Pfizer Experimental Drug Reduces Autism Signs in Mouse Study

An experimental drug for depression being developed by Pfizer Inc. reduced signs of autism in mice in a study, a finding that may shed light on the cause of the disorder and jump-start research into ways to treat it.

The treatment, named GRN-529, targets the brain chemical glutamate, tied to socialization and behavior. When given to mice displaying signs of autism, it suppressed repetitive actions and anti-social behavior, according to the research published today in the journal Science Translational Medicine. Pfizer and the National Institutes of Health funded the study.

There are no drugs on the market to treat the symptoms or causes of autism. Until recently, scientists believed the brains of autistic patients were hard-wired before diagnosis, making treatment with a drug difficult, said Daniel Smith, an autism researcher at Pfizer. That changed with findings the disorder may be caused by malfunctioning genes that have a real-time effect on the brain, said Smith, a co-author of the study.

“It is only in the past five years that there have been large human studies identifying the genetic mutations linked to autism,” Smith said in a telephone interview. “From that we can now explore those genetic pathways in more detail.”

Pfizer shares rose 1.5 percent to $22.96 in New York time. The stock has gained 14 percent in the past 12 months.

In the latest research, mice that were given the Pfizer drug displayed less repetitive grooming and jumping, and spent more time interacting with other animals.

Autism, a disorder that may affect one in 88 U.S. children, hurts brain development and is linked to poor social interaction and communication skills, repeated body movements, and unusual attachments to objects.

Pfizer, based in New York, plans to continue studying the drug in animals, Smith said.

Novartis AG and Roche Holding AG, both based in Basel, Switzerland, and the closely held U.S. company Seaside Therapeutics LLC are testing similar medicines in humans with fragile X syndrome, a genetic condition characterized by mental retardation, behavior problems, and delayed speech.

About 30 percent of patients with fragile X meet the diagnostics criteria for autism, the study said.