Process Development for Production of Photoreceptor Cells to Treat Blindness
Millions of people worldwide live with severe degenerative diseases of the eye leading to progressive vision impairment and eventually total blindness. The majority of these inherited and acquired degenerative diseases affect the light- sensing tissue at the back of the eye, the retina, that contains the rod and cone photoreceptors.
The study furthered research into the possibility of restoring vision for patients with blinding eye disease, using stem cell transplantation. A renewable source of stem cells and an unlimited supply of stem cell-derived products, such as the photoreceptors, will replenish the cells lost during disease progression and restore the vision. To date the research team have demonstrated proof-of-concept for the transplantation of mouse and human stem cell-derived photoreceptors to rescue visual perception in mice. In this project the research team will move this therapy closer to implementation.
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This project provides preliminary data which will one day help to restore the sight of patients with degeneration or loss of the light-sensing photoreceptor cells by replacement of photoreceptor cells derived from good manufacturing practice (GMP)-compatible allogeneic induced pluripotent stem cells (iPSCs). The availability of the stem cell product and the research team’s experience in stem cell biology and cell transplantation will add to the pre- clinical data of cell therapy as the supportive evidence for a clinical trial application in the future.
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Lead Investigator
- Dr Anai Gonzalez Cordero, Group Leader Stem Cell Medicine Group, Children’s Medical Research Institute, The University of Sydney
Research Team
- Prof. Patrick Tam, Embryology Research Unit, Children’s Medical Research Institute
- Dr Ngaire Elwood, BMDI Cord Blood Bank, Cord Blood Stem Cell Research Group, Murdoch Children’s Research Institute
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This initial research provided the supportive evidence to attract further funding from the Medical Research Future Fund (MRFF)
The MRFF provided $2.5m funding for the Development of photoreceptor cell therapy to treat blindness 2022-2027.
This research lays the foundation of retinal cell therapy for the treatment of blinding eye diseases caused by the degeneration of the light-sensing cells in the eye. A renewable source of human stem cells will be created from which transplantable photoreceptor cells will be produced for regenerative therapy of the retina. This pre-clinical process development enables translational research in stem cell medicine and cell therapy and offers a path to clinical trials of retinal cell therapy.Milestone initiatives of the MRFF funding to date include:
- 23 March 2024 inaugural Inherited Retinal Disease Patient and Family Engagement Day, held in partnership with CMRI and UNSW Sydney and supported by Luminesce Alliance
- 24 September 2024 World Retina Day launch of the top 10 research priorities for Inherited Eye Disease (IHD), supported by Vision 2020, the national peak body for eye health and vision care, along with the Parliamentary Friends for Eye Health and Vision Care.
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