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Medicine The Almighty Buck United States Science

FDA Approves First-Ever Gene Therapy For Inherited Form of Blindness (sciencealert.com) 58

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.
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FDA Approves First-Ever Gene Therapy For Inherited Form of Blindness

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  • "The approval signals a new era for gene therapy...likely to cost as much as $1 million for both eyes."

    When a solution that affects many is only affordable for the few, the only thing it signals is a new era of Greed.

    • by Anonymous Coward

      First steps are always expensive.

      • 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.

        • by Higaran ( 835598 )
          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.
          • by sjbe ( 173966 ) on Thursday December 21, 2017 @07:30AM (#55782227)

            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 [bloomberg.com] than almost anywhere else.

            • 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.
              • by geekmux ( 1040042 ) on Thursday December 21, 2017 @07:56AM (#55782289)

                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.

                • by Mashiki ( 184564 )

                  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 t

                  • 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 U

                    • by Mashiki ( 184564 )

                      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 b

                    • by IMightB ( 533307 )

                      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

                    • by IMightB ( 533307 )

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

                    • 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 att

                    • by Mashiki ( 184564 )

                      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/... [wikipedia.org]

                      Don't let the stupid hurt you.

                    • by Mashiki ( 184564 )

                      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 ver

                    • by Mashiki ( 184564 )

                      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 s

                • 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 [healio.com]

              • by XXongo ( 3986865 )

                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.

              • 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 m

            • "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%....

              • 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.

                • 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....
    • You could use the Tleilaxu Eyes model where the eyes are free but they're monetized in various ways which are a trade secret and probably not entirely in the users' best interests.

      A bit like Google do with Android.

    • 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 k

    • 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 [sciencealert.com]

      Spark Therapeutics chief executive Je

    • 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.
  • How many zombie movies have we had where gene splicing was the cause of a new breed of human that behaved identically to zombies?

  • Huh? (Score:1, Troll)

    by Freischutz ( 4776131 )

    Yet the products, should they reach patients, are likely to cost as much as $1 million for both eyes.

    Ah, the wonders and joys of a privatised helthcare systems run by Wall Street hyenas out to make the biggest possible profit off of your misery with the help of your elected repesentatives in Congress. You pay as you go and it is much cheaper for the individual citizen than the socialist horrors of a universal single payer healthcare system and it is so much more humane.

    • Re:Huh? (Score:4, Insightful)

      by Opportunist ( 166417 ) on Thursday December 21, 2017 @06:58AM (#55782157)

      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.

      • 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 syst

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

  • by zoefff ( 61970 ) on Thursday December 21, 2017 @08:27AM (#55782415)

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

    • by jblues ( 1703158 )

      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.

    • 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.
  • 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.

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

Nothing recedes like success. -- Walter Winchell

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