Download November 2012 - Retina New Zealand

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.
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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
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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.
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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)
-
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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.
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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 –
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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.
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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
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(“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.
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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.
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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.
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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.
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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.
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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.
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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.
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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
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