16, 2021
Nicolaus Kroeger, MD – Speaker Nicole Prokopishyn, PhD – Speaker
• ADVISORY BOARD/COMMITTEE – EBMT, Novartis, Kite, • ADVISORY BOARD/COMMITTEE – FACT, CTTC
Celgene
• PAYMENT – Sanofi, Novartis, Celgene, Kite Jonathan Rayment, MDCM, MSc, FRCPC – Speaker
• GRANT/HONORARIA – Riemser • ADVISORY BOARD/COMMITTEE – Polarean LLC
• CLINICAL TRIAL – University Hospital Hamburg • CLINICAL TRIAL – Vertex Pharmaceuticals, Boeringher
Deepali Kumar, MD, MSc, FRCPC – Speaker Ingleheim
• ADVISORY BOARD/COMMITTEE – Roche, Sanofi, GSK, Stanley Riddell, MD – Speaker
Takeda, American Society of Transplantation • ADVISORY BOARD/COMMITTEE – Lyell Immunopharma
• CLINICAL TRIAL – Roche, GSK, Merck, Takeda, Qiagen • PAYMENT/GRANT/HONORARIA – Lyell Immunopharma
John Kuruvilla, MD, FRCPC – Moderator • INVESTMENTS – Lyell Immunopharma
• ADVISORY BOARD/COMMITTEE – Lymphoma Canada Guy Sauvageau, MD, PhD, FRCPC – Speaker
• GRANT/HONORARIA – BMS/Celgene, Kite/Gilead, Novartis • INVESTMENTS – ExcellThera Inc.
• CLINICAL TRIAL – BMS/Celgene, Kite/Gilead
Akshay Sharma, MBBS – Speaker
Wilsom Lam, MD, FRCPC – Moderator, Planning Committee
• CONSULTANT FEE – Spotlight Therapeutics
• ADVISORY BOARD/COMMITTEE – Jazz Pharmaceuticals • CLINICAL TRIAL – CRISPR Therapeutics, Vertex
• CLINICAL TRIALS – Takeda Pharmaceuticals Pharmaceuticals, Novartis
Jonas Mattsson, MD, PhD – Speaker
Craig Speziali, MD, MSc, FRCPC – Planning Committee
• GRANT/HONORARIA – Jazz, Merck, IBD, Gilead, Takeda,
• ADVISORY BOARD/COMMITTEE – Celgene
Therakos/Mallinkrodt
Luca Vago, MD, PhD – Speaker
Kristjan Paulson, MD, MSc, FRCPC – Speaker
• GRANT/HONORARIA – Moderna Therapeutics, GEN-DX
• ADVISORY BOARD/COMMITTEE – Pfizer, Amgen, Astellas,
• PATENT – GEN-DX
Jazz, Novartis
Gizelle Popradi, MD, FRCPC – Planning Committee
• GRANT/HONORARIA – Jazz, Seattle Genetics, Sanofi
Genzyme, Lundbeck, Teva, Otsuka, Baxalta, Merck, Janssen,
Servier, Pendopharm, Novartis, Pfizer, Kite, Abbvie
ANNUAL CONFERENCE OF CELL THERAPY TRANSPLANT CANADA 6
CTTC Head Office: 750 West Pender Street, Suite 301, Vancouver, BC, V6C 2T7 • T: 604-874-4944 F: 604-874-4378 E: info@cttcanada.org W: www.cttcanada.orgSCIENTIFIC PROGRAM
JUNE 13-16, 2021
INVITED SPEAKERS, CHAIRS, AND PANELISTS
Harold Atkins, MD, The Ottawa Hospital Research Institute, Ontario Susie Joron, BSc, Héma-Québec, Québec
Nadia Baillargeon, MT, Héma-Québec, Québec Mike Kennah, MD, FRCPC, Ottawa Hospital, Ontario
Frédéric Barabé, MD, FRCPC, Université Laval, Québec Donald Kohn, MD, University of California, Los Angeles, Los Angeles, USA
Renée Bazin, PhD, Héma-Québec, Québec Nicolaus Kröger, MD, Universitiy Medical Center Hamburg, Hamburg,
Germany
Julie Bergeron, MD, FRCPC, Hôpital Maisonneuve-Rosemont, Québec
Deepali Kumar, MD, MSc, FRCPC, University Health Network, Ontario
Susan Berrigan, MLT, Alberta Precision Labortories, Alberta
John Kuruvilla, MD, FRCPC, Princess Margaret Cancer Centre, Ontario
Guy Cantin, MD, FRCPC, CHU de Quebec – Hôpital de l’Enfant-Jésus, Québec
Josée Laganière, PhD, Héma-Québec, Québec
Marina Cavazzana, MD, Biotherapy Department and Clinical Investigation
Center, Assistance Publique Hopitaux de Paris, Paris, France Wilson Lam, MD, FRCPC, Princess Margaret Cancer Centre, Ontario
Sandra Cohen, MD, FRCPC, Hôpital Maisonneuve-Rosemont/Université de Peter Malone, Cell Therapy Transplant Canada, British Columbia
Montréal, Québec Jonas Mattsson, MD, PhD, Princess Margaret Cancer Centre, Ontario
Meredith Cowden, LPCC-S, Meredith A. Cowden Foundation, Ohio, USA Luciana Melo Garcia, MD, MD Anderson Cancer Center, Texas, USA
Jean-Sébastien Delisle, MD, FRCPC, PhD, Hôpital Maisonneuve- Sylvain Moineau, OC, OQ, PhD, FRSC, Université Laval, Québec
Rosemont/Université de Montréal, Québec
Kristjan Paulson, MD, MSc, FRCPC, University of Manitoba, Manitoba
Mélanie Dieudé, PhD, Université de Montréal, Québec
Erin Plenert, MPH, Peter Gilgan Centre for Research & Learning at the
Mai Duong, BAA, Swab The World, Québec Hospital for Sick Children, Ontario
Isabelle Fleury, MD, MSc, FRCPC, Maisonneuve-Rosemont Hospital, Nicole Prokopishyn, PhD, Alberta Health Services, Alberta
Québec
Jonathan Rayment, MDCM, MSc, FRCPC, BC Children’s Hospital;
Ronan Foley, MD, FRCPC, Juravinski Hospital, Ontario University of British Columbia, British Columbia
Diane Fournier, PhD, Héma-Québec, Québec Stanley Riddell, MD, Fred Hutchinson Cancer Research Center, Seattle, USA
Leeza Friedman-Prokopishyn, MSW, RCSW, Alberta Health Services, Christiane Rochon, BAA, Swab The World, Québec
Alberta
Guy Sauvageau, MD, Ph D, FRCP(C), Université de Montréal, Québec
Genevieve Gallagher, MD, FRCPC Université Laval/CHU de Quebec,
Québec
Tal Schechter-Finkelstein, Hospital for Sick Children, Ontario
Akshay Sharma, MBBS, St. Jude Children’s Research Hospital, Memphis,
Martin, Giroux, PhD, CETC, Québec USA
Kevin, Hay, MD, MSc, FRCPC, L/BMT Program of BC and Terry Fox Laboratory, Pierre Teira, MD, MSc, University Hospital (CHU) Sainte Justine, Québec
BC Cancer, BC
Patrick Trépanier, PhD, MBA, Héma-Québec, Québec
Jelena Holovati, PhD, University of Alberta, Canadian Blood Services,
Alberta Luca Vago, MD, PhD, IRCCS San Raffaele Scientific Institute, Milan, Italy
ANNUAL CONFERENCE OF CELL THERAPY TRANSPLANT CANADA 7
CTTC Head Office: 750 West Pender Street, Suite 301, Vancouver, BC, V6C 2T7 • T: 604-874-4944 F: 604-874-4378 E: info@cttcanada.org W: www.cttcanada.orgSCIENTIFIC PROGRAM
JUNE 13-16, 2021
CONFERENCE SCHEDULE All times in this program are listed in Eastern Time.
Please note that on the virtual meeting platform,
PRE-CONFERENCE: JUNE 13, 2021 times will automatically display in your timezone.
9:45am – 10:00am WELCOME REMARKS
BMT101 – STREAM 1: HEALTHCARE BMT101 – STREAM 2: PATIENT, FAMILY, &
PROFESSIONALS CAREGIVERS
ACCREDITATION HOURS: 1.5 Chair: Peter Malone
Chairs: Wilson Lam, MD, FRCPC, Jelena Holovati, PhD, MLT
• Introduction
• Introduction • Chronic GVHD: What Matters from the Patients’ View
10:00am – 11:30am • Those Magical Cells – What They Are & What – Meredith Cowden, LPCC-S
They Do – Nicole Prokopishyn, PhD • Using Mindfulness to Support Wellness –
• Conditioning Regimens in Transplantation and Leeza Friedman-Prokopishyn, MSW, RCSW
Cellular Therapy – Michael Kennah, MD, MSc • Therapeutic Innovations in Cell Therapy and
• Societal Impact of GVHD – Jonas Mattsson, MD, PhD Transplantation – Jean-Sébastien Delisle, MD, FRCPC, PhD
• Q&A • Q&A
11:30pm – 12:00pm Break
FAIR FIGHT FOR ALL SYMPOSIUM
ACCREDITATION HOURS: 1.0
Chair: Susie Joron, BSc
12:00pm – 1:00pm • Introduction
• Diversity Saves Lives – Mai Duong, BAA, & Christiane Rochon, BAA
• Population Study in Partnership with First Nations to Address the Challenge of Diversity – Nadia Baillargeon, MT
• Impactful Patient Donor and Family Partnership in Research: The CDTRP experience – Mélanie Dieudé, PhD
• Q&A
3:00pm - 3:30pm General Networking
COVID-19 SYMPOSIUM
ACCREDITATION HOURS: 1.5
Chairs: Wilson Lam, MD, FRCPC, Tal Schechter-Finkelstein, MD
3:30pm – 5:00pm • COVID-19 in HCT and Chimeric Antigen Receptor CAR-T-cell Therapy Recipients – Akshay Sharma, MBBS
• COVID-19 Vaccines in Stem Cell Transplants – Deepali Kumar, MD, MSc, FRCPC
• COVID-19 and the Health & Wellbeing of Healthcare Workers in a Pediatric Haematology/Oncology Setting –
Erin Plenert, MPH
• Q&A
PRESIDENTIAL SYMPOSIUM
ACCREDITATION HOURS: 1.5
Chair: John Kuruvilla, MD, FRCPC
7:00pm – 8:30pm • Evolving National Strategies for CAR-T – Kristjan Paulson, MD, MSc, FRCPC
• Practical Considerations of CAR-T Therapy in Adult Lymphoma – Ronan Foley, MD, FRCPC
• Canadian-Led Immunotherapies in Cancer: The CLIC Experience – Kevin Hay, MD, MSc, FRCPC
• Q&A
ANNUAL CONFERENCE OF CELL THERAPY TRANSPLANT CANADA 8
CTTC Head Office: 750 West Pender Street, Suite 301, Vancouver, BC, V6C 2T7 • T: 604-874-4944 F: 604-874-4378 E: info@cttcanada.org W: www.cttcanada.orgSCIENTIFIC PROGRAM
JUNE 13-16, 2021
All times in this program are listed in Eastern Time.
Please note that on the virtual meeting platform,
DAY 1: JUNE 14, 2021 times will automatically display in your timezone.
HANS MESSNER LECTURESHIP SYMPOSIUM
ACCREDITATION HOURS: 2.25
Chairs: Guy Cantin, MD, FRCPC, Genevieve Gallagher, MD, FRCPC
10:00am – 1:00pm • Hans Messner Lectureship: Allogeneic Stem Cell Transplantation in Acute Leukemia and High Risk MDS –
Break: Nicolaus Kröger, MD
10:50am – 11:15am • Approaches in MDS and AML for Patients Not-Eligible for Transplantation – Julie Bergeron, MD
12:00pm – 12:15pm • Q&A with Nicolaus Kröger & Julie Bergeron
• Hans Messner New Investigator Award 2019: Longitudinal MBW in Pediatric BMT Patient – Jonathan Rayment,
MDCM, MSc, FRCPC
• Q&A with Jonathan Rayment
Administrators
Advanced BMT
Nursing Pharmacist and BMT Laboratory
2:15pm – 3:30pm Practitioners Physicians
Networking Networking Coordinators Networking
Networking Networking
Networking
3:30pm – 4:00pm General Networking
CAR-T AND THE FUTURE OF CELL THERAPY SYMPOSIUM
ACCREDITATION HOURS: 2
Chair: John Kuruvilla, MD, FRCPC
4:00pm – 6:00pm • State of the Nation of CAR-T – Isabelle Fleury, MD
• NK Cells as a Novel Alternative for Immunotherapy: Towards Effective, Off-the-Shelf and Safe Options for
Cancer Treatment – Luciana Melo Garcia, MD
• Translating Engineered T-Cells from Bench to Bedside – Stanley Riddell, MD
• Q&A
INNOVATION IN GENOME EDITING SYMPOSIUM
ACCREDITATION HOURS: 1
Chair: Renée Bazin, PhD
7:45pm – 8:45pm
• CRISPR-Cas systems: from Humble Beginnings to Today’s Headlines – Sylvain Moineau, OC, OQ, PhD, FRSC
• Genomic Editing in Hematopoietic Stem Cells – Josée Laganière, PhD
• Q&A
DAY 2: JUNE 15, 2021
FRED SAUNDERS LECTURESHIP SYMPOSIUM
ACCREDITATION HOURS: 1
10:00am – 11:00am Chair: Pierre Teira, MD, MSc
• Gene Therapy for β-Hemoglobinopathies: Lentiviral and Genome Editing Approaches – Marina Cavazzana, MD
• Q&A
11:00am – 11:45am General Networking
ANNUAL CONFERENCE OF CELL THERAPY TRANSPLANT CANADA 9
CTTC Head Office: 750 West Pender Street, Suite 301, Vancouver, BC, V6C 2T7 • T: 604-874-4944 F: 604-874-4378 E: info@cttcanada.org W: www.cttcanada.orgSCIENTIFIC PROGRAM
JUNE 13-16, 2021
All times in this program are listed in Eastern Time.
Please note that on the virtual meeting platform,
DAY 2: JUNE 15, 2021 times will automatically display in your timezone.
ORAL ABSTRACT PRESENTATIONS
11:45am – 1:15pm ACCREDITATION HOURS: 1.5
Chairs: Wilson Lam, MD, FRCPC, Ronan Foley, MD, FRCPC
1:15pm – 1:45pm General Networking
1:45pm – 2:45pm ABSTRACT POSTER PRESENTATION
3:15pm – 4:00pm Networking: Self-Led Roundtable Discussions
4:00pm – 5:00pm CTTC AGM
DAY 3: JUNE 16, 2021
BASIC SCIENCE SYMPOSIUM LABORATORY SYMPOSIUM
ACCREDITATION HOURS: 2.25 Chairs: Diane Fournier, PhD,
Chairs: Frédéric Barabé, MD, FRCPC, Patrick Trepanier, PhD, MBA
Jean-Sébastien Delisle, MD, FRCPC, PhD • Advanced Regenerative Medicine and Cell
• Expanding Stem Cells: From Lab to Clinic – Guy Therapies and the Role of Canadian Cell Therapy
Sauvageau, MD Centers – Martin Giroux, PhD
• Q&A • Management of Laboratory Nightmares – Jelena
Holovati, PhD
10:50am – 11:15am Break • Processing Using the Miltenyi CliniMACS Plus-
10:00am – 1:00pm
• Mechanisms of Leukemia Immune Evasion Understanding and Optimizing Cell Recoveries
and Relapse After Hematopoietic Stem Cell – Susan Berrigan, MLT
Transplantation – Luca Vago, MD, PhD • Q&A
• Q&A
11:30am – 1:00pm Break
12:00am – 12:15pm Break
• Hematopoietic Stem Cell Gene Therapy for Primary
Immune Deficiencies – Donald Kohn, MD
• Q&A
1:00pm – 1:15pm General Networking
2:15pm – 2:45pm General Networking
ENABLING INNOVATION SYMPOSIUM
ACCREDITATION HOURS: 1.5
Chair: Jean-Sébastien Delisle, MD, FRCPC, PhD
2:45pm – 4:15pm
• Cord Blood Transplantation Using Expanded Stem Cells Using UM171 – Sandra Cohen, MD
• Autologous Hematopoietic Stem Cell Transplantation for Autoimmune Disease – Harold Atkins, MD
• Q&A
4:15pm – 4:30pm CLOSING REMARKS
ANNUAL CONFERENCE OF CELL THERAPY TRANSPLANT CANADA 10
CTTC Head Office: 750 West Pender Street, Suite 301, Vancouver, BC, V6C 2T7 • T: 604-874-4944 F: 604-874-4378 E: info@cttcanada.org W: www.cttcanada.orgSCIENTIFIC PROGRAM
JUNE 13-16, 2021
SESSION SUMMARIES
PRE-CONFERENCE SESSIONS (JUNE 13):
BMT101 STREAM 1 – HEALTHCARE Learning Objectives:
PROFESSIONALS • Review the principles and modalities of pre-transplant
preparative conditioning regimens.
June 13, 2021 | 10:00AM – 11:30AM Eastern
• Describe the differences in regimen intensity on outcomes.
• Highlight the considerations in choosing an appropriate
THOSE MAGICAL CELLS – WHAT THEY ARE & WHAT
conditioning regimen.
THEY DO
Nicole Prokopishyn, PhD SOCIETAL IMPACT OF GVHD
The presentation will focus on the essentials of cellular therapy from Jonas Mattsson, MD, PhD
the perspective of the collection and processing of hematopoietic Chronic graft versus host disease (cGVHD) is a debilitating and costly
stem cells and other cell types. Our journey will include key basics on complication following haemopoietic stem cell transplantation (HSCT).
collection of stem cells used in transplant and T-cells used in CAR-T Treatment with HSCT is paradoxical since we want some cGVHD (i.e.
therapy. We will also journey into the laboratory and learn about the mild cGVHD) to avoid relapse of the underlying disease, but it is pivotal to
various processes that prepare the cells for optimal use in transplants, avoid more severe forms. This presentation will look at the background
including cryopreservation, red cell reduction, cell enrichments and of cGVHD, risk factors, increasing numbers of patients suffering from
depletions, and genetic modification. cGVHD and the societal impact of this severe complication following
HSCT. I aim to present a recent study of the societal impact of cGVHD
Learning Objectives:
in the Swedish population, which also has healthcare implications in
• General understanding of the sources and types of cells used in Canada. Direct costs associated with specialized healthcare utilization
hematopoietic stem cell transplants and other cellular therapies. (inpatient admissions and outpatient visits), as well as indirect costs
• Overview of processing procedures and quality controls used in due to illness-related absences and resulting productivity loss were
the cellular therapy processing facility. estimated in patients who underwent allogeneic HSCT in Sweden
• Introduction to cell manipulation and manufacturing for the between 2006 and 2015, linking population-based health and
generation of personality cellular therapy products for treatment economic registers. To capture the period of cGVHD, patients were
and cure of disease. included who survived more than 182 days post-HSCT (start of follow-
up), and cGVHD was classified based on patient treatment records
CONDITIONING REGIMENS IN TRANSPLANTATION to correct for any diagnosis underreporting. Patients were classified
AND CELLULAR THERAPY as “non-cGVHD” if they received no immunosuppressive treatment,
Michael Kennah, MD FRCPC “mild-cGVHD” if they received only systemic corticosteroid treatment
or immunosuppressive treatment, or “moderate-severe cGVHD” if
Conditioning regimens serve an essential role in hematopoietic cell they received extracorporeal photopheresis (ECP) only, corticosteroid
transplantation in providing tumor eradication, aiding engraftment treatment and immunosuppressive treatment, or systemic
and preventing rejection. Many patient, disease and treatment corticosteroid treatment and ECP treatments. Patients with moderate-
variables must be considered in optimal selection of a preparative severe cGVHD spent more time in healthcare, had higher healthcare
regimen. This presentation will review the principles of conditioning resource costs and higher illness-related absence productivity loss
and the critical elements during selection. compared to patients with non- or mild cGVHD.
ANNUAL CONFERENCE OF CELL THERAPY TRANSPLANT CANADA 11
CTTC Head Office: 750 West Pender Street, Suite 301, Vancouver, BC, V6C 2T7 • T: 604-874-4944 F: 604-874-4378 E: info@cttcanada.org W: www.cttcanada.orgSCIENTIFIC PROGRAM
JUNE 13-16, 2021
Learning Objectives: USING MINDFULNESS TO SUPPORT WELLNESS
Leeza Friedman-Prokopishyn, MSW, RCSW
• Describe risk factors for chronic GvHD.
• Treatment options. The use of meditation as an adjunctive support to traditional medical
• Societal impact of chronic GvHD. care was a new area of study when Jon Kabat-Zinn founded the
Stress Reduction Clinic and the Center for Mindfulness Medicine at
the University of Massachusetts Medical School. Since then, research
BMT101 STREAM 2 – PATIENT, FAMILY, into the benefits of mindfulness for patients and clinicians alike, has
AND CAREGIVERS exploded in clinical research. That said, how does the average person
begin to use mindfulness to support stress reduction and traditional
June 13, 2021 | 10:00AM – 11:30AM Eastern medical care? This presentation hopes to provide information and an
introduction to mindfulness practice.
CHRONIC GVHD: WHAT MATTERS FROM THE
PATIENTS’ VIEW Learning Objectives:
Meredith Cowden, LPCC-S
• Learn some of the practice uses of mindfulness to support
A discussion about the patient experience of living long-term with the wellness.
after-effects of a bone marrow transplant, with a focus on the importance • Engage in experiential mindfulness practice.
of enhancing communication and collaboration between providers and • Learn about next steps to Learn more about mindfulness
patients. This presentation identifies the long-term implications of being practice.
a bone marrow transplant survivor, and how gaps in communication
lead to lack of understanding and awareness of patients’ experiences. THERAPEUTIC INNOVATIONS IN CELL THERAPY AND
Meredith proposes ideas to facilitate increased communication and TRANSPLANTATION
collaboration to further increase understanding of patients as well as Jean-Sebastien Delisle, MD, FRCPC, PhD
promote efforts to develop and maintain long-term support for patients
following a transplant. The presentation will summarize in accessible terms the dominant
trends in the field of cellular therapies and transplantation. Concrete
Learning Objectives: examples of these leading therapeutic innovations will be described,
highlighting recent contributions from Canada. Finally, the basics
• Create increased awareness around patient experience concerning the logistics and scientific challenges to be overcome to
following transplantation. make these therapies more available, more efficacious and less toxic
• Identify areas that create gaps in communication between will be described.
providers and patients.
• Recognize ways in which these gaps decrease understanding Learning Objectives:
and increase disparity among providers and patients regarding
perspective of patients’ health and wellbeing. • To understand the current landscape of cell therapy approaches
• Identify ways of facilitating open discussion to increase effective to treat blood cancers.
communication and collaboration between providers and • To identify the major limitations in the design and implementation
patients. of cell therapies and novel transplantation approaches.
ANNUAL CONFERENCE OF CELL THERAPY TRANSPLANT CANADA 12
CTTC Head Office: 750 West Pender Street, Suite 301, Vancouver, BC, V6C 2T7 • T: 604-874-4944 F: 604-874-4378 E: info@cttcanada.org W: www.cttcanada.orgSCIENTIFIC PROGRAM
JUNE 13-16, 2021
FAIR FIGHT FOR ALL POPULATION STUDY IN PARTNERSHIP WITH
FIRST NATIONS TO ADDRESS THE CHALLENGE OF
June 13, 2021 | 12:00PM – 1:00PM Eastern
DIVERSITY
DIVERSITY SAVES LIVES Nadia Baillargeon, MT
Mai Duong, BAA & Christiane Rochon, BAA The Stem Cell Donor Registry is made up primarily of Caucasian
During this presentation, we will discuss: individuals, as is the case with international registries. This is a major
issue since the characteristics of transplanted stem cells must match
• How Swab The World’s patient-led initiative can contribute to those of the patient as closely as possible. Because of a genetic profile
a concrete patient partnership between Swab The World and that is unique in the world, First Nations are very poorly represented
health professionals. in Canadian registries. The limited existing data on their HLA typing
• The lack of diversity in stem cell registries around the world. makes searches even more complex, as it is difficult to evaluate the
• How Swab The World was born. various compatible combinations. A research study in partnership
• How Swab The World diversifies stem cell registries, educates with First Nations was launched few years ago to address this issue.
young people about stem cell donation, and advocates for
patients looking for stem cell donors. IMPACTFUL PATIENT DONOR AND FAMILY
• How establishing a partnership between Swab The World and PARTNERSHIP IN RESEARCH- THE CDTRP
health professionals will increase patient advocacy and tackle EXPERIENCE
the lack of ethnic diversity in stem cell registries.
Mélanie Dieudé, PhD
At the end of this presentation, participants will be able to: Since 2015 the Canadian Donation and Transplantation Research
• Outline Swab The World’s mission and goals. Program has developed a strategy to give patients, caregivers and
• Explain how our stem cell registries’ lack of ethnic diversity living donors a voice while offering an environment to collaborate
makes it much more challenging for patients of underrepresented with investigators, project leads and CDTRP as a whole. Now a
ethnic groups to find stem cell donors. central feature of the CDTRP, meaningful patient, family and donor
• Describe how Swab The World’s patient advertising campaigns participation in research is now a priority in all CDTRP projects,
can empower patients by helping them reach potential stem cell contributing to the transfer of knowledge and positive impact on
donors and enabling them to share their stories with people transplantation and donation in Canada.
around the world.
• Identify several ways in which, by partnering up with Swab The
World, health professionals can continue empowering patients
and tackling the lack of ethnic diversity in our stem cell registries.
• Use the information provided in this presentation to encourage
patients to work with Swab The World if they would like
assistance with their patient campaign to find a stem cell donor.
ANNUAL CONFERENCE OF CELL THERAPY TRANSPLANT CANADA 13
CTTC Head Office: 750 West Pender Street, Suite 301, Vancouver, BC, V6C 2T7 • T: 604-874-4944 F: 604-874-4378 E: info@cttcanada.org W: www.cttcanada.orgSCIENTIFIC PROGRAM
JUNE 13-16, 2021
COVID-19 SYMPOSIUM COVID-19 AND THE HEALTH & WELLBEING
OF HEALTHCARE WORKERS IN A PEDIATRIC
June 13, 2021 | 3:30PM – 5:00PM Eastern
HAEMATOLOGY/ONCOLOGY SETTING
COVID-19 IN HCT AND CAR-T CELL THERAPY Erin Plenert, MPH
RECIPIENTS Healthcare workers have been at the heart of the COVID pandemic
Akshay Sharma, MBBS for over a year now, facing unprecedented challenges both at work
and in their homes. This presentation will provide an overview of
Dr. Sharma will discuss the results of a CIBMTR study describing
a mixed-methods research study that was done with over 200
outcomes of HCT recipients who developed COVID-19. He will
healthcare workers from a pediatric haematology/oncology unit in the
summarize the results of studies that evaluate risk factors and
first year of the pandemic. This presentation will explore the impact
variables affecting survival in HCT and CAR-T cell therapy recipients
of COVID on individuals’ mental health and wellbeing, identify both
who develop COVID-19. He will also share other updates in the field
protective and risk factors associated with emotional distress, as well
of transplantation and cellular therapy as they relate to COVID-19.
as illustrate potential strategies for managing unexpected stress and
Describe the outcomes of HCT and CAR-T cell therapy recipients who
supporting those workers most vulnerable to experiencing it.
developed COVID-19.
Learning Objective: Learning Objectives:
• To describe the impact of COVID-19 on healthcare workers’
• Discuss risk factors and variables affecting survival in HCT and
mental health and wellbeing.
CAR-T cell therapy recipients who develop COVID-19.
• To identify factors that may increase or decrease a healthcare
COVID-19 VACCINES IN STEM CELL TRANSPLANTS worker’s risk of experiencing emotional distress during the
pandemic.
Deepali Kumar, MD, MSc, FRCPC
• To discuss potential strategies.
There are two types of COVID-19 vaccines approved in Canada - mRNA
and adenoviral vector vaccines. Other types of vaccines are under
development. All vaccines show excellent efficacy in the prevention
of severe COVID in the general population. However, their efficacy
and safety in immunosuppressed populations is largely unknown.
Immunogenicity is likely to be lower in such populations. Timing of
vaccination in relation to transplant is an important consideration. In
this presentation, we will review the science behind COVID vaccines
and data that led to their licensure. We will discuss vaccination of
immunosuppressed populations, and any data that is available to
assess immunogenicity and safety of these vaccines in HSCT.
Learning Objectives:
• Learn about the development and availability of COVID-19
vaccines.
• Be aware of the considerations for COVID-19 vaccine use in the
transplant setting.
• Review up-to-date data on immunogenicity and safety of vaccine
in the HSCT setting.
ANNUAL CONFERENCE OF CELL THERAPY TRANSPLANT CANADA 14
CTTC Head Office: 750 West Pender Street, Suite 301, Vancouver, BC, V6C 2T7 • T: 604-874-4944 F: 604-874-4378 E: info@cttcanada.org W: www.cttcanada.orgSCIENTIFIC PROGRAM
JUNE 13-16, 2021
PRESIDENTIAL SYMPOSIUM CANADIAN LED IMMUNOTHERAPIES IN CANCER:
THE CLIC EXPERIENCE
June 13, 2021 | 7:00PM – 8:30PM Eastern
Kevin Hay, MD, MSc, FRCPC
PRESIDENTIAL SYMPOSIUM CAR-T cells have revolutionized our standard approach to the
Kristjan Paulson, MD, MSc, FRCPC treatment of relapsed/refractory B-cell malignancies, with newer
CAR-T cells for diseases such as myeloma and solid tumours either
Dr. Paulson will review the CTTC organization’s vision for incorporating
approaching regulatory approval or in the clinical trial development
novel cell therapies, such as CAR-T, into all CTTC activities.
stage. Canadian cancer immunology researchers are at the forefront
Learning Objectives: of developing improved approaches for these T-cell therapies, but
translation of their work to Canadian patients often is delayed by
• Review the CTTC vision for cellular therapy in Canada. lack of access to a Canadian clinical cell therapy network. CLIC, or
• Discuss the CTTC white paper for the implementation of CAR-T Canadian Led Immunotherapies in Cancer, was born out of this need
therapy in Canada. and supported by BioCanRx. Using a basic CD19 CAR as a starting
point, we have established centralized plasmid and lentiviral vector
PRACTICAL CONSIDERATIONS OF CAR-T THERAPY manufacturing capacity with point-of-care T-cell manufacturing for
IN ADULT LYMPHOMA a fully “made in Canada” CAR-T cell therapy. This initial trial, CLIC-
Ronan Foley, MD, FRCPC 01, is ongoing and enrolling patients with relapsed/refractory B-cell
Onboarding for CAR-T therapy is complex and requires a multi- malignancies in Vancouver and Ottawa. Working with the National
disciplinary strategy to “set up.” Education, policies and procedures Research Council of Canada, we have developed a novel camelid
are required to meet FACT/HC and industry standards. Beyond the single domain-based CD22 CAR which is on track to be in the clinic
cell therapy institution and emphasis on referring centers to ensure in 2022. Future directions of our program include targeting multiple
best patient selection. Moreover important management decisions antigens for B-cell malignancies as well as the development of CARs
post day 30 including management of infectious complications and and transgenic T-cell receptors (TCRs) for solid tumours. We are
how to best evaluate any residual disease post day 30 CT/PET. This actively onboarding new sites for both manufacturing and clinical
session will focus on clinical CAR-T including the latest products administration, with a goal of making a TransCanada Highway for
available in Canada. CARs.
Learning Objectives: Learning Objectives:
• Review basic concepts in CAR-T cell therapy. • Identify the challenges and opportunities associated with
• Evaluate steps and experience in the onboarding process. setting up academic CAR-T cell manufacturing in Canada.
• Discuss patient selection and early referral. • Describe the CLIC point-of-care manufacturing model.
• Understand the clinical needs of patients post d 30. • Discuss the ongoing CD19 (CLIC-01) and upcoming CD22 (CLIC-
02) CAR-T cell trials.
ANNUAL CONFERENCE OF CELL THERAPY TRANSPLANT CANADA 15
CTTC Head Office: 750 West Pender Street, Suite 301, Vancouver, BC, V6C 2T7 • T: 604-874-4944 F: 604-874-4378 E: info@cttcanada.org W: www.cttcanada.orgSCIENTIFIC PROGRAM
JUNE 13-16, 2021
DAY 1 SESSIONS (JUNE 14):
HANS MESSNER LECTURESHIP Learning Objectives:
SYMPOSIUM • Define indication for allogeneic stem cell transplantation in AML
and MDS.
June 14, 2021 | 10:00AM – 1:00PM Eastern
• Define risk factors for non-relapse mortality and relapse.
• Getting familiar with treatment options to reduce risk of relapse.
ALLOGENEIC STEM CELL TRANSPLANTATION IN
• Impact of intensity of pre-transplant conditioning regimen.
AKUTE LEUKEMIA AND HIGH RISK MDS
Nicolaus Kröger, MD APPROACHES IN MDS AND AML FOR PATIENTS NOT-
Allogeneic stem cell transplantation (AHSCT) is the most effective post- ELIGIBLE FOR TRANSPLANTATION
remission therapy for patients with Acute Myeloid Leukemia (AML) and Julie Bergeron, MD, FRCPC
the only curative treatment for high risk MDS. However, due to the high
We will explore the sequencial options available for patients who are
morbidity and also mortality associated with AHSCT, this procedure is
not transplant candidates. Options that are available now, others that
clearly indicated for patients with poor and very poor risk AML, while for
are hopefully coming soon, in terms of accessibility, and a bit of what
intermediate risk patients patient-related factors such as comorbidities
lies ahead.
and age as well as donor availability has to be taken into account.
However results of using alternative donor such as mismatched Learning Objectives:
unrelated, cord blood and more recently haploidentical donor has
been improved substantially and the issue of the optimal donor is • Be (more) familiar with the standard and putatively soon to be
currently a matter of debate. Especially the results of haploidentical standard treatment approaches for AML patients that are not
stem cell transplantation by using cyclophophamide post-transplant as transplant candidates.
GvHD prophylaxis has shown very encouraging outcome. AML patients • Be (more) familiar with some specific adverse effects of the new
with favorable risk receive in general chemotherapy as consolidation, agents that are part of the above-implied ormamentarium.
although more recent studies have shown a very favorable outcome • Learning (more) about new agents that are the most promising in
after AHSCT. The role of the intensity of the conditioning regimen is still the field of acute leukemia and MDS.
not solved. Randomized studies resulted in inconsistent results. The
value of pre-transplant treatment with chemotherapy or HMA in MDS is MULTIPLE BREATH WASHOUT TESTING TO MONITOR
still a matter of debate. The transplant community is preferring reduced LUNG FUNCTION AFTER BMT
intensity regimens in older patients or those with comorbidities. Due Jonathan Rayment, MDCM, MSc, FRCPC
to better management of infectious complication, toxicity as well as Pulmonary chronic graft versus host disease (cGVHD) contributes
graft-versus-host disease the non-relapse mortality has substantially significantly to morbidity and mortality after blood and bone marrow
decreased in the last years. The major treatment failure after AHSCT transplantation (BMT). Early identification of this condition can lead
is now relapse and MRD pre-transplant is one of the major risk factors to earlier intervention and better outcomes. Current pulmonary
for relapse post allograft. Current clinical studies are focusing on function testing modalities, specifically spirometry, are often too
prevention of relapse by monitoring minimal residual disease and difficult for young children to perform and are also likely insensitive
using post-transplant cellular or drug approaches. Recent published to early physiologic abnormalities in pulmonary cGVHD. Better tools
studies with FLT3 inhibitors in FLT3 positive AML or low doses HMA in are needed to monitor for lung disease in children after BMT. Multiple
combination with DLI have shown encouraging results. breath washout (MBW) testing is a non-invasive measure lung function.
Is has been demonstrated to be feasible in young children and is highly
ANNUAL CONFERENCE OF CELL THERAPY TRANSPLANT CANADA 16
CTTC Head Office: 750 West Pender Street, Suite 301, Vancouver, BC, V6C 2T7 • T: 604-874-4944 F: 604-874-4378 E: info@cttcanada.org W: www.cttcanada.orgSCIENTIFIC PROGRAM
JUNE 13-16, 2021
sensitive to ventilation inhomogeneity. We hypothesised that this tool the implementation of CAR-T in Maisonneuve-Rosemont Hospital and
would be feasible in the paediatric BMT population and would be unmet needs for CAR-T in Canada.
sensitive to the physiologic changes seen in cGVHD. In this talk, I will
present data from two studies. First I will present a pilot cross sectional Learning Objectives:
study looking at the ability of this technology to distinguish patients • Review the implementation of CAR-T in clinical practice in
with clinically diagnosed pulmonary cGVHD from those without. Quebec.
Second, I will present preliminary data from an ongoing longitudinal • Discuss patient selection and referral.
study, further characterizing the utility of this tool to monitor lung • Describe the challenges of toxicity management Identify unmet
function in children after BMT. needs for CAR-T in Canada.
Learning Objectives:
NK CELLS AS A NOVEL ALTERNATIVE FOR
• Understand the physiology behind the multiple breath washout IMMUNOTHERAPY: TOWARDS EFFECTIVE, OFF-
(MBW) test. THE-SHELF AND SAFE OPTIONS FOR CANCER
• Review the discriminative power of MBW to identify pulmonary TREATMENT
cGVHD.
Luciana Melo Garcia, MD
• Understand the potential role of MBW to monitor lung function
longitudinally after BMT. Adoptive cell therapy has had impressive outcomes in patients with
hematologic malignancies. CAR-T cell therapy has revolutionized
outcomes of patients with B-cell malignancies, but has notable
CAR-T AND THE FUTURE OF CELL drawbacks including manufacturing time, autologous T-cell
THERAPY SYMPOSIUM harvesting and genetic manipulation from heavily pre-treated
patients, and cost. In this context, NK cells are an effective and safe
June 14, 2021 | 4:00PM – 6:00PM Eastern alternative to T-cells. Allogeneic sources, such as cord-blood NK
cells, are a feasible and renewable off-the-shelf option. They can
STATE OF THE NATION OF CAR-T be genetically manipulated to express CARs and have directed anti-
Isabelle Fleury, MD, MSc, FRCPC tumor activity without causing graft-versus-host disease or eliciting
Immune effector cells (IEC) have changed the therapeutic landscape cytokine release syndrome. In addition, they can be genetically
in many hematologic malignancies. Initial reports of the success modified to express cytokines that improve their cytotoxicity and
of CAR-T cell in refractory or relapsing B-cell acute lymphoblastic persistence. In this presentation, we will explore the novel genetic
leukemia and large B-cell non-Hodgkin lymphomas have launched and non-genetic approaches to improve NK cell engineering for the
a new era. A countless number of clinical trials involving IEC now treatment of cancers.
addresses an extensive field of new clinical indications. Enhancement
Learning Objectives:
of IEC construct and manufacturing process provides further
opportunities. IEC however carry a new toxicity profile representing • Understand NK cell biology.
a challenge in implementation. Cytokine release syndrome and • Describe the relevance of NK and T-cell as options for cellular
immune effector cell-associated neurotoxicity syndrome are some therapy.
of the acute toxicities that deserve specific interventions in order to • Explore possible NK cell-based strategies to improve adoptive
maintain CAR-T benefit and limit life-threatening toxicities. Access cellular therapy.
to CAR-T is actually restricted to a minority of patient, mainly due
to limitations related to patient comorbidities and disease kinetics.
Improvement in our understanding of disease escape to CAR-T will
likely improve the efficacy of IEC-based therapy. Dr Fleury will review
ANNUAL CONFERENCE OF CELL THERAPY TRANSPLANT CANADA 17
CTTC Head Office: 750 West Pender Street, Suite 301, Vancouver, BC, V6C 2T7 • T: 604-874-4944 F: 604-874-4378 E: info@cttcanada.org W: www.cttcanada.orgSCIENTIFIC PROGRAM
JUNE 13-16, 2021
TRANSLATING ENGINEERED T-CELLS FROM BENCH GENOME EDITING IN HEMATOPOIETIC STEM CELLS
TO BEDSIDE Josée Laganière, PhD
Stanley Riddell, MD Autologous hematopoietic cell transplantation coupled to
The presentation will discuss issues related to the clinical translation genome editing holds promise for curing patients affected by
of T-cells engineered with synthetic receptors for adoptive hemoglobinopathies, such as sickle cell disease. Experimental
immunotherapy of cancer. Topics will include target selection, models are important research tools to assess the impact of the
receptor optimization, cell composition, preclinical models and ever-growing number of genome editing modalities and to develop
design and interpretation of correlative studies that inform next new treatments. There remains a need for refining in vitro human
generation therapies. The focus will be on hematologic malignancies erythropoiesis to assay the functionality and integrity of gene-edited
including multiple myeloma. red blood cells (RBCs). We developed a feeder-free, cell culture
protocol for the erythroid differentiation of human hematopoietic stem
Learning Objectives: and progenitor cell (HSPCs) that is compatible with genome editing
approaches. The method enabled an intense erythroid progenitor
• Principles to consider in designing tumor targeting receptors.
proliferation, a high enucleation efficiency, and a near-complete
• The role of T-cell subsets and differentiation for persistence of
reticulocyte maturation into erythrocytes. These conditions allowed
transferred T-cells.
for the non-viral, selection-free insertion of the sickle mutation at the
• Novel methods of cell manufacturing to maintain metabolic
beta globin locus (HbB) and resulted in RBCs with a distinct crescent
fitness.
shape cell morphology, recapitulating an important hallmark of the
• Design and interpretation of first in human clinical trials and
disease. The editing conditions developed are also compatible with
utility of correlative laboratory studies.
other HSPC culture conditions. We foresee that the distinct features
of this system will facilitate the functional study of cultured RBCs
INNOVATION IN GENOME EDITING following the introduction of various traits via gene editing.
SYMPOSIUM Learning Objectives:
June 14, 2021 | 7:45PM – 8:45PM Eastern • Learn about genome editing.
• Review hematopoietic stem cell biology and differentiation
CRISPR-CAS SYSTEMS: FROM HUMBLE BEGINNINGS concepts.
TO TODAY’S HEADLINES • Understand current limitations of erythropoiesis or in vitro red
Sylvain Moineau, OC, OQ, PhD, FRSC blood cell production.
Fighting viruses is no easy task. Bacterial cells have survived phage • Discover the potential of CRISPR-Cas9 for the study of gene
attacks by evolving sophisticated defence strategies that enable function and for the modelling of hematological traits.
them to thrive even in virus-rich ecosystems. Clustered regularly
interspaced short palindromic repeats (CRISPR) and their associated
cas genes protect microbial cells against foreign nucleic acids such
as phage genomes and plasmids. Exploiting this natural system has
resulted in the development of the much-publicized CRISPR-Cas9
technology for precise genome manipulation of various organisms.
This seminar will present the differences between the CRISPR-
Cas systems and the CRISPR-Cas9 technology as well as their
applications.
ANNUAL CONFERENCE OF CELL THERAPY TRANSPLANT CANADA 18
CTTC Head Office: 750 West Pender Street, Suite 301, Vancouver, BC, V6C 2T7 • T: 604-874-4944 F: 604-874-4378 E: info@cttcanada.org W: www.cttcanada.orgSCIENTIFIC PROGRAM
JUNE 13-16, 2021
DAY 2 SESSIONS (JUNE 15):
FRED SAUNDERS LECTURESHIP Learning Objectives:
SYMPOSIUM • Theoretical advantages of gene therapy over allogeneic
transplantation.
June 15, 2021 | 10:00AM – 11:00AM Eastern
• Theoretical advantages of gene editing over addition of
therapeutic genes.
GENE THERAPY FOR β-HEMOGLOBINOPATHIES:
• Gene therapy treatment for beta thalassemia and sickle cell
LENTIVIRAL AND GENOME EDITING APPROACHES disease.
Marina Cavazzana, MD • Results of ongoing clinical trials.
β-thalassemia and sickle cell disease (SCD) are the most widespread •
monogenic diseases, and are caused by quantitative or qualitative
defects in the adult hemoglobin production. Gene therapy approaches
represent an alternative to allogenic hematopoietic stem cell (HSC)
transplantation in the absence of a compatible donor. The generation
of lentiviral vectors (LVs) carrying a β-globin like gene revolutionized
this field, as they allow an effective HSC transduction. Several
clinical trials are ongoing all over the world for both the diseases,
employing different kind of vectors; the first results are promising
in terms of improvement of biological parameters and quality of life,
with sustained production of therapeutic hemoglobin and reduced
requirement of transfusions. Technical improvements are in progress
to further ameliorate the transduction process. In terms of safety
issues, no genotoxicity event has been reported for the patients
affected by transfusion dependent thalassemia. Conversely, three
severe adverse events (SAE) have been reported in SCD patients (ie
1 LAM, 1 MDS). In two of the three cases the lentiglobin vector did
not seem to be the driver element while in the third we are awaiting
for more detailed information by the sponsor of the clinical study.
These SAE likely pinpoint a particular risk for patients affected by
SCD, which deserves further deep investigations. Novel LV-based
strategies aiming at reactivating endogenous fetal hemoglobin
(HbF) represent another promising approach, as elevated HbF levels
can ameliorate the severity of both β-thalassemia and SCD. Finally,
genome editing approaches aimed at correcting the disease-causing
mutation or reactivating HbF are currently under investigation. Here,
we discuss the clinical outcomes of current LV-based gene addition
trials and the potential advantages of novel alternative therapeutic
strategies.
ANNUAL CONFERENCE OF CELL THERAPY TRANSPLANT CANADA 19
CTTC Head Office: 750 West Pender Street, Suite 301, Vancouver, BC, V6C 2T7 • T: 604-874-4944 F: 604-874-4378 E: info@cttcanada.org W: www.cttcanada.orgSCIENTIFIC PROGRAM
JUNE 13-16, 2021
DAY 3 SESSIONS (JUNE 16):
BASIC SCIENCE SYMPOSIUM MECHANISMS OF LEUKEMIA IMMUNE EVASION
AND RELAPSE AFTER HEMATOPOIETIC STEM CELL
June 16, 2021 | 10:00AM – 1:00PM Eastern
TRANSPLANTATION
EXPANDING STEM CELLS: FROM THE LAB TO THE Luca Vago, MD, PhD
CLINIC Despite the constant improvement in the outcome of allogeneic
Guy Sauvageau, MD, PhD, FRCPC hematopoietic stem cell transplantation (allo-HSCT), post-
transplantation relapses remain frequent. No consensus to date
The mechanisms that govern the expansion of human blood stem cells
is available on the optimal therapy for relapses, and most of the
are poorly characterized, with the consequence that bone marrow
strategies currently in use (infusion of donor lymphocytes, salvage
transplantation remains unavailable to a subset of patients for which
chemotherapy, second transplantation) have yielded discouraging
graft size is too small, resulting in prolonged engraftment and often
results. There is growing scientific evidence that post-transplantation
lethal complications. We previously identified and developed a small
relapse might represent the expression of mechanisms of immune-
molecule called UM171, which acts as a potent mediator of stem cell
resistance enacted by leukemic cells to evade the control mediated
expansion with important clinical benefits, as proven in a series of
donor-derived immune system. During the presention, we will review
completed and ongoing clinical trials. More recently, we identified a
current knowledge regarding genomic and epigenetic mechanisms of
novel protein complex called CRL3KBTBD4 which, under exposure to
immune escape and relapse, discuss how to integrate this biological
UM171, is responsible for the degradation of another protein complex
information into clinical decision-making, and propose innovative
called CoREST1 that plays an important role in the “epigenetic”
strategies to circumvent immune escape with precision medicine
control of key stem cell genes. Epigenetic marks (together referred
approaches.
to as the epigenome) are modifications to DNA and proteins that turn
genes on or off. CRL3KBTBD4 activation by UM171 prevents the loss Learning Objectives:
of key epigenetic marks in human blood stem cells that undergo
amplification in culture. This work positions the KBTBD4-COREST1 • Gain knowledge on known mechanisms of post-transplantation
axis as a critical mediator of stem cell amplification. We will present relapse, and on assays used for their differential diagnosis.
basic and clinical data indicating that UM171 provide rejuvenation of • Gain new principles for the selection of salvage treatments in
stem cell grafts. patients with post-transplantation relapse.
Learning Objectives: HEMATOPOIETIC STEM CELL GENE THERAPY FOR
• Learn the potential benefits of stem cell expansion.
PRIMARY IMMUNE DEFICIENCIES
• Identify patients who may receive amplified stem cell grafts. Donald Kohn, MD
• Learn the epigenetic marks associated with stem cell attrition. Approaches to genetically modifying autologous hematopoietic stem
cells (HSC) for gene therapy of primary immune deficiencies (PID)will
be presented. Results from trials using lentiviral vectors for ADA SCID
and other PID will be presented. New approaches using gene editing
to make precise genetic changes in HSC will also be described.
ANNUAL CONFERENCE OF CELL THERAPY TRANSPLANT CANADA 20
CTTC Head Office: 750 West Pender Street, Suite 301, Vancouver, BC, V6C 2T7 • T: 604-874-4944 F: 604-874-4378 E: info@cttcanada.org W: www.cttcanada.orgYou can also read
