4 Promising Autism Treatments, From Vitamin B12 to Alzheimer’s Drug Namenda

Medicine hasn’t come up with a cure for autism, the often-devastating developmental disorder that now affects 1 in 150 children, and one big reason is that doctors don’t yet know what causes it. Parents frustrated by the lack of options often turn to the Internet for help, where dozens of medical and behavioral treatments are promoted.

Unfortunately, most of the treatments out there have not been tested to find out if they work, making it tough for parents to figure out what might help. Those that have been rigorously tested so far have failed to measure up. That includes secretin, a hormone affecting liver and pancreas function that was popular until a 2003 trial found it did nothing to alleviate symptoms.

Yet treatments for autism do exist. Those proven to work include structured behavioral interventions that teach children social and language skills, as well as medications that reduce disabling symptoms such as anxiety, depression, and gastrointestinal disorders. Increasingly, researchers are looking at autism as a “state” that could be changed rather than a “trait,” according to Martha Herbert, a pediatric neurologist at Massachusetts General Hospital in Boston. And researchers are starting to rigorously test other potential treatments, including methyl vitamin B12 and an Alzheimer’s drug known as Namenda.

“What we’re really beginning to understand is that autism is not one disease but that there are many different autisms,” says Geri Dawson, chief science officer for the advocacy group Autism Speaks, which funds research. “There’s a strong push to use genetic markers and genetic testing, but also medical testing to identify the specific causes of children’s autism. The treatment could be very different, depending on cause.”

Robert Hendren, a child psychiatrist who is executive director of the MIND Institute at the University of California–Davis, is among the researchers using biological markers—rather than behavioral observations—to measure how autistic children respond to experimental treatments. (Observational assessments of behavioral change tend to be less consistent from one person to the next and therefore less reliable as research measures.)

MIND researchers recently tested injections of methyl B12 in a controlled trial on 30 children, since prior recent findings had shown that some children with autism have altered biomarkers for oxidative stress. The results weren’t statistically significant, but Hendren says nine of the children did improve in language and socialization, and those children had changes in biomarkers for oxidative stress. The institute will run a larger trial this summer with 50 children in an effort to figure out if the treatment really does have a benefit.

The MIND researchers also have just finished a double-blind placebo-controlled trial of omega-3 fatty acids, which may have an anti-inflammatory effect. “We’ve been using a model where autism is a static-brain, brain-based, mostly genetic disorder,” says Hendren. “That model is shifting to where different things in the body are involved, like the gastrointestinal tract interacting with the brain, or the immune system interacts with the brain. That gives way to new models—new ways of subtyping autism and new ways of treating it.”

Other researchers are investigating not just better treatment for painful physical symptoms that are common in children with autism, such as gastrointestinal problems, but for problematic behaviors associated with the disorder itself. “So far most of our drug treatments have really been treating associated symptoms: irritability, depression, aggression,” says Dawson. “It’s been really challenging to develop drugs that target the core symptoms of autism: social behavior and language.”

Dawson gives the example of fenobam, a drug in phase-1 clinical trials for a genetic disorder called fragile X syndrome; about half of people with that syndrome also develop autism. Fenobam calmed behavior and reduced hyperactivity a small study with 12 people, published in the January issue of the Journal of Medical Genetics. And a multicenter clinical trial is being launched to assess the effect of the Alzheimer’s drug Namenda, which slows the rate of decline in Alzheimer’s patients.