September 29, 2015

expert reaction to announcement of start of trial investigating safety and efficacy of potential new embryonic stem cell-derived treatment for wet age-related macular degeneration (AMD), at Moorfields Eye Hospital, as part of the ‘London Project to Cure Blindness’ project

A patient at Moorfields Eye Hospital in London has become the first to receive an experimental embryonic stem cell therapy for wet age-related macular degeneration (AMD), as part of an 18 month clinical trial to find a cure for wet AMD.

Prof. Anthony Hollander, Professor of Stem Cell Biology, University of Liverpool, said:

“The UK should be very proud of its long-term investment in stem cell research and the benefits are starting to come through.  Until now the potential of stem cell therapy has been tested almost entirely using adult stem cells.   The safety and efficacy of embryonic stem cells remains unknown and will only be understood through clinical trials such as this one.  Therefore the start of a trial using cells derived from embryonic stem cells for wet AMD is an important landmark and in time will help to establish the true potential of this type of therapy.”

Prof. Graham McGeown, Reader in Physiology and Deputy Head of School of Medicine, Dentistry & Biomedical Sciences, Queen’s University Belfast, said:

“Wet AMD is one of the commonest causes of sight loss in the ageing population. It results from the growth of blood vessels into the back of the retina, where light is detected. These new vessels are leaky and can cause scarring, blurring the image seen. Current treatment uses antibodies which help reduce the formation of these vessels into the eye but this isn’t always successful and requires regular injections into the eye itself. Retinal pigment epithelium cells normally help separate the retina from underlying blood vessels and retinal pigment epithelium stem cells might re-inforce this barrier, preventing further new blood vessels invading the retina itself. The hope would be that this might result in a long term improvement in symptoms, with fewer uncomfortable injections. The planned study is too small to establish whether this really will work as a mainline treatment but such a pilot study is a necessary first step and, if this is successful, a larger clinical trial would be justified.”

Prof. Chris Mason, Professor of Regenerative Medicine, UCL, said:

“This clinical trial is important for two reasons; firstly, it is potentially a big step forward towards curing a major cause of blindness. Secondly, this study will enable a much better understanding of the use of embryonic stem cells to treat disease in general. If the AMD trials are successful, then by using embryonic stem cells as the starting material, the therapy can then be affordably manufactured at large scale. This will enable all patients to benefit and not just be an expensive bespoke therapy for a select few.”

Declared interests

Prof. Anthony Hollander: “I have no conflicts of interest in relation to the stem cells for wet AMD story.”

Prof. Graham McGeown: “I am an academic who researches retinal physiology and pathophysiology but I am not involved directly with Moorfields or UCL and have no conflicts of interest.”

Prof. Chris Mason: “Employed at UCL.”