
Macular degeneration: 'I've been given my sight back' - obeone
http://www.bbc.com/news/health-43458365
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Brakenshire
This is a phase 1 trial (usually just undertaken to check safety before larger
trials are done to test efficacy and dose), so it seems all the more amazing
that such a big improvement was seen.

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epmaybe
From the article, 29- and 21- letters improvement in visual acuity. I believe
this means at least 4-6 rows improvement from the chart you read off of.

Think about that for a second. Nearly blind, or actually blind, to being able
to read again.

I'm seriously debating specializing in ophthalmology, and sub-specializing in
retina surgery just became an even more appealing career plan for me.

~~~
philbarr
helping people be able to see again? sounds like a definition of "job
satisfaction"

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Mithaldu
Really depends on the person. I have very small sight problems (+.5/-.75)
which don't affect me most of the time, but are a danger in traffic. The
doctor that diagnosed that really didn't care to answer any of my questions.

~~~
Mithaldu
Did i kick someone's puppy here or what? I could understand getting this level
of disagreement if i claimed all doctors are psychopaths, but all i did was
point out that not all eye doctors enjoy their job. Not like i give a shit
about karma, but i am honestly curious what caused this seemingly extremely
irrational reaction.

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ropeadopepope
It's not nice to pee in your neighbor's new pool.

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Mithaldu
What.

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ropeadopepope
The tone of the thread was positive. Everybody knows there are doctors who
don't care about their patients. It didn't add anything to the conversation
and was needlessly negative. In other words: you peed in the pool. Don't pee
in the pool.

~~~
Mithaldu
I don't agree that that is a rational reaction, but it explains the reaction.
Thanks for the explanation. Allows me to know i didn't do anything wrong.

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maroonblazer
I'd love to know if similar work is being done for loss of hearing/deafness.
Is there an auditory equivalent to retinal pigment epithelium that nourishes
the hair cell stereocilia?

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robohoe
I would love to see if there are any updates/trials going on in regards to
hearing/deafness. Suffering from Tinnitus and hearing loss is not pretty.

~~~
todd102030
I'm by no means experienced in this field, but wouldn't cochlear implants
count to some extent? I know those sidestep the hearing loss rather than
fixing it directly, but the end result is essentially the same. My mom has
dealt with hearing loss since her early years of childhood, and peaking in
total hearing loss about 15 years ago. Just a year or two later, she was
included in a study and was able to get a cochlear implant for free. Not too
long after, she was lucky enough to be offered a second one at no charge (the
benefits of being a beta tester), and to this date, she's still being given
new speech processors that constantly improve the quality of sound as well as
adding new features. It honestly blows me away with what they've done already
with hearing, and I won't be surprised if in 10-15 years, those implants
surpass a normal human's capabilities, and people who still have perfectly
working ears start getting them.

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reasonattlm
Most stem cell therapies produce benefits through the signaling of the
transplanted cells, near all of which die off rather than integrate. This is
an example of an approach that goes beyond that, providing a set of more
organized cells in a thin tissue segment that looks more like the native
tissue. When this was done for the heart, 10% of cells survived (this is a
large number in the scope of stem cell therapies).

So it is interesting to speculate on the degree to which the benefits here are
signaling versus cell integration. Clearly the big difference between past
attempts is that a tissue-like set of organized cells are delivered rather
than just free-floating unorganized cells.

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logronoide
My mother had macular degeneration. She lost almost all vision in a couple of
years. She was a very active women all her life, a business woman when women
had to ask permission to her husband to open a bank account. It was devasting
for such an independent person. She was deeply depressed and being in her
early seventies her health deteriorated quickly, finally dying.

If researchers find a solution it will improve the quality of life of millions
elder people.

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bennyg
I have had 4 retinal detachment surgeries on my left eye, with the last
rendering it blind.

I had one retinal detachment surgery and a subsequent cataract surgery (since
vitrectomy almost always results in a cataract) on my right eye. It's fairly
stable but my vision is not great.

I'm hoping for stem cell therapy sometime in my life to help repair areas of
my right eye that are no longer great (retinoschisis and general rod/cone
dystrophy) and to make sure I'm seeing well into my later years of life.

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tombert
About a year and a half ago, my wife was showing symptoms of early-onset
macular degeneration, and it led to a rabbit hole of me searching for
treatments, and I remember the earlier stages of this being discussed.

While it turned out that my wife's issue was (fortunately) something less
severe, it still makes me happy to see that this treatment has had some
success.

~~~
abledon
I'm really curious to see what the statistics of early-onset macular
degeneration will be in 30-40 years, when a large portion of the population
will have been looking at screens 10+ hours a day, for 30+ years. I suspect
humans current eye-usage habits will affect the numbers.

results in this area of the field can't come fast enough!

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davidw
Wow, that's pretty cool. One of the things I worked on a couple companies back
was this:
[https://www.centervue.com/products/compass/](https://www.centervue.com/products/compass/)
which is a system used to measure how much people are losing their vision from
things like macular degeneration.

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pmarreck
> Cells from a human embryo

Are they sure? They can now harvest stem cells from regular tissue and blood,
as I understand it

~~~
Brakenshire
> Are they sure? They can now harvest stem cells from regular tissue and
> blood, as I understand it

There are adult stem cells which are differentiated or partly differentiated
to particular systems, eyes, skin, blood etc, and there are induced
pluripotent stem cells, where chemical or viral factors have been used to
'wind back the clock' for adult, differentiated cells back to completely non-
differentiated (pluripotent) stem cells, equivalent to embryonic stem cells.

In the long run, it seems that embryonic stem cells won't be needed, but for
the moment induced pluripotent cells are not 100% understood, everywhere apart
from Japan clinical researchers are wary of using them in human trials,
because they are not completely certain a particular preparation doesn't have
abnormalities. Embryonic stem cells for the moment are the gold standard, and
some work is needed to iron out the technology to standardize and check the
process for inducing pluripotency.

But in principle any cure proven with embryonic stem cells could be 'ported'
over to induced pluripotent stem cells once the iPSC process is standardized,
it's just a question of whether you want to pursue these two research paths in
parallel, or wait for the iPSC problems to be ironed out before even starting
the process of running human trials.

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hcknwscommenter
Very well put. I would add that it could take decades to "iron out" the issues
with iPSCs. It could also never actually happen. For example, the iPSC
generation process induces oncogenic properties in the cells (
[https://www.ncbi.nlm.nih.gov/pubmed/22998387](https://www.ncbi.nlm.nih.gov/pubmed/22998387)
). It could be that in the next few decades, iPSCs just turn out to be a lot
less safe than embryonic stem cells. Considering the hundreds of millions of
dollars it could take to move stem cell therapies like this through the
validation and regulatory approval process, it would be foolish to handicap
the process by using "unproven" iPSCs in favor of "gold standard" embryonic
stem cells.

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etskinner
>Both patients in the trial had "wet" age-related macular degeneration. This
form of the disease is caused by abnormal blood vessels growing through the
retinal pigment epithelium and damaging the macula. Dry age-related macular
degeneration is more common and caused by the retinal pigment epithelium
breaking down.

If this is a viable treatment method, it would help only the minority of
macular degeration patients.

~~~
pinum
>It is hoped the patch will be able to treat both forms of the disease.

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jtl999
What's the latest on optic nerve regeneration with stem cells?

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mosselman
“He was one of two patients given pioneering stem" I find the tiny test groups
in medical ‘research’ so strange.

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sp332
It's a test to see if they should bother moving forward. You don't want to
gear up a big study just to find out that the treatment gives everyone cancer
(which is a concern mentioned in the article) or that it doesn't work at all.
You do a pilot study to see if there is potential before you waste a lot of
work and maybe hurt people.

Anyway if the effect size is big enough, you don't need a really large study
to get statistical significance. I mean, what are the odds of macular
degeneration reversing by chance?

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sametmax
We do the same in web ergonomics. Testing you Ui on 3 people is enough to see
the biggest flaws of the tool and engaging in more expenses. See the excellent
book "don't make me think" for a quick and efficient write up on the topuc.

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chapill
>Cells from a human embryo were grown into a patch that was delicately
inserted into the back of the eye.

I assume the baby didn't survive the process since there's no mention of that.

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philbarr
It's not a baby, it's an embryo. Lots of embryos don't survive for lots of
reasons.

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gottebp
It's a philosophical dilemma that demands rigour. When does a human person
begin? If a few cells cannot be a person, well, adults are only bigger clumps
of cells. What is the threshold? If personhood begins when the cells are
independently viable, well, most infants are not independently viable and some
elderly aren't either. What is the threshold? By contrast: The moment when it
all begins and the DNA is first unique, and the potentiality for an adult is
present is very simple and philosophically elegant. If personhood comes later,
such as implantation, birth, 7th birthday, etc. it is difficult to give a
basis in the philosophical sense. Many philosophers have struggled with this
and it's no simple thing.

