“Stratifying treatment for diabetic macular oedema using iPS cells"
Research type
Research Study
Full title
“STRatifying trEatment for diAbetic MacuLar oedema using INduced pluripotEnt stem (iPS) cells” (STREAMLINE)
IRAS ID
162254
Contact name
Noemi Lois
Contact email
Sponsor organisation
Belfast Health and Social Care Trust
Duration of Study in the UK
1 years, 2 months, 30 days
Research summary
Diabetic macular oedema (DMO) is a common cause of blindness linked to breakdown of the blood retinal barrier. For the last 20 years the main treatment has been laser therapy but recent clinical studies have show that for patients with central retinal thickness of 400 microns or over, intra-ocular injections of antibodies against vascular endothelial growth factor (anti-VEGF, Ranibizumab) are not only effective, but more cost effective than laser therapy.
Ranibizumab is expensive and requires monthly injections which, due to the incidence of DMO, is a major burden for UK health authorities. Only ~50-60% of patients respond to Ranibizumab in randomised clinical trials but it is not currently possible to predict which patients will benefit from it and all eligible patients are injected with Ranibizumab for the first 24 months.
There is a clear unmet clinical need to stratify DMO patients and provide early clinical decisions that limit exposure of patients to costly, ineffective and risky treatments. The targeted challenge of STREAMLINE is to provide a means for assessing individual responsiveness to Ranibizumab and predict treatment effectiveness.
Induced pluripotent stem (iPS) cell technology will be used in this project to generate stem cells from individuals, which can then be differentiated into particular cell lineages known to be affected by DMO (blood retinal barrier cells of the vascular endothelium and retinal pigment epithelium). Key aspects of DMO can then be reproduced in the laboratory and patient-specific responses to Ranibizumab evaluated. The predicted "laboratory-based response" will be compared with the response observed following treatment.
An additional benefit will be derived by providing opportunities to identify novel therapeutic targets based on patient-linked experimental models, especially for patients who do not respond to VEGF blockade.REC name
HSC REC A
REC reference
14/NI/1109
Date of REC Opinion
28 Nov 2014
REC opinion
Further Information Favourable Opinion