Project title: Restoring glucose control in T1D patients with genetically engineered GARV-AAV2-A20-islet cells - a first in Human safety and efficacy trial
Inspired by ground-breaking discoveries in human immunology, this project offers patients with type 1 diabetes a new approach to restoring normal glucose control, developed at the Garvan Institute of Medical Research.
Type 1 diabetes (T1D) is one of the top 10 diseases of childhood and impact about 127, 000 Australians. Loss of glucose control has major long-term health consequences including kidney and cardiovascular disease, blindness and more.
Restoring normal glucose control in patients with T1D with insulin-producing islet cells therapy represents the holy grail, and while clinical islet-transplantation shows therapeutic promise, it is limited by donor cell availability and immune attack. As for all transplants, islet-cell transplant patients need to receive life-long immunosuppressive drugs to prevent immune rejection of the newly transplanted islet cells and this can lead to later health complications.
Identification of a new approach that allows islet cell transplantation without needing immunosuppression would represent a major advance in diabetes therapy.
We have studied how activity of the immune system is controlled by the gene 'A20'. Our studies led to the discovery that by increasing A20 levels in islet transplants we could prevent their immune rejection and provide life-long graft survival with minimal immunosuppression. These 'A20-islet-grafts' reversed diabetes and provided lifelong normal glucose control.
Development of a new gene therapy (GARV-AAV2-A20) that allows islet-cell transplantation with reduced or no immunosuppression would improve patient outcomes, and also increase patient access to this life-changing therapy.
TTRA Project Round: Two
State: New South Wales
- CMRI Westmead Sydney
- Sydney Children's Hospitals Network
- Royal Adelaide Hospital
- Western Sydney Health
- Westmead Health Precinct
- Sydney University
- Westmead Kids
- Children's Hospital Westmead
- Cell Technologies
- TTRA: $749,979
- Industry Contribution: $1,546,336 in-kind
Duration: September 2022 – October 2024