Public-Private Effort Seeks to Expedite Discovery of Autism Drugs

Under a contract with the institute, U.C.L.A. will form a network of researchers at other academic centers that will try to identify promising new and older drug compounds quickly, and conduct early tests to see if they merit additional investment.

The program, part of the “Fast Fail” initiative at the institute, aims to determine within weeks whether a drug works, rather than the years it traditionally takes to evaluate a new drug.

“The whole idea is just getting much better in these early phases at identifying drugs that are going to be efficacious and safe, and thereby greatly speeding the development of effective new therapies and reducing the overall cost,” said Dr. James McCracken, who is leading the effort at U.C.L.A. as director of the division of child and adolescent psychiatry at the Semel Institute for Neuroscience and Human Behavior.

The number of diagnosed cases of autism, Asperger’s syndrome and related disorders in children has been growing in recent years, largely because of increased awareness. A recent report by the Centers for Disease Control and Prevention and the Health Resources and Services Administration concluded that one in 50 children aged 6 to 17 had been found to have autism or a related disorder, a 72 percent increase since 2007.

Although more cases are being diagnosed, no drugs are approved to treat the core symptoms of the disorders, which are characterized by delays in developing effective communication and social skills. Other drugs often prescribed to people with the disorders treat difficult behaviors like aggressiveness, hyperactivity and irritability.

Dr. McCracken said developing effective treatments had been difficult because the underlying causes were poorly understood until the last few years, and some prominent efforts had failed. In 2004, the experimental drug secretin, developed by RepliGen, did not show that it worked in an advanced clinical trial, disappointing parents of children with autism who had placed their hopes in the drug.

Several major drug companies, including GlaxoSmithKline and AstraZeneca, have scaled back their research in the neurosciences because of the high failure rate, Dr. McCracken said.

Developing drugs to treat neurological disorders is difficult, in part because brain science is still evolving. The field is littered with drugs that scientists had hoped would be effective against diseases like Alzheimer’s and schizophrenia but that performed poorly in clinical trials.

Despite the setbacks, scientific advances in understanding the genetic underpinnings of autism have accelerated, leaving the door open for new drug discoveries, said Robert H. Ring, vice president of translational research at Autism Speaks, a patient advocacy group.

“Autism spectrum disorder is the brave new world of medicine development, and most companies out there — despite a lot of the retraction you’re seeing — they do recognize autism as a clear area of opportunity,” said Mr. Ring, who serves on a committee that helps select which compounds the U.C.L.A. program will test.

Some companies are pursuing treatments. Seaside Therapeutics, a private company in Cambridge, Mass., is developing drugs to treat autism and a form of mental retardation known as fragile X syndrome in a partnership with Roche.

“The approach that people have taken over the years is, ‘This person looks anxious, I’ll give them a drug I use to treat anxiety,’ ” said Dr. Randall L. Carpenter, a co-founder of Seaside and its chief executive. “We hope to treat the underlying molecular abnormality.”

Dr. McCracken said the program would identify four to eight compounds and run them through small trials in humans, testing how the drugs are absorbed and how they affect brain wave patterns that scientists say they believe are linked to autism.

“It’s taken a really long time to kind of crack open and begin to understand part of the disorder of brain biology that underpins autism,” Dr. McCracken said. “This is, to me, the most exciting time because we understand so much more than we did even five or 10 years ago.”

Article source: http://www.nytimes.com/2013/04/25/business/public-private-effort-seeks-to-expedite-discovery-of-autism-drugs.html?partner=rss&emc=rss