Why Aren’t There Good Drugs for Autism? Ex-MDRNA Exec Takes a Shot at Pharma’s Neglected Disease

[Corrected: 09/14/09, 12:40 pm. See below.] Autism has stumped drug developers for a long time. Scientists say they don’t really know what causes it. There’s a long list of symptoms, from social isolation, to obsessive behaviors like staring at ceiling fans, to difficulty with language. So it’s hard for drug developers to form a strategy to treat this disease, and not surprisingly, the condition doesn’t rank high on the research agendas of most drug companies.

That was true at Bothell, WA-based Nastech Pharmaceuticals (now called MDRNA), which long had a project on the back burner that held some promise for autism. When the company did some major downsizing a year ago to conserve cash, president Gordon Brandt was let go, and negotiated to take that neglected piece of intellectual property with him. That license has now formed into a business plan for a company Brandt is calling Anatrope Pharmaceuticals, which he told me about the other day at a Starbucks near Xconomy’s office.

“This always rattled around as number 4 on our priority list at Nastech, and never really got the attention it deserved,” Brandt says.

Autism has been riding a wave of increased public awareness, and is now said to affect about one out of every 150 kids in the U.S. Celebrities like Jenny McCarthy have pushed the idea that preservatives in childhood vaccines are causing the rising incidence, although scientists say there isn’t convincing data to support this. Yet despite all this popular attention (Bruce Springsteen and Jerry Seinfeld are scheduled to headline a fundraiser for Autism Speaks, a patient advocacy group, in New York on Nov. 17), there’s been surprisingly little progress, or competition, in the biotech and pharma industries to create effective new therapies. Right now, some children with irritability from autism get atypical antipsychotics like Johnson & Johnson’s risperidone (Risperdal), or depression meds, but behavioral therapy appears to be the best option for kids diagnosed with autism, Brandt says.

The idea at Anatrope is to build on research into oxytocin, the “hormone of bonding and trust,” as Brandt puts it. Levels of oxytocin spike for both mothers and their babies during breastfeeding; the hormone is supposed to play a role in the bonding process. [Editor’s note: Previous sentence revised to clarify role of oxytocin in breastfeeding]. A 2005 paper in Nature lent more credence

Author: Luke Timmerman

Luke is an award-winning journalist specializing in life sciences. He has served as national biotechnology editor for Xconomy and national biotechnology reporter for Bloomberg News. Luke got started covering life sciences at The Seattle Times, where he was the lead reporter on an investigation of doctors who leaked confidential information about clinical trials to investors. The story won the Scripps Howard National Journalism Award and several other national prizes. Luke holds a bachelor’s degree in journalism from the University of Wisconsin-Madison, and during the 2005-2006 academic year, he was a Knight Science Journalism Fellow at MIT.