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

Posted by BeauHD on from the first-of-its-kind dept.

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.

9 of 58 comments (clear)

  1. 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.
  2. 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 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.

    3. 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.

  3. 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>
  4. 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

  5. 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.