Why Aren’t There Good Drugs for Autism? Ex-MDRNA Exec Takes a Shot at Pharma’s Neglected Disease
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to this concept of bonding and trust, by showing that adult subjects given oxytocin were more willing to trust financial advisors with their money, Brandt says.
Since one of the main symptoms of autism is social isolation and lack of ability to empathize with other people’s emotions, it was hypothesized that oxytocin might be a decent drug candidate, Brandt says. The problem is it’s hard to deliver in drug form, because it only lasts in the bloodstream for a few minutes. That’s why his former company worked on carbetocin, a similar peptide molecule that can last four hours in the body instead of about four minutes, Brandt says. Carbetocin has been made into a nasal spray form, which could provide convenient dosing.
No other company has a drug like this in clinical trials, and Brandt says his research can find only three other competitors testing the clinical effects of their drugs on patients with autism. Bristol-Myers Squibb and Takeda Pharmaceuticals are testing a new use of aripaprazole (Abilify) for the severe irritability of some autism patients, BioMarin has a small trial of sapropterin (Kuvan), and Neuropharm failed earlier this year in a pivotal trial of generic, low-dose form of fluoxetine (Prozac).
Nastech had already done the work formulating carbetocin into a drug, ran it through animal studies, and got the drug candidate to the point where Anatrope can start a mid-stage clinical trial to demonstrate whether it works in people, before it was shelved. The challenge for Brandt is raising at least $5 million to run that trial.
Even though the Anatrope drug is further along in development than many of the other therapies venture capitalists typically invest in, Brandt says he’s run into a lot of VCs who don’t know anything about autism, and seem too preoccupied with propping up their existing portfolios to think about making new investments. (That didn’t surprise me much, but I was surprised to hear Brandt say that unlike patient advocacy groups for other conditions like cystic fibrosis or muscular dystrophy, autism foundations are full of highly motivated parents, yet they don’t appear to have much money for clinical trials.)
Brandt is collaborating on the company with Eric Hollander, a psychiatry researcher at Albert Einstein College of Medicine in New York, and three other people he worked with previously at Nastech who are familiar with the autism program. [Correction: an earlier version of the story included an outdated affiliation for Hollander.] The team has the outline of a clinical trial design in hand—a high and a low dose, placebo-controlled, 90 patients in the U.S., four weeks of observation, and a budget of $5 million. “It’s a gigantic potential market, there’s nothing out there for it, and it takes little money,” to run such a small trial, Brandt says.
It’s unclear whether Brandt can raise the cash he needs or not, but I got the sense he’s really passionate about taking the program forward. He doesn’t have a child with autism, but he’s gotten intense support from those who do.
I saw it firsthand the morning we met at Starbucks. A woman sitting at a nearby table overheard our interview, and after about 45 minutes, she stood up and introduced herself to Brandt. She flipped open her cell phone, showed it to him, and said, “This is a picture of my daughter. She has autism. Keep up the good work.”