EyeTherapy
Newsletter
The Gene and Cell Therapy Group at
the UCL Institute of Ophthalmology
Winter 2013
Prof. Ali’s welcome
Welcome to the First Edition of the EyeTherapy Newsletter,
bringing you news from the Gene and Cell Therapy Group
at the UCL Institute of Ophthalmology.
We start what will be an exciting year in 2013 with a look
back at significant developments in 2012, including major
advances in gene and cell therapy research from our group
and elsewhere, updates from our clinical trials, and our first
ever Retina Patient Day. This is the first in a series of
regular Newsletters, through which you can engage with
our research - you can also stay up-to-date through our
website, follow us via Facebook and Twitter, and contact us
via email; details are on the last page. We hope you find the
information here both interesting and useful!
Research Highlights 2012
May 2012 saw the publication of a landmark study by Dr.
Rachael Pearson, leader of our group’s cell transplantation team,
in the prestigious journal Nature. The study showed that
transplanting immature light-sensitive photoreceptor cells can
restore vision in a model of sight loss. We were able to show for
the first time that cell transplantation can improve vision as
measured by the ability to respond to a visual stimulus by
navigating a water maze... Continued on page 2
We also began Europe’s first clinical trial investigating the use of
stem cells, which are grown into retinal pigment epithelium (RPE)
cells before being injected into the eyes of participants with
Stargardt Macular Dystrophy… Continued on page 2
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Research Highlights 2012
Stem cell transplants restore vision in mice
(Continued from page 1…) Dr. Pearson’s study provides the first convincing evidence
that transplanted immature photoreceptors (the light-sensitive cells in the retina) can
mature into fully-functioning cells that detect light, produce a nerve signal in response to
it, and transmit this signal to the brain as efficiently as normal photoreceptors. These
encouraging results confirm that photoreceptor cell transplantation is a feasible therapy
for sight loss, repairing the degenerating retina and restoring vision in this model of
disease.
We tested vision in mice that received cell transplants using a head-tracking test (left) and a water-maze
(right). More details, including a video of the water-maze test, can be found on the Gene and Cell
Therapy website.
Clinical trial testing the safety of stem cell transplants
(Continued from page 1…) Prof. James Bainbridge began Europe’s first clinical trial
testing the safety of transplanting retinal cells derived from stem cells into the eye. The
surgery involves injecting a suspension of retinal pigment epithelial cells, which have
been grown from stem cells in a dish. The study, which will last approximately 2 years, is
designed to determine whether the technique is safe. Future studies will be required to
find out if the technique can help protect or improve sight.
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Retina Patient Day 2012
We held the first “Retina Patient Day” in London on April 21st 2012. Over 250 people with
retinal degeneration and their family members met with more than 40 clinicians and
scientists from UCL Institute of Ophthalmology Department of Genetics and Moorfields
Eye Hospital Biomedical Research Centre for Ophthalmology. We provided updates on
our efforts to develop effective treatments for sight loss through information stalls, a
chance to ‘meet the doctor’, ‘meet the scientist’ and ‘meet the counsellor,’ an artist’s
workshop, and most importantly, over 30 poster presentations explaining our research in
terms that attendees could relate to.
Through these posters our researchers explained how their area of research fits into the
broader effort of seeking therapies for visual disorders. They answered many excellent
questions put to them by the attendees, who were keen to understand more about their
conditions and about the research into treatments. This opportunity to engage with
scientists at the forefront of delivering innovative therapies is not often afforded to people
with vision loss, and the interaction proved very welcome.
Commenting on the day, Prof. Robin Ali, Head of Department of Genetics and the BRC
Gene Therapy Theme leader, said “The day has been a great success. We aim to hold a
“Retina Patient Day” regularly. Patient engagement is an essential part of developing an
effective translational research programme. Our team not only look forward to further
opportunities to explain our work to patients and their families, but to involve and learn
from those who may benefit from it in the future.”
For further details, photographs and feedback from Retina Patient Day 2012, please visit
https://www.ucl.ac.uk/ioo/genetics/gene-and-cell-therapy/patient-engagement/patientengagement-day - we will also provide information on the planned Patient Day in 2013 in
future newsletters and on our website.
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Research commentary
Restoring vision in mice
Scientists at Columbia University, New York have successfully restored retinal function
for at least five months in a model of sight loss caused by damage to the PDE6-alpha
gene. By delivering a working copy of that gene using an engineered virus, they showed
that light-sensitive photoreceptor cells can survive longer and improve retinal function.
This is further evidence that AAV-mediated gene therapy is valid for various forms of sight
loss, and lead researcher Dr. Stephen Tsang says that clinical trials may follow soon.
Dr. Tsang’s group also published a study in which they turned human skin cells into cells
that resemble stem cells (so-called iPS cells), then grew retinal pigment epithelium (RPE)
cells from these iPS cells. Transplanting these RPE cells into a model of sight loss,where
the RPE doesn’t work properly but is largely intact, partially restored some elements of
vision. These results are promising, and if such cells can be shown to improve vision
where RPE cells are lost, such as in Stargardt disease, they may be a welcome source of
RPE cells for use in clinical trials.
Stem cell transplants
In January 2013 the BBC reported that Professor Pete Coffey and Mr Lyndon da Cruz are
preparing a clinical trial testing the safety and efficacy of injecting patches of RPE cells,
grown from stem cells, into the eyes of people with Age-Related Macular Degeneration
(AMD). The trial will take place at Moorfields Eye Hospital and will add to our
understanding of how to treat advanced sight loss.
Further laboratory evidence of how useful cell transplantation can be in restoring sight
was provided in a study by Professor Robert MacLaren and colleagues at Oxford
University. They used a similar approach to our previous studies on photoreceptor
transplantation, showing that sight can be restored even in an animal model of very
advanced sight loss.
We will report on further advances on our website and in future Newsletters.
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Get involved!
Stay up-to-date with our research and contact us
Website: http://www.ucl.ac.uk/ioo/genetics/gene-and-cell-therapy/
Blog: http://blogs.ucl.ac.uk/eyetherapy/
Email: eye.info@ucl.ac.uk
Twitter: https://twitter.com/Eye_Therapy
Facebook: https://www.facebook.com/UCLeyetherapy
Contribute to our work
Developing therapies for inherited and acquired sight loss requires significant investment,
and we are seeking substantial support from charities and research councils. But we also
need your support. Your generous donation – however big or small – will help us reach
our goal of bringing a pipeline of gene therapies for sight loss to clinical trial. If you would
like to support our work, please visit our JustGiving page where you can select which area of our research you would like to contribute to.
All support, however big or small, is welcome - your contribution will help us invest in vital
equipment and people to advance our work. Just £5 or £10 would help pay for tests that
show how well our gene and cell therapies are working in the lab.
You can even donate by text message! Just text EYES48, followed by £5 or any amount,
to 70070.
We appreciate all contributions of any size. Should you wish to discuss your donation, or
set up regular monthly payments to support our work, please email eye.info@ucl.ac.uk or
call 0207 608 7982
Future EyeTherapy Newsletters and the EyeTherapy blog
We hope you enjoyed the first edition of our Newsletter. If there are features you would
like to see in future editions, or you want to share your thoughts on treatments for sight
loss on our blog, please contact us using the details on this page. We would really
appreciate your input, as our aim with the Newsletter and the blog is to provide content
you would like to see - please get in touch with Dr. Prateek Buch who manages our
patient and public engagement programme and share your ideas using the contact details
above!