Production of iPSC derived RPE Cells for transplantation in AMD
Research type
Research Study
Full title
Feasibility of Production of induced Pleuripotent Stem Cell derived Retinal Pigment Epithelial Cells fulfilling regulatory requirements for human transplantation in dry Age-related Macular Degeneration.
IRAS ID
158357
Contact name
Lyndon da Cruz
Contact email
Sponsor organisation
Moorfields Eye Hospital NHS Foundation Trust
Duration of Study in the UK
1 years, 0 months, 1 days
Research summary
It has been shown that adult human skin cells or human blood cells can be reprogrammed to become stem cells. This type of stem cell is called an induced Pleuripotent Stem Cell (iPSC). Theoretically, stem cells are cells that can be converted into any other cell type of the body. It has been shown specifically that this type of stem cell can be produced from adult human skin cells or blood cells and can in turn be converted into Retinal Pigment Epithelial (RPE) Cells. These RPE cells are a very important cell type in the human retina that are abnormal and ultimately lost in Age-related Macular Degeneration (AMD). Although it has been shown to be possible to make RPE cells from adult human skin, it has not been done to a level to fulfill regulatory requirements for human transplantation in AMD. In this project we aim to demonstrate that we can produce RPE cells of sufficient quality and safety for human transplantation from iPSC derived from human skin or blood from patients with early dry macular degeneration. The end point for this study is the production of RPE cells that would fulfill the regulatory requirement for transplantation.
Patients will be identified at Moorfields Eye Hospital, consented for the study and a full thickness skin biopsy and blood sample taken. The biopsies will then be transferred to Dr Nathwani (Cancer Institute) to produce iPSC in GMP Cellular Therapeutics Facility (Royal Free Hospital) and transferred to The Institute of Ophthalmology for production of RPE and safety testing.
Although outside the remit of this ethics application, we intend to make a subsequent application to the appropriate regulatory body to consider transplanting the RPE as sheets back into the patient that they were taken from and potentially into a tissue matched individual with the same condition.
REC name
London - Chelsea Research Ethics Committee
REC reference
15/LO/0622
Date of REC Opinion
16 Jul 2015
REC opinion
Further Information Favourable Opinion