Apr 26, 2013 7:39 PM by Connie Murphy
LOS ANGELES (AP) - In an effort to find a treatment for autism, scientists at the University of California, Los Angeles, are leading a $9 million experiment that aims to evaluate the effectiveness of drugs within weeks rather than years.
It's part of a push by the National Institutes of Health to fast-track the pace of discovery that hopefully will lead to new therapies for several neurological disorders. Besides autism, the federal agency is also backing rapid efforts to seek treatments for schizophrenia and mood and anxiety disorders.
Typically, the path to getting drugs approved is long and costly and often littered with setbacks. The goal of this new approach is to test several potential drugs in small groups of people and then decide which one merits further investigation.
"We need to quickly and accurately identify which ones are really ready" for prime time, UCLA lead investigator Dr. James McCracken said in a statement.
UCLA is working with other medical centers to pinpoint promising drugs for autism, a spectrum of disorders affecting a person's ability to communicate and interact with others.
Autism advocacy groups welcomed the latest effort, saying it may lead to new treatments faster.
"The earlier you can know whether or not something has the potential to go all the way, the better," said Robert Ring, a vice president at Autism Speaks who previously oversaw autism research at Pfizer Inc.
Ring said he hoped promising drugs identified through rapid clinical trials would spur interest among drug companies to take it to the next level.
Federal statistics show one child out of 88 in the U.S. is believed to have autism or a related disorder. The number of cases has jumped in recent years mainly because of wider screening and better diagnosis. Children with autism may make poor eye contact or exhibit repetitive movements such as rocking or hand-flapping.
The causes of autism are unknown, but scientists lately have focused on genetics, which is thought to account for roughly 20 percent of cases. People with autism are often prescribed drugs to manage hyperactivity and aggressiveness, but the medications do not target the core problems, doctors said.
Enrollment in the UCLA-led project has yet to begin but researchers expect it to happen soon. Over a three-year period, scientists will test several compounds and study how they interact with the brain.
The federal government tried this in the 1950s and 1960s, but later decided to do more focused studies, said Dr. Lon Schneider, a professor of psychiatry at the University of Southern California.
Schneider said such work is useful, but researchers need to be careful about the limitations of studying small populations.
"I'm thrilled to see quick and dirty early development trials, but nothing comes free and the devil is in the details in how these trials are managed and carried out," he said.