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Autism drug to be tested on children

(50 Posts)
CFSKate Thu 14-Mar-13 10:38:00

"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.

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.'
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.'"

sickofsocalledexperts Thu 14-Mar-13 15:55:47

I think the reason why this study gives me hope is that I have always wondered whether the damage in my son's brain is to the hard drive (iykwim) and therefore incorrectable, or whether the basic structure of the brain is ok but it is how the different bits connect, the synapses I think they are called. I am no scientist but this study seems to be pointing to some of the autism features being caused by faulty connections, or the way cells in the brain "talk to each other" .

That is what I found hopeful, though of course we are a long way off yet, as its only a first clinical trial.

Also, it is a new use for a drug that has been safely in use for decades.

Maybe I am just an optimist!

CFSKate Thu 14-Mar-13 15:58:33

ISTR one mouse model for autism involves epilepsy medicine being given to the mother mouse, and the baby mice get autism. I don't know if this study used that model though.

Here's an earlier study that used that model, they knew VPA gave babies autism so they tried giving it to rats.

"For the animal work they used an autism model called the VPA rat. This model is based on dozens of case studies of children whose mothers took the
anticonvulsant and mood-stabilising drug valproic acid (VPA) while pregnant. A frighteningly high proportion of these children ended up with some form of autism - around 10 per cent, compared with some 0.08 per cent of the general population.
In the mid-1990s, researchers working on the adverse effects of VPA tried
exposing rat fetuses to the drug and found that giving it on the 12th day of
gestation - equivalent to the early part of the first trimester in humans - caused major damage to the rats' developing brainstem. This has far-reaching effects on later brain development and results in socially withdrawn behaviour that looks eerily similar to humans with autism. The VPA rat is now an established animal model of autism."

willowthecat Thu 14-Mar-13 16:01:32

It sounds very interesting - I hope we can be kept updated about progress and to see if there is any chance of it reaching us here in the UK ? I wouldn't want to take any unnecessary risks but for my ds any improvement in functioning would significantly increase his chance of living a better quality of life later on when we're gone and not able to protect him - still maybe is just a daydream.

LimboLil Thu 14-Mar-13 16:07:21

Can't they find a cure for the NT population to be a bit bloody nicer about autism?!! My DS is a big source of worry for me but I find him much more interesting than most kids his age! Mind boggling images of autistic mice!!

sickofsocalledexperts Thu 14-Mar-13 16:08:26

I think maybe there are many years between clinical tests and us actually getting the drug. But if it works, I wouldn't hesitate. It is unthinkable for me to say I wouldn't change him - he isn't just a bit different, he s limited in almost every way possible by his autism. I would leap on a drug for autism, just as I would if his life were seriously affected by a medical issue.

LimboLil Thu 14-Mar-13 16:10:56

Is anyone else sitting here wondering how long before some well meaning berk friend/relatives sends me the clip from the DM?! Sorry, feeling touchy today!

CFSKate Sun 17-Mar-13 11:16:39

There seem to be various types of autistic mice models.

1. The one I mentioned above, the VPA epilepsy medicine given to the mother mouse.

2. The triggered immune response in the mother mouse.
"The first step in the work was establishing a mouse model that tied the autism-related behaviors together with immune changes. Several large epidemiological studies -- including one that involved tracking the medical history of every person born in Denmark between 1980 and 2005 -- have found a correlation between viral infection during the first trimester of a mother's pregnancy and a higher risk for autism spectrum disorder in her child. To model this in mice, the researchers injected pregnant mothers with a viral mimic that triggered the same type of immune response a viral infection would.

"In mice, this single insult to the mother translates into autism-related behavioral abnormalities and neuropathologies in the offspring," says Elaine Hsiao, a graduate student in Patterson's lab and lead author of the PNAS paper."

3 "Mice with a defective version of a single gene..CNTNAP2 "

If you search that sciencedaily website for autism mice gene it gets loads of results.

MerryCouthyMows Sun 17-Mar-13 14:41:58

Concerns me about the epilepsy drugs - most women with epilepsy now know that there is a high risk of their baby having Autism if they take sodium valproate during pregnancy - bit that's because it's a well established AED.

Thing is - there is also an increased risk of having a baby with Autism if you HAVE epilepsy - even if you aren't using any medication - not as high as if you take Epilim , but significantly increased compared to the general population.

My epilepsy was undxd / misdxd as depression (!) till I was 22 - by which point I already HAD 3 DC's. All are somewhere on the spectrum.

I can't see how an I he croon or something like that can change something that may well have been caused in the first trimester of my pregnancies, and also has a genetic link because even my Dbro has been dxd with Aspergers, and the father of my youngest two DC's was ALSO dxd with Autism when he was 12yo.

How can an injection solve something that they were born with? My DC's all had obvious differences that were evident either from birth or within the first 3 months of their life.

The side effects also concern me - messing with the brainwaves?! My DD AND my DS2 have both already been tested for epilepsy with 'inconclusive' results for DS2 (but a watchful wait), And my DD has been dxd with 'mild epilepsy' that is unmedicated as her szs are currently only about one per year.

Their brain waves aren't entirely 'NT' already, I wouldn't want to mess with that!

And also, why can't SOCIETY adapt to our DC's, instead of us parents having to inject our DC's with god knows what in order to make them 'fit in'?!

saintlyjimjams Sun 17-Mar-13 14:46:02

interesting blog post mentioning this paper

couthy - autism is many things, so it may be that it wouldn't be suitable in your case iyswim. But might be suitable for some.

In ds1's case I'm not bothered about him fitting in but if a drug meant he could function then I think he would want it (at his current level of functioning he is expected to need lifelong 14 hour 1:1 care - he needs 2:1 out and about at the moment). My main concern would be potential side effects.

saintlyjimjams Sun 17-Mar-13 14:46:36

24 hour - not 14 confused blush

CFSKate Sun 17-Mar-13 14:48:17

willowthecat - thanks for that original article link, it says they used the maternal immune activation mouse model.
So it says this is based on "purinergic signaling", it says "Hyperpurinergia", and "antipurinergic therapy" fixes it so I guess that means the problem is too much signalling?

I'm trying to read the full article now, interestingly it says about the autistic mice "Females generally displayed fewer and milder abnormalities than males"

There seem to be a lot of women with ME (or other autoimmune) who have children with ASD. I know they have been discussing purinergic signalling in ME research as well, so if this drug works for autism I wonder if it might signpost towards some kind of help for us too.

CFSKate Sun 17-Mar-13 15:11:33

saintlyjimjams - thanks for the link. It links to this. It is discussing autistic mice created by activating the immune system of the mother mouse, and talking about the different effects depending on the breed of mouse. It seems the breed of mouse can affect how severe the autism is. It is about how genes and environment combine.

HotheadPaisan Sun 17-Mar-13 15:19:29

Message withdrawn at poster's request.

MareeyaDolores Sun 17-Mar-13 21:09:46

I nearly posted something scathing about mouse models, 'autism isn't one category, silly' and overexcited press releases. Having read the linked research paper though, I'm glad I didn't.

Mitochondria are clever, complex, generally-overlooked little organelles. The so-many-causes but such-similar-symptoms problem with doing ASD research might be overcome if this hypothesis holds.

And so multiple causes might be overcome with one treatment if the mitochondria really are the 'missing link'.

PipinJo Mon 18-Mar-13 00:34:06

Message withdrawn at poster's request.

MareeyaDolores Mon 18-Mar-13 20:45:20

ah, you need this guy

saintlyjimjams Mon 18-Mar-13 20:50:10

DS1 saw an NHS paediatric neurologist who was very open to him having mt dysfunction (and quite up to date with the American research). It helped that a guy I had been talking to in the States hooked me up with a neurologist at Cambridge who had worked in his lab. And he recommended seeing a local paediatric neurologist. But the paper trail of emails meant it was taken seriously.

He was willing to muscle biopsy - we decided not to yet as it's so invasive and there's no clear treatment protocol, so it would be an invasive procedure just for information really, but it's something he said he'd be happy to revisit in the future.

MareeyaDolores Mon 18-Mar-13 20:54:02

Almost impossible to do a proper double blind trial on bumetanide though
How can parents not notice the active drug, when the dc wee a lot more wink

<pedant alert>

zzzzz Mon 18-Mar-13 21:06:34

God it's such a seductive idea though isn't it?

Find the pill that allows the layers of dysfunction to fall away leaveing you with the child you have caught fleeting glimpses of all these years.

Toni27 Tue 19-Mar-13 11:06:50

I don't think the daily mail has anything against autism in fact as a parent of a child who has been recently diagnosed Im grateful to the newspaper for taking an interest in this condition that has affected my son so greatly it has struck me there are a lot of articles by the paper on the subject of autism maybe someone who works there has an autistic child and has an interest in the subject and I am always pleased to see that autism has made the press. Any greater understanding or even just talking about autism in the general public is surely a good thing? I love my son with all my heart and I would give anything to alleviate his autism and make life easier for him.

zzzzz Tue 19-Mar-13 11:58:42

It's not a paper I have ever read much, but certainly doesn't have he reputation for positive portrayal of sn or sn related topics, and no, I don't agree with the "any publicity is good publicity" concept.

CFSKate Fri 26-May-17 20:18:29

Four years later, the suramin study has been published.

CFSKate Sat 27-May-17 07:54:24

Parents point of view of the drug effects

Chasingmytail17 Sat 27-May-17 09:48:25

This is very interesting reading, I had not come across suramin before. I wonder how long it will take to do the further testing they need. The parents comments certainly seemed positive commenting on how much happier their child seemed, not just the difference in language etc

CFSKate Sat 27-May-17 13:02:38

Chasingmytail17 - the problem is money not time.
It says near the end here that if money was no problem it would take 5 years. "As a result of going into debt to fund the study, I've had to dramatically shrink my lab and slow our research progress greatly. We are currently working at only about 20% of our capacity if we actually had adequate funding."

QUESTION: What is the rate-limiting factor to progress right now? Where are the bottlenecks?

ANSWER: The rate-limiting factor is money. Lack of funding has slowed our research progress on the CDR and purinergic signaling in autism for the past nine years. We can’t do the next studies without new funding.

We have plans for five additional studies over the next five years to collect all the data the FDA will need to decide about the approval of suramin for autism. With adequate funding, culminating in a multicenter, phase III, registrational trial, these studies can be completed without further delay.

Usually, the multimillion dollar cost of new drug development is covered by the Big Pharma that will benefit from FDA approval. Unfortunately, since suramin is 100 years old, the usual patent laws don’t apply and the next clinical trials will require grass roots support from families and foundations and other approaches to raise the needed funding.

further up it also says "We did not have enough funding to do a larger study. And even with the funding we were able to raise, we had to go $500,000 in debt to complete the SAT-1 trial."

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