15 ms·

Macular degeneration: 'I've been given my sight back'

295 points by obeone 9y ago

mosselman 9y ago
“He was one of two patients given pioneering stem" I find the tiny test groups in medical ‘research’ so strange.
- sp332 9y ago
  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?
  - sametmax 9y ago
    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.
- bluGill 9y ago
  unfortunately that is all we have. There is nobody on earth with your exact genetic makeup (even identical twins, though then the random mutation is small enough to ignore: consider it an exception). As a result nothing exactly will behave correctly for you. A drug that causes death in 99% of those it is tried on might be the one you need, but because it is so deadly trials will ensure it never makes it to market.
- ainiriand 9y ago
  It is normal in phase 1 trials involving complicated surgeries.
pmarreck 9y ago
> Cells from a human embryo Are they sure? They can now harvest stem cells from regular tissue and blood, as I understand it
- deleted 9y ago
  [deleted]
- matte_black 9y ago
  Yea but they probably need to be young versions of your stem cells. iPS stem cells from old cells will still be fraught with DNA damage and can be cancerous.
- maxerickson 9y ago
  It's the BBC. They make mistakes, but it is entirely reasonable to start from the assumption that they have not made such a big mistake. Anyway, here's a publication about the study that makes it pretty clear that they got it right (abstract only, paywall): https://www.nature.com/articles/nbt.4114 https://www.nature.com/articles/nbt.4114 (I found the study by assuming a page like http://www.thelondonproject.org/ http://www.thelondonproject.org/ would exist and then following the link provided)
  - ars 9y ago
    http://sci-hub.tw/10.1038/nbt.4114 http://sci-hub.tw/10.1038/nbt.4114
- Brakenshire 9y ago
  > 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.
Brakenshire 9y ago
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.
- epmaybe 9y ago
  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 9y ago
    helping people be able to see again? sounds like a definition of "job satisfaction"
    - pstrazzulla 9y ago
      My dad is an eye doctor and loves it for this reason.
    - darkarmani 9y ago
      > sounds like a definition of "job satisfaction" Depends if you like dealing with people, right?
    - Mithaldu 9y ago
      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 9y ago
        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.
reasonattlm 9y ago
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.
chapill 9y ago
>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.
- jackhack 9y ago
  This is important because, for some, the moral dilemma would preclude this treatment.
  - chapill 9y ago
    It's also important because a baby was killed to give a rich old guy a few more years of sight. A whole new underclass of children worthy of murdering for medical treatment arises.
    - eganist 9y ago
      http://www.dictionary.com/browse/baby http://www.dictionary.com/browse/baby Embryo is not included in any standard definition of the word, so you may wish to reconsider your word choice. Or not. Your call.
      - nsxwolf 9y ago
        "Human" is harder to dodge though.
        tialaramex 9y ago
        Sure, we know that humans expect small pieces of human to be treated as irrelevant (e.g. you leave tiny skin particles and hairs basically everywhere and all you usually ask is that people vacuum them up to avoid the place being covered in dust) and that larger pieces (e.g. a toe, or in this case an embryo) should be treated with extra care and consideration of people's emotional reaction. But it's not kidnapping if you keep someone's toenail clippings. It's creepy, but it's not kidnapping. It's not murder when the barber sweeps all the hair into a furnace, it's called tidying up.
mjh2539 9y ago
I regularly see comments on YC about Signal-to-Noise ratio, and how YC votes are meant to distinguish the "signal" from the "noise", but in this case, it's clear that this isn't what they're being used for. They're being used to enforce a preconceived bias. Of the two comments that addressed the moral indignity of the treatment (that it requires the death (murder, in most cases) of an innocent human person for its success) they were considered to be "noise".
- telchar 9y ago
  Misleading comment. You're talking about murder but this only involved embryos. Many/most people don't consider that murder any more than the spilling of seed is. If you want to push a political view don't couch it as a systemic complaint.
  - mjh2539 9y ago
    You're begging the question that one of the following two is false: 1. The embryos in question are human, innocent, and alive. 2. Murder is the intentional killing of an innocent human life. I'm going to infer that you're begging the question that (1) is false. Your argument seems to be an appeal to popularity: most people disbelieve x, therefore x is false. But this doesn't follow. I'd like you to give your reasons as to why it's not true.
    - dsr_ 9y ago
      This is the fourth time in this thread that you have attempted to reiterate this argument. Nearly everyone disagreeing with you is contesting your implication that an embryo is "a human" as opposed to "a potential human", and they further believe that "a potential human" does not have the same rights as "a human". You don't appear to have a follow-up to that argument other than "isn't that just your opinion." In order to convince people, you will probably need a better answer.
      - mjh2539 9y ago
        > You don't appear to have a follow-up to that argument other than "isn't that just your opinion." This is patently false. I have tried to give arguments but people are generally incapable of understanding what "begging the question" means. I cannot convince people that are not rational. Something is either a human, or it isn't. If it isn't a human, it has to be something else. Assume that the embryo is not a human. It follows that it has to be something else. If it is something else, what is it? If you say "an embryo", this is circular (the embryo is an embryo). If you say "not a human", you're begging the question. What's left? You have to attribute it as some other species of being, but if you do this, you're committing yourself to the absurd proposition that humans conceive something other than humans, and these non-humans then become humans. This is complex (and absurd) in a way that saying that human beings conceive other human beings is not.
maroonblazer 9y ago
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?
- robohoe 9y ago
  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 9y ago
    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.
  - DoreenMichele 9y ago
    Alternative remedy groups recommend magnesium supplements to treat tinnitus. I know at least one person whose tinnitus improved and became more bearable after improving their magnesium status. I rapidly get noise sensitive when I become magnesium deficient due to fever or vomiting and it rapidly resolves with consuming magnesium rich foods. If you are magnesium deficient, you may also need calcium, vitamin D and vitamin K to fully resolve the issue.
- snegu 9y ago
  This lab at Stanford is working on some similar things (there are also other research centers I can't recall right now): https://hearinglosscure.stanford.edu/ https://hearinglosscure.stanford.edu/ I'm watching them like a hawk hoping that some day my 2 year old son might benefit from their work.
etskinner 9y ago
>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 9y ago
  >It is hoped the patch will be able to treat both forms of the disease.
- AnimalMuppet 9y ago
  If I understand correctly, "wet" progresses much more rapidly than "dry". You're much more likely to unexpectedly lose your sight to "wet" (as opposed to just slowly having your sight deteriorate). Feel free to correct if I'm wrong. I'm not an expert. This is just what I've gathered from having two family members with it.
tombert 9y ago
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 9y ago
  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!
jtl999 9y ago
What's the latest on optic nerve regeneration with stem cells?
logronoide 9y ago
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.
- deleted 9y ago
  [deleted]
bennyg 9y ago
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.
davidw 9y ago
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.

295 points by obeone 9y ago

mosselman 9y ago

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

sp332 9y ago
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?
- sametmax 9y ago
  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.
bluGill 9y ago
unfortunately that is all we have. There is nobody on earth with your exact genetic makeup (even identical twins, though then the random mutation is small enough to ignore: consider it an exception). As a result nothing exactly will behave correctly for you. A drug that causes death in 99% of those it is tried on might be the one you need, but because it is so deadly trials will ensure it never makes it to market.
ainiriand 9y ago
It is normal in phase 1 trials involving complicated surgeries.

pmarreck 9y ago

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

deleted 9y ago
[deleted]
matte_black 9y ago
Yea but they probably need to be young versions of your stem cells. iPS stem cells from old cells will still be fraught with DNA damage and can be cancerous.
maxerickson 9y ago
It's the BBC. They make mistakes, but it is entirely reasonable to start from the assumption that they have not made such a big mistake. Anyway, here's a publication about the study that makes it pretty clear that they got it right (abstract only, paywall): https://www.nature.com/articles/nbt.4114 https://www.nature.com/articles/nbt.4114 (I found the study by assuming a page like http://www.thelondonproject.org/ http://www.thelondonproject.org/ would exist and then following the link provided)
- ars 9y ago
  http://sci-hub.tw/10.1038/nbt.4114 http://sci-hub.tw/10.1038/nbt.4114
Brakenshire 9y ago
> 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.

Brakenshire 9y ago

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.

epmaybe 9y ago
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 9y ago
  helping people be able to see again? sounds like a definition of "job satisfaction"
  - pstrazzulla 9y ago
    My dad is an eye doctor and loves it for this reason.
  - darkarmani 9y ago
    > sounds like a definition of "job satisfaction" Depends if you like dealing with people, right?
  - Mithaldu 9y ago
    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 9y ago
      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.

reasonattlm 9y ago

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.

chapill 9y ago

>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.

jackhack 9y ago
This is important because, for some, the moral dilemma would preclude this treatment.
- chapill 9y ago
  It's also important because a baby was killed to give a rich old guy a few more years of sight. A whole new underclass of children worthy of murdering for medical treatment arises.
  - eganist 9y ago
    http://www.dictionary.com/browse/baby http://www.dictionary.com/browse/baby Embryo is not included in any standard definition of the word, so you may wish to reconsider your word choice. Or not. Your call.
    - nsxwolf 9y ago
      "Human" is harder to dodge though.
      - tialaramex 9y ago
        Sure, we know that humans expect small pieces of human to be treated as irrelevant (e.g. you leave tiny skin particles and hairs basically everywhere and all you usually ask is that people vacuum them up to avoid the place being covered in dust) and that larger pieces (e.g. a toe, or in this case an embryo) should be treated with extra care and consideration of people's emotional reaction. But it's not kidnapping if you keep someone's toenail clippings. It's creepy, but it's not kidnapping. It's not murder when the barber sweeps all the hair into a furnace, it's called tidying up.

mjh2539 9y ago

I regularly see comments on YC about Signal-to-Noise ratio, and how YC votes are meant to distinguish the "signal" from the "noise", but in this case, it's clear that this isn't what they're being used for. They're being used to enforce a preconceived bias. Of the two comments that addressed the moral indignity of the treatment (that it requires the death (murder, in most cases) of an innocent human person for its success) they were considered to be "noise".

telchar 9y ago
Misleading comment. You're talking about murder but this only involved embryos. Many/most people don't consider that murder any more than the spilling of seed is. If you want to push a political view don't couch it as a systemic complaint.
- mjh2539 9y ago
  You're begging the question that one of the following two is false: 1. The embryos in question are human, innocent, and alive. 2. Murder is the intentional killing of an innocent human life. I'm going to infer that you're begging the question that (1) is false. Your argument seems to be an appeal to popularity: most people disbelieve x, therefore x is false. But this doesn't follow. I'd like you to give your reasons as to why it's not true.
  - dsr_ 9y ago
    This is the fourth time in this thread that you have attempted to reiterate this argument. Nearly everyone disagreeing with you is contesting your implication that an embryo is "a human" as opposed to "a potential human", and they further believe that "a potential human" does not have the same rights as "a human". You don't appear to have a follow-up to that argument other than "isn't that just your opinion." In order to convince people, you will probably need a better answer.
    - mjh2539 9y ago
      > You don't appear to have a follow-up to that argument other than "isn't that just your opinion." This is patently false. I have tried to give arguments but people are generally incapable of understanding what "begging the question" means. I cannot convince people that are not rational. Something is either a human, or it isn't. If it isn't a human, it has to be something else. Assume that the embryo is not a human. It follows that it has to be something else. If it is something else, what is it? If you say "an embryo", this is circular (the embryo is an embryo). If you say "not a human", you're begging the question. What's left? You have to attribute it as some other species of being, but if you do this, you're committing yourself to the absurd proposition that humans conceive something other than humans, and these non-humans then become humans. This is complex (and absurd) in a way that saying that human beings conceive other human beings is not.

maroonblazer 9y ago

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?

robohoe 9y ago
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 9y ago
  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.
- DoreenMichele 9y ago
  Alternative remedy groups recommend magnesium supplements to treat tinnitus. I know at least one person whose tinnitus improved and became more bearable after improving their magnesium status. I rapidly get noise sensitive when I become magnesium deficient due to fever or vomiting and it rapidly resolves with consuming magnesium rich foods. If you are magnesium deficient, you may also need calcium, vitamin D and vitamin K to fully resolve the issue.
snegu 9y ago
This lab at Stanford is working on some similar things (there are also other research centers I can't recall right now): https://hearinglosscure.stanford.edu/ https://hearinglosscure.stanford.edu/ I'm watching them like a hawk hoping that some day my 2 year old son might benefit from their work.

etskinner 9y ago

>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 9y ago
>It is hoped the patch will be able to treat both forms of the disease.
AnimalMuppet 9y ago
If I understand correctly, "wet" progresses much more rapidly than "dry". You're much more likely to unexpectedly lose your sight to "wet" (as opposed to just slowly having your sight deteriorate). Feel free to correct if I'm wrong. I'm not an expert. This is just what I've gathered from having two family members with it.

tombert 9y ago

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 9y ago
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!

jtl999 9y ago

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

logronoide 9y ago

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.

deleted 9y ago
[deleted]

bennyg 9y ago

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.

davidw 9y ago

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.