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The first patient has been treated with a new
stem-cell-derived treatment for ‘wet’ age-related
macular degeneration (AMD) as part of a pioneering
clinical trial in London.
Using technology developed with MRC funding, the
trial will test if it is safe and effective to
transplant a type of eye cell called retinal pigment
epithelial (RPE) cells, grown from stem cells in the
lab, to restore sight in people with severe visual
loss from wet AMD. The cells are used to replace
diseased ones at the back of the eye using a
specially engineered patch. The patch is inserted
behind the retina in a surgical operation which
lasts one to two hours.
A woman was successfully treated last month and to
date there have been no complications. The research
team hope to assess the extent to which she has
recovered her vision by early December.
Professor Pete Coffey of the University College
London Institute of Ophthalmology (http://www.ucl.ac.uk/ioo),
who is co-leading the research, said: “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.”
The trial will recruit another nine patients over 18
months, each of whom will be followed for a year to
assess the safety and stability of the cells and
whether there is an effect in restoring vision.
Retinal surgeon and project co-lead Professor Lyndon
Da Cruz from Moorfields Eye Hospital (http://www.moorfields.nhs.uk/),
who is performing the operations, added: “There is
real potential that people with wet age-related
macular degeneration will benefit in the future from
transplantation of these cells.”
The MRC funded Professor Coffey in 2004 to carry out
the basic research and preclinical development
behind the cell transplants for retinal repair. With
further MRC funding, Professor Coffey and his team
have developed imaging techniques to allow them to
look at the survival and function of the cells after
transplant. The MRC also funded Professor Harry
Moore at the University of Sheffield to establish a
bank of human embryonic stem cell lines, one of
which has been used to develop this technique.
The MRC has recently made a £1.5m award as part of
the UK Regenerative Medicine Platform (http://www.ukrmp.org.uk/)
to further support the Coffey team in developing
this technology for use in the clinic.
The clinical trial is part of the London Project to
Cure Blindness, a partnership between Moorfields Eye
Hospital, the UCL Institute of Ophthalmology, the
National Institute for Health Research (NIHR) and
Pfizer Inc.
Dr Rob Buckle, the MRC’s Director of Science
Programmes, said: “This London Project to Cure
Blindness/Pfizer trial is an exciting development in
regenerative medicine and a great example of how
funding discovery science in stem cell biology has
led to a clinical trial with commercial partnership.
This development would neither have been possible
without the MRC’s long-standing support for the
groundwork behind this technology, nor the hard work
and investment from the London Project to Cure
Blindness and Pfizer and others to bring the therapy
to patients.”
For more information
University College London Institute of Ophthalmology
Link...
Moorfields Eye Hospital
Link...
UK Regenerative Medicine Platform
Link...
Medical Research Council - MRC
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