We are backing a campaign run by the National Institute for Health Research (NIHR) called “It’s OK to ask,” which encourages patients to ask their doctor about clinical research.
Clinical research is a vital tool for gathering evidence, which we can use to develop better treatments. Our group focuses on therapies for sight loss, but the process of promoting, conducting and using clinical research to improve healthcare is crucial to the whole of the NHS and to medical science in general.
In many cases doctors will approach patients about taking part in research, but the Gene and Cell Therapy Group agrees with the NIHR that patients and carers should feel empowered to ask about clinical research too. It is crucial that as patients, we all feel confident to ask about the latest research into conditions that affect us or our friends and families, and about opportunities to participate in future studies.
Asking your doctor or healthcare professional about research will enable you as a patient to engage with progress in medicine by staying up-to-date with the latest findings and by helping to contribute to tomorrow’s advances.
If you have a medical condition and are undergoing treatment, they would like you to ask your family doctor, nurse or consultant about clinical research, and whether it might be right for you.
Many of you already ask us about research into treatments for sight loss and we endeavour to provide as much information as we can about our work and how to engage with it through our website, this blog, and by directly answering your queries. In endorsing this NIHR drive for more patients to ask about research, we would like you to encourage others to do the same – there are many ways in which you can ask us about our research, including on Facebook, Twitter and by email.
You can also let the NIHR know if you do ask your doctor about research into any condition, and what happens when you do. You can find the NIHR’s campaign on Facebook, on Twitter using #NIHRoktoask, by email or by telephone (0300 311 99 66). You could even share your story about how you got in touch with the EyeTherapy group at UCL Institute of Ophthalmology!
We hope that through this campaign, which coincides with International Clinical Trials Day on May 20th, you feel encouraged to ask your doctor about clinical research – remember, “it’s OK to ask!”
Scientific research has enhanced our understanding of the causes of sight loss, enabling the development of novel treatments. For this research to be effective, it is crucial that investigators listen to with people affected by sight loss, to help set priorities in the future direction of research. Such engagement was evident at our first Retina Patient Day last year, and we’re pleased to note a further opportunity to help shape the future of eye research – the James Lind Alliance is bringing together patients, carers and researchers to identify and prioritise the top 10 ‘unanswered questions’ relating to sight loss.
The Alliance has set up the Sight Loss and Vision Priority Setting Partnership together with a wide range of organisations involved in tackling sight loss. This Partnership is seeking to understand your priorities for future research into eye health. In consultation with people affected by sight loss, their partners, relatives and carers and eye health professionals, the Partnership has built lists of ‘unanswered questions’ relating to many types of sight loss. You now have a chance to rank these questions in order of priority – to help shape the future direction of sight loss research.
This vital exercise will inform groups like the Gene and Cell Therapy Group at the UCL Institute of Ophthalmology as we investigate sight loss in the future. To participate in this consultation, please visit http://www.sightlosspsp.org.uk/. There, you will find a list of different types of sight loss in the right-hand side menu. Clicking on the type of sight loss you are interested in will download a Word document with two forms, A and B. Form A is a list of ‘uncertainties,’ or unanswered questions, from which you are asked to choose ten as priorities for future research. Form B asks you to rank the questions you’ve chosen from 1 to 10 in order of priority, and has instructions on how to complete and return both Forms including deadlines for submission.
Some important deadlines include:
Age-related macular degeneration (AMD) – April 12th 2013
Inherited retinal disease (including retinitis pigmentosa (RP), achromatopsia, Leber congenital amaurosis (LCA) and Stargardt disease) – April 15th 2013
Childhood-onset eye disorders (including amblyopia, coloboma and aniridia) – April 17th 2013
Ocular inflammatory diseases (including uveitis, birdshot retinopathy and Behçet’s disease) – March 28th 2013
This consultation is an excellent chance for your voice to be heard by the researchers investigating causes of and treatments for sight loss – and the funding bodies that support their research. Here’s Professor James Bainbridge, leading consultant ophthalmologist at Moorfields Eye Hospital and Professor of Retinal Studies, on the importance of engaging with people affected by sight loss when setting research priorities:
“Knowing which questions people with sight loss want answered helps us direct our research efforts into areas that will improve peoples’ lives. It is vital to engage people affected by sight loss in the design of future research to ensure that our efforts are closely directed to their needs.”
Transplanted photoreceptor cells can integrate into the retina and can restore vision in models of sight loss
Here’s Dr. Pearson on what she plans to present as part of one of the largest science festivals in Europe:
“I am very much looking forward to presenting our most recent findings on developing stem cell-based therapies for the treatment of retinal degeneration and discussing these with the audience. It is a great honour to have been asked by the Royal Society to show case the research we are conducting at UCL Institute of Ophthalmology”.
The talk takes place at the National Museum of Scotland at 5.30pm on March 27th, and is presented in association with the Royal Society.
We are pleased to have been awarded a major research grant from the Medical Research Council (MRC), to develop gene therapy for achromatopsia. Affecting around 1/30000 people, achromatopsia is an inherited condition that causes the complete absence of colour vision from birth, a severe reduction in central visual acuity, extreme sensitivity to light and significant impairment of daylight vision from an early age.
Roughly half of all achromatopsia cases in the UK are associated with damage to the CNGB3 gene – the 4 other genes associated with the disease are much rarer, making the CNGB3 form of the condition amenable to treatment.
We will use the funding (£2.1 million) over the next five years to produce and test an engineered viral vector suitable for use in the clinic, and conduct a Phase I/II clinical trial of gene therapy in patients with achromatopsia caused by defects in the CNGB3 gene.
In laboratory models, CNGB3 gene replacement using AAV is one of the most effective therapies being developed for sight loss caused by defects in the light-sensitive photoreceptor cells of the retina. In a study published in the journal Human Molecular Genetics we showed that gene delivery using an engineered adeno-associated virus (AAV) can restore vision in a model of achromatopsia lacking CNGB3. Our study showed long-term restoration of sight as measured by improvements in the ability of treated mice to track a visual stimulus. Dogs with a similar defect have been treated using AAV gene therapy by a group in the United States.
As well as the MRC funding for the trial, we have secured ethical approval for the study from the National Research Ethics Committee (NRES). We will soon begin production and safety testing of the clinical grade virus to be used in the clinic, after which we can seek full regulatory approval from the Medicines and Healthcare products Regulatory Agency (MHRA) to begin the trial.
Commenting on the prospect of a new trial for achromatopsia, Professor Robin Ali said: “This funding from the MRC is crucial for our programme. It will help us demonstrate that gene therapy can be used to treat photoreceptor cell defects, having shown it can be safe and effective in treating RPE defects.”
We will provide further updates on progress towards a clinical trial for achromatopsia both here and on our website.
Stem cells – cells that have the capacity to turn into virtually any cell in the human body – are an exciting source of potential treatments for a huge range of medical conditions. Stem cells are already being used in the treatment of disorders of the blood. Academics, clinical specialists and companies throughout the world are developing treatments for other conditions, including many that cause sight loss. Our own group has begun testing the safety of transplanting retinal cells grown from stem cells in patients with Stargardt disease.
Unfortunately there are a number of ‘stem cell therapies’ that are being promoted as proven treatments, without scientific evidence or appropriate approval. Here we aim to help those considering stem cell therapy for sight loss to distinguish between the approved experimental techniques in clinical trials, and the bogus treatments being offered elsewhere.
It’s important to remember that no stem cell treatment has been approved for eye disease by the medical authorities. This is because clinical trials involving stem cells in the eye are at the very earliest stages, and it is too early to know whether their use is safe or effective. Until such trials are completed, any treatments on offer are likely to be unproven.
There are a number of clinics and companies that offer what they describe as stem cell transplants for a wide range of conditions, often at significant cost. These transplants have not been subject to the rigorous safety checks that are part of properly-conducted clinical trials. The clinics offering them are usually located in countries with less stringent standards of safety and regulations than we expect in the UK, enabling them to sell such treatments in the absence of published scientific evidence to support them.
The treatment typically involves the injection of stem cells (or rather cells that come from bone marrow, or the umbilical cords of newborn babies) without first turning them into a particular cell type. The difference between these cells and cells we are using in our clinical trial is a subject for another day, but for now it’s worth remembering that the cells in our studies are first made into retinal cells in a dish before being delivered into the eye. There is no evidence that stem cells injected into the body can reach the eye, or that once they do they can repair any damage. Furthermore, there is a very real possibility that such cells could cause harm by proliferating uncontrollably.
In our cell transplantation clinical trial we take stem cells and turn them into retinal cells in a dish – it is these retinal cells, not the stem cells themselves, that we inject. The injection of stem cells remains an unproven and potentially dangerous technique.
In the absence of any scientific evidence supporting the direct injection of stem cells, we cannot recommend any of the treatments currently on offer. Should you be considering any such treatments, we strongly recommend that you check our Frequently Asked Questions and contact us first for advice.
A number of recent mediastories have highlighted two studies examining the link between long-term use of aspirin and the risk of developing the “wet” form of age-related macular degeneration (AMD). Because some of the headlines are misleading, we would urge you to consider the following details about the studies and the advice that follows.
In a study published last December in the Journal of the American Medical Association, specialists at the University of Wisconsin followed the vision of nearly 5000 patients over a period of fifteen years. They found that those who were prescribed daily aspirin to protect against heart disease were more likely to develop the “wet” form of AMD (in which the growth of new blood vessels damages the retina) than those who didn’t take aspirin. However, the risk of developing wet AMD rose from around 1-in-100 to just over 1-in-60 – which is a small difference in a rare risk.
A study from the University of Sydney, published in January 2013 in the separate journal JAMA Internal Medicine, showed that 4% of people who did not take aspirin regularly developed wet AMD, whilst 9% of those who took aspirin daily for ten years developed the condition.
While these studies suggest an association between long-term aspirin use and increased risk of AMD, they do not tell us whether any relationship is causative. Previous studies looking at the link between aspirin and AMD have been inconclusive. There is not yet enough evidence to know whether the increased risk of wet AMD is caused by aspirin itself or by other health factors for which aspirin is used.
Commenting on the media stories linking aspirin and AMD, Professor James Bainbridge said, “Daily aspirin use is well-established as an effective way to protect against heart disease in people at high risk. We therefore support the advice given by the Macular Society and others, which advises those who take aspirin to lessen their risk of heart attacks to continue to do so. Given that wet AMD can be managed using drug therapy, and that aspirin use can significantly reduce the risk of heart disease, there is no need as yet to change aspirin use based on these studies.”