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FDA Approves First-Ever Gene Therapy For Inherited Form of Blindness (sciencealert.com)

schwit1 shares a report from ScienceAlert: In a historic move, the Food and Drug Administration on Tuesday approved a pioneering gene therapy for a rare form of childhood blindness, the first such treatment cleared in the United States for an inherited disease. The approval signals a new era for gene therapy, a field that struggled for decades to overcome devastating setbacks but now is pushing forward in an effort to develop treatments for haemophilia, sickle-cell anaemia, and an array of other genetic diseases. Yet the products, should they reach patients, are likely to cost as much as $1 million for both eyes.

35 of 58 comments (clear)

  1. Re:A new era of Greed by Anonymous Coward · · Score: 1

    First steps are always expensive.

  2. Re:Huh? by Opportunist · · Score: 3, Insightful

    Why do you hate freedom of choice? Instead of being condemned to have health care, you can freely choose between having health insurance and eating.

    --
    We used to have a Bill of Rights. Now, with the rights gone, all we have left is the bill.
  3. Re:A new era of Greed by geekmux · · Score: 1

    First steps are always expensive.

    If this logic held true, diabetics would be able to buy insulin at the local dollar store, and QVC would be running a holiday special on cancer treatments.

  4. Re:A new era of Greed by Higaran · · Score: 1

    Currently stats say that only less than 10 percent of people in the USA have diabetes, if that number was closer to 50% then hell yes you would be able to get all your supplies at the dollar store. If you look it is a large enough percentage of people to be alarmed, but it's not enough of an issue to make enough people need those medical supplies to make the massive scale of production worth it.

  5. It needs to be more permanent by Baron_Yam · · Score: 1, Flamebait

    Wake me when they update the gonads so the repair is heritable.

    1. Re:It needs to be more permanent by Baron_Yam · · Score: 1

      Correcting the defect in the parent in a heritable way is less expensive - once you have the treatment designed, of course. But ultimately a needle is going to be a lot less expensive than IVF, and that's before recognizing that a percentage of people will simply have kids and hope the gene isn't passed on.

  6. Drug prices and production scale by sjbe · · Score: 3, Insightful

    Currently stats say that only less than 10 percent of people in the USA have diabetes, if that number was closer to 50% then hell yes you would be able to get all your supplies at the dollar store.

    "Only 10%"? That's over 30 million people. WELL past minimum efficient scale for production and distribution. Anything that affects double digit percentages of the US population is a gigantic market for a single drug.

    The reasons medications are expensive is because in the US we have a completely retarded system for buying them that gives all the power in the relationship to the drug company. They charge a lot because they can. Most countries solve this by having a single payer system so drug prices get regulated to reasonable prices. Evidently we aren't so smart in the US so we pay far more than almost anywhere else.

    1. Re:Drug prices and production scale by geekmux · · Score: 4, Informative

      When you are trying to be a righteous asshole, the first step is to get your facts fucking right. Most diabetics dont use insulin. Full fucking stop. Dipshit.

      Almost a century ago, scientists discovered insulin, and found it could be used to treat diabetes. They sold that patented idea for $1 as a goodwill gesture because they knew their discovery could save millions of lives. Greed took over and turned insulin into a $24 billion dollar global industry by 2014, and it targeted to be almost a $50 billion dollar industry by 2020.

      Greed is the only fucking reason this product still costs so much in the US. And regardless of how many people use insulin, diabetes is still one of the largest killers in our society. Get your fucking facts straight next time.

    2. Re:Drug prices and production scale by Mashiki · · Score: 1

      Greed is the only fucking reason this product still costs so much in the US. And regardless of how many people use insulin, diabetes is still one of the largest killers in our society. Get your fucking facts straight next time.

      Yeah not really. The reason the product still costs so much in the US is because of a variety of things, everything ranging from the source of said insulin and groups like PETA that try to shut it down. To the new types of insulin that are tested and developed because of insulin resistance. Diabetes isn't one of the largest killers in your society. That's heart disease, cancer and diseases related to weakened immune systems from secondary factors(old age, etc). You're more likely to die of malaria in the US then diabetes. FYI the reason it was sold for $1 wasn't because of a goodwill gesture, you should go read up some more about Frederick Banting and Rickard Macleod. There's far more to that story then you understand, or go take a trip to London, Ontario visit Banting House where they figured it all out.

      And before you or some sad AC troll replies with a "but u don't know anything about diabetics" or something else, I'll just add that having a sister who was a juvenile diabetic back in the 1980's, I do know a few things. You know those lovely 1980's where the child mortality rate from undiagnosed diabetes in young children still had a 50% death rate. Look at how far we've come, it's less then 5% now in the west...in 30 years.

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      Om, nomnomnom...
    3. Re:Drug prices and production scale by CrimsonAvenger · · Score: 1

      "Only 10%"? That's over 30 million people. WELL past minimum efficient scale for production and distribution. Anything that affects double digit percentages of the US population is a gigantic market for a single drug.

      That 10% number is for both Type 1 and Type 2 (and presumably Type 3 included with the Type 1). Type 1 diabetes (the kind you need insulin for) amounts to about 1.25 million people, so rather less than 0.5%....

      --

      "I do not agree with what you say, but I will defend to the death your right to say it"
    4. Re:Drug prices and production scale by XXongo · · Score: 1

      Most diabetics dont use insulin. Full fucking stop. Dipshit.

      "Diabetes" is actually two entirely different diseases, "type 1 diabetes" and "type 2 diabetes". Despite the same name, they have entirely different causes. Type 1 diabetes patients don't produce insulin. Type 2 diabetes patients are resistant to insulin.

      Type 1 diabetes was fatal before insulin.

    5. Re:Drug prices and production scale by geekmux · · Score: 2

      Greed is the only fucking reason this product still costs so much in the US. And regardless of how many people use insulin, diabetes is still one of the largest killers in our society. Get your fucking facts straight next time.

      Diabetes isn't one of the largest killers in your society. That's heart disease, cancer and diseases related to weakened immune systems from secondary factors(old age, etc).

      I didn't say it was our largest killer, I said it was one of our largest killers, and 80,000 deaths per year makes that fact pretty damn clear. It was ranked 7th on our list in 2014, and I doubt much has changed since then. And you haven't even identified our actual largest killer, which is a product we call cigarettes. Greed again clarifies why this top killer is a legal product today.

      You're more likely to die of malaria in the US then diabetes.

      You're really degrading your fact-checking capability now. There are less than 2,000 cases of malaria reported in the US each year. That's a far cry from diabetes.

      FYI the reason it was sold for $1 wasn't because of a goodwill gesture, you should go read up some more about Frederick Banting and Rickard Macleod. There's far more to that story then you understand, or go take a trip to London, Ontario visit Banting House where they figured it all out.

      This fact has already been clarified here, and $1 back in 1921 is as much of a goodwill gesture as $1000 would be today.

      https://www.healio.com/hematology-oncology/news/print/hemonc-today/%7Bb3848683-e962-43ac-b23b-5b2c10f711a8%7D/frederick-banting-discovered-insulin-in-1921

      And before you or some sad AC troll replies with a "but u don't know anything about diabetics" or something else, I'll just add that having a sister who was a juvenile diabetic back in the 1980's, I do know a few things. You know those lovely 1980's where the child mortality rate from undiagnosed diabetes in young children still had a 50% death rate. Look at how far we've come, it's less then 5% now in the west...in 30 years.

      My argument has nothing to do with how far we've come. My argument has to do with Greed, which adds to those 80,000 deaths per year. When people cannot afford the very chemical required to sustain life, it's a death sentence, which sadly still rings true today. You know this.

    6. Re:Drug prices and production scale by Mashiki · · Score: 1

      I didn't say it was our largest killer, I said it was one of our largest killers, and 80,000 deaths per year makes that fact pretty damn clear. It was ranked 7th on our list in 2014, and I doubt much has changed since then. And you haven't even identified our actual largest killer, which is a product we call cigarettes. Greed again clarifies why this top killer is a legal product today. You're really degrading your fact-checking capability now. There are less than 2,000 cases of malaria reported in the US each year. That's a far cry from diabetes.

      Except that your actual largest killer isn't cigarettes. It's not even the underlying cause. More people die from strokes unrelated to smoking then strokes directly related to smoking. You should probably look up those stats a bit more.

      This fact has already been clarified here, and $1 back in 1921 is as much of a goodwill gesture as $1000 would be today.

      No, because they sold the patent to the university for $1 to allow open production by multiple companies. Because it was an actual "treatable health threat" and an easy one at that. That link doesn't even explain the reasons "why" it was sold for $1 to UoT, go on read a bit more.

      My argument has to do with Greed, which adds to those 80,000 deaths per year. When people cannot afford the very chemical required to sustain life, it's a death sentence, which sadly still rings true today. You know this

      Except it's not. Those 80k deaths are not the primary cause, it's an underlying cause. It's not even secondary, it's usually 3rd or 4th underlying. Now here's the interesting party, you can bet that many of those people are on insulin. Many of those people simply didn't or refused to take actual care of themselves. I know diabetics that are in their 30's and have lost fingers, toes, feet, an entire leg all because of their own lack of action, or because they simply didn't care. Either not eating right, not caring to take insulin, eating foods that spike their blood sugar, and so on. That's *in* Canada, where insulin is covered. Primary deaths by diabetes are under the number of deaths in the US by malaria.

      Insulin isn't a chemical either, not even close to one. If you're going to argue with someone at least get your shit right.

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      Om, nomnomnom...
    7. Re:Drug prices and production scale by IMightB · · Score: 1

      I don't even know how to respond to this one... Half of me wants to agree with you on some points... My father did die of diabetes related issues, he was a type 1 who didn't take care of himself following a stroke. Is it his fault or is it the brain damage... Because a stroke IS brain damage.

      The other half of me wants to call you a full blown retard. The patent was sold for 1 dollar for altruistic reasons. Tell me, where is the altruism today? There is none in the corps involved in pharmaceutical manufacturing today. Corps have these We meet guidelines, feel good, publicity programs. But tell me why they simply don't lower prices on drugs that have been around for 50-60 years? Then you have humanitarians like Martin Shkreli, who seem to represent the whole of the industry.

    8. Re:Drug prices and production scale by IMightB · · Score: 1

      Hahahhahahah Insulin isn't a chemical? When was the last time you looked up that definition? You must be a Canadian Trump Voter.

    9. Re:Drug prices and production scale by geekmux · · Score: 2

      I didn't say it was our largest killer, I said it was one of our largest killers, and 80,000 deaths per year makes that fact pretty damn clear. It was ranked 7th on our list in 2014, and I doubt much has changed since then. And you haven't even identified our actual largest killer, which is a product we call cigarettes. Greed again clarifies why this top killer is a legal product today. You're really degrading your fact-checking capability now. There are less than 2,000 cases of malaria reported in the US each year. That's a far cry from diabetes.

      Except that your actual largest killer isn't cigarettes. It's not even the underlying cause. More people die from strokes unrelated to smoking then strokes directly related to smoking. You should probably look up those stats a bit more.

      The CDC still classifies cigarettes/tobacco use as the #1 cause of preventable death. Smoking causes heart disease and cancer, and with over 400,000 deaths per year, it's pretty safe to say it's one hell of a contributor to our top killers as an "underlying" cause. Little point in splitting hairs over that.

      My argument has to do with Greed, which adds to those 80,000 deaths per year. When people cannot afford the very chemical required to sustain life, it's a death sentence, which sadly still rings true today. You know this

      Except it's not. Those 80k deaths are not the primary cause, it's an underlying cause. It's not even secondary, it's usually 3rd or 4th underlying. Now here's the interesting party, you can bet that many of those people are on insulin. Many of those people simply didn't or refused to take actual care of themselves. I know diabetics that are in their 30's and have lost fingers, toes, feet, an entire leg all because of their own lack of action, or because they simply didn't care. Either not eating right, not caring to take insulin, eating foods that spike their blood sugar, and so on.

      Given your logic, you would also argue that it wasn't the drugs or obesity that killed Elvis, it was a toilet that did him in. Go argue with the CDC and their facts when it comes to deaths directly attributed to diabetes. No shit people die from not taking care of themselves; diabetes is literally caused by not taking care of yourself. It's completely preventable in the vast majority of people. Needless to say, laziness and don't-give-a-shit syndrome aren't tracked by the CDC/WHO. If they were, it would be the overwhelming cause of death for all humans by a long shot.

    10. Re:Drug prices and production scale by Mashiki · · Score: 1

      Hahahhahahah Insulin isn't a chemical? When was the last time you looked up that definition? You must be a Canadian Trump Voter.

      https://en.wikipedia.org/wiki/...

      Don't let the stupid hurt you.

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      Om, nomnomnom...
    11. Re:Drug prices and production scale by Mashiki · · Score: 1

      My father did die of diabetes related issues, he was a type 1 who didn't take care of himself following a stroke. Is it his fault or is it the brain damage... Because a stroke IS brain damage.

      While you have my sympathies on your father, that's a case where your father lacked fundamental and proper care. Either from a lack of proper teaching, or a lack of not having organizations like CCAS(as we call it here in Ontario), who are nurses who check on patients who are unable to properly care for themselves. But I'll also bet your father was: A drinker, didn't give two whits about their cholesterol, didn't have regular checkups and ignored symptoms thinking that "it'll just go away." Which is very common in men. Ask yourself how many times you'll feel something wrong and go "well if it goes away in a few, I won't worry about it."

      The patent was sold for 1 dollar for altruistic reasons. Tell me, where is the altruism today?

      No it really didn't, the reasons were far beyond that. Regular R-insulin is dirt cheap, I can cross the border into Michigan and buy it for $9/mo at walmart. It's $11.50/mo from walmart in most locations in Ohio for instance. That altruism is still around, you don't hear it. Just like you don't hear about the cop, ems, firefighter that's paying out of their own pocket to put some family kicked out of their house into a motel. Or the cop who refused to file a charge against someone who was caught stealing food at a grocery store, instead paid for it out of their own pocket and took the person(and/or their family) to their house for meals.

      Then you have humanitarians like Martin Shkreli, who seem to represent the whole of the industry.

      Far more to that story, far more.

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      Om, nomnomnom...
    12. Re:Drug prices and production scale by Mashiki · · Score: 1

      The CDC still classifies cigarettes/tobacco use as the #1 cause of preventable death. Smoking causes heart disease and cancer, and with over 400,000 deaths per year, it's pretty safe to say it's one hell of a contributor to our top killers as an "underlying" cause. Little point in splitting hairs over that.

      But the CDC doesn't classify that as the cause. Rather it's the underlying cause, those things cause other issues. Heart disease, cancer are what kill the people.

      Given your logic, you would also argue that it wasn't the drugs or obesity that killed Elvis, it was a toilet that did him in. Go argue with the CDC and their facts when it comes to deaths directly attributed to diabetes. No shit people die from not taking care of themselves; diabetes is literally caused by not taking care of yourself.

      And there's the part where you go from making some sense to none at all. You should go read the CDC's facts, because they don't their secondary, 3rd, 4th, as the underlying cause. Oh, diabetes is literally caused by not taking care of yourself? I should let me sister know that when her pancreas just went up and stopped producing insulin when she was 4. I'm sure she'll be relieved to hear of this from such a practiced expert. You should really let Sick Kids know about such a earth shattering medical breakthrough, I'm sure all those kids will be happy to hear it too.

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      Om, nomnomnom...
  7. sickle cell and malaria by zoefff · · Score: 2

    Before someone rushes of to cure sickle-cell disease, there is a reason it exists: better protection against malaria

    1. Re:sickle cell and malaria by jblues · · Score: 2

      It is now particularly common in the middle-east despite that mosquitos aren't much of a problem there. People evolved the trait then brought it with them to where it was no longer especially useful. A very effective prophylaxis against Malaria in East Africa was to encourage people to sleep with a mosquito net.

      --
      If it acquires resources on instantiation like a duck, then its a shared_ptr<Duck>
    2. Re:sickle cell and malaria by Anonymous+Cow+Ward · · Score: 1

      None of the cures for sickle cell that are in the works right now will be heritable, and some of the sickling hemoglobin will still be produced. And frankly, for an individual, it's better to not have sickle cell and be less resistant to malaria, even if heterozygotes have some protection.

      --
      Examine even your most deeply held beliefs. Nobody is always right.
  8. Re:Huh? by drinkypoo · · Score: 1

    However, there is a group of low-income citizens that have serious issues paying the heavy monthly insurance fee. And instead of helping them out somehow with a low-income emergency fund, our gov in all there wisdom decided to penalize them with an extra 50% monthly fine _until all late payments are solved_ (!).
    The USA values individual freedom a bit higher than we do. Hence, i do understand the critisism towards obliged insurances like obamacare.

    Because the USA is a republic of states, and obamacare implementation details were left up to them, the details vary significantly here. In California at this point, if you have less than $1,300-ish/mo in income as a single individual, you get ACA coverage provided by Medi-Cal and in general get no-cost health care with full prescription coverage. That's a pretty paltry amount of money, but if you live in one of the dinkier and dirtier counties around the periphery, you can live on a job like that so long as you don't live alone.

    --
    "You're right," Fisheye says. "I should have set it on 'whip' or 'chop.'"
  9. Insulin history by XXongo · · Score: 2

    Almost a century ago, scientists discovered insulin, and found it could be used to treat diabetes. They sold that patented idea for $1 as a goodwill gesture because they knew their discovery could save millions of lives.

    Fact-checking this one, and it turns out to be true. The researchers who discovered insulin, Banting, Collip and Best, did sell the patent for one dollar.
    https://www.healio.com/hematology-oncology/news/print/hemonc-today/%7Bb3848683-e962-43ac-b23b-5b2c10f711a8%7D/frederick-banting-discovered-insulin-in-1921

  10. Huh? by argStyopa · · Score: 1

    I'm confused?

    I was told from 2000-2007 that Bush II's ban on stem cell research was going to "forever put the US behind in medical technology" and genetic science?

    Has "forever" passed already? I'm older than I thought.

    --
    -Styopa
  11. Chill out by sjbe · · Score: 2

    When you are trying to be a righteous asshole, the first step is to get your facts fucking right.

    Who peed in your cereal this morning? The only thing I'm being righteous about is the stupid system we have for paying for drugs in the US and if you aren't pissed off about that there is something wrong with you.

    Most diabetics dont use insulin. Full fucking stop. Dipshit.

    First off before you turn into a green rage monster please notice that I DID NOT SAY INSULIN even once. You are trying to put words in my mouth I didn't say. Second, we have millions of people in the US with diabetes and that's a huge market for drugs no matter how you slice it. Well past the market size where economies of scale are realized. Any drug or treatment used by more than a million people (and insulin falls into that category fyi) should not be hugely expensive to make and can be sold for reasonable prices under a rational health care system.

    Seriously dude, try decaf.

  12. Read before responding by sjbe · · Score: 1

    That 10% number is for both Type 1 and Type 2 (and presumably Type 3 included with the Type 1). Type 1 diabetes (the kind you need insulin for) amounts to about 1.25 million people, so rather less than 0.5%....

    Please note that I never wrote the word "insulin" even once. So I'm not sure who you think you are responding to with your pedantry but it wasn't anything I wrote.

    1. Re:Read before responding by CrimsonAvenger · · Score: 1

      When one considers that Type 2 diabetes requires nothing more than controlling one's carb intake, and no other meds, it's pretty hard to see a connection between 30 million Type 2 diabetics and a need for meds that should be (more or less automatically) met by the manufacturers of meds....

      --

      "I do not agree with what you say, but I will defend to the death your right to say it"
  13. Price of a Body Part by Kamiza+Ikioi · · Score: 1

    Well, $1 million for each eye... maybe we'll advance research enough to finally know how much costs an arm and a leg!

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    I8-D
  14. Re:Huh? by Freischutz · · Score: 1

    Why do you hate freedom of choice? Instead of being condemned to have health care, you can freely choose between having health insurance and eating.

    I don't hate freedom, it is you wing-nuts who get off on demonising the concept of insurance. Another thing is that even though I live in a country that has implemented many of the blasphemous ideas of social democracy I can afford to BOTH pay for health insurance AND eat. Now ask yourself: why do people in your country have to choose between health insurance and eating? ... and this even though you have such a wonderful for maximum possible profit healthcare system and I have a single payer healthcare system conceived of in the socialist bowes of hell by lucifer himself. And why do such systems all over Europe costs significantly less to run per patient than your for maximum possible profit system does?

  15. Re: A new era of Greed by ArmoredDragon · · Score: 1

    You ought to be grateful that the thing exists at all to begin with. Billions of dollars were spent just to get here, with many, many big failures along the way (including some people who have died during the experimental phases.)

    Even if we exclude all of that, you have to think about what it takes just to execute this treatment on just one person: They have to customize the vector's (a virus) genome to fit the patients genome, and then they have to produce millions of them. If it's not done right, it can kill the patient.

    Besides, people are speculating that this will be the price based on what it takes to do this; they haven't actually announced any price.

  16. Re:A new era of Greed by Swave+An+deBwoner · · Score: 1

    Greed indeed.

    Normally I'd think that setting a price on some good or service would factor in things like the cost of producing it, reasonable return to the provider, and liability in case things go wrong. But here we see that the price depends on how much the customer stands to gain from using the product. So it's a case of controlling the supply to raise the price. That is greed.

    http://www.sciencealert.com/first-fda-gene-therapy-inherited-disease-childhood-blindness

    Spark Therapeutics chief executive Jeffrey Marrazzo has talked repeatedly about the challenge of setting a price for a treatment that is designed to be administered once but provide benefits over years or even a lifetime.

    He has said he was considering several factors in weighing the price, including the value of a patient being able to work because of improved vision and a reduced need for caregiving.

  17. Re:Huh? by Opportunist · · Score: 1

    You might want to have your sarcasm detector checked.... jeeeeeesh...

    --
    We used to have a Bill of Rights. Now, with the rights gone, all we have left is the bill.
  18. Re:A new era of Greed by Anonymous+Cow+Ward · · Score: 1

    This solution only works for a very particular kind of childhood blindness. It doesn't affect many people, which is one reason it has to be so expensive. All the R&D money that would normally have been recouped from tens of thousands of patients (or more) has to be recouped from a few hundred. It's hardly greed to not want to lose money on a breakthrough therapy, especially for a disease that had no treatments before this.

    --
    Examine even your most deeply held beliefs. Nobody is always right.
  19. Re:Correction by Anonymous+Cow+Ward · · Score: 1

    The total cost isn't likely to be much different in other places, but the proportion paid by the individual (or their family, since it's a degenerative disease and early treatment is best) will definitely vary by country.

    --
    Examine even your most deeply held beliefs. Nobody is always right.