Autism/ADHD News stories, Technology, breakthroughs, treatments

New drug that may help reverse autism
March 14, 2013 at 11:47 AM

New drug that may help reverse autism is to be tested on children for the first time after successful clinical trials on mice

  • Drug called suramin is already used to treat sleeping sickness in Africa
  • Scientists in the U.S. have found it corrects autism-like symptoms in mice
  • A small clinical trial on children with the condition will be started this year

By Suzannah Hills


A drug that may reverse autism is to be tested on children with the condition for the first time, scientists have revealed.

Preliminary results show that the drug called suramin, which is already used to treat sleeping sickness in Africa, corrects autism-like symptoms in mice.

At the molecular level, it normalises faulty brain connections, cell-to-cell signalling, and metabolic effects thought to underlie the disorder.

Groundbreaking: Testing of the drug suramin on mice in America has found it can help to correct autism-like symptoms (stock image)

Groundbreaking: Testing of the drug suramin on mice in America has found it can help to correct autism-like symptoms (stock image)

The drug targets a cell messaging system that produces a metabolic response to stress.

According to a new theory, autism is strongly linked to this pathway, known as purinergic signalling.

Scientists in the U.S. found that the drug corrected 17 types of abnormality linked to autism in genetically modified mice, including social behaviour problems.


Autism is a wide ranging condition, mostly seen in boys, that affects a person's ability to socialise and communicate and can have a devastating lifelong impact. Around 600,000 children and adults in the UK are thought to have the disorder.

Professor Robert Naviaux, co-director of the Mitochondrial and Metabolic Disease Centre at the University of California in San Diego, said: 'Our theory suggests that autism happens because cells get stuck in a defensive metabolic mode and fail to talk to each other normally, which can interfere with brain development and function.

'We used a class of drugs that has been around for almost a century to treat other diseases to block the 'danger' signal in a mouse model, allowing cells to return to normal metabolism and restore cell communication.'

New hope: A small clinical trial on children could be carried out within the year at the Mitochondrial and Metabolic Disease Centre at the University of California in San Diego, pictured

New hope: A small clinical trial on children could be carried out within the year at the Mitochondrial and Metabolic Disease Centre at the University of California in San Diego, pictured

He added: 'Of course, correcting abnormalities in a mouse is a long way from a cure for humans. But we are encouraged enough to test this approach in a small clinical trial of children with autism spectrum disorder in the coming year.

'This trial is still in the early stages of development. We think this approach, called antipurinergic therapy or APT, offers a fresh and exciting new path that could lead to development of a new class of drugs to treat autism.'

The findings are published in the online journal Public Library of Science ONE.

Professor Naviaux and his team believe both genetic and environmental causes of autism can be traced to a sustained cell danger response linked to immunity and inflammation.

'When cells are exposed to classical forms of dangers such as a virus, infection or toxic environmental substance, a defence mechanism is activated,' said the professor.

'This results in changes to metabolism and gene expression (activity) and reduces the communication between neighbouring cells. Simply put, when cells stop talking to each other, children stop talking.'

Suramin is an inhibitor of purinergic signalling that has been used to treat African sleeping sickness since shortly after it was first synthesised in 1916.

The 'striking effectiveness' of the drug in mice could pave the way to a 'completely new class of anti-inflammatory drugs to treat autism and other disorders', said Professor Naviaux.

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  • saTX1234
    April 14, 2013 at 4:07 PM
    to be very honest i did not read the post/article. One word: mice. (rhetorical question) How is it possible that charateristics where found in mice?. That sounds very comical. Do they stem.?. Have self injurious behavior?. I couldn't take this serious.
  • NeneMama
    April 17, 2013 at 2:39 AM

    I think this is fascinating.  I can't believe that they would start testing it on children so soon though, but I don't really know anything about drug testing. 

    I agree that it seems funny to have autistic characteristics in mice :) 

  • Corinne770
    May 13, 2013 at 7:39 PM

    I don't think they are talking about autistic traits. I think they are talking about the brain connection in the mice with the communicating cells that talk to each other. How do they find this out, I don't know. Do they give the mouse an MRI of sort or what? I don't know anything about test either. My guess is that they will try monkys before humans. I know my family won't be the first to try this. I think something needs to be out there for a while. Look what Riddillin (sp?) did to kids overtime. It cause either shrinking in the brain or no growth. I can't remember exactly what was in my college book, but yea, it was in my book :/  All of this is so scary. 

  • Corinne770
    May 13, 2013 at 7:43 PM

    Thank you for posting this. I will be keeping watch to see where this goes. Lets hope and pray:)))))))

  • lancet98
    October 13, 2013 at 10:32 AM

    Mice do not behave like people.   But they have many of the same genes, for the same chemicals, same enzymes, proteins(protein being a very general type of chemical, not something you eat at Burger King), etc.   And in many cases, that substance (gene, enzyme, whatever) has the same function as in a human.

    THIS IS A DIFFERENT KIND OF RESEARCH.   THIS IS RESEARCH ON THE GENE ITSELF.   Mice and humans share many genes and the genes govern the same processes.   That is all we need to have, for THIS type of research.

    This is VERY VERY basic research, into the processes BEHIND the behavior.   So it is not necessary that the mice behave the same as the human.  

    Genes are not just inherited.   They are functioning constantly, telling the cells of our body what to do, what to make, sending messages.   Genes exist in every cell of the body and are at work all the time.

    In any disorder or illness, some of those processes are going wrong.  

    What a researcher can do, is find abnormalities in the function of the human body, and trace that down to a specific gene.   

    THEN they work on what's wrong with that gene.   Is it missing?   Is it malformed?  Were too many copies, or too few, made?  What's going wrong with that particular process?   Is the gene causing too much, or too little, of a speicific protein or enzyme to be made?  

    Then, perhaps someday a medication is designed that deals with that specific enzyme or protein.   Is too little or too much made?   Then the medication prevents that.  But unless we have this much, much more basic knowledge first, we can't create treatments, cures, etc.

    NOW....since so many genes in the human are identical in a mouse, and do the same function, the medication can be tested in the mouse.   

    What if a gene is missing in the human?   How to duplicate that in a mouse?   The gene can be 'knocked out' in the mouse.   Whole generations of mice can be bred with that gene 'knocked out'.

    Now research can go ahead on the mouse.  

    The EFFECT of the process that gene governs, may not be the same in the mouse.   His little tiny brain is going to be different from a human's.

    Mice don't get autism.   In a mouse, that same gene problem isn't going to cause autism.   It may cause a mouse to play less, or to run around more, or not socialize with other mice as much.

    We're still good.   We need to study the genes, and that's what the mouse research allows us to do.  He's got the genes, we need to study those genes.

    And yes, it's possible, that there is a group of genes that have to do with the repetitive motions of autism, that are in mice as well as humans - and yes it's possible that those genes affect mouse behavior.   So those genes may make mice wash their little faces repeatedly or skitter around more.

    The brain has a ton of function in it that basically its whole job, is that it says, 'Don't do that again'.   Very slight errors in those functions allow a person to 'do that again'..and again...and again.   That can lead to repetitive actions.   There are other 'pathways' to repetitive actions too.   That can be studied too.

    Or maybe that group of genes would have a somewhat different effect on the mouse.   He might simply play more, or run around more.

    It isn't necessary for the mouse to have autism or behave anything like a child with autism, to allow THIS kind of research.

    There are many other kinds of research, but for THIS research, having a 'mouse model' with even just ONE biological process in common, is fine. 

    The gene or genes are going to affect the mouse differently, than the human.

    The point is it's the same gene doing the same function - making the same protein, the same enzyme, whatever.  That allows us to study the process in mice.   It doesn't matter if mice don't get autism.

    Not for THIS type of research.

    The point of THIS type of research is to research the gene and what it does, and if there is anything that affects what that gene does, so we can develop a treatment some day. 

    People are making a lot of unwarranted conclusions based on the article, but the article is also kinda stupid, especially its title.   This isn't going to result in a complete cure for autism.   It may, however, help us understand what is going on in autism.   And we desperately need that. 

    But it is dealing only with one tiny part of autism, one process, one chemical.   And there are many processes and genes involved in autism. 

    Sure.   We could get incredibly lucky and this one tiny process could turn out to be really central to autism.  So I never say never, but yes, a cure from this is not likely.

    A treatment?   Yeah, that's possible.   Or not.   Since it's so important to understand the processes involved in autism, this type of research has to be done, whether it leads to any cure or treatment or not.

    Scientists have to focus on research that leads to better understanding.   Their business is inquiry.   That's what they do.

    That understanding might turn out to be a treatment or even a cure, or it might not.   They still have to do it, regardless.   This kind of really, extremely basic stuff, this is what leads to the treatments that help.   So it has to be done.  

    But with  curing autism entirely, you'd have to be curing it in utero in the first few weeks of life as that's when it starts.  

    Quoting saTX1234:

    to be very honest i did not read the post/article. One word: mice. (rhetorical question) How is it possible that charateristics where found in mice?. That sounds very comical. Do they stem.?. Have self injurious behavior?. I couldn't take this serious.



  • lancet98
    October 13, 2013 at 11:02 AM

    I did a little research on this research, LOL.

    Here is a quote from the lead:    "I am very concerned that our work is being oversold," he wrote in an email.

    Here's the situation.   There is some preliminary research that raises an interesting question: "COULD autism start due to some subtle problem in mitochondria, a problem we don't yet know about or have the ability to measure or test for?"

    It's not at all clear how many autistic children might have defects in mitochondria, or if that would be a result or cause of autism. 

    But there is new interest in mitochondrial diseases and there are some researchers who wonder if this might have something to do with autism.

    The medication mentioned, has an effect on all cells of the body.   It alters the cell surface so the African Sleeping Sickness cannot get into the cell.

    The question is, could this medication help cells fight any mitochondrial problems?  

    The question would not have come up except that the medication seemed to help mice with the 'mouse model' of autism.   No, they don't have autism, but some of the gene changes are similar.   That's all we need for this type of research.

    And there is more.   There is one type of mitochondrial disorder, where the kids look like any other autistic kids.  In fact they're indistinguishable, by behavior, symptoms.  

     That got some people thinking.   Could mitochondrial abnormalities cause ALL cases of autism?  

    Now that kind of thinking can cause you to waste an awful lot of research time and money - you just can't assume that and always be right.   It doesn't always pan out.   But we have to ask the question.   With a disorder like autism that affects so many families so profoundly, there is a risk in NOT asking the question.  

    We don't know, but we sure would LIKE to know!!!   Either a yes or no answer would be very, very important to have in hand.

    How can mice help>  Think of it this way.   Gene A makes chemical B.   In the human, chemical B causes behavior C.   In the mouse, you have the same Gene A, and chemical B being made, but it causes behavior D.   But we're still fine, we are ONLY studying the part that goes from A to B in this type of research.

    That's what I mean by VERY basic research.  

    SO.   DO mitochondrial defects cause any cases of autism?   Do they cause ALL of autism?   Could this medication prevent or reduce autism?

    We do not know.   We don't know ANY of that, in fact.  

    But the researchers of the world who keep plugging away at this til we CAN answer those questions.   And it won't be one study, but many, to answer all these many questions.

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