Comments on: Two clinical trials for autism

By: Ashritha infoalcpl@gmail.com

Ashritha infoalcpl@gmail.com — Fri, 29 Oct 2010 09:40:02 +0000

My colleagues and I have just concluded one of India’s most in-depth studies on “Clinical Trials in India”. We have looked at various areas such as: – Market Trends – Growth Drivers – Regulatory Bodies and Framework – Major Players.
-Etc.
We interviewed over 200 individuals and firms to collect the data in what we believe is one of the most detailed study on the subject in India. If you are interested in a copy, you may email me at infoalcpl@gmail.com. It is a paid report.

Ashritha
infoalcpl@gmail.com

By: Carlos Useche

Carlos Useche — Sat, 20 Mar 2010 17:07:49 +0000

Rose, Rapamycin enter because we are talking about autism drugs.

http://www.sciencedaily.com/re.....151333.htm

By: Rose

Rose — Sat, 20 Mar 2010 13:21:22 +0000

Tuberous Sclerosis, yes. It’s a possibility for my son, but somewhat doubtful. It seems the benign tumours affect the drainage of cerebral spinal fluid (http://neuroanimations.com/Hyd.....truct.html ) a notion I just became aware of today via this study. ( http://www.ncbi.nlm.nih.gov/pubmed/16453317 )

I am not a scientist (duh), but how did rampamycin enter the discussion?

By: dr treg

dr treg — Fri, 19 Mar 2010 21:58:10 +0000

Rapamycin or sirolimus is
1. An anti-fungal treatment.
2. An immuno-suppressant.
http://en.wikipedia.org/wiki/Sirolimus
which suggests that the immunogenic response is important in autism.

Interestingly rapamycin increases the immuno-suppressive regulatory T cells or TREGS in diabetes, another auto-immune disease.
http://www.jimmunol.org/cgi/co.....77/12/8338

By: daedalus2u

daedalus2u — Fri, 19 Mar 2010 13:49:26 +0000

Interesting, tetrahydrobiopterin is an essential cofactor for nitric oxide synthase. NOS can produce either NO or superoxide, or both. Superoxide destroys NO forming peroxynitrite which oxidizes a zinc-thiol couple in NOS causing it to make only superoxide. Critical factors in this regulation are the level of tetrahydrobiopterin and the ratio of the tetrahydro to the dihydro.

In the short term, supplementation with tetrahydrobiopterin would probably increase NO levels. Tetrahydrobiopterin is synthesized by the body, so its levels are regulated. It is also consumed and recycled. I suspect (but this is just on a casual looking at the tetrahydrobiopterin literature) that any change in NO levels would be temporary and that the body would adjust itself to get back to where it was before the tetrahydrobiopterin was started. There might be temporary resolution of some autism symptoms which would then come back. The time constant for NO changes is much shorter than for changes in the level of phenylalanine, so the positive experience with PKU might not translate into a positive effect with autism.

Rapamycin is a compound that binds to mammalian target of rapamycin, mTOR. This is a major major control pathway that regulates autophagy and other stress responses, particularly nutrient stress. A number of the gene targets found in tuberous sclerosis are in the mTOR pathway.

By: farmwifetwo

farmwifetwo — Fri, 19 Mar 2010 12:57:14 +0000

I’m with the comment about picking kids that have gastro issues.

Since I completed my own mini-study at home… The one with gastro issues definately improved on dietary changes. The one without the issues did not.

Makes sense… so if you were using a small sampling… did you make a point of using those you were certain were going to prove the study failed?? Or did you pick half/half?? Did you make a comparison btwn the 2 groups??

I need more than a handful of kids, and no data, to convince me it did nothing…

By: Science Mom

Science Mom — Fri, 19 Mar 2010 02:28:07 +0000

pD, They didn’t specifically enroll children with GI issues and only 19% of the total study group were on special diets.

By: passionlessDrone

passionlessDrone — Fri, 19 Mar 2010 01:52:38 +0000

Hi Sullivan –
Very nicely done.

I saw the enzyme paper hit a week or so ago. Do you (or anyone) know if they used random kids with autism off the street, or kids with autism and gastro issues? The problem with doing this study ‘right’, to my mind, is finding a subset of kids with gastro issues and autism whose parents haven’t already tried the diet and/or enzymes to try to get their child feeling well.

As far as Sapropterin I don’t know enough to even comment.

– pD

By: Sullivan

Sullivan — Fri, 19 Mar 2010 00:41:09 +0000

Prometheus,

I’m pretty sure there are two studies. One by Frye and one from Japan. Both were small and both were inconclusive.

By: Carlos Useche

Carlos Useche — Fri, 19 Mar 2010 00:33:31 +0000

There are hope maybe in these drugs

1) STX209 (arbaclofen) is a selective gamma-amino butyric acid type B (GABA-B) receptor agonist.
http://www.clinicaltrials.gov/.....038;rank=1

and

2) Rapamycin

Do you have some information about that?