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Medicine Science

Century-Old Drug Reverses Signs of Autism In Mice 207

Posted by Soulskill
from the good-news-for-master-splinter dept.
sciencehabit writes: A single dose of a century-old drug has eliminated autism symptoms in adult mice with an experimental form of the disorder. Originally developed to treat African sleeping sickness, the compound, called suramin, quells a heightened stress response in neurons that researchers believe may underlie some traits of autism. The finding raises the hope that some hallmarks of the disorder may not be permanent, but could be correctable even in adulthood.
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Century-Old Drug Reverses Signs of Autism In Mice

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  • by i kan reed (749298) on Tuesday June 17, 2014 @02:55PM (#47256653) Homepage Journal

    Any idea what will be at this domain when its entire userbase is cured?

  • by mi (197448) <slashdot-2012@virtual-estates.net> on Tuesday June 17, 2014 @02:57PM (#47256683) Homepage

    Gaining a drug's approval by the Food and Drug Administration in the US — and similar government agencies in other countries — is a very expensive process. The expense is normally offset for by the patent(s) granted to the pharmaceutical company, that developed the drug, which make it an exclusive maker/seller of the medicine for decades.

    However, if the drug is long-known — and only needs an approval for new application — who will undertake to pay for the approval, if there is no way to patent it and the approval will allow all drug-makers (both domestic and foreign) to put their own versions on the market?

    • It shouldn't be patentable, but our patent system is a huge disaster. However, assuming this shows legitimate promise, one of those autism related charities should be able to front the bill.
      • by mi (197448)

        one of those autism related charities should be able to front the bill.

        Are there autism-related charities capable of putting forth the $150 million [fastcompany.com] typically required to pay for FDA's approval?

        And even if there are, I suspect, some of them might not want to to do that — under some legitimate-sounding reason — because it might eliminate their very reason for existing... Just as I would not trust "anti-poverty" politicians to do anything to really eliminate it — thus ending their political

        • Ignoring the money raised through walks and such, Autism Speaks noted that a bill with $230 million for autism research was passed a few years back, and Big Pharma would probably consider funding it worthwhile just to shut Jenny Mccarthy up, plus the great PR this would have.
          • by mi (197448)

            Autism Speaks noted that a bill with $230 million for autism research was passed a few years back

            The bulk — if not all — of that is already committed, no doubt.

            Big Pharma would probably consider funding it worthwhile just to shut Jenny Mccarthy up, plus the great PR this would have.

            That may be...

            • The bulk — if not all — of that is already committed, no doubt

              It probably is, but bills like that are pretty common. Given the number of people on the Autistic spectrum, it's not going to be hard to get that amount of money if this drug shows promise. NIH and similar sources drop about half the money already, and about half of the drugs are low priority me too drugs. So, all we need is an impassioned plea form the wife of a Senator with an autistic child and the grant for this can be fast-tra

          • Does big pharma hate Jenny McCarthy?

            She's giving them more customers...

            • Anyone in their right mind hates Jenny McCarthy for convincing parents to skip vaccinations over [scary voice] Autism and toxins [/scary voice]. (Botox is perfectly fine, though.)

              Since I have a child with Autism (Asperger's Syndrome so he's very high functioning), I double-hate her not only for her insinuation that a child is better off dead than Autistic, but for her claims of curing her son's Autism with a Gluten free diet (or whatever other nonsense she's advocating right now). Either her son didn't ha

              • Anyone in their right mind hates Jenny McCarthy for convincing parents to skip vaccinations

                Unless you're selling alternative treatments to the sicknesses those vaccinations cause.

              • by russotto (537200)

                Imagine not being able to communicate your fears, frustrations, desires, etc to anybody... ever. This drug might offer a path to reduce their symptoms to the point that they could communicate and possibly function in neurotypical society.

                Joke's on them. Once they have the ability, they'll find out that no one cares about their fears, frustrations, or desires anyway.

            • by Ol Olsoc (1175323)

              Does big pharma hate Jenny McCarthy?

              She's giving them more customers...

              Does Big Pharma make embalming fluid?

    • by larwe (858929)
      Off-label use of a drug currently approved for a given clinical use is very easy to achieve. However, looking at the side effects of this pharmafossil, it's looking like the side effects are worse than the condition it is being proposed to treat. I can't see an adult consenting to take it. Parents could force their children to be dosed with it, I guess.
      • Risking a 50% chance of toasted adrenals is a bit much to ask to treat a non-life-threatening condition.

        • by medv4380 (1604309)
          Given that the Adrenal Cortex is responsible for the stress response it probably works to combat autism stress by slightly damaging the gland responsible for over stressing. And only a small portion of that 50% actually need any life long treatment for the damage which does exist. So your "toasted" adrenals is a bit of an overstatement. Finally, Autism is pretty much life threatening in that they have much shorter life expectancy, and a far lower quality of life.
    • by Thagg (9904)

      Yes, one can often patent a drug for a new purpose. You probably can't charge $1,000/dose for a repurposing, but it can be done.

      The absolutely shocking prevalence of autism today (currently estimated at 1 in 68 births, probably 1 in 40 boys) will make any drug that has a good effect profitable.

      As the parent of an autistic teenager, I'm hoping for the best. It does appear that, like Tolstoy said, all autistic kids are autistic in their own way; so I'm not holding out a lot of hope. Some, though...

    • Re: (Score:3, Interesting)

      by medv4380 (1604309)
      The drug is still in use, and there is nothing preventing a doctor from prescribing it for an off label use.
    • by godel_56 (1287256)

      Gaining a drug's approval by the Food and Drug Administration in the US — and similar government agencies in other countries — is a very expensive process. The expense is normally offset for by the patent(s) granted to the pharmaceutical company, that developed the drug, which make it an exclusive maker/seller of the medicine for decades.

      However, if the drug is long-known — and only needs an approval for new application — who will undertake to pay for the approval, if there is no way to patent it and the approval will allow all drug-makers (both domestic and foreign) to put their own versions on the market?

      Or, the parents can just take their kids to Mexico for a week.

  • by jellomizer (103300) on Tuesday June 17, 2014 @02:58PM (#47256697)

    How about people?

    • I'm thinking we'll find that out really soon, as desperate parents buy the drug on the black market, and try it out on their kids.

      • No need to go for the black market, it's already widely available.

        • More of a gray market, although I don't think it's available without a prescription, and it's a bit hard to fake parasites (two of the primary uses of this drug)
      • I find it funny with the vaccine nonsense, and your comment. It makes it seem that having autism is worse then death. Now there are many degrees of autism (Hence why it is the autism spectrum)

        Most people with Autism will fall on the more mild areas, chances are with some different education the child will grow up to become a productive member of society. Even people with extreme cases are not completely out of it.

        • I'm the parent of a child with Autism (albeit High Functioning Autism/Asperger's Syndrome) and know plenty of parents of children with more severe Autism. None of us would rather our children be dead (of vaccine preventable illness or anything else) than have Autism.

    • Good news for them too, autistic mice can really be a pest.

    • Flowers for Algernon.
  • Godsend (Score:5, Insightful)

    by jklovanc (1603149) on Tuesday June 17, 2014 @03:01PM (#47256729)

    As someone with Aspergers Syndrome this would be a godsend for me. There are many times where my adrenaline level is far above what is needed for the situation. It is extremely frustrating when most of my body is in full fight/flight response and there is a small voice in the back of my head saying "chill out dude, it's not that important". The problem is that the adrenal response usually overrides the cognitive response and bad things happen.

    I just hope it does not impact the good things about autism such as the heightened ability to find and keep track of details.

    • There are days when I'm thankful that I'm an Aspie - like when my (neurotypical) wife is fretting over some body language that other people exhibited that I was completely blind to - and days when I'm not. In the case of the latter, when my Aspie fixations clash with my son's Aspie fixations, it can get quite stressful in our house. We feed off each other not doing what we're fixated on doing and everything quickly spirals out of control with my wife caught in the middle.

      Of course, like you said, I wouldn

    • by HiThere (15173)

      There's a good chance you are correct, but don't wrap your life around it. This is evidence based on mice, and people are significantly different. Many things that work on mice don't work on people.

      Also, it's not certain that it would have precisely the effect that you are hoping for. It *might*.

      That said, check out the side effects of suramin, and *if* they aren't too bad you might see if you can get a doctor to prescribe some for "off label" use. Your insurance probably won't pay, but the patents have

    • by Atrox666 (957601)

      I don't know if I'd take this drug. I'm over 40 and have learned to manage the downsides. I've also learned to use the super powers associated with mild cases of ASD.
      I've had to learn to override the adrenal response which gives me the ability to act when most people just freeze up(e.g. artillery strike). I'm always having a stress reaction when I'm outside my own head so a little more doesn't really matter that much. I doubt it would fix my voice modulation (flat affect)and other mannerisms that have been

  • provided it works on people (which is a big if, given that mice can't be autistic; they can only either exhibit behavior that when displayed in a human would be considered autistic, or otherwise have brain structures similar to those of people with autism. Also keep in mind that neural correlates of anything, though, are still rather tricky in contemporary neuroscience), I would take it if it alleviated the symptoms I experience negatively. If it would reduce the stress and anxiety I experience simply being

    • by radtea (464814) on Tuesday June 17, 2014 @04:24PM (#47257587)

      From TFA: "Second, suramin is a poor drug choice for chronic use because of potentially toxic side effects that can occur with prolonged treatment."

      And from the Wikipedia page on the drug (http://en.wikipedia.org/wiki/Suramin#Adverse_reactions):

      The most frequent adverse reactions are nausea and vomiting. About 90% of patients will get an urticarial rash that disappears in a few days without needing to stop treatment. There is a greater than 50% chance of adrenal cortical damage, but only a smaller proportion will require lifelong corticosteroid replacement. It is common for patients to get a tingling or crawling sensation of the skin with suramin. Suramin will cause clouding of the urine which is harmless: patients should be warned of this to avoid them becoming alarmed.

      Kidney damage and exfoliative dermatitis occur less commonly.

      Suramin has been applied clinically to HIV/AIDS patients resulting in a significant number of fatal occurrences and as a result the application of this molecule was abandoned for this condition.

      So while this is an important piece of work that identifies purine metabolism as a critical set of pathways related to ASD, it should be viewed primarily as a starting point for a more precisely targeted drug that will have the same effect on the pathways that matter without also messing up the ones that cause the side-effects.

  • How did this happen? Which idiot directly tested a century old drug without making a cosmetic change, or at least a name change to make it patentable? Guys like this never think of broader implications of their work. Narrowly focused on useless things like welfare of the people or some such thing. Must have contracted a very severe case of anthropophilia. They should have thought about how it would impact the boni of pharma executives and they should be constantly asking themselves how we could make this ye
  • the chill pill will actually be able to be taken.

  • by WormholeFiend (674934) on Tuesday June 17, 2014 @03:28PM (#47256999)

    in the form of a vaccine?

    • Or the does the vaccine itself cancel out the effects of autism vaccine? Would it even be worth getting?

    • by ignavus (213578)

      in the form of a vaccine?

      Vaccines cause neurotypicality - you heard it here first!

  • ... it is revealed the cure for Autism is pure, undiluted Thimerosal.

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