NIH gives UCLA $9 million to fast-track studies of new autism drugs

The National Institute of Mental Health is giving UCLA $9 million to create a national network of research centers that will fast-track studies of new autism drugs. While there are drugs on the market that manage the behavior of children with autism, the UCLA project hopes to lead to the development of treatments that would help restore normal brain function and development.

A typical large-scale drug study can take years and cost hundreds of millions of dollars. The UCLA-led effort will involve simultaneous experiments at the centers in the research network. That approach will give scientists the ability to determine within months whether a new drug shows promise.

"It's a challenge," said the project's principal investigator, Dr. James McCracken, a professor of psychiatry and director of the division of child and adolescent psychiatry at the Semel Institute for Neuroscience and Human Behavior at UCLA.  "There are now so many possible experimental medicines and approaches from basic science for ASD [autism spectrum disorders] that we find ourselves way behind. We need a new paradigm to test the many possible compounds, and we need to quickly and accurately identify which ones are really ready for 'prime time.'" 

Researchers have made a lot of progress in identifying the genes and biological components involved in autism spectrum disorders, said McCracken, raising hopes for the identification of breakthrough drugs. 

"This is definitely the most exciting time yet to be involved in treatment research for ASD," he added. "Our basic science colleagues are generating enormous information on the likely underlying causes" of the condition. "We are well positioned to apply the basic science and find drugs that make a difference.” 

The Centers for Disease Control and Prevention estimates that one in 88 children in the U.S. has been identified with ASD, which delays the development of communication and social skills.

At UCLA, testing will involve scientists and clinicians from the fields of psychiatry, radiology and biostatistics. The UCLA Clinical and Translational Science Institute will measure brain and behavioral responses to a drug; positive findings could lead to more large-scale studies. 

 The National Institute of Mental Health grant is for a three-year period.