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Retina New Zealand Inc. Retina New Zealand Inc. The Newsletter of Retina NZ Inc A Member of Retina International NOVEMBER 2012 No. 55 From the Editor/ From the President’s Desk .......................................... 2 Research News ...................................................................................... 3 World Congress report: ......................................................................... 5 Dr Harry Bradshaw: vitreo-retinal surgeon on the cutting edge ........... 10 Cane and Able: When the going gets bumpy ....................................... 13 Branch News ........................................................................................ 14 From our mailbag ................................................................................. 15 Media Review: Bardowl - new bird in the audiobook nest.................... 15 Charities Commission Reg No 23240 Mission Statement To promote public awareness of retinal degenerative disorders; To provide information and support; and to foster research leading to treatment and an eventual cure. 1 From the Editor Our Dunedin AGM was an inspiring occasion. I particularly enjoyed Dr Harry Bradshaw’s talk on genetic treatments for retinal disorders, summarised in this issue. He was also helpful explaining the difference between the two drugs currently used to treat wet MD. Dr Diane Sharp advises that there is a third drug in the pipeline that will require less frequent injections. It is not yet available here, so we will try to keep you posted about that one. Zane Bartlett gave a stimulating talk about the youth congress he attended. Dr Lynley Hood held her audience with her brilliant account of the challenge of becoming partially sighted/visually impaired/low vision. That was followed by animated discussion amongst the audience. We hope to include some of this in our next issue. Also in this issue is a very thorough account of the Retina International World Congress that Fraser Alexander attended in Germany this year. This has been a promising year in terms of new treatments for retinal disorders and most of us will be struggling to keep up with them. The same could be said of adaptive technology which we have not covered this time, but never fear, we are monitoring it constantly. We really welcome feedback from readers about what they like and dislike in our newsletter. We also like to know what you would like to read in forthcoming issues. Have a great summer! Camille Guy From the President’s Desk Our first AGM and public meeting in Dunedin in September turned out to be a most heartening and inspiring experience for me. Retina has always enjoyed strong support from that city’s clinical and scientific community and it is most pleasing to also see our voluntary patient effort go from strength to strength in recent years. We welcomed Karen Manning to our executive and were inspired at our Public Meeting by Dr Lynley Hood who gave one of the most professional and well-researched patient advocacy presentations I have seen in my 14 years of involvement with our society. It is my hope that a number of positive developments this year will greatly enhance our awareness among eye care professionals. The growth of our Retina Youth patient cohort, the upgrade of our publications, the continuing confidence being expressed by our financial supporters and the emergence of human clinical trials for retinal degenerations, should be important positive factors in achieving higher levels of engagement between Retina New Zealand and the clinical community - vital conduits as we seek to achieve our missions around public awareness, peer support and access to treatments. I wish all members, friends and family, eye care professionals, rehabilitation professionals and associated stakeholders a relaxing and safe festive season. Fraser Alexander President 2 Research News AMD sufferer can read again after telescope implant in eye An 89-year-old woman in Northern California, who was blind for seven years, has spoken of her joy at being able to see better than ever, after receiving a pioneering pea-sized telescope implant. Surgeons at UC Davis Medical Centre successfully implanted a tiny telescope implant in the eye of Dorothy Bane in May, the Daily Mail reported. She has suffered from endstage age-related macular degeneration (AMD), one of the leading causes of blindness. Bane is among the first in the world to receive the implant, and it has let her read again. “I can see better than ever now. Colors are more vibrant, beautiful and natural, and I can read large print with my glasses,” she said. Bane, who was a watercolour artist, said “I haven’t been able to read for the past seven years, and I look forward to being able to paint again.” The implant is able to focus images on the undamaged areas of the retina. The groundbreaking operation could lead to the procedure becoming commonplace. Mark Mannis, professor and chair of ophthalmology and vision sciences and director of the Eye Center at UC Davis Health System, said the telescopic implant restores vision by projecting images onto an undamaged portion of the retina, which makes it possible for patients to again see people’s faces and the details of objects located directly in front of them,” he said. Bane is the first in her state and among the first 50 individuals in the nation to receive the implant. Since her surgery, Bane has been working with Society for the Blind optometrists and UC Davis occupational therapists to learn how to use her new telescopic eye. FDA approves retinal prosthesis. There is talk of a “first ever bionic eye for the blind” in the United States after the FDA approved Second Sight’s Argus® II Retinal Prosthesis System. It was concluded that its probable benefit outweighs the risk to health. This is a first step toward market approval. Argus® II is an innovative retinal implant that, for the first time ever, partially restores vision to patients who are blind due to Retinitis Pigmentosa. The system works by converting video images captured by a miniature camera, housed in the patient’s glasses, into a series of small electrical pulses that are transmitted wirelessly to an array of electrodes on the surface of the retina. These pulses are intended to stimulate the retina’s remaining cells resulting in the corresponding perception of patterns of light in the brain. Patients then learn to interpret these visual patterns thereby regaining some visual function independence. It has taken three trials, $100m, 20 years in public investment and the same in private investment to reach this stage. 3 Singapore: Scientists have isolated three new genes linked to Primary Angle Closure Glaucoma (PACG), a leading cause of blindness, which affects 15 million people worldwide, 80 percent of whom live in Asia. Scientists did a genome-wide association study of 1,854 PACG cases and 9,608 controls (who did not suffer from PACG) of over five sample collections in Asia. They performed validation experiments in another 1,917 PACG cases and 8,943 controls collected from a sample from around the world. Lucentis v Avastin row ends in a draw Some New Zealand AMD patients have been wondering in recent years about the choices made here between two drugs used to treat wet MD. The drugs are injected into the retina to try to prevent progression of retinal damage. Within our public system the drug of choice has been the much cheaper Avastin, a drug used originally to treat bowel cancer. Its use for retinal treatment came about when one Florida doctor noticed that his cancer patients receiving Avastin found their macula degeneration also improved. Some ophthalmologists began using Avastin to treat wet MD. It seemed that it stopped the growth of the fragile new blood vessels that develop and later bleed. The molecule in Avastin was a large one and so a new drug with a molecule only one quarter the size was developed. The expectation was that when injected into the eye it could more easily penetrate into the retina to greater control the new vessels. This is Lucentis. Although Avastin was not a cheap drug for treating bowel cancer, such small amounts were needed for treatment of the retina that its price for that application was relatively small. As Lucentis had been hugely expensive to develop, both with the fractioning of the antibody and the clinical trials, it retailed for a great deal more. Some patients, aware that private NZ ophthalmologists preferred to treat them with Lucentis injections, believing that it was more effective, wondered whether public patients receiving shots of Avastin were being short changed. It has been seen as a matter of medicines being priced too high by multinational corporations for them to be affordable for all who need them. In the UK, those who could benefit could be given Lucentis, but the bill to the NHS is enormous and there is a danger that some sort of rationing would take place, such as treating only those who are most likely to improve. And a soaring drugs bill means less money for the NHS to spend on other sorts of care. Dunedin-based vitreo-retinal surgeon Dr Harry Bradshaw puts it into the New Zealand context. “Lucentis costs NZ$1500 per vial. If formulated at Dunedin Hospital pharmacy Avastin costs NZ$32. This means you could only treat a fraction of the people with Lucentis “My feeling is that there is no difference between Lucentis and Avastin. I would happily have Avastin if I needed it. There are a very few patients who don’t respond to Avastin. There is scope within Pharmac for these few people to receive Lucentis instead as some will respond to Lucentis.” (Learn more about Dr Bradshaw on page 14) - 4 Camille Guy $2 Million Prize Offered in Quest to End Blindness by 2020 Art Garfunkel and two of his college roommates, Jerry Speyer and Sanford Greenberg, described on their venture’s www.endblindnessby2020.com website as part of “a group of dreamers,” have announced they will give $2 million in gold bullion to whoever does the most to end blindness by 2020. Their Sanford and Susan Greenberg Prize to End Blindness by 2020 even has a specific final date - Dec. 13, 2020. The winner will be selected by a Governing Council whose members are listed on the site, and the Wilmer Eye Institute at Johns Hopkins University School of Medicine in Baltimore will administrate the prize. 17th Retina International World Congress outlines many paths to cures By Fraser Alexander The 17th Retina International World Congress was characterised by high quality speakers, accessible and concise presentations, a uniquely innovative conference venue and typically German operational precision. It was a real triumph for our hosts Pro-Retina Deutschland who were supported by the German Ophthalmological Society and the Professional Association of Ophthalmologists in Germany. ‘Gateway To Vision’, Hamburg, July 2012 attracted more than 700 people during the open-session days with a significant number of people travelling from distant cities and from a total of around 40 countries. Researchers and Clinicians speaking at the congress generally structured presentations in order to give patients an account of the recent progress made in their particular area of investigation, the results in the laboratory and the clinic and what the future holds in terms of developing clinical applications. The congress was scheduled using themed parallel sessions and included 52 speakers representing countries including Germany, United Kingdom, USA, Switzerland, Canada, Italy, Ireland, France and the Netherlands. All sessions ran concurrently and were translated into English, German, French and Spanish using personal headsets. Preceding all the exciting scientific and medical updates were the two days dedicated to patient group objectives. Member Organisations from 27 countries attended the Retina International General Assembly. Applications for Candidate Membership were unanimously approved from organisations in Argentina and Chile. Organisations from Belgium, Iceland and Taiwan were accepted as full members. Christina Fasser was reelected as president and MC members elected were: Fraser Alexander, Anna Maria Leopoldi, Claudette Medefindt, Caisa Ramshage, Lorna Rosenstein and KP Tsang. The GA approved the kind offer of RP Fighting Blindness, Ireland to start the process of establishing a secretariat for RI in Dublin. The main focus of the The Continuing Education Program Day was on clinical trials. I gave a presentation of what a clinical trial is, what patients should expect, what questions should be asked and a reminder that not all clinical trials lead to therapy. Avril Daly, Ireland, examined the history of clinical trials and the ethical issues that seem to have lagged behind the science. 5 As the pace of advancement in our understanding of retinal disease and the development of treatments appears to increase with each Biennial Retina International Conference - and you will have now probably read quite a lot about each particular area of research – I will highlight some “emerging trends”, “summary quotes” and patient-focused implications from Hamburg concluding with an overview of research areas, as has been the case in other World Congress Reports. Early on during the first day, the need for interdisciplinarity in order to progress in developing therapies for retinal degeneration was highlighted. Professor Eberhart Zrenner Tübingen, Germany, said there needed to be a great deal of interdisciplinary cooperation between doctors, patients, biologists, neurosurgery special clinics, laboratories, companies, and also health departments, research institutions, psychologists, engineers and pharmacologists. “As patients and patient groups, we need to be conscious of the fact that everyone has to work together to find a solution for every individual patient – “above all, there needs to be a mutual understanding of the outcome being sought”. Personal accounts by patients who have participated in a human clinical trial is a recent trend at Retina International conferences. Probably the most thought-provoking and positive perspective was the continuous education day presentation by Miikka Terho, Finland. Dr Stephen Rose, Chief Scientific Officer United States Foundation Fighting Blindness, in explaining how the therapeutic pipeline works, said “Academia understands the pathways of the visual cycle and identifies opportunities for treatments then ‘discovery research’ precedes pre-clinical research and finally into phase 1 human trials. Between discovery research and phase 1 trials is often called ‘the valley of death’ because huge funding is needed. We estimate in the US it takes $8-10m to take an identified treatment target to human clinical trial stage.” Professor Stephen Daiger, University of Texas, Houston, USA was asked about the complexities of the genetics of retinal disorders “We are hearing of so many more complications to genotyping and genotype-phenotype matching – is it getting worse?” Stephen replied “ No, 30 years ago we didn’t have the faintest idea about the complexities, the fact that I can get up here and talk about gene after gene after gene and DNA technology means we have caught up with the complexity. The problem is complex but the tools we possess to deal with this complexity are also now complex – I think there has been a huge advance, even in the last 10 years”. Prof Daiger underlined the rationale for gene testing “Firstly, I don’t know one family that isn’t pleased to know what is causing their sight loss – it makes it real for them - the pathogenesis and inheritance pattern can be defined. The diagnosis might change previously it might have been thought to be recessive and it turns out to be dominant. The second reason is that gene-specific therapies are around today. There are mutation-specific therapies and I get calls from investigators asking if I have patients on my database with a specific mutation. Thirdly, a new gene mutation being discovered in your DNA means that you have the opportunity to become an advocate for the development of therapies specific to you – 6 we see this happening in the US. While this puts pressure on scientists, it is fair to say that the charitable foundations (for RDs) have driven this field forward faster than many other inherited disease areas and ultimately both the patients and the scientists benefit.” Prof Daiger went on to illustrate the complexity of genetics associated with RDs, highlighting the case of autosomal dominant RP. “There are now more than 50 genes that are associated with this condition. In the case of genes coding for the protein Rhodopsin, there are now 250 mutations identified. A recent study estimated the total number of mutations identified in retinal degenerative disease to be around 8,000. It turns out that in some cases a mutation in a gene can lead to recessive disease and some cases dominant disease. To round off the complications, we have seen in a family of siblings with the same mutation, a range of diagnoses – two diagnosed with RP, two with MD. “In our laboratory at this time we are able to identify the mutation in around 70% of patients we test who have a diagnosis of autosomal dominant RP.” In similar fashion, Professor Frans Cremers, Netherlands, reinforced the difficulties and importance of genotyping. 160 Genes for Retinal Degeneration had been identified with around 10 new genes being found each year. He stressed the importance of researchers having access to patient registries. Professor Robin Ali spoke of his work in photoreceptor regeneration involving transplanting progenitor retinal cells from mice into a mouse model with rod photoreceptor loss. Progress has been made in that the transplanted cells are now making connections but challenges remain in getting more cells to integrate, achieving success in cone integration, growing photoreceptor cells from stem cells, finding more animal models, and more funding. Professor Gerald Chader from Los Angeles, USA, in the final plenary session, presented his perspectives in inherited RD research by summarising progress in research. He commented that in the past, no gene mutations for RP were known, there was very little idea about the mechanisms of photoreceptor cell death and there were no agents known that could slow photoreceptor degeneration and death. However, about half of the RP mutations are now known and much is understood about the basic mechanism of photoreceptor cell death and how to inhibit it. Electronic implants are available to patients and basic work in the fields of stem cell biology and optogenetics show promise for future treatment. So, what therapies will be effective when in the first case, some photoreceptor cells remain alive or, in the second case, when most or all photoreceptor cells are dead? When at least some photoreceptors remain alive, there are three approaches that can be used: 1) Gene Therapy 2) Pharmaceutical Therapy and 3) Nutritional Therapy. When most or all photoreceptor cells are dead, these three therapies cannot be effective so we must consider approaches that actually replace the photoreceptors or at least their function. These are 4) Stem Cell Transplantation 5) Optogenetics or 6) Electronic Prosthetic Devices. The following is a short synopsis of each of the promising areas for future therapy. 7 1) Gene Replacement Therapy This therapy replaces a defective (mutant) gene in a cell, such as a photoreceptor neuron, with a normal copy of the gene. The replacement gene is transported into the target cell by a vehicle that is called a “vector”. Vectors are modified viruses that have been altered such that they cannot replicate but they yet can effectively penetrate a target cell to deliver the gene cargo. In this way, the new normal gene acts as a blueprint to synthesize a normal protein (gene product) and restore the lost function. Gene Therapy has shown great success (efficacy and safety) in experiments on animal models of retinal degeneration (RD). Long term positive effects have been seen as well as positive results in treating older animals with relatively advanced RP. Clinical Trials are already taking place for a specific form of Leber Congenital Amaurosis. Reports on these trials are positive with relatively young children being treated. Several clinical trials for other forms of RP including Usher, Leber, Choroideremia and even Stargardt diseases are in progress or are being planned. Gene Therapy can also be used to deliver a therapeutic gene whose protein product prolongs the life of the photoreceptor neuron. This is called Pharmaceutical Gene Therapy. Thus, neuron-survival agents (neurotrophic agents) can be delivered to retinal cells. An example of the latter is the use of a virus vector to deliver agents such as CNTF or BDNF to the retina. 2) Pharmaceutical Therapy Pharmaceutical Therapy can be defined as the use of a chemical agent or protein that prolongs the life of a retinal cell. As mentioned above, these are called neuron-survival agents or neurotrophic agents that can be used when photoreceptors are damaged but some yet survive. Many of these agents have been identified and have been shown to be effective in prolonging the life of photoreceptor cells or even improving function in RD animal models. Great progress has been made with many neurotrophic agents such as the Rod-Derived Cone Viability Factor. A Clinical Trial is in progress (Neurotech Co.) using a Pharmaceutical Therapeutic approach. In this case, a neurotrophic factor (CNTF) is supplied to the retina using a tiny capsule implanted within the eye to slow photoreceptor degeneration. Early phases of this trial have been successfully completed and photoreceptor survival is enhanced for both RP and for dry AMD. Other pharmaceutical approaches are being pursued. For example, certain drugs already in use for other conditions may slow photoreceptor cell degeneration. In specific cases of RP where retinoid (vitamin A) metabolism is affected, pharmaceutical therapy seems to be able to successfully replenish supplies of the retinoid to the photoreceptor cell and improve vision. Some of these treatments could be applied to the RPs in general while others are specifically tailored to specific RP types. 3) Nutritional Therapy Various nutrition strategies, mostly using antioxidants, are being developed mainly based on the fact that severe oxidative damage occurs in the RD retina. This is true both in RP and in AMD. In animal models of RP, oxidative damage has been found to be a primary reason for photoreceptor cell damage and death. Antioxidants can markedly slow photoreceptor cell death in these RD animal models. Based on these findings, a small Clinical Trial has taken place using a special group of antioxidants 8 (“RetinaComplex”). Reported results to date have been favourable but more work is needed. Many other antioxidants are available for future study. 4) Photoreceptor/Stem Cell Transplants When all or most photoreceptor cells are dead, an obvious possibility for replacing the cells is transplanting new, normal photoreceptors into the retinal space occupied by the degenerated cells. Unfortunately, years have been spent on this effort in RP animal models with only marginal success. A clinical trial for photoreceptor transplantation has taken place but results have been inconclusive. Conversely, stem cell transplantation offers great hope for future treatment of both RP and AMD. Stem cells are primitive cells that have the potential of multiplying (replication) and ultimate development (differentiation) into any cell type found within the body. Stem cells are found in embryos but have also been shown to be present in many adult tissues (e.g., the retina) in small numbers. Several investigators are now conducting research on defining the conditions that will grow stem cells and direct them to differentiate into mature, functioning retinal photoreceptor or pigment epithelial cells. Other investigators are attempting to redirect cells such as retinal glial cells into a photoreceptor-like developmental pathway. Although some success is being obtained in preclinical work with animal models, this effort has far to go before it is shown to be effective and safe for human clinical trials. 5) Optogenetics Many animals and plants have light-sensing molecules within their cells. Optogenetics is a new field of research that combines optics with methods of molecular biology to place light-sensing molecules from one cell type into another cell type to make it photosensitive. For example, genes for a photosensitive protein called channelrhodopsin can be inserted using molecular biological techniques into remaining (non-photoreceptor) cells in a degenerating retina to make these cells sensitive to light. In this way, the lack of photoreceptors can be circumvented and vision restored. Inner retinal neurons such as bipolar and ganglion cells have already been targeted in animal models with insertion of light-sensitive molecules. Investigators are using many different approaches but already blind mice with inherited retinal degeneration have had vision restored. Several challenges still need to be overcome using some of the photosensitive proteins in animal experiments before human clinical trials can be undertaken. Some of the photosensitive proteins used only function at very high light levels and are thus potentially damaging to sensitive retinal cells while other proteins react too slowly to be useful in human vision. In spite of these problems though, optogenetics has great potential in human sight restoration. 6) Retinal Electronic Prostheses In cases where all photoreceptors are dead, the Retinal Electrical Prosthesis could “electronically” take their place and restore functional vision. With this device, a small external camera sends a visual image to a tiny electronic implant (called an electronic array) placed close to the retina – e.g., inserted into the subretinal space, tacked to the front surface of the retina or placed in the suprachoroidal space within the eye. This array stimulates remaining retinal neurons according to the external visual image it receives. The cells that receive the stimulus begin processing the electronic signal and pass it down the optic nerve to the brain for final synthesis as a visual image. Results on different prosthesis designs from several groups around the world are encouraging. 9 Much excellent preclinical work has led to human implantation with different types of the prosthesis. Alpha IMS, produced by Retina Implant AG, has a subretinal device with 1500 light-sensitive photodiodes with favourable patient implant results already published in the scientific literature. Second Sight Medical Products has completed a clinical trial with a device containing 60+ electrodes. This device has received the European CE mark and is being implanted into RP patients in several centres in Europe. Another electronic approach involves transcorneal electric stimulation. This technique shows promise in preserving vision without the need for invasive surgical intervention. Summary Much is now known about the inherited retinal degenerations. Specifically, enough is known to demonstrate scientific “Proof of Principle” in RD animal models that such interventions can be both efficacious and safe. Based on this, human Clinical Trials for these diseases are now taking place with many more to come in the next few years. Thus, we have now moved from basic work at the laboratory bench to human testing of a variety of treatments for retinal degenerative diseases. Dr Harry Bradshaw: vitreo-retinal surgeon on the cutting edge A highlight of the AGM held in Dunedin on August 22 was a talk by Dr Harry Bradshaw, a vitreo-retinal surgeon who has worked on the Moorfields genereplacement human clinical trial program in the UK. He recently returned to New Zealand and is currently based in Dunedin. Harry undertook the daunting task of explaining to our largely lay audience the genetic treatments being trialed for inherited retinal disorders. These disorders are primarily Retinitis Pigmentosa, Stargardts and Ushers. Vitreo-retinal surgery entails removing the vitreous jelly from the eyeball in order to undertake surgical intervention on the retina. “It is pretty tricky kind of work and takes a long time to learn,” Harry said, adding that it took him three-and-a-half years. “The dream has been to repair the [defective] genetic information or replace it, and prevent progression of the disease or perhaps even improve it.” The first challenge is to identify the particular genetic mutation that has caused, say, some variant of Retinitis Pigmentosa. Harry points out that there are three billion bits of information in our DNA, and it may be only two bits that are wrong. There are probably thousands of slightly different mutations responsible for RP alone. Laboratories all around the world are working on aspects of particular mutations, so the prospective trialist’s blood will be sent off to various labs for examination. “It’s quite a palaver,” he adds. This information all goes into a database which so far contains around 400,000 participants. Essentially most of these genetic retinal disorders come down to an excess of a certain protein, not enough of the protein, or one that is simply wrong. 10 Once the exact mutation is identified a correct genetic sequence is inserted into DNA to be put into the trialist’s eye via the medium of a virus. The hope then is that it will be accepted into the retina and the new genetic information will replace the defective protein. This process has been tried out on five patients at Moorfields so far. Since this work is still experimental, why do patients agree to participate in these clinical trials, Harry pondered. “The main reason people take part in these trials is that they often feel completely desperate, when given a diagnosis, particularly if the condition is progressive, and they are still quite young. So most of the patients who take part are in their 20s and 30s. A few older ones took part on the basis that it might help someone else. It involves a huge amount of work for the patient who is seen once a month, for three years, and not all dwell in London. And it is not just a brief procedure. Quite apart from the surgery, the pre and postoperative tests are extensive, and take one to two days of continual testing”. At present most trials are still examining whether the procedures are safe. Harry explains that several participants have such severe sight loss that they have little to lose. Unfortunately this means it is difficult to establish whether there is a benefit from the intervention. Ideally the researchers recruit patients who still have some vision so they can test whether they can prevent further vision loss. Harry believes that over the next 10 or 20 years more and more of the mutations that cause sight loss will be tackled using genetic treatments. One of the problems facing the researchers is that transferring corrective genetic material into the eye is done using viruses as vectors. “That involves an uncomfortable balance between destroying the cells and putting new ‘plans’ into them,” he says. When this work is done on mice, the mammal’s body eventually weeds out the intruding material. So it is still problematic how long the benefits of such procedures will last and whether they can be repeated. Harry also discussed stem cell work on Stargardts patients (this is an inherited retinal dystrophy marked by night vision then central vision loss). The United States holds a database of stem cells taken from four embryos which have been differentiated by a research based company to form Retinal Pigment Epithelium cells RPE. It is hoped those cells will not contain any mutations. These can be injected into suitable patients in the expectation that they will replace the degenerating RPE cells and preserve retinal function structurally and functionally. When Harry left London a third person had just been injected with these. “Because the stem cells do not belong to the patient, they [the patients] have to remain on life long immunosuppressant medication, essentially low dose chemotherapy. That’s quite an ask, when we don’t know whether the treatment will make any difference,” he says. Even more concerning, those stem cells could lose their cell cycle regulation and grow unrestrained – essentially turning into a tumour. Ideally stopping the anti-rejection drugs would allow the patients’ immune system to kill the implanted cells. But all this is very much an unknown so this research too is safety testing. 11 Fraught as it sounds, Harry describes it as “very exciting”. When he first went to London to do this work he hoped stem cells could be used to reverse the effects of AMD. But that has not turned out to be the case. By the time atrophy can be seen in the RPE,it has also affected the cells in the retinal layers above and below. So even if pristine RPE cells could be inserted into the eye they would effect little improvement in vision. One wet MD complication might in fact be receptive to this treatment, he says. In one case in 100 of wet MD there is blistering of the RPE, which can cause an RPE rip. One group of researchers in London has developed a technique where they make a wafer of collagen, on which they grow stem cells to replace the RPE. They detach the retina where it has been affected and slide the collagen wafer in. That dissolves and the RPE is replaced. Harry asks how all this research being undertaken in London can help his Retina NZ audience. He sees organisations and networks like ours as useful for tracking down potential patients who have the appropriate mutations. He comments that Dr Andrea Vincent in Auckland has started testing to see if she can identify people with genetic mutations. This can help the patient understand their prognosis. It will also give researchers some idea what sort of problems are here in NZ, when treatments do become available. Camille Guy QLT Inc has announced the sale of its drug Visudyne for $112.5 million to Valeant Pharmaceuticals International Inc. Visudyne helped QLT become one of Canada’s most successful biotechnology firms on the back of the treatment for age-related macular degeneration. But sales of the drug, which uses a combination of injections and light therapy to treat AMD, plunged after the launch of Genentech’s Lucentis and rising off-label use of cancer treatment Avastin. 12 Cane and Able : When the going gets bumpy by Trevor Plumbly For the lucky ones, sight loss is a pretty slow process and generally noticed more by others than by the victims themselves. Certainly the first indications I had came at me rather suddenly. I was sitting down, quietly enjoying a cup of tea when Pam said “we really need to talk”. Now anyone with experience of married life or full-time employment, knows that when someone says that, good news is not about to follow. The conversation started innocently enough along the lines of “do you realise how often you’re bumping into things lately?” I put on what I hoped was a pensive, reasonable expression, gathering reserves for the final verdict, which was “You need to get your eyes tested again, but go to someone a bit more high-tech than old Charlie”. I thought that, that was a bit harsh, Charlie had done my glasses for years and never given me any grief, anyway, if there was bad news out there why go looking for it. So I tried the usuals like, “I’ll think about it” and “as soon as work eases off a bit”. Occasionally this ploy works, but not this time and a couple of days later off I went, feet dragging, to the high-tech optometrist, mentally debating the difference between an optometrist and an optician and deciding it was probably the size of the bill. The high-tech optometrists looked more like a cemetry for posh spectacle frames than a place to get your sight fixed. The decor was a floor to ceiling sort of glaucoma grey which matched the hair colour of the chirpy, competent looking woman behind the counter. My optometrist shuffled and dealt the test lenses with all the panache of a croupier. While I gained confidence from this dexterity, she, obviously not satisfied, followed up the first act by shining a torch into my eyes and muttering ‘Hmm’. It’s a personal opinion of course, but coming from anyone in the medical game ‘Hmm’ has got to be one of scariest expressions in the English language. It has only two meanings. (A) ‘I haven’t got a clue what’s wrong with you’. Or (B) ‘I don’t want to be the one to tell you’. We talked about my vision and I mentioned having problems driving at night. She trumped this by saying “I don’t think you should be driving in daylight either but you definately need to see a specialist”. Not really what I wanted to hear, ‘seeing’ a specialist implied that what I had was beyond her ability, and then there was the cost. With Charlie it was in and out with glasses for around $300 but this little number was looking like $600 plus. The specialist oozed professional competence. He sat me in front of an ancient looking machine, explained the process, gave me a buzzer and said “press this when you see the lights”. Anxious to please I finally I spotted one and stabbed the button in triumph. From there we moved into the surgery which really looked as if it could do the biz bright lights, lots of neatly laid-out implements that looked essential and expensive, with a patient chair that could have been salvaged from a space shuttle. He did a more measured shuffle and deal with the test lenses as if he was catering to a high roller. After the magnifying light elicited another ‘hmm’ he rolled back his chair and said ‘you’ve got RP’. I chewed on this medical morsel for a moment then said ‘is that good?’ He gave me a short, honest opinion on the ‘joys’ of the condition. Briefly, I could lose my sight or some of it, quickly or slowly and it was inoperable. Somewhat poorer and not exactly buoyed with hope I left, with an appointment to the Dunedin Eye Clinic. 13 The clinic was all business, lots of fellow patients dotted around the waiting area, halfhoping and half-dreading the appointment and the result. The staff here are front line troops, a mix of eager younger types and battle-worn professionals who’ve seen it all before. Similar tests, same diagnosis, same prognosis, but good, helpful advice on what to expect and how to cope with it. Top marks to the public health system. I left quite upbeat and half-convinced there wasn’t too much wrong with my sight. But on reflection I guess it was more wishful thinking. Branch News Dunedin: At our July meeting, Dunedin VIPs and students from the University of Otago Design for Technology course brainstormed in small animated groups about the need for mainstream products that make life easier for people with low vision (instead of more difficult, as is often the case at present). The meeting was fun and the outcome has been immensely rewarding. The design concepts and prototypes presented to us by the students this week show that they listened carefully to our concerns and put a lot of time, thought and work into coming up with practical solutions. Lynley Hood Tauranga: The Tauranga branch of Retina NZ held its second meeting on 6 October, there were 11 of us. We had our first guest speaker librarian Harley who spoke about the services available for those with a print disability and the computer training available at the Library, we enjoyed a hot coffee and a bite to eat at a local cafe after the meeting. Come join us. We meet the first Saturday of each month at the Library in the Okahukura Room. For info contact Sara Ash on 07 5700917. Napier: Hawkes Bay held its first Retina meeting on Thursday 16 August. We held our first get together at the Silky Oak Chocolate Factory Cafe at 10am. Jean Martin and her partner John, Greta Hankins, John and Kathy Hands, Lloyd and Barbara Stokes, Evelyn Bennett and Lily Goslin and myself were in attendance. It was a beautiful day and really informal but a great way for a few of our members and friends to get together. Some members even stayed on for lunch. Linda Bartlett Kapiti: 25 people have regularly attended the Kapiti VIP support group over the last three months. The buzz of conversation in the room each month suggests that many friendships have been formed. We were visited by third year medical students doing a community week and we managed to share some valuable insights with them about patient communication. Other meetings hosted the regular RNZFB equipment display and a pearl threading specialist, who also demonstrated magnetic clasps which make putting on and taking off necklaces so much easier. It is planned to hold a Christmas function on 17 December, which will be a shared lunch starting 12.30 pm. Elizabeth East Auckland: A meeting of around 25 members attended on 9 September in Parnell. We heard an inspiring talk from Maria Williams who trained in nursing, psychology and molecular biology and now works as regional disability advisor at WINZ. Tea and much discussion followed. 14 From our mailbag Dear Camille, just a note to congratulate you on an excellent newsletter. You give us so much information on all sorts of aspects of vision loss, from current research to helpful hints around the home which make our life so much simpler. As a very active 76 year-old with the wet Macular Degeneration (currently having Avastin injections) I can only admire all those with impaired vision who can live life to the full, and be an inspiration to those of us who are losing our sight. The advances in technology are amazing, and you keep us informed as to what is available. I am looking forward to the recorded conference speakers, and wish you well as you help so many of us. Judy Campbell Hi Camille, I really enjoyed the August Retina newsletter and am still laughing over Trevor’s trips to restaurants. Just great and very informative as usual. Sally Coutts Media Review: Bardowl - new bird in the audiobook nest Bardowl, which is being touted as a Spotify for audiobooks, is currently limited to business and self-help titles. The best thing about Bardowl is its name. Partly it’s the cute animal logo but mostly it’s because the press pack reveals it was dreamt up by the founder’s wife after a Specials reunion concert in the UK. It works like this: users pay £9.99 a month for unlimited access to Bardowl’s library of audiobooks, which they stream via their iPhone or iPad. And, yes, you can listen offline. At the moment it’s limited to business and self-help titles – How to Get Rich by Felix Dennis, Overcoming Anxiety for Dummies, that sort of thing – but later this year it will start to offer fiction and a wider selection of non-fiction. The main difference between Bardowl and its main rival, Amazon-owned Audible, is that you’re streaming rather than downloading – there’s no need to invest in a whole audiobook and you can dip in and out of as many titles as you like. Bardowl also lets you share soundbites on Twitter, Facebook and LinkedIn, though I’m not sure why anyone would want to do that. Its app (which you can try free for a week) is a bit of a drain on the iPhone battery but is amazingly easy to use, and, once the range expands, should be well worth the subscription fee. For Bardowl to take off, though, it will have to overcome a little image problem, summed up by the top FAQ on Audible’s website: “Are audiobooks not just for the elderly and the blind?” Not at all, they answer, before offering some enlightening advice: “Unlike reading, you can listen to a book while doing other things, like household chores, walking, cooking and commuting to and from work.” We’re going to see a lot of “Spotify for x” startups over the next year or so: x being any content or entertainment that can be offered on an all-you-can-eat basis for a monthly subscription. 15 So, just as Lekiosk is seen as a Spotify for magazines, British startup Bardowl is pitching itself as a “Spotify for audiobooks”, aiming to disrupt the business of Audible, which has built its business around selling audiobook downloads. Bardowl launched its iPhone app earlier in June, with two key differences. First, it streams audiobooks to the device rather than downloads them, although the app uses local cacheing to store up to three hours of audio on the iPhone to last “a long tube journey or a short flight” for offline use. Second, it charges £9.99 a month for access to its entire catalogue, rather than charging by the book. There are no limits to the amount of hours or the amount of books that the user can listen to each month. It’s access not ownership, says chief executive Chris Book, who cofounded Bardowl. Social features are also built into Bardowl with a feature that makes it easier for people to share what they’re listening to with friends. “Users can extract 30 seconds of narrated audio from a book and share it as a link within messages sent to Twitter, Facebook and LinkedIn,” says Book. “We think this is an excellent way to increase awareness of the availability of audiobooks alongside their printed and digital counterparts and a way to enable our publisher partners to market titles and enter into social conversations with their end customers.” Reprinted from The Observer 16