Controversial autism drug gets ambitious new trial

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firemonkey
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28 Nov 2016, 1:00 pm

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The makers of a drug designed to correct an enzyme shortage seen in some children with autism are testing the treatment in children with autism who do not have that deficiency. If the drug works in these children, it will come as a surprise to scientists who say the trial has no scientific basis.

The New York-based drug company Curemark developed the drug, known as CM-AT, for children with autism who have unusually low levels of chymotrypsin, an enzyme needed to break down dietary protein into amino acids the body cannot make on its own. The drug is a powdered blend of chymotrypsin and similar enzymes that parents sprinkle on their child’s food. (Curemark representatives declined to identify any of the other components of the drug).

According to the company’s scientific officers, the idea underlying the drug is this: Amino acids serve as building blocks for chemical messengers in the brain that some children with autism may lack. By releasing essential amino acids, CM-AT restores the necessary levels of these chemicals in the children’s brains.

Joan Fallon, the company’s chief executive officer, says the drug improved irritability in the participants of a trial completed in 2011. The trial included 182 children with autism who had low chymotrypsin levels. After 90 days of treatment, these children had become less socially withdrawn, Fallon says.

Outside experts have long been skeptical of the therapy’s premise. Their reason: Curemark has yet to publish any of the results.

https://spectrumnews.org/news/controver ... new-trial/


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AJisHere
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28 Nov 2016, 2:24 pm

I'd love for it to work, but this sounds like snake oil.


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29 Nov 2016, 9:45 pm

I would give anything to be less irritable, but yes not naming the other components makes me a bit sceptical.


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johnnyh
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29 Nov 2016, 9:56 pm

This drug sounds like snake oil. A true drug is maybe a decade away since they just cracked some of the autistic epigenome. ADHD medication often does help, at least it does for me. Adderall doesn't give me meltdowns or screaming fits, no no no, the opposite, it can make me a bit too chill that i don't look concerned enough on the outside.


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30 Nov 2016, 11:45 am

johnnyh wrote:
......no no no, the opposite, it can make me a bit too chill that i don't look concerned enough on the outside.
I actually got yelled at by a boss once for 'not expressing enough concern for a job in front of the customer."

Interestingly, the customer told me she was glad I showed up because they were freaking her out by not being calm. She actually thanked me for being calm.

Oddly, I think that's one of my Aspie superpowers. Calm when everybody else is freaking out because I go straight into problem solving mode. But, the flipside is that little things can send me into panic. Especially if it's a sensory issue that affects me only, but is nothing to the others I am with.

As far as the drug trial goes, it may help in one very, very specific area as they even admit the theory is to try and correct a situation that is measured in a small percentage of Autistic children. But, no real evidence that it is a cause in the rest of the Autistic population.

It also seems to be oppositional to other studies that point to an over abundance of connective tissue and neuro transmission, even in lower functioning autistic people.


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