Comments on: Human Clinical Trials Underway for Fragile X Drug

By: clinical drug trials

clinical drug trials — Thu, 05 Mar 2009 12:08:24 +0000

The xperimental drugs that are said to “correct” symptoms of Fragile X, Rett Syndrome, and tuberous sclerosis complex are now in early-stage human trial.the MIT Technology Review reports more informative for Drugs canditate…..

By: Top Posts from the Past Two Weeks

Top Posts from the Past Two Weeks — Sun, 14 Dec 2008 02:37:15 +0000

[...] Human Clinical Trials Underway for Fragile X Drug Experimental drugs that are said to “correct” symptoms of Fragile X, Rett Syndrome, and tuberous sclerosis complex are now in early-stage human trials, the MIT Technology Review reports. [...]

By: Tyler

Tyler — Thu, 11 Dec 2008 19:51:02 +0000

>> I did not say that the drug if successful would only help those with Fragile X, I said their was no guarantee that it would be effective on other forms of autism.

Oops, sorry. I missed the word “likely”, as in “isn’t likely”. At least I’d hope someone would have been looking at this specific biochemical location in the general autistic population (and this drug seems very specific in it’s action) and finding any sort of connection would be BIG NEWS to say the least. The hope is really better placed in looking for things like this and the tech being somewhat transferable.

By: Phil Schwarz

Phil Schwarz — Thu, 11 Dec 2008 04:09:07 +0000

Roger has fallen prey to a stupid but pernicious strawman leveled at pro-neurodiversity autistic self-advocates and their allies.

If an intervention such as this mitigates impairments in people with Fragile X, that’s good, and saying so is not at all at odds with what pro-neurodiversity advocacy is all about.

The Occam’s razor here is whether an intervention mitigates true impairments, or simply makes people “look less autistic”. The former is truly worthwhile; time and energy invested in the latter are better spent making society more accepting of the aspects of autism that are not intrinsically disabling.

By: mayfly

mayfly — Wed, 10 Dec 2008 23:37:43 +0000

I did not say that the drug if successful would only help those with Fragile X, I said their was no guarantee that it would be effective on other forms of autism.

A year, perhaps more, there were stories about inhibiting an enzyme known as protein 21-activated kinease, and its effect on Fragile X during prenatal development

By: Tyler

Tyler — Wed, 10 Dec 2008 16:33:04 +0000

Here’s a local newspaper story that gives a rough overview of that U of A work.

http://www.canada.com/edmontonjournal/story.html?id=6a3f23da-9187-427b-9252-506f2fcddf8d

By: Tyler

Tyler — Wed, 10 Dec 2008 16:26:17 +0000

Has anyone solidly made a connection between the protein problems within Fragile-X and any other cases on the spectrum that are not Fragile-X? Fragile-X is a 1 in 2000-4000 range occurrence and even then not all of them have symptoms that fit the ASD definitions.

I think mayfly is dead on in that this isn’t a drug that can be applied to autism in general, if there will ever be any that can. But it does show where the state of the art is in biology were we can bring this level of precision in influence.

Incidentally there is another drug somewhat like this being worked on at the University of Edmonton, but not nearly far along, that’s been effective at recovering long term memory in fruit flies after Fragile-X protein physiology was induced in them. http://www.nature.com/neuro/journal/v11/n10/suppinfo/nn.2175_S1.html I don’t have a subscription there but I gather that in that case they are concerned with stopping excess protein production. So it seems to be something different than the MIT work.

P.S. Note that, if I understand it correctly, there is an implication in what they are doing that if they could figure out a [safe] way to dose the fetus ongoing they could counter-act some of the Fragile-X effects from very near the beginning. But that’d be quite the feat I’d think. I doubt you could do it via the mother’s blood system, and poking around the womb at all is a very risky business. Just getting a genetic sample from the amniotic fluid is risky.

By: siliconmom

siliconmom — Tue, 09 Dec 2008 23:19:42 +0000

Hmmm…I was about to say I’d rather someone give their child something like this than subject them to chelation or other types of therapies that are claimed to “cure” autism but I’m not sure I actually would. Mayfly makes a good point and aside from the side effects this medication may cause I’m not sure it would be good to encourage people to spend even more money on a medication that might not work on their child at all.

By: mayfly

mayfly — Tue, 09 Dec 2008 23:12:20 +0000

The drug shows remarkable promise for those with Fragile X. Autism has myriad of etiologies. It is an umbrella term. If the drug proves successful with Fragile X, and I say “God speed.”,
there is no guarantee it will do the same for autism in general.

One can but hope.

By: Rose

Rose — Tue, 09 Dec 2008 22:07:50 +0000

This could be helpful to some people with autism. It is “forward looking”, but then so was insulin at one time. Like with diabetes, PKU, Wilson’s Disease…it could have profound implications.