Nastech, the biotech company that has proprietary technology for intranasal drug delivery, plans to launch phase 2 clinical trials of a synthetic oxytocin treatment for autism within 12 months.
The plan is to use carbetocin, a synthetic used to control bleeding after labor and delivery, to control the core symptoms of autism, such as repetitive behavior and lack of sociability.
The company uses what it calls "tight junction biology" to squeeze molecules through cells in order to deliver larger molecules, such as the peptide oxytocin, and deliver them to the central nervous system via the epithelial membranes inside the nose.
In the company's earnings call today, CEO Steven Quay said Nastech has entered into an exclusive license of the intellectual property of Eric Hollander of the Seaver and New York Autism Center of Excellence. Hollander recently reported that he and colleague Jennifer Bartz had reduced some of the symptoms in adults with autism spectrum disorder with intravenous oxytocin.
Nastech's inhalant technology removes the needle barrier in oxytocin therapy.
Quay said, "We think current the scientific evidence provides useful insights: Autistic children have lower levels of oxytocin than normal children. In theory, we may be able to overcome this deficit through a nasal spray that uses our tight junction technology."
Nastech will work with Hollander on clinical trials. The plan is to conduct studies on dosage this year, and then, in 12 months, move into stage 2 trials with adults with ASD. Hollander will conduct the studies.
I recently spoke to Sue Carter of the University of Illinois' Brain Body Center. She is one of the foremost oxytocin researchers, and she's written extensively about the possible role of oxytocin and vasopressin in ASD.
She thinks we're moving too fast with oxytocin therapies. She pointed out several issues with dosing humans with oxytocin. First, it's not clear how much relation there is between serum levels of oxytocin and levels in the central nervous system. Second, it's possible that the problem with ASD is related to vasopressin rather than oxytocin. Finally, she said that with any hormone, there is an optimal level and a balance with other chemicals in the body. We don't know whether increasing the level of oxytocin will throw other things out of balance, or cause a shut-down in the body's natural production.
But, as Quay said during the call, so far there is no drug approved to treat the core symptoms of autism -- and one that worked could be a blockbuster. The CDC recently announced that the rate of autism in children was higher than its previous estimate: One in 150 kids will be diagnosed as on the spectrum.