UCL INSTITUTE OF OPHTHALMOLOGY
DIVISION OF MOLECULAR THERAPY
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NEWS


2012

First Retina Patient Day a great success
18th May 2012
Patients with retinal degeneration and their families gathered in London on April 21st to attend the first UCL/Moorfields Biomedical Research Centre for Ophthalmology “Retina Patient Day”. Over 250 attendees had the opportunity to interact with more than 40 clinicians and scientists from Moorfields Eye Hospital and UCL Institute of Ophthalmology Department of Genetics, who provided updates on their research into developing effective treatments for blinding retinal conditions.

The day began with a series of short talks delivered by the senior investigators leading the research effort. Professors Robin Ali, James Bainbridgeand Tony Moore were joined by Drs.Lyndon da Cruz,Michel Michaelides, Rachael Pearson and Andrew Webster in setting out how laboratory and clinical studies are rapidly enhancing our understanding of how best to treat inherited diseases that cause blindness.

The researchers delivered informative explanations of how advances in basic science are gradually being translated into clinical trials – the audience was told of the progress that was being made but that further rigorous work was stillrequired in order to develop more effective treatments.

The centrepiece of the “Retina Patient Day” was a unique and much-appreciated opportunity for the attendees to interact with dozens of clinicians and scientists, which we arranged in four broad ways: 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. The scientists presenting their posters explained their area of research and how it 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.

‘Very enlightening and educational, really delighted this patient day has been put on. Worth travelling from S.Wales!!!’

‘Extremely informative, one to one discussions with the professionals. Definitely will come again’

‘Well organised event with very helpful speakers. Like the poster rooms for specific questions’

‘It’s a great event and a long time coming’.


Attendees were also given an opportunity throughout the day to submit questions to the lead investigators – these questions covered a range of topics and were answered by our panel of experts.

Following these fruitful and energetic interactive sessions, the day was brought to a close with an insight into the work of RP Fighting Blindness and Moorfields Eye Charity, two of the organisations whose generous support make the research being discussed (and the awareness day itself) possible.

The Retina Patient Day 2012 was an excellent opportunity for patients with retinal degeneration and their families to engage with researchers involvedin developing new treatments.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 PatientDay” every year. 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.”




Photoreceptor transplant restores vision in mice
18th April 2012
Scientists funded by the Medical Research Council (MRC) have shown for the first time that transplanting light-sensitive photoreceptors into the eyes of visually impaired mice can restore their vision.

The research, published in Nature, suggests that transplanting photoreceptors –light-sensitive nerve cells that line the back of the eye – could form the basis of a new treatment to restore sight in people with degenerative eye diseases.

Scientists from UCL Institute of Ophthalmology injected cells from young healthy mice directly into the retinas of adult mice that lacked functional rod-photoreceptors. Loss of photoreceptors is the cause of blindness in many human eye diseases including age-related macular degeneration, retinitis pigmentosa and diabetes-related blindness.

There are two types of photoreceptor in the eye – rods and cones. The cells transplanted were immature (or progenitor) rod-photoreceptor cells. Rod cells are especially important for seeing in the dark as they are extremely sensitive to even low levels of light.

After four to six weeks, the transplanted cells appeared to be functioning almost as well as normal rod-photoreceptor cells and had formed the connections needed to transmit visual information to the brain.

The researchers also tested the vision of the treated mice in a dimly lit maze. Those mice with newly transplanted rod cells were able to use a visual cue to quickly find a hidden platform in the maze whereas untreated mice were able to find the hidden platform only by chance after extensive exploration of the maze.

Professor Robin Ali at UCL Institute of Ophthalmology, who led the research, said:

“We’ve shown for the first time that transplanted photoreceptor cells can integrate successfully with the existing retinal circuitry and truly improve vision. We’re hopeful that we will soon be able to replicate this success with photoreceptors derived from embryonic stem cells and eventually to develop human trials.

“Although there are many more steps before this approach will be available to patients, it could lead to treatments for thousands of people who have lost their sight through degenerative eye disorders. The findings also pave the way for techniques to repair the central nervous system as they demonstrate the brain’s amazing ability to connect with newly transplanted neurons.”

Dr Rachael Pearson from UCL Institute of Ophthalmology and principal author, said:

“We are now finding ways to improve the efficiency of cone photoreceptor transplantation and to increase the effectiveness of transplantation in very degenerate retina. We will probably need to do both in order to develop effective treatments for patients.”

Dr Rob Buckle, head of regenerative medicine at the MRC said:

“This is a landmark study that will inform future research across a wide range of fields including vision research, neuroscience and regenerative medicine. It provides clear evidence of functional recovery in the damaged eye through cell transplantation, providing great encouragement for the development of stem cell therapies to address the many debilitating eye conditions that affect millions worldwide.”

The researchers demonstrated previously, in another study published in Nature, that it is possible to transplant photoreceptor cells into an adult mouse retina, provided the cells from the donor mouse are at a specific stage of development - when the retina is almost, but not fully, formed. In this study they optimised the rod transplantation procedure to increase the number of cells integrated into the recipient mice and so were able to restore vision.

The research was funded by the MRC, the Wellcome Trust, the Royal Society, the British Retinitis Pigmentosa Society, Alcon Research Institute and The Miller’s Trust. Robin Ali is a senior investigator of the National Institute for Health Research and carries out research at the NIHR Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of Ophthalmology. Rachael Pearson is a Royal Society University Research Fellow.

Notes to editors

Video footage of the mouse maze is available here: http://youtu.be/GJCVr5pjeEc and images can be downloaded here: http://flickr.com/gp/uclnews/72Uqne/. For unedited video footage, please contact the UCL press office on the number below.

For more information, contact:

Hannah Isom,
Senior press officer, Medical Research Council
T: 0207 395 2345 (out of hours: 07818 428 297)
E: press.office@headoffice.mrc.ac.uk

Clare Ryan,
Media relations manger, UCL
Office: +44 (0) 20 3108 3846
Mob: +44 (0) 7747 565 056
Email: clare.ryan@ucl.ac.uk

1. The paper, ‘Restoration of vision following transplantation of photoreceptors’, by Pearson et al, is published in Nature: http://dx.doi.org/ 10.1038/nature10997.

2. For almost 100 years the Medical Research Council has improved the health of people in the UK and around the world by supporting the highest quality science. The MRC invests in world-class scientists. It has produced 29 Nobel Prize winners and sustains a flourishing environment for internationally recognised research. The MRC focuses on making an impact and provides the financial muscle and scientific expertise behind medical breakthroughs, including one of the first antibiotics penicillin, the structure of DNA and the lethal link between smoking and cancer. Today MRC funded scientists tackle research into the major health challenges of the 21st century. www.mrc.ac.uk

3. Founded in 1826, UCL was the first English university established after Oxford and Cambridge, the first to admit students regardless of race, class, religion or gender, and the first to provide systematic teaching of law, architecture and medicine. We are among the world's top universities, as reflected by performance in a range of international rankings and tables. UCL currently has 24,000 students from almost 140 countries, and more than 9,500 employees. Our annual income is over £800 million. www.ucl.ac.uk | Follow us on Twitter @uclnews

4. The National Institute for Health Research provides the framework through which the research staff and research infrastructure of the NHS in England is positioned, maintained and managed as a national research facility. The NIHR provides the NHS with the support and infrastructure it needs to conduct first-class research funded by the Government and its partners alongside high-quality patient care, education and training. Its aim is to support outstanding individuals (both leaders and collaborators), working in world class facilities (both NHS and university), conducting leading edge research focused on the needs of patients. Website: www.nihr.ac.uk

ITV: How mice are giving new hope to patients fighting blindness

BBC: Scientists restore sight in blind mice

BBC Radio4 Today programme: Success in blind mice trials

Independent: Major step towards cure for blindness

Daily Mail: Blindness cure step closer after scientists replace lost light-sensitive cells in back of the eye

Guardian:Transplanted cells allow mice with night blindness to see in dark


ACT trial of stem-cell derived RPE cells in Stargardt disease:
First patient transplant taken place
23rd January 2012
The first subject in this phase 1/2 UK trial received a transplant of retinal cells on Friday 20th January. He is 34 years old and has severe sight impairment as a result of Stargardt disease, which is the commonest form of macular degeneration that affects young people. Chief Investigator of the trial, Prof. James Bainbridge said “There is real potential that people with blinding disorders of the retina, including Stargardt disease and age-related macular degeneration, might benefit in the future from transplantation of retinal cells. Testing the safety of retinal cell transplantation in this clinical trial is an important step towards achieving this aim. We are very pleased that the first transplant surgery has gone smoothly and look forward to seeing the results as the trial progresses over the next 2 years. While this is primarily a safety trial, we will have the opportunity to monitor engraftment of retinal cells and to assess any impact on sight.”

FURTHER INFORMATION:

The purpose of the current trial is test the safety of retinal cell transplantation in people with Stargardt disease, a form of macular degeneration that causes loss of sight in young people and is currently untreatable.

The retinal cells to be used in this trial have been grown from stem cells and are transplanted during an operation that lasts up to one hour. The cells have been developed by Advanced Cell Technology (ACT), a company that is conducting similar trials in the United States.

This trial is designed to test the safety of the procedure in people who are already severely sight-impaired. People may be eligible to participate in the trial if they have Stargardt disease, severe sight-impairment and are otherwise well. The trial will involve 12 patients and will involve Aberdeen Royal Infirmary as well as Moorfields.

Future trials will find out if cell transplantation can help protect sight.

Media enquiries should be directed to Julia Jones Julia.Jones@moorfields.nhs.uk


For further information about the trial please visit: http://clinicaltrials.gov/ct2/show/NCT01469832?term=stargardts+london&rank=1

For further information on ACT please visit www.advancedcell.com



2011

NIHR Biomedical Research Centre at Moorfields Eye Hospital and UCL Institute of Ophthalmology
ACT Trial of stem-cell derived RPE cells in Stargardt disease
26th September 2011
Researchers at Moorfields Eye Hospital and UCL Institute of Ophthalmology have received approval from the Medicines and Healthcare Prodicts Regulatory Agency (MHRA) to begin a clinical trial investigating the safety of using transplanted retinal cells derived from stem cells to treat patients with advanced Stargardt disease.

Stargardt disease, a form of macular degeneration, causes loss of sight in around one in 10,000 young people and is currently untreatable. This trial is designed to test the safety of the procedure in people who are already severely sight impaired. People may be eligable to participate in the trial if they have Stargardt disease, severe signt impairment and are otherwise well.

The reintal cells to be used in this trial have been grown from stem cells and will be transplanted during an operation expected to last up to one hour. The cells have been developed by Advanced Cell Technology (ACT), a company that is conducting similar trials in the United States. Gary Rabin, chairman and CEO of ACT described the Moorfields trial as "another milestone for the field of regenerative medicine".

Professor James Bainbridge, who will be leading the trial at the National Institute for Health Research (NIHR) Biomedical Research Centre based at Moorfields and the UCL Institute of Ophthalmology says "There is real potential that people with blinding disorders of the retina, including Stargardt disease and age-related macular degeneration, might benefit from transplantation of retinal cells."

Earlier this year Moorfields Eye Hospital and UCL Institute of Ophthalmology were awarded £26m for work aimed at the translation of scientific advances into novel treatments for people with eye disease by the National Institute of Health Research (NIHR).

LINKS
http://www.moorfields.nhs.uk/Aboutus/Mediaoffice/Mediareleases/J6nP
Retinal stem cell trial - useful information for patients

http://www.rpfightingblindness.org.uk/
For information about Stargardt disease and other retinal disorders

http://www.brcophthalmology.org/
Biomedical Research Centre for Ophthalmology


2009

Clinical trial of gene therapy Leber’s congenital amaurosis: new phase includes higher dose and inclusion of children.

1 st July 2009
Researchers at UCL Institute of Ophthalmology and Moorfields Eye Hospital have begun the next step in its Phase 1 / 2 dose escalation clinical trial to treat a form of Leber’s Congenital Amaurosis caused by defects in the gene encoding the RPE65 protein. Leber’s Congenital Amaurosis, or LCA, is an inherited disease that typically results in blindness...read more.


2008

Results of world's first gene therapy for inherited blindness show sight improvement
27th April 2008

UK researchers from the UCL Institute of Ophthalmology and Moorfields Eye Hospital NIHR Biomedical Research Centre have announced results from the world’s first clinical trial to test a revolutionary gene therapy treatment for a type of inherited blindness. The results, published today in the New England Journal of Medicine, show that the experimental treatment is safe and can improve sight. The findings are a landmark for gene therapy technology and could have a significant impact on future treatments for eye disease...read more


BBC Clinical Trial News Feature 2008

 

 

GENE THERAPY TRIAL MAZE VIDEO

Read more about the clinical trial and view video footage on the following sites

 

For archived news click here

This page last modified 18 December, 2012 by xxx


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