Autism and Baclofen

Autism and Baclofen

Recent studies in autism have shown the effectiveness of STX209, a drug which is chemically similar to Baclofen in the treatment of autism.  It is 10 times more potent than Baclofen and target the same Gaba receptor.  Not available to the public until at least 2013 it give hope for the treatment of autism and possibly addiction.  The involvement of the Gaba B receptor and the use of Baclofen to treat autism highlights the significance of the Gaba B receptor in understanding the underlying causes of addiction.

Results of the study were published in on 1 December 2010.

Hoffman-La Roche Ltd began trials of arbaclofen but then decided not to proceed with them and have issued the following statement:

Roche Statement

Roche position on the decision not to exercise the option for Seaside Therapeutics’ compound arbaclofen for autism and fragile X

In recent weeks, Roche has received numerous letters from parents and patient advocates regarding the discontinuation of our collaboration with Seaside Therapeutics on the compound arbaclofen for autism and fragile X. While Roche neither owns nor develops arbaclofen, the questions raised in the letters we have received are very important to us. We therefore want to ensure that our position and the reasons for our decision not to exercise the option to license arbaclofen are clearly understood.

We take very seriously the concerns of all the patients, in this particular case those of parents for their children enrolled in clinical trials with arbaclofen. No parent ever wants to see their children suffer and will do whatever is needed to ensure their safety, health and well-being.

Roche had hoped for a much different outcome, one that would show a clear benefit for those patients affected with these disorders and their families. In the case of the Seaside Therapeutics trials, we regret that the clinical data collected in the autism and fragile X trials did not show the benefit we had hoped for. While it may appear that some patients had a favourable response to this experimental compound, this did not appear to be the case when we looked at the clinical trial results in their entirety.1

While Roche is not continuing its support for Seaside’s development of arbaclofen, it is important to note that Roche is currently one of only a few companies committed to finding innovative treatment options for individuals with neurodevelopmental conditions such as autism, fragile X and Down syndrome. Indeed, we continue to investigate three compounds in clinical trials, V1A (RG7314) for autism, mGluR5 (RG7090) for fragile X and GABA-A a5 (RG1662) for Down syndrome. More information on these trials can be accessed via and

Roche wants to reassure all patients, parents and patient advocates that our company understands the urgent need for effective treatment options for neurodevelopmental conditions and that our scientists are working tirelessly to discover and develop such treatment options.

1) For detailed questions on the study results, please contact the sponsor and owner of the results Seaside Therapeutics directly(
2) Autism trial:
Fragile X trials:  and


Read the full story at




New Version of an Old Drug Could Treat Autism (and Addiction Too)

Charly Franklin / Getty Images


One night in 2006, Kathy Roberts rushed her autistic daughter,

Jenny, to the hospital. Nothing had been able to stop the young woman, then in her mid-20s,

from vomiting. Jenny had recently suffered several major seizures and her entire

gastrointestinal system was going haywire.

To try to calm Jenny’s GI tract, doctors at Massachusetts General Hospital prescribed baclofen,

an antispasmodic drug that is also being studied as a potential treatment for alcoholism and

other addictions. The drug relieved Jenny’s vomiting, but it did something else too — a

completely unexpected and welcome side effect. (More on Could Anorexia Be

a ‘Female’ Form of Autism?)

“Within 24 hours, I saw a change,” says Roberts. “Right away, I saw that it was globally

calming. I’ve always described a state that she would get into where it seemed like she wasn’t

comfortable in her own skin, and was trying to crawl out. I saw that calmed down.”

Roberts, founder of the Giant Step school for children with autism in Southport, Conn., called

Mark Bear, professor of neuroscience at MIT and advisory board member of Giant Step. In

2005, Bear had co-founded a drug company called Seaside Therapeutics to develop treatments

for autism and other developmental disorders. Roberts told Bear about baclofen’s effect on her

daughter, and a new line of research was born. (More on Picky Eating May Be

Early Sign of Autism)

In September, Seaside announced positive results from a phase II clinical trial of STX209, an

experimental drug that is chemically related to baclofen. In the trial, which was not blinded or

placebo controlled, STX209 led to a reduction in agitation and related emotional outbursts in

autistic people. Such behavior is common in people with autism — often, a result of anxiety

caused by extreme sensory oversensitivity or frustration over being unable to communicate

their needs. To cope, autistic people often develop behavioral mechanisms, include tantrums,

social withdrawal or repetitive behaviors like rocking or hand flapping.

STX209, while not a cure, appeared to ease anxiety. “We’re seeing reductions in a lot of types

of outbursts and irritable behavior, along with increased communication and social behavior,”

says Dr. Randall Carpenter, co-founder, president and CEO of Seaside. …

Carpenter says that participants in both of Seaside’s STX209 trials were offered the option to

switch to baclofen once the trial was complete, if they thought their children had been helped.

“After about eight or 10 people tried it, the parents and clinicians were up in arms because they

didn’t think [baclofen] was working as well as what they’d seen in trial,” he says. “The FDA

allowed us to continue [treating people with STX209] under its provisions for compassionate

use. Some are coming up on a year now and they continue to see improvement and stay on.

Very few have dropped out.”

Whether the drug will prove safe and effective in the long term, of course, is yet to be seen.

But with no drugs approved for Fragile X and only two to treat autism — both aimed at

relieving symptoms rather than treating the underlying problem — the development of

STX209 will undoubtedly be closely watched, both by parents and the pharmaceutical


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