Up and Coming: Medications Under Research Part 1

It may seem like a pipe dream: a pill that could erase autism’s most debilitating symptoms. Children speaking, making friends, trying new foods. Families able to go out into the world without fearing meltdowns or stares. Too good to be true? Perhaps, but there are several medications currently being tested that may bring this dream a step closer to reality.

The medications covered in these articles are not yet approved for use in children with autism. Many are currently used for other medical conditions, but are showing promise in lab tests with animals, or even in clinical trials with human subjects. It remains to be seen whether any of them will end up being the miracle drug so many would love to have, but in the interest of keeping our readers informed, here are the latest contenders.

Bumetanide

Bumentanide is a diuretic currently used to help patients with heart failure, kidney disease, and liver disease reduce fluid buildup. A 2012 study in France followed 60 children, ages 3 to 11, with Asperger syndrome who were given bumentanide or a placebo for 90 days. The subjects’ symptoms were scored by individuals who did not know which group they fell into. Parents and teachers were also surveyed before and after the trial.
By the end of the study, three-fourths of the children in the treatment group shifted from average autism scores in the severe range to mild or moderate, compared to one-third of the placebo group.

“They were making more eye contact, more spontaneous speech, and more two-way conversation,”

says Daniel Coury, MD, a pediatric psychiatrist in Columbus Ohio who is medical director for the Autism Speaks Treatment Network.

Further testing on rats suggests that bumentanide works by “flipping a switch” in the brain. Side effects included lower potassium levels and bedwetting.

Most of the parents of the children in the study asked to continue the medication, according to researcher Yehezkel Ben-Ari, Phd, director of research at the National Institute of Health and Medical Research in Marseilles, France.

Arbaclofen

Arbaclofen is a derivative of the FDA-approved drug baclofen, which is used to treat muscle spasticity in conditions such as cerebral palsy, and is being studied as a treatment for alcoholism and other addictions. A study published in Science Translational Medicine suggests that it may treat social withdrawal in people with Fragile X syndrome.

The double-blind, six-week trial included 63 people with Fragile X, ages 6 to 39. The drug did little to improve irritability, which the researchers were testing for, but they did find significant improvements in problem behaviors and social avoidance.

Dr. Elizabeth Berry-Kravis of Rush University Medical Center in Chicago, says,

“[People with autism] would like to socialize, but they can’t make themselves do it because it’s so overwhelming. Arbaclofen lowers that over-responsiveness to the social environment so that they can now tolerate going into it.”

Additional trials are being conducted in the hopes of gaining FDA approval for arbaclofen.

Part 2 continues here.

To read articles in our five part series on autism and medication, click here.