New Research: Protein Blocking Medications Could Treat Symptoms of Autism

According to an article on MSNBC.com,

Scientists could be on the verge of a new treatment for autism, if the results of animal research hold up in people. A study in mice suggests that several drugs, including one that is poised to enter trials in human patients as soon as next year, could improve brain function and reverse the symptoms of some autistic patients.

Based on this research, which focuses specifically on Fragile X (a form of autism caused by a genetic anomaly), researchers are looking at three possible drugs to treat symptoms of autism spectrum disorders in general. All three of these block a brain protein called metabotropic glutamate receptor 5 - and all are already in development, which means approved medications could be available within the next few years.

According to the article:

[It may be too early to know whether a drug called STX107] will improve autistic behaviors in people. The drug could reduce mGluR5 levels, but autism is such a complex disease, this may not be enough to restore normal behavior in patients...

If the drug does fail, there still may be hope for patients and their families. The Fragile X Research Foundation (FRAXA), which co-funded STX107 research, is supporting investigations involving two other drugs that block the same protein.

Lithium, which is used for treating depression and bipolar disorder, is being investigated for its potential to treat autism and fragile X by researchers at Rush University in Chicago.

And another compound called fenobam — initially developed as an anti-anxiety medication in the 1970s and then abandoned — is being revitalized for fragile X by Neuropharm, a Surrey, UK-based pharmaceutical company.

“We believe that drugs which block mGluR5 have enormous potential for the treatment of fragile X and related developmental disorders, including many cases of autism,” said Katie Clapp, president and executive director of FRAXA.

Of course, there's no way to way whether protein blockers could really make the same difference for humans as they do for mice. And while Fragile X is considered part of the autism spectrum, its etiology may be quite different. Still, there is real reason to hope that at least some cases of autism may be directly treatable - and that would be a huge step forward for the autism community.