[disability scoop&new york times]Drugs for fragile x/autism

[AKAConlang]

They are being marketed as drugs for autism, but they are really for fragile X

From the disability scoop summary:

Fragile X is the most common form of inherited intellectual disability and many with the condition have autism as well. Accordingly, scientists believe that treatment for fragile X could yield significant progress in addressing autism and intellectual disability too.

Under the deal, Roche will get access to patents held by Seaside, a move that may give the company an edge over rival drug maker Novartis which is considered to be farther along in development of such medications.

Scientists at Roche told The New York Times that the partnership will “establish the biggest effort to date” to develop autism drugs. To read more click here.

From the new york times article

Competitors Form Partnership to Develop Autism Drugs
By ANDREW POLLACK
Published: June 19, 2012

Two of the front-runners in the race to develop drugs to treat mental retardation and autism are joining forces, hoping to save money and get to the market sooner.

A deal, expected to be announced on Tuesday, will pool the resources of Roche, the Swiss pharmaceutical giant, and Seaside Therapeutics, a private 30-employee company based in Cambridge, Mass.

“This deal will establish the biggest effort to date” in autism drugs, Luca Santarelli, head of neuroscience for Roche, said before the announcement. Financial terms are not being disclosed.

There is rising excitement that drugs might be able to relieve some of the behavioral problems associated with autism and in particular a cause of autism and mental retardation known as fragile X syndrome. About 100,000 Americans have fragile X syndrome.

Some parents of children being treated with new drugs in clinical trials have said they see positive changes in behavior.

etc

Still, Ms. Zorovic said that until the clinical trial comparing the drug to placebo was completed, it was difficult to say whether the effects were from the drugs or merely a result of parents’ perceptions.
The mechanism that has perhaps shown the most promise, at least in mice, is to damp signaling in the brain by blocking a receptor called mGluR5.

I crossposted this from AFF

[AKAConlang]

I know I haven't been around much, but I saw this article and I knew I had to post it here.

[Sophist]

Thanks, AKA. I wonder what the drug is. I know there've been promising animal models of Fragile X utilizing lithium, but since this is a whole big deal I'm guessing it's a new medication they've designed. I wouldn't be surprised if it's similar to lithium though in some of its chemical effects.

I don't know how helpful it'll be for autism in general though. The main problem is that FMRP which is the protein associated with Fragile X has a number of different functions, some of which have overlap with other forms of autism but some of which may not and therefore something that treats Fragile X may not apply to non-FX autism. My guess is, as we come to further understand all the variants of autism etiologically, subgroup by subgroup would be susceptible to different treatments.