Download cell and gene therapy - BC Children`s Hospital Research Institute

Who am I? A Brief Bio
• Undergrad at Simon Fraser University
Genetics: 3 summers in a Drosophila genetics lab
• PhD with John Schrader at The Biomedical Research Centre (UBC)
Biochemistry: Cytokine signal transduction
• PDF with Maria Grazia Roncarolo at the Telethon Institute for
Gene Therapy (TIGET-HSR) in Milano
Cellular immunology: cell and gene therapy
• 2003 UBC Department of Surgery (transplant immunology)
and VCHRI
• 2010 Moved to CFRI to join the Diabetes & Immunology Clusters
What is my area of research?
• how do Tregs normally work?
• how can Tregs become dysfunctional
and contribute to diseases?
• can we find “biomarkers” to track
the function of Tregs in patients
• can Tregs be given back as a cellular
therapy to restore immune regulation?
Primary immunodeficiencies, transplantation (organs & stem cells), diabetes,
obesity, Inflammatory bowel disease, other autoimmune diseases….
Why did I accept this position?
I love working at CFRI
Great colleagues
Core support (RTOD, training, core facilities etc)
Amazing opportunities for research & impact
Want to “give back” and contribute towards:
- enabling research across the campus
- the evolution of CFRI into internationally-known research
institute
- making CFRI an even more dynamic and fun place to work
CFRI Strategic Framework
• The combined activities of all the groups within
a theme should enable the over-arching institute goals:
1. Increase research capacity
2. Increase research excellence
3. Impact on children’s health
Childhood Disorders Theme
• includes Groups that are focused on studying
childhood diseases and disorders.
• focus on understanding mechanisms of health
versus disease, as well as identifying and
testing new diagnostic and therapeutic approaches.
Theme Opportunities
Specific goals to be defined during the planning process,
some initiatives to explore:
• Increase national & international recognition
- Start a new theme level seminar series
- Support participation in international conferences
• Enable investigator-initiated clinical trials
- Interventional (therapies) & observational
(biomarkers & diagnostics)
Theme Opportunities
• Facilitate multi-disciplinary research
- Enable cross-group interactions
• Support existing and new cores/platforms
- Biobanking
- Standardized immune monitoring
- iPS cells
- Exome sequencing and bioinformatics
- Humanized mouse models, other model organisms
• Training in translational research (not only for
trainees)
Purpose of today: start the transition from clusters
to groups under over arching themes
• Aim is for this to be a “grass roots” process
As of May 4th:
• 52 PIs selected Childhood Disorders as their
primary theme (35% of respondents)
• 145 out of ~300 CFRI members responded to the
visioning survey
Of the 300 CFRI members:
• 232 are on site
• 152 hold funding with CFRI as primary institution
The Main Task ahead: defining “groups”
A group is:
• ~10-20 investigators who want to work together
because they see the potential benefit for themselves
and the success of a larger program of research
• some examples of reasons to form a group:
 Enable research in the context of a large scale funding
opportunities (Foundations, CIHR-SPOR, Genome Canada, CFI, etc)
 Optimize the potential for multi-disciplinary
Research, training and mentoring in a common area of research
 Optimize the development, implementation and use of a
common technology (e.g. immune monitoring, iPS cells, imaging)
Keep in mind as we begin to define groups
• Groups are dynamic and meant to evolve
• Groups can cross themes
• Each group will have a leader
• Each group will outline specific activities they
wish to accomplish:
Type 1 activity: feasible with current resources
Type 2 activity: made possible with new resources
Possible Groups
~ 30 groups suggested on the visioning web site
Cancer
Diabetes
Childhood Cancer and Blood Research
Diabetes
Childhood cancer and blood disorders
Childhood Obesity &
Cardiometabolic Disease
Childhood Cancer
Cancer and Blood Research
Michael Cuccione Childhood Cancer
Possible Groups
Variations on infection & immunity in kids
Primary Immune Deficiency Diseases
Childhood Immunity
Inflammatory disorders
Rheumatology Research Unit
Infectious Diseases
Chronic Inflammatory Diseases
CUPIC
Possible Groups
Variations on therapies
Variations on rare diseases
Cell therapy and transplantation
Systems Biology and Rare Disease
Pediatric Transplantation
Translational Research Hub
for Rare Mendelian Disease
Cell and Gene Therapy
Therapeutic discovery
Rare Diseases
Other Group Suggestions
How best to group other suggestions:
Mechanisms of Disease
- cross-cutting groups? Theme level activity?
Obstructive Sleep Disorders
- where to place specific diseases/disorders that fall
outside large grouping (need to understand approaches
and what is needed to enable research)
Surgery
- how to best group surgeons (critical aspect of
disease research [e.g. biobanking] and many therapeutic
approaches [e.g. transplantation])
What will happen from here ?
1. Series of facilitated focus sessions with the goal of:
- Formalizing groups and identifying group leads
- Identifying the first type 1 (possible now) and type 2
(possible with more resources) activities
2. Facilitated session with group leads to:
- Define theme level initiatives / goals
- Ensure we do not re-create cluster “silos”
- Ensure we do not have theme “silos”