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Autism drug fails in late-stage trials

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ASPartOfMe
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09 Sep 2021, 8:53 am

Biopharmadive

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French pharma Servier and biotech partner Neurochlore have agreed to end early a pair of late-stage studies of an experimental medicine for autism after finding no benefit to treatment, the companies said in a statement Wednesday.

The drugmakers joined forces in 2017 after a smaller study conducted by Neurochlore suggested bumetanide could improve symptoms in children with autism spectrum disorders. But initial results from Servier's two Phase 3 studies showed no significant difference between the drug and a placebo.

Neurochlore President Yehezkel Ben-Ari said his team will continue to explore whether bumetanide can help specific subgroups of people on the spectrum, noting in the Tuesday statement that it's likely no one treatment will work for all patients because autism spectrum disorders are so different.


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magz
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09 Sep 2021, 9:06 am

Bumetanide

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Bumetanide, sold under the trade name Bumex among others, is a medication used to treat swelling and high blood pressure.[1] This includes swelling as a result of heart failure, liver failure, or kidney problems.[1] It may work for swelling when other medications have not.[1] For high blood pressure it is not a preferred treatment.[1] It is taken by mouth, or by injection into a vein or muscle.[1] Effects generally begin within an hour and lasts for about six hours.[1]
How is anti-swelling drug supposed to cure autism is a mystery for me.


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09 Sep 2021, 9:08 am

magz wrote:
Bumetanide
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Bumetanide, sold under the trade name Bumex among others, is a medication used to treat swelling and high blood pressure.[1] This includes swelling as a result of heart failure, liver failure, or kidney problems.[1] It may work for swelling when other medications have not.[1] For high blood pressure it is not a preferred treatment.[1] It is taken by mouth, or by injection into a vein or muscle.[1] Effects generally begin within an hour and lasts for about six hours.[1]
How is anti-swelling drug supposed to cure autism is a mystery for me.
"Improve Symptoms" does not mean "Cure".



magz
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09 Sep 2021, 9:12 am

True.
Still, how an anti swelling drug should do anything about social and sensory processing?
The clinical trials say, it doesn't.


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09 Sep 2021, 9:13 am

magz wrote:
True.  Still, how an anti swelling drug should do anything about social and sensory processing?
Maybe someone noticed some side-effects in a few patients and said, "How odd ... I should check this out".

Science is punctuated more often by "That's odd" than by "Eureka".



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09 Sep 2021, 9:25 am

magz wrote:
True.
Still, how an anti swelling drug should do anything about social and sensory processing?
The clinical trials say, it doesn't.

I'd guess maybe to do with the inflammatory based diseases that causes or exacerbate symptoms of AS.


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magz
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09 Sep 2021, 9:27 am

I have a suspicion that in autistic people, any health problem can exacerbate symptoms of ASD.


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09 Sep 2021, 9:43 am

A drug that would make me permanently drunk with none of the health effects would be a good cure for my autism that's for sure.

Medication for psychiatric problems irks me too. Anti-depressants for example are the at the top of my list. Even to this day the jury is out as to whether they work and they seemingly have no pick me effect. If you want to treat a mood disorder why not make a drug that actually effects the mood? If they want to treat poor social skills why not have a drug that attempts to imitate alcohol. It's not called a social lubricant for nothing.

Making such a drug would be difficult but it would be great to have before social events. At least for me.



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09 Sep 2021, 10:43 am

The future of autism treatments is to stop treating Autism as one condition rather as multiple autisms with multiple causes.

An example of this exists already with treating the subset of autistic kids with Cerebral folate receptor autoantibodies with leucovorin which has shown to improve outlook reducing symptoms from severe autism to high functional autism. In other words similar clusters, not perfect matches, and treat the autism by cluster.

https://www.researchgate.net/publicatio ... Prevention

I think Einstein once said "Insanity is doing the same thing over and over again and expecting different result"


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09 Sep 2021, 12:16 pm

Bumetanide clearly works for some kids as it wouldn’t have reached stage 3 in the first place.

An intelligent approach would be to study the subtype of autistic kids where it does work and create a marker to identify other kids of that subtype, where it has a high chance of working.

They already do a multiple drug approach with anti depressants / anxiety where one patent will have a bad reaction to one drug that works fine on another. The dr would simply try an alternative.


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magz
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09 Sep 2021, 12:33 pm

Or it could have been just a random fluctuation on a small sample.
Searching for sub-types, there is always risk of this:
Image
https://xkcd.com/882/


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