OptiNose US Inc. has announced that its Norwegian affiliate was awarded $2.1 million by the Research Council of Norway to study its nasal drug delivery technology in the treatment of autism spectrum disorders (ASDs).
OptiNose will use this research grant to investigate "nose-to-brain" transport of oxytocin via the patented OptiNose Bi-Directional delivery technology for the treatment of ASDs. Partners who have agreed to collaborate with OptiNose in the project include the Department of Psychiatry at Oslo University Hospital, SINTEF and Smerud Medical Research and Norwegian academic insitutions.
"The opportunity to investigate nose-to-brain drug transport with the OptiNose technology in an effort to develop a new treatment for autism spectrum disorders is very exciting," said Per G. Djupesland, M.D., Ph.D, Chief Scientific Officer (CSO) of OptiNose. "Autism spectrum disorders are growing in prevalence and there are no drugs approved to treat the core symptoms which burden children, adults and families with these conditions. We hope to see significant benefits from delivering treatment with our innovative nasal technology."
Autism spectrum disorders (ASDs) are a group of complex disorders of brain development, including autism. According to the Centers for Disease Control and Prevention, approximately 1 out of every 110 children in the United States have ASD, as well as millions of children worldwide. These disorders are characterized, in varying degrees, by difficulties in social interaction, verbal and nonverbal communication and repetitive behaviors. ASD can also be associated with intellectual disability, difficulties in motor coordination and attention and physical health issues, but social behavior dysfunction is the core clinical characteristic.
With prevalence rates increasing from 10 to 17 percent in recent years, autism appears to have its roots in very early brain development abnormalities. There are no available effective treatments for ASD and most symptoms are rarely detected before 2-3 years of age, when demands for social interaction increase.
Oxytocin is a small, naturally occurring peptide currently safely used to stimulate lactation in breastfeeding women. It has recently attracted attention as a potential novel treatment alternative in several psychiatric disorders, including autism (Bartz 2008, Ishak 2010, Feifel 2010, Neumann 2008, Kosfeld 2005). Oxytocin has very poor oral bioavailability and with standard nasal delivery (liquid spray) approximately 3 percent of the drug reaches the systemic circulation blood. However, because it is estimated that only a tiny fraction (less than 0.01 percent) of oxytocin in the blood enters the brain across the blood brain barrier, achieving direct "nose to brain" delivery is an exciting possibility.
The effects of conventional intranasal spray delivery of oxytocin on multiple psychiatric disorders have been examined in a number of small clinical studies (Hollender 2007, Gaustella 2009, Andari 2010). Most of these studies suggest beneficial effects, mainly on social, aggressive and paranoid behavior.