The London Project to Cure Blindness, launched by Professor Pete Coffey, Professor of Cellular Therapies at the Institute of Ophthalmology, University College London, has announced the treatment of a first patient in a Phase I clinical trial of a stem cell therapy to treat age-related macular degeneration. The study, commenced at Moorfields Eye Hospital, expects to have preliminary results on the first patient in December. According to a press release from the London Project, the milestone is the result of a partnership between the hospital, the UCL Institute of Ophthalmology and the National Institute for Health Research (NIHR). Originally funded by an anonymous US donor, the London Project has received additional support from Pfizer Inc. to support the translation of the research from bench to bedside.
The open-label, safety and feasibility study, formally entitled, “A Study Of Implantation Of Retinal Pigment Epithelium In Subjects With Acute Wet Age Related Macular Degeneration”, sponsored by Pfizer Inc., will inject human embryonic stem cell derived retinal pigment epithelium cells (RPE) into approximately 10 patients with acute AMD in which there has been recent rapid vision decline. According to the registered trial information, the therapy will be provided “as a Retinal Pigment Epithelium living tissue equivalent for intraocular use in the form of a monolayer of Retinal Pigmented Epithelial (RPE) cells immobilized on a polyester membrane. The membrane is approximately 6 mm x 3 mm and will contain a confluent layer of RPE cells, at a nominal dose of 17 mm2. The implant is intended to be life-long.” The primary outcome measures include a change in baseline in ETDRS best corrected visual acuity (BCVA) (proportion of subjects with an improvement of 15 letters or more at week 24) while secondary measures will include changes in BCVA at several time-points, change in leakage or perfusion in normal fundal vasculature and presence of abnormal vasculature by fundus fluorescein angiography. In addition, clinicians will assess the change in the central 30 degree of visual function by Humphrey Field test and the change in thickness of RPE layer by B-mode orbital ultrasound.
Commenting on the significant achievement, Prof. Pete Coffey of UCL Institute of Ophthalmology, stated: “We are tremendously pleased to have reached this stage in the research for a new therapeutic approach. Although we recognise this clinical trial focuses on a small group of AMD patients who have experienced sudden severe visual loss, we hope that many patients may benefit in the future.” Also commenting on the milestone, Dr Berkeley Phillips, UK Medical Director, Pfizer Ltd stated that: “great science comes through collaboration; no one person has all the answers and together, we can achieve more and move faster. Stem cell-derived therapy was only a theory until recent years, and to be part of a project that is applying the latest scientific breakthroughs to help restore patients’ eyesight is truly rewarding.”