Retina Day 2
Saturday 10th October 2015
About the event
Attendees were able to:
- Hear about some of the latest innovations in research around gene and cell therapies for inherited retinal conditions from world-leading researchers at UCL and Moorfields Eye Hospital and hear from RP Fighting Blindness;
- Meet with our world-leading research team and some of the charities who support their work in our exhibitor and poster session;
- Take part in a range of interactive activities and share your personal experiences and views about inherited retinal conditions to help shape our future work and priorities;
- Get answers to the research questions that matter most to you.
Retina Day was free, and open to everyone including patients, carers, the public and those who provide support for people with visual impairment. Retina Day was supported by a Wellcome People Award.
An overview and follow-up report of the day can be found here.
01. Retina Day - How is RP Fighting Blindness supporting research?
Summary: Sue Drew provides an overview of the patient support and research efforts of the charity to make improvements for those living with inherited retinal conditions.
Biog: Engagement Manager, RP Fighting Blindness
Sue works collaboratively to reach individuals and families affected by RP, to improve knowledge of inherited eye conditions, and to develop and improve high quality information and support services for those living with sight loss. Sue understands the impact of RP on families due to her own personal experience of supporting family with this condition.
The presentation slides for this talk can be downloaded by clicking here:How is RP Fighting Blindness supporting research?
02. Retina Day - Has research into retinal disease really progressed?
Summary: James Bainbridge talks about why research is important and what we need to understand if we are to address the impact of sight impairment.
- Professor of Retinal Studies, UCL Institute of Ophthalmology
- NIHR Research Professor, NIHR Moorfields BRC
- Consultant Ophthalmologist, Moorfields Eye Hospital
James was the lead clinician for the world's first gene therapy trial for Leber Congenital Amaurosis caused by mutation in the RPE65 gene and for Europe's first in-human clinical trial to assess transplantation of human embryonic stem cell-derived retinal pigment epithelium (RPE) in patients with Stargardt disease.
03. Retina Day - Are my personal experiences valuable to researchers?
Summary: Saruban Pasu and Roy Smith (PIMS Clinical Trial Steering Committee Member) discuss the PIMS Study - a clinical trial looking into the role of positioning following macular hole surgery to aid closure; and the role that patients have played in the design and outcome setting for this research.
Biog: Clinical Research Fellow, NIHR Moorfields BRC
Saruban is co-investigator for the PIMS trial at Moorfields Eye Hospital - a study to assess whether advice to position face down as opposed to face forward after standard surgery has an impact on macular hole closure rates. The trial has involved patients in helping to design the study.
The presentation slides for this talk can be downloaded by clicking here: Are my personal experiences valuable to researchers?
04. Retina Day - How do observational studies help research?
Summary: Michel Michaelides talks about some of the other types of research studies, other then clinical trials, which are essential to help us make more informed diagnoses; to better understand disease cause and progression as well as making future clinical trials more meaningful.
- Professor of Ophthalmology, UCL Institute of Ophthalmology
- Consultant Ophthalmic Surgeon, Moorfields Eye Hospital
Michel is interested in research aimed at improving the care and long-term outcomes for patients with inherited retinal diseases, with a focus on studies that link clinical research with laboratory findings, especially in the form of clinical trials for novel gene and cell therapy for sight loss.
05. Retina Day - What has been happening in gene therapy research?
Summary: Sander Smith provides an update into gene therapy research into interventions for inherited retinal diseases including an update on the results of UCL's clinical trial into RPE65 gene therapy for the treatment of Leber Congenital Amaurosis Type 2 (LCA2)
Biog: Senior Research Fellow, UCL Institute of Ophthalmology
Sander is one of the lead scientists in the world's first gene therapy trial for inherited blindness (Leber Congenital Amaurosis caused by mutations in the RPE65 gene). He is also the research fellow responsible for the pre-clinical development of gene therapy for inherited retinal dystrophies.
06. Retina Day - Will there ever be gene therapy for my condition?
Summary: Tassos Georgiadis discusses how researchers decide if gene therapy is a viable option for an intervention and what are some of the challenges or alternative approaches which are currently being investigated.
Biog: Senior Research Associate, UCL Institute of Ophthalmology
Tassos is working in gene therapy research with a focus on viral gene therapy including pre-clinical studies of possible interventions for inherited conditions causing retinitis pigmentosa and macular degeneration as well as pre-clinical optimisation of gene therapy for conditions such as LCA caused by mutations in the RPE65 gene.
The presentation slides for this talk can be downloaded by clicking here: Will there ever be gene therapy for my condition?
07. Retina Day - What is the potential for stem cell in treating and modelling disease?
Summary: Emma discusses stem cells and their potential to treat disease but also to help us model diseases of the eye, improve our understanding of how these conditions progress and how they might respond to different interventions; providing insights beyond animal models alone.
Biog: Senior Research Associate, UCL Institute of Ophthalmology
Emma is conducting research into the generation of retinal cells from stem cells and the transplantation of these cells to the diseased retina with the aim of developing this strategy into a potential cell therapy for retinal disorders in the future.
08. Retina Day - Q&A
Sponsors and support
Gene and Cell Therapy Group, UCL Institute of Ophthalmology
The Gene and Cell Therapy Group, part of the Department of Genetics, UCL Institute of Ophthalmology, is focused on reserach into new techniques to treat inherited disorders of the eye - those diseases that are caused by harmful mutations (defects) in the genetic code (the instructions which control how our bodies work) and which can be passed down through families. They have a growing clinical programme which aims to apply these techniques to patients and are responsible for the world’s first gene replacement therapy trial in Leber’s Congenital Amaurosis caused by defects in gene RPE65 and the first European clinical trial using stem cells to treat a specific form of macular degeneration called Stargardt’s disease.
The Wellcome Trust is a global charitable foundation dedicated to achieving extraordinary improvements in human and animal health. It supports the brightest minds in biomedical research and the medical humanities. The Trust’s breadth of support includes public engagement, education and the application of research to improve health. It is independent of both political and commercial interests.
NIHR Moorfields Biomedical Research Centre
"improving the health and wealth of the nation through research"
The goal of the National Institute for Health Research (NIHR) is to create a health research system in which the NHS supports outstanding individuals, working in world class facilities, conducting leading edge research focused on the needs of patients and the public.
RP Fighting Blindness
RP Fighting Blindness was founded in 1975 by a number of people with retinitis pigmentosa (RP). The group was concerned at the lack of knowledge about RP in the medical profession, the lack of a treatment or cure, and the lack of support for people with RP, and so decided to do something about it.
RP Fighting Blindness has since evolved into a respected medical research charity and a nationwide organisation providing support and information. We remain grounded in our volunteer roots as we seek to help patients live with RP and fund cutting edge research into the causes of and potential treatments for the disease.
Moorfields Eye Hospital NHS Foundation Trust
Moorfields is one of the world’s leading eye hospitals, providing expertise in clinical care, research and teaching and education.
Our main focus is the treatment and care of NHS patients with a wide range of eye problems, from common complaints to rare conditions, which require treatments not available anywhere else in the UK.
Moorfields Eye Charity
Moorfields Eye Charity is raising donations for ground-breaking research that is helping to transform the lives of people. Their charitable support makes it possible to buy new equipment that helps improve treatments and to enhance the hospital’s facilities for patient, adding value to the services paid for through the NHS.